Cover Page
Cover Page - USD ($) | 12 Months Ended | ||
Dec. 31, 2020 | Feb. 24, 2021 | Jun. 30, 2020 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Amendment Flag | false | ||
Document Period End Date | Dec. 31, 2020 | ||
Document Fiscal Year Focus | 2020 | ||
Document Fiscal Period Focus | FY | ||
Trading Symbol | TBIO | ||
Entity Registrant Name | Translate Bio, Inc. | ||
Entity Central Index Key | 0001693415 | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Current Reporting Status | Yes | ||
Entity Shell Company | false | ||
Entity Filer Category | Large Accelerated Filer | ||
Entity Small Business | true | ||
Entity Emerging Growth Company | false | ||
Entity Address, State or Province | MA | ||
Title of 12(b) Security | Common Stock | ||
Entity Interactive Data Current | Yes | ||
Security Exchange Name | NASDAQ | ||
Entity Public Float | $ 825,813,865 | ||
Entity Voluntary Filers | No | ||
Entity Common Stock, Shares Outstanding | 75,211,832 | ||
Entity Well-known Seasoned Issuer | Yes | ||
ICFR Auditor Attestation Flag | true | ||
Document Annual Report | true | ||
Document Transition Report | false | ||
Entity File Number | 001-38550 | ||
Entity Incorporation, State or Country Code | DE | ||
Entity Tax Identification Number | 61-1807780 | ||
Entity Address, Address Line One | 29 Hartwell Avenue | ||
Entity Address, City or Town | Lexington | ||
Entity Address, Postal Zip Code | 02421 | ||
City Area Code | 617 | ||
Local Phone Number | 945-7361 |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Current assets: | ||
Cash and cash equivalents | $ 342,027 | $ 84,580 |
Investments | 312,001 | 104,098 |
Collaboration receivables | 26,598 | 4,596 |
Prepaid expenses and other current assets | 11,741 | 9,391 |
Restricted cash | 4,826 | 950 |
Total current assets | 697,193 | 203,615 |
Property and equipment, net | 15,372 | 12,539 |
Right-of-use assets, net | 72,957 | 10,400 |
Goodwill | 21,359 | 21,359 |
Intangible assets, net | 79,127 | 85,536 |
Other assets | 3,928 | 2,752 |
Total assets | 889,936 | 336,201 |
Current liabilities: | ||
Accounts payable | 8,839 | 15,968 |
Accrued expenses | 13,202 | 7,072 |
Current portion of deferred revenue | 67,563 | 18,100 |
Current portion of operating lease liability | 11,733 | 530 |
Total current liabilities | 101,337 | 41,670 |
Contingent consideration | 152,230 | 103,655 |
Deferred revenue, net of current portion | 228,659 | 25,256 |
Operating lease liability, net of current portion | 50,953 | 12,084 |
Total liabilities | 533,179 | 182,665 |
Commitments and contingencies (Notes 3 and 13) | ||
Stockholders' equity: | ||
Preferred stock, $0.001 par value; 10,000,000 shares authorized as of December 31, 2020 and 2019; no shares issued and outstanding as of December 31, 2020 and 2019 | ||
Common stock, $0.001 par value; 200,000,000 shares authorized as of December 31, 2020 and 2019; 75,029,625 shares and 60,022,067 shares issued and outstanding as of December 31, 2020 and 2019, respectively | 75 | 60 |
Additional paid-in capital | 769,965 | 512,231 |
Accumulated deficit | (413,283) | (359,496) |
Accumulated other comprehensive income | 741 | |
Total stockholders' equity | 356,757 | 153,536 |
Total liabilities and stockholders' equity | $ 889,936 | $ 336,201 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2020 | Dec. 31, 2019 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.001 | $ 0.001 |
Preferred stock, shares authorized | 10,000,000 | 10,000,000 |
Preferred stock, shares Issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value | $ 0.001 | $ 0.001 |
Common stock, shares authorized | 200,000,000 | 200,000,000 |
Common stock, shares issued | 75,029,625 | 60,022,067 |
Common stock, shares outstanding | 75,029,625 | 60,022,067 |
Consolidated Statements of Oper
Consolidated Statements of Operations - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Income Statement [Abstract] | ||
Collaboration revenue | $ 138,811 | $ 7,804 |
Operating expenses: | ||
Research and development | 109,629 | 76,369 |
General and administrative | 35,922 | 28,632 |
Change in fair value of contingent consideration | 48,575 | 13 |
Impairment of intangible asset | 0 | 18,559 |
Total operating expenses | 194,126 | 123,573 |
Loss from operations | (55,315) | (115,769) |
Other income, net | 1,528 | 1,990 |
Loss before benefit from income taxes | (53,787) | (113,779) |
Benefit from income taxes | 486 | |
Net loss | $ (53,787) | $ (113,293) |
Net loss per share—basic and diluted | $ (0.80) | $ (2.20) |
Weighted average common shares outstanding—basic and diluted | 67,520,886 | 51,445,539 |
Consolidated Statements of Comp
Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Statement of Comprehensive Income [Abstract] | ||
Net loss | $ (53,787) | $ (113,293) |
Other comprehensive loss: | ||
Unrealized gains (losses) on available-for-sale securities, net of tax of $0 | (741) | 545 |
Comprehensive loss | $ (54,528) | $ (112,748) |
Consolidated Statements of Co_2
Consolidated Statements of Comprehensive Loss (Parenthetical) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Statement of Comprehensive Income [Abstract] | ||
Unrealized gains (losses) on available-for-sale securities, net of tax | $ 0 | $ 0 |
Consolidated Statements Stockho
Consolidated Statements Stockholders' Equity - USD ($) $ in Thousands | Total | Common Stock [Member] | Additional Paid-in Capital [Member] | Accumulated Deficit [Member] | Accumulated Other Comprehensive Income [Member] |
Beginning balances at Dec. 31, 2018 | $ 125,295 | $ 45 | $ 371,257 | $ (246,203) | $ 196 |
Beginning balance, Shares at Dec. 31, 2018 | 45,139,955 | ||||
Issuance of common stock in connection with private placement, net of placement agent fees and offering costs | 44,134 | $ 6 | 44,128 | ||
Issuance of common stock in connection with private placement, net of placement agent fees and offering costs, Shares | 5,582,940 | ||||
Issuance of common stock in connection with public offerings, net of underwriting discounts and commissions and offering costs | 84,011 | $ 9 | 84,002 | ||
Issuance of common stock in connection with public offerings, net of underwriting discounts and commissions and offering costs, Shares | 9,000,000 | ||||
Issuance of common stock in connection with a former employee letter agreement | 847 | 847 | |||
Issuance of common stock in connection with a former employee letter agreement, Shares | 67,406 | ||||
Forfeited restricted common stock | (2) | (2) | |||
Forfeited restricted common stock, Shares | (1,783) | ||||
Exercise of stock options | 1,512 | 1,512 | |||
Exercise of stock options, Shares | 233,549 | ||||
Stock-based compensation expense | 10,487 | 10,487 | |||
Unrealized gains (losses) on available-for-sale securities | 545 | 545 | |||
Net loss | (113,293) | (113,293) | |||
Ending balances at Dec. 31, 2019 | 153,536 | $ 60 | 512,231 | (359,496) | 741 |
Ending balance, shares at Dec. 31, 2019 | 60,022,067 | ||||
Issuance of common stock in connection with public offerings, net of underwriting discounts and commissions and offering costs | 153,611 | $ 9 | 153,602 | ||
Issuance of common stock in connection with public offerings, net of underwriting discounts and commissions and offering costs, Shares | 8,544,982 | ||||
Issuance of common stock in connection with Securities Purchase Agreement (Shares) | 4,884,434 | ||||
Issuance of common stock in connection with Securities Purchase Agreement | 73,754 | $ 5 | 73,749 | ||
Exercise of stock options | 12,398 | $ 1 | 12,397 | ||
Exercise of stock options, Shares | 1,551,178 | ||||
Issuance of common stock under employee stock purchase plan | 366 | 366 | |||
Issuance of common stock under employee stock purchase plan, Shares | 26,964 | ||||
Stock-based compensation expense | 17,620 | 17,620 | |||
Unrealized gains (losses) on available-for-sale securities | (741) | $ (741) | |||
Net loss | (53,787) | (53,787) | |||
Ending balances at Dec. 31, 2020 | $ 356,757 | $ 75 | $ 769,965 | $ (413,283) | |
Ending balance, shares at Dec. 31, 2020 | 75,029,625 |
Consolidated Statements of Cash
Consolidated Statements of Cash flows - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Cash flows from operating activities: | ||
Net loss | $ (53,787) | $ (113,293) |
Adjustments to reconcile net loss to net cash provided by (used in) operating activities: | ||
Depreciation and amortization expense | 9,270 | 4,732 |
Stock-based compensation expense | 17,620 | 11,334 |
Impairment of intangible asset | 0 | 18,559 |
Change in fair value of contingent consideration | 48,575 | 13 |
Deferred income tax benefit | (486) | |
Changes in operating assets and liabilities: | ||
Collaboration receivables | (22,002) | (3,763) |
Prepaid expenses and other assets | (6,278) | (5,743) |
Right-of-use assets | 4,315 | 484 |
Long-term prepaid rent | (10,058) | (2,752) |
Accounts payable | (6,572) | 9,928 |
Accrued expenses | 6,358 | 257 |
Lease liability | (3,991) | (375) |
Deferred revenue | 252,866 | (1,057) |
Net cash provided by (used in) operating activities | 236,316 | (82,162) |
Cash flows from investing activities: | ||
Purchases of investments | (339,410) | (174,271) |
Sales and maturities of investments | 130,765 | 159,622 |
Purchases of property and equipment | (6,477) | (3,537) |
Net cash used in investing activities | (215,122) | (18,186) |
Cash flows from financing activities: | ||
Proceeds from public offerings, net of underwriting discounts and commissions | 154,292 | 84,600 |
Payments of public offering costs | (681) | (589) |
Proceeds from Securities Purchase Agreement | 73,754 | |
Proceeds from private placement, net of placement agent fees | 44,608 | |
Payments of private placement offering costs | (477) | |
Proceeds from option exercises | 12,398 | 1,512 |
Proceeds from issuance of common stock under employee stock purchase plan | 366 | |
Net cash provided by financing activities | 240,129 | 129,654 |
Net increase in cash, cash equivalents and restricted cash: | 261,323 | 29,306 |
Cash, cash equivalents and restricted cash at beginning of period | 85,530 | 56,224 |
Cash, cash equivalents and restricted cash at end of period | 346,853 | 85,530 |
Cash, cash equivalents and restricted cash at end of period: | ||
Cash and cash equivalents | 342,027 | 84,580 |
Restricted cash | 4,826 | 950 |
Total cash, cash equivalents and restricted cash at end of period | 346,853 | 56,224 |
Supplemental disclosure of non-cash investing and financing activities: | ||
Purchases of property and equipment included in accounts payable and accrued expenses | $ 406 | 1,189 |
Issuance of common stock in connection with a former employee letter agreement | $ 847 |
Nature of the Business and Basi
Nature of the Business and Basis of Presentation | 12 Months Ended |
Dec. 31, 2020 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Nature of the Business and Basis of Presentation | 1. Nature of the Business and Basis of Presentation Translate Bio, Inc. (the “Company”) is a clinical-stage messenger RNA (“mRNA”) therapeutics company developing a new class of potentially transformative medicines to treat diseases caused by protein or gene dysfunction, or to prevent infectious diseases by generating protective immunity. Using its proprietary mRNA therapeutic platform (“MRT platform”), the Company creates mRNA that encodes functional proteins. The Company’s mRNA is designed to be delivered to the target cell where the cell’s own machinery recognizes it and translates it, restoring or augmenting protein function to treat or prevent disease. The Company is primarily focused on applying its MRT platform to treat pulmonary diseases caused by insufficient protein production or where production of proteins can modify disease. In addition, the Company is also pursuing discovery efforts in diseases that affect the liver. The Company is also pursuing the applicability of its MRT platform for the development of mRNA vaccines for infectious diseases under a collaboration with Sanofi Pasteur Inc. (“Sanofi”), the vaccines global business unit of Sanofi S.A. The outbreak of a novel strain of coronavirus named SARS-CoV-2, which causes COVID-19, and the resulting COVID-19 pandemic has presented a substantial public health and economic challenge around the world and continues to affect the Company’s employees, patients, communities and business operations, as well as economies and financial markets worldwide. In 2020, the Company progressed certain of its preclinical programs, specifically in therapeutics for pulmonary diseases and in vaccine development under its collaboration with Sanofi, as further discussed below. However, as a consequence of the COVID-19 pandemic, we announced that enrollment and dosing in the Company’s ongoing Phase 1/2 clinical trial in patients with cystic fibrosis (“CF”) was paused in April 2020 and then resumed in September 2020. The full extent to which the COVID-19 pandemic will directly or indirectly impact the Company’s business, results of operations and financial condition will depend on future developments that are highly uncertain and cannot be accurately predicted, including new information that may emerge concerning COVID-19, the actions taken in an effort to contain it or to potentially treat or vaccinate against COVID-19 and the economic impact on local, regional, national and international markets. The Company actively monitors this situation and the possible effects on its financial condition, liquidity, operations, suppliers, industry and workforce. The Company is developing MRT5005 for the treatment of CF. The Company is conducting a Phase 1/2 clinical trial to evaluate the safety and tolerability of single- and multiple-ascending doses of MRT5005. The clinical trial is investigating several groups receiving five once-weekly doses, as well as a group receiving five daily doses. In April 2019, the Company completed dosing of all patients in the originally planned single-ascending dose (“SAD”) portion of the Phase 1/2 clinical trial and in July 2019, the Company reported interim data from the SAD portion of the clinical trial through one-month follow up post dosing. In January 2021, the Company announced that it completed enrollment and dosing in the dose cohorts comprising the second interim data analysis. The Company anticipates reporting interim clinical data from these cohorts early in the second quarter of 2021. The Company is leveraging its lung delivery platform and focusing its preclinical research efforts on identifying lead product candidates for a next-generation CF program, as well as beyond CF in additional pulmonary diseases with unmet medical needs, including primary ciliary dyskinesia and pulmonary arterial hypertension. The Company has a collaboration with Sanofi to develop infectious disease vaccines using the Company’s mRNA technology. Under the collaboration, the Company and Sanofi are jointly conducting research and development activities to advance mRNA vaccines targeting up to seven infectious disease pathogens (see Note 3). Two of the target pathogens under development are SARS-CoV-2 and influenza. MRT5500 has been selected as the lead candidate for a vaccine against SARS-CoV-2. mid- y The Company is subject to risks 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 and the ability to secure additional capital to fund operations. Product candidates currently under development will require significant additional research and development efforts, including preclinical and clinical testing and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel and infrastructure and extensive compliance-reporting capabilities. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will realize significant revenue from product sales. The accompanying consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States of America (“GAAP”) and include the accounts of the Company and its two wholly owned subsidiaries, Translate Bio MA, Inc. and Translate Bio Securities Corporation, from their date of incorporation. All intercompany accounts and transactions have been eliminated in consolidation. The Company’s financial statements have been prepared on the basis of continuity of operations, realization of assets and the satisfaction of liabilities in the ordinary course of business. Through December 31, 2020, the Company has funded its operations primarily through sales of equity securities and upfront payments received under a collaboration and license agreement with Sanofi. The Company has incurred recurring losses and cash outflows from operations since its inception, including net losses of $53.8 million and $113.3 million for the years ended December 31, 2020 and 2019, respectively. In addition, the Company had an accumulated deficit of $413.3 million as of December 31, 2020. The Company expects to continue to generate operating losses for the foreseeable future. As of March 1 Sales of Common Stock On March 13, 2020, the Company filed a universal shelf registration statement on Form S-3 No. 333-237159). The Company is party to an Open Market Sale Agreement SM On June 24, 2020, the Company filed a registration statement on Form S-3ASR, No. 333-239405) Statement”). The June 2020 Registration Statement registered for sale from time to time common stock, preferred Sanofi Pasteur Collaboration and Licensing Agreement In 2018, the Company entered into a collaboration and license agreement with Sanofi (the “Original Sanofi Agreement”) to develop mRNA vaccines for up to five infectious disease pathogens (the “Licensed Fields”). On March 26, 2020, the Company and Sanofi amended the Original Sanofi Agreement (the “First Sanofi Amendment”) to include vaccines against SARS-CoV-2 Pursuant to the Amended Sanofi Agreement, the Company and Sanofi are jointly conducting pathogens. Under the terms of the Amended Sanofi Agreement, the Company know-how Pursuant to the Second Sanofi Amendment, Sanofi paid the Company an additional upfront payment of $300.0 million in August 2020. Additionally, in connection with the execution of the Second Sanofi Amendment, the Company and an affiliate of Sanofi (the “Sanofi Investor”) entered into a securities purchase agreement (the “Secur i |
Summary of Significant Accounti
Summary of Significant Accounting Policies | Nov. 03, 2020 |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Use of Estimates The preparation of the Company’s consolidated financial COVID-19 within its financial statements and have determined them to be immaterial. There may be changes to those estimates in future periods periodically changes estimates are recorded in the period in which they become known. Actual results could differ from those estimates. Cash and Cash Equivalents All highly liquid investments purchased with an original maturity date of three months or less at the date of purchase are considered to be cash equivalents. Cash equivalents consisted of money market funds as of December 31, 2020 and 2019. Investments Available-for-sale securities consist of investments with original maturities greater than 90 days at acquisition date. The Company has classified its investments with maturities beyond one year as short term, based on their highly liquid nature and because such available-for-sale securities represent the investment of cash that is available for current operations. The Company’s debt security investments are classified as available-for-sale and are carried at fair value, with the unrealized gains and losses reported as a component of accumulated other comprehensive income (loss) in stockholders’ equity (deficit). Realized gains and losses and declines in value determined to be other than temporary are based on the specific identification method and are included as a component of other income (expense), net in the consolidated statements of operations. The Company evaluates its investments with unrealized losses for other-than-temporary impairment. When assessing investments for other-than-temporary declines in value, the Company considers such factors as, among other things, how significant the decline in value is as a percentage of the original cost, how long the market value of the investment has been less than its original cost, the Company’s ability and intent to retain the investment for a period of time sufficient to allow for any anticipated recovery in fair value and market conditions in general. If any adjustment to fair value reflects a decline in the value of the investment that the Company considers to be “other than temporary,” the Company reduces the investment to fair value through a charge to the statement of operations. No such adjustments were necessary during the periods presented Concentrations of Credit Risk Financial instruments that potentially subject the Company to concentrations of credit risk consist principally of cash and cash equivalents as well as investments. Cash, cash equivalents and investments consist of demand deposits, money market funds, U.S. treasuries and U.S. government agency bonds. The Company generally maintains balances in various operating accounts with financial institutions that management believes to be of high credit quality, in amounts that may exceed federally insured limits. The Company has not experienced any losses related to its cash, cash equivalents and investments and does not believe that it is subject to unusual credit risk beyond the normal credit risk associated with commercial banking relationships. Restricted Cash In connection with its operating lease commitments, the Company issued letters of credit collateralized by cash deposits that are classified as restricted cash in the consolidated balance sheets. Restricted cash amounts have been classified as current assets based on the release dates of the restrictions under the letters of credit, which occur annually. Property and Equipment Property