Document and Entity Information
Document and Entity Information - USD ($) | 12 Months Ended | ||
Dec. 31, 2021 | Mar. 28, 2022 | Jun. 30, 2021 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Amendment Flag | false | ||
Document Period End Date | Dec. 31, 2021 | ||
Document Fiscal Year Focus | 2021 | ||
Document Fiscal Period Focus | FY | ||
Document Annual Report | true | ||
Document Transition Report | false | ||
Entity Registrant Name | 4D Molecular Therapeutics, Inc. | ||
Entity Central Index Key | 0001650648 | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Well-known Seasoned Issuer | No | ||
Entity Current Reporting Status | Yes | ||
Entity Voluntary Filers | No | ||
Entity Filer Category | Non-accelerated Filer | ||
Entity Small Business | true | ||
Entity Emerging Growth Company | true | ||
Entity Ex Transition Period | false | ||
ICFR Auditor Attestation Flag | false | ||
Entity Shell Company | false | ||
Entity Common Stock, Shares Outstanding | 32,237,496 | ||
Entity Public Float | $ 444,521,977 | ||
Entity Interactive Data Current | Yes | ||
Entity File Number | 001-39782 | ||
Entity Tax Identification Number | 47-3506994 | ||
Entity Address, Address Line One | 5858 Horton Street #455 | ||
Entity Address, City or Town | Emeryville | ||
Entity Address, State or Province | CA | ||
Entity Address, Postal Zip Code | 94608 | ||
City Area Code | (510) | ||
Local Phone Number | 505-2680 | ||
Entity Incorporation, State or Country Code | DE | ||
Title of 12(b) Security | Common Stock, par value $0.0001 per share | ||
Trading Symbol | FDMT | ||
Security Exchange Name | NASDAQ | ||
Documents Incorporated by Reference | DOCUMENTS INCORPORATED BY REFERENCE Portions of the registrant’s definitive Proxy Statement relating to the 2022 Annual Meeting of Stockholders are incorporated herein by reference in Part III of this Annual Report on Form 10-K to the extent stated herein. The proxy statement will be filed with the Securities and Exchange Commission within 120 days of the registrant’s fiscal year ended December 31, 2021. | ||
Auditor Firm ID | 238 | ||
Auditor Name | PricewaterhouseCoopers LLP | ||
Auditor Location | San Jose, California |
Balance Sheets
Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Current assets: | ||
Cash and cash equivalents | $ 153,001 | $ 276,726 |
Marketable securities | 94,776 | 0 |
Accounts receivable | 47 | 1,486 |
Prepaid expenses and other current assets (includes $285 and $169 at December 31, 2021 and 2020, respectively, attributable to related parties) | 8,456 | 4,444 |
Total current assets | 256,280 | 282,656 |
Marketable securities, long-term | 67,652 | 0 |
Property and equipment, net | 14,391 | 5,073 |
Operating lease, right-of-use assets, net | 14,562 | 0 |
Other assets | 602 | 602 |
Total assets | 353,487 | 288,331 |
Current liabilities | ||
Accounts payable (includes $0 and $173 at December 31, 2021 and 2020, respectively, attributable to related parties) | 4,764 | 1,787 |
Accrued and other current liabilities | 7,744 | 8,371 |
Deferred revenue | 2,599 | 6,586 |
Operating lease liabilities, current portion | 1,231 | 0 |
Total current liabilities | 16,338 | 16,744 |
Deferred revenue, net of current portion | 2,491 | 13,226 |
Derivative liability | 214 | 122 |
Operating lease liabilities, long -term portion | 15,217 | 0 |
Other liabilities | 120 | 1,852 |
Total liabilities | 34,380 | 31,944 |
Commitments and contingencies (Note 9) | ||
Stockholders' equity | ||
Preferred stock, $0.0001 par value, 10,000,000 shares authorized at December 31, 2021 2020; no shares issued and outstanding at December 31, 2021 and 2020, respectively. | 0 | 0 |
Common stock, $0.0001 par value, 300,000,000 shares authorized at December 31, 2021 and 2020; 32,224,524 and 26,681,983 shares issued and outstanding at December 31, 2021 and 2020, respectively. | 3 | 3 |
Additional paid-in-capital | 526,523 | 392,063 |
Accumulated other comprehensive loss | (423) | 0 |
Accumulated deficit | (206,996) | (135,679) |
Total stockholders' equity | 319,107 | 256,387 |
Total liabilities and stockholders' equity | $ 353,487 | $ 288,331 |
Balance Sheets (Parenthetical)
Balance Sheets (Parenthetical) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Statement of Financial Position [Abstract] | ||
Prepaid expenses to related parties | $ 285 | $ 169 |
Accounts payable to related parties | $ 0 | $ 173 |
Preferred stock, par value | $ 0.0001 | $ 0.0001 |
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 | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 300,000,000 | 300,000,000 |
Common stock, shares, issued | 32,224,524 | 26,681,983 |
Common stock, shares, outstanding | 32,224,524 | 26,681,983 |
Statements of Operations
Statements of Operations - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Revenue: | ||
Collaboration and license revenue | $ 18,038 | $ 13,363 |
Collaboration and license revenue, related parties | 249 | |
Total revenue | 18,038 | 13,612 |
Operating expenses: | ||
Research and development (includes $935 and $579 for the years ended December 31, 2021, and 2020, respectively, attributable to related parties) | 61,360 | 53,038 |
General and administrative | 28,011 | 17,238 |
Total operating expenses | 89,371 | 70,276 |
Loss from operations | (71,333) | (56,664) |
Other income (expense): | ||
Interest income | 137 | 152 |
Other income (expense), net | (121) | (181) |
Total other income (expense) | 16 | (29) |
Net loss | $ (71,317) | $ (56,693) |
Net loss per share, basic and diluted | $ (2.57) | $ (8.82) |
Weighted-average shares outstanding used in computing net loss per share, basic and diluted | 27,730,420 | 6,430,555 |
Statements of Operations (Paren
Statements of Operations (Parenthetical) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Income Statement [Abstract] | ||
Research and development expense, related party | $ 935 | $ 579 |
Statements of Comprehensive Los
Statements of Comprehensive Loss - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Statement of Comprehensive Income [Abstract] | ||
Net loss | $ (71,317) | $ (56,693) |
Other comprehensive loss: | ||
Gross unrealized loss on marketable securities | (423) | 0 |
Total comprehensive loss | $ (71,740) | $ (56,693) |
Statements of Redeemable Conver
Statements of Redeemable Convertible Preferred Stock and Stockholders' Equity (Deficit) - USD ($) $ in Thousands | Total | Common Stock | Additional Paid-in Capital | Additional Paid-in CapitalCumulative Effect, Period of Adoption, Adjustment | Accumulated Other Comprehensive Loss | Accumulated Deficit | Accumulated DeficitCumulative Effect, Period of Adoption, Adjustment |
Stockholders’ Equity (Deficit), beginning balances at Dec. 31, 2019 | $ (72,970) | $ 1 | $ 6,054 | $ (39) | $ (79,025) | $ 39 | |
Redeemable Convertible Preferred Stock, beginning balances (in shares) at Dec. 31, 2019 | 7,375,631 | ||||||
Redeemable Convertible Preferred Stock, beginning balances at Dec. 31, 2019 | $ 102,980 | ||||||
Stockholders’ Equity (Deficit), beginning balances (in shares) at Dec. 31, 2019 | 5,178,955 | ||||||
Accounting Standards Update [Extensible List] | Accounting Standard Update ("ASU") 2018-07 | ||||||
Issuance of redeemable convertible preferred stock, net of $3,138 of issuance cost | $ 72,468 | ||||||
Issuance of redeemable convertible preferred stock, net of $3,138 of issuance cost (in shares) | 4,200,353 | ||||||
Conversion of redeemable convertible preferred stock into common stock | $ 175,448 | $ 1 | 175,447 | ||||
Conversion of redeemable convertible preferred stock into common stock (in shares) | 11,575,984 | ||||||
Conversion of redeemable convertible preferred stock into common stock | $ (175,448) | ||||||
Conversion of redeemable convertible preferred stock into common stock (in shares) | (11,575,984) | ||||||
Issuance of common stock upon initial public offering, net of issuance cost | $ 204,713 | $ 1 | 204,712 | ||||
Issuance of common stock upon initial public offering, net of issuance cost (in shares) | 9,660,000 | ||||||
Issuance of common stock upon exercise of stock options | 857 | 857 | |||||
Issuance of common stock upon exercise of stock options (in shares) | 267,044 | ||||||
Stock-based compensation expense | 4,984 | 4,984 | |||||
Vesting of common stock warrants issued for services | 48 | 48 | |||||
Net loss | (56,693) | (56,693) | |||||
Stockholders’ Equity (Deficit), ending balances at Dec. 31, 2020 | $ 256,387 | $ 3 | 392,063 | (135,679) | |||
