UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

 

 

FORM 10-Q

 

 

(Mark One)

QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the quarterly period ended September 30,March 31, 20212022

OR

TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM TO

Commission File Number 001-39208

 

Beam Therapeutics Inc.

(Exact name of Registrant as specified in its Charter)

 

 

Delaware

81-5238376

(State or other jurisdiction of

incorporation or organization)

(I.R.S. Employer

Identification No.)

 

 

26 Landsdowne238 Main Street

Cambridge, MA

0213902142

(Address of principal executive offices)

(Zip Code)

 

Registrant’s telephone number, including area code: (857) 327-8775

Securities registered pursuant to Section 12(b) of the Act:

 

Title of each class

Trading

Symbol(s)

Name of each exchange

on which registered

Common Stock, par value $0.01 per share

BEAM

Nasdaq Global Select Market

 

Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ☒ NO ☐

 

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ☒ NO ☐

 

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act.

 

Large accelerated filer

Accelerated filer

 

 

 

 

Non-accelerated filer

Smaller reporting company

 

 

 

 

 

 

Emerging growth company

 

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.

 

Indicate by check mark whether the Registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). YES ☐ NO

 

The number of shares of registrant’s common stock outstandingoutstanding as of November 1, 2021May 2, 2022 was 68,135,79870,266,761.

 


 

CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS

This Quarterly Report on Form 10-Q contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, or the Securities Act, and Section 21E of the Securities Exchange Act of 1934, as amended, or the Exchange Act. Such forward-looking statements reflect, among other things:

our current expectations and anticipated results of operations;
our expectations regarding the initiation, timing, progress and results of our research and development programs and preclinical studies, including our expectationexpectations that we will nominate our first development candidatesubmit an Investigational New Drug application, or IND, for in vivo base editing in the liver using lipid nanoparticle, or LNP, deliveryBEAM-102 for the treatment of patientssickle cell disease and BEAM-201 for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia, that we will initiate IND-enabling studies for BEAM-301 for the treatment Glycogen Storage Disease Type IA (R83C mutation) byand that we will nominate a CAR-T development candidate and an additional in vivo liver development candidate during 2022;
our expectations regarding the end of 2021,initiation, timing, progress and results of our clinical trials, including our Phase 1/2 clinical trial designed to assess the safety and efficacy of BEAM-101 for the treatment of sickle cell disease, which we refer to as our BEACON-101 trial;
our ability to develop and maintain a sustainable portfolio;portfolio of product candidates;
our ability to develop life-long, curative, precision genetic medicines for patients through base editing;
our ability to create a hub for partnering with other companies;
our plans for pre-clinicalpreclinical studies for product candidates in our pipeline;
our ability to advance any product candidates that we may develop and successfully complete any clinical trials or preclinical studies, including the manufacture of any such product candidates;
our ability to pursue a broad suite of clinically validated delivery modalities;
our expectations regarding our ability to generate additional novel LNPslipid nanoparticles that we believe could accelerate novel nonviral delivery of gene editing or other nucleic acid payloads to tissues beyond the liver and our ability to expand the reach of gene editing,our programs, including as a result of our acquisition of Guide Therapeutics, Inc., or Guide;
the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates and technology;
developments related to our competitors and our industry;
the expected timing, progress and success of our collaborations with third parties, including any future payments we may receive under our collaboration and license agreements, and our ability to identify and enter into future license agreements and collaborations;
developments related to base editing technologies;
our ability to successfully develop our three distinct pipelinesdelivery modalities and obtain and maintain approval for our product candidates;
our ability to successfully establish and maintain a commercial-scale current Good Manufacturing Practice, or cGMP, manufacturing facility and our expectations that this facility will be operational in the first quarter of 2023;
regulatory developments in the United States and foreign countries;
our ability to attract and retain key scientific and management personnel;
our expectations regarding the strategic and other potential benefits of our acquisition of Guide,Guide; and
the impact of the coronavirus disease of 2019, or COVID-19, pandemic on our business.

All of these statements are subject to known and unknown important risks, uncertainties and other factors that may cause our actual results, performance or achievements, market trends, or industry results to differ materially from those expressed or implied by such forward-looking statements. Therefore, any statements contained herein that are not statements of historical fact may be forward-looking statements and should be evaluated as such. Without limiting the foregoing, the words “anticipate,” “expect,” “suggest,” “plan,” “believe,” “intend,” “project,” “forecast,” “estimates,” “targets,” “projections,” “should,” “could,” “would,” “may,” “might,” “will,” and the negative thereof and similar words and expressions are intended to identify forward-looking statements. These forward-looking statements are subject to a number of risks, uncertainties and assumptions, including those described in “Risk Factors” in Part II, Item 1A of this report and “Risk Factors Summary” and “Risk Factors” in Part I, Item 1A. of our Annual Report on Form 10-K for the fiscal year ended December 31, 2020,2021, or the 20202021 Form 10-K. Unless legally required, we assume no obligation to


update any such forward-looking information to reflect actual results or changes in the factors affecting such forward-looking information.

When we use the terms “Beam,” the “Company,” “we,” “us” or “our” in this Quarterly Report on Form 10-Q, we mean Beam Therapeutics Inc. and its subsidiaries on a consolidated basis, unless the context indicates otherwise.


 

Table of Contents

 

 

 

Page

PART I

Financial Information

 

Item 1.

Financial Statements (Unaudited)

1

 

Condensed Consolidated Balance Sheets

1

 

Condensed Consolidated Statements of Operations and Other Comprehensive Loss

2

 

Condensed Consolidated Statements of Redeemable Convertible Preferred Stock and Stockholders’ Equity (Deficit)

3

 

Condensed Consolidated Statements of Cash Flows

5

 

Notes to Condensed Consolidated Financial Statements

7

Item 2.

Management’s Discussion and Analysis of Financial Condition and Results of Operations

2322

Item 3.

Quantitative and Qualitative Disclosures About Market Risk

3735

Item 4.

Controls and Procedures

3736

 

 

 

PART II

Other Information

 

Item 1.

Legal Proceedings

3937

Item 1A.

Risk Factors

39

Item 2.

Unregistered Sales of Equity Security and Uses of Proceeds

4237

Item 6.

Exhibits

4341

Signatures

 

4442

 

 

 


 

PART I. FINANCIAL INFORMATION

Item 1. Financial Statements (Unaudited)

Beam Therapeutics Inc.

Condensed Consolidated Balance Sheets

(Unaudited)

(in thousands, except share and per share amounts)

 

 

September 30,
2021

 

 

December 31,
2020

 

 

March 31,
2022

 

 

December 31,
2021

 

Assets

 

 

 

 

 

 

 

 

 

 

 

 

Current assets:

 

 

 

 

 

 

 

 

 

 

 

 

Cash and cash equivalents

 

$

612,023

 

$

162,171

 

 

$

296,821

 

$

559,994

 

Marketable securities

 

321,382

 

137,500

 

 

925,779

 

405,653

 

Collaboration receivable

 

 

300,000

 

Prepaid expenses and other current assets

 

 

8,019

 

 

 

8,650

 

 

 

16,884

 

 

 

7,360

 

Total current assets

 

941,424

 

308,321

 

 

1,239,484

 

1,273,007

 

Property and equipment, net

 

71,533

 

38,513

 

 

94,288

 

84,258

 

Restricted cash

 

14,840

 

14,840

 

 

12,746

 

12,746

 

Operating lease right-of-use assets

 

103,169

 

86,859

 

 

105,543

 

102,718

 

Long-term investments

 

24,538

 

2,577

 

Other assets

 

 

1,051

 

 

 

567

 

 

 

1,341

 

 

 

1,724

 

Total assets

 

$

1,156,555

 

 

$

451,677

 

 

$

1,453,402

 

 

$

1,474,453

 

Liabilities and stockholders’ equity

 

 

 

 

 

 

 

 

 

 

 

 

Current liabilities:

 

 

 

 

 

 

 

 

 

 

 

 

Accounts payable

 

$

7,282

 

$

6,314

 

 

$

6,266

 

$

7,474

 

Accrued expenses and other current liabilities

 

22,965

 

18,463

 

 

24,062

 

28,921

 

Accrued sub-license fees

 

33,125

 

38,743

 

Derivative liabilities

 

49,600

 

71,200

 

 

28,600

 

42,200

 

Current portion of deferred revenue

 

12,822

 

24

 

 

115,049

 

86,270

 

Current portion of lease liability

 

6,484

 

4,218

 

 

9,359

 

7,540

 

Current portion of equipment financing liability

 

 

2,238

 

 

 

2,118

 

 

 

2,337

 

 

 

2,287

 

Total current liabilities

 

101,391

 

102,337

 

 

218,798

 

213,435

 

Long-term lease liability

 

133,120

 

96,014

 

 

136,034

 

134,810

 

Long-term equipment financing liability

 

3,598

 

5,294

 

 

2,404

 

3,007

 

Contingent consideration liabilities

 

26,960

 

0

 

 

30,915

 

31,367

 

Long-term portion of deferred revenue

 

36,820

 

394

 

 

225,093

 

262,303

 

Other liabilities

 

 

852

 

 

 

2,077

 

 

 

11,090

 

 

 

2,793

 

Total liabilities

 

302,741

 

206,116

 

 

624,334

 

647,715

 

Commitments and contingencies (See Note 7, Leases, Note 9, License agreements and Note 10, Collaboration and license agreements)

 

 

 

 

 

 

 

 

 

 

 

 

Stockholders’ equity:

 

 

 

 

 

 

 

 

 

 

 

 

Preferred stock, $0.01 par value; 25,000,000 shares authorized, and 0 shares issued or outstanding at September 30, 2021 and December 31, 2020, respectively

 

 

 

Common stock, $0.01 par value; 250,000,000 shares authorized, 68,126,713 and 58,446,016 issued, and 67,734,484 and 57,254,178 outstanding at September 30, 2021 and December 31, 2020, respectively

 

677

 

573

 

Preferred stock, $0.01 par value; 25,000,000 shares authorized, and 0 shares issued or outstanding at March 31, 2022 and December 31, 2021, respectively

 

 

 

Common stock, $0.01 par value; 250,000,000 shares authorized, 69,785,836 and 68,581,251 issued, and 69,753,023 and 68,389,425 outstanding at March 31, 2022 and December 31, 2021, respectively

 

698

 

684

 

Additional paid-in capital

 

1,556,685

 

642,633

 

 

1,668,567

 

1,594,378

 

Accumulated other comprehensive income (loss)

 

19

 

(9

)

Accumulated other comprehensive (loss) income

 

(2,709

)

 

(50

)

Accumulated deficit

 

 

(703,567

)

 

 

(397,636

)

 

 

(837,488

)

 

 

(768,274

)

Total stockholders’ equity

 

 

853,814

 

 

 

245,561

 

 

 

829,068

 

 

 

826,738

 

Total liabilities and stockholders’ equity

 

$

1,156,555

 

 

$

451,677

 

 

$

1,453,402

 

 

$

1,474,453

 

 

The accompanying notes are an integral part of these condensed consolidated financial statements.

1


 

Beam Therapeutics Inc.

Condensed Consolidated Statements of Operations and Other Comprehensive Loss

(Unaudited)

(in thousands, except share and per share amounts)

 

 

Three Months Ended September 30,

 

 

Nine Months Ended September 30,

 

 

Three Months Ended March 31,

 

 

2021

 

 

2020

 

2021

 

2020

 

 

2022

 

 

2021

 

License and collaboration revenue

 

$

763

 

$

6

 

 

$

775

 

 

$

18

 

 

$

8,432

 

$

6

 

Operating expenses:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Research and development

 

 

54,623

 

29,825

 

290,306

 

70,728

 

 

 

65,410

 

190,106

 

General and administrative

 

 

15,774

 

 

 

7,502

 

 

 

39,450

 

 

 

21,251

 

 

 

19,247

 

 

 

10,273

 

Total operating expenses

 

 

70,397

 

 

 

37,327

 

 

 

329,756

 

 

 

91,979

 

 

 

84,657

 

 

 

200,379

 

Loss from operations

 

 

(69,634

)

 

(37,321

)

 

(328,981

)

 

(91,961

)

 

 

(76,225

)

 

(200,373

)

Other income (expense):

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Change in fair value of derivative liabilities

 

 

35,800

 

2,700

 

(8,400

)

 

(8,700

)

 

 

13,600

 

(1,900

)

Change in fair value of long-term investments

 

 

(4,892

)

 

 

21,960

 

517

 

Change in fair value of non-controlling equity investments

 

 

(7,685

)

 

1,039

 

Change in fair value of contingent consideration liabilities

 

 

10,599

 

 

9,553

 

 

 

 

452

 

(305

)

Interest and other income (expense), net

 

 

9

 

 

 

169

 

 

 

(63

)

 

 

1,016

 

 

 

644

 

 

 

(21

)

Total other income (expense)

 

 

41,516

 

 

 

2,869

 

 

 

23,050

 

 

 

(7,167

)

 

 

7,011

 

 

 

(1,187

)

Net loss

 

$

(28,118

)

 

$

(34,452

)

 

$

(305,931

)

 

$

(99,128

)

 

$

(69,214

)

 

$

(201,560

)

Unrealized gain (loss) on marketable securities

 

 

(12

)

 

 

(132

)

 

 

28

 

 

 

25

 

 

 

(2,659

)

 

 

(15

)

Comprehensive loss

 

$

(28,130

)

 

$

(34,584

)

 

$

(305,903

)

 

$

(99,103

)

 

$

(71,873

)

 

$

(201,575

)

Reconciliation of net loss to net loss attributable to common stockholders:

 

 

 

 

 

 

 

 

 

 

 

 

Net loss

 

$

(28,118

)

 

$

(34,452

)

 

$

(305,931

)

 

$

(99,128

)

Accretion of redeemable convertible preferred stock to redemption value, including dividends on preferred stock

 

 

 

 

 

 

 

 

 

 

 

(1,277

)

Net loss attributable to common stockholders

 

$

(28,118

)

 

$

(34,452

)

 

$

(305,931

)

 

$

(100,405

)

Net loss per common share attributable to common stockholders, basic and diluted

 

$

(0.42

)

 

$

(0.69

)

 

$

(4.86

)

 

$

(2.31

)

Weighted-average common shares used in net loss per share attributable to common stockholders, basic and diluted

 

 

66,377,611

 

 

 

50,087,747

 

 

 

62,960,219

 

 

 

43,438,919

 

Net loss per common share, basic and diluted

 

$

(1.01

)

 

$

(3.35

)

Weighted-average common shares outstanding, basic and diluted

 

 

68,703,864

 

 

 

60,210,120

 

The accompanying notes are an integral part of these condensed consolidated financial statements.

2


 

Beam Therapeutics Inc.

Condensed Consolidated Statements of Redeemable Convertible Preferred Stock and Stockholders’ (Deficit) Equity

(Unaudited)

(in thousands, except share amounts)

 

 

Redeemable Convertible
Preferred Stock

 

 

 

Common Stock

 

 

Additional
Paid-in

 

 

Accumulated
Other
Comprehensive

 

 

Accumulated

 

 

Total
Stockholders’
(Deficit)

 

 

Common Stock

 

 

Additional
Paid-in

 

 

Accumulated
Other
Comprehensive

 

 

Accumulated

 

 

Total
Stockholders’

 

 

Shares

 

 

Amount

 

 

 

Shares

 

 

Amount

 

 

Capital

 

 

Income (Loss)

 

 

Deficit

 

 

Equity

 

 

Shares

 

 

Amount

 

 

Capital

 

 

Income (Loss)

 

 

Deficit

 

 

Equity

 

Balance at December 31, 2019

 

130,616,784

 

$

302,049

 

 

 

7,326,185

 

$

73

 

$

1,851

 

$

16

 

$

(203,044

)

 

$

(201,104

)

Accretion of redeemable convertible preferred stock to redemption value

 

 

1,277

 

 

 

 

 

(1,277

)

 

 

 

(1,277

)

Conversion of redeemable convertible preferred stock to common stock upon closing of initial public offering

 

(130,616,784

)

 

(303,326

)

 

 

29,127,523

 

291

 

303,035

 

 

 

303,326

 

Issuance of common stock from initial public offering, net of issuance costs of $18.7 million

 

 

 

 

 

12,176,471

 

122

 

188,201

 

 

 

188,323

 

Balance at December 31, 2020

 

57,254,178

 

$

573

 

$

642,633

 

$

(9

)

 

$

(397,636

)

 

$

245,561

 

Issuance of common stock from private placement, net of issuance costs of $8.0 million

 

2,795,700

 

28

 

251,977

 

 

 

252,005

 

Issuance of common stock to acquire Guide

 

1,087,153

 

10

 

120,022

 

 

 

120,032

 

Vesting of restricted common stock

 

 

 

 

 

387,866

 

4

 

(4

)

 

 

 

 

 

398,804

 

4

 

(4

)

 

 

 

 

Stock-based compensation

 

 

 

 

 

 

 

2,792

 

 

 

2,792

 

 

 

 

4,648

 

 

 

4,648

 

Exercise of common stock options

 

 

 

 

 

59,305

 

1

 

151

 

 

 

152

 

 

199,284

 

2

 

1,756

 

 

 

1,758

 

Other comprehensive income (loss)

 

 

 

 

 

 

 

 

(360

)

 

 

(360

)

 

 

 

 

(15

)

 

 

(15

)

Net loss

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

(30,458

)

 

 

(30,458

)

 

 

 

 

 

 

 

 

 

 

 

 

 

 

(201,560

)

 

 

(201,560

)

Balance at March 31, 2020

 

 

 

$

 

 

 

 

49,077,350

 

$

491

 

$

494,749

 

$

(344

)

 

$

(233,502

)

 

$

261,394

 

Vesting of restricted common stock

 

 

 

 

 

387,870

 

4

 

(4

)

 

 

 

 

Stock-based compensation

 

 

 

 

 

 

 

2,769

 

 

 

2,769

 

Exercise of common stock options

 

 

 

 

 

180,517

 

1

 

359

 

 

 

360

 

Other comprehensive income (loss)

 

 

 

 

 

 

 

 

517

 

 

517

 

Net loss

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

(34,218

)

 

 

(34,218

)

Balance at June 30, 2020

 

 

 

$

 

 

 

 

49,645,737

 

$

496

 

$

497,873

 

$

173

 

$

(267,720

)

 

$

230,822

 

Vesting of restricted common stock

 

 

 

 

 

387,867

 

4

 

(4

)

 

 

 

 

Issuance of common stock related to license agreement

 

 

 

 

 

175,000

 

2

 

262

 

 

 

264

 

Stock-based compensation

 

 

 

 

 

 

 

3,012

 

 

 

3,012

 

Exercise of common stock options

 

 

 

 

 

230,136

 

2

 

555

 

 

 

557

 

Other comprehensive income (loss)

 

 

 

 

 

 

 

 

(132

)

 

 

(132

)

Net loss

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

(34,452

)

 

 

(34,452

)

Balance at September 30, 2020

 

 

 

$

 

 

 

 

50,438,740

 

$

504

 

$

501,698

 

$

41

 

$

(302,172

)

 

$

200,071

 

Balance at March 31, 2021

 

 

61,735,119

 

 

$

617

 

 

$

1,021,032

 

 

$

(24

)

 

$

(599,196

)

 

$

422,429

 

 

3


 

Beam Therapeutics Inc.

Condensed Consolidated Statements of Redeemable Convertible Preferred Stock and Stockholders’ (Deficit) Equity - Continued

(Unaudited)

(in thousands, except share amounts)

 

 

Common Stock

 

 

Additional
Paid-in

 

 

Accumulated
Other
Comprehensive

 

 

Accumulated

 

 

Total
Stockholders’

 

 

Common Stock

 

 

Additional
Paid-in

 

 

Accumulated
Other
Comprehensive

 

 

Accumulated

 

 

Total
Stockholders’

 

 

Shares

 

 

Amount

 

 

Capital

 

 

Income (Loss)

 

 

Deficit

 

 

Equity

 

 

Shares

 

 

Amount

 

 

Capital

 

 

Income (Loss)

 

 

Deficit

 

 

Equity

 

Balance at December 31, 2020

 

57,254,178

 

$

573

 

$

642,633

 

$

(9

)

 

$

(397,636

)

 

$

245,561

 

Issuance of common stock from private placement, net of issuance costs of $8.0 million

 

2,795,700

 

28

 

251,977

 

 

 

252,005

 

Issuance of common stock to acquire Guide

 

1,087,153

 

10

 

120,022

 

 

 

120,032

 

Balance at December 31, 2021

 

68,389,425

 

$

684

 

$

1,594,378

 

$

(50

)

 

$

(768,274

)

 

$

826,738

 

Purchase of common stock under ESPP

 

28,990

 

 

1,412

 

 

 

1,412

 

Issuance of common stock from At-the-Market offering, net of issuance costs of $1.3 million

 

874,770

 

9

 

53,927

 

 

 

53,936

 

Vesting of restricted common stock

 

398,804

 

4

 

(4

)

 

 

 

 

 

283,186

 

3

 

(3

)

 

 

 

 

Stock-based compensation

 

 

 

4,648

 

 

 

4,648

 

 

 

 

18,035

 

 

 

18,035

 

Exercise of common stock options

 

199,284

 

2

 

1,756

 

 

 

1,758

 

 

176,652

 

2

 

818

 

 

 

820

 

Other comprehensive income (loss)

 

 

 

 

(15

)

 

 

(15

)

 

 

 

 

(2,659

)

 

 

(2,659

)

Net loss

 

 

 

 

 

 

 

 

 

 

 

 

 

 

(201,560

)

 

 

(201,560

)

 

 

 

 

 

 

 

 

 

 

 

 

 

 

(69,214

)

 

 

(69,214

)

Balance at March 31, 2021

 

 

61,735,119

 

 

$

617

 

 

$

1,021,032

 

 

$

(24

)

 

$

(599,196

)

 

$

422,429

 

Issuance of common stock from At-the-Market offering, net of issuance costs of $5.5 million

 

1,761,285

 

$

18

 

$

157,767

 

 

 

 

 

 

 

157,785

 

Vesting of restricted common stock

 

200,403

 

2

 

(2

)

 

 

 

 

Issuance of common stock for success payment liability

 

349,650

 

4

 

29,996

 

 

 

 

 

 

 

30,000

 

Stock-based compensation

 

 

 

10,452

 

 

 

10,452

 

Exercise of common stock options

 

406,225

 

4

 

4,079

 

 

 

4,083

 

Other comprehensive income (loss)

 

 

 

 

55

 

 

55

 

Net loss

 

 

 

 

 

 

 

 

 

 

 

 

 

 

(76,253

)

 

 

(76,253

)

Balance at June 30, 2021

 

 

64,452,682

 

 

$

645

 

 

$

1,223,324

 

 

$

31

 

 

$

(675,449

)

 

$

548,551

 

Issuance of common stock from At-the-Market offering, net of issuance costs of $8.6 million

 

2,912,557

 

$

29

 

$

318,580

 

$

 

$

 

318,609

 

Vesting of restricted common stock

 

200,402

 

2

 

(2

)

 

 

 

 

Issuance of common stock for success payment liability

 

 

 

 

 

 

 

Stock-based compensation

 

 

 

12,968

 

 

 

12,968

 

Exercise of common stock options

 

168,843

 

1

 

1,815

 

 

 

1,816

 

Other comprehensive income (loss)

 

 

 

 

(12

)

 

 

(12

)

Net loss

 

 

 

 

 

 

 

 

 

 

 

 

 

 

(28,118

)

 

 

(28,118

)

Balance at September 30, 2021

 

 

67,734,484

 

 

$

677

 

 

$

1,556,685

 

 

$

19

 

 

$

(703,567

)

 

$

853,814

 

Balance at March 31, 2022

 

 

69,753,023

 

 

$

698

 

 

$

1,668,567

 

 

$

(2,709

)

 

$

(837,488

)

 

$

829,068

 

The accompanying notes are an integral part of these condensed consolidated financial statements.

4


 

Beam Therapeutics Inc.

Condensed Consolidated Statements of Cash Flows

(Unaudited)

(in thousands)

 

 

Nine Months Ended September 30,

 

 

Three Months Ended March 31,

 

 

2021

 

 

2020

 

 

2022

 

 

2021

 

Operating activities

 

 

 

 

 

 

 

 

 

 

 

 

Net loss

 

$

(305,931

)

 

$

(99,128

)

 

$

(69,214

)

 

$

(201,560

)

Adjustments to reconcile net loss to net cash used in operating activities:

 

 

 

 

 

 

 

 

 

 

 

 

Depreciation and amortization

 

4,817

 

3,463

 

 

3,312

 

1,398

 

Amortization of investment discount (premiums)

 

(28

)

 

49

 

 

(390

)

 

15

 

In-process research and development charge

 

154,953

 

 

 

 

154,953

 

Stock-based compensation expense

 

28,068

 

8,573

 

 

18,035

 

4,648

 

Change in operating lease right-of-use assets

 

6,781

 

3,065

 

 

1,997

 

2,359

 

Non-cash research and development license expense, net

 

 

5,164

 

Change in fair value of derivative liabilities

 

8,400

 

8,700

 

 

(13,600

)

 

1,900

 

Change in fair value of contingent consideration liabilities

 

(9,553

)

 

 

 

(452

)

 

305

 

Change in fair value of non-controlling equity investments

 

(21,960

)

 

(517

)

 

7,685

 

(1,039

)

Other

 

63

 

 

 

3

 

63

 

Changes in operating assets and liabilities:

 

 

 

 

 

 

 

 

 

 

 

 

Prepaid expenses and other current assets

 

733

 

(3,667

)

 

(9,185

)

 

(2,365

)

Other long-term assets

 

(197

)

 

(56

)

 

 

(185

)

Accounts payable

 

(1,341

)

 

(134

)

 

(1,252

)

 

(666

)

Accrued expenses and other liabilities

 

2,818

 

4,219

 

 

(16,533

)

 

(4,458

)

Operating lease liabilities

 

15,208

 

(3,249

)

 

(1,780

)

 

6,111

 

Collaboration receivable

 

300,000

 

 

Deferred revenue

 

49,224

 

 

 

(8,432

)

 

(6

)

Other long-term liabilities

 

 

(153

)

 

 

2,075

 

 

 

8,296

 

 

 

(51

)

Net cash used in operating activities

 

(68,098

)

 

(71,443

)

Net cash provided by (used in) operating activities

 

218,490

 

(38,578

)

Investing activities

 

 

 

 

 

 

 

 

 

 

 

 

Purchases of property and equipment

 

(33,442

)

 

(8,232

)

 

(7,259

)

 

(11,478

)

Purchases of marketable securities

 

(606,746

)

 

(167,094

)

 

(690,390

)

 

(289,218

)

Maturities of marketable securities

 

422,920

 

157,380

 

 

160,310

 

20,450

 

Net cash acquired from Guide

 

620

 

 

 

 

 

 

 

620

 

Purchase of long-term investment

 

 

 

 

 

(750

)

Net cash used in investing activities

 

(216,648

)

 

(18,696

)

 

(537,339

)

 

(279,626

)

Financing activities

 

 

 

 

 

 

 

 

 

 

 

 

Proceeds from initial public offering, net of underwriting discount

 

 

192,510

 

Proceeds from issuance of common shares, net of commissions

 

737,205

 

 

 

54,003

 

260,000

 

Proceeds from issuances of stock under ESPP

 

1,412

 

 

Payment of equity offering costs

 

(8,688

)

 

(1,717

)

 

(6

)

 

(7,955

)

Proceeds from equipment financings

 

 

1,625

 

Repayment of equipment financings

 

(1,576

)

 

(1,158

)

 

(553

)

 

(529

)

Proceeds from exercise of stock options

 

 

7,657

 

 

 

1,069

 

 

 

820

 

 

 

1,758

 

Net cash provided by financing activities

 

734,598

 

192,329

 

 

55,676

 

253,274

 

 

 

 

 

 

 

 

 

 

 

 

 

Net change in cash, cash equivalents and restricted cash

 

449,852

 

102,190

 

 

(263,173

)

 

(64,930

)

Cash, cash equivalents and restricted cash—beginning of period

 

 

177,011

 

 

 

50,553

 

 

 

572,740

 

 

 

177,011

 

Cash, cash equivalents and restricted cash—end of period

 

$

626,863

 

 

$

152,743

 

 

$

309,567

 

 

$

112,081

 

The accompanying notes are an integral part of these condensed consolidated financial statements.

 

 

5


 

Beam Therapeutics Inc.

