Cover Page
Cover Page - shares | 3 Months Ended | |
Mar. 31, 2023 | Apr. 28, 2023 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Document Quarterly Report | true | |
Document Period End Date | Mar. 31, 2023 | |
Document Fiscal Year Focus | 2023 | |
Document Transition Report | false | |
Entity File Number | 001-39319 | |
Entity Registrant Name | GENERATION BIO CO. | |
Entity Incorporation, State or Country Code | DE | |
Entity Tax Identification Number | 81-4301284 | |
Entity Address, Address Line One | 301 Binney Street | |
Entity Address, City or Town | Cambridge | |
Entity Address, State or Province | MA | |
Entity Address, Postal Zip Code | 02142 | |
City Area Code | 617 | |
Local Phone Number | 655-7500 | |
Title of 12(b) Security | Common Stock, $0.0001 Par Value | |
Trading Symbol | GBIO | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Accelerated Filer | |
Entity Small Business | true | |
Entity Emerging Growth Company | false | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding | 65,674,928 | |
Entity Central Index Key | 0001733294 | |
Current Fiscal Year End Date | --12-31 | |
Document Fiscal Period Focus | Q1 | |
Amendment Flag | false |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets - USD ($) $ in Thousands | Mar. 31, 2023 | Dec. 31, 2022 |
Current assets: | ||
Cash and cash equivalents | $ 95,650 | $ 93,171 |
Marketable securities | 192,999 | 185,920 |
Collaboration receivable | 47,500 | 0 |
Tenant receivable | 1,814 | 395 |
Prepaid expenses and other current assets | 6,770 | 7,530 |
Total current assets | 344,733 | 287,016 |
Property and equipment, net | 22,030 | 22,215 |
Operating lease right-of-use assets | 57,945 | 59,208 |
Restricted cash | 5,692 | 5,692 |
Deferred offering costs | 434 | 434 |
Other long-term assets | 583 | 1,699 |
Total assets | 431,417 | 376,264 |
Current liabilities: | ||
Accounts payable | 1,504 | 662 |
Accrued expenses and other current liabilities | 7,647 | 11,402 |
Contract with customer liability, current | 5,681 | 0 |
Operating lease liability | 7,905 | 7,086 |
Total current liabilities | 22,737 | 19,150 |
Contract with customer liability, noncurrent | 55,085 | 0 |
Operating lease liability, net of current portion | 74,456 | 74,621 |
Total liabilities | 152,278 | 93,771 |
Commitments and contingencies (Note 9) | ||
Stockholders' equity: | ||
Preferred stock, $0.0001 par value; 5,000,000 shares authorized and no shares issued or outstanding at March 31, 2023 and December 31, 2022 | 0 | 0 |
Common stock, $0.0001 par value; 150,000,000 shares authorized at March 31, 2023 and December 31, 2022; 65,535,663 and 59,505,437 shares issued and outstanding at March 31, 2023 and December 31, 2022, respectively | 7 | 6 |
Additional paid-in capital | 755,957 | 727,335 |
Accumulated other comprehensive income | 34 | (83) |
Accumulated deficit | (476,859) | (444,765) |
Total stockholders' equity | 279,139 | 282,493 |
Total liabilities and stockholders' equity | $ 431,417 | $ 376,264 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares | Mar. 31, 2023 | Dec. 31, 2022 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value per share | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized | 5,000,000 | 5,000,000 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value per share | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 150,000,000 | 150,000,000 |
Common stock, shares issued | 65,535,663 | 59,505,437 |
Common stock, shares outstanding | 65,535,663 | 59,505,437 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations and Comprehensive Loss - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2023 | Mar. 31, 2022 | |
Operating expenses: | ||
Research and development | $ 22,000 | $ 25,554 |
General and administrative | 12,866 | 9,790 |
Total operating expenses | 34,866 | 35,344 |
Loss from operations | (34,866) | (35,344) |
Other income: | ||
Other income and interest income, net | 2,772 | 345 |
Net loss and net loss attributable to common stockholders | $ (32,094) | $ (34,999) |
Net loss per share attributable to common stockholders, basic | $ (0.53) | $ (0.61) |
Net loss per share attributable to common stockholders, diluted | $ (0.53) | $ (0.61) |
Weighted average common shares outstanding, basic | 60,230,077 | 56,996,495 |
Weighted average common shares outstanding, diluted | 60,230,077 | 56,996,495 |
Comprehensive loss: | ||
Net loss | $ (32,094) | $ (34,999) |
Other comprehensive loss: | ||
Unrealized gains on marketable securities | 117 | 0 |
Comprehensive loss | $ (31,977) | $ (34,999) |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands | Common Stock [Member] | Additional Paid-in Capital [Member] | Accumulated Other Comprehensive Income [Member] | Accumulated Deficit [Member] | Total |
Beginning balance at Dec. 31, 2021 | $ 6 | $ 689,866 | $ (308,126) | $ 381,746 | |
Beginning balance, shares at Dec. 31, 2021 | 56,969,618 | ||||
Issuance of common stock upon exercise of stock options | 102 | 102 | |||
Issuance of common stock upon exercise of stock options, shares | 21,787 | ||||
Vesting of restricted common stock, shares | 12,723 | ||||
Stock-based compensation expense | 6,066 | 6,066 | |||
Net loss | (34,999) | (34,999) | |||
Ending balance at Mar. 31, 2022 | $ 6 | 696,034 | (343,125) | 352,915 | |
Ending balance, shares at Mar. 31, 2022 | 57,004,128 | ||||
Beginning balance at Dec. 31, 2022 | $ 6 | 727,335 | $ (83) | (444,765) | 282,493 |
Beginning balance, shares at Dec. 31, 2022 | 59,505,437 | ||||
Issuance of common stock in connection with the Moderna Agreement | $ 1 | 22,555 | 22,556 | ||
Issuance of common stock in connection with the Moderna Agreement, shares | 5,859,375 | ||||
Vesting of restricted common stock | (199) | (199) | |||
Vesting of restricted common stock, shares | 170,851 | ||||
Stock-based compensation expense | 6,266 | 6,266 | |||
Unrealized losses on marketable securities | 117 | 117 | |||
Net loss | (32,094) | (32,094) | |||
Ending balance at Mar. 31, 2023 | $ 7 | $ 755,957 | $ 34 | $ (476,859) | $ 279,139 |
Ending balance, shares at Mar. 31, 2023 | 65,535,663 |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2023 | Mar. 31, 2022 | |
Cash flows from operating activities: | ||
Net loss | $ (32,094) | $ (34,999) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Stock-based compensation expense | 6,266 | 6,066 |
Depreciation and amortization expense | 1,330 | 1,197 |
Amortization (accretion) of premium (discount) on marketable securities, net | (2,101) | 0 |
Loss on sale of property and equipment | 24 | 28 |
Changes in operating assets and liabilities: | ||
Collaboration receivable | (47,500) | 0 |
Tenant receivable | (1,419) | 0 |
Prepaid expenses and other current assets | 761 | (531) |
Operating lease right-of-use assets | 1,263 | 2,260 |
Other noncurrent assets | 1,115 | (5,176) |
Accounts payable | 816 | (684) |
Accrued expenses and other current liabilities | (4,292) | (3,407) |
Deferred revenue | 47,500 | 0 |
Operating lease liability | 654 | (189) |
Net cash used in operating activities | (27,677) | (35,435) |
Cash flows from investing activities: | ||
Purchases of property and equipment | (755) | (2,784) |
Purchases of marketable securities | (87,861) | 0 |
Maturities of marketable securities | 83,000 | 0 |
Net cash used in investing activities | (5,616) | (2,784) |
Cash flows from financing activities: | ||
Payment of share issuance costs | (29) | (50) |
Proceeds from issuance of common stock in connection with the Moderna Agreement | 36,000 | 0 |
Proceeds from exercise of stock options and other types of equity, net | 0 | 102 |
Tax withholding payments related to net share settlements of restricted stock units | (199) | 0 |
Net cash provided by financing activities | 35,772 | 52 |
Net increase (decrease) in cash, cash equivalents and restricted cash | 2,479 | (38,167) |
Cash, cash equivalents and restricted cash at beginning of period | 98,863 | 380,837 |
Cash, cash equivalents and restricted cash at end of period | 101,342 | 342,670 |
Supplemental disclosure of noncash investing and financing information: | ||
Purchases of property and equipment included in accounts payable and accrued expenses | 438 | 1,928 |
Unrealized gains on marketable securities | 117 | 0 |
Issuance costs included in accrued expenses | $ 149 | $ 0 |
Nature of the Business and Basi
Nature of the Business and Basis of Presentation | 3 Months Ended |
Mar. 31, 2023 | |
Accounting Policies [Abstract] | |