and equipment are stated at cost less accumulated depreciation and amortization. Depreciation and amortization expense is recognized using the straight-line method over the estimated useful lives of each asset. Estimated useful lives are periodically assessed to determine if changes are appropriate. Upon retirement accumulated Estimated Useful Life Laboratory equipment 5 years Computer equipment 3 years Office equipment 5 years Leasehold improvements Shorter of lease term or 10 years Costs for capital assets not yet placed into service are capitalized as construction-in-progress amortized Property and equipment are tested for recoverability whenever events or changes in business circumstances Leases The Company adopted Accounting Standards Codification (“ASC”) Topic 842, Leases (ASC 842), using a modified retrospective approach, as of January 1, 2019, with no restatement of prior periods or cumulative adjustment to retained earnings. The Company elected the package of practical expedients, which permits the Company not to reassess under the new standards for prior conclusions about lease identification, lease classification and initial direct costs. The Company did not apply the hindsight practical expedient when determining the lease term for existing leases and assessing impairment of expired or existing non-lease The Company accounts for a contract as a lease when it has the right to control the asset for a period of time while obtaining substantially all of the asset’s economic benefits. The Company determines if an arrangement is a lease or contains an embedded lease at inception. For arrangements that meet the definition of a lease, the Company determines the initial classification and measurement of its operating right-of-use In addition to rent, the leases may require the Company to pay additional amounts for taxes, insurance, maintenance, and other expenses, which are generally referred to as non-lease has elected to not separate lease and non-lease non-lease new drug application in the United States; and sales-based milestones, generally based on meeting specific thresholds of sales in certain geographic areas. The Company is also eligible to receive from Sanofi tiered royalty payments on worldwide net sales of mRNA vaccines. For each collaboration that includes development milestone payments, the Company evaluates whether it is probable that the consideration associated with each milestone will not be subject to a significant reversal in the cumulative amount of revenue recognized. Amounts that meet this threshold are included in the transaction price using the most likely amount method, whereas amounts that do not meet this threshold are considered constrained and excluded from the transaction price until they meet this threshold. Milestones tied to regulatory approval, and therefore not within the Company’s control, are considered constrained until such approval is received. Upfront and ongoing development milestones per the collaboration agreements are not subject to refund if the development activities are not successful. At the end of each subsequent reporting period, the Company re-evaluates catch-up ASC 606 requires the Company to allocate the arrangement consideration on a relative standalone selling price basis for each performance obligation after determining the transaction price of the contract and identifying the performance obligations to which that amount should be allocated. The relative standalone selling price is defined in ASC 606 as the price at which an entity would sell a promised good or service separately to a customer. If other observable transactions in which the Company has sold the same performance obligation separately are not available, the Company is required to estimate the standalone selling price of each performance obligation. Key assumptions to determine the standalone selling price may include forecasted revenues, development timelines, reimbursement rates for personnel costs, discount rates and probabilities of technical and regulatory success. Whenever the Company determines that a contract should be accounted for as a combined performance obligation, which is recognized over time, it will utilize the cost-to-cost cost-to-cost The Company evaluates its collaborative agreements for proper classification in the consolidated statements of operations based on the nature of the underlying activity. Transactions between collaborators recorded in the Company’s consolidated statements of operations are recorded on either a gross or net basis, depending on the characteristics of the collaborative relationship. For revenue generating arrangements where the Company estimate the fair value of the benefit received. Payments to a customer that are deemed a reduction of the transaction price are recorded first as a reduction of revenue, to the extent of both cumulative revenue recorded to date and probable future revenues, which include any unamortized deferred revenue balances, under all arrangements with such customer, and then as an expense. Payments that are not deemed to be a reduction of the transaction price are recorded as an expense. Consideration that does not meet the requirements to satisfy the above revenue recognition criteria is a contract liability and is recorded as deferred revenue in the consolidated balance sheets. Although the Company follows detailed guidelines in measuring revenue, certain judgments affect the application of its revenue policy. For example, in connection with the Amended Sanofi Agreement, the Company has recorded short-term and long-term deferred revenue on its consolidated balance sheets based on the Company’s best estimate of when such revenue will be recognized. Short-term deferred revenue consists of amounts that are expected to be recognized as revenue in the next 12 months. Amounts that the Company expects will not be recognized within the next 12 months are classified as long-term deferred revenue. The estimate of deferred revenue also reflects management’s estimate of the periods of the Company’s involvement in certain of its collaborations. The Company’s performance obligations under these collaborations consist of participation on steering committees and the performance of other research and development services. In certain instances, the timing of satisfying these obligations can be difficult to estimate. Accordingly, the Company’s estimates may change in the future. Such changes to estimates would result in a change in revenue recognition amounts. If these estimates and judgments change over the course of these agreements, it may affect the timing and amount of revenue that the Company will recognize and record in future periods. At December 31, 2020, the Company had short-term and long-term deferred revenue of $67.6 million and $228.7 million, respectively, related to the Sanofi collaboration. Under ASC 606, the Company will recognize revenue and record In-Process The fair value of in-process a The fair value of an IPR&D intangible asset is typically determined using a discounted cash flow analysis based on various assumptions, including the amount and timing of future cash flows and the probability of technical success. The net cash flows are then adjusted to present value by applying an appropriate discount rate that reflects the risk factors associated with the cash flow streams. Indefinite-lived IPR&D is not subject to amortization, but is tested annually for impairment or more frequently if there are indicators of impairment. The Company tests its indefinite-lived IPR&D annually for impairment on October 1st. In testing indefinite-lived IPR&D for impairment, the Company has the option to first assess qualitative factors to determine whether the existence of events or circumstances would indicate that it is more likely than not that its fair value is less than its carrying amount, or the Company can perform a quantitative impairment analysis to determine the fair value of the indefinite-lived IPR&D without performing a qualitative assessment. Qualitative factors that the Company considers include significant underperformance of the business in relation to expectations, significant negative industry or economic trends and significant changes or planned changes in the use of the assets. If the Company chooses to first assess qualitative factors and the Company determines that it is more likely than not that the fair value of the indefinite-lived IPR&D is less than its carrying amount, the Company would then determine the fair value of the indefinite-lived IPR&D. Under either approach, if the fair value of the indefinite-lived IPR&D is less than its carrying amount, an impairment charge is recognized in the consolidated statements of operations. Definite-lived IPR&D is recorded at fair value and amortized over the greater of economic consumption or on a straight-line basis over its estimated useful life. Definite-lived IPR&D is tested for recoverability whenever events or changes in business circumstances indicate that the carrying amount of the assets may not be fully recoverable. If an impairment review is performed to evaluate an asset group for recoverability, the Company compares the forecasted undiscounted cash flows expected to result from the use and eventual disposition of the asset group to its carrying value. An impairment loss would be recognized when estimated undiscounted future cash flows expected to result from the use and eventual disposition of an asset group are less than its carrying amount. The impairment loss would be based on the excess of the carrying value of the impaired asset group over its fair value, determined based on discounted cash flows using market participant assumptions. Goodwill Goodwill represents the excess of the fair value of the consideration transferred over the fair value In testing goodwill for impairment, the Company has the option to first assess qualitative factors to determine whether it is necessary to perform a quantitative impairment test. If the Company elects this option and determine, as a result of the qualitative assessment, that it is more likely than not that the fair value of a reporting unit is less than its carrying amount, the quantitative impairment test is required; otherwise, no further testing is required. Among other relevant events and circumstances that affect the fair value of reporting units, the Company considers individual factors, such as microeconomic conditions, changes in the industry and the markets in which it operates as well as historical and expected future financial performance. Alternatively, the Company may elect to not first assess qualitative factors and instead immediately perform the quantitative impairment test. In 2020, the Company elected to early adopt guidance Fair Value Measurements Certain assets and liabilities of the Company are carried at fair value under GAAP. Fair value is defined as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. Valuation techniques used to measure fair value must maximize the use of observable inputs and minimize the use of unobservable inputs. Financial assets and liabilities carried at fair value are to be classified and disclosed in one of the following three levels of the fair value hierarchy, of which the first two are considered observable and the last is considered unobservable: • Level 1—Quoted prices in active markets for identical assets or liabilities. • Level 2—Observable inputs (other than Level 1 quoted prices), such as quoted prices in active markets for similar assets or liabilities, quoted prices in markets that are not active for identical or similar assets or liabilities, or other inputs that are observable or can be corroborated by observable market data. • Level 3—Unobservable inputs that are supported by little or no market activity that are significant to determining the fair value of the assets or liabilities, including pricing models, discounted cash flow methodologies and similar techniques. The Company’s cash equivalents and investments are carried at fair value, determined based on Level 2 inputs in the fair value hierarchy described above (see Note 5). The Company’s contingent consideration liability is carried at fair value, determined based on Level 3 inputs in the fair value hierarchy described above (see Note 5). The carrying values of the Company’s short-term collaboration receivables, prepaid expenses and other current assets, accounts payable, accrued expenses and other current liabilities approximate their fair values due to the short-term nature of these assets and liabilities. Segment Information The Company manages its operations as a single operating segment for the purposes of assessing performance and making operating decisions. The Company’s primary focus is on applying its MRT platform to treat pulmonary diseases caused by insufficient protein production or where production of proteins can modify disease. The Company is also pursuing the applicability of its MRT platform for the development of mRNA vaccines for infectious diseases under a collaboration with Sanofi. Research and Development Costs Costs associated with internal research and development and external research and development services, including drug development and preclinical studies, are expensed as incurred. Research and development expenses include costs for salaries, employee benefits, subcontractors, facility-related expenses, depreciation and amortization, including amortization related to definite-lived IPR&D intangible assets, stock-based compensation, third-party license fees, laboratory supplies, and external costs of outside vendors engaged to conduct discovery, preclinical and clinical development activities and clinical trials as well as to manufacture clinical trial materials, and other costs. Research and development costs also include costs related to performing the Company’s obligations under the Amended Sanofi Agreement. The Company recognizes external research and development costs based on an evaluation of the progress to completion of specific tasks using information provided to the Company by its service providers. Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are recorded as prepaid expenses. Such prepaid expenses are recognized as an expense when the services have been performed or the goods have been delivered, or when it is no longer expected that the goods will be delivered or the services rendered. Upfront payments, milestone payments (other than those deemed contingent consideration in a business combination) and annual maintenance fees under license agreements are expensed in the period in which they are incurred. Research and Development Contract Costs and Accruals The Company has entered into various research and development-related contracts with companies both inside and outside of the United States. The related costs are recorded as research and development expenses as incurred. The Company records accruals for estimated ongoing research and development costs. When actual costs. Patent Costs All patent-related costs incurred in connection with filing and prosecuting patent applications are expensed as incurred due to the uncertainty about the recovery of the expenditure. Amounts incurred are classified as general and administrative expenses. Stock-Based Compensation The Company measures all stock-based awards granted to employees, directors and non-employee The Company classifies stock-based compensation expense in its consolidated statements of operations in the same manner in which the award recipient’s payroll costs are classified or in which the award recipient’s service payments are classified. Comprehensive Income (Loss) Comprehensive income (loss) includes net loss as well as other changes in stockholders’ equity (deficit) that result from transactions and economic events other than those with stockholders. The Company’s only element of other comprehensive income (loss) was unrealized gains (losses) on investments, available-for-sale-securities. Income Taxes The Company accounts for income taxes using the asset and liability method, which requires the recognition of deferred tax assets and liabilities for the expected future tax consequences of events that have been recognized in the consolidated financial statements or in the Company’s tax returns. Deferred tax assets and liabilities are determined on the basis of the differences between the financial statement and tax basis of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse. Changes in deferred tax assets and liabilities are recorded in the provision for income taxes. The Company assesses the likelihood that its deferred tax assets will be recovered from future taxable income and, to the extent it believes, based upon the weight of available evidence, that it is more likely than not that all or a portion of the deferred tax assets will not be realized, a valuation allowance is established through a charge to income tax expense. Potential for recovery of deferred tax assets is evaluated by estimating the future taxable profits expected and considering prudent and feasible tax planning strategies. The Company accounts for uncertainty in income taxes recognized in the consolidated financial statements by applying two-step more-likely-than-not Net Income (Loss) per Share The Company follows the two-class two-class two-class Basic net income (loss) per share attributable to common stockholders is computed by dividing the net income (loss) attributable to common stockholders by the weighted average number of shares of common stock outstanding for the period. Diluted net income (loss) attributable to common stockholders is computed by adjusting net income (loss) attributable to common stockholders to reallocate undistributed earnings based on the potential impact of dilutive securities. Diluted net income (loss) per share attributable to common stockholders is computed by dividing the diluted net income (loss) attributable to common stockholders by the weighted average number of shares of common stock outstanding for the period, including potential dilutive shares of common stock. For purposes of this calculation, outstanding stock options and unvested restricted common stock are considered potential dilutive shares of common stock. In periods in which the Company reports a net loss attributable to common stockholders, diluted net loss per share attributable to common stockholders is the same as basic net loss per share attributable to common stockholders, since dilutive shares of common stock are not assumed to have been issued if their effect is anti-dilutive. The Company reported a net loss attributable to common stockholders for the years ended December 31, 2020 and 2019. Recently Adopted Accounting Pronouncements In January 2017, the FASB issued Accounting Standards Update (“ASU”) No. 2017-04, Intangibles—Goodwill and Other: Simplifying the Test for Goodwill Impairment (Topic 350) In August 2018, the FASB issued ASU No. 2018-13, Fair Value Measurement (Topic 820): Disclosure Framework—Changes to the Disclosure Requirements for Fair Value Measurement In November 2018, the FASB issued ASU No. 2018-18, Collaborative Arrangements (Topic 808): Clarifying the Interaction between Topic Revenue from Contracts with Customers Collaborative Arrangements Recently Issued Accounting Pronouncements In June 2016, the FASB issued ASU 2016-13, Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments available-for-sale In December 2019, the FASB issued ASU No. 2019-12, Income Taxes-Simplifying the Accounting for Income Taxes step-up statements |
Collaboration Agreement
Collaboration Agreement | 12 Months Ended |