Condensed Consolidated Statements of Cash Flows - Continued

(Unaudited)

(in thousands)

 

 

Nine Months Ended September 30,

 

 

Three Months Ended March 31,

 

 

2021

 

 

2020

 

 

2022

 

 

2021

 

Supplemental disclosure of cash flow information:

 

 

 

 

 

 

 

 

 

 

 

 

Cash paid for interest

 

$

443

 

 

$

422

 

 

$

113

 

 

$

159

 

 

 

 

 

 

 

 

 

 

 

 

 

Supplemental disclosure of noncash investing and financing activities:

 

 

 

 

 

 

 

 

 

 

 

 

Conversion of redeemable convertible preferred stock to common stock upon closing of the initial public offering

 

$

 

 

$

303,326

 

Property and equipment additions in accounts payable and accrued expenses

 

$

7,515

 

 

$

2,809

 

 

$

15,306

 

 

$

6,671

 

Operating lease liabilities arising from obtaining right-of-use assets

 

$

24,164

 

 

$

5,795

 

 

$

4,822

 

 

$

23,366

 

Issuance of common stock for research and development license

 

$

 

 

$

264

 

Equity issuance costs in accounts payable and accrued expenses

 

$

118

 

 

$

342

 

 

$

67

 

 

$

250

 

Fair value of common shares issued to settle success payment liability

 

$

30,000

 

 

$

 

Contingent consideration liabilities assumed in asset acquisition

 

$

36,513

 

 

$

 

 

$

 

 

$

36,513

 

Fair value of equity instruments issued in connection with asset acquisition

 

$

120,032

 

 

$

 

 

$

 

 

$

120,032

 

Accretion of redeemable convertible preferred stock to redemption value, including dividends on preferred stock

 

$

 

 

$

1,277

 

 

The accompanying notes are an integral part of these condensed consolidated financial statements

6


 

Beam Therapeutics Inc.

Notes to Condensed Consolidated Financial Statements (Unaudited)

1. Nature of the business and basis of presentation

Organization

Beam Therapeutics Inc., which we refer to herein as the “Company” or “Beam,” is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. Beam’s vision is to provide life-long cures to patients suffering from genetic diseases. The Company was incorporated on January 25, 2017 as a Delaware corporation and began operations in July 2017. Its principal offices are in Cambridge, Massachusetts.

In February 2021, the Company entered into an Agreement and Plan of Merger, or the Guide Merger Agreement, to acquire Guide Therapeutics, Inc., or Guide. Pursuant to the Guide Merger Agreement, the Company paid Guide’s former stockholders and optionholders upfront consideration in an aggregate amount of $120.0 million, excluding customary purchase price adjustments, in shares of its common stock, based upon the volume-weighted average price of the Company’s common stock over the ten-trading day period ending on February 19, 2021.In addition, Guide’s former stockholders and optionholders are eligible to receive up to an additional $100.0 million in technology milestone payments and $220.0 million in product milestone payments, payable in the Company’s common stock.

Liquidity and capital resources

Since its inception, the Company has devoted substantially all of its resources to building its base editing platform and advancing development of its portfolio of programs, establishing and protecting its intellectual property, conducting research and development activities, making arrangements to conduct manufacturing activities with contract manufacturing organizations, research and development costs including preclinical studies and IND-enabling studies, organizing and staffing the Company, maintaining its facilities and new facility build-outs, business planning, raising capital and providing general and administrative support for these operations. The Company is also in the process of developing internal manufacturing capabilities. The Company is subject to risks and uncertainties common to early-stage companies in the biotechnology industry including, but not limited to, technical risks associated with the successful research, development and manufacturing of product candidates, development by competitors of new technological innovations, dependence on key personnel, protection of proprietary technology, compliance with government regulations and the ability to secure additional capital to fund operations. Current and future programs will require significant research and development efforts, including extensive preclinical and clinical testing and regulatory approval prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel and infrastructure. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will realize significant revenue from product sales.

In February 2020, the Company completed its initial public offering, or IPO, in which the Company issued and sold 12,176,471 shares of its common stock, including 1,588,235 shares pursuant to the full exercise of the underwriters’ option to purchase additional shares, at a public offering price of $17.00 per share, for aggregate gross proceeds of $207.0 million. The Company received approximately $188.3 million in net proceeds after deducting underwriting discounts and offering expenses payable by the Company. In connection with the IPO, all outstanding shares of the Company’s redeemable convertible preferred stock converted into 29,127,523 shares of its common stock.

In October 2020, the Company issued and sold 5,750,000 shares of its common stock, including 750,000 shares pursuant to the full exercise of the underwriters’ option to purchase additional shares, at a public offering price of $23.50 per share, for aggregate gross proceeds of $135.1 million. The Company received approximately $126.6 million in net proceeds after deducting underwriting discounts and offering expenses payable by the Company.

On January 16, 2021, the Company entered into a Securities Purchase Agreement with certain purchasers, pursuant to which the Company agreed to sell and issue to the purchasers, in a private placement, shares of common stock of the Company. The closing of the private placement occurred on January 21, 2021. The Company issued and sold 2,795,700 shares of its common stock at a purchase price of $93.00 per share for aggregate gross proceeds of $260.0 million, before deducting fees to the placement agents and other offering expenses payable by the Company (See Note 11, Preferred and common stock). The Company received approximately $252.0 million in net proceeds after deducting fees to the placement agents and offering expenses payable by the Company.

In April 2021, the Company entered into an at the market, or ATM, sales agreement, or the Sales Agreement, with Jefferies LLC, or Jefferies, pursuant to which the Company was entitled to offer and sell, from time to time at prevailing market prices, shares of the Company’s common stock having aggregate gross proceeds of up to $300.0 million. The Company agreed to pay Jefferies a commission of up to 3.0% of the aggregate gross sale proceeds of any shares sold by Jefferies under the Sales Agreement. As of September 30,Between April and July 2021, the Company has sold 2,908,009 shares of its common stock under the Sales Agreement at an average price of $103.16 per share for aggregate gross proceeds of $300.0 million, before deducting commissions and offering expenses payable by the Company.

In July 2021, the Company and Jefferies entered into an amendment to the Sales Agreement to provide for an increase in the aggregate offering amount under the Sales Agreement,Agreement, such that as of July 7, 2021, the Company may offer and sell shares of common stock having an aggregate offering price of an additional $500.0 million. As of September 30, 2021,March 31, 2022, the Company has sold 1,765,8332,873,956

7


additional shares of its common stock under the amended Sales Agreement at an average price of $107.8892.71 per share for aggregate gross proceeds of $190.5266.5 million, before deducting commissions and offering expenses payable by the Company, resulting in an aggregate of $490.5 million in gross proceeds received under the Sales Agreement as of September 30, 2021.Company.

Since its inception, the Company has incurred substantial losses and had an accumulated deficit of $703.6837.5 million as of September 30, 2021.March 31, 2022. The Company expects to generate operating losses and negative operating cash flows for the foreseeable future.

The Company expects that its cash, cash equivalents, and marketable securities as of September 30, 2021March 31, 2022 of $933.41.2 millionbillion will be sufficient to fund its operations for at least the next 12 months from the date of issuance of these financial statements. The Company will need additional financing to support its continuing operations and pursue its growth strategy. Until such time as the Company can generate significant revenue from product sales, if ever, it expects to finance its operations through a combination of equity offerings, debt financings, collaborations, strategic alliances and licensing arrangements. The Company may be unable to raise additional funds or enter into such other agreements when needed on favorable terms or at all. The inability to raise capital as and when needed would have a negative impact on the Company’s financial condition and its ability to pursue its business strategy. The Company will need to generate significant revenue to achieve profitability, and it may never do so.

COVID-19-related significant risks and uncertainties

With the ongoing concernconcerns related to the COVID-19 pandemic, during 2020 and in the first nine months of 2021, the Company has maintained and expanded its business continuity plans to address and mitigate the impact of the COVID-19 pandemic on its business. In March 2020, to protect the health of its employees, and their families and communities, the Company restricted access to its offices to personnel who performed critical activities that must be completed on-site, limited the number of such personnel that could be present at its facilitiesfacilities at any one time, and requested that most of its employees work remotely. In May 2020,2021, as certain states eased restrictions, the Company established new protocols to better allow its full laboratory staff access to the Company’s facilities. These protocols included several shifts working over a seven-day-week protocol. In June 2021, as certain states continued to ease restrictions, the Company started to allow for all of its employeesentire workforce the ability to work on-site at the Company’s facilities, with fewer restrictions, particularly for vaccinated employees. The CompanyCompany

7


expects to continue incurring additional costs to ensure it adheres to the COVID-19 guidelines instituted by the Centers for Disease Control and Prevention and to provide a safe working environment to its onsite employees.

The extent to which the COVID-19 pandemic impacts the Company’s business, its corporate development objectives, and its results of operations and financial condition, includingand the value of and market for its common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the ultimate duration scope and severity of the pandemic, the duration and extent of travel restrictions, andquarantines, social distancing in the United States and other countries, business closures and business disruptions,closure requirements, and the effectiveness of actions taken in the United States and other countriesglobally to contain and treat the disease, periodic spikes in infection rates, new strains of the virus that cause outbreaks of COVID-19, and the broad availability of effective vaccines.disease. Disruptions to the global economy and supply chain, disruption of global healthcare systems, and other significant impacts of the COVID-19 pandemic could have a material adverse effect on the Company’s business, financial condition, results of operations and growth prospects.

While the COVID-19 pandemic did not significantly impact the Company’s business or results of operations during the ninethree months ended September 30, 2021,March 31, 2022, the length and extent of the pandemic, its consequences, and containment efforts will determine the future impact on the Company’s operations and financial condition.

2. Summary of significant accounting policies

The Company’s significant accounting policies are disclosed in the audited consolidated financial statements for the year ended December 31, 2020,2021, and notes thereto, which are included in the Company’s Annual Report on Form 10-K that was filed with the Securities and Exchange Commission on March 15, 2021,February 28, 2022, or the 20202021 Form 10-K. Since the date of those financial statements, there have been no material changes to Beam’s significant accounting policies except as noted below.policies.

Basis of presentation

The accompanying condensed consolidated financial statements have been prepared in accordance with United States generally accepted accounting principles, or GAAP. Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAP as found in the Accounting Standards Codification, or ASC, and Accounting Standards Update, or ASU, of the Financial Accounting Standards Board, or FASB.

Principles of consolidation

The accompanying condensed consolidated financial statements include the results of operations of the Company and its wholly-owned subsidiaries. All intercompany transactions and balances have been eliminated in consolidation.

In September 2021, the Company's wholly-owned subsidiary Blink Therapeutics Inc., or Blink, merged with and into Beam, such that Blink’s separate corporate existence ceased and Beam continued as the surviving corporation.

8


Use of estimates

The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, revenues and expenses, the determination of the fair value equity instruments and intangible assets acquired in an asset acquisition, and the disclosure of contingent assets and liabilities as of and during the reporting period. The Company bases its estimates and assumptions on historical experience when available and on various factors that it believes to be reasonable under the circumstances. The Company evaluates itsSignificant estimates and assumptions on an ongoing basis.reflected in these condensed consolidated financial statements include, but are not limited to, incremental borrowing rate used in the calculation of lease liabilities, research and development expenses, stock-based compensation, contingent consideration liabilities, success payments and certain judgements regarding revenue recognition. Actual results could differ from these estimates.

Cash, cash equivalents, and restricted cash

Cash and cash equivalents consist of standard checking accounts, money market accounts, and all highly liquid investments with a remaining maturity of three months or less at the date of purchase. Restricted cash represents collateral provided for letters of credit issued as security deposits in connection with the Company’s leases of its corporate and manufacturing facilities.

The following table reconciles cash, cash equivalents, and restricted cash reported within the Company’s condensed consolidated balance sheets to the total of the amounts shown in the condensed consolidated statements of cash flows (in thousands):

 

September 30,
2021

 

 

September 30,
2020

 

 

March 31,
2022

 

 

March 31,
2021

 

Cash and cash equivalents

 

$

612,023

 

$

137,903

 

 

$

296,821

 

$

97,241

 

Restricted cash

 

 

14,840

 

 

 

14,840

 

 

 

12,746

 

 

 

14,840

 

Total cash, cash equivalents, and restricted cash

 

$

626,863

 

 

$

152,743

 

 

$

309,567

 

 

$

112,081

 

Asset acquisitions

In 2018, the Company adoptedASU 2017-01, Business Combinations, or ASU 2017-01, which clarified the definition of a business. The Company measures and recognizes asset acquisitions that are not deemed to be business combinations based on the cost to acquire the assets, which includes transaction costs, and the consideration is allocated to the items acquired based on a relative fair value methodology. Goodwill is not recognized in asset acquisitions. In an asset acquisition, the cost allocated to acquire in-process research and development with no alternative future use is charged to research and development expense at the acquisition date.8


At the time of acquisition, the Company determines if a transaction should be accounted for as a business combination or acquisition of assets.

Contingent consideration liabilities

The estimated fair value of contingent consideration liabilities, initially measured and recorded on the acquisition date, are considered to be a Level 3 measurement and are reviewed quarterly, or whenever events or circumstances occur that indicate a change in fair value. The contingent consideration liabilities are recorded at fair value at the end of each reporting period with changes in estimated fair values recorded in other income (expense) in the condensed consolidated statements of operations and other comprehensive loss.

The estimated fair value is determined based on probability adjusted discounted cash flow models that include significant estimates and assumptions pertaining to technology and product development. Significant changes in any of the probabilities of success would result in a significantly higher or lower fair value measurement. Significant changes in the probabilities as to the periods in which milestones will be achieved would result in a significantly lower or higher fair value measurement. 

3. Property and equipment, net

Property and equipment consist of the following (in thousands):

 

September 30,
2021

 

 

December 31,
2020

 

 

March 31,
2022

 

 

December 31,
2021

 

Leasehold improvements

 

$

58,041

 

$

57,760

 

Lab equipment

 

$

24,374

 

$

17,201

 

 

35,489

 

29,905

 

Leasehold improvements

 

12,769

 

12,706

 

Furniture and fixtures

 

1,081

 

1,078

 

 

3,699

 

3,679

 

Computer equipment

 

576

 

547

 

 

1,866

 

1,646

 

Construction in process

 

 

46,255

 

 

 

15,880

 

 

 

14,527

 

 

 

7,349

 

Total property and equipment

 

85,055

 

47,412

 

 

113,622

 

100,339

 

Less accumulated depreciation

 

 

(13,522

)

 

 

(8,899

)

 

 

(19,334

)

 

 

(16,081

)

Property and equipment, net

 

$

71,533

 

 

$

38,513

 

 

$

94,288

 

 

$

84,258

 

 

The following table summarizes depreciation expense incurred (in thousands):

 

 

 

Three Months Ended September 30,

 

 

Nine Months Ended September 30,

 

 

 

2021

 

 

2020

 

 

2021

 

 

2020

 

Depreciation expense

 

$

1,675

 

 

$

1,218

 

 

$

4,642

 

 

$

3,463

 

9


 

 

Three Months Ended March 31,

 

 

 

2022

 

 

2021

 

Depreciation expense

 

$

3,262

 

 

$

1,398

 

 

4. fair value of financial instruments

The Company’s financial instruments that are measured at fair value on a recurring basis consist of cash equivalents, marketable securities, equity securities of Verve Therapeutics, Inc, or Verve, contingent consideration liabilities related to the agreement and plan of merger pursuant to which the Company acquired Guide, or the Guide Merger Agreement, equity securities of Verve Therapeutics, Inc., or Verve, and success payment derivative liabilities pursuant to the license agreement, or the Harvard License Agreement, between President and Fellows of Harvard University, or Harvard, and the Company, and the license agreement, or the Broad License Agreement, between The Broad Institute, Inc., or Broad Institute, and the Company.

The Company also holds an investment in privately issued corporate equity securities, which are accounted for as investments in equity securities. This investment does not have a readily determinable fair value and the Company values the investment based on the cost of the equity securities adjusted for observable market transactions or impairments, if any, and records any changes in value through earnings.

The following table setstables set forth the fair value of the Company’s financial assets and liabilities by level within the fair value hierarchy at September 30, 2021March 31, 2022 (in thousands):

 

 

Carrying
amount

 

Fair
value

 

Level 1

 

Level 2

 

Level 3

 

 

Carrying
amount

 

 

Fair
value

 

 

Level 1

 

 

Level 2

 

 

Level 3

 

Assets

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Cash equivalents:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Money market funds

 

$

583,524

 

$

583,524

 

$

583,524

 

$

 

$

 

 

$

152,020

 

$

152,020

 

$

152,020

 

$

 

$

 

Commercial paper

 

28,499

 

28,499

 

 

28,499

 

 

 

144,801

 

144,801

 

 

144,801

 

 

Marketable securities:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Commercial paper

 

317,324

 

317,324

 

 

317,324

 

 

 

730,967

 

730,967

 

 

730,967

 

 

Corporate notes

 

4,058

 

4,058

 

 

4,058

 

 

 

18,125

 

18,125

 

 

18,125

 

 

Equity securities included in other long-term investments:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

U.S. Treasury securities

 

164,205

 

164,205

 

 

164,205

 

 

Equity securities included in marketable securities:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Corporate equity securities

 

 

24,438

 

 

24,438

 

 

 

 

24,438

 

 

 

 

 

12,482

 

 

 

12,482

 

 

 

12,482

 

 

 

 

 

 

 

Total assets

 

$

957,843

 

$

957,843

 

$

583,524

 

$

374,319

 

$

 

 

$

1,222,600

 

 

$

1,222,600

 

 

$

164,502

 

 

$

1,058,098

 

 

$

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Liabilities

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Success payment liability – Harvard

 

$

24,700

 

$

24,700

 

$

 

$

 

$

24,700

 

 

$

14,200

 

$

14,200

 

$

 

$

 

$

14,200

 

Success payment liability – Broad Institute

 

24,900

 

24,900

 

 

 

24,900

 

 

14,400

 

14,400

 

 

 

14,400

 

Contingent consideration liability – Technology

 

19,910

 

19,910

 

$

 

$

 

$

19,910

 

 

24,531

 

24,531

 

$

 

$

 

$

24,531

 

Contingent consideration liability – Product

 

 

7,050

 

 

7,050

 

 

 

 

 

 

7,050

 

 

 

6,384

 

 

 

6,384

 

 

 

 

 

 

 

 

 

6,384

 

Total liabilities

 

$

76,560

 

$

76,560

 

$

 

$

 

$

76,560

 

 

$

59,515

 

 

$

59,515

 

 

$

 

 

$

 

 

$

59,515

 

9


 

The following table setstables set forth the fair value of the Company’s financial assets and liabilities by level within the fair value hierarchy at December 31, 20202021 (in thousands):

 

 

 

Carrying
amount

 

 

Fair
value

 

 

Level 1

 

 

Level 2

 

 

Level 3

 

Assets

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Cash equivalents:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Money market funds

 

$

88,259

 

 

$

88,259

 

 

$

88,259

 

 

$

 

 

$

 

Commercial paper

 

 

60,494

 

 

 

60,497

 

 

 

 

 

 

60,497

 

 

 

 

Corporate notes

 

 

12,314

 

 

 

12,308

 

 

 

 

 

 

12,308

 

 

 

 

Marketable securities:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Commercial paper

 

 

113,622

 

 

 

113,622

 

 

 

 

 

 

113,622

 

 

 

 

Corporate notes

 

 

7,836

 

 

 

7,836

 

 

 

 

 

 

7,836

 

 

 

 

U.S. Treasury securities

 

 

11,009

 

 

 

11,009

 

 

 

 

 

 

11,009

 

 

 

 

Government securities

 

 

5,033

 

 

 

5,033

 

 

 

 

 

 

5,033

 

 

 

 

Total assets

 

$

298,567

 

 

$

298,564

 

 

$

88,259

 

 

$

210,305

 

 

$

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Liabilities

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Success payment liability – Harvard

 

$

35,500

 

 

$

35,500

 

 

$

 

 

$

 

 

$

35,500

 

Success payment liability – Broad Institute

 

 

35,700

 

 

 

35,700

 

 

 

 

 

 

 

 

 

35,700

 

Total liabilities

 

$

71,200

 

 

$

71,200

 

 

$

 

 

$

 

 

$

71,200

 

10


 

 

Carrying
amount

 

 

Fair
value

 

 

Level 1

 

 

Level 2

 

 

Level 3

 

Assets

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Cash equivalents:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Money market funds

 

$

540,094

 

 

$

540,094

 

 

$

540,094

 

 

$

 

 

$

 

Commercial paper

 

 

13,997

 

 

 

13,997

 

 

 

 

 

 

13,997

 

 

 

 

Corporate notes

 

 

5,903

 

 

 

5,903

 

 

 

 

 

 

5,903

 

 

 

 

Marketable securities:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Commercial paper

 

 

368,743

 

 

 

368,743

 

 

 

 

 

 

368,743

 

 

 

 

Corporate notes

 

 

16,743

 

 

 

16,743

 

 

 

 

 

 

16,743

 

 

 

 

Equity securities included in marketable securities

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Corporate equity securities

 

 

20,167

 

 

 

20,167

 

 

 

20,167

 

 

 

 

 

 

 

Total assets

 

$

965,647

 

 

$

965,647

 

 

$

560,261

 

 

$

405,386

 

 

$

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Liabilities

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Success payment liability – Harvard

 

$

21,000

 

 

$

21,000

 

 

$

 

 

$

 

 

$

21,000

 

Success payment liability – Broad Institute

 

 

21,200

 

 

 

21,200

 

 

 

 

 

 

 

 

 

21,200

 

Contingent consideration liability – Technology

 

 

24,359

 

 

$

24,359

 

 

 

 

 

 

 

 

 

24,359

 

Contingent consideration liability – Product

 

 

7,008

 

 

 

7,008

 

 

 

 

 

 

 

 

 

7,008

 

Total liabilities

 

$

73,567

 

 

$

73,567

 

 

$

 

 

$

 

 

$

73,567

 

 

Cash equivalents – Money market funds included within cash equivalents are classified within Level 1 of the fair value hierarchy because they are valued using quoted market prices in active markets. Commercial paper and corporate notes are classified within Level 2 of the fair value hierarchy because pricing inputs are other than quoted prices in active markets, which are either directly or indirectly observable as of the reporting date, and fair value is determined through the use of models or other valuation methodologies.

Marketable securities and long-term investments– Marketable securities, excluding corporate equity securities, are classified within Level 2 of the fair value hierarchy because pricing inputs are other than quoted prices in active markets, which are either directly or indirectly observable as of the reporting date, and fair value is determined using models or other valuation methodologies.

During the nine months ended September 30, 2021, theThe Company heldholds an investment in Verve consisting of shares of Verve’s common and preferred stock. Prior to Verve's initial public offering in June 2021, the Company valued such investment based on the cost of the equity securities adjusted for any observable market transactions. Following theVerve's initial public offering, the equity securities have a readily determinable fair value; however, they arewere subject to transfer restrictions.restrictions for 6 months following Verve's initial public offering. As of September 30, 2021March 31, 2022, the Company owned 546,970 shares of Verve's common stock the value ofvalued at $12.5 million, which is included in long-term investmentsmarketable securities in the condensed consolidated balance sheet. TheIn addition the Company recorded other expense of $7.7 million and other income of $1.0 million for the investment atthree months ended March 31, 2022, and 2021 respectively, related to the changes in fair value of $24.4 million as of September 30, 2021, which resulted in the recognition of $4.9 million of other expense and $22.0 million of other income for the three and nine months ended September 30, 2021, respectively.Verve's stock.

Pursuant to ASU No. 2016-01, Recognition and Measurement of Financial Assets and Financial Liabilities, the Company records changes in the fair value of its investments in equity securities to “Otherother income (expense), net” in the Company’s condensed consolidated statements of operations.

Success payment liabilities – As discussed further in Note 9, License agreements, the Company is required to make payments determinedto Harvard and Broad Institute based upon the achievement of specified multiples of the initial weighted average value of the Company’s redeemable convertible Series A-1 Preferred Stock and the Company's redeemable convertible Series A-2 Preferred Stock, or collectively the Series A Preferred Stock or, subsequent to the Company’s initial public offering, or IPO, the market value of Beam’sthe Company's common stock, at specified valuation dates. The Company’s liability for the share-based success payments under the Harvard License Agreement and Broad License Agreement areAgreements is carried at fair value. To determine the estimated fair value of the success payment liability, the Company uses a Monte Carlo simulation methodology, which models the future movement of stock prices based on several key variables.

The following variables were incorporated in the calculation of the estimated fair value of the Harvard and Broad Institute success payment liabilities:

 

 

Harvard

 

 

Broad Institute

 

 

Harvard

 

 

Broad Institute

 

 

September 30,
2021

 

 

December 31,
2020

 

 

September 30,
2021

 

 

December 31,
2020

 

 

March 31,
2022

 

 

December 31,
2021

 

 

March 31,
2022

 

 

December 31,
2021

 

Fair value of common stock (per share)

 

$

87.01

 

$

81.64

 

$

87.01

 

$

81.64

 

 

$

57.30

 

$

79.69

 

$

57.30

 

$

79.69

 

Expected volatility

 

77

%

 

74

%

 

76

%

 

74

%

 

80

%

 

76

%

 

80

%

 

76

%

Expected term (years)

 

0.10-7.75

 

 

0.35-8.49

 

 

0.10-8.61

 

 

0.35-9.36

 

 

0.10-7.25

 

 

0.10-7.49

 

 

0.10-8.11

 

 

0.10-8.36

 

10


 

The computation of expected volatility was estimated using available information about the historical volatility of stocks of similar publicly traded companies in addition to the Company's own data for a period matching the expected term assumption. In addition, the Company incorporated the estimated number, timing, and probability of valuation measurement dates in the calculation of the success payment liability.

The following table reconciles the change in the fair value of success payment liabilities based on Level 3 inputs (in thousands):

 

 

 

Nine Months Ended September 30, 2021

 

 

 

Harvard

 

 

Broad Institute

 

 

Total

 

Balance at December 31, 2020

 

$

35,500

 

 

$

35,700

 

 

$

71,200

 

Payments

 

 

(15,000

)

 

 

(15,000

)

 

 

(30,000

)

Change in fair value

 

 

4,200

 

 

 

4,200

 

 

 

8,400

 

Balance at September 30, 2021

 

$

24,700

 

 

$

24,900

 

 

$

49,600

 

11


 

 

Three Months Ended March 31, 2022

 

 

 

Harvard

 

 

Broad Institute

 

 

Total

 

Balance at December 31, 2021

 

$

21,000

 

 

$

21,200

 

 

$

42,200

 

Change in fair value

 

 

(6,800

)

 

 

(6,800

)

 

 

(13,600

)

Balance at March 31, 2022

 

$

14,200

 

 

$

14,400

 

 

$

28,600

 

 

Contingent consideration liabilities – As discussed further in Note 8, Guide acquisitionacquisition, under the Guide Merger Agreement, Guide’s former stockholders and optionholders are eligible to receive up to an additional $100.0 million in technology milestone payments and $220.0 million in product milestone payments, payable in the Company’s common stock valued using the volume-weighted average price of the Company’s stock over the ten-day trading period ending two trading days prior to the date on which the applicable milestone is achieved. As these milestones are payable in the Company’s common stock, the milestone payments result in liability classification under ASC 480, Distinguishing Liabilities from Equity. These contingent consideration liabilities are carried at fair value which was estimated by applying a probability-based model, which utilized inputs based on timing of achievement that were unobservable in the market. These contingent consideration liabilities are classified within Level 3 of the fair value hierarchy.