Nature of the Business and Basis of Presentation | 1. Nature of the Business and Basis of Presentation Generation Bio Co., or Generation Bio, was incorporated on October 21, 2016 as Torus Therapeutics, Inc. and subsequently changed its name to Generation Bio Co. Generation Bio Co. and its consolidated subsidiary, or the company, we, our or us, are innovating genetic medicines to provide durable, redosable treatments for potentially hundreds of millions of patients living with rare and prevalent diseases. Our non-viral genetic medicines platform incorporates our high-capacity DNA construct called closed-ended DNA, or ceDNA; our cell-targeted lipid nanoparticle delivery system, or ctLNP; and our highly scalable capsid-free manufacturing process that uses our proprietary cell-free rapid enzymatic synthesis, or RES, to produce ceDNA. Using our approach, we are developing novel genetic medicines to provide targeted delivery of genetic payloads that include large and multiple genes to a range of cell types across a broad array of diseases. We are also engineering our genetic medicines to be redosable, which may enable individualized patient titration to reach the desired therapeutic expression and to maintain efficacy throughout a patient’s life. We are headquartered in Cambridge, Massachusetts. We are subject to risks and uncertainties common to early-stage companies in the biotechnology industry, including, but not limited to, development by competitors of new technological innovations, dependence on key personnel, protection of proprietary technology, compliance with government regulations, the ability to establish clinical- and commercial-scale manufacturing processes and the ability to secure additional capital to fund operations. Programs currently under development will require significant additional research and development efforts, including extensive preclinical and clinical testing and regulatory approval prior to commercialization of a product. These efforts require significant amounts of additional capital, adequate personnel and infrastructure and extensive compliance-reporting capabilities. Even if our development efforts are successful, it is uncertain when, if ever, we will realize significant revenue from product sales. The accompanying condensed consolidated financial statements have been prepared on the basis of continuity of operations, realization of assets and the satisfaction of liabilities and commitments in the ordinary course of business. Since inception, we have funded our operations with proceeds from the sale of instruments convertible into convertible preferred stock (which converted into convertible preferred stock in 2017), sales of convertible preferred stock (which converted into common stock in 2020), and sales of common stock in underwritten public offerings, “at-the-market” offerings, and in a private placement, as well as payments pursuant to our collaboration with ModernaTX, Inc., or Moderna. We have incurred recurring losses, including net losses of $32.1 million for the three months ended March 31, 2023 and $35.0 million for the three months ended March 31, 2022. As of March 31, 2023, we had an accumulated deficit of $476.9 million. We expect to continue to generate operating losses in the foreseeable future. As of May 10, 2023, the issuance date of these condensed consolidated financial statements, we expect that our cash, cash equivalents and marketable securities will be sufficient to fund our operating expenses and capital expenditure requirements for at least 12 months. We will need to obtain additional funding through public or private equity offerings, debt financings, collaborations, strategic alliances and/or licensing arrangements. We may not be able to obtain financing on acceptable terms, or at all, and we may not be able to enter into additional collaborative or strategic alliances or licensing arrangements. The terms of any financing may adversely affect the holdings or the rights of our stockholders. Arrangements with collaborators or others may require us to relinquish rights to certain of our technologies or programs. If we are unable to obtain funding, we could be forced to delay, reduce or eliminate some or all of our research and development programs, pipeline expansion or commercialization efforts, which could adversely affect our business prospects. Although management will continue to pursue these plans, there is no assurance that we will be successful in obtaining sufficient funding on terms acceptable to us to fund continuing operations when needed or at all. The accompanying condensed consolidated financial statements reflect the operations of Generation Bio and our wholly owned subsidiary, Generation Bio Securities Corporation. Intercompany balances and transactions have been eliminated in consolidation. The accompanying condensed consolidated financial statements have been prepared in conformity with generally accepted accounting principles in the United States of America, 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. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 3 Months Ended |
Mar. 31, 2023 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies 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 and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of expenses during the reporting periods. Significant estimates and assumptions reflected in these condensed consolidated financial statements include, but are not limited to, the accrual of research and development expenses and stock-based compensation expense. We base our estimates on historical experience, known trends and other market-specific or other relevant factors that we believe to be reasonable under the circumstances. On an ongoing basis, management evaluates its estimates, as there are changes in circumstances, facts and experience. Changes in estimates are recorded in the period in which they become known. Actual results may differ from those estimates or assumptions. Unaudited interim financial information The condensed consolidated balance sheet as of December 31, 2022 was derived from audited financial statements but does not include all disclosures required by GAAP. The accompanying unaudited financial statements as of March 31, 2023 and for the three months ended March 31, 2023 and 2022 have been prepared by us pursuant to the rules and regulations of the Securities and Exchange Commission, or SEC, for interim financial statements. Certain information and footnote disclosures normally included in financial statements prepared in accordance with GAAP have been condensed or omitted pursuant to such rules and regulations. These financial statements should be read in conjunction with our audited financial statements included in our Annual Report on Form 10-K that was most recently filed with the SEC. In the opinion of management, all adjustments, consisting only of normal recurring adjustments necessary for a fair presentation of our financial position as of March 31, 2023, the results of operations for the three months ended March 31, 2023 and 2022, and cash flows for the three months ended March 31, 2023 and 2022 have been made. The results of operations for the three months ended March 31, 2023 are not necessarily indicative of the results of operations that may be expected for the year ending December 31, 2023 or any other period. Our significant accounting policies are described in Note 2 of the Notes to Consolidated Financial Statements included in our Annual Report on Form 10-K for the year ended December 31, 2022. Concentrations of credit risk and of significant suppliers Financial instruments that potentially expose us to concentrations of credit risk consist primarily of cash, cash equivalents, and marketable securities. We believe that we are not exposed to significant credit risk due to the financial strength of the national depository institutions in which our cash, cash equivalents, and marketable securities are held. We maintain our cash equivalents in money market funds that invest in U.S. treasury securities. We have adopted an investment policy that limits the amounts that we may invest in the securities of a single issuer with the exclusion of the U.S. government. We have not experienced any credit losses. We are dependent on a small number of third-party suppliers for our drug substance and drug product. In particular, we rely, and expect to continue to rely, on third-party suppliers for certain materials and components required for the production of any product candidates we may develop for our programs. These programs could be adversely affected by a significant interruption in the supply process. Revenue Recognition We enter into collaboration agreements that are within the scope of ASC Topic 606, “Revenue from Contracts with Customers”, or ASC 606, under which we license rights to certain of our potential product candidates and perform research and development services. The terms of these contracts typically include payment of the following: non-refundable, upfront fees; reimbursement of research and development costs; development, regulatory, and commercial milestone payments; royalties on net sales of licensed products, and a premium or discount on the sale of our common stock. Under ASC 606, an entity recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the entity expects to receive in exchange for those goods or services. To determine the appropriate amount of revenue to be recognized for contracts determined to be within the scope of ASC 606, we perform the following five steps: (i) identification of the promised goods or services in the contract; (ii) determination