Document and Entity Information
Document and Entity Information - shares | 3 Months Ended | |
Mar. 31, 2021 | Apr. 30, 2021 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Mar. 31, 2021 | |
Document Fiscal Year Focus | 2021 | |
Document Fiscal Period Focus | Q1 | |
Trading Symbol | NTLA | |
Entity Registrant Name | INTELLIA THERAPEUTICS, INC. | |
Entity Central Index Key | 0001652130 | |
Current Fiscal Year End Date | --12-31 | |
Entity Filer Category | Large Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Common Stock, Shares Outstanding | 68,153,597 | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Shell Company | false | |
Entity File Number | 001-37766 | |
Entity Tax Identification Number | 36-4785571 | |
Entity Address, Address Line One | 40 Erie Street | |
Entity Address, Address Line Two | Suite 130 | |
Entity Address, City or Town | Cambridge | |
Entity Address, State or Province | MA | |
Entity Address, Postal Zip Code | 02139 | |
City Area Code | 857 | |
Local Phone Number | 285-6200 | |
Entity Incorporation, State or Country Code | DE | |
Title of 12(b) Security | Common Stock, par value $0.0001 per share | |
Security Exchange Name | NASDAQ | |
Document Quarterly Report | true | |
Document Transition Report | false |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets (Unaudited) - USD ($) $ in Thousands | Mar. 31, 2021 | Dec. 31, 2020 |
Current Assets: | ||
Cash and cash equivalents | $ 141,538 | $ 160,020 |
Marketable securities | 442,525 | 437,351 |
Accounts receivable | 953 | 2,130 |
Prepaid expenses and other current assets | 16,068 | 17,016 |
Total current assets | 601,084 | 616,517 |
Marketable securities - noncurrent | 16,735 | |
Property and equipment, net | 16,157 | 15,943 |
Operating lease right-of-use assets | 77,912 | 39,114 |
Other assets | 5,003 | 4,748 |
Total Assets | 716,891 | 676,322 |
Current Liabilities: | ||
Accounts payable | 8,688 | 10,460 |
Accrued expenses | 21,215 | 25,554 |
Current portion of operating lease liability | 8,255 | 5,696 |
Current portion of deferred revenue | 22,544 | 22,544 |
Total current liabilities | 60,702 | 64,254 |
Deferred revenue, net of current portion | 45,829 | 51,387 |
Long-term operating lease liability | 64,487 | 33,609 |
Commitments and contingencies (Note 6) | ||
Stockholders’ Equity: | ||
Common stock, $0.0001 par value; 120,000,000 shares authorized; 67,890,334 and 66,234,056 shares issued and outstanding at March 31, 2021 and December 31, 2020, respectively | 7 | 7 |
Additional paid-in capital | 1,027,192 | 962,173 |
Accumulated other comprehensive (loss) income | (12) | 1 |
Accumulated deficit | (481,314) | (435,109) |
Total stockholders’ equity | 545,873 | 527,072 |
Total Liabilities and Stockholders’ Equity | $ 716,891 | $ 676,322 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Unaudited) (Parenthetical) - $ / shares | Mar. 31, 2021 | Dec. 31, 2020 |
Statement Of Financial Position [Abstract] | ||
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 120,000,000 | 120,000,000 |
Common stock, shares issued | 67,890,334 | 66,234,056 |
Common stock, shares outstanding | 67,890,334 | 66,234,056 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations and Comprehensive Loss (Unaudited) - USD ($) shares in Thousands, $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Income Statement [Abstract] | ||
Collaboration revenue | $ 6,445 | $ 12,916 |
Type of Revenue [Extensible List] | us-gaap:LicenseAndServiceMember | us-gaap:LicenseAndServiceMember |
Operating expenses: | ||
Research and development | $ 39,276 | $ 34,650 |
General and administrative | 13,594 | 11,314 |
Total operating expenses | 52,870 | 45,964 |
Operating loss | (46,425) | (33,048) |
Interest income | 220 | 1,242 |
Net loss | $ (46,205) | $ (31,806) |
Net loss per share, basic and diluted | $ (0.69) | $ (0.63) |
Weighted average shares outstanding, basic and diluted | 67,183 | 50,491 |
Other comprehensive (loss) income: | ||
Unrealized (loss) gain on marketable securities | $ (13) | $ 112 |
Comprehensive loss | $ (46,218) | $ (31,694) |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Cash Flows (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | 71 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | Mar. 31, 2021 | |
CASH FLOWS FROM OPERATING ACTIVITIES: | |||
Net loss | $ (46,205) | $ (31,806) | |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Depreciation and amortization | 1,591 | 1,541 | |
Equity-based compensation | 6,424 | 4,157 | |
Amortization/accretion of investment premiums/discounts | 1,209 | (200) | |
Changes in operating assets and liabilities: | |||
Accounts receivable | 1,177 | (8,748) | |
Prepaid expenses and other current assets | (5,448) | (448) | |
Operating right-of-use assets | 1,596 | 1,566 | |
Other assets | (255) | 97 | |
Accounts payable | (917) | (370) | |
Accrued expenses | (4,588) | 177 | |
Deferred revenue | (5,558) | (3,151) | |
Operating lease liabilities | (1,402) | (1,360) | |
Net cash used in operating activities | (52,376) | (38,545) | |
CASH FLOWS FROM INVESTING ACTIVITIES: | |||
Purchases of property and equipment | (2,412) | (845) | |
Purchases of marketable securities | (148,330) | (31,207) | |
Maturities of marketable securities | 125,200 | 89,500 | |
Net cash (used in) provided by investing activities | (25,542) | 57,448 | |
CASH FLOWS FROM FINANCING ACTIVITIES: | |||
Proceeds from issuance of common stock through at-the-market offerings, net of issuance costs | $ 1,165,300 | ||
Proceeds from options exercised | 13,340 | 336 | |
Net cash provided by financing activities | 58,595 | 4,864 | |
Net (decrease) increase in cash and cash equivalents and restricted cash and cash equivalents | (19,323) | 23,767 | |
Cash and cash equivalents and restricted cash and cash equivalents, beginning of period | 164,606 | 57,226 | |
Cash and cash equivalents and restricted cash and cash equivalents, end of period | 145,283 | 80,993 | 145,283 |
Reconciliation of cash and cash equivalents and restricted cash and cash equivalents to condensed consolidated balance sheet: | |||
Cash and cash equivalents | 141,538 | 80,993 | 141,538 |
Restricted cash and cash equivalents, included in prepaids and other current assets and other assets | 3,745 | 3,745 | |
Cash and cash equivalents and restricted cash and cash equivalents, end of period | 145,283 | 80,993 | 145,283 |
SUPPLEMENTAL DISCLOSURES OF CASH FLOW INFORMATION: | |||
Purchases of property and equipment unpaid at period end | 901 | 750 | |
Right-of-use assets acquired under operating leases | 40,394 | 7,347 | |
Proceeds from at-the-market offerings unpaid at period end | 551 | ||
At The Market Offerings [Member] | |||
CASH FLOWS FROM FINANCING ACTIVITIES: | |||
Proceeds from issuance of common stock through at-the-market offerings, net of issuance costs | $ 45,255 | $ 4,528 | $ 196,500 |
Overview and Basis of Presentat
Overview and Basis of Presentation | 3 Months Ended |
Mar. 31, 2021 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Overview and Basis of Presentation | 1. Overview and Basis of Presentation Intellia Therapeutics, Inc. (“Intellia” or the “Company”) is a leading clinical-stage genome editing company, focused on developing proprietary, potentially curative CRISPR/Cas9-based therapeutics. CRISPR/Cas9, an acronym for C lustered, R egularly I nterspaced S hort P alindromic R epeats (“CRISPR”)/CRISPR associated 9 (“Cas9”), is a technology for genome editing, the process of altering selected sequences of genomic deoxyribonucleic acid (“DNA”). transform medicine by both producing therapeutics that permanently edit and/or correct disease-associated genes in the human body with a single dose of treatment and creating enhanced engineered cell therapies. The Company’s combination of deep scientific, technical and clinical development experience, and proprietary innovations in genome editing and delivery technologies, along with its intellectual property (“IP”) portfolio, puts it in a position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create new classes of therapeutic products. The condensed consolidated financial statements of the Company included herein have been prepared, without audit, pursuant to the rules and regulations of the Securities and Exchange Commission (“SEC”). Certain information and footnote disclosures normally