The following table reconciles the change in fair value of the contingent consideration liabilities based on level 3 inputs (in thousands):

 

 

 

Nine Months Ended September 30, 2021

 

 

 

Technology Milestones

 

 

Product Milestones

 

 

Total

 

Balance at February 23, 2021 (inception)

 

$

29,403

 

 

$

7,110

 

 

$

36,513

 

Change in fair value

 

 

(9,493

)

 

 

(60

)

 

 

(9,553

)

Balance at September 30, 2021

 

$

19,910

 

 

$

7,050

 

 

$

26,960

 

 

 

Three Months Ended March 31, 2022

 

 

 

Technology Milestones

 

 

Product Milestones

 

 

Total

 

Balance at December 31, 2021

 

$

24,359

 

 

$

7,008

 

 

$

31,367

 

Change in fair value

 

 

172

 

 

 

(624

)

 

 

(452

)

Balance at March 31, 2022

 

$

24,531

 

 

$

6,384

 

 

$

30,915

 

 

The following variables were incorporated in the calculation of the estimated fair value of the contingent consideration liabilities:

 

 

Technology Milestones

 

 

Product Milestones

 

 

Technology Milestones

 

 

Product Milestones

 

 

September 30,
2021

 

 

September 30,
2021

 

 

March 31,
2022

 

December 31,
2021

 

 

March 31,
2022

 

December 31,
2021

 

Discount Rate

 

7.50

%

 

7.50

%

 

8.50

%

 

 

7.50

%

 

8.50

%

 

 

7.50

%

Probability of Achievement

 

10-50%

 

 

2-15%

 

 

10-75%

 

10-75%

 

 

2-15%

 

2-15%

 

Projected Year of Achievement

 

2022

 

 

2023-2028

 

 

2022-2023

 

2022-2023

 

 

2024-2029

 

2023-2029

 

 

5. Marketable securities

The following table summarizes the Company’s marketable securities held at September 30, 2021March 31, 2022 (in thousands):

 

Amortized Cost

 

 

Gross
Unrealized
Gains

 

 

Gross
Unrealized
Losses

 

 

Fair Value

 

 

Amortized Cost

 

 

Gross
Unrealized
Gains

 

 

Gross
Unrealized
Losses

 

 

Fair Value

 

Commercial paper

 

$

317,303

 

$

26

 

$

(5

)

 

$

317,324

 

 

$

733,068

 

$

 

$

(2,101

)

 

$

730,967

 

Corporate notes

 

 

4,060

 

 

 

0

 

 

 

(2

)

 

 

4,058

 

 

18,249

 

 

(124

)

 

18,125

 

U.S. Treasury securities

 

164,689

 

2

 

(486

)

 

164,205

 

Corporate equity securities

 

 

12,482

 

 

 

 

 

 

 

 

 

12,482

 

Total

 

$

321,363

 

 

$

26

 

 

$

(7

)

 

$

321,382

 

 

$

928,488

 

 

$

2

 

 

$

(2,711

)

 

$

925,779

 

The following table summarizes the Company’s marketable securities held at December 31, 20202021 (in thousands):

 

Amortized Cost

 

 

Gross
Unrealized
Gains

 

 

Gross
Unrealized
Losses

 

 

Fair Value

 

 

Amortized Cost

 

 

Gross
Unrealized
Gains

 

 

Gross
Unrealized
Losses

 

 

Fair Value

 

Commercial paper

 

$

113,628

 

$

11

 

$

(17

)

 

$

113,622

 

 

$

368,778

 

$

32

 

$

(67

)

 

$

368,743

 

Corporate notes

 

7,839

 

2

 

(5

)

 

7,836

 

 

16,758

 

 

(15

)

 

16,743

 

U.S. Treasury securities

 

11,009

 

0

 

 

11,009

 

Government securities

 

 

5,033

 

 

 

0

 

 

 

0

 

 

 

5,033

 

Corporate equity securities

 

 

20,167

 

 

 

 

 

 

 

 

 

20,167

 

Total

 

$

137,509

 

 

$

13

 

 

$

(22

)

 

$

137,500

 

 

$

405,703

 

 

$

32

 

 

$

(82

)

 

$

405,653

 

11


The amortized cost of marketable securities is adjusted for amortization of premiums and accretion of discounts to maturity. At September 30, 2021,March 31, 2022, the balance in accumulated other comprehensive loss(loss) income was comprised solely of activity related to marketable securities. There were 0 realized gains or losses recognized on the sale or maturity of marketable securities for the ninethree months ended September 30,March 31, 2022 and 2021 and 2020 and, as a result, the Company did not reclassify any amounts out of accumulated other comprehensive loss(loss) income for the same periods.

The Company holds debt securities of companies with high credit quality and has determined that there was no material change in the credit risk of any of its debt securities. The contractual maturity dates of all the investments are less than one year.

6. Accrued expenses and other current liabilities

Accrued expenses and other current liabilities consist of the following (in thousands):

 

12


 

September 30,
2021

 

 

December 31,
2020

 

 

March 31,
2022

 

 

December 31,
2021

 

Employee compensation and related benefits

 

$

5,841

 

$

7,591

 

 

$

6,771

 

$

11,661

 

Research costs

 

4,987

 

2,423

 

 

6,545

 

3,133

 

Professional fees

 

3,733

 

1,948

 

 

3,542

 

3,330

 

Process development and manufacturing costs

 

3,285

 

2,272

 

 

2,299

 

3,833

 

Other

 

 

5,119

 

 

 

4,229

 

 

 

4,905

 

 

 

6,964

 

Total

 

$

22,965

 

 

$

18,463

 

 

$

24,062

 

 

$

28,921

 

 

7. Leases

Operating leases

The Company’s operating leases are as follows:

A February 2018 lease for 38,203 square feet of office and laboratory space, which commenced in March 2018 and terminates in September 2028. The lease is subject to fixed-rate rent escalations and provided for $6.1 million in tenant improvements and a term extension option, which is not reasonably certain of exercise.
An October 2018 lease for laboratory space as amended, which commenced in April 2019 and was amended in March 2020 and April 2020. The amended lease commenced in April 2020 and terminates in December 2025. The amended lease is subject to fixed-rate rent escalations and providedprovides an option to extend the lease for 2 additional two-year periods through December 31, 2029, which are not reasonably certain of being exercised. Upon commencement of theThrough March 2020 amendment,31, 2022, the Company has recorded an operating lease right-of-use, or ROU assetassets and a lease liabilityliabilities of $4.214.1 million. Upon commencement of the April 2020 amendment, the Company recorded an operating lease ROU assetmillion and a lease liability of $1.814.0 million.million related to this lease.
Leases in June 2019 and July 2019 for office and laboratory space, both of which commenced in October 2019 and terminate in December 2021. The leases are subject to fixed-rate rent escalations.
An April 2019 lease for office and laboratory space, to be built.that was built over the course of 2020 and 2021. Pursuant to the terms of the original lease agreement, the first phase of the lease commenced in October 2020 (rent payments for the first phase beginningbegan in August 2021) and the second phase of the lease commenced in January 2021 (rent payments for the second phase are expected to begin at the earliestbegan in the first half of 2022)February 2022). The lease is subject to fixed-rate rent escalations and provides for $23.4 million in tenant improvements and the option to extend the lease for 2 terms of five years each, which are not reasonably certain of exercise. The Company determined that it is the accounting owner of all tenant improvements. Upon executing the lease, theThe Company mademaintains a security deposit of $11.89.7 million in the form of a letter of credit, which is included in restricted cash as of September 30, 2021March 31, 2022 and December 31, 2020.2021. Upon commencement of the first phase of this lease in October 2020, the Company recorded an operating lease ROU asset of $66.8 million and a lease liability of $68.8 million and upon commencement of the second phase of this lease in January 2021, the Company recorded an operating lease ROU asset of $22.0 million and a corresponding lease liability of $23.0 million. Subsequently, during the second quarter of 2021, the Company amended the rent commencement dates of the first and second phases of this lease. Pursuant to the terms of the amendment, the lease will terminate onin February 28, 2034, which is 12 years years from the amended second phase rent commencement date. As a result, the Company recorded an increase in the ROU asset of $0.5 million and an increase in lease liability of $0.5 million.

The following table summarizes operating lease costs and sublease income (in thousands):

 

 

Three Months Ended
September 30,

 

 

Nine Months Ended
September 30,

 

 

Three Months Ended
March 31,

 

 

2021

 

 

2020

 

 

2021

 

 

2020

 

 

2022

 

 

2021

 

Operating lease costs

 

$

4,557

 

$

1,644

 

13,660

 

4,946

 

 

$

4,386

 

$

4,558

 

Variable lease costs

 

588

 

249

 

1,162

 

786

 

 

1,136

 

210

 

Short-term lease costs

 

383

 

 

761

 

0

 

 

232

 

 

Sublease income

 

 

(331

)

 

 

0

 

Total

 

$

5,528

 

 

$

1,893

 

 

$

15,583

 

 

$

5,732

 

 

$

5,423

 

 

$

4,768

 

12


 

The following table summarizes the lease term and discount rate for operating leases:

 

 

September 30,
2021

 

 

December 31,
2020

 

 

March 31,
2022

 

 

December 31,
2021

 

Weighted-average remaining lease term (years)

 

11.4

 

 

11.5

 

 

10.6

 

 

11.1

 

Weighted-average discount rate

 

7.1

%

 

7.4

%

 

7.0

%

 

7.0

%

13


 

The following table summarizes the lease costs for amounts included in the measurement of lease liabilities (in thousands):

 

 

Three Months Ended
September 30,

 

 

Nine Months Ended
September 30,

 

 

Three Months Ended
March 31,

 

 

2021

 

 

2020

 

 

2021

 

 

2020

 

 

2022

 

 

2021

 

Operating cash flows used for operating leases

 

$

3,058

 

$

2,014

 

$

7,053

 

$

5,132

 

 

$

4,509

 

$

1,685

 

Operating lease liabilities arising from obtaining ROU assets

 

 

 

24,164

 

5,795

 

 

4,822

 

23,366

 

 

At September 30, 2021,March 31, 2022, the future minimum lease payments for the Company’s operating leases for each of the next five years ending December 31and total thereafter were as follows (in thousands):

 

Remainder of 2021

 

$

4,159

 

2022

 

16,616

 

Remainder of 2022

 

$

13,865

 

2023

 

17,262

 

 

19,006

 

2024

 

17,787

 

 

19,586

 

2025

 

18,260

 

 

20,066

 

2026

 

17,143

 

Thereafter

 

 

132,835

 

 

 

115,692

 

Undiscounted lease payments

 

206,919

 

 

205,358

 

Less: imputed interest

 

 

(67,315

)

 

 

(59,965

)

Total operating lease liabilities

 

$

139,604

 

 

$

145,393

 

 

In August 2020, the Company entered into a lease agreement with Alexandria Real Estate Equities, Inc., or the Landlord, to build a 100,000 square foot manufacturing facility in Research Triangle Park, North Carolina intended to support a broad range of clinical programs. The lease has a term of 15 years years following the commencement date and provides the Company the option to extendthe lease term for 2 five-year terms. It is subject to fixed rate escalation increases and also provides up to $20.0 million for reimbursement of tenant improvements. As the lease had not commenced as of September 30, 2021,March 31, 2022, the Company has not recorded an operating lease ROU asset or lease liability for this lease in the accompanying condensed consolidated balance sheets. The lease payments are subject to adjustment following the determination of the total project costs of the landlord. The initial estimate of the minimum amount of undiscounted lease payments due under this lease is $81.169.0 million. The Company anticipates that the facility will be operational in the first quarter of 2023.2023 at which time it would begin making rent payments. The tabular disclosure of minimum lease payments above does not include payments due under this lease.

In August 2021, the Company executed a lease amendment to its April 2019 lease for office and laboratory space in Cambridge, Massachusetts to occupy additional space. The term of this lease will run concurrent with the term of the April 2019 lease. The initial estimate of the minimum amount of undiscounted lease payments due under this lease is $11.1 million. As the lease had not yet commenced as of September 30, 2021,March 31, 2022, the Company has not recorded an operating lease ROU asset or lease liability for this lease in the accompanying condensed consolidated balance sheets and the tabular disclosure of minimum lease payments above does not include the payments under this lease.

In October 2021, the Company executed a lease for additional office and laboratory space at an existing facility. The term of this lease will run through December 31, 2025. The Company took access to part of this space in October 2021 and will record an operating lease ROU asset and lease liability during the fourth quarter of 2021. The initial estimate of the minimum amount of undiscounted lease payments due under this segment of the lease is $1.9 million. As part of this lease, the Company will take access to the remainder of this space in January 2022 and will record an operating lease ROU asset and lease liability during the first quarter of 2022. The initial estimate of the minimum amount of undiscounted lease payments due under this lease is $2.6 million.sheets. The tabular disclosure of minimum lease payments above does not include the payments due under this segment of the lease.

8. Guide acquisition

On February 23, 2021, the Company entered into the Guide Merger Agreement. Under the Guide Merger Agreement, the Company paid Guide’s former stockholders and optionholders upfront consideration in an aggregate amount of $120.0 million, excluding customary purchase price adjustments and closing costs, in shares of the Company’s common stock, based upon the volume-weighted average price of the Company’s stock over the ten trading day period ending on February 19, 2021. Pursuant to the Guide Merger Agreement, Beamthe Company acquired all of the issued and outstanding shares of Guide. The Company issued a total of 1,087,153 shares of its common stock valued at $120.0 million in connection with the upfront payment to Guide’s former stockholders and optionholders. The Guide transaction resulted in the acquisition of certain know-how and intellectual property assets related to Guide’s proprietary in vivo LNP screening technology and its library of lipids and lipid nanoparticle formulations identified using the screening technology. Management determined that the acquired assets do not meet the definition of a business pursuant to ASC 805, Business Combinations, as substantially all of the fair value of the acquired assets is concentrated into one identifiable asset, the LNP screening technology and associated lipid library. As of the date of closing of the transactions contemplated by the Guide Merger Agreement, or the Guide Merger Agreement Date, the asset acquired had no alternative future use and had not reached a stage of technological feasibility. As a result, all share-based and cash payment obligations have been recorded as research and development expense in the

14


accompanying condensed consolidated statements of operations and other comprehensive loss in the amount of $155.0 million. The total transaction price was allocated to the assets acquired and liabilities assumed on a relative fair value basis.

13


In addition, Guide’s former stockholders and optionholders are eligible to receive up to an additional $100.0 million in technology milestone payments and $220.0 million in product milestone payments, payable in the Company’s common stock valued using the volume-weighted average price of the Company’s stock over the ten-day tradingten trading-day period ending two trading days prior to the date on which the applicable milestone is received.

The Company determined that all future technology and product milestone payments are classified as contingent consideration liabilities under ASC 480 and therefore the Company recorded a liability for these milestonesmilestone payments as of the Guide Merger Agreement Date at fair value of $36.5 million. These contingent consideration liabilities will beare remeasured at fair value each financial reporting period, with the resulting impact reflected in the Company’s condensed consolidated statements of operations and other comprehensive loss, presented within other income (expense).

The transaction price was determined and allocated as follows (in thousands):

 

Transaction price

 

 

 

 

Fair value of equity instruments issued

 

$

120,032

 

 

Technology and product contingent consideration liabilities

 

 

36,513

 

 

Transaction costs

 

 

2,531

 

 

Total transaction price

 

$

159,076

 

 

Transaction price allocated

 

 

 

 

In-process research and development

 

$

154,953

 

 

Cash acquired

 

 

3,151

 

 

Prepaid expenses and other assets

 

 

264

 

 

Property and equipment

 

 

1,835

 

 

Assembled workforce

 

 

300

 

 

Other liabilities assumed

 

 

(1,427

)

 

Total transaction price

 

$

159,076

 

 

 

 

9. License agreements

Harvard license agreement

Under the terms of the Harvard License Agreement, Harvard is entitled to receive success payments, in cash or shares of Company stock, determined based upon the achievement of specified multiples of the initial weighted average value of the Company’s Series A Preferred Stock at specified valuation dates. The success payments range from $5.0 million to a maximum of $105.0 million and have valuation multiples that range from 5 times to 40 times the initial weighted average value of the Series A Preferred Stock. Subsequent to the Company’s February 2020 IPO, the amount of success payments is based on the market value of Beam’sthe Company’s common stock.

The Company is required to make success payments to Harvard during a period of time, or the Harvard Success Payment Period, which has been determined to be the later of (1) the ninth anniversary of the Harvard License Agreement or (2) the earlier of (a) the twelfth anniversary of the Harvard License Agreement and (b) the third anniversary of the first date on which a licensed product receives regulatory approval in the United States. During the Harvard Success Payment Period and beginning one year after the Company’s IPO, the Company will perform a calculation of any amounts owed to Harvard on each rolling 90-day period, commencing one year after the Company’s IPO, with the first success payment becoming due in period.May 2021.

The following table summarizes the Company’s success payment liability for Harvard (in thousands):

 

 

September 30,
2021

 

 

December 31,
2020

 

Harvard success payment liability

 

$

24,700

 

 

$

35,500

 

The following table summarizes the expense resulting from the change in the fair value of the success payment liability for Harvard (in thousands):

 

 

Three Months Ended September 30,

 

 

Nine Months Ended September 30,

 

 

 

2021

 

 

2020

 

 

2021

 

 

2020

 

Change in fair value of Harvard success payment liability

 

$

(17,900

)

 

$

(1,400

)

 

$

4,200

 

 

$

4,300

 

15


In May 2021, the first success payment measurement occurred and amounts due to Harvard were calculated to be $15.0 million. The Company elected to make the payment in shares of the Company’s common stock and issued 174,825 shares of the Company’s common stock to settle this liability on June 10, 2021. The Company may owe Harvard success payments of up to an additional $90.0 million. As of March 31, 2022, 0 success payments were due to Harvard.

The following table summarizes the Company’s success payment liability for Harvard (in thousands):

 

 

March 31,
2022

 

 

December 31,
2021

 

Harvard success payment liability

 

$

14,200

 

 

$

21,000

 

The following table summarizes the expense resulting from the change in the fair value of the success payment liability for Harvard (in thousands):

 

 

Three Months Ended March 31,

 

 

 

2022

 

 

2021

 

Change in fair value of Harvard success payment liability

 

$

(6,800

)

 

$

1,000

 

14


The annual maintenance fee under the Harvard License Agreement is recorded as research and development expense. Patent prosecutionAnnual patent costs are recognizedwill be expensed as expense in the period incurred. As of September 30, 2021,March 31, 2022, the Company determined that product development and regulatory approval milestones and royalties under the Harvard License Agreement were not probable and, as such, 0 amounts were recognized for the ninethree months ended September 30, 2021. As of September 30, 2021,March 31, 2022. There was 0 success payments were dueexpense recorded for these milestones for the three months ended March 31, 2021. During the three months ended March 31, 2022 the Company incurred $0.5 million of expense related to non-royalty sublicense fees owed to Harvard. As of March 31, 2022, the Company has accrued $33.1 million of non-royalty sublicense fees owed to Harvard.

Broad license agreement

Under the terms of the Broad License Agreement, Broad Institute is entitled to receive success payments, in cash or shares of Company common stock, determined based upon the achievement of specified multiples of the initial weighted average value of the Series A Preferred Stock at specified valuation dates. The success payments range from $5.0 million to a maximum of $105.0 million and have valuation multiples that range from 5 times to 40 times the initial weighted average value of the Series A Preferred Stock. Subsequent to the Company’s February 2020Company's IPO, the amount of success payments is based on the market value of Beam’sthe Company’s common stock.

The Company is required to make success payments to Broad Institute during a period of time, or the Broad Success Payment Period, which has been determined to be the earliest of (1) the twelfth anniversary of the Broad License Agreement or (2) the third anniversary of the first date on which a licensed product receives regulatory approval in the United States. During the Broad Success Payment Period and beginning one year after the Company’s IPO, the Company will perform a calculation of any amounts owed to Broad Institute on each rolling 90-day period, commencing one year after the Company’s IPO, with the first success payment becoming due in period.May 2021.

The following table summarizes the Company’s success payment liability for Broad Institute (in thousands):

 

 

September 30,
2021

 

 

December 31,
2020

 

Broad Institute success payment liability

 

$

24,900

 

 

$

35,700

 

The following table summarizes the expense resulting from the change in the fair value of the success payment liability for Broad Institute (in thousands):

 

 

Three Months Ended September 30,

 

 

Nine Months Ended September 30,

 

 

 

2021

 

 

2020

 

 

2021

 

 

2020

 

Change in fair value of Broad Institute success payment liability

 

$

(17,900

)

 

$

(1,300

)

 

$

4,200

 

 

$

4,400

 

In May 2021, the first success payment measurement occurred and amounts due to Broad Institute were calculated to be $15.0 million. The Company elected to make the payment in shares of the Company’s common stock and issued 174,825 shares of the Company’s common stock to settle this liability on June 10, 2021. The Company may owe Broad Institute success payments of up to an additional $90.0 million. As of March 31, 2022, 0 success payments were due to Broad Institute.

The following table summarizes the Company’s success payment liability for Broad Institute (in thousands):

 

 

March 31,
2022

 

 

December 31,
2021

 

Broad Institute success payment liability

 

$

14,400

 

 

$

21,200

 

The following table summarizes the expense resulting from the change in the fair value of the success payment liability for Broad Institute (in thousands):

 

 

Three Months Ended March 31,

 

 

 

2022

 

 

2021

 

Change in fair value of Broad Institute success payment liability

 

$

(6,800

)

 

$

900

 

The annual maintenance fee under the Broad License Agreement is recorded as research and development expense. Patent prosecutionAnnual patent costs are recognizedwill be expensed as expense in the period incurred. As of September 30, 2021,March 31, 2022, the Company determined that product development and regulatory approval milestones and royalties under the Broad License Agreement were not probable and, as such, 0 amounts were recognized for the ninethree months ended September 30, 2021. As of September 30, 2021,March 31, 2022. There was 0 success payments were dueexpense recorded for these milestones for the three months ended March 31, 2021. The Company paid $6.1 million of non-royalty sublicense fees to Broad Institute.Institute during the three months ended March 31, 2022.

Editas license agreement

In May 2018, the Company entered into a license agreement, or the Editas License Agreement, with Editas Medicine, Inc., or Editas. Pursuant to the Editas License Agreement, Editas granted to the Company licenses and options to acquire licenses to certain intellectual property rights owned or controlled by Editas, for specified uses.

The annual maintenance fees under the Editas License Agreement are recorded as research and development expense. Annual patent costs are expensed as incurred. In addition, the Company is required to make certain development, regulatory and commercial milestone payments to Editas upon the achievement of specified milestones. As of September 30, 2021, theThe Company determined that it owed a regulatory milestone payment to Editas under the License Agreement and recognizedrecorded $0.1 million of expense forrelated to these milestones during the three and nine months ended September 30, 2021.

Bio Palette license agreement

In March 2019, the Company entered into a license agreement, or the Bio Palette License Agreement, with Bio Palette Co., Ltd., or Bio Palette, pursuant to which the Company received an exclusive (even as to Bio Palette), sublicensable license under certain patent rights related to base editing owned or controlled by Bio Palette to exploit products for the treatment of human disease throughout the world, but excluding products in the microbiome field in Asia. In addition, the Company granted Bio Palette an exclusive (even as to the Company) license under certain patent rights related to base editing and gene editing owned or controlled by the Company to

16


exploit products in the microbiome field in Asia. Each party to the agreement retains non-exclusive rights to develop and manufacture products in the microbiome field worldwide for the sole purpose of exploiting those products in its own territory. Each party agrees to certain coordination obligations in the microbiome field if either party determines not to exploit their rights in such field. Upon the execution of the Bio Palette License Agreement, the Company paid Bio Palette an upfront fee of $0.5 million and issued to Bio Palette 16,725 shares of its common stock valued at $0.1 million.

Unless earlier terminated, the Bio Palette License Agreement will expire on a licensed product-by-licensed product and country-by-country basis upon the expiration of the applicable royalty term for each such licensed product and country.

Upon the issuance of a certain Bio Palette patent in the United States in June 2020, the Company made a milestone payment of $2.0 million and, in July 2020, issued to Bio Palette 175,000 shares of its common stock valued at $0.3 million. The fair value of the common stock issued to Bio Palette under the Bio Palette License Agreement was measured at the inception of arrangement and expensed when the issuance of shares became probable.31, 2022.

Management concluded that the licenses acquired from each transaction above did not meet the accounting definition of a business as inputs, but no processes or outputs were acquired with the licenses, and the licensed technology had not achieved technological feasibility. As the inputs that were acquired along with the licenses do not constitute a “business,” the transactions have been accounted as asset acquisitions. As of the date of each license agreement, the assets acquired had no alternative future use and the assets had not reached a stage of technological feasibility. As a result, all share-based and cash payment obligations have been recorded as research and development expense in the accompanying condensed consolidated statements of operations and other comprehensive loss.

10. Collaboration and license agreements

Apellis PharmaceuticalsPfizer

15


On June 30,In December 2021, the Company entered into a masterresearch collaboration agreement, or the Pfizer Agreement, with Pfizer Inc., or Pfizer, focused on the use of certain of the Company’s base editing technology to develop in vivo therapies for rare genetic diseases of the liver, muscle, and central nervous system. Under the terms of the Pfizer Agreement, the Company will conduct all research activities through development candidate selection for three base editing programs that target specific genes corresponding to specific diseases that are the subject of such programs. Pfizer will have exclusive rights to license each of the three programs at no additional cost, each an Opt-In Right, and will assume responsibility for subsequent development and commercialization. At the end of the Phase 1/2 clinical trials, the Company may elect to enter into a global co-development and co-commercialization agreement with Pfizer with respect to one program licensed under the collaboration for an option exercise fee equal to a percentage of the applicable development costs incurred by Pfizer, or the Participation Election. In the event the Company elects to exercise its Participation Election, upon the payment of its option exercise fee, Pfizer and the Company would share net profits as well as development and commercialization costs in a 65%/35% (Pfizer/Company) split for such program. The research collaboration is managed on an overall basis by a Joint Research Committee, or JRC, formed by an equal number of representatives from the Company and Pfizer.

At the inception of the Pfizer Agreement, the Company was entitled to receive a nonrefundable upfront payment of $300.0 million in consideration for the rights granted to Pfizer under the collaboration. Should Pfizer exercise its Opt-In Right for any of the three programs, the Company would be eligible to receive development, regulatory, and commercial milestones of up to $350.0 million per program, for potential total consideration of up to $1.35 billion, plus royalty payments on global net sales for each licensed program, if any. If Pfizer does not exercise its Opt-In Right for a program, the Company’s rights in such program revert to the Company and the Company will be required to pay Pfizer earn-out payments equal to a low single digit percentage of net sales earned on such program for a ten-year period, if any. As the $300.0 million upfront fee was not received by the Company as of December 31, 2021, the Company recorded a collaboration receivable for $300.0 million with a corresponding deferred revenue liability. The Company received the $300.0 million in January 2022.

During the collaboration term, Pfizer has a one-time option to substitute a disease that is the subject of a specific program with one pre-defined substitute disease. The collaboration has an initial term of four years and may be extended for an additional year on a program-by-program basis. Pfizer may terminate the Pfizer Agreement for convenience on any or all of the programs by providing 90 days’ prior written notice.

The Company accounts for the Pfizer Agreement under ASC 606, Revenue from Contracts with Customers, or ASC 606, as it includes a customer-vendor relationship as defined under ASC 606 and meets the criteria to be considered a contract.

The overall transaction price as of the inception of the contract was determined to be $300.0 million, which is comprised entirely of the nonrefundable upfront payment. There is no variable consideration included in the transaction price at inception as the future milestone payments are fully constrained and the Company is not required to estimate variable consideration for the royalty payments at contract inception. The Company will re-evaluate the transaction price in each reporting period.

The Company has concluded that the licenses to its base editing technology, including the exclusive development and commercialization rights, are not capable of being distinct from the other performance obligations, and as such the Company has determined that the licenses combined with the other research and development services represent performance obligations and no up-front revenue was recognized for the licenses.

The selling price of each performance obligation was determined based on the Company’s estimated standalone selling price, or the ESSP. The Company developed the ESSP for all of the performance obligations included in the Pfizer Agreement with the objective of determining the price at which it would sell such an item if it were to be sold regularly on a standalone basis. The Company allocated the stand-alone selling price to the performance obligations based on the relative standalone selling price method.

The Company recognizes revenue for each performance obligation as it is satisfied during the term of the agreement using an input method. The Company allocated the transaction price of $300.0 million to each of the three performance obligations, which includes each of the three base editing programs combined with the research and development services, licenses, and exclusive development and commercialization rights. Revenue is recognized using an input method based on the actual costs incurred as a percentage of total budgeted costs towards satisfying the performance obligation as this method provides the most faithful depiction of the entity’s performance in transferring control of the goods and services promised to Pfizer and represents the Company’s best estimate of the period of the obligation. For the three months ended March 31, 2022, the Company recognized $6.3 million of revenue related to the Pfizer Agreement. As of March 31, 2022, there is $95.6 million and $198.1 million of current and long-term deferred revenue, respectively, related to the Pfizer Agreement.

Apellis Pharmaceuticals

In June 2021, the Company entered into a research collaboration agreement, or the Apellis Agreement, with Apellis Pharmaceuticals, Inc., or Apellis, focused on the use of certain of the Company’s base editing technology to discover new treatments for complement system-driven diseases. Under the terms of the Apellis Agreement, the Company will apply certain of its base editing technology and conduct preclinical research on up to six base editing programs that target specific genes within the complement system in various organs, including the eye, liver, and brain. Apellis will havehas an exclusive rightsoption to license eachany or all of the six programs, (eachor in each case, an “Opt-In Right”)Opt-In Right, and will assume responsibility for

16


subsequent development. The Company may elect to enter into a 50-50 U.S. co-development and co-commercialization agreement with Apellis with respect to one program licensed under the collaboration.instead of a license. The collaboration will beis managed on an overall basis by an Alliance Steering Committee, or ASC,alliance steering committee formed by an equal number of representatives from the Company and Apellis.

As part of the collaboration, the Company is eligible to receive a total of $75.0 million in upfront and near-term milestones from Apellis, which is comprised of $50.0 million received upon signing and an additional $25.0 million payment on June 30, 2022, the one-year anniversary of the signingeffective date of the Apellis Agreement, or the First Anniversary Payment. Following any exercise of an Opt-In Right for any of the six programs, the Company will be eligible to receive development, regulatory, and sales milestones from Apellis, as well as royalty payments on sales. The collaboration has an initial term of five years and may be extended up to two yearson a per year and program-by-program basis. During the collaboration term, Apellis may, subject to certain limitations, substitute a specific complement gene and/or organ for any of the initial base editing programs. Apellis may terminate the Apellis Agreement for convenience on any or all of the programs by providing prior written notice. The Company received the $50.0 million in upfront payment from Apellis in July 2021.

The Company accounts for the Apellis Agreement under ASC 606, Revenue from Contracts with Customers, or ASC 606 as it includes a customer-vendor relationship as defined under ASC 606 and meets the criteria to be considered a contract.