Dec. 31, 2020 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Sanofi Collaboration and License Agreement | 3. Collaboration Agreement Sanofi Collaboration and License Agreement In 2018, the Company and Sanofi entered into the Original Sanofi Agreement to develop mRNA vaccines and an mRNA vaccine platform for up to five infectious disease pathogens. In March 2020, the Company and Sanofi entered into the First Sanofi Amendment to include vaccines against SARS-CoV-2 Under the terms of the Amended Sanofi Agreement, the Company has agreed to grant to Sanofi exclusive, worldwide licenses under applicable patents, patent applications, know-how Pursuant to the Amended Sanofi Agreement, the Company and Sanofi are jointly conducting research and development activities to advance mRNA vaccines targeting up to seven infectious disease pathogens. The term of the research collaboration (the “Collaboration Term”) expires in June 2022, with an option for Sanofi to extend the Collaboration Term for one additional year, followed by a technology transfer to Sanofi. If Sanofi elects to extend the Collaboration Term, the collaboration may be further expanded to jointly conduct research and development activities to advance mRNA vaccines for up to an additional three infectious disease pathogens, bringing the total to up to ten pathogens. In addition to the research and developme n non-clinical products, related materials and investigational products as required by the collaboration plan. Pursuant to the Amended Sanofi Agreement, the Company and Sanofi agreed to a governance structure to manage the activities under the collaboration. If the Company and Sanofi do not mutually agree on certain decisions, Sanofi will be able to break a deadlock without the Company’s consent under certain conditions. The collaboration includes an estimated budget. Sanofi is responsible for paying reimbursable development costs, including the Company’s employee costs, manufacturing costs, and out-of-pocket costs paid to third parties, up to a specified amount for each Licensed Field. The Company and Sanofi retain the rights to perform their respective obligations and exercise their respective rights under the Amended Sanofi Agreement. Sanofi also granted the Company non-exclusive, Pursuant to the Original Sanofi Agreement, Sanofi paid the Company an upfront payment of $45.0 million in 2018. Pursuant to the Second Sanofi Amendment, Sanofi paid the Company an additional upfront payment of $300.0 million in August 2020. If Sanofi chooses to exercise its option to extend the Collaboration Term for an additional year, Sanofi has agreed to pay the Company an additional payment of $ million. Amended Sanofi Agreement provides that the Company is eligible to receive aggregate potential payments of up to $ billion upon the achievement of additional specified development, regulatory, manufacturing and commercialization milestones, inclusive of the fee to exercise the option to extend the Collaboration Term. In particular, the Company is entitled to receive development, regulatory and sales milestone payments of up to $ million for each Licensed Field, other than the SARS-CoV-2 Licensed Field, development, regulatory and sales milestone payments of up to $ million in the SARS-CoV-2 Licensed Field, and one-time manufacturing milestone payments of up to $ million. In addition, the Company is entitled to receive a $ million milestone payment from Sanofi following completion of the technology and process transfer. Under the terms of the Amended Sanofi Agreement, Sanofi has also agreed to pay the Company royalties on net sales of mRNA vaccines in the SARS-CoV-2 The Amended Sanofi Agreement provides t h payment obligations. In connection with the execution of the Second Sanofi Amendment, the Company and the Sanofi Investor entered into the Securities Purchase Agreement for the sale and issuance of 4,884,434 shares of the Company’s common stock to the Sanofi Investor at a price of $25.59 per share for an aggregate purchase price of approximately $125.0 million. The closing of the transaction contemplated by the Securities Purchase Agreement was consummated in July 2020, following early termination of the waiting period under the Hart-Scott Rodino Act. Pursuant to the terms of the Securities Purchase Agreement, the Sanofi Investor agreed not to, without the prior written approval of the Company and subject to specified conditions, directly or indirectly acquire shares of the Company’s outstanding common stock, make a tender, exchange, or other offer to acquire shares of the Company’s outstanding common stock, solicit proxies or consents with respect to any matter, or undertake other specified actions related to the potential acquisition of additional equity interests in the Company (the “Standstill Restrictions”). Further, the Sanofi Investor agreed not to, and to cause its affiliates not to, sell or transfer the shares without the prior written approval of the Company subject to specified conditions (the “Lock-Up Lock-Up Sanofi has sole responsibility for all commercialization activities for mRNA vaccines in the Licensed Fields and is obligated to bear all costs in connection with any commercialization in the Licensed Fields. The Company and Sanofi also entered non-clinical non-clinical SARS-CoV-2 Accounting for the Sanofi Collaboration For accounting purposes, the Company has combined the Amended Sanofi Agreement, Securities Purchase Agreement and Supply Agreement because the contracts were negotiated as a package with a single commercial objective, the amount of consideration to be paid in one contract depends on the price or performance of the other contracts, and the goods and services promised in the contracts are a single performance obligation in accordance with ASC 606. The Company accounts for the Amended Sanofi Agreement under ASC 606. In determining the appropriate amount of revenue to be recognized under ASC 606, the Company performed the following steps: (i) identified the promised goods or services in the contract; (ii) determined 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) the Company satisfies each performance obligation. The Company identified the following promised goods or services contained in the Amended Sanofi Agreement: (i) the license it conveyed to Sanofi with respect to the Licensed Fields, (ii) the licensed know-how non-clinical transfer. The Company assessed whether each of these promised goods or services are distinct performance obligations on their own or if they need to be combined with other promises to create a bundle that is a distinct performance obligation. The Company determined that the promised goods and services do not have standalone value and are highly interrelated. Accordingly, the promised goods and services represent one performance obligation. Additionally, Sanofi’s right to exercise its option to extend the research term and have the Company conduct research and development activities to advance mRNA vaccines for up to an additional three infectious disease pathogens were determined at the time of the modification to not represent material rights, based on the criteria of ASC 606, and therefore do not represent a separate performance obligation. Under ASC 606, the Company recognized revenue using the cost-to-cost cost-to-cost out-of-pocket During the year ended December 31, 2020, the Company increased the overall transaction price by $ million. The transaction price as of December 31, 2020 includes the upfront, non-refundable payments of $ million for the transfer of the combined license, supply and development obligations under the Original Sanofi Agreement and Second Sanofi Amendment, the premium paid in consideration for common stock of $ million, which represents the excess of the price paid compared to the fair value of the Company’s common stock on the closing date, under the Securities Purchase Agreement, an estimated $ million in reimbursable employee costs, an estimated $ million in reimbursable development costs including manufacturing costs and out-of-pocket costs paid to third parties and an estimated $ million in milestone payments. Under ASC 606, at the end of each reporting period, the Company re-evaluates the variable consideration determined using either the expected value or most likely outcome approach and re-evaluates the probability that the consideration associated with each milestone or reimbursement will not be subject to a significant reversal in the cumulative amount of revenue recognized, and, if necessary, adjusts the estimate of the overall transaction price. The estimated collaboration budget is consistently re-evaluated and changes to the budget, if any, require approval by the Joint Steering Committee. If an approved change occurs, the Company will re-evaluate the transaction price which could potentially affect the cumulative amount of revenue recognized. As a result of the Second Sanofi Amendment, the Company revised the budget and collaboration plan. The Company has accounted for the Second Sanofi Amendment as a modification to the existing agreement and not as a separate agreement because the additional goods and services are not distinct and therefore form a single performance obligation that is partially satisfied at the date of the contract modification. Changes as cumulative revenue catch-up adjustment $37.3 million. The following table summarizes the Company’s collaboration revenue (in thousands): Year Ended December 31, 2020 2019 Collaboration revenue $ 138,811 $ 7,804 The following table presents the balance of the Company’s contract liabilities (in thousands): December 31, December 31, Contract liabilities Deferred revenue $ 296,222 $ 43,356 Deferred revenue is classified as short-term or long-term in the consolidated balance sheets based on the Company’s estimate of revenue that will be recognized within the next twelve months which is determined by the cost-to-cost |
Intangible Assets and Goodwill
Intangible Assets and Goodwill | 12 Months Ended |
Dec. 31, 2020 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
Intangible Assets and Goodwill | 4. Intangible Assets and Goodwill Acquisition of Shire’s MRT Program In December 2016, the Company entered into an asset purchase agreement (as amended in June 2018) with Shire Human Genetic Therapies, Inc. (“Shire”), a subsidiary of Takeda Pharmaceutical Company Ltd., pursuant to which Shire sold equipment to and assigned to the Company all of its rights to certain patent rights, permits, real property leases, contracts, regulatory documentation, books and records, and materials related to Shire’s mRNA therapy platform (the “MRT Program”), including its cystic fibrosis transmembrane conductance regulator program. Intangible Assets, Net The acquisition of Shire’s MRT Program was accounted for in accordance with the acquisition method of accounting for business combinations. The total purchase consideration transferred was allocated to the tangible and identifiable intangible assets acquired based on their estimated fair values. The tables below present the Company’s definite-lived intangible assets that are subject to amortization and indefinite-lived intangible assets: December 31, 2020 Estimated Life Gross Carrying Amount Accumulated Amortization Impairment Charge Net Carrying Amount (In thousands) Definite-lived intangible assets: MRT 6 years $ 45,992 $ (9,156 ) $ — $ 36,836 Indefinite-lived intangible assets: IPR&D - CF Indefinite 42,291 — — 42,291 Total intangible assets, net $ 88,283 $ (9,156 ) $ — $ 79,127 December 31, 2019 Estimated Life Gross Carrying Amount Accumulated Amortization Impairment Charge Net Carrying Amount (In thousands) Definite-lived intangible assets: MRT 8 years $ 45,992 $ (2,747 ) $ — $ 43,245 Indefinite-lived intangible assets: IPR&D - CF Indefinite 42,291 — — 42,291 IPR&D - OTC Indefinite 18,559 — (18,559 ) — Total intangible assets, net $ 106,842 $ (2,747 ) $ (18,559 ) $ 85,536 Identifiable intangible assets acquired acquisition Upon commencement of the Original Sanofi Agreement, the IPR&D – MRT intangible asset was reclassified from indefinite-lived to definite-lived intangible assets and the Company began amortization of this intangible asset. Amortization will be recorded over the intangible asset’s estimated life based on an economic consumption model. The Company recorded amortization expense of $6.4 million and $ million during the years ended December , and , respectively, related to the definite-lived MRT intangible asset. The estimated aggregate amortization expense for the remainder of the useful life is $ million, $ million, $ million and $ million for the years ending December , , , and , respectively. Indefinite annually to this program. During the year ended December 31, 2020, the Company did not recognize any impairment charges related to its definite-lived IPR&D. Goodwill The excess of the fair value of the consideration transferred over the fair value of identifiable assets acquired in the acquisition of Shire’s MRT Program was allocated to goodwill in the amount of $21.4 million. There have been no changes to the carrying amount of goodwill during the year ended December 31, 2020. Goodwill is not subject to amortization, but is tested annually for impairment or more frequently if there are indicators of impairment. The Company tests its goodwill annually for impairment on October 1 st goodwill |
Fair Value of Financial Assets
Fair Value of Financial Assets and Liabilities | 12 Months Ended |
Dec. 31, 2020 | |
Fair Value Disclosures [Abstract] | |
Fair Value of Financial Assets and Liabilities | 5. Fair Value of Financial Assets and Liabilities The following tables present information Fair Value Measurements as of December 31, 2020 Using: Level 1 Level 2 Level 3 Total Assets: Money market funds $ — $ 273,827 $ — $ 273,827 U.S. treasuries — 292,001 — 292,001 U.S. government agency bonds — 20,000 — 20,000 $ — $ 585,828 $ — $ 585,828 Liabilities: Contingent consideration $ — $ — $ 152,230 $ 152,230 $ — $ — $ 152,230 $ 152,230 Fair Value Measurements as of December 31, 2019 Using: Level 1 Level 2 Level 3 Total Assets: Money market funds $ — $ 56,591 $ — $ 56,591 U.S. government agency bonds — 104,098 — 104,098 $ — $ 160,689 $ — $ 160,689 Liabilities: Contingent consideration $ — $ — $ 103,655 $ 103,655 $ — $ — $ 103,655 $ 103,655 During the years ended December 31, 2020 and 2019, there were no t Cash equivalents as of December 31, 2020 and 2019 consisted of money market funds totaling $273.8 million and $56.6 million, respectively. The money market funds were valued using inputs observable in active markets for similar securities, which represent a Level 2 measurement in the fair value hierarchy. The U.S. treasuries and U.S. The estimated amortized costs and fair value of the Company’s available-for-sale December 31, 2020 December 31, 2019 Amortized Cost Fair Value Amortized Cost Fair Value Due within one year $ 201,606 $ 201,596 $ 103,357 $ 104,098 Due after one year through two years 110,395 110,405 — — Total available-for-sale $ 312,001 $ 312,001 $ 103,357 $ 104,098 Valuation of Contingent Consideration The contingent consideration liability related to the acquisition of Shire’s MRT Program in 2016 was classified as a Level 3 measurement within the fair value hierarchy. The Company may be required to pay future consideration to Shire contingent upon the achievement of potential future milestones and earnout payments. The fair value of the liability to make potential future milestone and earnout payments was estimated by the Company at each reporting date based, in part, on the results of a valuation using a discounted cash flow analysis based on various assumptions, including the amount and timing of cash flows, probability of achieving specified events, discount rate, and the period of time until earnout payments are payable and the conditions triggering the milestone payments are met. The actual settlement of contingent consideration could differ from current estimates based on the actual occurrence of these specified events. The following table presents the unobservable inputs and fair value of the components of the contingent consideration (dollar amounts in thousands): Unobservable Inputs Fair Value at Projected Year of Payment December 31, December 31, Earnout payments 2026 - 2039 142,250 $ 96,097 Milestone payments 2026 - 2032 9,980 7,558 $ 152,230 $ 103,655 The discount rate used in the valuation was 11.0% and 13.5% as of December 31, 2020 and 2019, respectively. The following table presents a roll-forward of the total acquisition-related contingent consideration liability (in thousands): Fair Value Balance as of December 31, 2019 $ 103,655 Increase in fair value of contingent consideration 48,575 Balance as of December 31, 2020 $ 152,230 The increase in the fair value of contingent consideration during the year ended December 31, 2020 was primarily due to the time value of money due to the passage of time and a decrease in the discount rate. |
Property and Equipment, Net
Property and Equipment, Net | 12 Months Ended |
Dec. 31, 2020 | |
Property, Plant and Equipment [Abstract] | |
Property and Equipment, Net | 6. Property and Equipment, Net Property and equipment, net consisted of the following (in thousands): December 31, December 31, 2020 2019 Laboratory equipment $ 12,710 $ 9,044 Computer equipment 922 779 Office equipment 941 883 Leasehold improvements 5,730 5,635 Construction in progress 5,189 3,460 25,492 19,801 Less: Accumulated depreciation and amortization (10,120 ) (7,262 ) $ 15,372 $ 12,539 Depreciation and amortization expense related to property and e |
Accrued Expenses
Accrued Expenses | 12 Months Ended |
Dec. 31, 2020 | |
Payables and Accruals [Abstract] | |
Accrued Expenses | 7. Accrued Expenses Accrued expenses consisted of the following (in thousands): December 31, December 31, 2020 2019 Accrued employee compensation and benefits $ 5,600 $ 3,547 Accrued external research and development expenses 2,805 1,763 Accrued consultant and professional fees 1,489 1,390 Other 3,308 372 $ 13,202 $ 7,072 Included in other accrued expenses as of December 31, 2020 are costs related to entering into the Second Sanofi Agreement. |
Common Stock
Common Stock | 12 Months Ended |
Dec. 31, 2020 | |
Equity [Abstract] | |
Common Stock | 8. Common Stock On July 2, 2018, the Company filed its restated certificate of incorporation, which authorizes the Company to issue up to 200,000,000 shares of common stock, $0.001 par value per share. As of December 31, 2020 and 2019, there were 75,029,625 and 60,022,067 shares of common stock issued and outstanding, respectively. Each share of common stock entitles the holder to one vote for each share of common stock held. Common stockholders are entitled to receive dividends, as declared by the Company’s Board of Directors (the “Board of Directors”). These dividends are subject to the preferential dividend rights of the holders of the Company’s preferred stock. Through December 31, 2020 and 2019, no cash dividends have been declared or paid. |
Incentive Stock Options and Res
Incentive Stock Options and Restricted Stock | 12 Months Ended |
Dec. 31, 2020 | |
Disclosure of Compensation Related Costs, Share-based Payments [Abstract] | |
Incentive Stock Options and Restricted Stock | 9. Incentive Stock Options and Restricted Stock 2018 Equity Incentive Plan On March 7, 2018, the Company’s (the “Board of Directors”), non-qualified The number of shares initially reserved for issuance under the 2018 Plan is the sum of 2,512,187, plus the number of shares (up to 1,013,167 shares) equal to the sum of (i) the number of shares remaining available for issuance under the 2016 Stock Incentive Plan, as amended, (the “2016 Plan”), upon the effectiveness of the 2018 Plan, which was 360,514 shares, and (ii) the number of shares of common stock subject to outstanding awards under the 2016 Plan that expire, terminate or are otherwise surrendered, canceled, forfeited or repurchased by the Company at their original issuance price pursuant to a contractual repurchase right. The number of shares of common stock that may be issued under the 2018 Plan will automatically increase on the first day of each fiscal year, beginning with the fiscal year ending December 31, 2019 and continuing for each fiscal year until, and including, the fiscal year ending December 31, 2028, by an amount equal to the lowest of (i) shares, (ii) % of the outstanding shares of common stock on such date and (iii) an amount determined by the Board of Directors. As of December 31, 2019, there were 4,829,847 shares of common stock reserved for issuance under the 2018 Plan. On January 1, 2020, the number of shares of common stock that may be issued under the 2018 Plan increased by shares of common stock. During the year ended December 31, 2020, a total of 226,495 shares issued under the 20 1 shares of common stock reserved for issuance under the 2018 Plan as of December 31, 2020. The shares of common stock underlying any awards that are forfeited, canceled, held back upon exercise or settlement of an award to satisfy the exercise price or tax withholding, repurchased or are otherwise terminated by the Company under the 2018 Plan will be added back to the shares of common stock available for issuance under the 2018 Plan. The 2018 Plan is administered by the Board of Directors. The exercise prices, vesting periods and other restrictions are determined at the discretion of the Board of Directors, except that the exercise price per share of options may not be less than 100% of the fair market value of the common stock on the date of grant. Stock options awarded under the 2018 Plan expire 10 years after the grant date, unless the Board of Directors sets a shorter term. Awards granted to employees, officers, members of the Board of Directors and consultants typically vest over a period of one Typically, unvested stock options are forfeited upon the recipient cea s 2018 Employee Stock Purchase Plan On March 7, 2018, the Board of Directors, subject to stockholder approval, adopted, and on June 15, 2018, the Company’s stockholders approved the 2018 Employee Stock Purchase Plan (the “2018 ESPP”), which became effective on June 27, 2018. A total of 418,697 shares of common stock were initially reserved for issuance under this plan. The number of shares of common stock that may be issued under the 2018 ESPP will automatically increase on the first day of each fiscal year, beginning with the fiscal year commencing on January 1, 2019 and continuing for each fiscal year until, and including, the fiscal year commencing on January 1, 2029, by an amount equal to the lowest of (i) 837,395 shares, (ii) 1% of the outstanding shares of common stock on such date and (iii) an amount determined by the Board of Directors. In December 2019, the Board of Directors elected to add no shares of common stock to the 2018 ESPP. As of December 31, 2020, 870,096 shares of common stock reserved for issuance under this plan. As of December 31, 2020, 26,964 shares have been issued under the 2018 ESPP. 2016 Stock Incentive Plan The 2016 Plan provided for the grant of stock options, stock appreciation rights, restricted stock and restricted stock units. Shares that are expired, terminated, surrendered or canceled under the 2016 Plan without having been exercised will be available for future grants of awards under the 2018 Plan. In addition, shares of common stock that are tendered to the Company by a participant to exercise an award are added to the number of shares of common stock available for the grant of awards under the 2018 Plan. The 2016 Plan is administered by the Board of Directors. The exercise prices, vesting periods and other restrictions were determined at the discretion of the Board of Directors, except that the exercise price per share of options could not be less than 100% of