included in annual financial statements prepared in accordance with accounting principles generally accepted in the United States of America (“U.S. GAAP”) have been condensed or omitted from this report, as is permitted by such rules and regulations. Accordingly, these condensed consolidated financial statements should be read in conjunction with the financial statements and notes thereto included in the Company’s Annual Report on Form 10-K (“Annual Report”) for the year ended December 31, 2020. The unaudited condensed consolidated financial statements include the accounts of Intellia Therapeutics, Inc. and its wholly owned, controlled subsidiary, Intellia Securities Corp. All intercompany balances and transactions have been eliminated in consolidation. Comprehensive loss is comprised of net loss and unrealized gain/loss on marketable securities. The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates, judgments and assumptions that affect the amounts reported in the financial statements and accompanying notes. Significant estimates in these condensed consolidated financial statements have been made in connection with the calculation of revenues, research and development expenses and equity-based compensation expense. The Company bases its estimates on historical experience and various other assumptions that management believes to be reasonable under the circumstances at the time such estimates are made. Actual results could differ from those estimates. The Company periodically reviews its estimates in light of changes in circumstances, facts and experience. The extent of the impact of the coronavirus disease 19 (“COVID-19”) pandemic on the Company’s operational and financial performance will depend on certain developments, including the length and severity of this pandemic, as well as its effect on our employees, collaborators and vendors, all of which are uncertain and cannot be predicted. The Company cannot reasonably estimate the extent to which the disruption may materially impact its consolidated results of operations or financial position. The effects of material revisions in estimates are reflected in the condensed consolidated financial statements prospectively from the date of the change in estimate. In the opinion of management, the information furnished reflects all adjustments, all of which are of a normal and recurring nature, necessary for a fair presentation of the results for the reported interim periods. The Company considers events or transactions that occur after the balance sheet date but before the financial statements are issued to provide additional evidence relative to certain estimates or to identify matters that require additional disclosure. The results of operations for interim periods are not necessarily indicative of results to be expected for the full year or any other interim period. Liquidity Since its inception through March 31, 2021, the Company has raised an aggregate of $1,165.3 million to fund its operations, of which $275.0 million was through its collaboration agreements, $170.5 million was from its initial public offering (“IPO”) and concurrent private placements, $438.3 million was from follow-on public offerings, $196.5 million was from at-the-market offerings and $85.0 million was from the sale of convertible preferred stock. The Company expects that its cash, cash equivalents and marketable securities as of March 31, 2021, as well as research and cost reimbursement funding from its collaboration agreement with Regeneron Pharmaceuticals, Inc. (“Regeneron”) (see Note 7), will enable the Company to fund its ongoing operating expenses and capital expenditure requirements for at least the twelve-month period following the issuance of these condensed consolidated financial statements. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 3 Months Ended |
Mar. 31, 2021 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies The Company’s significant accounting policies are described in Note 2, “Summary of Significant Accounting Policies” to the consolidated financial statements included in the Annual Report for the year ended December 31, 2020. There have been no material changes during the three months ended March 31, 2021, other than as noted below Recent Accounting Pronouncements – Adopted In December 2019, the FASB issued ASU 2019-12, Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes . The Company adopted ASU 2019-12 on January 1, 2021. The adoption did not have a material effect on the Company’s condensed consolidated financial statements. |
Marketable Securities
Marketable Securities | 3 Months Ended |
Mar. 31, 2021 | |
Investments Debt And Equity Securities [Abstract] | |
Marketable Securities | 3. Marketable Securities The following table summarizes the Company’s available-for-sale marketable securities as of March 31, 2021 and December 31, 2020 at net book value: March 31, 2021 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Estimated Fair Value (In thousands) Marketable securities: U.S. Treasury and other government securities $ 204,473 $ 49 $ (4 ) $ 204,518 Financial institution debt securities 169,979 7 (45 ) 169,941 Corporate debt securities 51,311 1 (13 ) 51,299 Other asset-backed securities 33,509 1 (8 ) 33,502 Total $ 459,272 $ 58 $ (70 ) $ 459,260 December 31, 2020 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Estimated Fair Value (In thousands) Marketable securities: U.S. Treasury and other government securities $ 245,666 $ 13 $ (11 ) $ 245,668 Financial institution debt securities 138,445 6 (8 ) 138,443 Corporate debt securities 41,765 3 (2 ) 41,766 Other asset-backed securities 11,474 1 (1 ) 11,474 Total $ 437,350 $ 23 $ (22 ) $ 437,351 The amortized cost of available-for-sale securities is adjusted for amortization of premiums and accretion of discounts to maturity. At March 31, 2021 and December 31, 2020, the balance in the Company’s accumulated other comprehensive (loss) income was composed of activity related to the Company’s available-for-sale marketable securities. There were no realized gains or losses in the three months ended March 31, 2021 or for the year ended December 31, 2020. The Company did not reclassify any amounts out of accumulated other comprehensive (loss) income during this period. The Company did not have any securities in a material unrealized loss position at March 31, 2021 or December 31, 2020. The Company's available-for-sale securities that are classified as short-term marketable securities in the condensed consolidated balance sheet mature within one year or less as of the balance sheet date. Available-for-sale securities that are classified as noncurrent in the condensed consolidated balance sheet are those that mature after one year but within five years from the balance sheet date and that the Company does not intend to dispose of within the next twelve months. At March 31, 2021 and December 31, 2020, the Company did not hold any investments that matured beyond five years of the balance sheet date. |
Fair Value Measurements
Fair Value Measurements | 3 Months Ended |