The overall transaction price as of the inception of the contract was determined to be $75.0 million, which is composed of the upfront payment of $50.0 million and the First Anniversary Payment of $25.0 million. The Company will re-evaluate the transaction price in each reporting period. The $25.0 million for the First Anniversary Payment represents both a contract asset and a contract liability and the Company has presented these amounts net in accordance with ASC 606 guidance for contract assets and liabilities.

The Company concluded that each of the six base editing programs combined with the research and development service, licenses, substitution rights and governance participation were material promises that were both capable of being distinct and were distinct within the context of the Apellis Agreement and represented separate performance obligations. Therefore, the Company did 0t recognize any upfront revenue related to the license. The Company further concluded that the Opt-In Rights and option to extend the collaboration term did not grant Apellis a material right. The Company determined that the term of the contract is five years, as this is the period during which both parties have enforceable rights.

The selling price of each performance obligation was determined based on the Company’s estimated standalone selling price, or the ESSP. The Company developed the ESSP for all of the performance obligations included in the Apellis Agreement with the objective

17


of determining the price at which it would sell such an item if it were to be sold regularly on a standalone basis. The Company allocated the stand alonestand-alone selling price to the performance obligations based on the relative standalone selling price method.

The Company recognizes revenue for each performance obligation as it is satisfied over the five-year term using an input method. The Company allocated the transaction price of $75.0 million to each of the six performance obligations, which includes each of the six base editing programs combined with the research and development service, licenses, substitution rights and governance participation, and is being recognized using an input method based on the actual costs incurred as a percentage of total budgeted costs towards satisfying the performance obligation as this method provides the most faithful depiction of the entity’s performance in transferring control of the goods and services promised to Apellis and represents the Company’s best estimate of the period of the obligation. For the three and nine months ended September 30, 2021,March 31, 2022, the Company recognized $0.82.1 million of revenue related to the Apellis Agreement. As of September 30, 2021,March 31, 2022, there is $12.819.4 million and $36.426.7 million of current and non-currentlong-term deferred revenue, liability, respectively, related to the Apellis Agreement.

Prime Medicine

In September 2019, the Company entered into a collaboration and license agreement, or the Prime Agreement, with Prime Medicine, Inc., or Prime, Medicine, to research and develop a novel gene editing technology developed by one of the Company’s founders.

Under the terms of the agreement, the Company granted Prime Medicine a non-exclusive license to certain of its clustered regulatory interspaced short palindromic repeats, or CRISPR technology (including Cas12b), delivery technology and certain other technology controlled by the Company to develop and commercialize gene editing products for the treatment of human diseases. ThePrime Medicine granted the Company is required to use commercially reasonable effortsan exclusive license to develop new product candidates usingand commercialize prime gene editing technology for the intellectual property licensed from Prime Medicine.creation or modification of any single base transition mutations, as well as any edits made for the treatment of sickle cell disease. Additionally, each party granted to the other party certain exclusive and non-exclusive licenses to certain technology developed after the effective date of the agreement and controlled by the granting party or jointly owned by the parties. Each party has an obligation to assign rights in certain technology developed under the collaboration to the other party.

The Company had an obligation to issue $5.0 million in shares of its common stock to Prime Medicine, and Prime Medicine had an obligation to issue 5,000,000 shares of its common stock to the Company, should the Company elect to extend the collaboration beyond one year. In September 2020, the Company elected to continue the collaboration and, in October 2020, issued 200,307 shares of its common stock to Prime Medicine. The Company recognized $5.0 million as research and development expense for the three and nine months ended September 30, 2020 as a result of its decision to extend its collaboration with Prime Medicine. Additionally, in October 2020, the Company received 5,000,000 shares of Prime Medicine’s common stock and recognized $0.1 million as an offset to research and development expense for the year ended December 31, 2020.

Additionally, the Company provided immaterial interim management and startup services to Prime Medicine through March 2021.2021 but did not provide any services during 2022.

As of September 30, 2021,March 31, 2022, the Company determined that future milestones and royalties under the Prime Agreement were not probable of recognition.

17


Verve

In April 2019, the Company entered into a collaboration and license agreement with Verve, or the Verve Agreement, to investigate gene editing strategies to modify genes associated with an increased risk of coronary diseases. Under the terms of the Verve Agreement, the Company granted Verve an exclusive license to certain base editor technology and certain delivery technology, and improvements and Verve granted Beam a non-exclusive license under certain know-how and patents controlled by Verve, an interest in joint collaboration technology and an exclusive license (except as to Verve) under certain delivery technology.

As of September 30, 2021,March 31, 2022, the Company determined that milestones and royalties under the Verve Agreement were not probable of recognition.

11. Preferred and commonCommon stock

In January 2020, the Company authorized the designation of 25,000,000 shares of preferred stock and increased its authorized common stock to 250,000,000 shares, each with a par value of $0.01 per share.

In February 2020, the Company completed its IPO in which the Company issued and sold 12,176,471 shares of its common stock, including 1,588,235 shares pursuant to the full exercise of the underwriters’ option to purchase additional shares, at a public offering price of $17.00 per share, for aggregate gross proceeds of $207.0 million. The Company received approximately $188.3 million in net proceeds after deducting underwriting discounts and offering expenses payable by the Company. In connection with the IPO, all outstanding shares of the Company’s redeemable convertible preferred stock converted into 29,127,523 shares of the Company’s common stock.

In October 2020, the Company issued and sold 5,750,000 shares of its common stock, including 750,000 shares pursuant to the full exercise of the underwriters’ option to purchase additional shares, at a public offering price of $23.50 per share, for aggregate gross proceeds of $135.1 million. The Company received approximately $126.6 million in net proceeds after deducting underwriting discounts and offering expenses payable by the Company.

In October 2020, due to its election to continue the Prime Agreement, the Company issued 200,307 shares of its common stock to Prime Medicine.

18


In January 2021, the Company issued and sold 2,795,700 shares of its common stock in a private placement at an offering price of $93.00 per share for aggregate gross proceeds of $260.0 million. The Company received $252.0 million in net proceeds after deducting fees to the placement agents and offering expenses payable by the Company.

In April 2021, the Company entered into the Sales Agreement, with Jefferies, pursuant to which the Company was entitled to offer and sell, from time to time at prevailing market prices, shares of the Company’s common stock having aggregate gross proceeds of up to $300.0 million. The Company agreed to pay Jefferies a commission of up to 3.0% of the aggregate gross sale proceeds of any shares sold by Jefferies under the Sales Agreement. As of September 30,Between April and July 2021, the Company has sold 2,908,009 shares of its common stock under the Sales Agreement at an average price of $103.16 per share for aggregate gross proceeds of $300.0 million, before deducting commissions and offering expenses payable by the Company.

In July 2021, the Company and Jefferies entered into an amendment to the Sales Agreement to provide for an increase in the aggregate offering amount under the Sales Agreement, such that as of July 7, 2021, the Company may offer and sell shares of common stock having an aggregate offering price of an additional $500.0 million. As of September 30, 2021,March 31, 2022, the Company has sold 1,765,8332,873,956 additional shares of its common stock under the amended Sales Agreement at an average price of $107.8892.71 per share for aggregate gross proceeds of $190.5266.5 million, before deducting commissions and offering expenses payable by the Company, resulting in an aggregate of $490.5 million in gross proceeds received under the Sales Agreement as of September 30, 2021.

In May 2021, the first success payment measurements under each of the Harvard License Agreement and Broad Institute License Agreement occurred and success payments to Harvard and Broad Institute were calculated to be $15.0 million and $15.0 million, respectively. The Company elected to make each payment in shares of the Company’s common stock and issued 174,825 shares of the Company’s common stock to each of Harvard and Broad Institute to settle these liabilities in June 2021.Company.

The holders of the Company’s common stock are entitled to one vote for each share of common stock. Subject to the payment in full of all preferential dividends to which the holders of the Company’s preferred stock are entitled, the holders of the Company’s common stock shall be entitled to receive ratably dividends out of funds legally available. In the event of any voluntary or involuntary liquidation, dissolution, or winding up of the Company, after the payment or provision for payment of all debts and liabilities of the Company and all preferential amounts to which the holders of Company’s preferred stock are entitled with respect to the distribution of assets in liquidation, the holders of common stock shall be entitled to share ratably in the remaining assets of the Company available for distribution.

12. Stock option and grant plan

Stock2017 stock option and grant plan

TheIn June 2017, the Company’s board of directors adopted the Beam Therapeutics Inc. 2017 Stock Option and Grant Plan, adopted byor the Company’s board of directors in June 2017 and amended in each of February 2019 and May 2019Plan, which provided for the grant of qualified incentive stock options and nonqualified stock options, restricted stock awards, restricted stock units, or other awards to the Company’s employees, officers, directors, advisors, and outside consultants for the issuance or purchase of shares of the Company’s common stock. In May 2019, the 2017 Plan was amended to provide up to 8,078,681 shares of common stock for the issuance of stock options and restricted stock.

2019 equity incentive plan

In October 2019, the Company’s board of directors adopted the Beam Therapeutics Inc. 2019 Equity Incentive Plan, or the 2019 Plan, and, subsequent tofollowing the IPO, all equity-based awards arehave been granted under the 2019 Plan. The 2019 Plan provides for the grant of qualified and nonqualified stock options, stock appreciation rights, restricted and unrestricted stock and stock units, performance awards, and other share-based awards to the Company’s employees, officers, directors, advisors, and outside consultants.

The maximum number of shares of the Company’s common stock that may be issued under the 2019 Plan was initially 3,700,000 shares, or the Share Pool, plus the number of shares of the Company’s common stock underlying awards under the 2017 Plan, not to exceed 5,639,818 shares, that become available again for grant under the 2017 Plan in accordance with its terms. The Share Pool will automatically increase on January 1st of each year from 2021 to 2029 by the lesser of (i) 4 percent of the number of shares of the Company’s common stock outstanding as of the close of business on the immediately preceding December 31st and (ii) the number of shares determined by the Company’s board of directors on or prior to such date for such year.

As of September 30, 2021,March 31, 2022, the Company had 9,019,80511,226,843 shares reserved including 1,985,9502,452,727 shares available for future issuance pursuant to the 2019 Plan.

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Stock-based compensation expense recorded as research and development and general and administrative expenses in the condensed consolidated statements of operations and other comprehensive loss is as follows (in thousands):

 

 

Three Months Ended
September 30,

 

 

Nine Months Ended
September 30,

 

 

 

2021

 

 

2020

 

 

2021

 

 

2020

 

Research and development

 

$

7,554

 

 

$

1,999

 

 

$

17,049

 

 

$

5,577

 

General and administrative

 

 

5,414

 

 

 

1,013

 

 

 

11,019

 

 

 

2,996

 

Total stock-based compensation expense

 

$

12,968

 

 

$

3,012

 

 

$

28,068

 

 

$

8,573

 

19


 

 

Three Months Ended
March 31,

 

 

 

2022

 

 

2021

 

Research and development

 

$

11,294

 

 

$

3,155

 

General and administrative

 

 

6,741

 

 

 

1,493

 

Total stock-based compensation expense

 

$

18,035

 

 

$

4,648

 

Stock options

The following table provides a summary of option activity under the Company’s equity award plans:plans:

 

Number
of options

 

 

Weighted
average
exercise
price

 

 

Number
of options

 

 

Weighted
average
exercise
price

 

Outstanding at December 31, 2020

 

5,336,441

 

$

9.70

 

Outstanding at December 31, 2021

 

6,034,192

 

$

32.40

 

Granted

 

1,638,143

 

88.57

 

 

1,427,500

 

69.13

 

Exercised

 

(774,352

)

 

9.89

 

 

(176,652

)

 

4.64

 

Forfeitures

 

 

(60,982

)

 

 

28.53

 

 

 

(20,637

)

 

36.41

 

Outstanding at September 30, 2021

 

 

6,139,250

 

 

 

30.53

 

Exercisable as of September 30, 2021

 

 

2,008,588

 

 

$

9.57

 

Outstanding at March 31, 2022

 

 

7,264,403

 

 

40.28

 

Exercisable as of March 31, 2022

 

 

2,602,709

 

 

$

15.83

 

The weighted-average grant date fair value per share of options granted in the ninethree months ended September 30, 2021March 31, 2022 was $58.8546.09. As of September 30, 2021,March 31, 2022, there was $107.3158.4 million of unrecognized compensation expense related to unvested stock options, which is expected to be recognized over a weighted-average period of approximately 2.42.6 years.

Restricted stock

The Company issues shares of restricted common stock, including both restricted stock units and restricted stock awards. Restricted common stock issued generally vests over a period of two to four years.

The following table summarizes the Company’s restricted stock activity:

 

 

Shares

 

 

Weighted-
average grant
date fair
value

 

Unvested as of December 31, 2020

 

 

1,275,338

 

 

$

10.95

 

Issued

 

 

819,830

 

 

 

88.13

 

Vested

 

 

(799,609

)

 

 

4.61

 

Cancelled

 

 

(8,725

)

 

 

80.04

 

Unvested as of September 30, 2021

 

 

1,286,834

 

 

$

63.59

 

 

 

Shares

 

 

Weighted-
average grant
date fair
value

 

Unvested as of December 31, 2021

 

 

1,126,206

 

 

$

74.32

 

Issued

 

 

711,025

 

 

 

57.30

 

Vested

 

 

(283,186

)

 

 

38.69

 

Forfeited

 

 

(11,519

)

 

 

86.15

 

Unvested as of March 31, 2022

 

 

1,542,526

 

 

$

72.93

 

At September 30, 2021,March 31, 2022, there was approximately $74.5106.9 million of unrecognized stock-based compensation expense related to restricted stock that is expected to vest. These costs are expected to be recognized over a weighted-average remaining vesting period of approximately 2.73.5 years.

2019 employee stock purchase plan

In February 2020, the Company’s board of directors adopted the Beam Therapeutics Inc. 2019 Employee Stock Purchase Plan, or ESPP, which was approved by the Company’s stockholders. Pursuant to the ESPP, certain employees of the Company, excluding consultants and non-employee directors, are eligible to purchase common stock of the Company at a reduced rate during offering periods. The ESPP permits participants to purchase common stock using funds contributed through payroll deductions, subject to a calendar year limit of $25,000 and at a purchase price of 85% of the lower of the fair market value of the Company’s common stock on the first trading day of the offering period or on the applicable purchase date, which will be the final trading day of the applicable purchase period. The first offering period commenced on October 1, 2021. The Company issued 28,990 shares under the ESPP during the three months ended March 31, 2022. There were 0 shares issued under the ESPP during the three months ended March 31, 2021. As of September 30, 2021,March 31, 2022, the Company had 1,049,4601,706,282 shares available for issuance under the ESPP.

The Company uses the straight-line attribution approach to record the expense over the offering period. Stock-based compensation for the ESPP for the three months ended March 31, 2022 was $0.3 million. There was 0 stock-based compensation expense recorded under the ESPP for the three months ended March 31, 2021.

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13. Net loss per share attributable to common stockholders

As noted above, for periods in which the Company reports a net loss, attributable to common stockholders, potentially dilutive securities have been excluded from the computation of diluted net loss per share as their effects would be anti-dilutive. Therefore, the weighted average number of common shares outstanding used to calculate both basic and diluted net loss per share attributable to common stockholders is the same. The Company excluded the following potential common shares, presented based on amounts outstanding at period end, from the computation of diluted net loss per share attributable to common stockholders because including them would have had an anti-dilutive effect:

 

 

As of September 30,

 

 

 

2021

 

 

2020

 

Unvested restricted stock

 

 

1,286,834

 

 

 

1,492,203

 

Outstanding options to purchase common stock

 

 

6,139,250

 

 

 

5,633,485

 

Total

 

 

7,426,084

 

 

 

7,125,688

 

20


 

 

As of March 31,

 

 

 

2022

 

 

2021

 

Unvested restricted stock

 

 

1,542,526

 

 

 

1,388,114

 

Outstanding options to purchase common stock

 

 

7,264,403

 

 

 

6,108,126

 

Total

 

 

8,806,929

 

 

 

7,496,240

 

The following table summarizes the computation of basic and diluted net loss per share attributable to common stockholders of the Company (in thousands, except share and per share amounts):

 

 

Three Months Ended September 30,

 

 

Nine Months Ended September 30,

 

 

 

2021

 

 

2020

 

 

2021

 

 

2020

 

Numerator:

 

 

 

 

 

 

 

 

 

 

 

 

Net loss attributable to common stockholders

 

$

(28,118

)

 

$

(34,452

)

 

$

(305,931

)

 

$

(100,405

)

Denominator:

 

 

 

 

 

 

 

 

 

 

 

 

Weighted average number of common shares, basic and diluted

 

 

66,377,611

 

 

 

50,087,747

 

 

 

62,960,219

 

 

 

43,438,919

 

Net loss per common share attributable to common stockholders, basic and diluted

 

$

(0.42

)

 

$

(0.69

)

 

$

(4.86

)

 

$

(2.31

)

 

 

Three Months Ended March 31,

 

 

 

2022

 

 

2021

 

Numerator:

 

 

 

 

 

 

Net loss

 

$

(69,214

)

 

$

(201,560

)

Denominator:

 

 

 

 

 

 

Weighted average common shares outstanding, basic and diluted

 

 

68,703,864

 

 

 

60,210,120

 

Net loss per common share, basic and diluted

 

$

(1.01

)

 

$

(3.35

)

 

14. Income taxes

During the three and nine months ended September 30,March 31, 2022 and 2021, and 2020, the Company recorded a full valuation allowance on federal and state deferred tax assets since management does not forecast the Company to be in a taxable position in the near future.

15. Related party transactions

Founders

The Company made payments of $0.1 million and $0.30.1 million to its 3 founder shareholders for scientific consulting and other expenses for the three and nine months ended September 30,March 31, 2022 and 2021, respectively.

Verve

The Company and Verve are parties to the Verve Agreementa collaboration and license agreement and have a common board member.

Prior to Verve’s initial public offering in June 2021, the Company owned both common and preferred shares of Verve and valued such investment based on the cost of the equity securities adjusted for any observable market transactions. Following theVerve’s initial public offering, and the conversion of the preferred stock to common stock and a stock split, the equity securities have a readily determinable fair value; however, they are subject to transfer restrictions. Following the initial public offering,value and the Company owned 546,970 shares of Verve's common stock, the value of which is included in long-term investmentsmarketable securities in the condensed consolidated balance sheet. The Company recorded the investment at fair value as of September 30, 2021,March 31, 2022, which resulted in thea recognition of $4.9 million of other expense andof $22.07.7 million during the three months ended March 31, 2022 . The Company recorded other income of $1.0 million of other income for the three and nine months ended September 30,March 31, 2021 respectively.related to the changes in fair value of Verve's stock. The value of this investment as of September 30, 2021March 31, 2022 is $24.412.5 million.

During the nine months ended September 30, 2020, the Company purchased shares of Verve's series A preferred stock valued at $0.8 million and recognized gains of $0.5 million, which is recorded in other income, on its investment in Verve stock.

The Company purchased certain materials from Verve amounting to $0.2 million, which are recorded as research and development expenses within the accompanying condensed consolidated statements of operations and other comprehensive loss, for the ninethree months ended September 30,March 31, 2021. The Company purchased certaindid 0t purchase any materials from Verve amounting to $during the 0.3 million for the ninethree months ended September 30, 2020. The Company also sold certain materials to Verve amounting to $0.2 million for the nine months ended September 30, 2020.March 31, 2022.

In October 2021, the Company entered into an agreement pursuant to which Verve will subleasesubleased 12,000 square feet of Beam'sthe Company’s existing office and laboratory space for a term of one year beginningwhich began in December 2021. Verve is expected to pay approximately $1.4 million in rental payments over the term of the sublease, as well as its proportionate costs for the landlord’s operating expense, insurance, property taxes, and utilities. The Company recorded $0.3 million of sublease income related to this sublease within the accompanying consolidated statements of operations and other comprehensive loss for the three months ended March 31, 2022.

Prime Medicine

The Company and Prime Medicine are parties to the Prime Agreement and have a common founder and a common board member.

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Additionally, in September 2019, in connection with the Prime Agreement, the Company executed a letter agreement, as amended, to provide certain interim management and startup services to Prime Medicine through March 2021. Prime Medicine was obligated to reimburse the Company’s out-of-pocket costs incurred in connection with performing these services and, beginning in October 2020 and ending in March 2021, paid the Company a $30,00030.0 thousand monthly service fee. ForThere were no interim management and startup services provided to Prime Medicine during the ninethree months ended September 30, 2021, the Company recognized $0.1 million for performing such services in interest and other income (expense), net, within the accompanying consolidated statements of operations and other comprehensive loss.March 31, 2022.

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16. Subsequent events

Sana Biotechnology

In October 2021, the Company entered into an option and license agreement with Sana Biotechnology, Inc., or Sana, to provide Sana with non-exclusive commercial rights to the Company’s CRISPR Cas12b nuclease system for certain ex vivo engineered cell therapy programs. The agreement excludes any rights to base editing using the Company’s CRISPR Cas12b system, which commercial rights remain with the Company. Under the terms of the agreement, Sana agreed to pay the Company an upfront payment of $50.0 million. The Company received this payment in October 2021. The Company is also eligible to receive certain target option exercise fees, certain milestone payments upon the achievement of certain development and commercial sale milestones, and certain royalties on net sales of royalty-bearing products by Sana.

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Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

The following discussion and analysis of our financial condition and results of operations should be read in conjunction with our condensed consolidated financial statements and the related notes to those statements included elsewhere in this Quarterly Report on Form 10-Q. In addition to historical financial information, the following discussion and analysis contains forward-looking statements that involve important risks, uncertainties and assumptions. Some of the numbers included herein have been rounded for the convenience of presentation. Our actual results may differ materially from those anticipated in these forward-looking statements as a result of many factors, including those discussed in “Risk Factors” in Part II, Item 1A. and elsewhere in this Quarterly Report on Form 10-Q, and in our Quarterly Reports on Form 10-Q for the quarters ended March 31, 2021, and June 30, 2021, and in the “Risk Factors Summary” and “Item 1A. Risk Factors” section of our Annual Report on Form 10-K for the fiscal year ended December 31, 2020,2021, or the 20202021 Form 10-K.

Overview

We are a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. Our vision is to provide life-long cures to patients suffering from serious diseases. To achieve this vision, we have assembled a platform that includes a suite of gene editing and delivery technologies and are in the process of developing internal manufacturing capabilities.

Our suite of gene editing technologies is anchored by our proprietary base editing technology, which potentially enables an entirely newa differentiated class of precision genetic medicines that target a single base in the genome without making a double-stranded break in the DNA. This approach uses a chemical reaction designed to create precise, predictable and efficient genetic outcomes at the targeted sequence. Our novelproprietary base editors have two principal components: (i) a clustered regulatoryregularly interspaced short palindromic repeats, or CRISPR, protein, bound to a guide RNA, that leverages the established DNA-targeting ability of CRISPR, but is modified to not cause a double-stranded break, and (ii) a base editing enzyme, such as a deaminase, which carries out the desired chemical modification of the target DNA base. We believe this design contributes to a more precise and efficient edit compared to traditional gene editing methods, which operate by creating targeted double-stranded breaks in the DNA; these breaksDNA that can result in unwanted DNA modifications. We believe that the precision of our editors will dramatically increase the impact of gene editing for a broad range of therapeutic applications.

To unlock the full potential of our base editing technology across a wide range of therapeutic applications, we are pursuing a broad suite of both clinically validated and novel delivery modalities. For a givenmodalities, depending on tissue type, we use the delivery modality with the most compelling biodistribution. Our current programs utilize three different delivery modalities:including: (1) electroporation for efficient delivery to blood cells and immune cells ex vivo;vivo; (2) lipid nanoparticles, or LNPs, for non-viral in vivo delivery to the liver and potentially other organs in the future; and (3) adeno-associated viral vectors, or AAV, for in vivo viral delivery to the eye.eye and potentially other organs.

The elegance of the base editing approach combined with a tissue specific delivery modality provides the basis for a targeted efficient, precise, and highly versatile gene editing system, capable of gene correction, gene modification, gene silencing or gene activation, and/or multiplex editing of several genes simultaneously. We are currently advancing a broad, diversified portfolio of base editing programs against distinct editing targets, utilizing the full range of our development capabilities. We believe

Furthermore, in addition to our portfolio, we are also pursuing an innovative, platform-based business model with the flexibilitygoal of further expanding our access to new technologies in genetic medicine and versatilityincreasing the reach of our base editorsprograms to more patients. Overall, we are seeking to build the leading integrated platform for precision genetic medicine, which may lead tohave broad therapeutic applicability and transformationalthe potential forto transform the field of precision genetic medicines.

We believe that building an integrated platform combining our gene editing capabilities with advanced delivery and manufacturing capabilities will give us the maximum flexibility to develop our own sustainable portfolio and to create a hub for partnering with other companies to unlock the full potential of precision genetic medicine across all possible applications.Ex Vivo

Hematology:HSCs: Sickle cell disease and beta-thalassemia

We are advancing ex vivo base editing programs in which hematopoietic stem cells, or HSCs are collected from a patient, edited using electroporation, a clinically validated technology for the delivery of therapeutic constructs into harvested cells. These cells are infused back into the patient following a myeloablative conditioning regimen, such as treatment with busulfan, the standard of care in HSC transplantation today. Once reinfused, the HSCs begin repopulating a portion of the bone marrow in a process known as engraftment. The engrafted, edited HSCs give rise to progenitor cell types with the corrected gene sequences.

We are pursuing a long-term, staged development strategy for our base editing approach to treat sickle cell disease that consists of advancing our ex vivo programs, BEAM-101 and BEAM-102, in Wave 1, improving patient conditioning regimens in Wave 2, and enabling in vivo base editing with delivery directly into HSCs of patients via LNPs in Wave 3. We believe this suite of technologies – base editing, improved conditioning and in vivo delivery for editing HSCs – can maximize the potential applicability of our sickle cell disease programs to patients as well as create a platform for the treatment of many other severe genetic blood disorders

Wave 1: Ex Vivo Base Editing via Autologous Transplant with BEAM-101 and BEAM-102

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We are using base editing to pursue the development of two complementary approaches to treating sickle cell disease, a severe inherited blood disease caused by a single point mutation, E6V, in the beta globin gene (BEAM-101 and BEAM-102), and one approach to treat beta-thalassemia, another inherited blood disorder characterized by severe anemia caused by reduced production of functional hemoglobin due to insufficient expression of the beta globin protein (BEAM-101).

BEAM-101: Recreating naturally-occurring protective mutations to activate fetal hemoglobin

In November 2021, we announced that our Investigational New Drug, or IND, application for BEAM-101 for the treatment of sickle cell disease was cleared by the U.S. Food and Drug Administration, or FDA. BEAM-101 is an investigational, a patient-specific, autologous hematopoietic investigational cell therapy which wasis designed to incorporate ex vivo base edits that mimic single nucleotide polymorphisms seen in individuals with hereditary persistence of fetal hemoglobin, or HPFH, to potentially alleviate the effects of mutations causing sickle cell disease or beta thalassemia. WeOur Investigational New Drug, or IND, application for BEAM-101 for the treatment of sickle cell disease has been cleared by the U.S. Food and Drug Administration, or FDA, and we are preparing to initiate a Phase 1/2 clinical trial designed to assess the safety and efficacy of BEAM-101 for the treatment of sickle cell disease, which we refer to as our BEACON-101 trial. The BEACON-101 trial is expected to include an initial “sentinel” cohort of three patients, treated one at a time to confirm successful engraftment, followed by dosing in up to a total of 45 patients. The clinical trial is designed to initially include patients between ages 18 and 35 with sickle cell disease who have received prior treatment with at least one disease-modifying agent with inadequate response or intolerance. Following mobilization, conditioning and HSC transplant with BEAM-101, patients will be assessed for safety and tolerability, with safety endpoints including the proportion of patients with successful neutrophil engraftment by day 42. Patients will also be assessed for efficacy, with efficacy endpoints including the change from baseline in severe vaso-occlusive events, transfusion requirements, hemoglobin F levels, and quality of life and ability to function. We have begun site selection and the institutional review board approval processes for the BEACON-101 trial and plan to enroll the first subject in the second half of 2022.

We have achieved proof-of-concept in vivo with long-term engraftment of base edited human CD34 cells in mice foradministered BEAM-101. Persistence of engraftment and high levels of editing have been confirmed in several preclinical studies, including in studies using material generated at a clinically relevant scale.