Redeemable Convertible Preferred Stock, ending balances (in shares) at Dec. 31, 2020 | 0 | ||||||
Stockholders’ Equity (Deficit), ending balances (in shares) at Dec. 31, 2020 | 26,681,983 | ||||||
Issuance of common stock upon exercise of stock options and warrants | $ 3,905 | 3,905 | |||||
Issuance of common stock upon exercise of stock options and warrants (in shares) | 646,417 | ||||||
Issuance of common stock upon initial public offering, net of issuance cost | $ 115,090 | 115,090 | |||||
Issuance of common stock upon initial public offering, net of issuance cost (in shares) | 4,875,715 | ||||||
Issuance of common stock upon exercise of stock options (in shares) | 563,387 | ||||||
Issuance of common stock warrant | $ 1,146 | 1,146 | |||||
Stock-based compensation expense | 13,799 | 13,799 | |||||
Issuance of common stock - 2020 ESPP | 433 | 433 | |||||
Issuance of common stock - 2020 ESPP (in shares) | 20,409 | ||||||
Vesting of common stock warrants issued for services | 87 | 87 | |||||
Gross unrealized loss on marketable securities | (423) | $ (423) | |||||
Net loss | (71,317) | (71,317) | (71,317) | ||||
Stockholders’ Equity (Deficit), ending balances at Dec. 31, 2021 | $ 319,107 | $ 3 | $ 526,523 | $ (423) | $ (206,996) | ||
Redeemable Convertible Preferred Stock, ending balances (in shares) at Dec. 31, 2021 | 0 | ||||||
Stockholders’ Equity (Deficit), ending balances (in shares) at Dec. 31, 2021 | 32,224,524 |
Statements of Redeemable Conv_2
Statements of Redeemable Convertible Preferred Stock and Stockholders' Equity (Deficit) (Parenthetical) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Initial Public Offering | ||
Issuance of common stock upon initial public offering, issuance cost | $ 7,660 | $ 17,468 |
Redeemable Convertible Preferred Stock | ||
Issuance cost for issuance of preferred stock | $ 3,138 |
Statements of Cash Flows
Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Cash flows from operating activities | ||
Net loss | $ (71,317) | $ (56,693) |
Adjustments to reconcile net loss to net cash used in operating activities | ||
Stock-based compensation expense | 13,799 | 4,984 |
Vesting of common stock warrant in return for services | 87 | 48 |
Change in fair value of derivative liability | 92 | 21 |
Depreciation and amortization | 1,523 | 1,443 |
Amortization of right-of-use assets | 1,497 | 0 |
Net amortization (accretion) of premium (discount) on marketable securities | 719 | 0 |
Changes in operating assets and liabilities | ||
Accounts receivable | 1,439 | (508) |
Prepaid expenses and other current assets | (4,225) | (2,566) |
Other assets | 0 | 75 |
Accounts payable | 2,285 | 43 |
Accrued and other liabilities | 689 | 1,899 |
Deferred revenue | (14,722) | 345 |
Operating lease liabilities | (1,000) | 0 |
Net cash used in operating activities | (69,134) | (50,909) |
Cash flows from investing activities | ||
Acquisition of property and equipment | (9,110) | (1,000) |
Purchase of marketable securities | (168,570) | 0 |
Maturities of marketable securities | 5,000 | 0 |
Net cash used in investing activities | (172,680) | (1,000) |
Cash flows from financing activities | ||
Issuance of redeemable convertible preferred stock, net of issuance costs | 0 | 72,468 |
Issuance of common stock upon offerings, net of issuance costs | 114,296 | 205,658 |
Issuance of common stock upon exercise of stock options and warrants | 3,360 | 857 |
Issuance of common stock through 2020 ESPP | 433 | 0 |
Net cash provided by financing activities | 118,089 | 278,983 |
Net (decrease) increase in cash and cash equivalents | (123,725) | 227,074 |
Cash and cash equivalents, beginning of period | 276,726 | 49,652 |
Cash and cash equivalents, end of period | 153,001 | 276,726 |
Supplemental disclosures of noncash investing and financing information | ||
Conversion of redeemable convertible preferred stock into common stock | 0 | 175,448 |
Purchases of property and equipment in accounts payable and accrued and other liabilities | 2,199 | 466 |
Unpaid offering costs | 152 | 946 |
Decrease in right-of-use assets due to reduction in operating lease liabilities upon modification | (478) | 0 |
Issuance of common stock warrant to settle liability | 1,146 | 0 |
Receivable for common stock issued upon exercise of stock options | $ 545 | $ 0 |
The Company
The Company | 12 Months Ended |
Dec. 31, 2021 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
The Company | 1. The Company 4D Molecular Therapeutics, Inc. (the “Company”) was formed as a limited liability company in September 2013 under the name 4D Molecular Therapeutics, LLC. The Company changed its name and converted into a corporation which was incorporated in the state of Delaware in March 2015. The Company is a clinical-stage gene therapy company pioneering the development of product candidates using its targeted and evolved adeno-associated viruses (“AAV”) vectors. Initial Public Offering In December 2020, the Company sold and issued 9,660,000 shares of common stock at a price to the public of $ 23.00 per share, which included shares sold upon the underwriters’ exercise of their overallotment option to purchase 1,260,000 additional shares. The Company received an aggregate of $ 204.7 million in net proceeds, after deducting underwriting discounts and commissions and offering costs. Upon the closing of the Company's initial public offering in December 2020 (the "IPO"), all outstanding shares of redeemable convertible preferred stock automatically converted into 11,575,984 shares of common stock. Subsequent to the closing of the IPO, there were no shares of redeemable convertible preferred stock outstanding. Follow On Public Offering In November 2021, the Company completed its second underwritten public offering ("Follow-on Offering") in which 4,750,000 shares of the Company's common stock were sold at an offering price of $ 25.00 per share pursuant to an effective Registration Statement on Form S-1. The net proceeds from the Follow-on Offering were $ 111.1 million, after deducting underwriting discounts and commissions and offering expenses. Liquidity The Company has incurred significant losses and negative cash flows from operations and had an accumulated deficit of $ 207.0 million as of December 31, 2021. The Company believes that its cash and cash equivalents and marketable securities as of December 31, 2021 are sufficient for the Company to fund planned operations for at least one year from the issuance date of these financial statements for the year ended December 31, 2021. The Company has historically financed its operations primarily through the sale of equity securities, and to a lesser extent, from cash received pursuant to its collaboration and license agreements. To date, none of the Company’s product candidates have been approved for sale, and therefore, the Company has not generated any revenue from product sales. Management expects operating losses and negative cash flows from operations to continue for the foreseeable future. The Company plans to raise additional funding as required based on the status of its clinical trials and projected cash flows. There can be no assurance that, in the event the Company requires additional financing, such financing will be available on terms acceptable to the Company, if at all. Failure to generate sufficient cash flows from operations, raise additional capital and reduce discretionary spending should additional capital not become available could have a material adverse effect on the Company’s ability to achieve its business objectives. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2021 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Basis of Presentation The accompanying financial statements have been prepared in accordance with United States Generally Accepted Accounting Principles (“U.S. GAAP”). Use of Estimates and Judgements The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and judgements that affect the reported amounts of assets, liabilities, revenue and expenses; and disclosure of contingent assets and liabilities as of the date of the financial statements. Such estimates include the determination of useful lives for property and equipment, the contract term, transaction price and costs of collaboration agreements, as well as estimates of the fair value of common stock (prior to the IPO), stock options and derivative instruments and income tax uncertainties. Actual results could differ from those estimates. Due to the coronavirus (“COVID-19”) pandemic, there has been uncertainty and disruption in the global economy and financial markets. The Company is not aware of any specific event or circumstance that would require an update to its estimates or judgments or a revision of the carrying value of its assets or liabilities as of December 31, 2021. While there was not a material impact to the Company’s financial statements as of December 31, 2021, these estimates may change, as new events occur and additional information is obtained, as well as other factors related to the COVID-19 pandemic that could result in material impacts to the financial statements in future reporting periods. Segment Information The Company operates and manages its business as one reportable and operating segment. The Company’s chief executive officer, who is the chief operating decision maker, reviews financial information on a company-wide basis for purposes of allocating resources and assessing financial performance. As of and for the years ended December 31, 2021 and 2020, all of the Company’s long-lived assets were located in the United States and all revenue was earned in the United States. Concentration of Credit Risk Financial instruments that potentially subject the Company to a concentration of credit risk consist of cash and cash equivalents, marketable securities and accounts receivable. The Company’s cash is held at two financial institutions in the United States of America. The Company’s cash equivalents are invested in money market funds. The Company also invests in U.S. Treasuries, commercial paper and corporate bonds. The Company has not experienced any losses on its deposits of cash and cash equivalents. Such deposits may, at times, exceed federally insured limits. The Company’s partners in collaboration and license agreements who represent 10% or more of the Company’s total revenue are as follows: Year Ended December 31, 2021 2020 Customer A 95 % 95 % Customer B * * Total 95 % 95 % * Less than 10 % The Company’s partners in collaboration and license agreements who represent 10% or more of the Company’s total accounts receivable are as follows: December 31, 2021 2020 Customer A 100 % 74 % Customer B 0 % 26 % Total 100 % 100 % The Company’s total revenues by geographic region, based on the location of the customer, are as follows (in thousands): Year Ended December 31, 2021 2020 Netherlands $ 736 $ 742 Switzerland 17,181 12,897 United States 121 ( 27 ) Total revenue $ 18,038 $ 13,612 Cash and Cash Equivalents The Company considers all highly liquid investments with original maturities of three months or less at the date of purchase to be cash equivalents. Cash equivalents consist of money market funds. Marketable Securities Marketable securities consist of commercial paper, corporate bonds and U.S. Treasuries and are included in current and noncurrent assets. The Company classifies its marketable securities as available-for-sale and carries them at fair value on its balance sheet. Fair value is estimated using independent pricing sources based on quoted prices in active markets for similar securities. Unrealized gains and losses on the marketable securities are reported as a component of stockholders' equity (deficit) in accumulated other comprehensive loss. The amortized cost of marketable securities is adjusted for amortization of premiums and accretion of discounts to maturity, which is included in interest income on the statements of operations. Realized gains and losses are included in interest income on the statements of operations. The Company periodically evaluates its marketable securities to assess whether those with unrealized loss positions are other than temporarily impaired. The Company considers various factors in determining whether to recognize an impairment charge. If the Company determines that the decline in an investment's fair value is other-than-temporary, the difference is recognized as an impairment loss in the statements of operations. Other Risks and Uncertainties The Company is subject to risks and uncertainties common to early-stage companies in the biotechnology industry, including, but not limited to, development by competitors of new technological innovations, protection of proprietary technology, dependence on key personnel, suppliers for key raw materials, contract manufacturing organizations (“CMOs”) and contract research organizations (“CROs”), compliance with government regulations and the need to obtain additional financing to fund operations. Product candidates currently under development will require significant additional research and development efforts, including extensive preclinical studies, clinical trials and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel and infrastructure and extensive compliance and reporting. There can be no assurance that the Company’s research and development will be successfully completed, that adequate protection for the Company’s intellectual property will be obtained or maintained, that any products developed will obtain necessary government regulatory approval or that any approved products will be commercially viable. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will generate significant revenue from product sales. The Company operates in an environment of rapid change in technology and substantial competition from other pharmaceutical and biotechnology companies. In addition, the Company is dependent upon the services of its employees, consultants and other third parties (including for clinical trials and some aspects of research and preclinical testing). The extent of the impact of the COVID-19 pandemic on the Company’s business will depend upon the duration and spread of the outbreak and the extent and severity of the impact on the Company’s clinical trial activities, research activities and suppliers, all of which are uncertain and cannot be predicted. The extent to which the coronavirus outbreak may materially impact the Company’s financial condition, liquidity or results of operations is uncertain. Fair Value Measurements The Company applies fair value accounting for all financial assets and liabilities and nonfinancial assets and liabilities that are recognized or disclosed at fair value in the financial statements on a recurring basis. Fair value is an exit price, representing the amount that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants on the measurement date. As such, fair value is a market-based measurement that should be determined based on assumptions that market participants would use in pricing an asset or liability. As a basis for considering such assumptions, a three-level fair value hierarchy has been established, which prioritizes the inputs used in measuring fair value as follows: • Level 1 — Observable inputs that reflect unadjusted quoted market prices in active markets for identical assets or liabilities that are accessible at the measurement date. • Level 2 — Inputs other than Level 1 that are observable, either directly or indirectly, such as quoted prices for similar assets or liabilities; quoted prices in markets that are not active; or other inputs that are observable or can be corroborated by observable market data for substantially the full term of the assets or liabilities. • Level 3 — Unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities. The Company accounts for transfers of financial instruments between levels of the fair value hierarchy on the date of the event or change in circumstance that caused the transfer. Accounts Receivable—Allowance for Doubtful Accounts The Company regularly reviews accounts receivable for collectability and establishes an allowance for probable credit losses and writes off uncollectible accounts as necessary. The Company has determined that no allowance was required at December 31, 2021 and 2020. The Company did no t have any write-offs relating to uncollectible accounts receivable during the years ended December 31, 2021 and 2020. Property and Equipment, Net Property and equipment are stated at cost less accumulated depreciation for acquired assets. Depreciation is computed using the straight-line method over the estimated useful lives of assets, ranging from three to five years . Leasehold improvements are amortized over the shorter of the useful life of the assets or the lease term. Upon sale or retirement of assets, the costs and related