Mar. 31, 2021 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | 4 . Fair Value Measurements The Company classifies fair value-based measurements using a three-level hierarchy that prioritizes the inputs used to measure fair value. This hierarchy requires entities to maximize the use of observable inputs and minimize the use of unobservable inputs. The three levels of inputs used to measure fair value are as follows: Level 1, quoted market prices in active markets for identical assets or liabilities; Level 2, observable inputs other than quoted market prices included in Level 1, such as quoted market prices for markets that are not active or other inputs that are observable or can be corroborated by observable market data; and Level 3, unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities, including certain pricing models, discounted cash flow methodologies and similar techniques that use significant unobservable inputs. As of March 31, 2021 and December 31, 2020, the Company’s financial assets recognized at fair value on a recurring basis consisted of the following: Fair Value as of March 31, 2021 Total Level 1 Level 2 Level 3 (In thousands) Cash equivalents and restricted cash equivalents $ 143,129 $ 143,129 $ - $ - Marketable securities: U.S. Treasury and other government securities 204,518 181,118 23,400 - Financial institution debt securities 169,941 - 169,941 - Corporate debt securities 51,299 - 51,299 - Other asset-backed securities 33,502 - 33,502 - Total marketable securities 459,260 181,118 278,142 - Total $ 602,389 $ 324,247 $ 278,142 $ - Fair Value as of December 31, 2020 Total Level 1 Level 2 Level 3 (In thousands) Cash equivalents and restricted cash equivalents $ 163,805 $ 163,805 $ - $ - Marketable securities: U.S. Treasury and other government securities 245,668 241,664 4,004 - Financial institution debt securities 138,443 - 138,443 - Corporate debt securities 41,766 - 41,766 - Other asset-backed securities 11,474 - 11,474 - Total marketable securities 437,351 241,664 195,687 - Total $ 601,156 $ 405,469 $ 195,687 $ - Certain of the Company’s financial assets, including cash equivalents, restricted cash equivalents and marketable securities, have been initially valued at the transaction price, and subsequently revalued at the end of each reporting period, utilizing third-party pricing services or other observable market data. The pricing services utilize industry standard valuation models and observable market inputs to determine value . After completing its validation procedures, the Company did not adjust or override any fair value measurements provided by the pricing services as of March 31, 2021 or December Other financial instruments, including accounts receivable, accounts payable and accrued expense, are carried at cost, which approximates fair value due to the short duration and term to maturity. |
Accrued Expenses
Accrued Expenses | 3 Months Ended |
Mar. 31, 2021 | |
Payables And Accruals [Abstract] | |
Accrued Expenses | 5 . Accrued Expenses Accrued expenses consisted of the following: March 31, December 31, 2021 2020 (In thousands) Accrued research and development $ 10,010 $ 11,008 Employee compensation and benefits 6,244 10,920 Accrued legal and professional expenses 2,833 1,876 Accrued other 2,128 1,750 Total accrued expenses $ 21,215 $ 25,554 |
Commitments and Contingencies
Commitments and Contingencies | 3 Months Ended |
Mar. 31, 2021 | |
Commitments And Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 6. Commitments and Contingencies Litigation There have been no material changes Caribou Arbitration On October 17, 2018, the Company initiated an arbitration proceeding against Caribou Biosciences, Inc. (“Caribou”) asserting that Caribou violated the terms and conditions of a license agreement the Company entered into with them in July 2014 related to certain IP (the “Caribou License”), as well as other contractual and legal obligations to the Company, by using and seeking to license to third parties two patent families relating to specific structural or chemical modifications of guide RNAs (“gRNAs”), that were purportedly invented or controlled by Caribou, in the Company’s exclusive human therapeutic field, before an agreed-upon cutoff date of January 30, 2018. On September 26, 2019, the Company announced that the arbitration panel issued an interim award concluding that both the structural and chemical gRNA modification technologies were exclusively licensed to the Company by Caribou pursuant to the Caribou License. Nevertheless, the arbitration panel, solely with respect to the clinically modified gRNAs, stated that it will declare that Caribou has an equitable “leaseback”, which it described as exclusive, perpetual and worldwide (the “Caribou Award”). The Caribou Award does not include the structural guide modifications IP also at issue in the arbitration, any other IP exclusively licensed or sublicensed by Caribou to the Company under the Caribou License (including but not limited to the foundational CRISPR/Cas9 IP co-owned by the Regents of the University of California, University of Vienna and Dr. Emmanuelle Charpentier), or any other of the Company’s IP. On February 6, 2020, the panel clarified that the Caribou Award is limited to a particular on-going Caribou program, which seeks to develop a chimeric antigen receptor T (“CAR-T”) product directed at CD19. As instructed by the panel, the parties have been negotiating the terms of the Caribou Award, including Caribou’s future payments to the Company. License Agreements The Company is party to license agreements, which include contingent payments. These payments will become payable if and when certain development, regulatory and commercial milestones are achieved. As of March 31, 2021, the satisfaction and timing of the contingent payments is uncertain and not reasonably estimable . |
Collaborations
Collaborations | 3 Months Ended |
Mar. 31, 2021 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Collaborations | 7 . Collaborations To accelerate the development and commercialization of CRISPR/Cas9-based products in multiple therapeutic areas, the Company has formed, and intends to seek other opportunities to form, strategic alliances with collaborators who can augment its leadership in CRISPR/Cas9 therapeutic development. As of March 31, 2021, the Company’s accounts receivable and contract liabilities were related to the Company’s collaboration with Regeneron. As of March 31, 2020, the Company’s accounts receivable and contract liabilities were related to the Company’s collaborations with Regeneron and Novartis Institutes for BioMedical Research (“Novartis”). The following table presents changes in the Company’s accounts receivable and contract liabilities during the three months ended March 31, 2021 and 2020 (in thousands): Balance at Beginning of Period Additions Deductions Balance at End of Period Three Months Ended March 31, 2021 Accounts receivable $ 2,130 $ 953 $ (2,130 ) $ 953 Contract liabilities: Deferred revenue $ 73,931 $ - $ (5,558 ) $ 68,373 Balance at Beginning of Period Additions Deductions Balance at End of Period Three Months Ended March 31, 2020 Accounts receivable $ 4,620 $ 9,765 $ (1,017 ) $ 13,368 Contract liabilities: Deferred revenue $ 28,810 $ - $ (3,151 ) $ 25,659 During the three months ended March 31, 2021 and 2020, the Company recognized the following revenues as a result of changes in the contract liability balance (in thousands): Three Months Ended March 31, Revenue recognized in the period from: 2021 2020 Amounts included in the contract liability at the beginning of the period $ 5,558 $ 3,151 Costs to obtain and fulfill a contract The Company did not incur any expenses to obtain collaboration agreements and costs to fulfill those contracts do not generate or enhance resources of the Company. As such, no costs to obtain or fulfill a contract have been capitalized in any period. Regeneron Pharmaceuticals, Inc. License and Collaboration Agreement In April 2016, the Company entered into a license and collaboration agreement with Regeneron (the “2016 Regeneron Agreement”). The 2016 Regeneron Agreement has two principal components: (i) a product development component under which the parties will research, develop and commercialize CRISPR/Cas-based therapeutic products primarily focused on genome editing in the liver, and (ii) a technology collaboration component, pursuant to which the Company and Regeneron will engage in research-related activities aimed at discovering and developing novel technologies and improvements to CRISPR/Cas technology to enhance the Company’s genome editing platform. Under this agreement, the Company also may access the Regeneron Genetics Center and proprietary mouse models to be provided by Regeneron for a limited number of the Company’s liver programs. On May 30, 2020, the Company entered into (i) amendment no. 1 (the “2020 Regeneron Amendment”) to the 2016 Regeneron Agreement, (ii) co-development and co-funding agreements for the treatment of hemophilia A and hemophilia B (the “Hemophilia Co/Co”) agreements and (iii) a stock purchase agreement. The collaboration expansion builds upon the jointly developed targeted transgene insertion capabilities designed to durably restore missing therapeutic protein, and to overcome the limitations of traditional gene therapy. The collaboration was extended until April 2024, at which point Regeneron has an option to renew for an additional two years. The 2020 Regeneron Amendment also grants Regeneron exclusive