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BEAM-102: Direct correction of the sickle cell mutation

Our second ex vivo base editing approach that we are developing for sickle cell disease, BEAM-102, is a direct correction ofdesigned to directly correct the causative sickle mutation at position 6 of the beta globin gene. This approach has been enabled by our inlaid base editors, or IBEs, which are architectural variants of base editors that have attributes of enhanced specificity and altered activity windows relative to foundational base editors. Our IBEs expand the breadth of potential base editing targets by extending the range of editing windows that can be created for any given CRISPR protein used to target the DNA. By inserting the deaminase into the CRISPR protein at various strategic positions, thereby repositioning the deaminase’s editing window, IBEs enable editing outside the traditional editing window. BEAM-102 directly corrects the causative mutation in sickle cell disease by recreating a naturally-occurring normal human hemoglobin variant, HbG-Makassar. By making a single A-to-G edit, we have demonstrated in primary human CD34+ cells isolated from sickle cell disease patients the ability to create the naturally occurring MakassarHbG or “Makassar” variant of hemoglobin. This variant, which was identified in humans and first published in 1970, has the same function as the wild-type variant and does not cause sickle cell disease. Distinct from other approaches, cells that are successfully edited in this way are fully corrected, no longer containing the sickle protein. We have initiated IND-enabling studies for BEAM-102 and expect to submit an IND to the FDA for the treatment of sickle cell disease during the second half of 2022.

During the second quarter of 2020, we published preclinical data on BEAM-102 demonstrating that our adenine base editors, or ABEs, can efficiently convert the causative Hemoglobin S, or HbS, point mutation, to HbG-Makassar, with high efficiency (more than 80%). In this preclinical study, the Makassar variant does not cause hemoglobin to polymerize and red blood cells to sickle and, therefore, edited cells are cured through elimination of the disease-causing protein. The resultsIn December 2021, we presented data from preclinical studies further characterizing the Makassar hemoglobin created by BEAM-102 and demonstrating biophysical and biochemical properties consistent with normal hemoglobin.

Wave 2: Improved Conditioning

In parallel with Wave 1 development, we also aim to improve the transplant conditioning regimen for sickle cell disease patients undergoing HSCT, reducing toxicity challenges associated with HSCT standard of care. Conditioning is a critical component necessary to prepare a patient’s body to receive the ex vivo edited cells that must engraft in the patient’s bone marrow in order to be effective. Today’s conditioning regimens rely on nonspecific chemotherapy or radiation, which are associated with significant toxicities. We are collaborating with Magenta Therapeutics, Inc., or Magenta, to evaluate the potential utility of MGTA-117, Magenta’s novel antibody drug conjugate, in combination with BEAM-101 and BEAM-102, as well as other base editing programs in hematology. MGTA-117 is designed to spare immune cells and precisely target hematopoietic stem and progenitor cells, or HSPCs, and has demonstrated high selectivity, potent efficacy, wide safety margins and broad tolerability in non-human primate, or NHP, models. We are also conducting our own research into novel conditioning strategies. Improved conditioning regimens could potentially be paired with BEAM-101 and BEAM-102, as well as other base editing programs in hematology.

Wave 3: In Vivo Base Editing via HSC-targeted LNPs

We are also exploring the potential for in vivo base editing programs for sickle cell disease, in which base editors would be delivered to the patient through an infusion of LNPs targeted to HSCs, eliminating the need for transplantation altogether. This approach could provide a more accessible option for patients, particularly in regions where ex vivo treatment is challenging. Building on our acquisition of Guide, we are using our proprietary DNA-barcoded LNP screening technology to enable high-throughput in vivo identification of LNPs with novel biodistribution and selectivity for target organs beyond the liver. In December 2021, we announced

23


we had screened more than 1,000 LNPs using this study confirmedtechnology for potential to deliver to HSCs and had identified LNP-HSC1 as the abilitymost potent, with efficient transfection in both mice and NHPs.

Achieving Understanding of the Makassar variantNatural History of Sickle Trait (AUNT) Study

In May 2022, we announced the initiation of a sickle cell trait, or SCT, focused natural history study. Carriers of sickle cell disease, or those with SCT, have only one copy of the hemoglobin gene, have HbS levels between 25-45%, and are thought to protecthave a benign condition. However, despite SCT impacting approximately 300 million people around the world, the key hematologic and clinical phenotypic characteristics and functional impacts from having SCT have been understudied in a prospective manner. As part of a long-term lifecycle strategy for our sickle cell disease programs, we, in collaboration with the National Alliance of Sickle Cell Centers, the University of Alabama, and Johns Hopkins Medical Center, have initiated the AUNT (Achieving Understanding of the Natural History of Sickle Trait) Study.

The AUNT Study is designed to establish an understanding of the hematologic and clinical phenotype of people with SCT, including blood rheology, potential complications and genetic modifiers, in an effort to better understand the hematologic phenotype that is associated with good health and lack of organ dysfunction. The study is designed to enroll approximately 1,000 participants with SCT in the United States who have been identified as family members of participants in the Global Research Network for Data and Discovery, a multi-institutional prospective registry comprising clinical and background data from more than 1,200 adult and pediatric individuals with sickle cell disease from 1999-2021.

Ex vivo T cell therapies

The starting material for our multiplex-edited allogeneic CAR-T cell products is white blood cells from sickling, even ina healthy donor, which are collected using a standard blood bank procedure known as leukapheresis. Using a single electroporation, we introduce the context of mono-allelic editing (with one sickle allelebase editor as mRNA, and one corrected allele). In November 2021, we announced that we initiated IND-enabling studies for BEAM-102.the guides encoding the target sequences. The edited cells are subsequently transduced with a lentivirus expressing the CAR. Once the T cells have been engineered, they are expanded and frozen. After the patient is lymphodepleted, the multiplex-edited, allogenic cell product is infused.

Oncology:We believe base editing is a powerful tool to simultaneously multiplex edit many genes without the unintended on-target effects that can result from simultaneous editing with nucleases through the creation of double-stranded breaks. The ability to create a large number of multiplex edits in T cells could endow CAR-T cells and other cell therapies with combinations of features that have the potential to dramatically enhance their therapeutic potential in treating hematological or solid tumors.

The initial indications that we plan to target with our chimeric antigen receptor T-cell, or CAR-T, product candidates are relapsed, refractory T-cell acute lymphoblastic leukemia /T cell lymphoblastic lymphoma, or T-ALL,T-ALL/T-LL, a severe disease affecting children and adults, and Acute Myeloid Leukemia, or AML. We believe that our approach has the potential to produce higher response rates and deeper remissions than existing approaches. Our proof-of-concept pre-clinical experimentspreclinical studies have demonstrated the ability of base editors to efficiently modify up to 8eight genomic loci simultaneously in primary human T cells with efficiencies ranging from 85-95% as measured by flow cytometry of target protein knockdown. Importantly, these results were achieved without the generation of observed chromosomal rearrangements, as detectedevaluated by sensitive methods such as UDiTaSTMor G-banded Karyotyping and with no observed loss of cell viability from editing. OurThe proof-of-concept experimentspreclinical studies have also demonstrated robust T-cellT cell killing of target tumor cells both in vitro and in vivo. We plan to nominate a second CAR-T development candidate, in addition to BEAM-201, in 2022.

BEAM-201: Universal CD7-targeting CAR-T cells

BEAM-201 is our potent and specific anti-CD7, multiplex edited, allogeneic CAR-Ta development candidate for the treatmentcomprised of relapsed/refractory T-ALL. BEAM-201 is produced using a Good Manufacturing Practice, or cGMP, compliant, clinical-scale process in which T-cellsT cells derived from healthy donors that are simultaneously base edited at four genomic loci,TRAC, CD7, CD52 and PDCD1 and then transduced with a lentivirus codingencoding for an anti-CD7 CAR. The resulting cells are universally-compatible, allogeneic (“off the shelf”) CD7-targetingCAR that is designed to create allogenic CD7 targeting CAR-T cells, resistant to both fratricide and immunosuppression. To our knowledge, BEAM-201 is the first investigational cell therapy featuring four simultaneous edits. In August 2021, we announced that we hadWe have initiated IND-enabling studies for BEAM-201.BEAM-201 and expect to submit an IND to the FDA for the treatment of relapsed, refractory T-ALL/T-LL and potentially other CD7+ malignancies during the second half of 2022.

CD5-targeting CAR-T cells

In October 2021, we announced preclinical data from our multiplex edited allogeneic CAR TCAR-T research program targeting CD5-positive hematologic malignancies which we intend to present later this year. Thismalignancies. These data demonstrated knockout of CD5 expression to be a general mechanism to enhance potency and potentially improve durability of highly multiplexed CAR TCAR-T cells.

In vivo LNP

LNPs are a clinically validated technology for delivery of nucleic acid payloads to the liver. LNPs are multi-component particles that encapsulate the base editor mRNA and one or more guides and protect them from degradation while in an external environment, enabling the transient delivery of the base editor in vivo. Multiple third-party clinical trials have demonstrated the effective delivery of silencing RNA to the liver using LNPs. Because only one dose of a base editing therapy may be needed in a course of treatment, LNPs are a suitable delivery modality that we believe is unlikely to face the complications seen with chronic use of LNPs, such as those

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observed when delivering oligonucleotides or mRNA for gene therapy. All of the components of the LNP, as well as the mRNA encoding the base editor, are well-defined and can be manufactured synthetically, providing the opportunity for scalable manufacturing.

We have developed several proprietary LNP formulations. In May 2021, we announced initial data from our evaluation of various LNP formulations and mRNA production processes using an mRNA-encoding ABE and guide RNA to target the ALAS1 gene, a surrogate payload for genetic liver diseases. These data showed improved in vivo editing in the livers of NHPs from less than 10% initially to 52% at a total RNA dose of 1.5 mg/kg. Continued optimization of our LNP formulations has demonstrated further increases in liver editing potency in NHPs. In September 2021, we presented data demonstrating up to 60% editing in NHPs at a total RNA dose of 1.0 mg/kg. Data from our preclinical studies demonstrated that these formulations were well tolerated by NHPs treated with doses up to 1.5 mg/kg. Minimal to mild and transient liver enzyme elevations were observed and resolved by day 15 post-treatment. Additionally, the formulations showed promising interim stability, maintaining potency after three months at -20⁰C and -80⁰C.

We are currently using LNP formulations to advance our programs for genetic liver diseases, including Glycogen Storage Disease Type Ia, or GSDIa, also known as Von Gierke disease, and Alpha-1 Antitrypsin Deficiency, or Alpha-1, and chronic hepatitis B infection. In December 2021, we nominated BEAM-301, a liver-targeting LNP formulation of base editing reagents designed to correct the R83C mutation, the most common disease-causing mutation of GSDIa, as our first in vivo development candidate. We anticipate initiating IND-enabling studies for BEAM-301 and nominating a second liver-targeted development candidate in 2022.

Liver diseases: Alpha-1 antitrypsin deficiency, glycogen storage disorder 1a, alpha-1 antitrypsin deficiency, and chronic hepatitis B infection

GSDIa

GSDIa is an inborn disorder of glucose metabolism caused by mutations in the G6PC gene, which results in low blood glucose levels that can be fatal if patients do not adhere to a strict regimen of slow-release forms of glucose, administered every one to four hours (including overnight). There are no disease modifying therapies available for patients with GSDIa.

Our approach to treating patients with GSDIa is to apply base editing via LNP delivery to repair the two most prevalent mutations that cause the disease, R83C and Q347X. It is estimated that these two point mutations account for 900 and 500 patients, respectively, in the United States, representing approximately 59% of all GSDIa patients in the United States. Third party animal studies have shown that as little as 11% of normal G6Pase activity in liver cells is sufficient to restore fasting glucose; however, this level must be maintained in order to preserve glucose control and alleviate other serious, and potentially fatal, GSDIa sequelae.

In October 2021, we reported data from preclinical studies that support the potential of base editing to durably correct disease-causing mutations of GSDIa. We are currently usingcreated a varietynovel, humanized R83C knockout mouse model (huR83C), mimicking the abnormal metabolic phenotype of cationic lipidshuman GSDIa, and collaborated with the National Institutes of Health, or NIH, to characterize the phenotype of these animals. The results demonstrated that newborn huR83C mice treated with our LNP-delivered ABE exhibited normal growth to the end of the study at three weeks of age without any hypoglycemia-induced seizures. In contrast, homozygous animals were unable to survive soon after birth in the absence of glucose supplementation. In addition, we observed editing efficiencies up to approximately 60% by next-generation sequencing of DNA isolated from various sourcesthe whole liver.

In May 2022, an abstract announcing new preclinical data to advance our programs for liver diseases,be presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting was published. The data, which include Alpha-1 Antitrypsin Deficiency, or Alpha-1, Glycogen Storage Disease Type IA, orbuild on previously released preclinical results, demonstrated that in a GSDIa (also knownmouse model, treated mice, which otherwise have poor survival outcomes if left untreated, grew normally to at least 35 weeks following administration of BEAM-301, with survival ongoing in the study. Notably, as Von Gierke disease)low as single digit percentage base-editing rates were sufficient to restore physiologically relevant levels of hepatic G6Pase activity, normalize serum metabolites and, chronic hepatitis B infection.most importantly, prevent hypoglycemia during a twenty-four hour fast in treated mice. In addition, preliminary assessments of observed off-target editing have suggested a favorable profile of BEAM-301.

Alpha-1

Alpha-1 is a severe inherited genetic disorder that can cause progressive lung and liver disease. The most severe form of Alpha-1 arises when a patient has a point mutation in both copies of the SERPINA1 gene at amino acid 342 position (E342K, also known as the PiZ mutation or the “Z” allele). With the high efficiency and precision of our base editors, we aim to utilize our ABEs to enable the programmable conversion of A-to-T and G-to-C base pairs and precisely correct the E342K point mutation back to the wild type sequence. In 2020, we showed the ability to directly correct the mutation causing Alpha-1, providing both in vitro and in vivo preclinical proof-of-concept for base editing to correct this disease.

GSDIa isIn May 2022, an inborn disorder of glucose metabolism caused by mutations inabstract to be presented at ASGCT detailed our efforts to optimize both the G6PC gene, which results in low blood glucose levels that can be fatal if patients do not adhereABE and the guide RNA used to correct the disease-causing PiZ mutation, with improvements over the original reagents leading to a strict regimen of slow-release forms of glucose, administered every onegreater than two-fold increase in observed editing potency and potentially therapeutically relevant increases in circulating alpha-1 antitrypsin in mice treated at doses that are expected to four hours (including overnight)be clinically relevant (<1mg/kg). Our approachFurther, similar results were observed in adult mice dosed at greater than 37 weeks, a treatment context more similar to treating patients with GSDIa is to apply base editing using lipid nanoparticle, or LNP, delivery to repair the two most prevalent mutations that cause the disease, R83C and Q347X. We have achieved editing levels what might be encountered in vivo, ina clinical setting.

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preclinical models, for the correction of the two most prevalent mutations causing GSDIa, which could be clinically relevant if reproduced in humans. Additionally, in October 2021, we presented new preclinical data demonstrating the ability of our liver-targeted base editing approach to directly correct R83C. To evaluate this approach, we created a novel humanized GSDIa R83C knockout mouse model in collaboration with the National Institutes of Health, or NIH, which mimicked the abnormal metabolic phenotype of human GSDIa. These data demonstrated that newborn huR38C mice treated with our LNP-delivered adenine base editors, or ABEs, exhibited normal growth to the end of the study at three weeks of age without any hypoglycemia-induced seizures. In contrast, homozygous animals were unable to survive soon after birth in the absence of glucose supplementation. In addition, we observed editing efficiencies up to approximately 60% by next generation sequencing of DNA isolated from the whole liver. Published studies suggest that a critical therapeutic threshold of approximately 11% of normal G6Pase activity in the liver is sufficient to mitigate fastinghypoglycemia in animal models of GSDIa. Based on these preclinical data and our LNP formulation progress discussed below, we expect to nominate our first development candidate for in vivo base editing in the liver using LNP delivery for the treatment of patients with GSDIa (R83C mutation) by the end of 2021.

Hepatitis B virus, or HBV, causes serious liver infection that can become chronic, increasing the risk of developing life-threatening health issues like cirrhosis, liver failure or liver cancer. Chronic HBV infection is characterized by the persistence of covalently closed circular DNA, or cccDNA, a unique DNA structure that forms in response to HBV infection in the nuclei of liver cells. Additionally, the HBV DNA can integrate into the human genome becoming a source of hepatitis B surface antigen, or HBsAg. While currently available treatments can manage HBV replication, they do not clear cccDNA from the infected liver cells. This inability to prevent HBV infection rebound from cccDNA is a key challenge to curing HBV. In September 2021, we presented preclinical data demonstratingthat demonstrated the potential of our cytosine base editors to reduce viral markers, including HBsAg expression, and prevent viral rebound of HBV in in vitro models.

An important next stepIn vivo AAV

AAV is a clinically validated technology that has been extensively used for gene delivery to a variety of tissues. AAV is a small, non-pathogenic virus that can be repurposed to carry a therapeutic payload, making it a suitable vector for delivery of gene editing therapies. Several clinical trials have been conducted or are in progress with different AAV variants for multiple diseases, including diseases of the eye, liver, muscle, lung and central nervous system. We have an option to in-license a variety of AAV variants that could be selected for optimal distribution to multiple organs. Because our liver disease programsDNA base editors are larger than the approximate 4.5kb packaging limit of AAV vectors, we use a novel split intein technology that is finalizing our LNP formulation, and we are making progress on developing a formulation using proof-of-concept targets. In May 2021, we announced initial data from our evaluation of various LNP formulations and mRNA production processes using an mRNA-encoding ABEdesigned to deliver the base editor and guide RNA to targetby co-infection with two viruses, where each virus contains approximately one half of the ALAS1 gene, a surrogate payload for genetic liver diseases. These data showed improved in vivo editing in the livers of non-human primates, or NHPs, from less than 10% initially to 52% at a total RNA dose of 1.5 mg/kg. Continued optimization of our LNP formulations has demonstrated further increases in liver editing potency in NHPs. In September 2021, we presented data demonstrating up to 60% editing at a total RNA dose of 1.0 mg/kg. Data from our studies demonstrated that these formulations were well tolerated by NHPs treated with doses up to 1.5 mg/kg. Minimal to mild and transient liver enzyme elevations were observed and resolved by day 15 post-treatment. Additionally, the formulations showed promising interim stability, maintaining potency after three months at -20⁰C and -80⁰C. These and other LNP advances continue to give us confidence in our delivery to the liver and support our advances towards identifying and selecting future development candidates.editor.

Ocular disorders: Stargardt disease

We are currently evaluating AAV technology to correct one of the most prevalent mutations in the ABCA4 gene causing Stargardt disease, a progressive macular degeneration.degeneration disease. This mutation is known as the G1961E point mutation.mutation and approximately 5,500 individuals in the United States are affected.

Our base editing approach is to repair the G1961E point mutation in the ABCA4 gene. Disease modeling using tiny spot stimuli, or light stimuli through holes that are equivalent in size to a single photoreceptor cell, suggests that only 12%-20% of these cells are sufficientnecessary to preserve vision. We anticipate, therefore, that editing percentages in the range of 12%-20% of these cells would be disease-modifying, since each edited cell will be fully corrected and protected from the biochemical defect.defect associated with Stargardt disease.

We have identified a base editor that is able to edit approximately 45% of the alleles in recombinant cells carrying the human mutated sequence. Given that the base editor is larger than the packaging capacity of a single AAV, we use a split AAV system that delivers the base editor via two AAV vectors. Once inside the cell, the two halves of the editor are recombined to create a functional base editor. In a human retinal pigment epithelial cell line (ARPE-19 cells) in which we have knocked in the ABCA4 G1961E point mutation, we have demonstrated the precise correction of approximately 75% of the disease alleles at 5five weeks after dual infection with the split AAV system. In November 2021, we announced that we have initiated preclinical studies in NHPs for our Stargardt program.

Delivery of genetic medicines

To complement our next-generation gene editing technologies, we are also making significant investments in a broad suite of delivery technologies designed to deliver our gene editing or other nucleic acid payloads to the right cells toand enable potentially curative therapy. These delivery technologies include ex vivoelectroporation, nonviral vectors such as LNPs, and viral vectors such as AAVs. In our pipeline, we have initially focused on applications of these technologies that arewhere their delivery capabilities have already been clinically-validated by third parties, such as ex vivo editing of blood stem cells orand LNP delivery to the liver. Longer term, we are also investing in more innovative delivery options, such as LNPs that could target other organs beyond the liver, or novel viral vectors beyond AAV. We have also developed critical enabling capabilities such as mRNA manufacturing and cell processing for autologous and allogeneic cell therapy.

Consistent with this approach, our acquisition of Guide Therapeutics, Inc., or Guide, expandsexpanded our ability to explore new tissues and disease indications with our editing technologies. With Guide’s proprietary screening technology, which utilizes DNA barcodes to enable

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high throughput in vivo LNP screening, provides us with access to an existingwe have a broad library of lipids and lipid formulations, and the ability to generatewe have generated additional novel LNPs that we believe couldcan accelerate novel nonviral delivery of gene editing or other nucleic acid payloads to tissues beyond the liver. In September 2021,For example, we provided an update on our strategy for developing LNPs to deliver base editors to tissues beyond the liver, including hematopoietic stem and progenitor cells, or HSPCs. Leveraginghave used our DNA barcoding technology we identifiedto identify a family of LNPs for delivery of base editors to HSPCs in mice, with administration at 1.0 mg/kg leading to 40% expression of mRNA cargo in cells. We are evaluating this delivery approach for potential application in hemoglobinopathies and other genetic blood disorders. In November 2021, we announced that we utilized our DNA barcoding technology to screenhave screened more than 1,000 LNPs and identified LNP-HSC1 as the most potent. LNP-HSC1 was validated in vivo, leading to identify LNPs that achieve durable, dose-dependent mRNA transfection in HSPCs of more than 40%, which was maintained for 10 weeks post-delivery. Further, LNP-HSC1 efficiently transfectedin mice at doses ranging from 0.3 mg/kg to 1.0 mg/kg, and NHPs in NHPs, a dose-dependent increase in mRNA in bone marrow-derived CD34+ HSPCs was observed.

Collaborations

We believe our base editing technology has potential across a broad array of genetic diseases. To fully realize this potential, we have established and will continue to seek out innovative collaborations, licenses, and strategic alliances with pioneering companies and with leading academic and research institutions. Additionally, we have and will continue to pursue relationships that potentially allow us to accelerate our preclinical research and development efforts. These relationships will allow us to aggressively pursue our vision of maximizing the potential of base editing to provide life-long cures for patients suffering from serious diseases.

Hematology and oncology

Sana Biotechnology

In October 2021, we entered into an option and license agreement with Sana Biotechnology, Inc., or Sana, pursuant to which we granted Sana non-exclusive commercial rights to our CRISPR Cas12b nuclease system for certain ex vivo engineered cell therapy programs. Cas12b is a CRISPR-based nuclease with a high degree of specificity and efficiency that can be used to knock out and/or knock in genes in certain cell types. Under the terms of the agreement, Sana received non-exclusive rights to utilize our Cas12b system with certain allogeneic T cell and stem cell-derived programs. The agreement excludes any rights to base editing using our Cas12b system, which commercial rights remain with Beam. Under the terms of the agreement, Sana made a $50.0 million upfront payment to us in cash in October 2021. According to the terms of our May 2018 license agreement with The Broad Institute, Inc., or Broad Institute, we owe certain sublicense fees to Broad Institute on the upfront payment. We are also eligible to receive certain target option exercise fees, milestone payments upon the achievement of certain development and commercial sale milestones, and royalties on net sales of royalty-bearing products by Sana.

Boston Children’s Hospital

In July 2020, we formed a strategic alliance with Boston Children’s Hospital, or Boston Children’s. Under the terms of the agreement, we will sponsor research programs at Boston Children’s to facilitate development of disease-specific therapies using our proprietary base editing technology. Boston Children’s will also serve as a clinical site to advance bench-to-bedside translation of our pipeline across certain therapeutic areas of interest, including programs in sickle cell disease and pediatric leukemias and exploration of new programs targeting other diseases.

Magenta Therapeutics

In June 2020, we announced a non-exclusive research and clinical collaboration agreement with Magenta Therapeutics Inc., or Magenta, to evaluate the potential utility of MGTA-117, Magenta’s novel targeted antibody-drug conjugate, for conditioning of patients with sickle cell disease and beta-thalassemia receiving our base editing therapies. Conditioning is a critical component necessary to prepare a patient’s body to receive the edited cells, which carry the corrected gene and must engraft in the patient’s bone marrow in order to be effective. Today’s conditioning regimens rely on nonspecific chemotherapy or radiation, which are associated with significant toxicities. MGTA-117 is designed to precisely target only hematopoietic stem and progenitor cells, to spare immune cells, and has shown high selectivity, potent efficacy, wide safety margins and broad tolerability in non-human primate models. MGTA-117 may be capable of clearing space in bone marrow to support long-term engraftment and rapid recovery in patients. Combining the precision of our base editing technology with the more targeted conditioning regimen enabled by MGTA-117 has the potential to further improve therapeutic outcomes for patients suffering from these severe diseases. We will be responsible for clinical trial costs related to development of our base editors when combined with MGTA-117, while Magenta will continue to be responsible for all other development costs of MGTA-117.

Liver diseases

Verve Therapeutics

In April 2019, we entered into a collaboration and license agreement, or the Verve Agreement, with Verve Therapeutics Inc., or Verve, a company focused on developing genetic medicines to safely edit the genome of adults to permanently lower LDL cholesterol and triglyceride levels and thereby treat coronary heart disease. This collaboration allows us to more fully realize the potential of base

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editing in treating cardiovascular diseases, an area outside of our core focus where the Verve team has significant expertise. Under the terms of the Verve Agreement, Verve received exclusive access to our base editing technology, gene editing, and delivery technologies for human therapeutic applications against certain cardiovascular targets. In exchange, we received shares of Verve common stock. Additionally, we will receive milestone payments for certain clinical and regulatory events and we retain the option, after the completion of Phase 1 studies, to participate in future development and commercialization, and share 50 percent of U.S. profits and losses, for any product directed against these targets. Verve granted to us a non-exclusive license under know-how and patents controlled by Verve, and an interest in joint collaboration technology. Either party may owe the other party other milestone payments for certain clinical and regulatory events related to the delivery technology products. Royalty payments may become due by either party to the other based on the net sales of any commercialized delivery technology products under the agreement. Per the terms of the Verve Agreement, we can exercise our right to participate in the future development and commercialization of any programs at the completion of Phase 1 studies.

In January 2021, Verve announced it had selected VERVE-101 as its lead product to be developed initially for the treatment of heterozygous familial hypercholesterolemia, or HeFH, a potentially fatal genetic heart disease. Individuals with HeFH have a genetic mutation causing extremely high LDL-C levels in the blood. Over time, high LDL-C builds up in the heart’s arteries, resulting in reduced blood flow or blockage, and ultimately heart attack or stroke. Inactivation of the proprotein convertase subtilisin/kexin type 9, or PCSK9, gene has been shown to up-regulate LDL receptor expression, which leads to lower LDL-C levels. By making a single A-to-G change in the DNA genetic sequence of PCSK9, VERVE-101 aims to inactivate the target gene.

In January 2021, Verve also reported additional preclinical proof-of-concept data in non-human primates that demonstrated the successful use of ABEs to turn off PCSK9. Utilizing ABE technology licensed from us and an optimized guide RNA packaged in an engineered LNP, Verve announced data demonstrating that in vivo base editing of the PCSK9 gene in the liver of non-human primates resulted in durable and consistent lowering of blood LDL-C and blood PCSK9 protein levels following a single course of treatment. In its pre-clinical studies, Verve reported that a single intravenous infusion achieved a 59% reduction in blood LDL-C at two weeks, which was maintained at six months post treatment, and that LDL-C reduction over this time period averaged 61%. Verve also disclosed that during this same six-month time period, the average blood PCSK9 protein level was reduced by 89%. Verve further reported that the treatment was well tolerated with no adverse events reported during the study and that in studies of primary human hepatocytes, clear evidence of on-target editing was observed with no evidence of off-target editing.

Verve announced the closing of its initial public offering in June 2021, at which time we owned 546,970 shares of its common stock. Following the initial public offering, the equity securities are included in long-term investments on the consolidated balance sheet. We recorded the investment at fair value as of September 30, 2021, which resulted in of the recognition of $4.9 million of other expense and $22.0 million of other income for the three and nine months ended September 30, 2021, respectively. During the nine months ended September 30, 2020, we recognized gains of $0.5 million, which is recorded in other income, on our investment in Verve stock. The value of this investment as of September 30, 2021 is $24.4 million.

Ophthalmology

Institute of Molecular and Clinical Ophthalmology Basel

In July 2020, we announced a research collaboration with the Institute of Molecular and Clinical Ophthalmology Basel, or IOB. Founded in 2018 by a consortium that includes Novartis, the University Hospital of Basel and the University of Basel, IOB is a leader in basic and translational research aimed at treating impaired vision and blindness. Clinical scientists at IOB have also helped to develop better ways to measure how vision is impacted by Stargardt disease.

Additionally, researchers at IOB have developed living models of the retina, known as organoids, which can be used to test novel therapies. Under the terms of the agreement, the companies will leverage IOB’s unique expertise in the field of ophthalmology along with our novel base editing technology to advance programs directed to the treatment of certain ocular diseases, including Stargardt disease.