the fair market value of the common stock on the date of grant. Stock options awarded under the 2016 Plan expire 10 years after the grant date, unless the Board of Directors set a shorter term. Stock options and restricted stock granted to employees, officers, members of the Board of Directors and consultants typically vest over a four-year period. Upon the effectiveness of the 2018 Plan on June 27, 2018, no further awards will be made under the 2016 Plan, but awards outstanding under the 2016 Plan will continue to be governed by their existing terms. Stock Options The following table summarizes the Company’s stock option activity since December 31, 2019 (in thousands, except share and per share amounts): Number of Shares Weighted Average Exercise Price Weighted Average Remaining Contractual Term Intrinsic Value (in years) Outstanding as of December 31, 2019 8,646,378 $ 8.06 8.42 $ 3,687 Granted 3,313,584 $ 10.26 Exercised (1,551,178 ) $ 7.99 Forfeited (851,393 ) $ 8.57 Outstanding as of December 31, 2020 9,557,391 $ 8.79 7.92 $ 93,256 Exercisable as of December 31, 2020 4,783,112 $ 7.86 7.11 $ 50,567 Vested and expected to vest as of December 31, 2020 9,557,391 $ 8.79 7.92 $ 93,256 The aggregate intrinsic value of options is calculated as the difference between the exercise price of the options and the fair value of the Company’s common stock for those options that had exercise prices lower than the fair value of the Company’s common stock. The aggregate intrinsic value of stock options exercised during the years ended December 31, 2020 and 2019 was $19.2 million and $0.6 million, respectively. The weighted average grant-date fair value per share of stock options granted was $6.31 and $5.59 during the years ended December 31, 2020 and 2019, respectively. Stock Option Valuation The fair value of stock option grants is estimated using the Black-Scholes option-pricing model. The Company completed its initial public offering in July 2018 and therefore lacks company-specific historical and implied volatility information before that date. Therefore, it estimates its expected stock volatility based on the historical volatility of a publicly traded set of peer companies and expects to continue to do so until such time as it has adequate historical data regarding the volatility of its own traded stock price. For options with service-based vesting conditions, the expected term of the Company’s stock options has been determined utilizing the “simplified” method for awards that qualify as “plain-vanilla” options. The expected term of stock options granted to non-employees The following table presents, on a weighted average basis, the assumptions used in the Black-Scholes option-pricing model to determine the grant-date fair value of stock options granted to employees and directors: Year Ended December 31, 2020 2019 Risk-free interest rate 0.75 % 2.36 % Expected term (in years) 6.1 6.0 Expected volatility 68.8 % 73.0 % Expected dividend yield 0 % 0 % Restricted Common Stock The following table summarizes the Company’s restricted stock activity since December 31, 2019: Number of Shares Weighted Average Grant-Date Fair Value Unvested restricted common stock outstanding as of December 31, 2019 34,168 $ 1.28 Forfeited restricted common stock — $ — Vested restricted common stock (34,168 ) $ 1.28 Unvested restricted common stock outstanding as of December 31, 2020 — $ — Stock-Based Compensation Stock-based compensation expense was classified in the consolidated statements of operations as follows (in thousands): Year Ended December 31, 2020 2019 Research and development expenses $ 8,818 $ 4,786 General and administrative expenses 8,802 6,548 $ 17,620 $ 11,334 Included in research and development stock-based compensation expense for the year ended December 31, 2020 was $2.4 million related to the modification of options in connection with the resignation of the Company’s former Executive Vice President and Founder (“EVP and Founder”). In connection with this resignation the Company entered into a separation agreement with the EVP and Founder. Under the terms of the separation agreement, vesting of options for the purchase of 176,266 shares of common stock held by the EVP and Founder was accelerated with no change to the exercise price of such options. Stock options for the purchase of 550,278 shares of common stock, representing all of the vested options held by the EVP and Founder as of the date of his resignation, are exercisable for 18 months following his resignation. Included in general and administrative stock-based compensation expense for the year ended December 31, 2020 was $1.0 million related to the modification of options in connection with the departure of the As of December 31, 2020, total unrecognized compensation cost related to the unvested stock-based awards was $25.5 million, which is expected to be recognized over weighted average periods of 2.5 years. |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2020 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 10. Income Taxes During the years ended December 31, 2020 and 2019, the Company recognized an income tax benefit of $ 0 MRT Program. All of the Company’s operating losses since inception have been g e A reconciliation of the U.S. federal statutory income tax rate to the Company’s effective income tax rate is as follows: Year Ended December 31, 2020 2019 U.S. federal statutory income tax rate (21.0 )% (21.0 )% State income taxes, net of federal benefit (9.4 ) (6.6 ) Research and development tax credits and orphan drug credit (11.6 ) (6.8 ) Stock-based compensation (3.5 ) 0.5 Other permanent differences 0.0 0.2 Change in deferred tax asset valuation allowance 37.0 33.6 Officers compensation 3.5 0.0 NOL and tax credit expiration under 382 4.8 0.0 Other 0.2 (0.3 ) Effective income tax rate 0.0 % (0.4 )% Components of the Company’s deferred tax assets were as follows (in thousands): December 31, 2020 2019 Deferred tax assets: Net operating loss carryforwards $ 64,639 $ 61,460 Research and development tax credit and orphan drug credit carryforwards 28,406 21,830 Intangibles 7,570 3,607 Deferred revenue 3,753 11,845 Depreciation and amortization 17,305 2,253 Accrued expenses and other 4,583 5,354 Total deferred tax assets 126,256 106,349 Valuation allowance (126,256 ) (106,346 ) Net deferred tax assets $ — $ 3 As of December 31, 2020, the Company had federal net operating loss carryforwards of $ 239.3 116.1 227.6 7.0 4.3 17.4 0.8 Utilization of the net operating loss carryforwards and research and development tax credit carryforwards may be subject to a substantial annual limitation under Section 382 of the Internal Revenue Code of 1986, as amended, due to ownership changes that have occurred previously or that could occur in the future. These ownership changes may limit the amount of carryforwards that can be utilized annually to offset future taxable income. In general, an ownership change, as defined by Section 382, results from transactions increasing the ownership of certain stockholders or public groups in the stock of a corporation by more than 50% over a three The Company has conducted a study to assess whether an ownership change has occurred and determined there have been multiple ownership changes since inception. As the Company has experienced an ownership change, as defined by Section 382, utilization of the net operating loss carryforwards and research and development tax credit carryforwards are subject to an annual limitation under Section 382, which is determined by first multiplying the value of the Company’s stock at the time of the ownership change by the applicable long-term tax-exempt a In addition, the Company has not conducted a study of its research and development tax credit carryforwards. This study may result in an adjustment to the Company’s research and development tax credit carryforwards. Until a study is completed and any adjustment is known, no amounts are being presented as an uncertain tax position. The Company has evaluated the positive and negative evidence bearing upon its ability to realize the deferred tax assets. Management has considered the Company’s history of cumulative net losses incurred since inception and its lack of commercialization of any products or generation of any revenue from product sales since inception and has concluded that it is more likely than not that the Company will not realize all of the benefits of the deferred tax assets. At December 31, 20 20 Changes in the valuation allowance for deferred tax assets during the years ended December 31, 2020 and 2019 related primarily to the increase in net operating loss carryforwards and research and development tax credit carryforwards in 2020 and 2019, and were as follows (in thousands): Year Ended December 31, 2020 2019 Valuation allowance at beginning of year $ (106,346 ) $ (68,143 ) Increases recorded to income tax provision (19,910 ) (38,203 ) Valuation allowance at end of year $ (126,256 ) $ (106,346 ) The Company had not recorded any amounts for unrecognized tax benefits as of December 31, 2020 and 2019. The Company files income tax returns in the U.S. and Massachusetts. The federal and state returns are generally subject to tax examinations for the tax years ended December 31, 2017 to the present. There are currently no pending tax examinations. To the extent the Company has tax attribute carryforwards, the tax years in which the attribute was generated may still be adjusted upon examination by the Internal Revenue Service and state tax authorities to the extent utilized in a future or prior period. The Company’s policy is to record interest and penalties related to income taxes as part of its income tax provision. |
Net Loss per Share
Net Loss per Share | 12 Months Ended |
Dec. 31, 2020 | |
Earnings Per Share [Abstract] | |
Net Loss per Share | 11. Net Loss per Share Net Loss per Share Basic and diluted net loss per share attributable to common stockholders was calculated as follows (in thousands, except share and per share amounts): Year Ended December 31, 2020 2019 Numerator: Net loss $ (53,787 ) $ (113,293 ) Denominator: Weighted average common shares outstanding—basic and diluted 67,520,886 51,445,539 Net loss per share—basic and diluted $ (0.80 ) $ (2.20 ) The Company excluded 124,697 shares of restricted common stock, presented on a weighted average basis, from the calculations of basic net loss per share attributable to common stockholders for the year ended December 31, 2019 because those shares had not vested. The Company’s potentially dilutive securities, which include stock options and unvested restricted common stock, have been excluded from the computation of diluted net loss per share attributable to common stockholders as the effect would be to reduce the net loss per share. Therefore, the weighted average number of shares of common stock outstanding used to calculate both basic and diluted net loss per share attributable to common stockholders is the same. The Company excluded the following potential shares of common stock, 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 because including them would have had an anti-dilutive effect: Year Ended December 31, 2020 2019 Options to purchase common stock 9,557,391 8,646,378 Unvested restricted common stock — 34,168 9,557,391 8,680,546 |
Leases
Leases | 12 Months Ended |
Dec. 31, 2020 | |
Leases [Abstract] | |
Leases | 12. Leases Suite Retention Agreements In September 2019, the Company entered into a suite retention and development agreement with Albany Molecular Research, Inc. (“AMRI”) under which a series of cleanroom suites were built at AMRI’s manufacturing facility in accordance with the Company’s objectives (“AMRI Agreement”). The AMRI Agreement continues for five years after the build-out build-out (“Build-Out Build-Out Build-Out Build-Out lease commencement date. Upon the build-out completion date of August , , the Company determined that it gained control of the space, in accordance with ASC , which resulted in the recording of an ROU asset and related lease liability of approximately $ million and $ million, respectively, with the difference being due to the elimination of previously recorded prepaid rent. As of August , , the Company began paying monthly fees of $ million, which are subject to a % increase on January of each calendar year following the first anniversary of the build-out completion. The option to extend the lease for an additional three years was not included in the lease liability as of December , as the Company is not reasonably certain it will exercise this option. In October 2020, the Company entered into a suite retention agreement with Biomedical Research Models, Inc. (“Biomere”) under which the Company will lease two exclusive procedure rooms and one housing and maintenance room in the Worcester, Massachusetts facility. The lease term is 13 months, commencing on December 1, 2020 (“Biomere Lease Commencement”). The Company can terminate the agreement for convenience, and without penalty, with 60 Real Estate Lease In June 2017, the Company entered into an operating lease for office and laboratory space at its headquarters in Lexington, Massachusetts. The Company occupies approximately 59,000 square feet of space under a 10-year Equipment Lease In March 2018, the Company entered into an operating lease for communications equipment for use at its office and laboratory space in Lexington, Massachusetts. The term of the lease is five years, expiring in March 2023 The Company excludes leases with an initial term of one year or less in the recognized ROU asset and lease liabilities. The Company recognizes lease expense for these leases on a straight-line basis over the lease term. For lease agreements entered into or reassessed after the adoption of ASC , lease and non-lease components are combined into a single lease component. The Company’s leases have remaining lease terms of up to , excluding two five-year options to extend the real estate lease after the expiration of the initial term. The Company believes the Lexington real estate lease, together with the anticipated relocation to a space in Waltham, Massachusetts as described below, will be sufficient to meet its needs for the foreseeable future and that suitable additional space will be available as and when needed. The components of lease cost were as follows (dollar amounts in thousands): Year Ended 2020 2019 Lease cost Operating lease cost $ 8,099 $ 2,692 Total lease cost $ 8,099 $ 2,692 Other information Operating cash flows from operating leases $ 7,775 $ 2,583 Operating lease liabilities arising from obtaining right-of-use $ 54,063 — Weighted-average remaining lease term 5 years 8 years Weighted-average discount rate 11.9 % 17.5 % As of December 31, 2020, maturities of operating lease liabilities are as follows (in thousands): December 31, 2020 2021 $ 18,067 2022 15,178 2023 15,591 2024 16,050 2025 12,029 2026 and thereafter 7,134 Total future minimum lease payments 84,049 Less: imputed interest (21,363 ) Present value of lease liabilities $ 62,686 As of December 31, 2019, maturities of operating lease liabilities are as follows (in thousands): December 31, 2019 2020 $ 2,659 2021 2,737 2022 2,818 2023 2,860 2024 2,937 2025 and thereafter 10,160 Total future minimum lease payments 24,171 Less: imputed interest (11,557 ) Present value of lease liabilities $ 12,614 As most of the Company’s leases do not provide an implicit rate, the Company uses its incremental borrowing rate which are the rates incurred to borrow on a collateralized basis over a term equal to the lease payments in a similar economic environment in determining the present value of lease payments. The Company used the incremental borrowing rate on January 1, 2019 for operating leases that commenced prior to that date and for all subsequent leases the Company used an appropriate incremental borrowing rate upon commencement date. In October 2020, the Company entered into a suite retention agreement with Azzur Cleanrooms-on-Demand months, commencing on July 1, 2021, with the option to extend the term with three months’ notice prior to the termination 2023. The Company can terminate the agreement for convenience, and without penalty, with three months’ written notice. The lease does not contain any lease incentives or renewal options. The Company has determined this is a lease under ASC 842. As of December 31, 2020, the Company has determined that it does not have control of the space, as defined in ASC 842, during the build-out and as such, this lease was On November 3, 2020 (the “Lease Commencement Date”) , ten-year build-out those. The Company expects to spend between $9.0 million and $11.0 million on the construction of lessor assets, which represents the cost of the project that exceeds the tenant allowances. Initial base rent, which commences 12 months after the Lease Commencement Date, shall be build-out |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2020 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 13. Commitments and Contingencies Research, Supply and License Agreements Roche Master Supply Agreement The Company is a party to a master supply agreement with Roche Diagnostics Corporation (“Roche”) pursuant to which Roche will custom manufacture certain products for the Company. The agreement requires the Company to purchase from Roche specified manufactured products and the related raw materials in an amount equal to the greater of (i) quantities of raw materials in the Company’s annual forecast to be purchased or (ii) 80% of the Company’s demand for products as the same or similar type (the “Purchase Commitment”). In June 2017, the Company exercised its option under the agreement to extend the agreement through December 31, 2024. In September 2018, the Company and Roche amended the agreement to remove and replace the Purchase Commitment for certain manufactured products and related raw materials supplied by Roche. The agreement, as amended, specifies a minimum purchase requirement for certain custom manufactured products. As of December 31, 2020, the Company’s purchase commitments under the agreement totaled $14.0 million, with $3.5 million committed as payments each year from 2021 to 2024. Research and development expenses related to this agreement totaled $3.3 million and $12.5 million during the years ended December 31, 2020 and 2019, respectively. MIT Research Agreement In September 2019, the Company entered into a research agreement with the Massachusetts Institute of Technology (“MIT”) pursuant to which the Company is obligated to reimburse MIT up to $4.1 million for specified direct and indirect costs to be incurred from January 2020 through December 2022 for specified research activities conducted for the Company (the “2019 MIT Agreement”). As of December 31, 2020 and 2019, the Company had paid MIT $1.7 million and $0.3 million, respectively, towards the total committed amount. Research and development expenses related to this agreement during the years ended December 31, 2020 and 2019 were $1.4 million and $0, respectively. There were no amounts payable by the Company under the agreement as of December 31, 2020. The 2019 MIT Agreement expires in December 2022 and may be extended thereafter by mutual agreem e MIT Exclusive Patent License Agreement The Company is a party to an exclusive patent license agreement with MIT pursuant to which the Company received an exclusive license under the licensed patent rights to develop, manufacture and commercialize any product containing both (i) any RNA sequences, including mRNA, that encode a protein or peptide suitable for human therapeutic use which may include operably linked non-coding non-coding The Company has the right to grant sublicenses under this license. The patent rights licensed to the Company by MIT include claims that cover certain of the Company’s customized lipid Under the license agreement, the Company is obligated to make annual license maintenance payments to MIT, payable on January 1 of each calendar year, of up to $0.2 million, which may be credited against royalties subsequently due on net sales of licensed products earned in the same calendar year. The Company paid annual license maintenance fees of $0.2 million to MIT during each of the years ended December 31, 2020 and 2019. The Company is also obligated to make milestone payments to MIT aggregating up to $1.375 million upon the achievement of specified clinical and regulatory milestones with respect to each licensed product and $1.250 million upon the Company’s first commercial sale of each licensed product, and to pay royalties of a low single-digit percentage to MIT based on the Company’s, and any of its affiliates’ and sublicensees’, net sales of licensed products. The royalties are payable on a product-by-product country-by-country last-to-expire MIT-licensed The The agreement obligates the Company to use commercially reasonable efforts and expend a minimum amount of resources each year to develop licensed products in accordance with a development plan, and a development milestone timetable specified in the agreement; to use commercially reasonable efforts to commercialize licensed products; and upon commercialization, to make the licensed products reasonably MIT has the right to terminate the agreement if the Company fails to pay amounts when due or otherwise materially breaches the agreement and fails to cure such nonpayment or breach within specified cure periods or in the event the Company ceases to carry on its business related to the agreement. In the event of a termination due to the Company’s breach caused by a due diligence failure of a licensed product, but where the Company has fulfilled its obligations with respect to a different licensed product, MIT may not terminate the agreement with respect to the different licensed product. MIT may immediately terminate the agreement if the Company or any of its affiliates brings specified patent challenges against MIT or assists others in bringing a patent challenge against MIT. The Company has the right to terminate the agreement for its convenience at any time on three months’ prior written notice to MIT and payment of all amounts due to MIT through the date of termination. The Company’s patent rights, and the rights of its affiliates and sublicensees, in specified licensed products may also terminate if the Company, its affiliates or MIT receives a request from a third party to develop such licensed product for which the Company is unable to, within nine months of receiving notice of any such request, either demonstrate that the Company has initiated a fully funded project for the commercial development of such licensed product, and provide a business plan with acceptable milestones; demonstrate that the licensed product proposed by such third party would be competitive with a licensed product for which the Company has initiated a fully funded project; or enter into a sublicense agreement with such third party on commercially reasonable terms, and, in each case, MIT, in its sole discretion, grants a license to such third party for the specified patent rights. Expenses related to this agreement totaled $0.2 million during each of the years ended December 31, 2020 and 2019. Indemnification Agreements In the ordinary course of business, the Company may provide indemnification of varying scope and terms to vendors, lessors, 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, the Company has entered into indemnification agreements with members of its board of directors that will require the Company, 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 the Company could be required to make under these indemnification agreements is, in many cases, unlimited. To date, the Company has not incurred any material costs as a result of such indemnifications. The Company does not believe that the outcome of any claims under indemnification arrangements will have a material effect on its financial position, results of operations or cash flows, and it has not accrued any liabilities related to such obligations in its consolidated financial statements as of December 31, 2020 and 2019. Legal Proceedings The Company is not a party to any litigation and does not have contingency reserves established for any litigation liabilities. |