rights to develop products for five additional in vivo CRISPR/Cas-based therapeutic liver targets and non-exclusive rights to independently develop and commercialize up to 10 ex vivo gene edited products made using certain defined cell types. Since December 31, 2020, there have been no material changes to the key terms of the 2016 Regeneron Agreement and the 2020 Regeneron Amendment (the “Amended Agreements”). For further information on the terms and conditions of these agreements, please see the notes to the consolidated financial statements included in the Company’s Annual Report for the year ended December 31, 2020. Revenue Recognition – Collaboration Revenue. Through March 31, 2021, excluding amounts allocated to Regeneron’s purchase of the Company’s common stock, the Company recorded $145.0 million in upfront payments under the Amended Agreements and $35.6 million primarily for research and development services under the ATTR Co/Co agreement. Through March 31, 2021, the Company has recognized $129.7 million of collaboration revenue under all arrangements, including $6.4 million and $7.9 million during the three months ended March 31, 2021 and 2020, respectively, in the condensed consolidated statements of operations and comprehensive loss. This includes $0.9 million and $4.8 million during the three months ended March 31, 2021 and 2020, respectively, primarily representing payments due from Regeneron pursuant to the ATTR Co/Co agreement. As of March 31, 2021, there was approximately $68.4 million of the aggregate transaction price of the Amended Agreements remaining to be recognized, which the Company expects to be recognized during the research term through April 2024. As of March 31, 2021 and December 31, 2020, the Company had accounts receivable of $1.0 million and $2.1 million, respectively, and deferred revenue of $68.4 million and $73.9 million, respectively, related to the Amended Agreements. Novartis Institutes for BioMedical Research, Inc. In December 2014, the Company entered into a strategic collaboration agreement with Novartis (the “2014 Novartis Agreement”), primarily focused on the research of new ex vivo Revenue Recognition – Milestone . During the three months ended March 31, 2020, the U.S. Food and Drug Administration (“FDA”) accepted the investigational new drug (“IND”) application submitted by Novartis for a CRISPR/Cas9-based engineered cell therapy for the treatment of sickle cell disease. As a result of meeting this milestone, the Company recognized $5.0 million as collaboration revenue within the condensed consolidated statement of operations and comprehensive loss. No other milestones under the 2014 Novartis Agreement and the Novartis Amendment were achieved during the three months ended March 31, 2021 or 2020. The Company is eligible to receive additional downstream success-based milestones and royalties. As of March 31, 2021 and December 31, 2020, the Company had no accounts receivable or deferred revenue related to the 2014 Novartis Agreement and the Novartis Amendment. |
Leases
Leases | 3 Months Ended |
Mar. 31, 2021 | |
Leases [Abstract] | |
Leases | 8 . Leases In March 2020, the Company entered into an agreement to lease approximately 39,000 square feet of office and laboratory space at 281 Albany Street in Cambridge, Massachusetts under an operating lease agreement (the “281 Albany Lease”). The Company’s obligation to pay rent will start on the date that is six months after the commencement date or the date on which the Company occupies the premises, whichever occurs earlier (the “Rent Commencement Date”). The initial term of the 281 Albany Lease is ten years following the Rent Commencement Date. As of March 31, 2021 the Company determined, in accordance with Accounting Standards Codification 842, “Leases (Topic 842)” 40.4 term of the lease. These restricted cash equivalents are reported in “Other Assets” in the Company’s condensed consolidated balance sheet. The Company has the option to extend the 281 Albany Lease for two successive five-year terms . The option for this extension is not included as part of the lease liability and right-of-use asset at March 31, 2021, as it is not reasonably certain that it will be exercised. |
Equity-Based Compensation
Equity-Based Compensation | 3 Months Ended |
Mar. 31, 2021 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Equity-Based Compensation | 9. Equity-Based Compensation In April 2016, the Company adopted the Amended and Restated 2015 Stock Option and Incentive Plan (the “2015 Plan”). The 2015 Plan provides for the grant of incentive stock options, non-qualified stock options, stock appreciation rights, restricted stock awards, restricted stock units (“RSUs”) and other stock-based awards. Recipients of incentive stock options and non-qualified stock options are eligible to purchase shares of the Company’s common stock at an exercise price equal to the fair value of such stock on the grant date. Stock options granted under the 2015 Plan generally vest 25% on the first anniversary of the original vesting date, with the balance vesting monthly over the remaining three years, unless they contain specific performance-based vesting provisions. The maximum term of stock options granted under the 2015 Plan is ten years. As of March 31, 2021, there were 2,890,540 shares available for future issuance. The number of shares reserved for issuance under the 2015 Plan shall be cumulatively increased by four percent of the number of shares of stock issued and outstanding on the immediately preceding December 31 or such lesser number of shares of stock as determined by the board of directors. Equity-based compensation expense is classified in the condensed consolidated statements of operations and comprehensive loss as follows: Three Months Ended March 31, 2021 2020 (In thousands) Research and development $ 3,491 $ 2,160 General and administrative 2,933 1,997 Total $ 6,424 $ 4,157 Restricted Stock Restricted stock is measured at fair value based on the quoted price of the Company’s common stock. The following table summarizes the Company’s restricted stock activity for the three months ended March 31, 2021: Number of Shares Weighted Average Grant Date Fair Value per Share Unvested restricted stock as of December 31, 2020 193,936 $ 23.98 Granted 259,839 57.71 Vested - - Cancelled (11,035 ) 27.59 Unvested restricted stock as of March 31, 2021 442,740 $ 43.69 In March 2021, the Company granted 259,839 RSUs with a service condition to executive and non-executive employees as part of their annual grant, which vest over a period of four years. The weighted average grant date fair value of these RSUs was $57.71. The vesting start date for these RSUs is January 1, 2021. Included in the unvested restricted stock as of March 31, 2021 are 107,360 RSUs that include a performance condition in addition to a service condition. The RSUs vest over a period of three years and are subject to accelerated vesting based on the Company’s programs achieving certain development milestones before December 1, 2022. The fair value of the RSUs at date of grant was $15.05. There has been no additional vesting of these shares in the three months ended March 31, 2021. As of March 31, 2021, there was $17.8 Stock Options The weighted average grant date fair value of options, estimated as of the grant date using the Black-Scholes option pricing model, was $36.64 7.96 0.3 Three Months Ended March 31, 2021 2020 Risk-free interest rate 0.9% 1.0% Expected life of options 6.0 years 6.0 years Expected volatility of underlying stock 72.0% 66.7% Expected dividend yield 0.0% 0.0% Risk-free Interest Rate. The risk-free interest rate is based on the U.S. Treasury yield curve in effect at the time of grant with maturities approximately equal to the option’s expected term. Expected Dividend Yield. The expected dividend yield assumption is based on the fact that the Company has never paid cash dividends and has no present intention to pay cash dividends. Expected Volatility. The expected volatility was derived from a blend of the Company’s historical volatility and an average of the historical stock volatilities of several peer companies within the Company’s industry, both over a period equivalent to the expected term of the stock option grants. Expected Term. The expected term represents the period that stock option awards are expected to be outstanding. For option grants that are considered to be “plain vanilla,” the Company determines the expected term using the simplified method. The simplified method deems the term to be the average of the time-to-vesting and the contractual life of the options. The Company uses the simplified method because it does not have sufficient historical option exercise data to provide a reasonable basis upon which to estimate the expected term. The Company uses the market closing price of its common stock as reported on the Nasdaq Global Select Market to determine the fair value of the shares of common stock underlying stock options. The following is a summary of stock option activity for the three months ended March 3 1 , 20 2 1 : Number of Options Weighted Average Exercise Price per Share Weighted Average Remaining Contractual Term Aggregate Intrinsic Value (In years) (In thousands) Outstanding at December 31, 2020 6,977,440 $ 15.43 Granted 1,645,823 57.94 Exercised (1,014,569 ) 13.15 Forfeited (129,393 ) 20.71 Outstanding at March 31, 2021 7,479,301 $ 25.00 8.25 $ 413,258 Exercisable at March 31, 2021 2,734,966 As of March 31, 2021, there was $86.9 |