of whether the promised goods or services are performance obligations, including whether they are distinct in the context of the contract; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations; and (v) recognition of revenue when, or as, we satisfy each performance obligation. We only apply the five-step model to contracts when it is probable that we will collect consideration we are entitled to in exchange for the goods or services we transfer to the customer. The promised goods or services in our arrangements typically consist of license rights to our intellectual property and research and development services. We provide options to additional items in the contract, which will be accounted for as separate contracts if and when the other party elects to exercise such options, unless the option provides a material right to such party. We evaluate the other party’s options for material rights, or options to acquire additional goods or services for free or at a discount. If the other party’s options are determined to represent a material right, the material right is recognized as a separate performance obligation at the outset of the contract. Performance obligations are promised goods or services in a contract to transfer a distinct good or service to the other party and are considered distinct when (i) the other party can benefit from the good or service on its own or together with other readily available resources and (ii) the promised good or service is separately identifiable from other promises in the contract. In assessing whether promised goods or services are distinct, we consider factors such as the stage of development of the underlying intellectual property, the capabilities of the other party to develop the intellectual property on its own or whether the required expertise is readily available and whether the goods or services are integral or dependent to other goods or services in the contract. We estimate the transaction price based on the amount expected to be received for transferring the promised goods or services in the contract. The consideration may include fixed consideration or variable consideration. At the inception of each contract that includes variable consideration, we evaluate the number of potential payments and the likelihood that the payments will be received. We utilize either the most likely amount method or expected amount method to estimate the amount expected to be received based on which method best predicts the amount expected to be received. The amount of variable consideration that is included in the transaction price may be constrained and is included in the transaction price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Our contracts include development, regulatory, and commercial milestone payments that will be assessed under the most likely amount method and constrained if it is probable that a significant revenue reversal would occur. Milestone payments that are not within our control or the counterparty’s control, such as regulatory approvals, are not considered probable of being achieved until those approvals are received. At the end of each reporting period, we re-evaluate the probability of achievement of such development, regulatory, and commercial milestones and any related constraint, and if necessary, we adjust our estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis, which would affect collaboration revenues in the period of adjustment. To date, we have not recognized any consideration related to the achievement of development, regulatory, or commercial milestone revenue resulting from our collaboration contracts. For contracts that include sales-based royalties, including milestone payments based on the level of sales, and where the license is deemed to be the predominant item to which the royalties relate, we recognize revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). To date, we have not recognized any consideration related to sales-based royalty revenue resulting from our collaboration contract. We allocate the transaction price based on the estimated stand-alone selling price of each of the performance obligations. We must develop assumptions that require judgment to determine the stand-alone selling price for each performance obligation identified in the contract. We utilize key assumptions to determine the stand-alone selling price for service obligations, which may include other comparable transactions, pricing considered in negotiating the transaction and the estimated costs. Additionally, in determining the standalone selling price for material rights, we utilize comparable transactions, clinical trial success probabilities, and estimates of option exercise likelihood. Variable consideration is allocated specifically to one or more performance obligations in a contract when the terms of the variable consideration relate to the satisfaction of the performance obligation and the resulting amounts allocated are consistent with the amounts we would expect to receive for the satisfaction of each performance obligation. The consideration allocated to each performance obligation is recognized as revenue when control is transferred for the related goods or services. For performance obligations that consist of licenses and other promises, we utilize judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, the appropriate method of measuring progress. We evaluate the measure of progress each reporting period and, if necessary, adjust the measure of performance and related revenue recognition. Upfront payments and fees are recorded as deferred revenue upon receipt or when due until we perform our obligations. Amounts are recorded as accounts receivable when our right to consideration is unconditional. |
Marketable Securities and Fair
Marketable Securities and Fair Value Measurements | 3 Months Ended |
Mar. 31, 2023 | |
Marketable Securities and Fair Value Measurements | |
Marketable Securities and Fair Value Measurements | 3. Marketable Securities and Fair Value Measurements The following tables present our marketable securities by security type: As of March 31, 2023 Gross Gross Amortized Unrealized Unrealized Fair (in thousands) Cost Gains Losses Value U.S. treasury securities $ 192,965 $ 38 $ (4) $ 192,999 As of December 31, 2022 Gross Gross Amortized Unrealized Unrealized Fair (in thousands) Cost Gains Losses Value U.S. treasury securities $ 186,003 $ 13 $ (96) $ 185,920 As of March 31, 2023 and December 31, 2022, our marketable securities consisted of investments that mature within one year. The following tables present our assets that are measured at fair value on a recurring basis and indicate the level within the fair value hierarchy of the valuation techniques that we utilized to determine such fair value: Fair Value Measurements at March 31, 2023 Using: (in thousands) Level 1 Level 2 Level 3 Total Cash equivalents: Money market funds $ 48,960 $ — $ — $ 48,960 Marketable securities: U.S. treasury securities — 192,999 — 192,999 Totals $ 48,960 $ 192,999 $ — $ 241,959 Fair Value Measurements at December 31, 2022 Using: (in thousands) Level 1 Level 2 Level 3 Total Cash equivalents: Money market funds $ 77,010 $ — $ — $ 77,010 Marketable securities: U.S. treasury securities — 185,920 — 185,920 Totals $ 77,010 $ 185,920 $ — $ 262,930 |
Collaboration and License Agree
Collaboration and License Agreement | 3 Months Ended |
Mar. 31, 2023 | |
Collaboration and License Agreement | |
Collaboration and License Agreement | 4. Collaboration and License Agreement Moderna Collaboration and License Agreement In March 2023, we entered into a Collaboration and License Agreement, or the Collaboration Agreement, with Moderna to collaborate on developing treatments for certain diseases by targeting delivery of nucleic acids to liver cells and certain cells outside of the liver. Under the Collaboration Agreement, the parties have agreed to collaborate on preclinical research programs relating to lipid nanoparticle, or LNP, delivery systems and nucleic acid payloads, with each party obtaining certain rights to intellectual property used in and arising out of such research programs. Each party will be solely responsible for its own clinical development and commercialization of products under the Collaboration Agreement. Moderna will reimburse us for the internal and external costs incurred by us in conducting the research programs, to the extent consistent with such research plans and budgets. Moderna has exclusive options, upon payment of option exercise fees, to obtain worldwide, exclusive, sublicensable licenses under specified company intellectual property to develop, manufacture and commercialize (a) products comprising LNP delivery systems and nucleic acid payloads that are directed to (i) up to two liver targets, (ii) up to two non-liver targets and (iii) a third liver or non-liver target and (b) “Exclusive Targets,” which are Independent Program Products (as defined below) that include messenger RNA, or mRNA ,that are directed to gene