accumulated depreciation are removed from the balance sheet and the resulting gain or loss is reflected within operating expenses in the statements of operations. Maintenance and repairs are charged to expense as incurred. Impairment of Long-Lived Assets The Company reviews long-lived assets for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. Recoverability is measured by comparison of the carrying amount to the future undiscounted net cash flows, which the assets are expected to generate. If such assets are considered to be impaired, the impairment to be recognized is typically measured by the amount by which the carrying amount of the assets exceeds the projected discounted future net cash flows arising from the asset. There have been no such impairments of long-lived assets in the years ended December 31, 2021 and 2020. Redeemable Convertible Preferred Stock The Company recorded all shares of redeemable convertible preferred stock at their respective fair values on the date of issuance, net of issuance costs. The redeemable convertible preferred stock was recorded outside of permanent equity because while it was not mandatorily redeemable, in certain events considered not solely within the Company's control, such as a merger or consolidation, sale, lease, or license of substantially all of the Company's assets (each, a "deemed liquidation event"), the convertible preferred stock would become redeemable at the option of the holders of a majority of the outstanding series of redeemable convertible preferred stock. The Company did not adjust the carrying values of the redeemable convertible preferred stock to the liquidation preference of such shares because a deemed liquidation event obligating the Company to pay the liquidation preference did not occur. All outstanding shares of the redeemable convertible preferred stock converted into common stock upon the close of the IPO in December 2020. Common Stock Warrants The Company accounts for common stock warrants which meet the definition of a derivative as liabilities if the warrant requires net cash settlement or gives the holder the option of net cash settlement. The Company accounts for common stock warrants as equity if the contract requires physical settlement or net physical settlement or if the Company has the option of physical settlement or net physical settlement. Common stock warrants classified as liabilities are initially recorded at fair value and remeasured at fair value each balance sheet date with the offset adjustments recorded in other income (expense), net within the statements of operations. Common stock warrants classified as equity are initially measured at fair value on the grant date and are not subsequently remeasured. Leases On December 31, 2021, the Company adopted Accounting Standards Update (“ASU”) No. 2016-02, Leases (Topic 842), ("ASC 842") using the alternative modified retrospective approach effective as of January 1, 2021 to recognize a cumulative-effect adjustment on the effective date and to not adjust financial information and disclosures required under the new lease standards for comparative prior periods. The Company has elected to apply the package of practical expedients requiring no reassessment of whether any expired or existing contracts are or contain leases, the lease classification of any expired or existing leases, or the capitalization of initial direct costs for any existing leases. Additionally, the Company has elected not to separate lease and non-lease components and not to recognize leases with an initial term of twelve months or less. Under ASC 842, at contract inception, the Company determines if an arrangement is or contains a lease. A contract is or contains a lease if the contract conveys the right to control the use of an identified asset for a period of time in exchange for consideration. If determined to be or contain a lease, the lease is assessed for classification as either an operating or finance lease at the lease commencement date, defined as the date on which the leased asset is made available for use by the Company, based on the economic characteristics of the lease. A right-of-use asset represents the economic benefit conveyed to the Company by the right to use the underlying asset over the lease term. A lease liability represents the obligation to make lease payments arising from the lease. Operating lease right-of-use assets represent the Company's right to use an underlying asset for the lease term and operating lease liabilities represent the Company's obligation to make payments arising from the lease. Operating right-of-use assets and liabilities are recognized at the commencement date of the lease and are measured at the present value of the fixed payments due over the expected lease term less the present value of any incentives, rebates, or abatements the Company expects to receive from the lessor. The Company records amortization of operating right-of-use assets and accretion of lease liabilities as a single lease cost on a straight-line basis over the lease term. No lease renewal options are recognized as part of the right-of-use assets and lease liabilities. The Company's operating leases are presented in the balance sheet as operating lease right-of-use assets, classified as noncurrent assets, and operating lease liabilities, classified as current and noncurrent based on the discounted lease payments to be made within the proceeding twelve months. As the Company’s leases do not provide an implicit rate , the Company uses its incremental borrowing rate to discount lease payments. The incremental borrowing rate represents an estimated rate of interest that the Company would have to pay to borrow equivalent funds on a collateralized basis at the lease commencement date. Upon adoption of the new lease standard effective January 1, 2021 , the Company recognized operating lease right-of-use assets of $ 16.5 million, and current and noncurrent operating lease liabilities of $ 2.0 million and $ 16.1 million, respectively. In connection with the adoption of this standard, deferred rent of $ 1.0 million and lease incentive liability of $ 0.5 million, which were previously recorded in prepaid expenses and other current assets, accrued and other current liabilities and other liabilities on the balance sheet as of December 31, 2020, were derecognized. The adoption of the standard did no t have an impact on the Company's statements of operations or comprehensive loss, stockholders' equity or cash flows. The impact of adoption of the new lease standard as of January 1, 2021 is summarized as follows: December 31, 2020 ASC 842 Adjustments January 1, 2021 Operating lease right-of-use assets $ — $ 16,537 $ 16,537 Prepaid expenses and other current assets 4,444 ( 214 ) 4,230 Accrued and other current liabilities ( 8,371 ) 68 ( 8,303 ) Other liabilities ( 1,852 ) 1,696 ( 156 ) Operating lease liabilities, current — ( 2,029 ) ( 2,029 ) Operating lease liabilities, long-term — ( 16,058 ) ( 16,058 ) Revenue Recognition The Company determines revenue recognition for arrangements within the scope of Accounting Standards Update 2014-09, Revenue from Contracts with Customers (Topic 606) ("ASC 606") by performing the following five steps: (i) identification of the promised goods or services in the contract; (ii) determination of whether the promised goods or services are performance obligations including whether they are distinct in the context of the contract; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations based on estimated selling prices; and (v) recognition of revenue when (or as) the Company satisfies each performance obligation. The Company’s revenue is primarily derived through its license, research, development and commercialization agreements. The terms of these types of agreements may include (i) licenses to the Company’s technology, (ii) research and development services, and (iii) services or obligations in connection with participation in research or steering committees. Payments to the Company under these arrangements typically include one or more of the following: nonrefundable upfront and license fees, research funding, milestone and other contingent payments to the Company for the achievement of defined collaboration objectives and certain preclinical, clinical, regulatory and sales-based events, as well as royalties on sales of any commercialized products. Arrangements that