Loss Per Share
Loss Per Share | 3 Months Ended |
Mar. 31, 2021 | |
Earnings Per Share [Abstract] | |
Loss Per Share | 10 . Loss Per Share The Company calculates basic loss per share by dividing net loss for each respective period by the weighted average number of common shares outstanding for each respective period. The Company computes diluted loss per share after giving consideration to the dilutive effect of stock options and unvested restricted stock that are outstanding during the period, except where such securities would be anti-dilutive. Basic and diluted loss per share was calculated as follows: Three Months Ended March 31, 2021 2020 (In thousands) Net loss $ (46,205 ) $ (31,806 ) Weighted average shares outstanding, basic and diluted 67,183 50,491 Net loss per share, basic and diluted $ (0.69 ) $ (0.63 ) The following common stock equivalents were excluded from the calculation of diluted loss per share because their inclusion would have been anti-dilutive: Three Months Ended March 31, 2021 2020 (In thousands) Unvested restricted stock 443 248 Stock options 7,479 7,243 7,922 7,491 |
Stockholders_ Equity
Stockholders’ Equity | 3 Months Ended |
Mar. 31, 2021 | |
Equity [Abstract] | |
Stockholders’ Equity | 11 . Stockholders’ Equity The following tables present changes in stockholders’ equity for the three-month periods ended March 31, 2021 and 2020 (in thousands, except share data): Additional Accumulated Other Total Common Paid-In Comprehensive Accumulated Stockholders’ Shares Amount Capital Income (Loss) Deficit Equity Balance at December 31, 2020 66,234,056 $ 7 $ 962,173 $ 1 $ (435,109 ) $ 527,072 Issuance of common stock through at-the-market offerings, net of issuance costs of $52 641,709 - 45,255 - - 45,255 Exercise of stock options 1,014,569 - 13,340 - - 13,340 Equity-based compensation - - 6,424 - - 6,424 Other comprehensive loss - - - (13 ) - (13 ) Net loss - - - - (46,205 ) (46,205 ) Balance at March 31, 2021 67,890,334 $ 7 $ 1,027,192 $ (12 ) $ (481,314 ) $ 545,873 Additional Accumulated Other Total Common Paid-In Comprehensive Accumulated Stockholders’ Shares Amount Capital Income Deficit Equity Balance at December 31, 2019 50,198,044 $ 5 $ 570,493 $ 261 $ (300,878 ) $ 269,881 Issuance of common stock through at-the-market offerings, net of issuance costs of $48 351,252 - 5,079 - - 5,079 Exercise of stock options 53,579 - 336 - - 336 Equity-based compensation - - 4,157 - - 4,157 Other comprehensive income - - - 112 - 112 Net loss - - - - (31,806 ) (31,806 ) Balance at March 31, 2020 50,602,875 $ 5 $ 580,065 $ 373 $ (332,684 ) $ 247,759 At-the-Market Offering Programs I n August 2019, the Company entered into an Open Market Sale Agreement (the “2019 Sales Agreement”) with Jefferies, under which Jefferies was able to offer and sell, from time to time in “at-the-market” offerings, common stock having aggregate gross proceeds of up to $150.0 million. The Company agreed to pay Jefferies cash commissions of 3.0% of the gross proceeds of sales of common stock under the 2019 Sales Agreement. During the three months ended March 31, 2021, the Company issued 641,709 shares of its common stock in a series of sales at an average price of $72.79 per 45.3 approximately million related to legal, accounting and other fees in connection with the sales. $15.05 per 5.1 approximately $0.1 million related to legal, accounting and other fees in connection with the sales. As of March 31, 2021, $47.4 million in shares of common stock remain eligible for sale under the 2019 Sales |
Related Party Transactions
Related Party Transactions | 3 Months Ended |
Mar. 31, 2021 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | 1 2 . Related Party Transactions In the ordinary course of business, the Company may purchase materials or supplies from entities that are associated with a party that meets the criteria of a related party of the Company. These transactions are reviewed quarterly and to date have not been material to the Company’s condensed consolidated financial statements. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 3 Months Ended |
Mar. 31, 2021 | |
Accounting Policies [Abstract] | |
Recent Accounting Pronouncements - Adopted | Recent Accounting Pronouncements – Adopted In December 2019, the FASB issued ASU 2019-12, Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes . The Company adopted ASU 2019-12 on January 1, 2021. The adoption did not have a material effect on the Company’s condensed consolidated financial statements. |
Marketable Securities (Tables)
Marketable Securities (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Investments Debt And Equity Securities [Abstract] | |
Summary of Available-for-sale Marketable Securities | The following table summarizes the Company’s available-for-sale marketable securities as of March 31, 2021 and December 31, 2020 at net book value: March 31, 2021 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Estimated Fair Value (In thousands) Marketable securities: U.S. Treasury and other government securities $ 204,473 $ 49 $ (4 ) $ 204,518 Financial institution debt securities 169,979 7 (45 ) 169,941 Corporate debt securities 51,311 1 (13 ) 51,299 Other asset-backed securities 33,509 1 (8 ) 33,502 Total $ 459,272 $ 58 $ (70 ) $ 459,260 December 31, 2020 Amortized Cost Gross Unrealized Gains Gross Unrealized Losses Estimated Fair Value (In thousands) Marketable securities: U.S. Treasury and other government securities $ 245,666 $ 13 $ (11 ) $ 245,668 Financial institution debt securities 138,445 6 (8 ) 138,443 Corporate debt securities 41,765 3 (2 ) 41,766 Other asset-backed securities 11,474 1 (1 ) 11,474 Total $ 437,350 $ 23 $ (22 ) $ 437,351 |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Fair Value Disclosures [Abstract] | |
Summary of Financial Assets Recognized at Fair Value on Recurring Basis | As of March 31, 2021 and December 31, 2020, the Company’s financial assets recognized at fair value on a recurring basis consisted of the following: Fair Value as of March 31, 2021 Total Level 1 Level 2 Level 3 (In thousands) Cash equivalents and restricted cash equivalents $ 143,129 $ 143,129 $ - $ - Marketable securities: U.S. Treasury and other government securities 204,518 181,118 23,400 - Financial institution debt securities 169,941 - 169,941 - Corporate debt securities 51,299 - 51,299 - Other asset-backed securities 33,502 - 33,502 - Total marketable securities 459,260 181,118 278,142 - Total $ 602,389 $ 324,247 $ 278,142 $ - Fair Value as of December 31, 2020 Total Level 1 Level 2 Level 3 (In thousands) Cash equivalents and restricted cash equivalents $ 163,805 $ 163,805 $ - $ - Marketable securities: U.S. Treasury and other government securities 245,668 241,664 4,004 - Financial institution debt securities 138,443 - 138,443 - Corporate debt securities 41,766 - 41,766 - Other asset-backed securities 11,474 - 11,474 - Total marketable securities 437,351 241,664 195,687 - Total $ 601,156 $ 405,469 $ 195,687 $ - |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Payables And Accruals [Abstract] | |