and protein targets in any of certain agreed-upon cell types, referred to as the Cell Target Types. Subject to the our exclusivity obligations described below, each party has granted to the other a worldwide, non-exclusive, sublicensable license under certain LNP-related intellectual property arising out of the non-liver ctLNP program, or the Joint Collaboration ctLNP Intellectual Property, to develop, manufacture and commercialize products comprising LNP delivery systems and nucleic acid payloads directed to gene and protein targets in any of the Cell Target Types, or Independent Program Products. Each party is obligated to use commercially reasonable efforts to complete the activities assigned to it under the research plans, and Moderna is further obligated to use commercially reasonable efforts to develop, seek regulatory approval for and commercialize at least one product directed to each target for which Moderna exercises its exclusive license option in at least one indication in the United States and in specified European countries. We have agreed not to, directly or indirectly, alone or with, for or through any third party, develop, manufacture, commercialize or exploit (a) products containing mRNA that are directed to any of the Cell Target Types, during an agreed-upon exclusivity period, which may be extended by payment of extension fees, (b) products directed to any liver target or non-liver target during the option periods for those targets, (c) products directed to any liver target or non-liver target for which Moderna has exercised its exclusive license option or (d) products containing mRNA that are directed to any Exclusive Target for which Moderna has exercised its exclusive license option. Under the terms of the Collaboration Agreement, in April 2023, Moderna made an upfront payment to us of $40.0 million, as well as $7.5 million in prepaid research funding. In addition, we are eligible to receive up to $1.8 billion in milestone payments upon the achievement of specified development, regulatory, commercial, and sales milestone events, research term extension fees and exclusivity extension fees. Subject to reductions in specified circumstances, we will also be entitled to receive tiered royalties: (i) ranging from high-single-digits to low-double-digits on sales of licensed products that are directed to any liver target or non-liver target with respect to which Moderna has exercised its exclusive license option, and (ii) in the single digits on sales of Independent Program Products, including the exclusively licensed Independent Program Products directed to the Exclusive Targets. In consideration for the non-exclusive license granted by Moderna to us under the Joint Collaboration ctLNP Intellectual Property, we have agreed to pay Moderna tiered royalties in the single digits on sales of Independent Program Products that include mRNA, subject to reductions in specified circumstances. Royalties will be paid by each party, on a licensed product-by-licensed product and country-by-country basis, until the latest to occur of: (i) expiration of the last-to-expire of specified licensed patent rights; (ii) expiration of regulatory exclusivity; or (iii) ten In addition, in connection with the execution of the Collaboration Agreement, we entered into a Share Purchase Agreement, or the Share Purchase Agreement, with Moderna, pursuant to which we issued and sold 5,859,375 shares of our common stock to Moderna, at a price of $6.14 per share, for an aggregate purchase price of $36.0 million, which closed concurrently with the execution of the Collaboration Agreement. Under the Share Purchase Agreement, Moderna has the right, subject to certain terms and conditions, to purchase up to 3.06% of the outstanding shares of our common stock (on a post-closing basis) in connection with a future equity financing of at least $25.0 million by us. Moderna Agreement Assessment We assessed the promised goods and services under the Collaboration Agreement, in accordance with ASC 606. At inception, the Collaboration Agreement included one combined performance obligation, which includes the license to the ctLNP technology to target indications outside of the liver and the related research services to develop such technology, as the two items are not distinct in context of the contract. The Collaboration Agreement also provides Moderna with options to receive additional research services and options to receive exclusive licenses. The options to receive exclusive licenses allow Moderna to develop and commercialize product candidates that utilize our ctLNP and ceDNA technology for targets within the liver, as well as utilizing the ctLNP technology to be developed as part of the Collaboration Agreement and our ceDNA technology for targets outside the liver. These options are considered to be a priced at a discount to its standalone selling price and therefore are considered to be material rights. The transaction price will initially include the $40.0 million upfront fee, the premium paid over the fair value of the common stock of $13.3 million in connection with shares issued and sold to Moderna under the Share Purchase Agreement, and the estimated revenue associated with the payment for research services, including $7.5 million in prepaid research services. We utilized the expected amount method to determine the amount of reimbursement for these activities. We utilized the most likely amount method to determine the amount of consideration to include in the transaction price related to any variable consideration related to exclusivity fees, and milestones, and the royalty payments are constrained based on the royalty constraint. No amounts are included in the transaction price related to these elements. The transaction price was allocated to the performance obligation and material rights based on the relative estimated standalone selling prices of each element, over which management has applied significant judgment. We developed the estimated standalone selling price for combined performance obligation and each of the options to receive licenses primarily based on the probability-weighted present value of expected future cash flows associated with each license related to each specific program and an estimate of the costs to provide services including a reasonable return. In developing such estimate, we also considered applicable market conditions and relevant entity-specific factors, including those factors contemplated in negotiating the agreement, the probability of success and the time needed to commercialize a product candidate pursuant to the associated license. We measure proportional performance of the combined performance obligation over time using an input method based on cost incurred relative to the total estimated costs on a quarterly basis by determining the proportion of effort incurred as a percentage of total effort we expect to expend. Any changes to these estimates will be recognized in the period in which they change as a cumulative catch up. All allocated consideration for the material rights is deferred until such time that Moderna exercises its options or the right to exercise the options expires. Upon exercise, we will determine the appropriate revenue recognition methodology and any other implications on the accounting treatment for the arrangement. As of March 31, 2023, no services have been performed under the combined performance obligation and therefore no amounts have been recognized. |
Property and equipment, net
Property and equipment, net | 3 Months Ended |
Mar. 31, 2023 | |
Property and equipment, net. | |
Property and equipment, net | 5. Property and equipment, net Property and equipment, net consisted of the following: March 31, December 31, (in thousands) 2023 2022 Laboratory equipment $ 13,595 $ 13,619 Computer equipment and software 1,262 1,189 Furniture and fixtures 1,146 1,146 Leasehold improvements 20,786 20,786 Construction in progress 1,068 13 37,857 36,753 Less: Accumulated depreciation and amortization (15,827) (14,538) Total $ 22,030 $ 22,215 Depreciation and amortization expense for the three months ended March 31, 2023 and 2022 was $1.3 million and $1.2 million, respectively. |
Accrued Expenses
Accrued Expenses | 3 Months Ended |
Mar. 31, 2023 | |
Accrued Expenses | |
Accrued Expenses | 6. Accrued Expenses Accrued expenses and other current liabilities consisted of the following: March 31, December 31, (in thousands) 2023 2022 Accrued employee compensation and benefits $ 3,015 $ 7,970 Accrued external research and development expenses 2,406 1,959 Accrued professional fees 1,253 1,047 Property and equipment 388 — Other 585 426 Total $ 7,647 $ 11,402 |
Equity
Equity | 3 Months Ended |
Mar. 31, 2023 | |
Equity | |