Complement-Driven Diseases

Apellis Pharmaceuticals

In June 2021, we entered into a research collaboration agreement, or the Apellis Agreement, with Apellis Pharmaceuticals, Inc., or Apellis, focused on the use of certain of our base editing technology to discover new treatments for complement system-driven diseases. Under the terms of the Apellis Agreement, we will supply certain of our base editing technology and conduct preclinical research on up to six base editing programs that target specific genes within the complement system in various organs, including the eye, liver, and brain. Apellis will have exclusive rights to license each of the six programs and will assume responsibility for subsequent development. We may elect to enter into a 50-50 U.S. co-development and co-commercialization agreement with Apellis with respect to one program licensed under the collaboration.

As part of the collaboration, we are eligible to receive a total of $75.0 million in upfront and near-term milestones from Apellis, which is comprised of $50.0 million received upon signing and an additional $25.0 million payment on June 30, 2022, the one-year

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anniversary of the signing date of the Apellis Agreement. Following any exercise of the opt-in license rights for any of the six programs, we will be eligible to receive development, regulatory, and sales milestones from Apellis, as well as royalty payments on sales. The collaboration has an initial term of five years and may be extended up to two years on a per year and program-by-program basis. We received the $50.0 million initial payment in July 2021. According to the terms of our June 2017 license agreement with Harvard University, or Harvard, we owe certain sublicense fees to Harvard on the upfront payment.

Manufacturing of genetic medicines

To realize the full potential of base editors as a newdifferentiated class of medicines and to enable our parallel investment strategy in multiple delivery modalities, we are building customized and integrated capabilities across discovery, manufacturing, and preclinical and clinical development. Due to the critical importance of high-quality manufacturing and control of production timing and know-how, we have taken steps toward establishing our own manufacturing facility, which will provide us the flexibility to manufacture a variety of different product modalities. We believe this investment will maximize the value of our portfolio and capabilities, the probability of technical success of our programs, and the speed at which we can provide potentially life-long cures to patients.

In August 2020, we entered into a lease agreement with Alexandria Real Estate Equities, Inc. to build a 100,000 square foot current cGMP compliant manufacturing facility in Research Triangle Park, North Carolina intended to support a broad range of clinical programs. The initial estimate of the minimum amount of undiscounted lease payments due under this lease is $81.1$69.0 million. The

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tabular disclosure of minimum lease payments above under Note 7, Leases, does not include payments due under this lease. We anticipate that the facility will be operational in the first quarter of 2023. The project will beis facilitated, in part, by a Job Development Investment Grant approved by the North Carolina Economic Investment Committee, which authorizes potential reimbursements based on new tax revenues generated through the project. The facility will beis designed to support manufacturing for our ex vivocell therapy programs in hematology and oncology and in vivo non-viral delivery programs for liver diseases, with flexibility to support manufacturing of our viral delivery programs, and ultimately, scale-up to support potential commercial supply.

For our initial waves of clinical programs,trials, we willexpect to use contract manufacturing organizations, or CMOs with relevant manufacturing experience in genetic medicines.

Collaborations

We believe our collection of base editing, gene editing and delivery technologies has significant potential across a broad array of genetic diseases. To fully realize this potential, we have established and will continue to seek out innovative collaborations, licenses, and strategic alliances with pioneering companies and with leading academic and research institutions. Additionally, we have and will continue to pursue relationships that potentially allow us to accelerate our preclinical research and development efforts. These relationships will allow us to aggressively pursue our vision of maximizing the potential of base editing to provide life-long cures for patients suffering from serious diseases.

In vivo collaborations

Pfizer

In December 2021, we entered into a research collaboration agreement with Pfizer Inc., or Pfizer, focused on in vivo base editing programs for three targets for rare genetic diseases of the liver, muscle and central nervous system. The collaboration has an initial term of four years and may be extended for an additional one year on a program-by-program basis. Under the terms of the agreement, we will conduct all research activities through development candidate selection for three pre-specified, undisclosed targets, which are not included in our existing programs. Pfizer may opt in to exclusive, worldwide licenses to each development candidate, after which it will be responsible for all development activities, as well as potential regulatory approvals and commercialization, for each such development candidate. We have a right to opt in, at the end of Phase 1/2 clinical trials, upon the payment of an option exercise fee, to a global co-development and co-commercialization agreement with respect to one program licensed under the collaboration pursuant to which we and Pfizer would share net profits as well as development and commercialization costs in a 35%/65% ratio (Beam/Pfizer).

Apellis Pharmaceuticals

In June 2021, we entered into a research collaboration agreement with Apellis Pharmaceuticals, Inc., or Apellis, focused on the use of certain of our base editing technology to discover new treatments for complement system-driven diseases. Under the terms of the agreement, we will conduct preclinical research on up to six base editing programs that target specific genes within the complement system in various organs, including the eye, liver, and brain. Apellis has an exclusive option to license any or all of the six programs and will assume responsibility for subsequent development. We may elect to enter into a 50-50 U.S. co-development and co-commercialization agreement with Apellis with respect to one program licensed under the collaboration.

Verve Therapeutics

In April 2019, we entered into a collaboration and license agreement, or the Verve Agreement, with Verve Therapeutics, Inc., or Verve, a company focused on gene editing for cardiovascular disease treatments. This collaboration allows us to more fully realize the potential of base editing in treating cardiovascular disease, a disease area outside of our core focus and where Verve has significant expertise. Under the terms of the Verve Agreement, Verve received exclusive worldwide licenses to use our base editing technology and certain gene editing and delivery technologies for human therapeutic applications against certain cardiovascular targets. In exchange, we received shares of Verve common stock. Additionally, we are eligible to receive milestone payments for certain clinical and regulatory events for licensed products, and we retain the option, after the completion of Phase 1 clinical trials, to participate in future development and commercialization, and share 50 percent of U.S. profits and losses, for any licensed product directed against these targets.

In January 2021, Verve announced it had selected VERVE-101 as its lead product to be developed initially for the treatment of heterozygous familial hypercholesterolemia, or HeFH, a potentially fatal genetic heart disease. Individuals with HeFH have a genetic mutation causing high LDL-C levels in the blood. Over time, high LDL-C builds up in the heart’s arteries, resulting in reduced blood flow or blockage, and ultimately heart attack or stroke. Inactivation of the proprotein convertase subtilisin/kexin type 9, or PCSK9, gene has been shown to up-regulate LDL receptor expression, which leads to lower LDL-C levels. By making a single A-to-G change in the DNA genetic sequence of PCSK9, VERVE-101 aims to inactivate the target gene. In January 2021, Verve also reported preclinical proof-of-concept data in NHPs that demonstrated the successful use of ABEs to turn off PCSK9, and in January 2022, Verve announced it expects to submit an IND application for VERVE-101 in the second half of 2022.

Institute of Molecular and Clinical Ophthalmology Basel

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In July 2020, we announced a research collaboration with the Institute of Molecular and Clinical Ophthalmology Basel, or IOB. Founded in 2018 by a consortium that includes Novartis, the University Hospital of Basel and the University of Basel, IOB is a leader in basic and translational research aimed at treating impaired vision and blindness. Clinical scientists at IOB have also helped to develop better ways to measure how vision is impacted by Stargardt disease.

Additionally, researchers at IOB have developed living models of the retina, known as organoids, which can be used to test novel therapies. Under the terms of the agreement with IOB, the parties will leverage IOB’s unique expertise in the field of ophthalmology along with our novel base editing technology to advance programs directed to the treatment of certain ocular diseases, including Stargardt disease.

Ex vivo collaborations

Sana Biotechnology

In October 2021, we entered into an option and license agreement, or the Sana Agreement, with Sana Biotechnology, Inc., or Sana, pursuant to which we granted Sana non-exclusive research and development and commercial rights to our CRISPR Cas12b technology to perform nuclease editing for certain ex vivo engineered cell therapy programs. Under the terms of the Sana Agreement, licensed products include certain specified allogeneic T cell and stem cell-derived products directed at specified genetic targets, with certain limited rights for Sana to add and substitute such products and targets. The Sana Agreement excludes the grant of any Beam-controlled rights to perform base editing.

Boston Children’s Hospital

In July 2020, we entered into an alliance agreement, or the BCH Agreement, with Boston Children’s Hospital, or Boston Children’s. Under the terms of the BCH Agreement, we will identify and sponsor research programs to be performed at Boston Children’s, either solely by Boston Children’s or by both Boston Children’s and us, to facilitate the development of certain disease-specific therapies using our proprietary base editing technology. Boston Children’s will also serve as a clinical site to advance bench-to-bedside translation of our pipeline across certain therapeutic areas of interest, including programs in sickle cell disease and pediatric leukemias and exploration of new programs targeting other diseases.

Magenta Therapeutics

In June 2020, we announced a non-exclusive research and clinical trial collaboration agreement with Magenta Therapeutics Inc., or Magenta, to evaluate the potential utility of MGTA-117, Magenta’s novel targeted antibody-drug conjugate, for conditioning of patients with sickle cell disease and beta-thalassemia receiving our base editing therapies. Conditioning is a critical component necessary to prepare a patient’s body to receive the edited cells, which carry the corrected gene and must engraft in the patient’s bone marrow in order to be effective. Today’s conditioning regimens rely on nonspecific chemotherapy or radiation, which are associated with significant toxicities. MGTA-117 is designed to precisely target only hematopoietic stem and progenitor cells, to spare immune cells, and has shown high selectivity, potent efficacy, wide safety margins and broad tolerability in NHP models. MGTA-117 may be capable of clearing space in bone marrow to support long-term engraftment and rapid recovery in patients. Combining the precision of our base editing technology with the more targeted conditioning regimen enabled by MGTA-117 has the potential to further improve therapeutic outcomes for patients suffering from these severe diseases.

Acquisitions

In February 2021, we entered into an Agreement and Plan of Merger, or the Guide Merger Agreement, to acquireacquired Guide Therapeutics, Inc., or Guide. Pursuant to the Guide, Merger Agreement, we paid Guide’s former stockholders and optionholdersfor upfront consideration in an aggregate amount of $120.0 million, excluding customary purchase price adjustments, in shares of our common stock, based upon the volume-weighted average price of the common stock over the ten trading daytrading-day period ending on February 19, 2021. In addition, Guide’s former stockholders and optionholders are eligible to receive up to an additional $100.0 million in technology milestone payments and $220.0 million in product milestone payments, payable in our common stock.

COVID-19

With the ongoing concern related to the COVID-19 pandemic, in the year ended December 31, 2020 and the nine months ended September 30, 2021, we have maintained and expanded our business continuity plans to address and mitigate the impact of the COVID-19 pandemic on our business. In March 2020, to protect the health of our employees, and their families and communities, we restricted access to our offices to personnel who performed critical activities that must be completed on-site, limited the number of such personnel that can be present at our facilities at any one time, and requested that most of our employees work remotely. In May 2020, as certain states eased restrictions, we established new protocols to better allow our full laboratory staff access to our facilities. These protocols included several shifts working over a seven-day week protocol. In June 2021, as states continued to ease restrictions, we started to allow for all of our employees to work on-site at our facilities, with fewer restrictions, particularly for vaccinated employees. We expect to continue incurring additional costs to ensure we adhere to the guidelines instituted by the Centers for Disease Control and to provide a safe working environment to our onsite employees.

The extent to which the COVID-19 pandemic impacts our business, our corporate development objectives, results of operations and financial condition, including the value of and market for our common stock, will depend on future developments that are highly uncertain and cannot be predicted with confidence at this time, such as the duration, scope and severity of the pandemic, the existence and duration and extent of any travel restrictions and social distancingor business restrictions in the United States and other countries, business closures and business disruptions, the effectiveness of actions taken in the United States and other countries to contain and treat the disease,periodic spikes in infection rates, new strains of the virus that cause outbreaks of COVID-19, and the broad availability of effective vaccines.vaccines and therapeutics.

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Disruptions to the global economy and supply chain, disruption of global healthcare systems, and other significant impacts of the COVID-19 pandemic could have a material adverse effect on our business, financial condition, results of operations and growth prospects.

While the COVID-19 pandemic did not significantly impact our business or results of operations during the ninethree months ended September 30, 2021,March 31, 2022, the length and extent of the pandemic, its consequences, and containment efforts will determine theits future impact on our operations and financial condition.

Critical accounting policies and significant judgements and estimates

Our management’s discussion and analysis of our financial condition and results of operations is based on our consolidated financial statements, which we have prepared in accordance with U.S. generally accepted accounting principles. The preparation of these financial statements requires us to make estimates, judgments and assumptions that affect the reported amounts of assets, liabilities, and expenses and the disclosure of contingent assets and liabilities in our financial statements. We base our estimates on historical experience, known trends and events and various other factors that we believe are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities that are not readily apparent from other sources. We evaluate our estimates and assumptions on an ongoing basis. Our actual results may differ from these estimates under different assumptions or conditions.

Our critical accounting policies are those policies which require the most significant judgments and estimates in the preparation of our condensed consolidated financial statements. We have determined that our most critical accounting policies are those relating to stock-based compensation, variable interest entities, fair value measurements, and leases. There have been no significant changes to our existing critical accounting policies and significant accounting policies discussed in the 20202021 Form 10-K, except as discussed below.

Asset Acquisitions

In 2018, we adopted ASU 2017-01, Business Combinations, or ASU 2017-01, which clarified the definition of a business. We measure and recognize asset acquisitions that are not deemed to be business combinations based on the cost to acquire the assets, which includes transaction costs, and the consideration is allocated to the items acquired based on a relative fair value methodology. Goodwill is not recognized in asset acquisitions. In an asset acquisition, the cost allocated to acquire in-process research and development with no alternative future use is charged to research and development expense at the acquisition date.

At the time of acquisition, we determine if a transaction should be accounted for as a business combination or acquisition of assets.

Contingent Consideration Liabilities

We may be required to make milestone payments to the former stockholders and optionholders of Guide in the form of our common stock based on the achievement of certain product and technology milestones. The payments are accounted for under ASC 480, Distinguishing Liabilities from Equity. These contingent consideration liabilities are carried at fair value which was estimated by applying a probability-based model, which utilized inputs primarily based upon the achievement and related timing of certain product and technology milestones that were unobservable in the market. The estimated fair value of contingent consideration liabilities, initially measured and recorded on the acquisition date, are considered to be a Level 3 measurement and are reviewed quarterly, or whenever events or circumstances occur that indicate a change in fair value. The contingent consideration liabilities are recorded at fair value at the end of each reporting period with changes in estimated fair values recorded in other income (expense) in the condensed consolidated statements of operations and other comprehensive loss.

The estimated fair value is determined based on probability adjusted discounted cash flow models that include significant estimates and assumptions pertaining to technology and product development. Significant changes in any of the probabilities of success would result in a significantly higher or lower fair value measurement. Significant changes in the probabilities as to the periods in which milestones will be achieved would result in a significantly lower or higher fair value measurement.10-K.

Financial operations overview

General

We were incorporated onfounded in January 25, 2017 and commencedbegan operations shortly thereafter.in July 2017. Since our inception, we have devoted substantially all of our resources to building our base editing platform and advancing development of our portfolio of programs, establishing and protecting our intellectual property, conducting research and development activities, organizing and staffing our company, business planning, raising capital and providing general and administrative support for these operations. To date, we have financed our operations primarily through the sales of our redeemable convertible preferred stock, proceeds from offerings of our common stock and payments received under collaboration and license agreements.

We are a development stagean early-stage company, and all of our programs are at a preclinical or early clinical stage of development. To date, we have not generated any revenue from product sales and do not expect to generate revenue from the sale of our products for the foreseeable future.Our revenue to date has been primarily derived from license and collaboration agreements with partners. Since inception we have incurred significant operating losses. Our net losses for the ninethree months ended September 30,March 31, 2022 and 2021, and 2020 were $305.9$69.2 million, and $99.1$201.6 million, respectively. As of September 30, 2021,March 31, 2022, we had an accumulated deficit of $703.6$837.5 million. We expect to continue to incur significant expenses and increasing operating losses in connection with ongoing development activities related to our portfolio ofinternal programs and collaborations as we continue our preclinical and clinical development of product candidates; advance theseadditional product candidates toward clinical development; build and operate our cGMP facility in North Carolina,Carolina; further develop our base editing platform; continue to make investments in delivery technology for our base editors, including in connection withthe LNP technology we acquired through our acquisition of Guide; conduct research activities as we seek to discover and develop additional product candidates; maintain, expand, enforce, defend and protect our intellectual property portfolio; and continue to hire research and development, clinical, technical operations and commercial personnel. In addition, we expect to continue to incur additionalthe costs associated with operating as a public company.

As a result of these anticipated expenditures, we will need to raise additional financingcapital to support our continuing operations and pursue our growth strategy. Until such time as we can generate significant revenue from product sales, if ever, we expect to finance our

29


operations through a combination of equity offerings, debt financings, collaborations, strategic alliances, and licensing arrangements. We may be unable to raise additional funds or enter into such other agreements when needed on favorable terms or at all. Our inability to raise capital as and when needed would have a negative impact on our financial condition and our ability to pursue our business strategy. We can give no assurance that we will be able to secure such additional sources of fundscapital to support our operations, or, if such funds arecapital is available to us, that such additional fundingcapital will be sufficient to meet our needs.needs for the short or long term.

Revenue Recognition

In April 2019, we entered into a collaboration and license agreement, or the Verve Agreement, with Verve Therapeutics, Inc., or Verve, a company focused on gene editing for cardiovascular disease treatments. In June 2021, we entered into a research collaboration agreement, or the Apellis Agreement, with Apellis Pharmaceuticals, Inc., or Apellis, focused on the use of certain of our base editing technology to discover new treatments for complement system-driven diseases. In October 2021, we entered into an option and license agreement, or the Sana Agreement, with Sana Biotechnology, Inc., or Sana, pursuant to which we granted Sana

29


non-exclusive research and development and commercial rights to our CRISPR Cas12b technology to perform nuclease editing for certain ex vivo engineered cell therapy programs. In December 2021, we entered into a research collaboration agreement, or the Pfizer Agreement, with Pfizer Inc., or Pfizer, focused on in vivo base editing programs for three targets for rare genetic diseases of the liver, muscle and central nervous system.

We have not generated any revenue to date from product sales and do not expect to do so in the near future. During the three months ended March 31, 2022 and 2021, we recognized $8.4 million and $6.0 thousand of revenue, respectively.

Research and development expenses

Research and development expenses consist of costs incurred in performing research and development activities, which include:

Expenses incurred in connection with investments in delivery technology for our base editors, including as a result ofthe LNP technology we acquired through our acquisition of Guide;
the cost to obtain licenses to intellectual property, such as those with Harvard University, or Harvard, The Broad Institute, of MIT and Harvard,Inc., or Broad Institute, and Editas Medicine, Inc, or Editas, and Bio Palette Co., Ltd., or Bio Palette, and related future payments should certain success, development and regulatory milestones be achieved;
personnel-related expenses, including salaries, bonuses, benefits and stock-based compensation for employees engaged in research and development functions;
expenses incurred in connection with the discovery and preclinical development of our research programs, including under agreements with third parties, such as consultants, contractors and contract research organizations;
expenses incurred in connection with the initiation of clinical trials, including contract research organization costs and costs related to study preparation;
expenses incurred in connection with regulatory filings;
expenses incurred in connection with the building of our base editing platform;
the cost of manufacturing product candidatesmaterials for use in our preclinical studies, IND-enabling studies and future clinical trials;
laboratory supplies and research materials; and
facilities, depreciation and other expenses which include direct and allocated expenses.

We expense research and development costs as incurred. Advance payments that we make for goods or services to be received in the future for use in research and development activities are recorded as prepaid expenses. The prepaid amounts are expensed as the benefits are consumed.

In the early phases of development, our research and development costs are often devoted to product platform and proof-of-concept preclinical studies that are not necessarily allocable to a specific target.

We expect that our research and development expenses will increase substantially in connection withas we advance our programs through their planned preclinical and future clinical development activities.development.

General and administrative expenses

General and administrative expenses consist primarily of salaries and other related costs, including stock-based compensation, for personnel in our executive, intellectual property, business development and administrative functions. General and administrative expenses also include legal fees relating to intellectual property and corporate matters, professional fees for accounting, auditing, tax and consulting services, insurance costs, travel, and direct and allocated facility related expenses and other operating costs.

We anticipate that our general and administrative expenses will increase in the future to support our increased research and development activities. We also expect to continue to incur increased costs associated with being a public company and implementingmaintaining controls over financial reporting, including costs of accounting, audit, legal, regulatory and tax-related services associated with maintaining compliance with the requirements of the Nasdaq Global Select Market and SEC requirements, director and officer insurance costs, and investor and public relations costs.

Other income and expenses

Other income and expenses consist of the following items:

Change in fair value of derivative liabilities consists primarily of remeasurement gains or losses associated with changes in success payment liabilities associated with our license agreement with Harvard, dated as of June 27, 2017, as amended, or the

30


Harvard License Agreement, and ourthe license agreement with The Broad Institute, as amended, dated as of May 9, 2018, as amended, or the Broad License Agreement.
Change in fair value of long-termnon-controlling equity investments consists of mark-to-market adjustments related to our investments in equity securities.

30


Change in fair value of contingent consideration liabilities consists of remeasurement of the fair market value associated with changes inof the technology and product contingent consideration liabilities as partrelated to the acquisition of the Guide Merger Agreement.Guide.
Interest and other income (expense), net consists primarily of interest income as well as interest expense related to our equipment financings.

Results of operations

Comparison of the three months ended September 30,March 31, 2022 and 2021 and 2020

The following table summarizes our results of operations (in thousands):

 

Three Months Ended September 30,

 

 

 

 

 

Three Months Ended March 31,

 

 

 

 

 

2021

 

 

2020

 

 

Change

 

 

2022

 

 

2021

 

 

Change

 

License and collaboration revenue

 

$

763

 

 

$

6

 

 

$

757

 

 

$

8,432

 

 

$

6

 

 

$

8,426

 

Operating expenses:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Research and development

 

 

54,623

 

 

 

29,825

 

 

 

24,798

 

 

 

65,410

 

 

 

190,106

 

 

 

(124,696

)

General and administrative

 

 

15,774

 

 

 

7,502

 

 

 

8,272

 

 

 

19,247

 

 

 

10,273

 

 

 

8,974

 

Total operating expenses

 

 

70,397

 

 

 

37,327

 

 

 

33,070

 

 

 

84,657

 

 

 

200,379

 

 

 

(115,722

)

Loss from operations

 

 

(69,634

)

 

 

(37,321

)

 

 

(32,313

)

 

 

(76,225

)

 

 

(200,373

)

 

 

124,148

 

Other income (expense):

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Change in fair value of derivative liabilities

 

 

35,800

 

 

 

2,700

 

 

 

33,100

 

 

 

13,600

 

 

 

(1,900

)

 

 

15,500

 

Change in fair value of long-term investments

 

 

(4,892

)

 

 

 

 

 

(4,892

)

Change in fair value of non-controlling equity investments

 

 

(7,685

)

 

 

1,039

 

 

 

(8,724

)

Change in fair value of contingent consideration liabilities

 

 

10,599

 

 

 

 

 

 

10,599

 

 

 

452

 

 

 

(305

)

 

 

757

 

Interest and other income (expense), net

 

 

9

 

 

 

169

 

 

 

(160

)

 

 

644

 

 

 

(21

)

 

 

665

 

Total other income (expense)

 

 

41,516

 

 

 

2,869

 

 

 

38,647

 

 

 

7,011

 

 

 

(1,187

)

 

 

8,198

 

Net loss

 

$

(28,118

)

 

$

(34,452

)

 

$

6,334

 

 

$

(69,214

)

 

$

(201,560

)

 

$

132,346

 

License and collaboration revenue

License and collaboration revenue was approximately $0.8$8.4 million and $6.0 thousand for the three months ended September 30,March 31, 2022 and 2021, and 2020, respectively. License and collaboration revenue represents revenue recorded under the Pfizer, Apellis, and Verve Agreements.

Research and development expenses

Research and development expenses were $54.6$65.4 million and $29.8$190.1 million for the three months ended September 30,March 31, 2022 and 2021, and 2020, respectively. The increasedecrease of $24.8$124.7 million was primarily due to the following:

A decrease of $155.0 million related to the write-off of in-process research and development asset acquired from Guide during the three months ended March 31, 2022, as it was determined to be of no alternative future use.
An increase of $6.6$10.8 million in personnel-related costs and $3.9$3.0 million in facility-related costs, including depreciation. These increases were due to the growth in the number of research and development employees from 136182 at September 30, 2020March 31, 2021 to 250321 at September 30, 2021,March 31, 2022, and their related activities, as well as the expense allocated to research and development related to our leased facilities.
An increase of $6.0 million in lab supplies due to the movement of our lead programs into IND enabling activities and continued investment in platform and discovery efforts.
An increase of $5.6$8.1 million in stock-based compensation from additional stock option awards due to the increase in the number of research and development employees as well as an increase in the value of our common stock.stock during 2021.
An increase of $4.1$4.6 million in lab supplies due to the movement of our lead programs into IND-enabling activities and continued investment in platform and discovery efforts.
An increase of $1.6 million in other expenses, primarily related to an increase in research and development specific software costs.
An increase of $1.5 million in outsourced services, driven by process development spend and IND enablingIND-enabling materials for BEAM-102, assay development and qualification for mRNA and gRNA for BEAM-101 and BEAM-201, toxicology studies related to BEAM-201 and initial clinical start-up activities for BEAM-101.

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An increase of $1.4$0.6 million in other expenses, primarily related to an increase in research and development specific software costs.
A decrease of $2.7 million in milestonelicense expenses. During the three months ended September 30, 2020, we recognized $5.0 million expense as a result of our decision to extend our collaboration with Prime Medicine. In September 2021, we recorded an additional $1.6 million in sublicense fees and $0.7 million of expense for milestones related to our technology license agreements.

Research and development expenses are expected to continue to increase as we start to initiate clinical trials for BEAM-101, continue IND enablingIND-enabling studies for BEAM-102 and BEAM-201, begin IND-enabling studies for BEAM-301, continue our current research programs, initiate new research programs, continue the preclinical and clinical development of our product candidates and conduct any future preclinical studies and begin to enroll patients in and conduct clinical trials for any of our product candidates.

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General and administrative expenses

General and administrative expenses were $15.8$19.2 million and $7.5$10.3 million for the three months ended September 30,March 31, 2022 and 2021, and 2020, respectively. The increase of $8.3$9.0 million was primarily due to the following:

An increase of $4.4$5.2 million in stock-based compensation due to an increase in the number of general and administrative employees, as well as an increase in the value of our common stock.stock during 2021.
An increaseincrease of $2.5$3.4 million in personnel related costs and $0.6 million$0.1 million in facility-related costs, including depreciation, due to an increase in general and administrative employees from 3034 employees as of September 30, 2020March 31, 2021 to 5673 employees as of September 30, 2021,March 31, 2022, as well as the expense allocated to general and administrative expenses related to our leased facilities.
An increase of $0.4$0.3 million in legal costs primarily due to legal fees incurred in connection with business development activities.
An increase of $0.2 million in insurance costs due to higher premiums attributable to the Company's directors and officers insurance policy following our IPO in February 2020 and insurance costs related to our acquisition of Guide in 2021.
An increaseA decrease of $0.5$0.2 million of other expenses primarily related to a decrease in legal costs primarily due to legal fees incurred in connection with the Apellis and Sana Agreements.software costs.

Change in fair value of derivative liabilities

During the three months ended September 30, 2021,March 31, 2022, we recorded $35.8$13.6 million of other income related to the change in fair value of success payment liabilities as compared to $2.7 million of other income for the three months ended September 30, 2020 due to decreasesa decrease in the price of our common stock for the three months ended September 30,March 31, 2022. During the three months ended March 31, 2021, and 2020.we recorded $1.9 million of other expense due to an increase in the price of our common stock for the three months ended March 31, 2021. A portion of the success payment obligations were paid in June 2021; the remaining success payment obligations are still outstanding as of September 30, 2021March 31, 2022 and will continue to be revalued at each reporting period.

Change in fair value of long-termnon-controlling equity investments

During the three months ended September 30,March 31, 2022 and 2021, we recorded $7.7 million of other expense and $1.0 million of $4.9 millionother income, respectively, as a result of a decreasechanges in the value of our investment in Verve's common stock.

Change in contingent consideration liabilities

During the three months ended September 30,March 31, 2022 and 2021, we recorded $10.6$0.5 million of other income and $0.3 million of other expense, respectively, related to the change in fair value of the Guide technology and product contingent consideration liabilities asliabilities. These changes are a result of an update in project timelines and the expected probability of achievement of the milestones.

Interest and other income (expense), net

The decrease in interestInterest and other income (expense), net was $0.6 million for the three months ended March 31, 2022 as compared to $21.0 thousand of expense for the three months ended March 31, 2021. The increase was primarily due to a decreaseincreases in interest income driven by decreasedincreased market rates.