Related Party Transactions
Related Party Transactions | 12 Months Ended |
Dec. 31, 2020 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | 14. Related Party Transactions Private Placement In connection with a private placement of the Company’s common stock in May 2019, entities affiliated with Baupost Group, L.L.C. (“Baupost”), a substantial stockholder, purchased 2,352,941 shares of the Company’s common stock at a price per share of $8.50 for an aggregate purchase price of $20.0 million. Public Offering In connection with a public offering of the Company’s common stock in September 2019, Baupost purchased 5,000,000 shares of the Company’s common stock at a price per share of $10.00 for an aggregate purchase price of $50.0 million. In connection with a public offering of the Company’s common stock in June 2020, Baupost purchased 500,000 shares of the Company’s common stock at a price per share of $22.00 for an aggregate purchase price of $11.0 million. |
Benefit Plans
Benefit Plans | 12 Months Ended |
Dec. 31, 2020 | |
Retirement Benefits [Abstract] | |
Benefit Plans | 15. Benefit Plans The Company has established a defined-contribution retirement plan under Section 401(k) of the Code. This plan covers all employees who meet minimum age and service requirements and allows participants to defer a portion of their annual compensation on a pre-tax |
Selected Quarterly Financial In
Selected Quarterly Financial Information (Unaudited) | 12 Months Ended |
Dec. 31, 2020 | |
Selected Quarterly Financial Information [Abstract] | |
Selected Quarterly Financial Information | 16. Selected Quarterly Financial Information (Unaudited) The following table contains quarterly financial information for 2020 and 2019 (in thousands, except per share amounts). The Company believes that the following information reflects all normal recurring adjustments necessary for a fair statement of the information for the periods presented. The operating results for any quarter are not necessarily indicative of results for any future period. 2020 First Quarter Second Quarter Third Quarter Fourth Quarter Total Collaboration revenue $ 4,654 $ 16,319 $ 66,446 $ 51,392 $ 138,811 Total operating expenses 19,445 52,950 49,697 72,034 194,126 Income (loss) from operations (14,791 ) (36,631 ) 16,749 (20,642 ) (55,315 ) Net income (loss) (14,282 ) (36,288 ) 17,344 (20,561 ) (53,787 ) Net income (loss) per share applicable to common $ (0.24 ) $ (0.58 ) $ 0.24 $ (0.22 ) $ (0.80 ) Net income (loss) per share applicable to common stockholders—diluted $ (0.24 ) $ (0.58 ) $ 0.23 $ (0.22 ) $ (0.80 ) 2019 First Quarter Second Quarter Third Quarter Fourth Quarter Total Collaboration revenue $ 1,474 $ 1,174 $ 1,266 $ 3,890 $ 7,804 Total operating expenses 35,679 29,364 22,901 35,629 123,573 Loss from operations (34,205 ) (28,190 ) (21,635 ) (31,739 ) (115,769 ) Net loss (33,198 ) (27,832 ) (21,227 ) (31,036 ) (113,293 ) Net loss per share applicable to common stockholders—basic and diluted $ (0.74 ) $ (0.57 ) $ (0.41 ) $ (0.48 ) $ (2.20 ) During |
Subsequent Event
Subsequent Event | 12 Months Ended |
Dec. 31, 2020 | |
Subsequent Events [Abstract] | |
Subsequent Event | 17. Subsequent Events On January 20, 2021, the Board of Directors adopted a 2021 Inducement Stock Incentive Plan (the “2021 Plan”), pursuant to which the Company may grant non-statutory units and other stock-based awards with respect to an aggregate of 2,612,550 shares of common stock. Awards under the 2021 Plan may only be granted to persons who (i) were not previously an employee or director of the Company or (ii) are commencing employment with the Company following a bona fide period of non-employment, |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2020 | |
Accounting Policies [Abstract] | |
Use of Estimates | Use of Estimates The preparation of the Company’s consolidated financial COVID-19 within its financial statements and have determined them to be immaterial. There may be changes to those estimates in future periods periodically changes estimates are recorded in the period in which they become known. Actual results could differ from those estimates. |
Cash and Cash Equivalents | Cash and Cash Equivalents All highly liquid investments purchased with an original maturity date of three months or less at the date of purchase are considered to be cash equivalents. Cash equivalents consisted of money market funds as of December 31, 2020 and 2019. |
Investments | Investments Available-for-sale securities consist of investments with original maturities greater than 90 days at acquisition date. The Company has classified its investments with maturities beyond one year as short term, based on their highly liquid nature and because such available-for-sale securities represent the investment of cash that is available for current operations. The Company’s debt security investments are classified as available-for-sale and are carried at fair value, with the unrealized gains and losses reported as a component of accumulated other comprehensive income (loss) in stockholders’ equity (deficit). Realized gains and losses and declines in value determined to be other than temporary are based on the specific identification method and are included as a component of other income (expense), net in the consolidated statements of operations. The Company evaluates its investments with unrealized losses for other-than-temporary impairment. When assessing investments for other-than-temporary declines in value, the Company considers such factors as, among other things, how significant the decline in value is as a percentage of the original cost, how long the market value of the investment has been less than its original cost, the Company’s ability and intent to retain the investment for a period of time sufficient to allow for any anticipated recovery in fair value and market conditions in general. If any adjustment to fair value reflects a decline in the value of the investment that the Company considers to be “other than temporary,” the Company reduces the investment to fair value through a charge to the statement of operations. No such adjustments were necessary during the periods presented |
Concentrations of Credit Risk | Concentrations of Credit Risk Financial instruments that potentially subject the Company to concentrations of credit risk consist principally of cash and cash equivalents as well as investments. Cash, cash equivalents and investments consist of demand deposits, money market funds, U.S. treasuries and U.S. government agency bonds. The Company generally maintains balances in various operating accounts with financial institutions that management believes to be of high credit quality, in amounts that may exceed federally insured limits. The Company has not experienced any losses related to its cash, cash equivalents and investments and does not believe that it is subject to unusual credit risk beyond the normal credit risk associated with commercial banking relationships. |
Restricted Cash | Restricted Cash In connection with its operating lease commitments, the Company issued letters of credit collateralized by cash deposits that are classified as restricted cash in the consolidated balance sheets. Restricted cash amounts have been classified as current assets based on the release dates of the restrictions under the letters of credit, which occur annually. |
Property and Equipment | Property and Equipment Property and equipment are stated at cost less accumulated depreciation and amortization. Depreciation and amortization expense is recognized using the straight-line method over the estimated useful lives of each asset. Estimated useful lives are periodically assessed to determine if changes are appropriate. Upon retirement accumulated Estimated Useful Life Laboratory equipment 5 years Computer equipment 3 years Office equipment 5 years Leasehold improvements Shorter of lease term or 10 years Costs for capital assets not yet placed into service are capitalized as construction-in-progress amortized Property and equipment are tested for recoverability whenever events or changes in business circumstances |
Leases | Leases The Company adopted Accounting Standards Codification (“ASC”) Topic 842, Leases (ASC 842), using a modified retrospective approach, as of January 1, 2019, with no restatement of prior periods or cumulative adjustment to retained earnings. The Company elected the package of practical expedients, which permits the Company not to reassess under the new standards for prior conclusions about lease identification, lease classification and initial direct costs. The Company did not apply the hindsight practical expedient when determining the lease term for existing leases and assessing impairment of expired or existing non-lease The Company accounts for a contract as a lease when it has the right to control the asset for a period of time while obtaining substantially all of the asset’s economic benefits. The Company determines if an arrangement is a lease or contains an embedded lease at inception. For arrangements that meet the definition of a lease, the Company determines the initial classification and measurement of its operating right-of-use In addition to rent, the leases may require the Company to pay additional amounts for taxes, insurance, maintenance, and other expenses, which are generally referred to as non-lease |
Revenue Recognition | Revenue Recognition The terms of the Company’s collaboration agreements may include consideration such as non-refundable The Company had no revenue prior to the Amended Sanofi Agreement, therefore the adoption of ASC 606, Revenue from Contracts with Customers The Company recognizes the transaction The Amended Sanofi Agreement entitles the Company to additional payments upon the achievement of performance-based milestones. These milestones are generally categorized into three types: development milestones, generally based on the advancement of the Company’s pipeline and initiation of clinical trials; regulatory milestones, generally based on the submission, filing or approval of regulatory applications such as a new drug application in the United States; and sales-based milestones, generally based on meeting specific thresholds of sales in certain geographic areas. The Company is also eligible to receive from Sanofi tiered royalty payments on worldwide net sales of mRNA vaccines. For each collaboration that includes development milestone payments, the Company evaluates whether it is probable that the consideration associated with each milestone will not be subject to a significant reversal in the cumulative amount of revenue recognized. Amounts that meet this threshold are included in the transaction price using the most likely amount method, whereas amounts that do not meet this threshold are considered constrained and excluded from the transaction price until they meet this threshold. Milestones tied to regulatory approval, and therefore not within the Company’s control, are considered constrained until such approval is received. Upfront and ongoing development milestones per the collaboration agreements are not subject to refund if the development activities are not successful. At the end of each subsequent reporting period, the Company re-evaluates catch-up ASC 606 requires the Company to allocate the arrangement consideration on a relative standalone selling price basis for each performance obligation after determining the transaction price of the contract and identifying the performance obligations to which that amount should be allocated. The relative standalone selling price is defined in ASC 606 as the price at which an entity would sell a promised good or service separately to a customer. If other observable transactions in which the Company has sold the same performance obligation separately are not available, the Company is required to estimate the standalone selling price of each performance obligation. Key assumptions to determine the standalone selling price may include forecasted revenues, development timelines, reimbursement rates for personnel costs, discount rates and probabilities of technical and regulatory success. Whenever the Company determines that a contract should be accounted for as a combined performance obligation, which is recognized over time, it will utilize the cost-to-cost cost-to-cost The Company evaluates its collaborative agreements for proper classification in the consolidated statements of operations based on the nature of the underlying activity. Transactions between collaborators recorded in the Company’s consolidated statements of operations are recorded on either a gross or net basis, depending on the characteristics of the collaborative relationship. For revenue generating arrangements where the Company estimate the fair value of the benefit received. Payments to a customer that are deemed a reduction of the transaction price are recorded first as a reduction of revenue, to the extent of both cumulative revenue recorded to date and probable future revenues, which include any unamortized deferred revenue balances, under all arrangements with such customer, and then as an expense. Payments that are not deemed to be a reduction of the transaction price are recorded as an expense. Consideration that does not meet the requirements to satisfy the above revenue recognition criteria is a contract liability and is recorded as deferred revenue in the consolidated balance sheets. Although the Company follows detailed guidelines in measuring revenue, certain judgments affect the application of its revenue policy. For example, in connection with the Amended Sanofi Agreement, the Company has recorded short-term and long-term deferred revenue on its consolidated balance sheets based on the Company’s best estimate of when such revenue will be recognized. Short-term deferred revenue consists of amounts that are expected to be recognized as revenue in the next 12 months. Amounts that the Company expects will not be recognized within the next 12 months are classified as long-term deferred revenue. The estimate of deferred revenue also reflects management’s estimate of the periods of the Company’s involvement in certain of its collaborations. The Company’s performance obligations under these collaborations consist of participation on steering committees and the performance of other research and development services. In certain instances, the timing of satisfying these obligations can be difficult to estimate. Accordingly, the Company’s estimates may change in the future. Such changes to estimates would result in a change in revenue recognition amounts. If these estimates and judgments change over the course of these agreements, it may affect the timing and amount of revenue that the Company will recognize and record in future periods. At December 31, 2020, the Company had short-term and long-term deferred revenue of $67.6 million and $228.7 million, respectively, related to the Sanofi collaboration. Under ASC 606, the Company will recognize revenue and record |
In-Process Research and Development | In-Process The fair value of in-process a The fair value of an IPR&D intangible asset is typically determined using a discounted cash flow analysis based on various assumptions, including the amount and timing of future cash flows and the probability of technical success. The net cash flows are then adjusted to present value by applying an appropriate discount rate that reflects the risk factors associated with the cash flow streams. Indefinite-lived IPR&D is not subject to amortization, but is tested annually for impairment or more frequently if there are indicators of impairment. The Company tests its indefinite-lived IPR&D annually for impairment on October 1st. In testing indefinite-lived IPR&D for impairment, the Company has the option to first assess qualitative factors to determine whether the existence of events or circumstances would indicate that it is more likely than not that its fair value is less than its carrying amount, or the Company can perform a quantitative impairment analysis to determine the fair value of the indefinite-lived IPR&D without performing a qualitative assessment. Qualitative factors that the Company considers include significant underperformance of the business in relation to expectations, significant negative industry or economic trends and significant changes or planned changes in the use of the assets. If the Company chooses to first assess qualitative factors and the Company determines that it is more likely than not that the fair value of the indefinite-lived IPR&D is less than its carrying amount, the Company would then determine the fair value of the indefinite-lived IPR&D. Under either approach, if the fair value of the indefinite-lived IPR&D is less than its carrying amount, an impairment charge is recognized in the consolidated statements of operations. Definite-lived IPR&D is recorded at fair value and amortized over the greater of economic consumption or on a straight-line basis over its estimated useful life. Definite-lived IPR&D is tested for recoverability whenever events or changes in business circumstances indicate that the carrying amount of the assets may not be fully recoverable. If an impairment review is performed to evaluate an asset group for recoverability, the Company compares the forecasted undiscounted cash flows expected to result from the use and eventual disposition of the asset group to its carrying value. An impairment loss would be recognized when estimated undiscounted future cash flows expected to result from the use and eventual disposition of an asset group are less than its carrying amount. The impairment loss would be based on the excess of the carrying value of the impaired asset group over its fair value, determined based on discounted cash flows using market participant assumptions. |
Goodwill | Goodwill Goodwill represents the excess of the fair value of the consideration transferred over the fair value In testing goodwill for impairment, the Company has the option to first assess qualitative factors to determine whether it is necessary to perform a quantitative impairment test. If the Company elects this option and determine, as a result of the qualitative assessment, that it is more likely than not that the fair value of a reporting unit is less than its carrying amount, the quantitative impairment test is required; otherwise, no further testing is required. Among other relevant events and circumstances that affect the fair value of reporting units, the Company considers individual factors, such as microeconomic conditions, changes in the industry and the markets in which it operates as well as historical and expected future financial performance. Alternatively, the Company may elect to not first assess qualitative factors and instead immediately perform the quantitative impairment test. In 2020, the Company elected to early adopt guidance |