include upfront payments are recorded as deferred revenue upon receipt or when due and are recognized as revenue as performance conditions are met. The event-based milestone payments, royalties and cost reimbursements represent variable consideration, and the Company uses the most likely amount method to estimate this variable consideration. Royalty payments are recognized when earned or as the sales occur. The Company records cost reimbursements as accounts receivable when right to consideration is unconditional. A performance obligation is a promise in a contract to transfer a distinct good or service to the customer and is the unit of account in ASC 606. The Company allocates the total transaction price to each performance obligation based on the estimated standalone selling price and recognizes revenue when, or as, the performance obligation is satisfied. The Company includes the unconstrained amount of estimated variable consideration in the transaction price. At the end of each reporting period, the Company re-evaluates the estimated variable consideration included in the transaction price and any related constraint, and if necessary, adjusts its estimate of the overall transaction price. Significant management judgment is required to determine the level of effort required under an arrangement and the period over which the Company expects to complete its performance obligations under the arrangement. Changes in these estimates can have a material effect on revenue recognized. Research and Development Expenses Costs related to research, design and development of programs are charged to research and development expense as incurred. Research and development costs include, but are not limited to, payroll and personnel expenses including stock-based compensation, materials, laboratory supplies, outside services and allocated overhead, including rent, insurance, repairs and maintenance, depreciation and utilities. The Company expenses all research and development costs in the period in which they are incurred. Costs incurred in obtaining technology licenses are charged to research and development expense as acquired in-process research and development if the technology licensed has not reached technological feasibility and has no alternative future use. Accrued Research and Development The Company has entered into various agreements with CROs and CMOs. The Company’s research and development accruals are estimated based on the level of services performed, progress of the studies, including the phase or completion of events, and contracted costs. The estimated costs of research and development provided, but not yet invoiced, are included in accrued and other current liabilities on the balance sheet. If the actual timing of the performance of services or the level of effort varies from the original estimates, the Company will adjust the accrual accordingly. Payments made to CROs or CMOs under these arrangements in advance of the performance of the related services are recorded as prepaid expenses and other current assets until the services are rendered. Stock-Based Compensation As of January 1, 2020 , the Company adopted ASU 2018-07, Compensation - Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting , which aligns the measurement and classification guidance for share-based payments to nonemployees with the guidance for share-based payments to employees. As of January 1, 2020, the Company accounts for stock-based compensation for stock options granted to employees, directors and nonemployees as measured at grant date, based on the fair value of the award. The Company measures the fair value of awards granted using the Black-Scholes option pricing model and recognizes the expense in the statements of operations over the requisite service period, generally four years , using the straight-line method. Forfeitures are accounted for as they occur. The Company’s policy for issuing stock upon stock option exercise is to issue new common stock. Prior to the adoption of ASU 2018-07 on January 1, 2020, stock-based compensation expense related to stock options granted to nonemployees was recognized based on the vesting date fair value of options as the stock options were earned. The Company remeasured the stock-based compensation at each reporting period end with the resulting change in fair value being recognized in the statements of operations over the period the related services were rendered. The Company estimated the service period for the options based on the time that would be required to satisfy the service condition, assuming the service condition would be satisfied. Stock-based compensation expense was recognized over the estimated service period but was accelerated if the performance condition was achieved earlier than estimated. Income Taxes The Company accounts for income taxes under the asset and liability method, which requires, among other things, that deferred income taxes be provided for temporary differences between the tax basis of the Company’s assets and liabilities and their financial statement reported amounts. In addition, deferred tax assets are recorded for the future benefit of utilizing net operating losses and research and development credit carryforwards and are measured using the enacted tax rates and laws that will be in effect when such items are expected to reverse. A valuation allowance is provided against deferred tax assets unless it is more likely than not that they will be realized. The Company accounts for uncertain tax positions by assessing all material positions taken in any assessment or challenge by relevant taxing authorities. Assessing an uncertain tax position begins with the initial determination of the position’s sustainability and is measured at the largest amount of benefit that is greater than fifty percent likely of being realized upon ultimate settlement. The Company’s policy is to recognize interest and penalties related to the underpayment of income taxes as a component of income tax expense or benefit. To date, there have been no interest or penalties charged in relation to the unrecognized tax benefits. Embedded Derivative Embedded derivatives that are required to be bifurcated from the underlying host instrument are accounted for and valued as a separate financial instrument. An embedded derivative exists in the award agreement with the Cystic Fibrosis Foundation (“CFF”). As described in Note 16, the embedded derivative has been bifurcated and is classified as a liability on the balance sheet and separately accounted for at its fair value. The derivative liability is subject to remeasurement to fair value each reporting period. Changes in the fair value of the derivative liability are recognized as a component of other income (expense), net within the statements of operations. Deferred Offering Costs The Company capitalizes certain legal, accounting and other third-party fees that are directly related to the Company’s in-process financings, until such financings are consummated. After consummation of the financing, these costs are recorded as a reduction of the proceeds received as a result of the offering. In the event that a planned offering does not occur or is significantly delayed, all related deferred offering costs will be expensed immediately within the Company’s statements of operations. Net Loss Per Share, Basic and Diluted The Company calculates basic and diluted net loss per share in conformity with the two-class method required for companies with participating securities. The Company considers all series of redeemable convertible preferred stock to be participating securities as the holders are entitled to receive non-cumulative dividends on a pari passu basis in the event the dividend is paid on common shares. Under the two-class method, the net loss is not allocated to the redeemable convertible preferred stock as the holders of redeemable convertible preferred stock do not have a contractual obligation to share in losses. Basic net loss per share is calculated by dividing the net loss by the weighted-average number of shares of common stock outstanding during the period, less shares subject to repurchase. Diluted net loss per share is computed by giving effect to all potentially dilutive common shares outstanding for the period. For purposes of this calculation, redeemable convertible preferred shares, stock options to acquire shares of common