Equity | 7. Equity As of March 31, 2023, our amended and restated certificate of incorporation authorizes us to issue 150,000,000 shares of common stock, par value $0.0001 per share, and 5,000,000 shares of preferred stock, par value $0.0001 per share, all of which preferred stock is undesignated. In August 2021, we entered into an “at-the-market” sales agreement pursuant to which we may, from time to time, sell shares of our common stock having an aggregate offering price of up to $250.0 million. As of May 10, 2023, the issuance date of these condensed consolidated financial statements, we have issued and sold 1,795,524 shares of our common stock pursuant to this sales agreement resulting in net proceeds of $12.3 million. In March 2023, in connection with the Share Purchase Agreement with Moderna, we issued and sold 5,859,375 shares of our common stock to Moderna at a price of $6.14 per share for an aggregate purchase price of $36.0 million. For additional information, refer to Note 4, Collaboration and License Agreements. Each share of common stock entitles the holder to one vote on all matters submitted to a vote of our stockholders. Holders of common stock are not entitled to receive dividends, unless declared by the board of directors. |
Stock-Based Compensation
Stock-Based Compensation | 3 Months Ended |
Mar. 31, 2023 | |
Stock-Based Compensation | |
Stock-Based compensation | 8. Stock-Based Compensation Stock incentive plans Our 2017 Stock Incentive Plan, or the 2017 Plan, provided for us to grant incentive stock options or nonstatutory stock options, restricted stock, restricted stock units and other equity awards to employees, non-employees, and directors. In May 2020, our board of directors adopted, and in June 2020, our stockholders approved, the 2020 Stock Incentive Plan, or the 2020 Plan, and, together with the 2017 Plan, the Plans, which became effective on June 11, 2020. The 2020 Plan provides for the grant of incentive stock options, nonstatutory stock options, stock appreciation rights, restricted stock awards, restricted stock units and other stock-based awards. The number of shares of common stock reserved for issuance under the 2020 Plan is the sum of (1) 2,547,698 shares; plus (2) the number of shares (up to a maximum of 7,173,014 shares) as was equal to the sum of (x) the number of shares of common stock reserved for issuance under the 2017 Plan that remained available for grant under the 2017 Plan on June 11, 2020 and (y) the number of shares of common stock subject to outstanding awards granted under the 2017 Plan that expire, terminate or are otherwise surrendered, cancelled, forfeited or repurchased by us at their original issuance price pursuant to a contractual repurchase right; plus (3) an annual increase, to be added on the first day of each fiscal year, beginning with the fiscal year ending December 31, 2021 and continuing until, and including, the fiscal year ending December 31, 2030, equal to the lesser of (i) 4% of the number of shares of common stock outstanding on such date, and (ii) an amount determined by the board of directors. In January 2021, 2022 and 2023, the number of shares of common stock authorized for issuance under the 2020 Plan was increased from 10,275,717 shares to 12,154,517 shares, from 12,154,517 shares to 14,433,745 shares, and from 14,433,745 shares to 16,813,962 shares, respectively. Upon the effectiveness of the 2020 Plan, we ceased granting additional awards under the 2017 Plan. The Plans are administered by the board of directors or, at the discretion of the board of directors, by a committee of the board of directors. The exercise prices, vesting and other restrictions on any award under the Plans are determined at the discretion of the board of directors, or its committee if so delegated. Stock options granted under the Plans with service-based vesting conditions generally vest over four years and expire after ten years. The exercise price for stock options granted is not less than the fair value of common stock as of the date of grant. Prior to our IPO, fair value of common stock was determined by the board of directors. Subsequent to our IPO, fair value of common stock is based on quoted market prices. As of March 31, 2023, 1,207,324 shares remained available for future issuance under the 2020 Plan. Shares subject to outstanding awards granted under the Plans that expire, terminate or are otherwise surrendered, cancelled, forfeited or repurchased by us at their original issuance price pursuant to a contractual repurchase right will be available for future awards under the 2020 Plan. Grant of stock options During the three months ended March 31, 2023, we granted time-based options to certain employees for the purchase of an aggregate of 1,365,391 shares of common stock with a weighted average grant date fair value of $3.68 per share that vest over a weighted average period of approximately four years. Restricted stock units During the three months ended March 31, 2023, we issued 695,595 restricted stock units with a fair value of $3.3 million that vest over a weighted average period of approximately 3.9 years. Employee stock purchase plan In May 2020, our board of directors adopted, and in June 2020, our stockholders approved, the 2020 Employee Stock Purchase Plan, or the 2020 ESPP, which became effective June 11, 2020. The 2020 ESPP is administered by our board of directors or by a committee appointed by the board of directors. The number of shares of common stock authorized for issuance under the 2020 ESPP automatically increases on the first day of each fiscal year, beginning with the fiscal year that commenced on January 1, 2021 and continuing for each fiscal year until, and including the fiscal year commencing on, January 1, 2030, in an amount equal to the lowest of (1) 1,302,157 shares of common stock, (2) 1% of the number of shares of common stock outstanding on such date, and (3) an amount determined by the board of directors. In January 2021, 2022, and 2023, the number of shares of common stock authorized for issuance under the 2020 ESPP was increased from 481,231 shares to 950,931 shares, from 950,931 shares to 1,520,738 shares, and from 1,520,738 shares to 2,115,792 shares, respectively. As of March 31, 2023, 1,933,830 shares remained available for issuance under the 2020 ESPP. Stock-based compensation Stock-based compensation expense was classified in the condensed consolidated statements of operations and comprehensive loss as follows: Three Months Ended March 31, (in thousands) 2023 2022 Research and development expenses $ 2,855 $ 3,151 General and administrative expenses 3,411 2,915 Total $ 6,266 $ 6,066 As of March 31, 2023, total unrecognized compensation cost related to unvested time-based stock options and restricted stock units was $42.6 million, with $35.8 million expected to be recognized over a weighted average period of 2.9 years and $6.8 million expected to be recognized over a weighted average period of 2.3 years, respectively. Additionally, as of March 31, 2023, we had unrecognized compensation cost related to unvested stock options with performance-based vesting conditions for which performance has not been deemed probable of $1.7 million. |
Commitments and Contingencies
Commitments and Contingencies | 3 Months Ended |
Mar. 31, 2023 | |
Commitments and Contingencies. | |
Commitments and Contingencies | 9. Commitments and Contingencies 401(k) Plan We have a defined-contribution plan under Section 401(k) of the Internal Revenue Code of 1986, as amended, or the 401(k) Plan. The 401(k) Plan covers all employees who meet defined minimum age and service requirements and allows participants to contribute a portion of their annual compensation on a pre-tax and/or after-tax basis. In September 2020, we adopted a match program, beginning on January 1, 2021, for employee contributions to the 401(k) Plan up to a maximum of four percent of the employee’s salary , subject to the maximums established under the Indemnification agreements In the ordinary course of business, we may provide indemnification of varying scope and terms to vendors, lessors, contract research organizations, business partners and other parties with respect to certain matters including, but not limited to, losses arising out of breach of such agreements or from intellectual property infringement claims made by third parties. In addition, we have entered into indemnification agreements with members of our board of directors and our officers that will require us, among other things, to indemnify them against certain liabilities that may arise by reason of their status or service as directors or officers. The maximum potential amount of future payments we could be required to make under these indemnification agreements is, in many cases, unlimited. We have not incurred any material costs as a result of such indemnifications and are not currently aware of any indemnification claims. Legal proceedings We, from time to time, may be party to litigation arising in the ordinary course of business. We were not subject to any material legal proceedings during the three months ended March 31, 2023. |
Net Loss per Share
Net Loss per Share | 3 Months Ended |
Mar. 31, 2023 | |
Net Loss per Share | |