Fair Value Measurements | Fair Value Measurements Certain assets and liabilities of the Company are carried at fair value under GAAP. Fair value is defined as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. Valuation techniques used to measure fair value must maximize the use of observable inputs and minimize the use of unobservable inputs. Financial assets and liabilities carried at fair value are to be classified and disclosed in one of the following three levels of the fair value hierarchy, of which the first two are considered observable and the last is considered unobservable: • Level 1—Quoted prices in active markets for identical assets or liabilities. • Level 2—Observable inputs (other than Level 1 quoted prices), such as quoted prices in active markets for similar assets or liabilities, quoted prices in markets that are not active for identical or similar assets or liabilities, or other inputs that are observable or can be corroborated by observable market data. • Level 3—Unobservable inputs that are supported by little or no market activity that are significant to determining the fair value of the assets or liabilities, including pricing models, discounted cash flow methodologies and similar techniques. The Company’s cash equivalents and investments are carried at fair value, determined based on Level 2 inputs in the fair value hierarchy described above (see Note 5). The Company’s contingent consideration liability is carried at fair value, determined based on Level 3 inputs in the fair value hierarchy described above (see Note 5). The carrying values of the Company’s short-term collaboration receivables, prepaid expenses and other current assets, accounts payable, accrued expenses and other current liabilities approximate their fair values due to the short-term nature of these assets and liabilities. |
Segment Information | Segment Information The Company manages its operations as a single operating segment for the purposes of assessing performance and making operating decisions. The Company’s primary focus is on applying its MRT platform to treat pulmonary diseases caused by insufficient protein production or where production of proteins can modify disease. The Company is also pursuing the applicability of its MRT platform for the development of mRNA vaccines for infectious diseases under a collaboration with Sanofi. |
Research and Development Costs | Research and Development Costs Costs associated with internal research and development and external research and development services, including drug development and preclinical studies, are expensed as incurred. Research and development expenses include costs for salaries, employee benefits, subcontractors, facility-related expenses, depreciation and amortization, including amortization related to definite-lived IPR&D intangible assets, stock-based compensation, third-party license fees, laboratory supplies, and external costs of outside vendors engaged to conduct discovery, preclinical and clinical development activities and clinical trials as well as to manufacture clinical trial materials, and other costs. Research and development costs also include costs related to performing the Company’s obligations under the Amended Sanofi Agreement. The Company recognizes external research and development costs based on an evaluation of the progress to completion of specific tasks using information provided to the Company by its service providers. Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are recorded as prepaid expenses. Such prepaid expenses are recognized as an expense when the services have been performed or the goods have been delivered, or when it is no longer expected that the goods will be delivered or the services rendered. Upfront payments, milestone payments (other than those deemed contingent consideration in a business combination) and annual maintenance fees under license agreements are expensed in the period in which they are incurred. |
Research and Development Contract Costs and Accruals | Research and Development Contract Costs and Accruals The Company has entered into various research and development-related contracts with companies both inside and outside of the United States. The related costs are recorded as research and development expenses as incurred. The Company records accruals for estimated ongoing research and development costs. When actual costs. |
Patent Costs | Patent Costs All patent-related costs incurred in connection with filing and prosecuting patent applications are expensed as incurred due to the uncertainty about the recovery of the expenditure. Amounts incurred are classified as general and administrative expenses. |
Stock-Based Compensation | Stock-Based Compensation The Company measures all stock-based awards granted to employees, directors and non-employee The Company classifies stock-based compensation expense in its consolidated statements of operations in the same manner in which the award recipient’s payroll costs are classified or in which the award recipient’s service payments are classified. |
Comprehensive Income (loss) | Comprehensive Income (Loss) Comprehensive income (loss) includes net loss as well as other changes in stockholders’ equity (deficit) that result from transactions and economic events other than those with stockholders. The Company’s only element of other comprehensive income (loss) was unrealized gains (losses) on investments, available-for-sale-securities. |
Income Taxes | Income Taxes The Company accounts for income taxes using the asset and liability method, which requires the recognition of deferred tax assets and liabilities for the expected future tax consequences of events that have been recognized in the consolidated financial statements or in the Company’s tax returns. Deferred tax assets and liabilities are determined on the basis of the differences between the financial statement and tax basis of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse. Changes in deferred tax assets and liabilities are recorded in the provision for income taxes. The Company assesses the likelihood that its deferred tax assets will be recovered from future taxable income and, to the extent it believes, based upon the weight of available evidence, that it is more likely than not that all or a portion of the deferred tax assets will not be realized, a valuation allowance is established through a charge to income tax expense. Potential for recovery of deferred tax assets is evaluated by estimating the future taxable profits expected and considering prudent and feasible tax planning strategies. The Company accounts for uncertainty in income taxes recognized in the consolidated financial statements by applying two-step more-likely-than-not |
Net Income (Loss) per Share | Net Income (Loss) per Share The Company follows the two-class two-class two-class Basic net income (loss) per share attributable to common stockholders is computed by dividing the net income (loss) attributable to common stockholders by the weighted average number of shares of common stock outstanding for the period. Diluted net income (loss) attributable to common stockholders is computed by adjusting net income (loss) attributable to common stockholders to reallocate undistributed earnings based on the potential impact of dilutive securities. Diluted net income (loss) per share attributable to common stockholders is computed by dividing the diluted net income (loss) attributable to common stockholders by the weighted average number of shares of common stock outstanding for the period, including potential dilutive shares of common stock. For purposes of this calculation, outstanding stock options and unvested restricted common stock are considered potential dilutive shares of common stock. In periods in which the Company reports a net loss attributable to common stockholders, diluted net loss per share attributable to common stockholders is the same as basic net loss per share attributable to common stockholders, since dilutive shares of common stock are not assumed to have been issued if their effect is anti-dilutive. The Company reported a net loss attributable to common stockholders for the years ended December 31, 2020 and 2019. |
Recently Adopted Accounting Pronouncement | Recently Adopted Accounting Pronouncements In January 2017, the FASB issued Accounting Standards Update (“ASU”) No. 2017-04, Intangibles—Goodwill and Other: Simplifying the Test for Goodwill Impairment (Topic 350) In August 2018, the FASB issued ASU No. 2018-13, Fair Value Measurement (Topic 820): Disclosure Framework—Changes to the Disclosure Requirements for Fair Value Measurement In November 2018, the FASB issued ASU No. 2018-18, Collaborative Arrangements (Topic 808): Clarifying the Interaction between Topic Revenue from Contracts with Customers Collaborative Arrangements |
Recently Issued Accounting Pronouncements | Recently Issued Accounting Pronouncements In June 2016, the FASB issued ASU 2016-13, Financial Instruments—Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments available-for-sale In December 2019, the FASB issued ASU No. 2019-12, Income Taxes-Simplifying the Accounting for Income Taxes step-up statements |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Accounting Policies [Abstract] | |
Property And Equipment Estimated Useful Life | The estimated useful lives of the Company’s property and equipment are as follows: Estimated Useful Life Laboratory equipment 5 years Computer equipment 3 years Office equipment 5 years Leasehold improvements Shorter of lease term or 10 years |
Collaboration Agreement (Tables
Collaboration Agreement (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Summary of Collaboration Revenue | The following table summarizes the Company’s collaboration revenue (in thousands): Year Ended December 31, 2020 2019 Collaboration revenue $ 138,811 $ 7,804 |
Balance of Contract Liabilities Related to Collaboration Agreements | The following table presents the balance of the Company’s contract liabilities (in thousands): December 31, December 31, Contract liabilities Deferred revenue $ 296,222 $ 43,356 |
Intangible Assets and Goodwill
Intangible Assets and Goodwill (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Goodwill and Intangible Assets Disclosure [Abstract] | |
Summary of Definite-Lived Intangible Assets Subject to Amortization and Indefinite-Lived Intangible Assets | December 31, 2020 Estimated Life Gross Carrying Amount Accumulated Amortization Impairment Charge Net Carrying Amount (In thousands) Definite-lived intangible assets: MRT 6 years $ 45,992 $ (9,156 ) $ — $ 36,836 Indefinite-lived intangible assets: IPR&D - CF Indefinite 42,291 — — 42,291 Total intangible assets, net $ 88,283 $ (9,156 ) $ — $ 79,127 December 31, 2019 Estimated Life Gross Carrying Amount Accumulated Amortization Impairment Charge Net Carrying Amount (In thousands) Definite-lived intangible assets: MRT 8 years $ 45,992 $ (2,747 ) $ — $ 43,245 Indefinite-lived intangible assets: IPR&D - CF Indefinite 42,291 — — 42,291 IPR&D - OTC Indefinite 18,559 — (18,559 ) — Total intangible assets, net $ 106,842 $ (2,747 ) $ (18,559 ) $ 85,536 |
Fair Value of Financial Asset_2
Fair Value of Financial Assets and Liabilities (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Fair Value Disclosures [Abstract] | |
Schedule of Financial Assets and Liabilities Measured at Fair Value on Recurring Basis | The following tables present information Fair Value Measurements as of December 31, 2020 Using: Level 1 Level 2 Level 3 Total Assets: Money market funds $ — $ 273,827 $ — $ 273,827 U.S. treasuries — 292,001 — 292,001 U.S. government agency bonds — 20,000 — 20,000 $ — $ 585,828 $ — $ 585,828 Liabilities: Contingent consideration $ — $ — $ 152,230 $ 152,230 $ — $ — $ 152,230 $ 152,230 Fair Value Measurements as of December 31, 2019 Using: Level 1 Level 2 Level 3 Total Assets: Money market funds $ — $ 56,591 $ — $ 56,591 U.S. government agency bonds — 104,098 — 104,098 $ — $ 160,689 $ — $ 160,689 Liabilities: Contingent consideration $ — $ — $ 103,655 $ 103,655 $ — $ — $ 103,655 $ 103,655 |
Schedule of Unobservable Inputs and Fair Value Components of Contingent Consideration | The following table presents the unobservable inputs and fair value of the components of the contingent consideration (dollar amounts in thousands): Unobservable Inputs Fair Value at Projected Year of Payment December 31, December 31, Earnout payments 2026 - 2039 142,250 $ 96,097 Milestone payments 2026 - 2032 9,980 7,558 $ 152,230 $ 103,655 |
Summary Of Estimated Amortized Costs And Fair Value Of The Debt Securities Available For Sale And Held To Maturity | The estimated amortized costs and fair value of the Company’s available-for-sale December 31, 2020 December 31, 2019 Amortized Cost Fair Value Amortized Cost Fair Value Due within one year $ 201,606 $ 201,596 $ 103,357 $ 104,098 Due after one year through two years 110,395 110,405 — — Total available-for-sale $ 312,001 $ 312,001 $ 103,357 $ 104,098 |
Schedule of Total Acquisition Related Contingent Consideration Liability | The following table presents a roll-forward of the total acquisition-related contingent consideration liability (in thousands): Fair Value Balance as of December 31, 2019 $ 103,655 Increase in fair value of contingent consideration 48,575 Balance as of December 31, 2020 $ 152,230 |
Property and Equipment, Net (Ta
Property and Equipment, Net (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Property, Plant and Equipment [Abstract] | |
Schedule of Property and Equipment, Net | Property and equipment, net consisted of the following (in thousands): December 31, December 31, 2020 2019 Laboratory equipment $ 12,710 $ 9,044 Computer equipment 922 779 Office equipment 941 883 Leasehold improvements 5,730 5,635 Construction in progress 5,189 3,460 25,492 19,801 Less: Accumulated depreciation and amortization (10,120 ) (7,262 ) $ 15,372 $ 12,539 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Expenses | Accrued expenses consisted of the following (in thousands): December 31, December 31, 2020 2019 Accrued employee compensation and benefits $ 5,600 $ 3,547 Accrued external research and development expenses 2,805 1,763 Accrued consultant and professional fees 1,489 1,390 Other 3,308 372 $ 13,202 $ 7,072 |
Incentive Stock Options and R_2
Incentive Stock Options and Restricted Stock (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Summary of Stock Option Activity | The following table summarizes the Company’s stock option activity since December 31, 2019 (in thousands, except share and per share amounts): Number of Shares Weighted Average Exercise Price Weighted Average Remaining Contractual Term Intrinsic Value (in years) Outstanding as of December 31, 2019 8,646,378 $ 8.06 8.42 $ 3,687 Granted 3,313,584 $ 10.26 Exercised (1,551,178 ) $ 7.99 Forfeited (851,393 ) $ 8.57 Outstanding as of December 31, 2020 9,557,391 $ 8.79 7.92 $ 93,256 Exercisable as of December 31, 2020 4,783,112 $ 7.86 7.11 $ 50,567 Vested and expected to vest as of December 31, 2020 9,557,391 $ 8.79 7.92 $ 93,256 |
Summary of Assumptions Used in Black-Scholes Option-Pricing Model to Determine Grant-Date Fair Value of Stock Option Granted | The following table presents, on a weighted average basis, the assumptions used in the Black-Scholes option-pricing model to determine the grant-date fair value of stock options granted to employees and directors: Year Ended December 31, 2020 2019 Risk-free interest rate 0.75 % 2.36 % Expected term (in years) 6.1 6.0 Expected volatility 68.8 % 73.0 % Expected dividend yield 0 % 0 % |
Summary of Restricted Stock Activity | Number of Shares Weighted Average Grant-Date Fair Value Unvested restricted common stock outstanding as of December 31, 2019 34,168 $ 1.28 Forfeited restricted common stock — $ — Vested restricted common stock (34,168 ) $ 1.28 Unvested restricted common stock outstanding as of December 31, 2020 — $ — |
Summary of Stock-Based Compensation Expense | Stock-based compensation expense was classified in the consolidated statements of operations as follows (in thousands): Year Ended December 31, 2020 2019 Research and development expenses $ 8,818 $ 4,786 General and administrative expenses 8,802 6,548 $ 17,620 $ 11,334 |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Income Tax Disclosure [Abstract] | |
Schedule of Reconciliation of U.S. Federal Statutory Income Tax Rate and Effective Income Tax Rate | A reconciliation of the U.S. federal statutory income tax rate to the Company’s effective income tax rate is as follows: Year Ended December 31, 2020 2019 U.S. federal statutory income tax rate (21.0 )% (21.0 )% State income taxes, net of federal benefit (9.4 ) (6.6 ) Research and development tax credits and orphan drug credit (11.6 ) (6.8 ) Stock-based compensation (3.5 ) 0.5 Other permanent differences 0.0 0.2 Change in deferred tax asset valuation allowance 37.0 33.6 Officers compensation 3.5 0.0 NOL and tax credit expiration under 382 4.8 0.0 Other 0.2 (0.3 ) Effective income tax rate 0.0 % (0.4 )% |
Schedule of Components of Deferred Tax Assets and Liabilities | Components of the Company’s deferred tax assets were as follows (in thousands): December 31, 2020 2019 Deferred tax assets: Net operating loss carryforwards $ 64,639 $ 61,460 Research and development tax credit and orphan drug credit carryforwards 28,406 21,830 Intangibles 7,570 3,607 Deferred revenue 3,753 11,845 Depreciation and amortization 17,305 2,253 Accrued expenses and other 4,583 5,354 Total deferred tax assets 126,256 106,349 Valuation allowance (126,256 ) (106,346 ) Net deferred tax assets $ — $ 3 |
Summary of Changes in Valuation Allowance for Deferred Tax Assets | Changes in the valuation allowance for deferred tax assets during the years ended December 31, 2020 and 2019 related primarily to the increase in net operating loss carryforwards and research and development tax credit carryforwards in 2020 and 2019, and were as follows (in thousands): Year Ended December 31, 2020 2019 Valuation allowance at beginning of year $ (106,346 ) $ (68,143 ) Increases recorded to income tax provision (19,910 ) (38,203 ) Valuation allowance at end of year $ (126,256 ) $ (106,346 ) |
Net Loss per Share (Tables)
Net Loss per Share (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Earnings Per Share [Abstract] | |
Summary of Basic and Diluted Net Loss Per Share Attributable to Common Stockholders | Basic and diluted net loss per share attributable to common stockholders was calculated as follows (in thousands, except share and per share amounts): Year Ended December 31, 2020 2019 Numerator: Net loss $ (53,787 ) $ (113,293 ) Denominator: Weighted average common shares outstanding—basic and diluted 67,520,886 51,445,539 Net loss per share—basic and diluted $ (0.80 ) $ (2.20 ) |
Schedule of Potential Securities Excluded from Computation of Earnings Per Share | The Company excluded the following potential shares of common stock, 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 because including them would have had an anti-dilutive effect: Year Ended December 31, 2020 2019 Options to purchase common stock 9,557,391 8,646,378 Unvested restricted common stock — 34,168 9,557,391 8,680,546 |
Leases (Tables)
Leases (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Leases [Abstract] | |
Schedule of Lease Cost | The components of lease cost were as follows (dollar amounts in thousands): Year Ended 2020 2019 Lease cost Operating lease cost $ 8,099 $ 2,692 Total lease cost $ 8,099 $ 2,692 Other information Operating cash flows from operating leases $ 7,775 $ 2,583 Operating lease liabilities arising from obtaining right-of-use $ 54,063 — Weighted-average remaining lease term 5 years 8 years Weighted-average discount rate 11.9 % 17.5 % |
Schedule of maturities of operating lease liabilities | As of December 31, 2020, maturities of operating lease liabilities are as follows (in thousands): December 31, 2020 2021 $ 18,067 2022 15,178 2023 15,591 2024 16,050 2025 12,029 2026 and thereafter 7,134 Total future minimum lease payments 84,049 Less: imputed interest (21,363 ) Present value of lease liabilities $ 62,686 As of December 31, 2019, maturities of operating lease liabilities are as follows (in thousands): December 31, 2019 2020 $ 2,659 2021 2,737 2022 2,818 2023 2,860 2024 2,937 2025 and thereafter 10,160 Total future minimum lease payments 24,171 Less: imputed interest (11,557 ) Present value of lease liabilities $ 12,614 |
Selected Quarterly Financial _2
Selected Quarterly Financial Information (Unaudited) (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Selected Quarterly Financial Information [Abstract] | |
Schedule of Selected Quarterly Financial Information | The following table contains quarterly financial information for 2020 and 2019 (in thousands, except per share amounts). The Company believes that the following information reflects all normal recurring adjustments necessary for a fair statement of the information for the periods presented. The operating results for any quarter are not necessarily indicative of results for any future period. 2020 First Quarter Second Quarter Third Quarter Fourth Quarter Total Collaboration revenue $ 4,654 $ 16,319 $ 66,446 $ 51,392 $ 138,811 Total operating expenses 19,445 52,950 49,697 72,034 194,126 Income (loss) from operations (14,791 ) (36,631 ) 16,749 (20,642 ) (55,315 ) Net income (loss) (14,282 ) (36,288 ) 17,344 (20,561 ) (53,787 ) Net income (loss) per share applicable to common $ (0.24 ) $ (0.58 ) $ 0.24 $ (0.22 ) $ (0.80 ) Net income (loss) per share applicable to common stockholders—diluted $ (0.24 ) $ (0.58 ) $ 0.23 $ (0.22 ) $ (0.80 ) 2019 First Quarter Second Quarter Third Quarter Fourth Quarter Total Collaboration revenue $ 1,474 $ 1,174 $ 1,266 $ 3,890 $ 7,804 Total operating expenses 35,679 29,364 22,901 35,629 123,573 Loss from operations (34,205 ) (28,190 ) (21,635 ) (31,739 ) (115,769 ) Net loss (33,198 ) (27,832 ) (21,227 ) (31,036 ) (113,293 ) Net loss per share applicable to common stockholders—basic and diluted $ (0.74 ) $ (0.57 ) $ (0.41 ) $ (0.48 ) $ (2.20 ) |