Comparison of the nine months ended September 30, 2021rates and 2020

The following table summarizes our results of operations, together with the change in dollars (in thousands):

 

 

Nine Months Ended September 30,

 

 

 

 

 

 

2021

 

 

2020

 

 

Change

 

License and collaboration revenue

 

$

775

 

 

$

18

 

 

$

757

 

Operating expenses:

 

 

 

 

 

 

 

 

 

Research and development

 

 

290,306

 

 

 

70,728

 

 

 

219,578

 

General and administrative

 

 

39,450

 

 

 

21,251

 

 

 

18,199

 

Total operating expenses

 

 

329,756

 

 

 

91,979

 

 

 

237,777

 

Loss from operations

 

 

(328,981

)

 

 

(91,961

)

 

 

(237,020

)

Other income (expense):

 

 

 

 

 

 

 

 

 

Change in fair value of derivative liabilities

 

 

(8,400

)

 

 

(8,700

)

 

 

300

 

Change in fair value of long-term investments

 

 

21,960

 

 

 

517

 

 

 

21,443

 

Change in fair value of contingent consideration liabilities

 

 

9,553

 

 

 

 

 

 

9,553

 

Interest and other income (expense), net

 

 

(63

)

 

 

1,016

 

 

 

(1,079

)

Total other income (expense)

 

 

23,050

 

 

 

(7,167

)

 

 

30,217

 

Net loss

 

$

(305,931

)

 

$

(99,128

)

 

$

(206,803

)

License and collaboration revenue

License and collaboration revenue was approximately $0.8 million and $18.0 thousand for the nine months ended September 30, 2021 and September 30, 2020, respectively. License and collaboration revenue represents revenue recorded under the Apellis and the Verve Agreements.

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Research and development expenses

Research and development expenses were $290.3 million and $70.7 million for the nine months ended September 30, 2021 and 2020, respectively. The increase of $219.6 million was primarily due to the following:

$155.0 million of expense related to in-process research and development asset acquired from Guide as there was determined to be no alternative future use.
An increase of $17.9 million in outsourced services, driven by process development, IND enabling materials and clinical readiness spend for BEAM-101, BEAM-201 and BEAM-102, assay development and qualification of gRNA and mRNA materials for BEAM-101 and BEAM-201, and toxicology studies and activities for BEAM-101 and BEAM-201.
Increases of $14.0 million in personnel-related costs and $10.8 million in facility-related costs, including depreciation. These increases were due to the growth in the number of research and development employees from 136 at September 30, 2020 to 250 at September 30, 2021, and their related activities, as well as the expense allocated to research and development related to our leased facilities.
An increase of $11.5 million in stock-based compensation due to the increase in the number of research and development employees, as well as an increase in the value of our common stock.
An increase of $10.6 million in lab supplies due to the movement of our lead programs into IND enabling activities and continued investment in platform and discovery efforts.
An increase of $3.8 million in other expenses, primarily related to an increase in research and development specific software costs.
A decrease of $4.0 million in milestone and license expenses. During the nine months ended September 30, 2020, we recognized $5.0 million in expense as a result of our decision to extend our collaboration with Prime Medicine. During the nine months ended September 30, 2020, we also recorded a $2.3 million expense for a milestone paid to Bio Palette upon the issuance of a certain patent in the United States becoming probable. During the nine months ended September 30, 2021, we recorded $2.7 million in sublicense fees. In September 2021, we recorded $0.7 million of expense for milestones related to our technology license agreements.

Research and development expenses are expected to continue to increase as we start to initiate clinical trials for BEAM-101, continue IND-enabling studies for BEAM-102, continue our current research programs, initiate new research programs, continue the preclinical development of our product candidates and conduct any future preclinical studies and begin to enroll patients and conduct clinical trials for any of our product candidates.

General and administrative expenses

General and administrative expenses were $39.5 million and $21.3 million for the nine months ended September 30, 2021 and 2020, respectively. The increase of $18.2 million was primarily due to the following:

An increase of $8.0 million in stock-based compensation due to an increase in the number of general and administrative employees, as well as an increase in the value of our common stock.
Increases of $6.6 million in personnel related costs and $1.4 million in facility related costs, including depreciation costs, due to an increase in general and administrative employees from 30 employees as of September 30, 2020 to 56 employees as of September 30, 2021, as well as the expense allocated to general and administrative expenses related to our leased facilities.
An increase of $1.2 million in insurance costs due to increased directors and officers insurance costs as a result of our IPO in February 2020 and insurance costs related to our acquisition of Guide in 2021.
An increase of $1.4 million in legal costs primarily due to legal fees incurred in connection with our acquisition of Guide and the completion of the Apellis and Sana Agreements.

Change in fair value of derivative liabilities

During the nine months ended September 30, 2021, we recorded $8.4 million of other expense related to the change in fair value of success payment liabilities as compared to $8.7 million of other expense for the nine months ended September 30, 2020 due to increases in the price of our common stock for the nine months ended September 30, 2021 and 2020. A portion of the success payments was paid in shares in June 2021; the remaining success payment obligations are still outstanding as of September 30, 2021 and will continue to be revalued at each reporting period.

Change in fair value of long-term investments

During the nine months ended September 30, 2021 and 2020, we recorded income of $22.0 million and $0.5 million as a result of increases in the value of our investment in Verve's common stock.

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Change in contingent consideration liabilities

During the nine months ended September 30, 2021, we recorded $9.6 million of other income related to the change in fair value of the Guide technology and product contingent consideration liabilities as a result of an update in project timelines and the expected probability of achievement of the milestones.

Interest and other income (expense), net

The decrease in interest and other income (expense), net was primarily due to a decrease in interest income driven by decreased market rates.portfolio.

Liquidity and capital resources

Since our inception in January 2017, we have not generated any revenue from product sales, have generated only limited license and collaboration revenue from the Verve Agreementour license and the Apellis Agreement,collaboration agreements, and have incurred significant operating losses and negative cash flows from our operations. We expect to incur significant expenses and operating losses for the foreseeable future as we advance the preclinical and if successful, the clinical development of our product candidates. In February 2020,

To date, we completedhave funded our IPO in which we issued and sold 12,176,471 shares of our common stock, including 1,588,235 shares of common stock sold pursuant to the underwriters’ full exercise of their option to purchase additional shares, at a public offering price of $17.00 per share. We received net proceeds from our IPO of $188.3 million, after deducting underwriting discounts and offering expenses payable by us. In October 2020, we issued and sold 5,750,000 shares of our common stock, including 750,000 shares pursuant to the full exercise of the underwriters’ option to purchase additional shares, at a public offering price of $23.50 per share, for aggregate gross proceeds of $135.1 million. We received approximately $126.6 million in net proceeds after deducting applicable underwriting discounts and offering expenses.operations primarily through equity offerings. In January 2021, we issued and sold 2,795,700 shares of our common stock in a private placement at an offering price of $93.00 per share for aggregate gross proceeds of $260.0 million. We received $252.0 million in net proceeds after deducting offering expenses payable by us. To date, we have funded our operations primarily through equity offerings.

In April 2021, we filed a universal shelf registration statement on Form S-3 with the SEC, or the 2021 Shelf, to register for sale an indeterminate amount of our common stock, preferred stock, debt securities, warrants and/or units in one or more offerings, which became effective upon filing with the SEC (File No. 333-254946).

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In April 2021, we entered into an at the market, or ATM, sales agreement, or the Sales Agreement, with Jefferies LLC, or Jefferies, pursuant to which we were entitled to offer and sell, from time to time at prevailing market prices, shares of our common stock having aggregate gross proceeds of up to $300.0 million. We agreed to pay Jefferies a commission of up to 3.0% of the aggregate gross sale proceeds of any shares sold by Jefferies under the Sales Agreement. As of September 30,Between April and July 2021, we have sold 2,908,009 shares of our common stock under the Sales Agreement at an average price of $103.16 per share for aggregate gross proceeds of $300.0 million, before deducting commissions and offering expenses payable by us.

In July 2021, we and Jefferies entered into an amendment to the Sales Agreement to provide for an increase in the aggregate offering amount under the Sales Agreement, such that as of July 7, 2021, we may offer and sell shares of common stock having an aggregate offering price of an additional $500.0 million. As of September 30, 2021,March 31, 2022, we have sold 1,765,8332,873,956 additional shares of our common stock under the amended Sales Agreement at an average price of $107.88$92.71 per share for aggregate gross proceeds of $190.5$266.5 million, before deducting commissions and offering expenses payable by us, resultingus.

In December 2021, we entered into the Pfizer Agreement, which is focused on in vivo base editing programs for three targets for rare genetic diseases of the liver, muscle and central nervous system. Under the terms of the Pfizer Agreement, we will conduct all research activities through development candidate selection for three undisclosed targets, which are not included in our existing programs. Pursuant to the Pfizer Agreement, we received an aggregateupfront payment of $490.5$300.0 million in gross proceeds received under the Sales Agreement as of September 30, 2021.January 2022.

As of September 30, 2021,March 31, 2022, we had $933.4 million$1.2 billion in cash, cash equivalents, and marketable securities.

We are required to make success payments to Harvard and Broad Institute based on increases in the per share fair market value of our Series A-1 Preferred Stock and Series A-2 Preferred Stock or, subsequent to our IPO, our common stock. The amounts due may be settled in cash or shares of our common stock, at our discretion. In May 2021, the first success payment measurements occurred and success payments to Harvard and Broad Institute were calculated to be $15.0 million and $15.0 million, respectively. We elected to make each payment in shares of our common stock and issued 174,825 shares to each of Harvard and Broad Institute to settle these liabilities in June 2021. We may additionally owe Harvard and Broad Institute success payments of up to an additional $90.0 million each.

We have not yet commercialized any of our product candidates, and we do not expect to generate revenue from the sale of our productsproduct candidates for the foreseeable future. We anticipate that we may need to raise additional capital in order to continue to fund our research and development, including our planned preclinical studies and clinical trials, building, maintaining and operating a commercial-scale cGMP manufacturing facility, and new product development, as well as to fund our general operations. As and if necessary, we will seek to raise these additional fundscapital through various potential sources, such as equity and debt financings or through corporate collaboration and license agreements. Especially in light of the COVID-19 pandemic, weWe can give no assurances that we will be able to secure such additional sources of fundscapital to support our operations, or, if such funds are available to us, that such additional financing will be sufficient to meet our needs. For a more detailed discussion of risks related to COVID-19, please see Part I, Item 1A, Risk Factors—Risks related to our relationships with third parties, included in our 2020 Form 10-K.

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Cash flows

The following table summarizes our sources and uses of cash (in thousands):

 

Nine Months Ended September 30,

 

 

Three Months Ended March 31,

 

 

2021

 

 

2020

 

 

2022

 

 

2021

 

Net cash used in operating activities

 

$

(68,098

)

 

$

(71,443

)

Net cash provided by (used in) operating activities

 

$

218,490

 

 

$

(38,578

)

Net cash used in investing activities

 

 

(216,648

)

 

 

(18,696

)

 

 

(537,339

)

 

 

(279,626

)

Net cash provided by financing activities

 

 

734,598

 

 

 

192,329

 

 

 

55,676

 

 

 

253,274

 

Net change in cash, cash equivalents and restricted cash

 

$

449,852

 

 

$

102,190

 

 

$

(263,173

)

 

$

(64,930

)

Operating activities

Net cash provided by operating activities for the three months ended March 31, 2022 was $218.5 million, consisting primarily of the collection of collaboration receivables of $300.0 million related to the Pfizer Agreement and an increase in other long-term liabilities of $8.3 million, in addition to noncash items consisting primarily of stock-based compensation expense of $18.0 million, a decrease in the fair value of a non-controlling equity investment of $7.7 million, depreciation and amortization expense of $3.3 million and a change in operating lease ROU assets of $2.0 million. These sources of cash were partially offset by our net loss of $69.2 million, decreases in accrued expenses and other liabilities of $16.5 million, deferred revenue of $8.4 million, operating lease liabilities of $1.8 million and accounts payable of $1.3 million, and an increase in prepaid expenses and other current assets of $9.2 million, as well as noncash items including decreases in the fair value of derivative liabilities of $13.6 million and in the fair value of contingent consideration liabilities of $0.5 million as well as amortization of investment premiums of $0.4 million.

Net cash used in operating activities for the ninethree months ended September 30,March 31, 2021 was $68.1$38.6 million, consisting primarily of our net loss of $305.9$201.6 million, a decrease in accounts payable of $1.3 million and noncash items including an increase in the fair value of a non-controlling equity investment of $22.0 million and a change in fair value of contingent consideration liabilities of $9.6 million. These uses of cash were offset in part by an increase in deferred revenue of $49.2 million, primarily related to the Apellis Agreement; an increase in operating lease liabilities of $15.2 million; an increase in accrued expenses and other liabilities of $2.8 million and a decrease in prepaid expenses and other current assets of $0.7 million and noncash items consisting primarily of in-process research and development expenses of $155.0 million, stock-based compensation expense of $28.1 million, change in fair value of derivative liabilities of $8.4 million, a change in operating lease ROU assets of $6.8 million and depreciation and amortization expense of $4.8 million.

Net cash used in operating activities for the nine months ended September 30, 2020 was $71.4 million, consisting primarily of our net loss of $99.1$4.5 million, an increase in prepaid expenses and other current assets of $3.7$2.4 million, a decrease in accounts payable of $0.7 million and other non-cash items of $1.0 million; offset by a change in operating lease liabilities of $3.2 million; offset by an increase in accrued expenses and other liabilities of $4.2$6.1 million, and noncash chargesexpenses consisting primarily of in-process research and development expense of $155.0 million related to our acquisition of Guide, stock-based compensation expense of $8.6$4.6 million, change

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in operating lease ROU assets of $2.4 million, change in fair value of derivative liabilities of $8.7 million, non-cash research and developmental license expense, net of $5.2$1.9 million, depreciation and amortization expense of $3.5$1.4 million and change in operating lease ROU assetscontingent consideration liabilities of $3.1$0.3 million.

Investing activities

For the ninethree months ended September 30,March 31, 2022, cash used in investing activities was primarily the net purchases of marketable securities of $530.1 million, and purchases of property and equipment of $7.3 million.

For the three months ended March 31, 2021, cash used in investing activities was primarily the net purchases of marketable securities of $183.8$268.8 million, and purchases of property and equipment of $33.4$11.5 million. We also received $0.6 million in net cash from our acquisition of Guide, after the payment of acquisition costs.

For the nine months ended September 30, 2020, cash used in investing activities was primarily the net purchases of marketable securities of $9.7 million, and purchases of property and equipment of $8.2 million.

Financing activities

Net cash provided by financing activities for the ninethree months ended September 30, 2021March 31, 2022 consisted primarily of proceeds from equity offerings of $737.2$54.0 million, $1.4 million of proceeds from the issuance of common stock under benefit plans, and proceeds from the exercise of stock options of $0.8 million, offset in part by repayments of equipment financing liabilities of $0.6 million.

Net cash provided by financing activities for the three months ended March 31, 2021 consisted primarily of proceeds from our private placement offering of $260.0 million and proceeds from the exercise of stock options of $7.7$1.8 million, offset in part by the payment of equity offering costs of $8.7$8.0 million and repayments of equipment financing liabilities of $1.6 million.

Net cash provided by financing activities for the nine months ended September 30, 2020 consisted primarily of proceeds from our IPO of $192.5 million, net of underwriting discounts, proceeds of $1.6 million from equipment financings, and proceeds from the exercise of stock options of $1.1 million, offset by the payment of equity offering costs of $1.7 million, and repayments of equipment financing liabilities of $1.2$0.5 million.

Funding requirements

Our operating expenses are expected to increase substantially as we continue to advance our portfolio of programs.

Specifically, our expenses will increase if and as we:

initiate clinical trials of our product candidates, including our BEACON-101 trial;
continue our current research programs and our preclinical development of product candidates from our current research programs;
seek to identify additional research programs and additional product candidates;
initiate preclinical studies and clinical trials for anyadditional product candidates we identify and develop;
maintain, expand, enforce, defend, and protect our intellectual property portfolio and provide reimbursement of third-party expenses related to our patent portfolio;
seek marketing approvals for any of our product candidates that successfully complete clinical trials;

35


establish a sales, marketing, and distribution infrastructure to commercialize any medicines for which we may obtain marketing approval;approval';
further develop our base editing platform;
further develop delivery technology for our base editors, resulting fromincluding the LNP technology we acquired through our acquisition of Guide;
continue to hire additional personnel including research and development, clinical and commercial personnel;
add operational, financial, and management information systems and personnel, including personnel to support our product development;
acquire or in-license products, intellectual property, medicines and technologies; and
build, maintain, and operate a commercial-scale cGMP manufacturing facility.

We expect that our cash, cash equivalents and marketable securities at September 30, 2021March 31, 2022 will enable us to fund our current and planned operating expenses and capital expenditures for at least the next 12 months.twelve calendar months beginning March 31, 2022 and beyond such twelve-month period. We have based these estimates on assumptions that may prove to be imprecise, and we may exhaust our available capital resources sooner thanthat we currently expect. Because of the numerous risks and uncertainties associated with the development our programs, we are unable to estimate the amounts of increased capital outlays and operating expenses associated with completing the research and development of our product candidates.

Our future funding requirements will depend on many factors including:

the cost of continuing to build our base editing platform;
the costs of acquiring licenses for the delivery modalities that will be used with our product candidates;

34


the scope, progress, results, and costs of discovery, preclinical development, laboratory testing, manufacturing and clinical trials for the product candidates we may develop;
the costs of preparing, filing, and prosecuting patent applications, maintaining and enforcing our intellectual property and proprietary rights, and defending intellectual property-related claims;
the costs, timing, and outcome of regulatory review of the product candidates we may develop;
the costs of future activities, including product sales, medical affairs, marketing, manufacturing, distribution, coverage and reimbursement for any product candidates for which we receive regulatory approval;
the success of our license agreements and our collaborations;
our ability to establish and maintain additional collaborations on favorable terms, if at all;
the achievement of milestones or occurrence of other developments that trigger payments under any collaboration agreements we are a party to or may become a party to;
the payment of success liabilities to Harvard and Broad Institute pursuant to the respective terms of the Harvard License Agreement and the Broad Institute License Agreement, should we choose to pay in cash;
the extent to which we acquire or in-license products, intellectual property, and technologies;
the costs of obtaining, building, operating and expanding our manufacturing capacity; andand;
the impacts of the COVID-19 pandemic and our response to it.

A change in the outcome of any of these or other variables with respect to the development of any of our product candidates could significantly change the costs and timing associated with the development of that product candidate. Further, our operating plans may change in the future, and we may need additional funds to meet operational needs and capital requirements associated with such operating plans.

Until such time, if ever, as we can generate substantial revenues from the sale of our product candidates,revenues, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances, and licensing arrangements. We do not have any committed external source of funds.capital. We have historically relied on equity issuances to fund our capital needs and will likely rely on equity issuances in the future. Debt financing, if available, may involve agreements that include restrictive covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends. In addition, debt financings would result in increased fixed payment obligations.

If we raise fundscapital through additional collaborations, strategic alliances, or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs, or product candidates, or we may have to grant licenses on terms that may not be favorable to us. If we are unable to raise additional fundscapital through equity or debt financings when needed, we may be required to delay, limit, reduce, or terminate our product development or, if approved, future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves. We can give

36


no assurance that we will be able to secure such additional sources of funds to support our operations, or, if such funds are available to us, that such additional funding will be sufficient to meet our needs.

Contractual obligations

We enter into contracts in the normal course of business with contract research organizations and other vendors to assist in the performance of our research and development activities and other services and products for operating purposes. These contracts generally provide for termination on notice, and therefore are cancelable contracts and not included in the tableour calculations of contractual obligations and commitments. During the ninethree months ended September 30, 2021,March 31, 2022, there were no material changes to our contractual obligations and commitments described under Management’s Discussion and Analysis of Financial Condition and Results of Operations in the 20202021 Form 10-K with the exception of the commitments as described below.10-K.

During the nine months ended September 30, 2021, we entered into the second phase of our April 2019 lease for office and laboratory space to be built. The minimum of undiscounted lease payments due under the second phase of this lease is $42.7 million.

In May 2021, the first success payment measurements under the Harvard License Agreement and Broad License Agreement occurred and success payments to Harvard and Broad Institute were calculated to be $15.0 million and $15.0 million, respectively. We elected to make each payment in shares of our common stock and issued 174,825 shares of our common stock to each of Harvard and Broad Institute to settle these liabilities in June 2021.

Off-balance sheet arrangements

We did not have during the periods presented and we do not currently have any off-balance sheet arrangements, as defined under the applicable regulations of the SEC.

Item 3. Quantitative and Qualitative Disclosures About Market Risk.

We are exposed to market risk related to changes in interest rates. As of September 30, 2021,March 31, 2022, we had cash, cash equivalents, and marketable securities of $933.4 million,$1.2 billion, which consisted of cash, money market funds, commercial paper, and corporate notes U.S. Treasury securities and government securities.a corporate equity security. Our primary exposure to market risk is interest rate sensitivity, which is affected by changes in the general level of U.S. interest rates, particularly because our investments are in short-term marketable securities. Due to the short-term duration of our investment portfolio and the low risk profile of our investments, we believe an immediate 10% change in interest rates would not have a material effect on the fair market value of our investment portfolio. We have the ability to hold our investments until maturity, and therefore, we would not expect our operating results or cash flows to be affected to any significant degree by the effect of a change in market interest rates on our investment portfolio.

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We are not currently exposed to significant market risk related to changes in foreign currency exchange rates; however, we do contract with vendors that are located outside of the United States and may be subject to fluctuations in foreign currency exchange rates. We may enter into additional contracts with vendors located outside of the United States in the future, which may increase our foreign currency exchange risk.

Inflation generally affects us by increasing our cost of labor and research, manufacturing and development costs. We believe that inflation has not had a material effect on our financial statements included elsewhere in this Quarterly Report on Form 10-Q. However, our operations may be adversely affected by inflation in the future.

Item 4. Controls and Procedures.

Evaluation of Disclosure Controls and Procedures

Our management, with the participation of our principal executive officer and our principal financial officer, evaluated, as of the end of the period covered by this Quarterly Report on Form 10-Q, the effectiveness of our disclosure controls and procedures as defined in Rules 13a-15(e) and 15d-15(e) under the Exchange Act. The term “disclosure controls and procedures,” as defined in Rules 13a-15(e) and 15d-15(e) under the Exchange Act, means controls and other procedures of a company that are designed to ensure that information required to be disclosed by a company in the reports that it files or submits under the Exchange Act is recorded, processed, summarized and reported, within the time periods specified in the SEC’s rules and forms. Disclosure controls and procedures include, without limitation, controls and procedures designed to ensure that information required to be disclosed by a company in the reports that it files or submits under the Exchange Act is accumulated and communicated to the company’s management, including its principal executive and principal financial officers, or persons performing similar functions, as appropriate to allow timely decisions regarding required disclosure. Management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving their objectives and management necessarily applies its judgment in evaluating the cost-benefit relationship of possible controls and procedures.

Based on the evaluation of our disclosure controls and procedures as of September 30, 2021,March 31, 2022, our principal executive officer and principal financial officer concluded that our disclosure controls and procedures as of such date are effective at the reasonable assurance level.

Changes in Internal Control over Financial Reporting

We continuously seek to improve the efficiency and effectiveness of our internal controls. This results in refinements to processes throughout our company. There were no changes in our internal control over financial reporting (as defined in Rules 13a-15(f) and

37


15d-15(f) under the Exchange Act) during the quarter ended September 30, 2021March 31, 2022 that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.

3836


 

PART II. OTHER INFORMATION

We are not currently subject to any material legal proceedings.

Item 1A. Risk Factors.

Investing in our common stock involves a high degree of risk. For a detailed discussion of the risks that affect our business, pleasebusiness. Please refer to the sections titled “Risk Factors Summary” and “Item 1A. Risk Factors” in the 20202021 Form 10-K and “Item 1A. Risk Factors” in our Quarterly Report on Form 10-Q for the quarters ended March 31, 2021 and June 30, 2021.10-K. The COVID-19 pandemic may also have the effect of heightening many of the other risks described in the section titled “Item 1A. Risk Factors” in each of the 20202021 Form 10-K and our quarterly reports, such as risks related to our need to raise additional funding, fluctuation of our quarterly financial results, and our ability to obtain regulatory approvals for our product candidates.

The risk factors set forth below represent new risk factors or those containing changes to the similarly titled risk factor included in “Item 1.A Risk Factors” of the 20202021 Form 10-K.

If any of the product candidates we may develop, or the delivery modes we rely on to administer them, cause serious adverse events, undesirable side effects, or unexpected characteristics, such events, side effects or characteristics could delay or prevent regulatory approval of the product candidates, limit the commercial potential, or result in significant negative consequences following any potential marketing approval.

We have not evaluated any product candidates in human clinical trials. Moreover, there have been only a limited number of clinical trials involving the use of gene editing technologies and none involving base editing technology similar to our technology. It is impossible to predict when or if any product candidates we may develop will prove safe in humans. In the genetic medicine field, there have been several significant adverse events from gene therapy treatments in the past, including reported cases of leukemia, serious blood disorders and death. There can be no assurance that base editing technologies, or components of our product candidates or methods of delivery, will not cause undesirable side effects, as improper editing of a patient’s DNA and other effects could lead to lymphoma, leukemia, or other cancers, other serious conditions or syndromes or other aberrantly functioning cells.

A significant risk in any base editing product candidate is that “off-target” edits may occur, which could cause serious adverse events, undesirable side effects or unexpected characteristics. For example, Erwei Zuo et al. reported that cytosine base editors generated substantial off-target edits, that is, edits in unintended locations on the DNA, when tested in mouse embryos. Such unintended edits are referred to as “spurious deamination.” We cannot be certain that off-target editing will not occur in any of our planned or future clinical studies, and the lack of observed side effects in preclinical studies does not guarantee that such side effects will not occur in human clinical studies. We have developed assays that can detect off-target edits, even when such edits occur at very low frequencies. Using these assays, we have observed off-target edits in our base editing product candidates. As the sensitivity of these assays increases, it is possible that we will continue to detect more such off-target edits. While we do not believe that the off-target edits we have observed to date have had a material adverse impact on the safety or benefit of our product candidates, if, in the future, we detect off-target edits for a product candidate that negatively impact safety or efficacy, our ability to develop the product candidate as a therapeutic could be adversely affected. There is also the potential risk of delayed adverse events following exposure to base editing therapy due to the permanence of edits to DNA or due to other components of product candidates used to carry the genetic material. Further, because base editing makes a permanent change, the therapy cannot be withdrawn, even after a side effect is observed. In addition, Rees et al. and Grunewald et al. have reported that the deaminases we currently use in our C base editors and our A base editors for use in DNA base editing also cause unintended mutations in RNA for as long as the editor is present in the cell.

Although we and others have demonstrated the ability to engineer base editors to improve the specificity of their edits in a laboratory setting, we cannot be sure that our engineering efforts will be effective in any product candidates that we may develop. For example, we might not be able to engineer an editor to make the desired change or a by-stander edit could diminish the effectiveness of an edit that we make.

In certain rare DNA sequence contexts, where more than one edit occurs on a contiguous piece of DNA, the repair of two or more nicks may lead to a deletion. For example, in our BEAM-101 program, where we are simultaneously editing two positions in the promoters of the HBG2 and HBG1 genes, which share >99% sequence identity and are contiguous due to a gene duplication event, we observed a 5 kb deletion in HBG2 at single digit percentages in animal studies. We do not believe that such a deletion represents a safety or efficacy concern because healthy individuals, including those with hereditary persistence of fetal hemoglobin, with naturally-occurring deletions at this locus, including some as large as 13 kb, have been documented. However, if we were to observe deletions that have a negative effect on HbF upregulation or on other important cellular attributes, our ability to develop BEAM-101 as a therapeutic could be adversely affected.

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In certain of our programs, we plan to use LNPs to deliver our base editors. LNPs have been shown to induce oxidative stress in the liver at certain doses, as well as initiate systemic inflammatory responses that can be fatal in some cases. While we aim to continue to optimize our LNPs, there can be no assurance that our LNPs will not have undesired effects. Our LNPs could contribute, in whole or in part, to one or more of the following: immune reactions; infusion reactions; complement reactions; opsonization reactions; antibody reactions including IgA, IgM, IgE or IgG or some combination thereof; or reactions to the PEG from some lipids or PEG otherwise associated with the LNP. Certain aspects of our investigational medicines may induce immune reactions from either the mRNA or the lipid as well as adverse reactions within liver pathways or degradation of the mRNA or the LNP, any of which could lead to significant adverse events in one or more of our future clinical trials. Many of these types of side effects have been seen for legacy LNPs. There may be uncertainty as to the underlying cause of any such adverse event, which would make it difficult to accurately predict side effects in future clinical trials and would result in significant delays in our programs.