Net Loss per Share | 10. Net Loss per Share We have generated a net loss in all periods presented, therefore the basic and diluted net loss per share attributable to common stockholders are the same as the inclusion of the potentially dilutive securities would be anti-dilutive. We excluded the following potential common shares, presented based on amounts outstanding at each period end, from the computation of diluted net loss per share attributable to common stockholders for the periods indicated: March 31, 2023 2022 Unvested restricted stock units 1,411,002 1,537,744 Stock options to purchase common stock 10,032,920 8,473,696 Total 11,443,922 10,011,440 |
Related Parties
Related Parties | 3 Months Ended |
Mar. 31, 2023 | |
Related Parties | |
Related Parties | 11. Related Parties In March 2023, we entered into the Collaboration Agreement with Moderna. In connection with the Share Purchase Agreement, we issued and sold 5,859,375 shares of our common stock to Moderna, which resulted in Moderna becoming the beneficial owner of 8.9% of our outstanding common stock and a related party. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 3 Months Ended |
Mar. 31, 2023 | |
Accounting Policies [Abstract] | |
Use of estimates | 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 and liabilities, the disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of expenses during the reporting periods. Significant estimates and assumptions reflected in these condensed consolidated financial statements include, but are not limited to, the accrual of research and development expenses and stock-based compensation expense. We base our estimates on historical experience, known trends and other market-specific or other relevant factors that we believe to be reasonable under the circumstances. On an ongoing basis, management evaluates its estimates, as there are changes in circumstances, facts and experience. Changes in estimates are recorded in the period in which they become known. Actual results may differ from those estimates or assumptions. |
Unaudited interim financial information | Unaudited interim financial information The condensed consolidated balance sheet as of December 31, 2022 was derived from audited financial statements but does not include all disclosures required by GAAP. The accompanying unaudited financial statements as of March 31, 2023 and for the three months ended March 31, 2023 and 2022 have been prepared by us pursuant to the rules and regulations of the Securities and Exchange Commission, or SEC, for interim financial statements. Certain information and footnote disclosures normally included in financial statements prepared in accordance with GAAP have been condensed or omitted pursuant to such rules and regulations. These financial statements should be read in conjunction with our audited financial statements included in our Annual Report on Form 10-K that was most recently filed with the SEC. In the opinion of management, all adjustments, consisting only of normal recurring adjustments necessary for a fair presentation of our financial position as of March 31, 2023, the results of operations for the three months ended March 31, 2023 and 2022, and cash flows for the three months ended March 31, 2023 and 2022 have been made. The results of operations for the three months ended March 31, 2023 are not necessarily indicative of the results of operations that may be expected for the year ending December 31, 2023 or any other period. Our significant accounting policies are described in Note 2 of the Notes to Consolidated Financial Statements included in our Annual Report on Form 10-K for the year ended December 31, 2022. |
Concentrations of credit risk and of significant suppliers | Concentrations of credit risk and of significant suppliers Financial instruments that potentially expose us to concentrations of credit risk consist primarily of cash, cash equivalents, and marketable securities. We believe that we are not exposed to significant credit risk due to the financial strength of the national depository institutions in which our cash, cash equivalents, and marketable securities are held. We maintain our cash equivalents in money market funds that invest in U.S. treasury securities. We have adopted an investment policy that limits the amounts that we may invest in the securities of a single issuer with the exclusion of the U.S. government. We have not experienced any credit losses. We are dependent on a small number of third-party suppliers for our drug substance and drug product. In particular, we rely, and expect to continue to rely, on third-party suppliers for certain materials and components required for the production of any product candidates we may develop for our programs. These programs could be adversely affected by a significant interruption in the supply process. |
Revenue Recognition | Revenue Recognition We enter into collaboration agreements that are within the scope of ASC Topic 606, “Revenue from Contracts with Customers”, or ASC 606, under which we license rights to certain of our potential product candidates and perform research and development services. The terms of these contracts typically include payment of the following: non-refundable, upfront fees; reimbursement of research and development costs; development, regulatory, and commercial milestone payments; royalties on net sales of licensed products, and a premium or discount on the sale of our common stock. Under ASC 606, an entity recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the entity expects to receive in exchange for those goods or services. To determine the appropriate amount of revenue to be recognized for contracts determined to be within the scope of ASC 606, we perform the following five steps: (i) identification of the promised goods or services in the contract; (ii) determination of whether the promised goods or services are performance obligations, including whether they are distinct in the context of the contract; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations; and (v) recognition of revenue when, or as, we satisfy each performance obligation. We only apply the five-step model to contracts when it is probable that we will collect consideration we are entitled to in exchange for the goods or services we transfer to the customer. The promised goods or services in our arrangements typically consist of license rights to our intellectual property and research and development services. We provide options to additional items in the contract, which will be accounted for as separate contracts if and when the other party elects to exercise such options, unless the option provides a material right to such party. We evaluate the other party’s options for material rights, or options to acquire additional goods or services for free or at a discount. If the other party’s options are determined to represent a material right, the material right is recognized as a separate performance obligation at the outset of the contract. Performance obligations are promised goods or services in a contract to transfer a distinct good or service to the other party and are considered distinct when (i) the other party can benefit from the good or service on its own or together with other readily available resources and (ii) the promised good or service is separately identifiable from other promises in the contract. In assessing whether promised goods or services are distinct, we consider factors such as the stage of development of the underlying intellectual property, the capabilities of the other party to develop the intellectual property on its own or whether the required expertise is readily available and whether the goods or services are integral or dependent to other goods or services in the contract. We estimate the transaction price based on the amount expected to be received for transferring the promised goods or services in the contract. The consideration may include fixed consideration or variable consideration. At the inception of each contract that includes variable consideration, we evaluate the number of potential payments and the likelihood that the payments will be received. We utilize either the most likely amount method or expected amount method to estimate the amount expected to be received based on which method best predicts the amount expected to be received. The amount of variable consideration that is included in the transaction price may be constrained and is included in the transaction price only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Our contracts include development, regulatory, and commercial milestone payments that will be assessed under the most likely amount method and constrained if it is probable that a significant revenue reversal would occur. Milestone payments that are not within our control or the counterparty’s control, such as regulatory approvals, are not considered probable of being achieved until those approvals are received. At the end of each reporting period, we re-evaluate the probability of achievement of such development, regulatory, and