Schedule of Accrued Expenses | Accrued expenses consisted of the following: March 31, December 31, 2021 2020 (In thousands) Accrued research and development $ 10,010 $ 11,008 Employee compensation and benefits 6,244 10,920 Accrued legal and professional expenses 2,833 1,876 Accrued other 2,128 1,750 Total accrued expenses $ 21,215 $ 25,554 |
Collaborations (Tables)
Collaborations (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Summary of Changes in Accounts Receivable and Contract Liabilities | The following table presents changes in the Company’s accounts receivable and contract liabilities during the three months ended March 31, 2021 and 2020 (in thousands): Balance at Beginning of Period Additions Deductions Balance at End of Period Three Months Ended March 31, 2021 Accounts receivable $ 2,130 $ 953 $ (2,130 ) $ 953 Contract liabilities: Deferred revenue $ 73,931 $ - $ (5,558 ) $ 68,373 Balance at Beginning of Period Additions Deductions Balance at End of Period Three Months Ended March 31, 2020 Accounts receivable $ 4,620 $ 9,765 $ (1,017 ) $ 13,368 Contract liabilities: Deferred revenue $ 28,810 $ - $ (3,151 ) $ 25,659 |
Summary of Revenues Recognized Resulting From Changes in Contract Liability Balance | During the three months ended March 31, 2021 and 2020, the Company recognized the following revenues as a result of changes in the contract liability balance (in thousands): Three Months Ended March 31, Revenue recognized in the period from: 2021 2020 Amounts included in the contract liability at the beginning of the period $ 5,558 $ 3,151 |
Equity-Based Compensation (Tabl
Equity-Based Compensation (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Schedule of Equity-Based Compensation Expense | Equity-based compensation expense is classified in the condensed consolidated statements of operations and comprehensive loss as follows: Three Months Ended March 31, 2021 2020 (In thousands) Research and development $ 3,491 $ 2,160 General and administrative 2,933 1,997 Total $ 6,424 $ 4,157 |
Summary of Restricted Stock Activity | The following table summarizes the Company’s restricted stock activity for the three months ended March 31, 2021: Number of Shares Weighted Average Grant Date Fair Value per Share Unvested restricted stock as of December 31, 2020 193,936 $ 23.98 Granted 259,839 57.71 Vested - - Cancelled (11,035 ) 27.59 Unvested restricted stock as of March 31, 2021 442,740 $ 43.69 |
Summary of Weighted Average Assumptions Used to Compute Fair Value of Option Granted | Weighted average assumptions used to apply this pricing model were as follows: Three Months Ended March 31, 2021 2020 Risk-free interest rate 0.9% 1.0% Expected life of options 6.0 years 6.0 years Expected volatility of underlying stock 72.0% 66.7% Expected dividend yield 0.0% 0.0% |
Summary of Stock Option Activity | The following is a summary of stock option activity for the three months ended March 3 1 , 20 2 1 : Number of Options Weighted Average Exercise Price per Share Weighted Average Remaining Contractual Term Aggregate Intrinsic Value (In years) (In thousands) Outstanding at December 31, 2020 6,977,440 $ 15.43 Granted 1,645,823 57.94 Exercised (1,014,569 ) 13.15 Forfeited (129,393 ) 20.71 Outstanding at March 31, 2021 7,479,301 $ 25.00 8.25 $ 413,258 Exercisable at March 31, 2021 2,734,966 |
Loss Per Share (Tables)
Loss Per Share (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Earnings Per Share [Abstract] | |
Schedule of Basic and Diluted Net Loss Per Share | Basic and diluted loss per share was calculated as follows: Three Months Ended March 31, 2021 2020 (In thousands) Net loss $ (46,205 ) $ (31,806 ) Weighted average shares outstanding, basic and diluted 67,183 50,491 Net loss per share, basic and diluted $ (0.69 ) $ (0.63 ) |
Potential Dilutive Securities Excluded from Computation of Diluted Net Loss Per Common Share | The following common stock equivalents were excluded from the calculation of diluted loss per share because their inclusion would have been anti-dilutive: Three Months Ended March 31, 2021 2020 (In thousands) Unvested restricted stock 443 248 Stock options 7,479 7,243 7,922 7,491 |
Stockholders_ Equity (Tables)
Stockholders’ Equity (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Equity [Abstract] | |
Schedule of Changes in Stockholders’ Equity | The following tables present changes in stockholders’ equity for the three-month periods ended March 31, 2021 and 2020 (in thousands, except share data): Additional Accumulated Other Total Common Paid-In Comprehensive Accumulated Stockholders’ Shares Amount Capital Income (Loss) Deficit Equity Balance at December 31, 2020 66,234,056 $ 7 $ 962,173 $ 1 $ (435,109 ) $ 527,072 Issuance of common stock through at-the-market offerings, net of issuance costs of $52 641,709 - 45,255 - - 45,255 Exercise of stock options 1,014,569 - 13,340 - - 13,340 Equity-based compensation - - 6,424 - - 6,424 Other comprehensive loss - - - (13 ) - (13 ) Net loss - - - - (46,205 ) (46,205 ) Balance at March 31, 2021 67,890,334 $ 7 $ 1,027,192 $ (12 ) $ (481,314 ) $ 545,873 Additional Accumulated Other Total Common Paid-In Comprehensive Accumulated Stockholders’ Shares Amount Capital Income Deficit Equity Balance at December 31, 2019 50,198,044 $ 5 $ 570,493 $ 261 $ (300,878 ) $ 269,881 Issuance of common stock through at-the-market offerings, net of issuance costs of $48 351,252 - 5,079 - - 5,079 Exercise of stock options 53,579 - 336 - - 336 Equity-based compensation - - 4,157 - - 4,157 Other comprehensive income - - - 112 - 112 Net loss - - - - (31,806 ) (31,806 ) Balance at March 31, 2020 50,602,875 $ 5 $ 580,065 $ 373 $ (332,684 ) $ 247,759 |
Overview and Basis of Present_2
Overview and Basis of Presentation - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | 71 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | Mar. 31, 2021 | |
Organization Consolidation And Presentation Of Financial Statements Disclosure [Line Items] | |||
Proceeds from common stock offering | $ 1,165,300 | ||
Proceeds from issuance of convertible preferred stock | 85,000 | ||
Collaborative Arrangement [Member] | |||
Organization Consolidation And Presentation Of Financial Statements Disclosure [Line Items] | |||
Proceeds from common stock offering | 275,000 | ||
Initial Public Offering and Concurrent Private Placements [Member] | |||
Organization Consolidation And Presentation Of Financial Statements Disclosure [Line Items] | |||
Proceeds from common stock offering | 170,500 | ||
Follow-on public Offerings [Member] | |||
Organization Consolidation And Presentation Of Financial Statements Disclosure [Line Items] | |||
Proceeds from common stock offering | 438,300 | ||
At The Market Offerings [Member] | |||
Organization Consolidation And Presentation Of Financial Statements Disclosure [Line Items] | |||
Proceeds from common stock offering | $ 45,255 | $ 4,528 | $ 196,500 |
Marketable Securities - Summary
Marketable Securities - Summary of Available -for-sale Marketable Securities (Detail) - USD ($) $ in Thousands | Mar. 31, 2021 | Dec. 31, 2020 |
Marketable Securities [Line Items] | ||
Amortized Cost | $ 459,272 | $ 437,350 |
Gross Unrealized Gains | 58 | 23 |
Gross Unrealized Losses | (70) | (22) |
Estimated Fair Value | 459,260 | 437,351 |
U.S. Treasury and Other Government Securities [Member] | ||
Marketable Securities [Line Items] | ||
Amortized Cost | 204,473 | 245,666 |
Gross Unrealized Gains | 49 | 13 |
Gross Unrealized Losses | (4) | (11) |
Estimated Fair Value | 204,518 | 245,668 |
Financial Institution Debt Securities [Member] | ||
Marketable Securities [Line Items] | ||
Amortized Cost | 169,979 | 138,445 |
Gross Unrealized Gains | 7 | 6 |
Gross Unrealized Losses | (45) | (8) |
Estimated Fair Value | 169,941 | 138,443 |
Corporate Debt Securities [Member] | ||
Marketable Securities [Line Items] | ||
Amortized Cost | 51,311 | 41,765 |
Gross Unrealized Gains | 1 | 3 |
Gross Unrealized Losses | (13) | (2) |
Estimated Fair Value | 51,299 | 41,766 |
Other Asset Backed Securities [Member] | ||
Marketable Securities [Line Items] | ||
Amortized Cost | 33,509 | 11,474 |
Gross Unrealized Gains | 1 | 1 |
Gross Unrealized Losses | (8) | (1) |
Estimated Fair Value | $ 33,502 | $ 11,474 |
Marketable Securities - Additio
Marketable Securities - Additional Information (Detail) - USD ($) | 3 Months Ended | 12 Months Ended |