Nature of the Business and Ba_2
Nature of the Business and Basis of Presentation - Additional Information (Detail) $ / shares in Units, $ in Thousands | Jul. 20, 2020USD ($)$ / sharesshares | Jun. 30, 2020USD ($)$ / sharesshares | Dec. 31, 2020USD ($)Disease | Sep. 30, 2020USD ($) | Jun. 30, 2020USD ($)$ / shares | Mar. 31, 2020USD ($) | Dec. 31, 2019USD ($) | Sep. 30, 2019USD ($) | Jun. 30, 2019USD ($) | Mar. 31, 2019USD ($) | Dec. 31, 2020USD ($)DiseaseSubsidiaryshares | Dec. 31, 2019USD ($) | Mar. 26, 2020Disease | Mar. 13, 2020USD ($) |
Initial Public Offering [Line Items] | ||||||||||||||
Number of wholly owned subsidiaries | Subsidiary | 2 | |||||||||||||
Accumulated deficit | $ (413,283) | $ (359,496) | $ (413,283) | $ (359,496) | ||||||||||
Net loss | (20,561) | $ 17,344 | $ (36,288) | $ (14,282) | $ (31,036) | $ (21,227) | $ (27,832) | $ (33,198) | (53,787) | (113,293) | ||||
Stock available to be sold under sales agreement | 62,100 | 62,100 | ||||||||||||
Cash, cash equivalents and short-term investments | $ 654,000 | 654,000 | ||||||||||||
Gross proceeds from issuance of public offering | $ 154,292 | $ 84,600 | ||||||||||||
Sale from time to time of equity and debt securities | $ 350,000 | |||||||||||||
Sanofi Pasteur Inc [Member] | ||||||||||||||
Initial Public Offering [Line Items] | ||||||||||||||
Maximum number of infectious disease pathogens for vaccine development | 10 | 10 | ||||||||||||
Securities Purchase Agreement [Member] | ||||||||||||||
Initial Public Offering [Line Items] | ||||||||||||||
Stock Issued During Period, Value, New Issues | $ 125,000 | |||||||||||||
Shares Issued, Price Per Share | $ / shares | $ 25.59 | |||||||||||||
Shares issued through public offering | shares | 4,884,434 | |||||||||||||
Second Sanofi Amendment Agreement [Member] | ||||||||||||||
Initial Public Offering [Line Items] | ||||||||||||||
Additional Upfront Payment | $ 300,000 | |||||||||||||
Sanofi Pasteur Collaboration and Licensing Agreement [Member] | Maximum [Member] | ||||||||||||||
Initial Public Offering [Line Items] | ||||||||||||||
Number of infectious disease pathogens for vaccine development | Disease | 7 | 7 | ||||||||||||
Sanofi Pasteur Collaboration and Licensing Agreement [Member] | Minimum [Member] | ||||||||||||||
Initial Public Offering [Line Items] | ||||||||||||||
Number of infectious disease pathogens for vaccine development | Disease | 3 | 3 | ||||||||||||
Sanofi Pasteur Collaboration and Licensing Agreement [Member] | Sanofi Pasteur Inc [Member] | ||||||||||||||
Initial Public Offering [Line Items] | ||||||||||||||
Number of infectious disease pathogens | Disease | 6 | |||||||||||||
Open Market Sale Agreement [Member] | ||||||||||||||
Initial Public Offering [Line Items] | ||||||||||||||
Payments of other offering expenses | $ 200 | |||||||||||||
Payment of issuance commissions | 1,100 | |||||||||||||
Gross proceeds from issuance of public offering | 37,900 | |||||||||||||
Market sale aggreement of common stock | $ 100,000 | $ 100,000 | ||||||||||||
Issuance of common, shares | shares | 2,863,163 | |||||||||||||
Public Offering [Member] | ||||||||||||||
Initial Public Offering [Line Items] | ||||||||||||||
Payments of other offering expenses | $ 500 | |||||||||||||
Gross proceeds from issuance of public offering | 125,000 | |||||||||||||
Underwriting discounts and commissions | $ 7,500 | |||||||||||||
Issuance of common, shares | shares | 5,681,819 | |||||||||||||
Common stock issued and sold, per share | $ / shares | $ 22 | $ 22 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Additional Information (Detail) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Indefinite-lived Intangible Assets [Line Items] | ||
Current portion of deferred revenue | $ 67,563 | $ 18,100 |
Deferred revenue, net of current portion | 228,659 | $ 25,256 |
ASU 2016-02 [Member] | ||
Indefinite-lived Intangible Assets [Line Items] | ||
Current portion of deferred revenue | 67,600 | |
Deferred revenue, net of current portion | $ 228,700 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Estimated Useful Lives of Property and Equipment (Detail) | 12 Months Ended |
Dec. 31, 2020 | |
Laboratory Equipment [Member] | |
Property Plant And Equipment Estimated Useful Lives [Line Items] | |
Property plant and equipment estimated useful life | 5 years |
Computer Equipment [Member] | |
Property Plant And Equipment Estimated Useful Lives [Line Items] | |
Property plant and equipment estimated useful life | 3 years |
Office Equipment [Member] | |
Property Plant And Equipment Estimated Useful Lives [Line Items] | |
Property plant and equipment estimated useful life | 5 years |
Leasehold Improvements [Member] | |
Property Plant And Equipment Estimated Useful Lives [Line Items] | |
Property plant and equipment estimated useful life | Shorter of lease term or 10 years |
Collaboration Agreement - Addit
Collaboration Agreement - Additional Information (Detail) $ / shares in Units, $ in Millions | Jul. 20, 2020USD ($)$ / sharesshares | Dec. 31, 2020USD ($)Disease | Dec. 31, 2019USD ($) |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Revenue recognized as a cumulative catch-up adjustment | $ 37.3 | ||
Sanofi Collaboration And License Agreement [Member] | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Maximum number of infectious disease pathogens for vaccine development | 10 | ||
Sanofi Collaboration And License Agreement [Member] | Maximum [Member] | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Number of infectious disease pathogens for vaccine development | Disease | 7 | ||
Sanofi Collaboration And License Agreement [Member] | Minimum [Member] | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Number of infectious disease pathogens for vaccine development | Disease | 3 | ||
Second Sanofi Amendment Agreement [Member] | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Additional Upfront Payment | $ 300 | ||
Securities Purchase Agreement [Member] | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Shares issued through public offering | shares | 4,884,434 | ||
Shares Issued, Price Per Share | $ / shares | $ 25.59 | ||
Shares issued during the period value | $ 125 | ||
Original Sanofi Agreement And Second Sanofi Amendment Agreement [Member] | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Increase in transaction price | $ 592.4 | ||
Non-refundable upfront payment | 345 | ||
Sanofi Agreement [Member] | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Remaining performance obligation, expected to be recognized as revenue | $ 595.6 | ||
Number of infectious disease pathogens for vaccine development | Disease | 5 | ||
Upfront payment received | $ 45 | ||
Technology and process transfer milestone payment receivable | 10 | ||
Estimated reimbursable employee cost | 75.2 | ||
Estimated reimbursable development cost | 160.2 | ||
Estimated milestone payments | 112 | ||
Revenue recognized from contract liabilities | $ 6 | $ 1.1 | |
Sanofi Agreement [Member] | Maximum [Member] | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Number of infectious disease pathogens for vaccine development | Disease | 6 | ||
Sublicense Second Amendment Agreement With Sanofi [Member] | Massachusetts Institute Of Technology Exclusive Patent License Agreement [Member] | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Additional Upfront Payment Receivable | $ 75 | ||
Milestone payment receivable upon the achievement of additional specified regulatory development manufacturing and commercial milestones | 1,900 | ||
Lumpsum or one time manufacturing milestone amount receivable | 200 | ||
Maximum development and regulatory milestone payment receivable | 148 | ||
Sublicense Second Amendment Agreement With Sanofi [Member] | Sars Cov2 Licensed Filed [Member] | Massachusetts Institute Of Technology Exclusive Patent License Agreement [Member] | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Maximum development and regulatory milestone payment receivable | 250 | ||
Additional Paid-in Capital [Member] | Securities Purchase Agreement [Member] | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Shares issued during the period value | $ 51.2 |
Collaboration Agreement - Summa
Collaboration Agreement - Summary of Collaboration Revenue (Detail) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | ||||||||
Dec. 31, 2020 | Sep. 30, 2020 | Jun. 30, 2020 | Mar. 31, 2020 | Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2020 | Dec. 31, 2019 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | ||||||||||
Collaboration revenue | $ 51,392 | $ 66,446 | $ 16,319 | $ 4,654 | $ 3,890 | $ 1,266 | $ 1,174 | $ 1,474 | $ 138,811 | $ 7,804 |
Collaboration Agreement - Balan
Collaboration Agreement - Balance of Contract Liabilities Related to Collaboration Agreements (Detail) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Contract liabilities | ||
Deferred revenue | $ 296,222 | $ 43,356 |
Intangible Assets and Goodwil_2
Intangible Assets and Goodwill - Summary of Definite-Lived Intangible Assets Subject to Amortization and Indefinite-Lived Intangible Assets (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Finite Lived Intangible Assets [Line Items] | ||
Definite-lived intangible assets, accumulated amortization | $ (9,156) | $ (2,747) |
Definite-lived intangible assets, Impairment Charge | 0 | (18,559) |
Total intangible assets, gross carrying amount | 88,283 | 106,842 |
Total intangible assets, net carrying amount | $ 79,127 | $ 85,536 |
In-Process Research and Development [Member] | MRT Product [Member] | Shire's MRT Program [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Definite-lived intangible assets, estimated life | 6 years | 8 years |
Definite-lived intangible assets, gross carrying amount | $ 45,992 | $ 45,992 |
Definite-lived intangible assets, accumulated amortization | (9,156) | (2,747) |
Definite-lived intangible assets, Impairment Charge | 0 | |
Definite-lived intangible assets, net carrying amount | 36,836 | 43,245 |
In-Process Research and Development [Member] | Cystic Fibrosis [Member] | Shire's MRT Program [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Definite-lived intangible assets, accumulated amortization | 0 | |
Definite-lived intangible assets, Impairment Charge | 0 | |
Indefinite-lived intangible assets, gross carrying amount/net carrying amount | $ 42,291 | 42,291 |
In-Process Research and Development [Member] | OTC Deficiency [Member] | Shire's MRT Program [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Definite-lived intangible assets, Impairment Charge | (18,600) | |
Indefinite-lived intangible assets, gross carrying amount/net carrying amount | $ 18,559 |
Intangible Assets and Goodwil_3
Intangible Assets and Goodwill - Additional Information (Detail) - USD ($) | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Finite Lived Intangible Assets [Line Items] | ||
indefinite lived intangible assets Impairment charge | $ 0 | $ 18,559,000 |
Goodwill, Impairment Charges | 0 | 0 |
Goodwill | 21,359,000 | 21,359,000 |
Goodwill changes | 0 | |
Shire's MRT Program [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Goodwill | 21,400,000 | |
Shire's MRT Program [Member] | Sanofi Agreement [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Estimated amortization expense of intangible assets for 2021 | 10,800,000 | |
Estimated amortization expense of intangible assets for 2022 | 11,700,000 | |
Estimated amortization expense of intangible assets for 2023 | 11,600,000 | |
Estimated amortization expense of intangible assets for 2024 | 2,800,000 | |
OTC Deficiency Program [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Amortization of Intangible Assets | 6,400,000 | 2,300,000 |
In Process Research and Development [Member] | OTC Deficiency [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
Impairment of Intangible Assets, Finite-lived | $ 0 | |
In Process Research and Development [Member] | OTC Deficiency [Member] | Shire's MRT Program [Member] | ||
Finite Lived Intangible Assets [Line Items] | ||
indefinite lived intangible assets Impairment charge | $ 18,600,000 |
Fair Value of Financial Asset_3
Fair Value of Financial Assets and Liabilities - Schedule of Financial Assets and Liabilities Measured at Fair Value on Recurring Basis (Detail) - Fair Value, Measurements, Recurring - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Assets: | ||
Total, assets | $ 585,828 | $ 160,689 |
Liabilities: | ||
Total, liabilities | 152,230 | 103,655 |
Fair Value, Inputs, Level 1 | ||
Assets: | ||
Total, assets | 0 | |
Liabilities: | ||
Total, liabilities | 0 | |
Fair Value, Inputs, Level 2 | ||
Assets: | ||
Total, assets | 585,828 | 160,689 |
Liabilities: | ||
Total, liabilities | 0 | |
Fair Value, Inputs, Level 3 | ||
Assets: | ||
Total, assets | 0 | |
Liabilities: | ||
Total, liabilities | 152,230 | 103,655 |
Money Market Funds | ||
Assets: | ||
Total, assets | 273,827 | 56,591 |
Money Market Funds | Fair Value, Inputs, Level 1 | ||
Assets: | ||
Total, assets | 0 | |
Money Market Funds | Fair Value, Inputs, Level 2 | ||
Assets: | ||
Total, assets | 273,827 | 56,591 |
Money Market Funds | Fair Value, Inputs, Level 3 | ||
Assets: | ||
Total, assets | 0 | |
U.S. treasuries | ||
Assets: | ||
Total, assets | 292,001 | |
U.S. treasuries | Fair Value, Inputs, Level 1 | ||
Assets: | ||
Total, assets | 0 | |
U.S. treasuries | Fair Value, Inputs, Level 2 | ||
Assets: | ||
Total, assets | 292,001 | |
U.S. treasuries | Fair Value, Inputs, Level 3 | ||
Assets: | ||
Total, assets | 0 | |
U.S. Government Agency Bonds | ||
Assets: | ||
Total, assets | 20,000 | 104,098 |
U.S. Government Agency Bonds | Fair Value, Inputs, Level 1 | ||
Assets: | ||
Total, assets | 0 | |
U.S. Government Agency Bonds | Fair Value, Inputs, Level 2 | ||
Assets: | ||
Total, assets | 20,000 | 104,098 |
U.S. Government Agency Bonds | Fair Value, Inputs, Level 3 | ||
Assets: | ||
Total, assets | 0 | |
Contingent Consideration | ||
Liabilities: | ||
Total, liabilities | 152,230 | 103,655 |
Contingent Consideration | Fair Value, Inputs, Level 1 | ||
Liabilities: | ||
Total, liabilities | 0 | |
Contingent Consideration | Fair Value, Inputs, Level 2 | ||
Liabilities: | ||
Total, liabilities | 0 | |
Contingent Consideration | Fair Value, Inputs, Level 3 | ||
Liabilities: | ||
Total, liabilities | $ 152,230 | $ 103,655 |
Fair Value of Financial Asset_4
Fair Value of Financial Assets and Liabilities - Schedule of Unobservable Inputs and Fair Value Components of Contingent Consideration (Detail) - Contingent Consideration - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Fair Value at | $ 152,230 | $ 103,655 |
Earnout Payments | ||
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Fair Value at | $ 142,250 | $ 96,097 |
Earnout Payments | Minimum [Member] | ||
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Projected Year of Payment | 2026 | 2026 |
Earnout Payments | Maximum [Member] | ||
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Projected Year of Payment | 2039 | 2039 |
Milestone Payments | ||
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Fair Value at | $ 9,980 | $ 7,558 |
Milestone Payments | Minimum [Member] | ||
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Projected Year of Payment | 2026 | 2026 |
Milestone Payments | Maximum [Member] | ||
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Projected Year of Payment | 2032 | 2032 |
Fair Value of Financial Asset_5
Fair Value of Financial Assets and Liabilities - Schedule of Total Acquisition Related Contingent Consideration Liability (Detail) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |
Mar. 31, 2020 | Dec. 31, 2020 | Dec. 31, 2019 | |
Business Acquisition, Contingent Consideration [Line Items] | |||
Beginning Balance | $ 103,655 | $ 103,655 | |
Discontinuation of MRT5201 and Increase in fair value of contingent consideration | $ 9,500 | 48,575 | $ 13 |
Ending Balance | 152,230 | $ 103,655 | |
Contingent Consideration | |||
Business Acquisition, Contingent Consideration [Line Items] | |||
Discontinuation of MRT5201 and Increase in fair value of contingent consideration | $ 48,575 |
Fair Value of Financial Asset_6
Fair Value of Financial Assets and Liabilities - Summary Of Estimated Amortized Costs And Fair Value Of The Debt Securities Available For Sale And Held To Maturity (Detail) - USD ($) $ in Millions | Dec. 31, 2020 | Dec. 31, 2019 |
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | $ 312,001 | $ 103,357 |
Fair Value | 312,001 | 104,098 |
Due Within One Year [Member] | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 201,606 | 103,357 |
Fair Value | 201,596 | $ 104,098 |
Due After One Year through Two Years [Member] | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 110,395 | |
Fair Value | $ 110,405 |
Fair Value of Financial Asset_7
Fair Value of Financial Assets and Liabilities - Additional Information (Detail) | 12 Months Ended | |
Dec. 31, 2020USD ($) | Dec. 31, 2019USD ($) | |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | $ 342,027,000 | $ 84,580,000 |
Contingent Consideration | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Discount Rate | 11 | 13.5 |
Money Market Funds | Fair Value, Inputs, Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash and cash equivalents | $ 273,800,000 | $ 56,600,000 |
Fair Value, Measurements, Recurring | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Fair Value, assets transfers into (out of) Level 3 | 0 | 0 |
Fair value,assets transfers from Level 2 to Level 1 | 0 | 0 |
Fair value,assets transfers from Level 1 to Level 2 | 0 | 0 |
Fair value, liabilities transfers from Level 1 to Level 2 | 0 | 0 |
Fair value, liabilities transfers from Level 2 to Level 1 | 0 | 0 |
Fair Value, liabilities transfers into (out of) Level 3 | $ 0 | $ 0 |
Property and Equipment, Net - S
Property and Equipment, Net - Schedule of Property and Equipment, Net (Detail) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | $ 25,492 | $ 19,801 |
Less: Accumulated depreciation and amortization | (10,120) | (7,262) |
Property and equipment, net | 15,372 | 12,539 |
Laboratory Equipment [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | 12,710 | 9,044 |
Computer Equipment [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | 922 | 779 |
Office Equipment [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | 941 | 883 |
Leasehold Improvements [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | 5,730 | 5,635 |
Construction In Progress [Member] | ||
Property Plant And Equipment [Line Items] | ||
Property and equipment, gross | $ 5,189 | $ 3,460 |
Property and Equipment, Net - A
Property and Equipment, Net - Additional Information (Detail) - USD ($) $ in Millions | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Property, Plant and Equipment [Abstract] | ||
Depreciation and amortization expense | $ 2.9 | $ 2.4 |
Accrued Expenses - Schedule of
Accrued Expenses - Schedule of Accrued Expenses (Detail) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Payables and Accruals [Abstract] | ||
Accrued employee compensation and benefits | $ 5,600 | $ 3,547 |
Accrued external research and development expenses | 2,805 | 1,763 |
Accrued consultant and professional fees | 1,489 | 1,390 |
Other | 3,308 | 372 |
Total accrued expenses | $ 13,202 | $ 7,072 |
Common Stock - Additional Infor
Common Stock - Additional Information (Detail) - USD ($) | 12 Months Ended | ||
Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Class of Stock [Line Items] | |||
Common stock, shares authorized | 200,000,000 | 200,000,000 | |
Common stock, par value | $ 0.001 | $ 0.001 | |
Common stock voting rights | Each share of common stock entitles the holder to one vote for each share of common stock held. | ||
Cash dividend paid shares | $ 0 | $ 0 | |
Common stock, shares issued | 75,029,625 | 60,022,067 | |
Common stock, shares outstanding | 75,029,625 | 60,022,067 | 60,022,067 |
Incentive Stock Options and R_3
Incentive Stock Options and Restricted Stock - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | Jun. 15, 2018 | Mar. 07, 2018 | Dec. 31, 2020 | Dec. 31, 2019 |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Stock-based compensation expense | $ 847 | |||
Common stock, shares issued | 75,029,625 | 60,022,067 | ||
Unrecognized compensation cost related to unvested stock-based awards | $ 25,500 | |||
Unrecognized compensation cost, period for recognition | 2 years 6 months | |||
Stock Options [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Vested Stock options for purchase of common stock | 9,557,391 | |||
Intrinsic value of stock options, exercised | $ 19,200 | $ 600 | ||
Weighted average grant-date fair value | $ 6.31 | $ 5.59 | ||
2018 Stock Incentive Plan [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Number of common shares reserved for issuance | 7,457,171 | |||
Number of shares remaining available for issuance | 360,514 | |||
Increase in common stock issued | 2,400,829 | |||
Percentage threshold of outstanding shares under the plan | 4.00% | |||
Percentage of exercise price per share of fair market value | 100.00% | |||
Expiration period of stock options after grant date | 10 years | |||
2018 Stock Incentive Plan [Member] | Minimum [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Stock options for purchase of common stock held, exercisable period | 1 year | |||
Number of stock issued during period under the plan | 3,349,582 | |||
2018 Stock Incentive Plan [Member] | Maximum [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Stock options for purchase of common stock held, exercisable period | 4 years | |||
2018 Stock Incentive Plan [Member] | Share-based Compensation Award, Tranche One [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Number of common shares reserved for issuance | 2,512,187 | |||