stock, common stock warrants, and unvested common stock subject to repurchase, are considered potentially dilutive common shares, but have been excluded from the calculation of diluted net loss per share as their effect is antidilutive. Recent Accounting Pronouncements New Accounting Pronouncements Recently Adopted In June 2018, the Financial Accounting Standards Board ("FASB") issued Accounting Standards Update ("ASU") No. 2018-07, Compensation-Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting. This ASU aligns the measurement and classification guidance for share-based payments to nonemployees with the guidance for share-based payment to employees. Under this ASU, the measurement of equity-classified nonemployee awards is fixed at the grant date, which may lower their cost and reduce volatility in the statements of operations. The transition method provided by this ASU is on a modified retrospective basis, which recognizes a cumulative-effect adjustment to the opening balance of retained earnings in the period of adoption. This ASU is effective for fiscal years beginning after December 15, 2019, and interim periods within fiscal years beginning after December 15, 2020, with early adoption permitted, but no earlier than a company's adoption date of ASC 606. The Company adopted this guidance effective January 1, 2020 using the modified retrospective method. The Company recorded a less than $ 0.1 million cumulative-effect adjustment reflected as a decrease to the opening balance of accumulated deficit and a decrease to additional paid-in capital In November 2018, the FASB issued ASU No. 2018-18, Collaborative Arrangements (Topic 808): Clarifying the Interaction Between Topic 808 and Topic 606 . This ASU clarifies that certain transactions between participants in a collaborative arrangement should be accounted for under ASC 606, Revenue from Contracts with Customers, when the counterparty is a customer. This ASU also precludes an entity from presenting consideration received from a transaction as revenue from contracts with customers if the counterparty is not a customer for that transaction. This ASU is effective for fiscal years beginning after December 15, 2020, and interim periods within fiscal years beginning after December 15, 2021. Early adoption is permitted for entities that have adopted ASC 606, Revenue from Contracts with Customers. The Company adopted this ASU on January 1, 2021 . The adoption of this ASU did not result in a material impact on the Company’s financial statements and related disclosures. In December 2019, the FASB issued ASU No. 2019-12, Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes . This ASU affects general principles within Topic 740 and is meant to simplify the accounting for income taxes by removing certain exceptions to the general framework. The ASU adds guidance to reduce complexity in certain areas, including recognizing a franchise (or similar) tax that is partially based on income as an income-based tax and incremental amounts incurred as a non-income-based tax and recognizing deferred taxes for tax goodwill. ASU 2019-12 also created an exception to the incremental approach for intraperiod tax allocation when there is a loss from continuing operations and income or a gain from other items (for example, other comprehensive income). The amendments in ASU 2019-12 are effective for the Company for fiscal years beginning after December 15, 2021, and interim periods within fiscal years beginning after December 15, 2022. Early adoption of the amendments is permitted. The Company adopted ASU 2019-12 effective January 1, 2021 on a prospective basis. The adoption of this ASU did not result in a material impact on the Company's financial statements and related disclosures. In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842) , as subsequently amended, which sets out the principles for the recognition, measurement, presentation and disclosure of leases for both parties to a contract (i.e., lessees and lessors), and replaces the existing guidance in ASC 840, Leases. The new standard requires lessees to apply a dual approach, classifying leases as either finance or operating leases based on the principle of whether or not the lease is effectively a financed purchase by the lessee. The classification will determine the recognition pattern of lease expense over the term of the lease. In addition, a lessee is required to record (i) a right-of-use asset and a lease liability on its balance sheet for all leases with accounting lease terms of more than twelve months regardless of whether it is an operating or financing lease and (ii) lease expense in its statements of operations for operating leases and amortization and interest expense in its statements of operations for financing leases. Leases with a term of twelve months or less may be accounted for similar to existing guidance for operating leases under Topic 840. In July 2018, the FASB issued ASU No. 2018-11, Leases (Topic 842) , which added an optional transition method that allows companies to adopt the standard as of the beginning of the year of adoption as opposed to the earliest comparative period presented. See earlier disclosure in this footnote on the impact on the Company of the adoption of this standard in 2021. New Accounting Pronouncements Not Yet Adopted As an “emerging growth company,” the Jumpstart Our Business Startups Act (“JOBS Act”) allows the Company to delay adoption of new or revised accounting pronouncements applicable to public companies until such pronouncements are made applicable to private companies. The Company has elected to use this extended transition period under the JOBS Act. The adoption dates discussed below reflect this election. In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments-Credit Losses (Topic 326), Measurement of Credit Losses on Financial Instr |
Fair Value Measurements
Fair Value Measurements | 12 Months Ended |
Dec. 31, 2021 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | 3. Fair Value Measurements The following tables represent the Company’s fair value hierarchy for financial assets and financial liabilities measured at fair value on a recurring basis as of December 31, 2021 and 2020 (in thousands): Basis for Fair Value Measurements Fair Value as of (Level 1) (Level 2) (Level 3) December 31, 2021 Assets Money market funds $ 36,556 $ — $ — $ 36,556 Commercial paper — 37,535 — 37,535 Corporate bonds — 94,864 — 94,864 US Treasuries — 30,029 — 30,029 Total $ 36,556 $ 162,428 $ — $ 198,984 Liabilities Derivative liability $ — $ — $ 214 $ 214 Total $ — $ — $ 214 $ 214 Basis for Fair Value Measurements Fair Value as of (Level 1) (Level 2) (Level 3) December 31, 2020 Assets Money market funds $ 276,726 $ — $ — $ 276,726 Total $ 276,726 $ — $ — $ 276,726 Liabilities Derivative liability $ — $ — $ 122 $ 122 Total $ — $ — $ 122 $ 122 Level 3 Inputs The fair value of the derivative liability is based on significant inputs not observable in the market, which represents a Level 3 measurement within the fair value hierarchy. The fair value of the derivative liability was determined using a present value analysis with multiple scenarios. In determining the fair value of the derivative liability, the inputs impacting fair value include the change of control payment to CFF, the probability of a change of control event, the product status at time of a change of control event and the discount rate. See Note 16 for further discussion on embedded derivative. There were no transfers between Level 1, 2 and 3 during the periods presented. The following table sets forth a summary of the changes in the fair value of the Company’s Level 3 derivative liability (in thousands): Balance as of December 31, 2019 $ 101 Change in fair value included in other income (expense), net 21 Balance as of December 31, 2020 $ 122 Change in fair value included in other income (expense), net 92 Balance as of December 31, 2021 $ 214 |
Marketable Securities
Marketable Securities | 12 Months Ended |
Dec. 31, 2021 | |
Investments, Debt and Equity Securities [Abstract] | |