commercial milestones and any related constraint, and if necessary, we adjust our estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis, which would affect collaboration revenues in the period of adjustment. To date, we have not recognized any consideration related to the achievement of development, regulatory, or commercial milestone revenue resulting from our collaboration contracts. For contracts that include sales-based royalties, including milestone payments based on the level of sales, and where the license is deemed to be the predominant item to which the royalties relate, we recognize revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). To date, we have not recognized any consideration related to sales-based royalty revenue resulting from our collaboration contract. We allocate the transaction price based on the estimated stand-alone selling price of each of the performance obligations. We must develop assumptions that require judgment to determine the stand-alone selling price for each performance obligation identified in the contract. We utilize key assumptions to determine the stand-alone selling price for service obligations, which may include other comparable transactions, pricing considered in negotiating the transaction and the estimated costs. Additionally, in determining the standalone selling price for material rights, we utilize comparable transactions, clinical trial success probabilities, and estimates of option exercise likelihood. Variable consideration is allocated specifically to one or more performance obligations in a contract when the terms of the variable consideration relate to the satisfaction of the performance obligation and the resulting amounts allocated are consistent with the amounts we would expect to receive for the satisfaction of each performance obligation. The consideration allocated to each performance obligation is recognized as revenue when control is transferred for the related goods or services. For performance obligations that consist of licenses and other promises, we utilize judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, the appropriate method of measuring progress. We evaluate the measure of progress each reporting period and, if necessary, adjust the measure of performance and related revenue recognition. Upfront payments and fees are recorded as deferred revenue upon receipt or when due until we perform our obligations. Amounts are recorded as accounts receivable when our right to consideration is unconditional. |
Marketable Securities and Fai_2
Marketable Securities and Fair Value Measurements (Tables) | 3 Months Ended |
Mar. 31, 2023 | |
Marketable Securities and Fair Value Measurements | |
Summary of marketable securities | The following tables present our marketable securities by security type: As of March 31, 2023 Gross Gross Amortized Unrealized Unrealized Fair (in thousands) Cost Gains Losses Value U.S. treasury securities $ 192,965 $ 38 $ (4) $ 192,999 As of December 31, 2022 Gross Gross Amortized Unrealized Unrealized Fair (in thousands) Cost Gains Losses Value U.S. treasury securities $ 186,003 $ 13 $ (96) $ 185,920 |
Summary of assets measured at fair value on a recurring basis | The following tables present our assets that are measured at fair value on a recurring basis and indicate the level within the fair value hierarchy of the valuation techniques that we utilized to determine such fair value: Fair Value Measurements at March 31, 2023 Using: (in thousands) Level 1 Level 2 Level 3 Total Cash equivalents: Money market funds $ 48,960 $ — $ — $ 48,960 Marketable securities: U.S. treasury securities — 192,999 — 192,999 Totals $ 48,960 $ 192,999 $ — $ 241,959 Fair Value Measurements at December 31, 2022 Using: (in thousands) Level 1 Level 2 Level 3 Total Cash equivalents: Money market funds $ 77,010 $ — $ — $ 77,010 Marketable securities: U.S. treasury securities — 185,920 — 185,920 Totals $ 77,010 $ 185,920 $ — $ 262,930 |
Property and equipment, net (Ta
Property and equipment, net (Tables) | 3 Months Ended |
Mar. 31, 2023 | |
Property and equipment, net. | |
Summary of Property and equipment, net | Property and equipment, net consisted of the following: March 31, December 31, (in thousands) 2023 2022 Laboratory equipment $ 13,595 $ 13,619 Computer equipment and software 1,262 1,189 Furniture and fixtures 1,146 1,146 Leasehold improvements 20,786 20,786 Construction in progress 1,068 13 37,857 36,753 Less: Accumulated depreciation and amortization (15,827) (14,538) Total $ 22,030 $ 22,215 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 3 Months Ended |
Mar. 31, 2023 | |
Accrued Expenses | |
Summary of accrued expenses | Accrued expenses and other current liabilities consisted of the following: March 31, December 31, (in thousands) 2023 2022 Accrued employee compensation and benefits $ 3,015 $ 7,970 Accrued external research and development expenses 2,406 1,959 Accrued professional fees 1,253 1,047 Property and equipment 388 — Other 585 426 Total $ 7,647 $ 11,402 |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 3 Months Ended |
Mar. 31, 2023 | |
Stock-Based Compensation | |
Summary of stock-based compensation expense was classified in the statements of operations and comprehensive loss | Three Months Ended March 31, (in thousands) 2023 2022 Research and development expenses $ 2,855 $ 3,151 General and administrative expenses 3,411 2,915 Total $ 6,266 $ 6,066 |
Net Loss per Share (Tables)
Net Loss per Share (Tables) | 3 Months Ended |
Mar. 31, 2023 | |
Net Loss per Share | |
Summary of potential dilutive securities, presented based on amounts outstanding | March 31, 2023 2022 Unvested restricted stock units 1,411,002 1,537,744 Stock options to purchase common stock 10,032,920 8,473,696 Total 11,443,922 10,011,440 |
Nature of the Business and Ba_2
Nature of the Business and Basis of Presentation - Additional information (Detail) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | ||
Aug. 31, 2021 | Mar. 31, 2023 | Mar. 31, 2022 | Dec. 31, 2022 | |
Date of incorporation | Oct. 21, 2016 | |||
Shares issued during the period | 1,795,524 | |||
Net proceeds from shares issued | $ 12,300 | |||
Net loss and net loss attributable to common stockholders | $ (32,094) | $ (34,999) | ||
Accumulated deficit | $ (476,859) | $ (444,765) | ||
Common Stock [Member] | ||||
Shares issued during the period | 5,859,375 |
Marketable Securities and Fai_3
Marketable Securities and Fair Value Measurements - Summary of marketable securities (Detail) - Fair Value, Recurring [Member] - U.S. treasury securities - USD ($) $ in Thousands | Mar. 31, 2023 | Dec. 31, 2022 |
Marketable Securities [Line Items] | ||
Amortized Cost | $ 192,965 | $ 186,003 |
Gross Unrealized Gains | 38 | 13 |
Gross Unrealized Losses | (4) | (96) |
Fair Value | $ 192,999 | $ 185,920 |
Marketable Securities and Fai_4
Marketable Securities and Fair Value Measurements - Summary of assets measured at fair value on a recurring basis (Detail) - Fair Value, Recurring [Member] - USD ($) $ in Thousands | Mar. 31, 2023 | Dec. 31, 2022 |
Marketable securities: | ||
Total | $ 241,959 | $ 262,930 |
Level 1 | ||
Marketable securities: | ||
Total | 48,960 | 77,010 |
Level 2 | ||
Marketable securities: | ||
Total | 192,999 | 185,920 |
Level 3 | ||
Marketable securities: | ||
Total | 0 | 0 |
Money Market Funds | ||
Cash equivalents: | ||
Cash equivalents | 48,960 | 77,010 |
Money Market Funds | Level 1 | ||
Cash equivalents: | ||
Cash equivalents | 48,960 | 77,010 |
Money Market Funds | Level 2 | ||
Cash equivalents: | ||
Cash equivalents | 0 | 0 |
Money Market Funds | Level 3 | ||
Cash equivalents: | ||
Cash equivalents | 0 | 0 |
U.S. treasury securities | ||
Marketable securities: | ||
Marketable securities | 192,999 | 185,920 |
U.S. treasury securities | Level 1 | ||
Marketable securities: | ||
Marketable securities | 0 | 0 |
U.S. treasury securities | Level 2 | ||
Marketable securities: | ||
Marketable securities | 192,999 | 185,920 |
U.S. treasury securities | Level 3 | ||
Marketable securities: | ||
Marketable securities | $ 0 | $ 0 |
Collaboration and License Agr_2
Collaboration and License Agreement (Details) $ / shares in Units, $ in Thousands | 1 Months Ended | 3 Months Ended | ||||
Mar. 31, 2023 USD ($) product item $ / shares shares | Aug. 31, 2021 shares | Mar. 31, 2023 USD ($) $ / shares | Mar. 31, 2022 USD ($) | Apr. 30, 2023 USD ($) | Dec. 31, 2022 USD ($) | |
Collaboration and License Agreement | ||||||
Issuance of common stock in connection with the Moderna Agreement, shares | shares | 1,795,524 | |||||
Proceeds from issuance of common stock in connection with the Moderna Agreement | $ 36,000 | $ 0 | ||||
Contract with customer liability, current | $ 5,681 | 5,681 | $ 0 | |||
Contract with customer liability, noncurrent | 55,085 | 55,085 | $ 0 | |||
Collaboration and License Agreement | ModernaTX, Inc | ||||||
Collaboration and License Agreement | ||||||
Upfront payments | 40,000 | 40,000 | $ 40,000 | |||
Prepaid research services funding | 7,500 | 7,500 | $ 7,500 | |||