Our viral vectors including AAV or lentiviruses, which are relatively new approaches used for disease treatment, also have known side effects, and for which additional risks could develop in the future. In past clinical trials that were conducted by others with non-AAV vectors, several significant side effects were caused by gene therapy treatments, including reported cases of leukemia and death. Other potential side effects could include an immunologic reaction and insertional oncogenesis, which is the process whereby the insertion of a functional gene near a gene that is important in cell growth or division results in uncontrolled cell division, which could potentially enhance the risk of malignant transformation. If the vectors we use demonstrate a similar side effect, or other adverse events, we may be required to halt or delay further clinical development of any potential product candidates. Furthermore, the FDA has stated that lentiviral vectors possess characteristics that may pose high risks of delayed adverse events. Such delayed adverse events may occur in other viral vectors, including AAV vectors, at a lower rate.

In addition to side effects and adverse events caused by our product candidates, the conditioning administration process or related procedures which may be used in our electroporation pipeline also can cause adverse side effects and adverse events. Additionally, we have and may continue to collaborate with third parties to develop alternative conditioning regimes. We cannot predict if alternative conditioning regimes will be compatible with our product candidates. If in the future we are unable to demonstrate that such adverse events were caused by the conditioning regimens used, or administration process or related procedure, the FDA, the European Commission, EMA or other regulatory authorities could order us to cease further development of, or deny or limit approval of, our product candidates for any or all target indications. Even if we are able to demonstrate that adverse events are not related to the drug product or the administration of such drug product, such occurrences could affect patient recruitment, the ability of enrolled patients to complete the clinical trial, or the commercial viability of any product candidates that obtain regulatory approval.

If any product candidates we develop are associated with serious adverse events, undesirable side effects, or unexpected characteristics, we may need to abandon their development or limit development to certain uses or subpopulations in which the serious adverse events, undesirable side effects or other characteristics are less prevalent, less severe, or more acceptable from a risk-benefit perspective, any of which would have a material adverse effect on our business, financial condition, results of operations, and prospects. Many product candidates that initially showed promise in early stage testing for treating cancer or other diseases have later been found to cause side effects that prevented further clinical development of the product candidates.

If in the future we are unable to demonstrate that any of the above adverse events were caused by factors other than our product candidate, the FDA, the EMA or other regulatory authorities could order us to cease further development of, or deny approval of, any product candidates we are able to develop for any or all targeted indications. Even if we are able to demonstrate that all future serious adverse events are not product-related, such occurrences could affect patient recruitment or the ability of enrolled patients to complete the trial. Moreover, if we elect, or are required, to delay, suspend or terminate any clinical trial of any product candidate we may develop, the commercial prospects of such product candidates may be harmed and our ability to generate product revenues from any of these product candidates may be delayed or eliminated. Any of these occurrences may harm our ability to identify and develop product candidates, and may harm our business, financial condition, result of operations, and prospects significantly.

Additionally, if we successfully develop a product candidate and it receives marketing approval, the FDA could require us to adopt a Risk Evaluation and Mitigation Strategy, or REMS, to ensure that the benefits of treatment with such product candidate outweighs the risks for each potential patient, which may include, among other things, a medication guide outlining the risks of the product for distribution to patients, a communication plan to health care practitioners, extensive patient monitoring, or distribution systems and processes that are highly controlled, restrictive, and more costly than what is typical for the industry. Furthermore, if we or others later identify undesirable side effects caused by any product candidate that we develop, several potentially significant negative consequences could result, including:

regulatory authorities may suspend or withdraw approvals of such product candidate;
regulatory authorities may require additional warnings on the label or limit the approved use of such product candidate;
we may be required to conduct additional clinical trials;

40


we could be sued and held liable for harm caused to patients; and
our reputation may suffer.

Any of these events could prevent us from achieving or maintaining market acceptance of any product candidates we may identify and develop and could have a material adverse effect on our business, financial condition, and results of operations.

Our owned patents and patent applications and in-licensed patents and patent applications and other intellectual property may be subject to priority disputes or to inventorship disputes and similar proceedings. If we or our licensors are unsuccessful in any of these proceedings, we may be required to obtain licenses from third parties, which may not be available on commercially reasonable terms or at all, or to cease the development, manufacture, and commercialization of one or more of the product candidates we may develop, which could have a material adverse impact on our business.

Although we have an option to exclusively license certain patents and patent applications directed to Cas9 and Cas12a from Editas, who in turn has licensed such patents from various academic institutions including the Broad Institute, we do not currently have a license to such patents and patent applications. Certain of the U.S. patents and one U.S. patent application to which we hold an option are co-owned by the Broad Institute and MIT, and in some cases co-owned by the Broad Institute, Massachusetts Institute of Technology, or MIT, and Harvard, which we refer to together as the Boston Licensing Parties, and were involved in U.S. interference No. 106,048 with one U.S. patent application co-owned by the University of California, the University of Vienna, and Emmanuelle Charpentier, which we refer to together as the University of California. On September 10, 2018, the Court of Appeals for the Federal Circuit, or the CAFC, affirmed the Patent Trial and Appeal Board of the USPTO’s, or PTAB’s, holding that there was no interference-in-fact. An interference is a proceeding within the USPTO to determine priority of invention of the subject matter of patent claims filed by different parties.

On June 24, 2019, the PTAB declared an interference (U.S. Interference No. 106,115) between 10ten U.S. patent applications ((U.S. Serial Nos. 15/947,680; 15/947,700; 15/947,718; 15/981,807; 15/981,808; 15/981,809; 16/136,159; 16/136,165; 16/136,168; and 16/136,175) that are co-owned by the University of California, and 13 U.S. patents and one U.S. patent application (U.S. Patent Nos. 8,697,359; 8,771,945; 8,795,965; 8,865,406; 8,871,445; 8,889,356; 8,895,308; 8,906,616; 8,932,814; 8,945,839; 8,993,233; 8,999,641; and 9,840,713, and U.S. Serial No. 14/704,551)) that are co-owned by the Boston Licensing Parties, which we have an option to under the Editas License Agreement. In the declared interference, the University of California has been designated as the junior party and the Boston Licensing Parties have been designated as the senior party.

As a result of the declaration of interference, an adversarial proceeding in the USPTO before the PTAB has been initiated, which is declared to ultimately determine priority, specifically and which party was first to invent the claimed subject matter. Following oral arguments on the parties’ motions in May 2020, the PTAB issued a decision in September 2020, which included, in part, denying the Boston Licensing Parties motion that the University of California should be estopped in the current proceeding by the PTAB’s decision in the prior interference proceeding between the parties (No. 106,048), finding that the Boston Licensing Parties remain the senior party in the proceeding, and holding that the interference will proceed to the second, priority phase. An interference is typically divided into two phases. The first phase is referred to as the motions or preliminary motions phase while the second is referred to as the priority phase. In the first phase, each party may raise issues including but not limited to those relating to the patentability of a party’s claims based on prior art, written description, and enablement. A party also may seek an earlier priority benefit or may challenge whether the declaration of interference was proper in the first place. Priority, or a determination of who first invented the commonly claimed invention, is determined in the second phase of an interference. Although we cannot predict with any certainty how long the priority phase will actually take, it may take approximately a year or longer before a decision is made by the PTAB.

The 10ten University of California patent applications and the 13 U.S. patents and one U.S. patent application co-owned by the Boston Licensing Parties involved in U.S. Interference No. 106,115 generally relate to CRISPR/Cas9 systems or eukaryotic cells comprising CRISPR/Cas9 systems having fused or covalently linked RNA and the use thereof in eukaryotic cells. On February 28, 2022, the PTAB issued a decision that the Boston Licensing Parties have priority of invention over University of California with respect to a single RNA CRISPR-Cas9 system that functions in eukaryotic cells. This decision is being appealed.

There can be no assurance that the U.S. interference will be resolved in favor of the Boston Licensing Parties.Parties on appeal. If the U.S. interference resolves in favor of University of California, or if the Boston Licensing Parties’ patents and patent application are narrowed, invalidated, or held unenforceable, we may lose the ability to license the optioned patents and patent application and our ability to commercialize our product candidates may be adversely affected if we cannot obtain a license to relevant third party patents that cover our product candidates. We may not be able to obtain any required license on commercially reasonable terms or at all. Even if we were able to obtain a license, it could be nonexclusive, thereby giving our competitors and other third parties access to the same technologies licensed to us, and it could require us to make substantial licensing and royalty payments. If we are unable to obtain a

37


necessary license to a third-party patent on commercially reasonable terms, we may be unable to commercialize our base editing platform technology or product candidates or such commercialization efforts may be significantly delayed, which could in turn significantly harm our business.

We or our licensors may be subject to similar interferences in the future with the same risks as described above. For example, on December 14, 2020, the PTAB declared an interference (U.S. Interference No. 106,126) between 14 U.S. patents and two U.S. patent applications (U.S. Patent Nos. 8,697,359; 8,771,945; 8,795,965; 8,865,406; 8,871,445; 8,889,356; 8,889,418; 8,895,308; 8,906,616; 8,932,814; 8,945,839; 8,993,233; 8,999,641; and 9,840,713, and U.S. Serial Nos. 14/704,551 and 15/330,876) that are co-owned by the Boston Licensing Parties, which we have an option to under the Editas License Agreement, and one U.S. patent application (U.S. Serial Nos.No. 14/685,510) that is owned by Toolgen, Inc, or Toolgen. In the declared interference, the Boston Licensing Parties have

41


been designated as the junior party and Toolgen has been designated as the senior party. In March 2021, the PTAB issued an order on preliminary motions, granting, in part, and denying, in part, certain motions proposed by the Boston Licensing Parties and Toolgen. Although we cannot predict with any certainty how long the preliminary motions phase will actually take, it may take approximately a year or longer before a decision on the motions is made by the PTAB. The 14 U.S. patents and two U.S. patent applications co-owned by the Boston Licensing Parties involved in U.S. Interference No. 106,126 generally relate to CRISPR/Cas9 systems or eukaryotic cells comprising CRISPR/Cas9 systems having fused or covalently linked RNA and the use thereof in eukaryotic cells.

On June 21, 2021, the PTAB declared an interference (U.S. Interference No. 106,133) between the same 14 U.S. patents and two U.S. patent applications (U.S. Patent Nos. 8,697,359; 8,771,945; 8,795,965; 8,865,406; 8,871,445; 8,889,356; 8,889,418; 8,895,308; 8,906,616; 8,932,814; 8,945,839; 8,993,233; 8,999,641; and 9,840,713, and U.S. Serial Nos. 14/704,551 and 15/330,876, co-owned by the Boston Licensing Parties) as named in the interference with Toolgen, and one U.S. patent application (U.S. Serial Nos.No. 15/456,204) that is owned by Sigma-Aldrich Co., LLC, or Sigma-Aldrich. In the declared interference, the Boston Licensing Parties have been designated as the junior party and Sigma-Aldrich has been designated as the senior party. In September 2021, the PTAB issued an order on preliminary motions, granting, deferring, dismissing, or denying, certain motions proposed by the Boston Licensing Parties and Sigma-Aldrich. Although we cannot predict with any certainty how long the preliminary motions phase will actually take, it may take approximately a year or longer before a decision on the motions is made by the PTAB.

We or our licensors may also be subject to claims that former employees, collaborators, or other third parties have an interest in our owned patents or patent applications or in-licensed patents or patent applications or other intellectual property as an inventor or co-inventor. If we are unable to obtain an exclusive license to any such third-party co-owners’ interest in such patents or patent applications, such co-owners may be able to license their rights to other third parties, including our competitors. In addition, we may need the cooperation of any such co-owners to enforce any patents that issue from such patent applications against third parties, and such cooperation may not be provided to us.

If we or our licensors are unsuccessful in any interference proceedings or other priority, validity (including any patent oppositions), or inventorship disputes to which we or they are subject, we may lose valuable intellectual property rights through the loss of one or more of our owned, licensed, or optioned patents, or such patent claims may be narrowed, invalidated, or held unenforceable, or through loss of exclusive ownership of or the exclusive right to use our owned or in-licensed patents. In the event of loss of patent rights as a result of any of these disputes, we may be required to obtain and maintain licenses from third parties, including parties involved in any such interference proceedings or other priority or inventorship disputes. Such licenses may not be available on commercially reasonable terms or at all, or may be non-exclusive. If we are unable to obtain and maintain such licenses, we may need to cease the development, manufacture, and commercialization of one or more of the product candidates we may develop. The loss of exclusivity or the narrowing of our patent claims could limit our ability to stop others from using or commercializing similar or identical technology and product candidates. Even if we or our licensors are successful in an interference proceeding or other similar priority or inventorship disputes, it could result in substantial costs and be a distraction to management and other employees. Any of the foregoing could result in a material adverse effect on our business, financial condition, results of operations, or prospects.

Item 2. Unregistered SalesThe intellectual property landscape around gene editing technology, including base editing and delivery technology, is highly dynamic, and third parties may initiate legal proceedings alleging that we are infringing, misappropriating, or otherwise violating their intellectual property rights, the outcome of Equity Securitieswhich would be uncertain and Uses of Proceedsmay prevent, delay or otherwise interfere with our product discovery and development efforts.

Unregistered salesThe field of equity securities

We did not sell any unregistered securitiesgene editing, especially in the three months ended September 30, 2021.

Usearea of proceeds from registered securities

In February 2020, we closed our IPObase editing technology, is still in which we issuedits infancy, and sold 12,176,471 shares of our common stock, including 1,588,235 shares of common stock sold pursuantno such product candidates have reached the market. Due to the underwriters’ full exercise of their option to purchase additional shares, at a public offering price of $17.00 per share, for aggregate gross proceeds of $207.0 million. We received approximately $188.3 millionintense research and development that is taking place by several companies, including us and our competitors, in net proceeds after deducting underwriting discountsthis field and offering expenses payable by us. None of the underwriting discounts and commissions or offering expenses were paid directly or indirectly to any directors or officers of ours or their associates or to persons owning 10% or more of any class of equity securities or to any affiliates of ours. All of the shares issued and sold in the IPO were registered underfield of delivery technology, the Securities Act pursuantintellectual property landscape is evolving and in flux, and it may remain uncertain for the coming years. There may be significant intellectual property related litigation and proceedings relating to a Registration Statement on Form S-1 (File No. 333-233985), which was declared effective byour owned and in-licensed, and other third party, intellectual property and proprietary rights in the SEC on February 5, 2020, and a Registration Statement on Form S-1 MEF (File No. 333-236284) filed pursuant to Rule 462(b) of the Securities Act. The offering commenced on February 5, 2020 and did not terminate until the sale of all the shares offered.future.

Our commercial success depends upon our ability and the ability of our collaborators and licensors to develop, manufacture, market, and sell any product candidates that we may develop and use our proprietary technologies without infringing, misappropriating, or otherwise violating the intellectual property and proprietary rights of third parties. The biotechnology and pharmaceutical industries are characterized by extensive litigation regarding patents and other intellectual property rights as well as administrative proceedings for challenging patents, including interference, derivation, inter partes review, post grant review, and reexamination proceedings

38


before the USPTO or oppositions and other comparable proceedings in foreign jurisdictions. We may be subject to and may in the future become party to, or threatened with, adversarial proceedings or litigation regarding intellectual property rights with respect to our base editing platform technology, delivery platform technology and any product candidates we may develop, including interference proceedings, post-grant review, inter partes review, and derivation proceedings before the USPTO and similar proceedings in foreign jurisdictions such as oppositions before the EPO. Numerous U.S. and foreign issued patents and pending patent applications that are owned by third parties exist in the fields in which we are developing our product candidates and they may assert infringement claims against us based on existing patents or patents that may be granted in the future, regardless of their merit.

As the biotechnology and pharmaceutical industries expand and more patents are issued, the risk increases that our base editing platform technology, delivery platform technology and product candidates may give rise to claims of infringement of the patent rights of others. Moreover, it is not always clear to industry participants, including us, which patents cover various types of therapies, products or their methods of use or manufacture. We are aware of certain third-party patents and patent applications that, if issued, may be construed to cover our base editing technology, delivery technology and product candidates. There may also be third-party patents of which we are currently unaware with claims to technologies, methods of manufacture or methods for treatment related to the use or manufacture of our product candidates. Because patent applications can take many years to issue, there may be currently pending patent applications that may later result in issued patents that our product candidates may infringe. In addition, third parties may obtain patents in the future and claim that use of our technologies infringes upon these patents.

Numerous third-party U.S. and foreign issued patents and pending patent applications exist in the net offering proceeds throughfields in which we are developing product candidates. Our product candidates make use of CRISPR-based technology, which is a field that is highly active for patent filings. The extensive patent filings related to CRISPR and Cas make it difficult for us to assess the full extent of relevant patents and pending applications that may cover our base editing platform technology and product candidates and their use or manufacture. There may be third-party patents or patent applications with claims to materials, formulations, methods of manufacture or methods for treatment related to the use or manufacture of our base editing platform technology and product candidates. For example, we are aware of a patent portfolio that is co-owned by the University of California, University of Vienna and Emmanuelle Charpentier, or the University of California Portfolio, which contains multiple patents and pending applications directed to gene editing. The University of California portfolio includes, for example, U.S. Patent Nos. 10,266,850; 10,227,611; 10,000,772; 10,113,167; 10,301,651; 10,308,961; 10,337,029; 10,351,878; 10,407,697; 10,358,659; 10,358,658; 10,385,360; 10,400,253; 10,421,980; 10,415,061; 10,428,352; 10,443,076; 10,487,341; 10,513,712; 10,519,467; 10,526,619; 10,533,190; 10,550,407; 10,563,227; 10,570,419; 10,577,631; 10,597,680; 10,612,045; 10,626,419; 10,640,791; 10,669,560; 10,676,759; 10,752,920; 10,774,344; 10,793,878; 10,900,054; 10,982,230; 10,982,231; 10,988,780; 10,988,782; 11,001,863; 11,008,589; 11,008,590; 11,028,412; 11,186,849; 11,242,543; 11,274,318; 11,293,034, which are expected to expire around March 2033, excluding any additional term for patent term adjustment, or PTA, or patent term extension, or PTE, and any disclaimed term for terminal disclaimers. The University of California portfolio also includes numerous additional pending patent applications. If these patent applications issue as patents, they are expected to expire around March 2033, excluding any PTA, PTE, and any disclaimed term for terminal disclaimers. As discussed above, certain applications in the University of California Portfolio are currently subject to U.S. Interference No. 106,115 with certain U.S. patents and one U.S. patent application that are co-owned by the Boston Licensing Parties to which we have an option under the Editas License Agreement. Although we have an option to exclusively license certain patents and patent applications directed to Cas9 and Cas12a from Editas, who in turn has licensed such patents from various academic institutions including Broad Institute, we do not currently have a license to such patents and patent applications. Certain members of the University of California Portfolio are being opposed in Europe by multiple parties. For example, the EPO Opposition Division has initiated opposition proceedings against European Patent Nos. EP2,800,811 B1, and EP3,241,902 B1and EP3401400 B1, which are estimated to expire in March 2033 (excluding any patent term adjustments or extensions). The opposition procedure before the EPO allows one or more third parties to challenge the validity of a granted European patent within nine months after grant date of the European patent. Opposition proceedings may involve issues including, but not limited to, priority, patentability of the claims involved, and procedural formalities related to the filing of the patent application. As a result of the opposition proceedings, the Opposition Division can revoke a patent, maintain the patent as granted, or maintain the patent in an amended form. Most of the claims of European patent EP 2,800,811 B1 were maintained without amendment by the Opposition Division, but this Quarterly Reportdecision is being appealed. In April 2021, the claims of European patent EP3,241,902 B1 were revoked in their entirety, and that decision is not being appealed. In February 2022, the claims of European patent EP3,401,400 B1 were maintained in amended form by the Opposition Division, and it is uncertain if this decision will be appealed. If these patents are maintained by the Opposition Division with claims similar to those that are currently opposed, our ability to commercialize our product candidates may be adversely affected if we do not obtain a license to these patents. We may not be able to obtain any required license on Form 10-Q, is consistent withcommercially reasonable terms or at all. Even if we were able to obtain a license, it could be nonexclusive, thereby giving our competitors and other third parties access to the usesame technologies licensed to us, and it could require us to make substantial licensing and royalty payments. If we are unable to obtain a necessary license to a third-party patent on commercially reasonable terms, we may be unable to commercialize our base editing platform technology or product candidates or such commercialization efforts may be significantly delayed, which could in turn significantly harm our business.

Numerous other patents and patent applications have been filed by other third parties directed to gene editing, guide nucleic acids, PAM sequence variants, split inteins, Cas12b or gene editing in the context of proceeds describedimmune therapy or chimeric antigen receptors.

39


Because of the large number of patents issued and patent applications filed in our final prospectus filed withfield, third parties may allege they have patent rights encompassing our product candidates, technologies or methods. Third parties may assert that we are employing their proprietary technology without authorization and may file patent infringement claims or lawsuit against us, and if we are found to infringe such third-party patents, we may be required to pay damages, cease commercialization of the SEC pursuantinfringing technology, or obtain a license from such third parties, which may not be available on commercially reasonable terms or at all.

Our ability to Rule 424(b)(4)commercialize our product candidates in the United States and abroad may be adversely affected if we cannot obtain a license on February 7, 2020, andcommercially reasonable terms to relevant third-party patents that cover our product candidates, delivery platform technology or base editing platform technology. Even if we believe third-party intellectual property claims are without merit, there has beenis no material changeassurance that a court would find in our planned usefavor on questions of infringement, validity, enforceability, or priority. A court of competent jurisdiction could hold that these third-party patents are valid, enforceable, and infringed, which could materially and adversely affect our ability to commercialize any product candidates we may develop and any other product candidates or technologies covered by the asserted third-party patents. In order to successfully challenge the validity of any such U.S. patent in federal court, we would need to overcome a presumption of validity. As this burden is a high one requiring us to present clear and convincing evidence as to the invalidity of any such U.S. patent claim, there is no assurance that a court of competent jurisdiction would invalidate the claims of any such U.S. patent. If we are found to infringe a third party’s intellectual property rights, and we are unsuccessful in demonstrating that such patents are invalid or unenforceable, we could be required to obtain a license from such third party to continue developing, manufacturing, and marketing any product candidates we may develop and our technology. However, we may not be able to obtain any required license on commercially reasonable terms or at all. Even if we were able to obtain a license, it could be non-exclusive, thereby giving our competitors and other third parties access to the same technologies licensed to us, and it could require us to make substantial licensing and royalty payments. If we are unable to obtain a necessary license to a third-party patent on commercially reasonable terms, we may be unable to commercialize our base editing platform technology, delivery platform technology or product candidates or such commercialization efforts may be significantly delayed, which could in turn significantly harm our business. We also could be forced, including by court order, to cease developing, manufacturing, and commercializing the infringing technology or product candidates. In addition, we could be found liable for significant monetary damages, including treble damages and attorneys’ fees, if we are found to have willfully infringed a patent or other intellectual property right. Claims that we have misappropriated the confidential information or trade secrets of third parties could have a similar material adverse effect on our business, financial condition, results of operations, and prospects.

Defense of third-party claims of infringement of misappropriation, or violation of intellectual property rights involves substantial litigation expense and would be a substantial diversion of management and employee time and resources from our business. Some third parties may be able to sustain the costs of complex patent litigation more effectively than we can because they have substantially greater resources. In addition, any uncertainties resulting from the initiation and continuation of any litigation could have a material adverse effect on our ability to raise the funds necessary to continue our operations or could otherwise have a material adverse effect on our business, financial condition, results of operations and prospects. There could also be public announcements of the balanceresults of hearings, motions, or other interim proceedings or developments, and if securities analysts or investors perceive these results to be negative, it could have a substantial adverse effect on the price of our common stock. Any of the net proceeds fromforegoing events could have a material adverse effect on our IPO described in such prospectus.business, financial condition, results of operations and prospects.

4240


 

Item 6. Exhibits.

 

 

 

If Incorporated by Reference

 

 

 

 

If Incorporated by Reference

 

Exhibit

Number

 

Description of Exhibit

 

 

Form

 

File

Number

 

Date of

Filing

 

Exhibit

Number

 

Filed

Herewith

 

Description of Exhibit

 

 

Form

 

File

Number

 

Date of

Filing

 

Exhibit

Number

 

Filed

Herewith

 

 

 

 

3.1

 

Fourth Amended Certificate of Incorporation of Beam Therapeutics Inc.

 

8-K

 

001-39208

 

02/11/2020

 

3.1

 

 

Fourth Amended Certificate of Incorporation of Beam Therapeutics Inc.

 

8-K

 

001-39208

 

02/11/2020

 

3.1

 

 

 

 

 

 

 

3.2

 

Amended and Restated By-laws of Beam Therapeutics Inc.

 

8-K

 

001-39208

 

02/11/2020

 

3.2

 

 

Amended and Restated By-laws of Beam Therapeutics Inc.

 

8-K

 

001-39208

 

02/11/2020

 

3.2

 

 

 

 

4.1

 

Form of Purchase Agreement, dated as of January 16, 2021, among Beam Therapeutics Inc. and each purchaser party thereto.

 

8-K

 

001-39208

 

01/19/2021

 

10.1

 

 

 

 

 

 

 

10.1

 

Amendment No. 1 to Sales Agreement, dated July 7, 2021, by and between Beam Therapeutics Inc. and Jefferies LLC.

 

8-K

 

001-39208

 

07/07/2021

 

1.1

 

 

Amended and Restated Non-employee Director Compensation Policy

 

X

 

 

 

 

 

 

31.1

 

Certification of Principal Executive Officer Pursuant to Rules 13a-14(a) and 15d-14(a) under the Securities Exchange Act of 1934, as Adopted Pursuant to Section 302 of the Sarbanes-Oxley Act of 2002.

 

X

 

Certification of Principal Executive Officer Pursuant to Rules 13a-14(a) and 15d-14(a) under the Securities Exchange Act of 1934, as Adopted Pursuant to Section 302 of the Sarbanes-Oxley Act of 2002.

 

X

 

 

 

 

 

 

31.2

 

Certification of Principal Financial Officer Pursuant to Rules 13a-14(a) and 15d-14(a) under the Securities Exchange Act of 1934, as Adopted Pursuant to Section 302 of the Sarbanes-Oxley Act of 2002.

 

X

 

Certification of Principal Financial Officer Pursuant to Rules 13a-14(a) and 15d-14(a) under the Securities Exchange Act of 1934, as Adopted Pursuant to Section 302 of the Sarbanes-Oxley Act of 2002.

 

X

 

 

 

 

 

 

32.1

 

Certification of Principal Executive Officer Pursuant to 18 U.S.C. Section 1350, as Adopted Pursuant to Section 906 of the Sarbanes-Oxley Act of 2002.

 

X

 

Certification of Principal Executive Officer Pursuant to 18 U.S.C. Section 1350, as Adopted Pursuant to Section 906 of the Sarbanes-Oxley Act of 2002.

 

X

 

 

 

 

 

 

32.2

 

Certification of Principal Financial Officer Pursuant to 18 U.S.C. Section 1350, as Adopted Pursuant to Section 906 of the Sarbanes-Oxley Act of 2002.

 

X

 

Certification of Principal Financial Officer Pursuant to 18 U.S.C. Section 1350, as Adopted Pursuant to Section 906 of the Sarbanes-Oxley Act of 2002.

 

X

 

 

 

 

 

 

101.INS

 

XBRL Instance Document - the instance document does not appear in the Interactive Data File because its XBRL tags are embedded within the Inline XBRL document

 

X

 

XBRL Instance Document - the instance document does not appear in the Interactive Data File because its XBRL tags are embedded within the Inline XBRL document

 

X

 

 

 

 

 

 

101.SCH

 

Inline XBRL Taxonomy Extension Schema Document

 

X

 

Inline XBRL Taxonomy Extension Schema Document

 

X

 

 

 

 

 

 

101.CAL

 

Inline XBRL Taxonomy Calculation Linkbase Document

 

X

 

Inline XBRL Taxonomy Calculation Linkbase Document

 

X

 

 

 

 

 

 

101.DEF

 

Inline XBRL Taxonomy Extension Definition Linkbase Document

 

X

 

Inline XBRL Taxonomy Extension Definition Linkbase Document

 

X

 

 

 

 

 

 

101.LAB

 

Inline XBRL Taxonomy Label Linkbase Document

 

X

 

Inline XBRL Taxonomy Label Linkbase Document

 

X

 

 

 

 

 

 

101.PRE

 

Inline XBRL Taxonomy Presentation Linkbase Document

 

X

 

Inline XBRL Taxonomy Presentation Linkbase Document

 

X

 

 

 

 

 

 

104

 

Cover Page Interactive Data File (embedded within the Inline XBRL document)

 

X

 

Cover Page Interactive Data File (embedded within the Inline XBRL document)

 

X

 

4341


 

SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.

 

 

BEAM THERAPEUTICS INC.

 

 

 

 

Date: November 8, 2021May 9, 2022

 

By:

/s/ John Evans

 

 

 

John Evans

 

 

 

Chief Executive Officer

 

 

 

(Principal executive officer)

 

Date: November 8, 2021May 9, 2022

 

By:

/s/ Terry-Ann Burrell

 

 

 

Terry-Ann Burrell

 

 

 

Chief Financial Officer and Treasurer

 

 

 

(Principal financial and accounting officer)

 

 

4442