Marketable Securities | 4. Marketable Securities Marketable securities, which are classified as available-for-sale, consisted of the following as of December 31, 2021 (in thousands): Amortized Cost Basis Gross Unrealized Gains Gross Unrealized Losses Fair Value as of December 31, 2021 Short-term marketable securities: Commercial paper $ 32,471 $ — $ ( 4 ) $ 32,467 Corporate bonds 57,315 — ( 79 ) 57,236 US Treasuries 5,082 — ( 9 ) 5,073 Total short-term marketable securities $ 94,868 $ — $ ( 92 ) $ 94,776 Long-term marketable securities: Commercial paper $ 5,089 $ — $ ( 21 ) $ 5,068 Corporate bonds 37,821 — ( 193 ) 37,628 US Treasuries 25,073 — ( 117 ) 24,956 Total long-term marketable securities $ 67,983 $ — $ ( 331 ) $ 67,652 All marketable securities held as of December 31, 2021, had contractual maturities of less than two years . There have been no realized gains or losses on marketable securities for the periods presented. None of the Company's investments in marketable securities has been in an unrealized loss position for more than one year. The Company determined that it has the ability and intent to hold all marketable securities that have been in a continuous loss position until maturity or recovery, thus there has been no recognition of any other-than-temporary impairment in the year ended December 31, 2021. As of December 31, 2020, the Company had no marketable securities. |
Property and Equipment, Net
Property and Equipment, Net | 12 Months Ended |
Dec. 31, 2021 | |
Property, Plant and Equipment [Abstract] | |
Property and Equipment, Net | 5. Property and Equipment, Net Property and equipment, net, consisted of the following (in thousands): December 31, 2021 2020 Machinery and equipment $ 5,971 $ 4,911 Leasehold improvements 2,527 2,527 Furniture and fixtures 566 473 Office equipment 165 101 Computer equipment and software 421 366 Construction in progress 10,066 539 Total property and equipment 19,716 8,917 Less: Accumulated depreciation and amortization ( 5,325 ) ( 3,844 ) Property and equipment, net $ 14,391 $ 5,073 All property and equipment are maintained in the United States. Depreciation expense was $ 1.5 million and $ 1.4 million for the years ended December 31, 2021 and 2020, respectively. |
Accrued and Other Liabilities,
Accrued and Other Liabilities, Current and Noncurrent | 12 Months Ended |
Dec. 31, 2021 | |
Payables and Accruals [Abstract] | |
Accrued and Other Liabilities, Current and Noncurrent | 6. Accrued and Other Liabilities, Current and Noncurrent Accrued liabilities consisted of the following (in thousands): December 31, 2021 2020 Payroll and related expenses $ 3,994 $ 3,437 Accrued clinical and preclinical study costs 407 869 Consulting and professional 1,573 3,574 Other accrued expenses 1,770 491 Total accrued and other current liabilities $ 7,744 $ 8,371 Other liabilities, noncurrent consisted of the following (in thousands): December 31, 2021 2020 Deferred rent $ — $ 1,237 Other payable 120 615 Total other liabilities, noncurrent $ 120 $ 1,852 |
Research and Collaboration Arra
Research and Collaboration Arrangements | 12 Months Ended |
Dec. 31, 2021 | |
Research And Collaboration Arrangements [Abstract] | |
Research and Collaboration Arrangements | 7. Research and Collaboration Arrangements Collaboration and license revenue for each period was as follows (in thousands): Year Ended December 31, 2021 2020 uniQure $ 736 $ 742 Roche 17,181 12,897 CFF 121 ( 27 ) $ 18,038 $ 13,612 Deferred revenue for each period was as follows (in thousands): Deferred Revenue 2021 2020 uniQure $ 3,661 $ 4,396 Roche — 14,318 CFF 1,429 1,098 $ 5,090 $ 19,812 The total amount of revenue in the year ended December 31, 2021, which was included in deferred revenue at January 1, 2021, was $ 15.1 million. The total amount of revenue in the year ended December 31, 2020, which was included in deferred revenue at January 1, 2020, was $ 3.2 million. uniQure In January 2014, the Company and uniQure biopharma B.V. (“uniQure”) entered into a Collaboration and License Agreement (the “uniQure Agreement”) to collaborate on the discovery and non-clinical research activities related to the Company’s Therapeutic Vector Evolution platform in order to generate and validate vectors for gene delivery to treat diseases within the central nervous system and liver (together, the “uniQure Field”). The uniQure Agreement provided uniQure with a research license as well as an exclusive development and commercialization license for each project variant selected for further development. The initial research term is three years with an option for uniQure to extend the research term one time for an additional year. Once the Company’s research plan has concluded, uniQure is solely responsible for the continued development, manufacturing and commercialization of the project variants as potential product candidates. In October 2016, uniQure exercised its option to extend the research term for an additional year to January 2018. The Company was also required to work exclusively with uniQure in the uniQure Field (the “uniQure Exclusivity Clause”). Pursuant to the uniQure Agreement, the Company received upfront payments of $ 0.2 million, and was entitled to receive (i) contingent payments for the achievement of research and development milestones of up to $ 5.0 million for each licensed product selected under the arrangement, and (ii) royalties in the single digit range on future sales of the potential product candidates and sublicense consideration in the low teens to low thirties range on any future sublicensing arrangements. The Company also received capped research and development service fees based on contractual full-time employee rates per year. In connection with the performance obligations under the uniQure Agreement, the founders of 4D Molecular Therapeutics, LLC received equity options to purchase an aggregate of 609,744 of uniQure ordinary shares that vest over the initial three-year term of the agreement. The upfront payment of $ 0.2 million was recorded as deferred revenue and was recognized on a ratable basis over the estimated performance period of four years . Payments and reimbursements for research costs were recognized on an as-incurred basis. The options to purchase uniQure shares were deemed to be a noncash component of the arrangement consideration, as the vesting of options is linked to the uniQure Agreement and there is a requirement for the holders of the options to provide services under the agreement. The fair value of the uniQure options, which was estimated to be $ 10.6 million, was recognized ratably as revenue over the estimated performance period of four years and the associated compensation expense related to the stock options were recorded as research and development expense. In August 2019, the Company and uniQure entered into an Amended and Restated Collaboration and License Agreement (the “Amended uniQure Agreement”), which amended and restated the uniQure Agreement, and a separate Collaboration and License Agreement (the “Second uniQure Agreement”). Under these agreements, the Company agreed to transfer incremental rights and services to uniQure in exchange for uniQure eliminating the uniQure Exclusivity Clause and transferring other rights back to the Company. Under the Amended uniQure Agreement, uniQure continues to have an exclusive license to select AAV capsid variants (the “Selected Variants”) in the uniQure Field. uniQure continues to be solely responsible, at its cost, to develop and commercialize the compounds and products containing the Selected Variants. The amended uniQure Agreement eliminated the uniQure Exclusivity Clause in the uniQure Agreement. Furthermore, the contingent payments that the Company was entitled to from uniQure for the achievement of research and development milestones of up to $ 5.0 million for each licensed product selected under the uniQure Agreement were eliminated and sublicense consideration on any future sublicensing arrangements was reduced from the low teens to low thirties percentages to mid-single digit to mid-twenties percentages. Under the Second uniQure Agreement, the parties agreed to research and develop new AAV capsid variants (the “New Variants”) that are not Selected Variants that affect certain targets selected by uniQure (the “uniQure Targets”) in the uniQure Field. The Company is solely responsible, at its cost, for the research of the New Variants. The Company granted uniQure an exclusive license to a certain number of the New Variants (the “uniQure New Variants”) that affect the uniQure Targets. uniQure is solely responsible, at its cost, to develop and commercialize the compounds and products containing the uniQure New Variants that affect the uniQure Targets (the “Licensed Products”). The