Mar. 31, 2021 | Dec. 31, 2020 | |
Marketable Securities [Line Items] | ||
Realized gains or losses on marketable securities | $ 0 | $ 0 |
Investments that matured beyond five years | $ 0 | $ 0 |
Minimum [Member] | ||
Marketable Securities [Line Items] | ||
Available-for-sales Securities, non-current, maturity period | 1 year | |
Maximum [Member] | ||
Marketable Securities [Line Items] | ||
Available-for-sales Securities, non-current, maturity period | 5 years |
Fair Value Measurements - Summa
Fair Value Measurements - Summary of Financial Assets Recognized at Fair Value on Recurring Basis (Detail) - Fair Value on Recurring Basis [Member] - USD ($) $ in Thousands | Mar. 31, 2021 | Dec. 31, 2020 |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash equivalents and restricted cash equivalents | $ 143,129 | $ 163,805 |
Marketable securities: | ||
Marketable securities | 459,260 | 437,351 |
Total | 602,389 | 601,156 |
U.S. Treasury and Other Government Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 204,518 | 245,668 |
Financial Institution Debt Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 169,941 | 138,443 |
Corporate Debt Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 51,299 | 41,766 |
Other Asset Backed Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 33,502 | 11,474 |
Level 1 [Member] | ||
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | ||
Cash equivalents and restricted cash equivalents | 143,129 | 163,805 |
Marketable securities: | ||
Marketable securities | 181,118 | 241,664 |
Total | 324,247 | 405,469 |
Level 1 [Member] | U.S. Treasury and Other Government Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 181,118 | 241,664 |
Level 2 [Member] | ||
Marketable securities: | ||
Marketable securities | 278,142 | 195,687 |
Total | 278,142 | 195,687 |
Level 2 [Member] | U.S. Treasury and Other Government Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 23,400 | 4,004 |
Level 2 [Member] | Financial Institution Debt Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 169,941 | 138,443 |
Level 2 [Member] | Corporate Debt Securities [Member] | ||
Marketable securities: | ||
Marketable securities | 51,299 | 41,766 |
Level 2 [Member] | Other Asset Backed Securities [Member] | ||
Marketable securities: | ||
Marketable securities | $ 33,502 | $ 11,474 |
Accrued Expenses - Schedule of
Accrued Expenses - Schedule of Accrued Expenses (Detail) - USD ($) $ in Thousands | Mar. 31, 2021 | Dec. 31, 2020 |
Payables And Accruals [Abstract] | ||
Accrued research and development | $ 10,010 | $ 11,008 |
Employee compensation and benefits | 6,244 | 10,920 |
Accrued legal and professional expenses | 2,833 | 1,876 |
Accrued other | 2,128 | 1,750 |
Total accrued expenses | $ 21,215 | $ 25,554 |
Collaborations - Additional Inf
Collaborations - Additional Information (Detail) - USD ($) | 3 Months Ended | 60 Months Ended | |||
Mar. 31, 2021 | Mar. 31, 2020 | Mar. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||
Collaboration revenue | $ 6,445,000 | $ 12,916,000 | |||
Deferred revenue | 68,373,000 | 25,659,000 | $ 68,373,000 | $ 73,931,000 | $ 28,810,000 |
Regeneron Pharmaceuticals Inc. [Member] | Regeneron Agreement [Member] | |||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||
Deferred revenue additions | 145,000,000 | ||||
Collaboration revenue | $ 6,400,000 | 7,900,000 | 129,700,000 | ||
Aggregate transaction price remaining to be recognized, period | Through March 31, 2021 | ||||
Payments due | $ 900,000 | $ 4,800,000 | |||
Regeneron Pharmaceuticals Inc. [Member] | Regeneron Agreement [Member] | Research and Development Services [Member] | |||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||
Collaboration revenue | 35,600,000 | ||||
Regeneron Pharmaceuticals Inc. [Member] | Regeneron Amendment [Member] | |||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||
Aggregate transaction price remaining to be recognized | 68,400,000 | 68,400,000 | |||
Accounts receivable | 1,000,000 | 1,000,000 | 2,100,000 | ||
Deferred revenue | $ 68,400,000 | 68,400,000 | 73,900,000 | ||
Novartis [Member] | |||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||
Strategic collaboration agreement amended date | 2018-12 | ||||
Novartis [Member] | Regeneron Agreement [Member] | |||||
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items] | |||||
Collaboration revenue | $ 5,000,000 | ||||
Deferred revenue | 0 | 0 | 0 | ||
Accounts receivable | $ 0 | $ 0 | $ 0 |
Collaborations - Summary of Cha
Collaborations - Summary of Changes in Accounts Receivable and Contract Liabilities (Detail) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Accounts receivable: | ||
Accounts receivable, Balance at Beginning of Period | $ 2,130 | $ 4,620 |
Accounts receivable, Additions | 953 | 9,765 |
Accounts receivable, Deductions | (2,130) | (1,017) |
Accounts receivable, Balance at End of Period | 953 | 13,368 |
Contract liabilities: | ||
Deferred revenue, Balance at Beginning of Period | 73,931 | 28,810 |
Deferred revenue, Deductions | (5,558) | (3,151) |
Deferred revenue, Balance at End of Period | $ 68,373 | $ 25,659 |
Collaborations - Summary of Rev
Collaborations - Summary of Revenues Recognized Resulting From Changes in Contract Liability Balance (Detail) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Revenue From Contract With Customer [Abstract] | ||
Amounts included in the contract liability at the beginning of the period | $ 5,558 | $ 3,151 |
Leases - Additional Information
Leases - Additional Information (Detail) $ in Thousands | 3 Months Ended | |
Mar. 31, 2021USD ($)ft²USD_per_sqft | Dec. 31, 2020USD ($) | |
Lessee Lease Description [Line Items] | ||
Operating lease right-of-use assets | $ 77,912 | $ 39,114 |
Albany Lease [Member] | ||
Lessee Lease Description [Line Items] | ||
Area of space leased | ft² | 39,000 | |
Operating lease, description | In March 2020, the Company entered into an agreement to lease approximately 39,000 square feet of office and laboratory space at 281 Albany Street in Cambridge, Massachusetts under an operating lease agreement (the “281 Albany Lease”). The Company’s obligation to pay rent will start on the date that is six months after the commencement date or the date on which the Company occupies the premises, whichever occurs earlier (the “Rent Commencement Date”). | |
Term of lease | 10 years | |
Base rent per square foot for first year | USD_per_sqft | 99 | |
Base rent per square foot for last year | USD_per_sqft | 128.87 | |
Operating lease right-of-use assets | $ 40,400 | |
Operating lease, liability | $ 34,800 | |
Incremental borrowing rate | 5.52% | |
Prepaid lease payments | $ 5,600 | |
Amount receivable on cost of construction and tenant improvement | $ 4,400 | |
Operating lease, existence of option to extend | true | |
Operating lease, options to extend | The Company has the option to extend the 281 Albany Lease for two successive five-year terms. | |
Albany Lease [Member] | Other Assets [Member] | ||
Lessee Lease Description [Line Items] | ||
Letter of credit | $ 1,900 |
Equity-Based Compensation - Add
Equity-Based Compensation - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Millions | 1 Months Ended | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2021 | Mar. 31, 2020 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Remaining vesting period | 4 years | ||
Weighted Average Grant Date Fair Value per Share, Granted | $ 57.71 | ||
Number of Shares, Granted | 259,839 | ||
Unrecognized equity-based compensation expense related to restricted stock | $ 17.8 | $ 17.8 | |
Weighted average grant date fair value per share | $ 36.64 | $ 7.96 | |
Total intrinsic value of stock options exercised | $ 54.8 | $ 0.3 | |
Unrecognized compensation cost related to stock options | $ 86.9 | $ 86.9 | |
Restricted Stock [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Weighted Average Grant Date Fair Value per Share, Granted | $ 57.71 | ||
Number of Shares, Granted | 259,839 | ||
Weighted average period of unrecognized compensation costs | 3 years 7 months 6 days | ||
RSUs [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Remaining vesting period | 3 years | ||
Weighted Average Grant Date Fair Value per Share, Granted | $ 15.05 | ||
Number of Shares, Granted | 107,360 | ||
Additional vesting of shares | 0 | ||