2018 Stock Incentive Plan [Member] | Share-based Compensation Award, Tranche Two [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Number of common shares reserved for issuance | 1,013,167 | |||
2018 Employee Stock Purchase Plan [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Number of common shares reserved for issuance | 418,697 | 870,096 | ||
Percentage threshold of outstanding shares under the plan | 1.00% | |||
Common stock, shares issued | 26,964 | |||
2018 Employee Stock Purchase Plan [Member] | Minimum [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Number of stock issued during period under the plan | 837,395 | |||
2016 Stock Incentive Plan [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Sharebased Compensation Arrangement by Sharebased Payment Award Shares Cancelled in Period | 226,495 | |||
Stock options for purchase of common stock held, exercisable period | 4 years | |||
Percentage of exercise price per share of fair market value | 100.00% | |||
Expiration period of stock options after grant date | 10 years | |||
Research and Development Expense [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Stock-based compensation expense | $ 2,400 | |||
Former Employee [Member] | General and Administrative Expense [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Stock-based compensation expense | $ 1,000 | |||
EVP and Founder [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Vested Stock options for purchase of common stock | 176,266 | |||
Stock options for purchase of common stock held | 550,278 | |||
Stock options for purchase of common stock held, exercisable period | 18 months | |||
Chief Financial Officer [Member] | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Vested Stock options for purchase of common stock | 71,374 | |||
Stock options for purchase of common stock held | 145,362 |
Incentive Stock Options and R_4
Incentive Stock Options and Restricted Stock - Summary of Stock Option Activity (Detail) - Stock Options [Member] - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Number of shares | ||
Number of shares, beginning balance | 8,646,378 | |
Number of shares, granted | 3,313,584 | |
Number of shares, exercised | (1,551,178) | |
Number of shares, forfeited | (851,393) | |
Number of shares, ending balance | 9,557,391 | |
Number of shares, exercisable | 4,783,112 | |
Number of shares, vested and expected to vest, ending balance | 9,557,391 | |
Weighted average exercise price | ||
Weighted average exercise price, beginning balance | $ 8.06 | |
Weighted average exercise price, granted | 10.26 | |
Weighted average exercise price, exercised | 7.99 | |
Weighted average exercise price, forfeited | $ 8.57 | |
Weighted average exercise price, ending balance | $ 8.79 | |
Weighted average exercise price, exercisable | 7.86 | |
Weighted average exercise price, vested and expected to vest, ending balance | $ 8.79 | |
Weighted average remaining contractual term | ||
Weighted average remaining contractual term, ending balance | 7 years 11 months 1 day | 8 years 5 months 1 day |
Weighted average remaining contractual term, exercisable | 7 years 1 month 9 days | |
Weighted average remaining contractual term, vested and expected to vest, ending balance | 7 years 11 months 1 day | |
Intrinsic value | ||
Intrinsic value, ending balance | $ 93,256 | $ 3,687 |
Intrinsic value, exercisable | 50,567 | |
Intrinsic value, vested and expected to vest, ending balance | $ 93,256 |
Incentive Stock Option and Rest
Incentive Stock Option and Restricted Stock - Summary of Assumptions Used in Black-Scholes Option-Pricing Model to Determine Grant-Date Fair Value of Stock Option Granted (Detail) - Employees And Directors [Member] - Stock Options [Member] | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Risk-free interest rate | 0.75% | 2.36% |
Expected term (in years) | 6 years 1 month 6 days | 6 years |
Expected volatility | 68.80% | 73.00% |
Expected dividend yield | 0.00% | 0.00% |
Incentive Stock Option and Re_2
Incentive Stock Option and Restricted Stock - Summary of Restricted Stock Activity (Detail) - Restricted Common Stock [Member] | 12 Months Ended |
Dec. 31, 2020$ / sharesshares | |
Number of shares | |
Number of shares, beginning balance | shares | 34,168 |
Number of shares, forfeited | shares | 0 |
Number of shares, vested | shares | (34,168) |
Number of shares, ending balance | shares | 0 |
Weighted average grant-date fair value | |
Weighted average grant-date fair value, beginning balance | $ / shares | $ 1.28 |
Weighted average grant-date fair value, forfeited | $ / shares | 0 |
Weighted average grant-date fair value, vested | $ / shares | 1.28 |
Weighted average grant-date fair value, ending balance | $ / shares | $ 0 |
Incentive Stock Option and Re_3
Incentive Stock Option and Restricted Stock - Summary of Stock-Based Compensation Expense (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Stock-based compensation expense | $ 17,620 | $ 11,334 |
Research and Development Expenses [Member] | ||
Stock-based compensation expense | 8,818 | 4,786 |
General and Administrative Expenses [Member] | ||
Stock-based compensation expense | $ 8,802 | $ 6,548 |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Detail) - USD ($) | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Operating Loss Carryforwards [Line Items] | ||
Income tax benefits | $ (486,000) | |
Orphan drug tax credit carryforwards | $ 17,400,000 | |
Orphan drug tax credit carryforwards expiration year | 2037 | |
Increase in ownership of certain stockholders or public groups in the stock as result of transactions, specified period | 3 years | |
Unrecognized tax benefits | $ 0 | 0 |
Income tax examination, description | The federal and state returns are generally subject to tax examinations for the tax years ended December 31, 2017 to the present. | |
Tax cuts and jobs act of 2017, net operating losses carryforward, maximum percentage of taxable income | 80.00% | |
Federal [Member] | ||
Operating Loss Carryforwards [Line Items] | ||
Operating loss carryforwards | $ 239,300,000 | |
Operating loss carryforwards subject to expiration | $ 116,100,000 | |
Operating loss carryforwards, expiration date | 2031 | |
Research and development tax credit carryforward, amount | $ 7,000,000 | |
Research and development tax credit carryforward expiration year | 2032 | |
State [Member] | ||
Operating Loss Carryforwards [Line Items] | ||
Operating loss carryforwards | $ 227,600,000 | |
Operating loss carryforwards, expiration date | 2031 | |
Research and development tax credit carryforward, amount | $ 4,300,000 | |
Research and development tax credit carryforward expiration year | 2028 | |
Investment tax credit carryforwards amount | $ 800,000 | |
Investment tax credit carryforward expiration year | 2020 | |
Shires Mrt Program [Member] | ||
Operating Loss Carryforwards [Line Items] | ||
Income tax benefits | $ 0 | $ 500,000 |
Minimum [Member] | ||
Operating Loss Carryforwards [Line Items] | ||
Increase in ownership of certain stockholders or public groups in the stock as result of transactions | 50.00% |
Income Taxes - Schedule of Reco
Income Taxes - Schedule of Reconciliation of U.S. Federal Statutory Income Tax Rate and Effective Income Tax Rate (Detail) | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
U.S. federal statutory income tax rate | (21.00%) | (21.00%) |
State income taxes, net of federal benefit | (9.40%) | (6.60%) |
Research and development tax credits and orphan drug credit | (11.60%) | (6.80%) |
Stock-based compensation | (3.50%) | 0.50% |
Other permanent differences | 0.00% | 0.20% |
Change in deferred tax asset valuation allowance | 37.00% | 33.60% |
Officers compensation | 3.50% | 0.00% |
NOL and tax credit expiration under 382 | 4.80% | 0.00% |
Other | 0.20% | (0.30%) |
Effective income tax rate | 0.00% | (0.40%) |
Income Taxes - Schedule of Comp
Income Taxes - Schedule of Components of Deferred Tax Assets and Liabilities (Detail) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 |
Deferred tax assets: | |||
Net operating loss carryforwards | $ 64,639 | $ 61,460 | |
Research and development tax credit and orphan drug credit carryforwards | 28,406 | 21,830 | |
Intangibles | 7,570 | 3,607 | |
Deferred revenue | 3,753 | 11,845 | |
Depreciation and amortization | 17,305 | 2,253 | |
Accrued expenses and other | 4,583 | 5,354 | |
Total deferred tax assets | 126,256 | 106,349 | |
Valuation allowance | (126,256) | (106,346) | $ (68,143) |
Net deferred tax assets | $ 0 | $ 3 |
Income Taxes - Summary of Chang
Income Taxes - Summary of Changes in Valuation Allowance for Deferred Tax Assets (Detail) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Valuation Allowance [Line Items] | ||
Valuation allowance at beginning of year | $ (106,346) | $ (68,143) |
Valuation allowance at end of year | (126,256) | (106,346) |
Increases Recorded to Income Tax Provision [Member] | ||
Valuation Allowance [Line Items] | ||
Increases/decreases in valuation allowance | $ (19,910) | $ (38,203) |
Net Loss per Share - Summary of
Net Loss per Share - Summary of Basic and Diluted Net Loss Per Share Attributable to Common Stockholders (Detail) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | ||||||||
Dec. 31, 2020 | Sep. 30, 2020 | Jun. 30, 2020 | Mar. 31, 2020 | Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2020 | Dec. 31, 2019 | |
Numerator: | ||||||||||
Net loss | $ (20,561) | $ 17,344 | $ (36,288) | $ (14,282) | $ (31,036) | $ (21,227) | $ (27,832) | $ (33,198) | $ (53,787) | $ (113,293) |
Denominator: | ||||||||||
Weighted average common shares outstanding—basic and diluted | 67,520,886 | 51,445,539 | ||||||||
Net loss per share—basic and diluted | $ (0.48) | $ (0.41) | $ (0.57) | $ (0.74) | $ (0.80) | $ (2.20) |
Net Loss per Share - Additional
Net Loss per Share - Additional Information (Detail) - shares | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Earnings Per Share, Basic, by Common Class, Including Two Class Method [Line Items] | ||
Antidilutive securities excluded from computation of basic net loss per share | 9,557,391 | 8,680,546 |
Restricted Common Stock [Member] | ||
Earnings Per Share, Basic, by Common Class, Including Two Class Method [Line Items] | ||
Antidilutive securities excluded from computation of basic net loss per share | 34,168 | |
Restricted Common Stock [Member] | Weighted Average [Member] | ||
Earnings Per Share, Basic, by Common Class, Including Two Class Method [Line Items] | ||
Antidilutive securities excluded from computation of basic net loss per share | 124,697 |
Net Loss per Share - Summary _2
Net Loss per Share - Summary of Potential Common Shares Excluded from Computation of Diluted Net Loss per Share Attributable to Common Stockholders (Detail) - shares | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Earnings Per Share, Basic, by Common Class, Including Two Class Method [Line Items] | ||
Antidilutive securities excluded from computation of basic net loss per share | 9,557,391 | 8,680,546 |
Stock Options [Member] | ||
Earnings Per Share, Basic, by Common Class, Including Two Class Method [Line Items] | ||
Antidilutive securities excluded from computation of basic net loss per share | 9,557,391 | 8,646,378 |
Restricted Common Stock [Member] | ||
Earnings Per Share, Basic, by Common Class, Including Two Class Method [Line Items] | ||
Antidilutive securities excluded from computation of basic net loss per share | 34,168 |
Leases - Additional Information
Leases - Additional Information (Details) | Nov. 03, 2020USD ($)ft² | Mar. 31, 2018 | Oct. 31, 2020USD ($) | Jun. 30, 2017USD ($)ft² | Dec. 31, 2020USD ($) | Dec. 31, 2019USD ($) |
Lessee Lease Description [Line Items] | ||||||
Lease expiration period | Mar. 31, 2023 | Mar. 31, 2023 | ||||
Remaining term of lease | 5 years | 8 years | ||||
Operating lease right of use assets | $ 72,957,000 | $ 10,400,000 | ||||
Operating lease, liability | 62,686,000 | 12,614,000 | ||||
Operating lease commitment | 84,049,000 | 24,171,000 | ||||
Operating lease liability due in first year | 18,067,000 | 2,659,000 | ||||
Operating lease liability due in second year | 15,178,000 | 2,737,000 | ||||
Opearting lease payment | 7,775,000 | $ 2,583,000 | ||||
Waltham Massachusetts [Member] | ||||||
Lessee Lease Description [Line Items] | ||||||
Office and laboratory space | ft² | 138,444 | |||||
Operating lease right of use assets | 0 | |||||
Operating lease, liability | 0 | |||||
Tenant improvement for lease | $ 26,300,000 | |||||
Additional tenant allownace per square foot | $ 15 | |||||
Operating lease liability due in first year | 5,700,000 | |||||
Operating lease liability due in second year | 8,000,000 | |||||
Restricted cash | $ 3,900,000 | |||||
Operating Lease Term Of Contract | 10 years | |||||
Percentage Of Increase In The Monthly Rental Expense | 3.00% | |||||
Lexington, Massachusetts [Member] | ||||||
Lessee Lease Description [Line Items] | ||||||
Office and laboratory space | ft² | 59,000 | |||||
Monthly lease payments | $ 200,000 | |||||
Percentage of annual increase in operating lease | 3.00% | |||||
Cash deposit collateral | $ 1,000,000 | |||||
Remaining term of lease | 9 years | |||||
Operating Lease Term Of Contract | 5 years | 10 years | ||||
Suite Retention And Development Agreement [Member] | ||||||
Lessee Lease Description [Line Items] | ||||||
Commitment to Build Out Cost | $ 6,000,000 | |||||
Shared Overage cost commitment | 11,000,000 | |||||
Maximum [Member] | Waltham Massachusetts [Member] | ||||||
Lessee Lease Description [Line Items] | ||||||
Opearting lease payment | 11,000,000 | |||||
Minimum [Member] | Waltham Massachusetts [Member] | ||||||
Lessee Lease Description [Line Items] | ||||||
Opearting lease payment | 9,000,000 | |||||
Albany Molecular Research, Inc. ("AMRI") [Member] | Suite Retention And Development Agreement [Member] | ||||||
Lessee Lease Description [Line Items] | ||||||
Monthly lease payments | 1,000,000 | |||||
Payments For Build Out Costs | 1,100,000 | |||||
Future Build Out Costs | 4,100,000 | |||||
Operating lease right of use assets | 66,600,000 | |||||
Operating lease, liability | $ 53,800,000 | |||||
Operating Lease Term Of Contract | 5 years | |||||
Operating Lease Renewal Term | 3 years | |||||
Operating lease option to extend description | the Company has the right to extend for an additional three years | |||||
Percentage Of Increase In The Monthly Rental Expense | 3.00% | |||||
Albany Molecular Research, Inc. ("AMRI") [Member] | Suite Retention And Development Agreement [Member] | Other Noncurrent Assets [Member] | ||||||
Lessee Lease Description [Line Items] | ||||||
Payments For Build Out Costs | $ 12,800,000 | |||||
Biomedical Research Models Inc [Member] | Suite Retention And Development Agreement [Member] | ||||||
Lessee Lease Description [Line Items] | ||||||
Monthly lease payments | $ 100,000 | |||||
Operating lease right of use assets | 300,000 | |||||
Operating lease, liability | $ 300,000 | |||||
Operating Lease Term Of Contract | 13 months | |||||
Azzur Cleanrooms on Demand Burlington LLC [Member] | Burlington Massachusetts [Member] | ||||||
Lessee Lease Description [Line Items] | ||||||
Monthly lease payments | 400,000 | |||||
Operating lease right of use assets | 0 | |||||
Operating lease, liability | 0 | |||||
Operating lease commitment | $ 8,800,000 | |||||
Operating Lease Term Of Contract | 24 months | |||||
Percentage Of Increase In The Monthly Rental Expense | 4.00% |
Leases - Schedule of Lease Cost
Leases - Schedule of Lease Cost (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Lease Cost | ||
Operating lease cost | $ 8,099 | $ 2,692 |
Total lease cost | 8,099 | 2,692 |
Operating cash flows from operating leases | 7,775 | $ 2,583 |
Operating lease liabilities arising from obtaining right-of-use assets | $ 54,063 | |
Weighted-average remaining lease term | 5 years | 8 years |
Weighted-average discount rate | 11.90% | 17.50% |
Leases - Schedule of Maturities
Leases - Schedule of Maturities of Operating Lease Liabilities (Detail) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Leases [Abstract] | ||
2020 and 2021 | $ 18,067 | $ 2,659 |
2021 and 2022 | 15,178 | 2,737 |
2022 and 2023 | 15,591 | 2,818 |
2023 and 2024 | 16,050 | 2,860 |
2024 and 2025 | 12,029 | 2,937 |
Thereafter | 7,134 | 10,160 |
Total future minimum lease payments | 84,049 | 24,171 |
Less: imputed interest | (21,363) | (11,557) |
Present value of lease liabilities | $ 62,686 | $ 12,614 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Detail) - USD ($) $ in Thousands | 1 Months Ended | 12 Months Ended | |
Sep. 30, 2019 | Dec. 31, 2020 | Dec. 31, 2019 | |
Loss Contingencies [Line Items] | |||
Research agreement, payable amount | $ 2,805 | $ 1,763 | |
Research and development | 109,629 | $ 76,369 | |
Research agreement, expiration period | 2022-12 | ||
Research agreement, payment | $ 1,700 | $ 300 | |
Roche Diagnostics Corporation Master Supply Agreement [Member] | |||
Loss Contingencies [Line Items] | |||
Raw material to purchase as percentage of demand | 80.00% | ||
Agreement extended date | Dec. 31, 2024 | ||
Commitment amount | $ 14,000 | ||
Purchase commitments, year 2021 | 3,500 | ||
Purchase commitments, year 2022 | 3,500 | ||
Purchase commitments, year 2023 | 3,500 | ||
Purchase commitments, year 2024 | 3,500 | ||
Research and development | 3,300 | 12,500 | |
MIT Research Agreement [Member] | |||
Loss Contingencies [Line Items] | |||
Research agreement, payable amount | 0 | ||
Research and development | 1,400 | 0 | |
Research agreement, committed amount | $ 4,100 | ||
Research agreement, expiration period | 2022-12 | ||
MIT Exclusive Patent License Agreement [Member] | |||
Loss Contingencies [Line Items] | |||
Research and development | 200 | 200 | |
Annual license maintenance payments | 200 | ||
Payments of annual license maintenance fees | 200 | $ 200 | |
MIT Exclusive Patent License Agreement [Member] | Sanofi Pasteur Inc [Member] | |||
Loss Contingencies [Line Items] | |||
Upfront payment received | 700 | ||
MIT Exclusive Patent License Agreement [Member] | Milestone Payment One [Member] | |||
Loss Contingencies [Line Items] | |||
License agreement, milestone payments | 1,375 | ||
MIT Exclusive Patent License Agreement [Member] | Milestone Payment Two [Member] | |||
Loss Contingencies [Line Items] | |||
License agreement, milestone payments | 1,250 | ||
Sublicense Second Amendment Agreement With Sanofi [Member] | MIT Exclusive Patent License Agreement [Member] | Sanofi Pasteur Inc [Member] | |||
Loss Contingencies [Line Items] | |||
Additional upfront payment payable | $ 300,000 |
Related Party Transactions - Ad
Related Party Transactions - Additional Information (Detail) - USD ($) $ / shares in Units, $ in Millions | 1 Months Ended | ||
Jun. 30, 2020 | Sep. 30, 2019 | May 31, 2019 | |
Private Placement [Member] | Baupost Group LLC [Member] | |||
Related Party Transaction [Line Items] | |||
Sale of Stock, Number of Shares Issued in Transaction | 2,352,941 | ||
Sale of Stock, Price Per Share | $ 8.50 | ||
Sale of Stock, Consideration Received on Transaction | $ 20 | ||
Public Offering [Member] | |||
Related Party Transaction [Line Items] | |||
Sale of Stock, Price Per Share | $ 22 | ||
Public Offering [Member] | Baupost Group LLC [Member] | |||
Related Party Transaction [Line Items] | |||
Sale of Stock, Number of Shares Issued in Transaction | 500,000 | 5,000,000 | |
Sale of Stock, Price Per Share | $ 22 | $ 10 | |
Sale of Stock, Consideration Received on Transaction | $ 11 | $ 50 |
Benefit Plans - Additional Info
Benefit Plans - Additional Information (Detail) - USD ($) $ in Millions | 12 Months Ended | |
Dec. 31, 2020 | Dec. 31, 2019 | |
Retirement Benefits [Abstract] | ||
Employer's discretionary contribution to the plan | $ 0.5 | $ 0.4 |
Selected Quarterly Financial _3
Selected Quarterly Financial Information (Unaudited) - Schedule of Quarterly Financial Information (Detail) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | ||||||||
Dec. 31, 2020 | Sep. 30, 2020 | Jun. 30, 2020 | Mar. 31, 2020 | Dec. 31, 2019 | Sep. 30, 2019 | Jun. 30, 2019 | Mar. 31, 2019 | Dec. 31, 2020 | Dec. 31, 2019 | |
Selected Quarterly Financial Information [Abstract] | ||||||||||
Collaboration revenue | $ 51,392 | $ 66,446 | $ 16,319 | $ 4,654 | $ 3,890 | $ 1,266 | $ 1,174 | $ 1,474 | $ 138,811 | $ 7,804 |
Total operating expenses | 72,034 | 49,697 | 52,950 | 19,445 | 35,629 | 22,901 | 29,364 | 35,679 | 194,126 | 123,573 |
Income (loss) from operations | (20,642) | 16,749 | (36,631) | (14,791) | (31,739) | (21,635) | (28,190) | (34,205) | (55,315) | (115,769) |
Net income (loss) | $ (20,561) | $ 17,344 | $ (36,288) | $ (14,282) | $ (31,036) | $ (21,227) | $ (27,832) | $ (33,198) | $ (53,787) | $ (113,293) |
Net income (loss) per share applicable to common stockholders—basic | $ (0.22) | $ 0.24 | $ (0.58) | $ (0.24) | $ (0.80) | |||||
Net income (loss) per share applicable to common stockholders—diluted | $ (0.22) | $ 0.23 | $ (0.58) | $ (0.24) | (0.80) | |||||
Net loss per share applicable to common stockholders—basic and diluted | $ (0.48) | $ (0.41) | $ (0.57) | $ (0.74) | $ (0.80) | $ (2.20) |
Selected Quarterly Financial _4
Selected Quarterly Financial Information (Unaudited) - Additional Information (Detail) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |
Mar. 31, 2020 | Dec. 31, 2020 | Dec. 31, 2019 | |
Selected Quarterly Financial Information [Abstract] | |||
Decrease in fair value of contingent consideration | $ 9,500 | $ 48,575 | $ 13 |
Subsequent Event - Additional I
Subsequent Event - Additional Information (Detail) | Jan. 20, 2021shares |
Subsequent Event [Member] | Inducement Stock Incentive Plan Two Thousand And Twenty One [Member] | |
Subsequent Event [Line Items] | |
Share-based Compensation Arrangement by Share-based Payment Award, Number of Shares Available for Grant | 2,612,550 |