Milestone payments receivable | $ 1,800,000 | $ 1,800,000 | ||||
Issuance of common stock in connection with the Moderna Agreement, shares | shares | 5,859,375 | |||||
Shares issue price (in dollars per share) | $ / shares | $ 6.14 | $ 6.14 | ||||
Proceeds from issuance of common stock in connection with the Moderna Agreement | $ 36,000 | |||||
Percent of shares to be issued | 3.06% | |||||
Equity financing | $ 25,000 | $ 25,000 | ||||
Number of performance obligations | item | 1 | |||||
Premium paid on common stock | $ 13,300 | 13,300 | ||||
Variable consideration | 0 | 0 | ||||
Revenue recognized | 0 | |||||
Contract with customer liability, current | 5,700 | 5,700 | ||||
Contract with customer liability, noncurrent | $ 55,100 | $ 55,100 | ||||
Collaboration and License Agreement | ModernaTX, Inc | Minimum | ||||||
Collaboration and License Agreement | ||||||
Number of products to be commercialized | product | 1 | |||||
Royalty payment term | 10 years |
Property and equipment, net - S
Property and equipment, net - Summary of Property and Equipment, Net (Detail) - USD ($) $ in Thousands | Mar. 31, 2023 | Dec. 31, 2022 |
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | $ 37,857 | $ 36,753 |
Less: Accumulated depreciation and amortization | (15,827) | (14,538) |
Property and equipment, net | 22,030 | 22,215 |
Laboratory equipment | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 13,595 | 13,619 |
Computer equipment and software | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 1,262 | 1,189 |
Furniture and fixtures | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 1,146 | 1,146 |
Leasehold Improvements | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | 20,786 | 20,786 |
Construction in progress | ||
Property, Plant and Equipment [Line Items] | ||
Property and equipment, gross | $ 1,068 | $ 13 |
Property and equipment, net - A
Property and equipment, net - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2023 | Mar. 31, 2022 | |
Property and equipment, net | ||
Depreciation and amortization expense | $ 1,330 | $ 1,197 |
Accrued Expenses - Summary of A
Accrued Expenses - Summary of Accrued expenses (Detail) - USD ($) $ in Thousands | Mar. 31, 2023 | Dec. 31, 2022 |
Accrued Expenses | ||
Accrued employee compensation and benefits | $ 3,015 | $ 7,970 |
Accrued external research and development expenses | 2,406 | 1,959 |
Accrued professional fees | 1,253 | 1,047 |
Property and equipment | 388 | |
Other | 585 | 426 |
Total | $ 7,647 | $ 11,402 |
Equity - Additional Information
Equity - Additional Information (Detail) $ / shares in Units, $ in Thousands | 1 Months Ended | 3 Months Ended | |||
Mar. 31, 2023 USD ($) $ / shares shares | Aug. 31, 2021 USD ($) shares | Mar. 31, 2023 USD ($) security $ / shares shares | Mar. 31, 2022 USD ($) | Dec. 31, 2022 $ / shares shares | |
Common stock, shares authorized | 150,000,000 | 150,000,000 | 150,000,000 | ||
Common stock, par value per share | $ / shares | $ 0.0001 | $ 0.0001 | $ 0.0001 | ||
Preferred stock, shares authorized | 5,000,000 | 5,000,000 | 5,000,000 | ||
Preferred stock, par value per share | $ / shares | $ 0.0001 | $ 0.0001 | $ 0.0001 | ||
Shares issued during the period | 1,795,524 | ||||
Proceeds from Issuance of Common Stock | $ | $ 36,000 | $ 0 | |||
Net proceeds from shares issued | $ | $ 12,300 | ||||
Number of vote per common share | security | 1 | ||||
Maximum | |||||
Aggregate gross offering price | $ | $ 250,000 | ||||
Common Stock [Member] | |||||
Shares issued during the period | 5,859,375 | ||||
Share Purchase Agreement | ModernaTX, Inc | |||||
Shares issued during the period | 5,859,375 | ||||
Shares issue price (in dollars per share) | $ / shares | $ 6.14 | $ 6.14 | |||
Proceeds from Issuance of Common Stock | $ | $ 36,000 |
Stock-Based Compensation - Addi
Stock-Based Compensation - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Millions | 1 Months Ended | 3 Months Ended | |||||||
Jun. 11, 2020 | Aug. 31, 2021 | Mar. 31, 2023 | Jan. 31, 2023 | Dec. 31, 2022 | Jan. 31, 2022 | Dec. 31, 2021 | Jan. 31, 2021 | Dec. 31, 2020 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||
Unrecognized stock compensation on account of unvested options | $ 42.6 | ||||||||
Unrecognized stock compensation on account of unvested options remaining period over which the compensation is to be recognized | 2 years 10 months 24 days | ||||||||
Shares issued during the period | 1,795,524 | ||||||||
Unvested Stock Awards for Which Performance is not Probable | |||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||
Unrecognized stock compensation on account of unvested options | $ 1.7 | ||||||||
Service Based Options [Member] | Certain Employees Directors And Consultants [Member] | |||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||
Share based compensation by share based payment arrangement number of options granted | 1,365,391 | ||||||||
Share based compensation by share based fair value | $ 3.68 | ||||||||
Share based compensation by share based payment weighted average period | 4 years | ||||||||
Restricted Stock [Member] | |||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||
Unrecognized stock compensation on account of unvested options remaining period over which the compensation is to be recognized | 2 years 3 months 18 days | ||||||||
Unrecognized stock compensation on account of restricted common stock | $ 6.8 | ||||||||
Shares issued | 695,595 | ||||||||
Fair value | $ 3.3 | ||||||||
Weighted average period | 3 years 10 months 24 days | ||||||||
2020 Stock Incentive Plan | |||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||
Stock reserved for future issuance | 2,547,698 | ||||||||
Stock based compensation vesting period service based | 4 years | ||||||||
Stock based compensation period of expiry | 10 years | ||||||||
Common stock shares available for future issuance | 1,207,324 | ||||||||
Unrecognized stock compensation on account of unvested options | $ 35.8 | ||||||||
2020 Stock Incentive Plan | Maximum | Additional From 2017 Plan | |||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||
Stock reserved for future issuance | 7,173,014 | ||||||||
2020 Stock Incentive Plan | Maximum | Annual Increase 2021 To 2030 | |||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||
Percentage of common stock shares additionally reserved for issuance | 4% | ||||||||
Employee Stock Purchase Plan 2020 | |||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||
Stock reserved for future issuance | 2,115,792 | 1,520,738 | 1,520,738 | 950,931 | 950,931 | 481,231 | |||
Shares issued | 1,933,830 | ||||||||
Employee Stock Purchase Plan 2020 | Maximum | Annual Increase 2021 To 2030 | |||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||
Percentage of common stock shares additionally reserved for issuance | 1% | ||||||||
Number of share instruments newly issued under a share-based compensation plan | 1,302,157 | ||||||||
Common Stock [Member] | 2020 Stock Incentive Plan | |||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||
Share based compensation by share based payment arrangement number of shares authorized for issuance | 16,813,962 | 14,433,745 | 14,433,745 | 12,154,517 | 12,154,517 | 10,275,717 |
Stock-Based Compensation - Summ
Stock-Based Compensation - Summary of Stock-Based Compensation Expense was Classified in the Statements of Operations and Comprehensive Loss (Detail) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2023 | Mar. 31, 2022 | |
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items] | ||
Stock-based compensation expense | $ 6,266 | $ 6,066 |
Research and development expense [Member] | ||
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items] | ||
Stock-based compensation expense | 2,855 | 3,151 |
General and administrative expenses | ||
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items] | ||
Stock-based compensation expense | $ 3,411 | $ 2,915 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) | 3 Months Ended |
Mar. 31, 2023 | |
Commitments and Contingencies. | |
Defined contribution plan, percentage | 4% |
Net Loss per Share - Summary of
Net Loss per Share - Summary of Potential Dilutive Securities, Presented Based on Amounts Outstanding (Detail) - shares | 3 Months Ended | |
Mar. 31, 2023 | Mar. 31, 2022 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive net loss per share | 11,443,922 | 10,011,440 |
Unvested restricted common stock units | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive net loss per share | 1,411,002 | 1,537,744 |
Stock option to purchase common stock | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive net loss per share | 10,032,920 | 8,473,696 |
Related Parties (Details)
Related Parties (Details) - shares | 1 Months Ended | |
Mar. 31, 2023 | Aug. 31, 2021 | |
Related Party Transaction [Line Items] | ||
Shares issued during the period | 1,795,524 | |
ModernaTX, Inc [Member] | Share Purchase Agreement | ||
Related Party Transaction [Line Items] | ||
Shares issued during the period | 5,859,375 | |
Beneficial ownership percentage | 8.90% |