Stock Options [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Weighted average period of unrecognized compensation costs | 3 years 2 months 12 days | ||
2015 Plan [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Remaining vesting period | 3 years | ||
Description of stock options granted under the Plan | Stock options granted under the 2015 Plan generally vest 25% on the first anniversary of the original vesting date, with the balance vesting monthly over the remaining three years, unless they contain specific performance-based vesting provisions. The maximum term of stock options granted under the 2015 Plan is ten years. | ||
Shares available for future issuance | 2,890,540 | 2,890,540 | |
Percentage of cumulative increase in number of shares for future issuance | 4.00% | ||
2015 Plan [Member] | First Anniversary of Original Vesting Date [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Vest percentage on the first anniversary | 25.00% | ||
Maximum [Member] | 2015 Plan [Member] | |||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||
Maximum term of stock options granted | 10 years |
Equity-Based Compensation - Sch
Equity-Based Compensation - Schedule of Equity-Based Compensation Expense (Detail) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||
Equity-based compensation expense | $ 6,424 | $ 4,157 |
Research and Development [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||
Equity-based compensation expense | 3,491 | 2,160 |
General and Administrative [Member] | ||
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items] | ||
Equity-based compensation expense | $ 2,933 | $ 1,997 |
Equity-Based Compensation - Sum
Equity-Based Compensation - Summary of Restricted Stock Activity (Detail) | 3 Months Ended |
Mar. 31, 2021$ / sharesshares | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Number of Shares, Unvested, Beginning balance | shares | 193,936 |
Number of Shares, Granted | shares | 259,839 |
Number of Shares, Cancelled | shares | (11,035) |
Number of Shares, Unvested, Ending balance | shares | 442,740 |
Weighted Average Grant Date Fair Value per Share, Unvested, Beginning balance | $ / shares | $ 23.98 |
Weighted Average Grant Date Fair Value per Share, Granted | $ / shares | 57.71 |
Weighted Average Grant Date Fair Value per Share, Cancelled | $ / shares | 27.59 |
Weighted Average Grant Date Fair Value per Share, Unvested, Ending balance | $ / shares | $ 43.69 |
Equity-Based Compensation - S_2
Equity-Based Compensation - Summary of Weighted Average Assumptions Used to Compute Fair Value of Option Granted (Detail) | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | ||
Risk-free interest rate | 0.90% | 1.00% |
Expected life of options | 6 years | 6 years |
Expected volatility of underlying stock | 72.00% | 66.70% |
Expected dividend yield | 0.00% | 0.00% |
Equity-Based Compensation - S_3
Equity-Based Compensation - Summary of Stock Option Activity (Detail) $ / shares in Units, $ in Thousands | 3 Months Ended |
Mar. 31, 2021USD ($)$ / sharesshares | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Number of Options, Outstanding, Beginning Balance | 6,977,440 |
Number of options, Granted | 1,645,823 |
Number of options, Exercised | (1,014,569) |
Number of options, Forfeited | (129,393) |
Number of Options, Outstanding, Ending Balance | 7,479,301 |
Number of Options, Exercisable | 2,734,966 |
Weighted Average Exercise Price per Share, Outstanding, Beginning Balance | $ / shares | $ 15.43 |
Weighted Average Exercise Price per Share, Granted | $ / shares | 57.94 |
Weighted Average Exercise Price per Share, Exercised | $ / shares | 13.15 |
Weighted Average Exercise Price per Share, Forfeited | $ / shares | 20.71 |
Weighted Average Exercise Price per Share, Outstanding, Ending Balance | $ / shares | $ 25 |
Weighted Average Remaining Contractual Term, Outstanding | 8 years 3 months |
Aggregate Intrinsic Value, Outstanding | $ | $ 413,258 |
Loss Per Share - Schedule of Ba
Loss Per Share - Schedule of Basic and Diluted Net Loss Per Share (Detail) - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Earnings Per Share [Abstract] | ||
Net loss | $ (46,205) | $ (31,806) |
Weighted average shares outstanding, basic and diluted | 67,183 | 50,491 |
Net loss per share, basic and diluted | $ (0.69) | $ (0.63) |
Loss Per Share - Potential Dilu
Loss Per Share - Potential Dilutive Securities Excluded from Computation of Diluted Net Loss Per Common Share (Detail) - shares shares in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Potential dilutive securities excluded from computation of diluted net loss per common share | 7,922 | 7,491 |
Unvested Restricted Stock [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Potential dilutive securities excluded from computation of diluted net loss per common share | 443 | 248 |
Stock Options [Member] | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Potential dilutive securities excluded from computation of diluted net loss per common share | 7,479 | 7,243 |
Stockholders' Equity - Schedule
Stockholders' Equity - Schedule of Changes in Stockholders Equity (Detail) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Class Of Stock [Line Items] | ||
Beginning balance | $ 527,072 | $ 269,881 |
Beginning balance, shares | 66,234,056 | |
Exercise of stock options | $ 13,340 | 336 |
Exercise of stock options, shares | 1,014,569 | |
Equity-based compensation | $ 6,424 | 4,157 |
Other comprehensive income (loss) | (13) | 112 |
Net loss | (46,205) | (31,806) |
Ending balance | $ 545,873 | 247,759 |
Ending balance, shares | 67,890,334 | |
At The Market Offerings [Member] | ||
Class Of Stock [Line Items] | ||
Issuance of common stock | $ 45,255 | 5,079 |
Common Stock [Member] | ||
Class Of Stock [Line Items] | ||
Beginning balance | $ 7 | $ 5 |
Beginning balance, shares | 66,234,056 | 50,198,044 |
Exercise of stock options, shares | 1,014,569 | 53,579 |
Ending balance | $ 7 | $ 5 |
Ending balance, shares | 67,890,334 | 50,602,875 |
Common Stock [Member] | At The Market Offerings [Member] | ||
Class Of Stock [Line Items] | ||
Issuance of common stock, shares | 641,709 | 351,252 |
Additional Paid-In Capital [Member] | ||
Class Of Stock [Line Items] | ||
Beginning balance | $ 962,173 | $ 570,493 |
Exercise of stock options | 13,340 | 336 |
Equity-based compensation | 6,424 | 4,157 |
Ending balance | 1,027,192 | 580,065 |
Additional Paid-In Capital [Member] | At The Market Offerings [Member] | ||
Class Of Stock [Line Items] | ||
Issuance of common stock | 45,255 | 5,079 |
Accumulated Other Comprehensive Income (Loss) [Member] | ||
Class Of Stock [Line Items] | ||
Beginning balance | 1 | 261 |
Other comprehensive income (loss) | (13) | 112 |
Ending balance | (12) | 373 |
Accumulated Deficit [Member] | ||
Class Of Stock [Line Items] | ||
Beginning balance | (435,109) | (300,878) |
Net loss | (46,205) | (31,806) |
Ending balance | $ (481,314) | $ (332,684) |
Stockholders' Equity - Schedu_2
Stockholders' Equity - Schedule of Changes in Stockholders Equity (Parenthetical) (Detail) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
At The Market Offerings [Member] | ||
Class Of Stock [Line Items] | ||
Stock issuance cost,net | $ 52 | $ 48 |
Stockholders' Equity - Addition
Stockholders' Equity - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Millions | 1 Months Ended | 3 Months Ended | 12 Months Ended | 71 Months Ended | |
Aug. 31, 2019 | Mar. 31, 2021 | Mar. 31, 2020 | Dec. 31, 2019 | Mar. 31, 2021 | |
Class Of Stock [Line Items] | |||||
Proceeds from common stock offering | $ 1,165.3 | ||||
2019 Sales Agreement [Member] | |||||
Class Of Stock [Line Items] | |||||
Proceeds from common stock offering | $ 5.1 | $ 45.3 | |||
Percentage of gross proceeds from common stock as sales agent cash commission | 3.00% | ||||
Number of common stock issued upon conversion of shares | 641,709 | 351,252 | |||
Common stock price per share | $ 15.05 | $ 72.79 | $ 15.05 | ||
Proceeds from common stock offering | $ 47.4 | $ 47.4 | |||
2019 Sales Agreement [Member] | General and Administrative Expenses [Member] | |||||
Class Of Stock [Line Items] | |||||
Legal accounting and other fees | $ 0.1 | $ 0.1 | |||
2019 Sales Agreement [Member] | Maximum [Member] | |||||
Class Of Stock [Line Items] | |||||
Proceeds from common stock offering | $ 150 |