Company retains all rights to New Variants in the uniQure Field that affect targets other than the uniQure Targets. Under both the Amended uniQure Agreement and the Second uniQure Agreement, uniQure will be required to pay the Company royalties on worldwide annual net sales of Licensed Products at a mid-single digit percentage rate, subject to certain specified reductions. uniQure will also be required to pay the Company sublicensing consideration for sublicensing the Company’s intellectual property rights licensed under the Amended uniQure Agreement or the Second uniQure Agreement to third parties at a rate between the mid-single digit to mid-twenties. The Company has reciprocal obligations, at the same percentage rates as uniQure, to pay uniQure royalties and sublicensing consideration for sublicensing certain intellectual property rights licensed under the Amended uniQure Agreement or the Second uniQure Agreement to third parties. The Company concluded that the Amended uniQure Agreement and the Second uniQure Agreement should be accounted for as one combined contract that should be accounted for as a separate contract from the uniQure Agreement given that the incremental licensed intellectual property rights and research and development services are distinct from the rights and services previously transferred to uniQure under the uniQure Agreement and the transaction price increased by an amount that equals the standalone selling price of the incremental rights and services to be transferred to uniQure under the Amended uniQure Agreement and Second uniQure Agreement. Neither party was required to pay monetary consideration in connection with the execution of the Amended uniQure Agreement or the Second uniQure Agreement or for subsequent performance by the parties under those agreements, notwithstanding the potential future royalty and sublicense consideration described above. The fair value of the non-monetary consideration given by uniQure to the Company, for the intellectual property right is $ 5.1 million. This intellectual property right is considered to be an in-process research and development asset with no alternative future use and, accordingly, was written off as acquired in-process research and development expense in the year ended December 31, 2019. The incremental transaction price described in the paragraph above was recorded as deferred revenue given that the Company identified one single combined performance obligation under ASC 606, which includes the licenses to the New Variants, research services and participation in the joint steering committee (“JSC”). Revenue is being recognized using the input method based on actual costs incurred as a percentage of total budgeted costs as the Company completes its performance obligation. Based on the current estimated timelines, the deferred revenue is expected to be recognized as revenue over approximately one to two years from December 31, 2021. The Company determined the transaction price using the risk adjusted net present value analysis (“rNPV”) methodology to value the elimination of the uniQure exclusivity clause and other material rights received by the Company, including the potential royalties the Company would receive from uniQure. The rNPVs incorporate estimates and assumptions including the number of products the Company and uniQure would develop, the risk-adjusted probability of successfully developing a biopharmaceutical product, the probability that uniQure will develop a product, the research and development costs, the potential worldwide sales and associated commercialization costs, corporate tax rate, and discount rate. During each of the years ended December 31, 2021 and 2020 the Company recognized revenue of $ 0.7 million under the Amended uniQure Agreement and the Second uniQure Agreement. As of December 31, 2021 and 2020, deferred revenue relating to uniQure was $ 3.7 million and $ 4.4 million, respectively. There were no amounts due from uniQure under the uniQure Agreement, Amended uniQure Agreement or Second uniQure Agreement as of December 31, 2021 and 2020. As of December 31, 2021 and 2020, the aggregate amount of the transaction price allocated to the remaining performance obligation was $ 3.7 million and $ 4.4 million, respectively. CRF In November 2015, the Company entered into a research funding and collaboration agreement (the “CRF Agreement”) with the Choroideremia Research Foundation (“CRF”), a non-profit organization dedicated to finding a cure for choroideremia, a rare inherited disorder that causes progressive vision loss, ultimately leading to complete blindness. The goal of the CRF Agreement is for CRF to contribute funding to help with the advancement of the Company’s choroideremia research program. The Company is responsible for all decision making and execution of any and all of the related activities to be completed in its sole discretion. The initial term of the CRF Research Plan is two years . The agreement includes contribution by the Company to CRF of up to $ 2.5 million upon certain development or approval milestones. The overall arrangement has automatic extensions of up to three additional years. As of December 31, 2021, no milestones have been achieved. Revenue was fully recognized for this agreement in the year ended December 31, 2017. There was no deferred revenue relating to the CRF Agreement as of December 31, 2021 and 2020. No amount was due from or to CRF under the CRF Agreement as of December 31, 2021 and 2020. Roche In November 2017, the Company entered into a collaboration and license agreement with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. (together, “Roche”) to discover and develop products containing optimized next generation AAV Vectors focused on ophthalmological diseases and disorders excluding select criteria (the “2017 Roche Agreement”). The Company and Roche both have the ability to nominate products to discover, develop and commercialize. At the effective date of the 2017 Roche Agreement, choroideremia was designated a Roche product class and the 4D-110 product was licensed to Roche. The Company was responsible for conducting research and development services prior to pivotal clinical studies, and Roche was responsible for conducting subsequent development and commercialization activities. In addition, Roche agreed to pay for research and development services at the agreed upon full-time employee rate for work performed for choroideremia under the 2017 Roche Agreement, and certain external costs, except for the costs associated with the manufacturing work for choroideremia. Under the 2017 Roche Agreement, Roche had an option, exercisable prior to pivotal clinical studies, to license 4D-125 (for the treatment of XLRP), which was not exercised. Pursuant to the 2017 Roche Agreement, the Company received a non-refundable upfront payment of $ 21.0 million as consideration. In addition, the Company was entitled to contingent payments including (i) $ 1.0 million for each Roche nominated product beyond the first three, (ii) up to $ 30.0 million upon exercise of the option to convert a product the Company nominated and developed prior to pivotal clinical studies, (iii) development milestone payments of up to $ 223.0 million, of which $ 86.0 million related to choroideremia and the rest related to other licensed products; and (iv) sales-based milestones of up to $ 123.0 million in connection with licensed products. Through December 31, 2021, the Company received $ 10.0 million for development milestone payments that are non-refundable. The 2017 Roche Agreement also included provisions that entitled the Company to receive royalty payments ranging from the mid-single digits to the mid-teens for the net sales of the licensed products, in each case subject to the reductions in accordance with the terms of the agreement. The Company identified a single combined performance obligation for the license, research services and participation in the JSC and concluded that Roche’s option did not represent a material right. The Company’s contract with Roche did not include a significant financing component. The Company further concluded that the transaction price should not include the variable consideration related to development milestones as they were considered to be constrained as it was probable that the inclusion of such variable consideration could result in a significant reversal of cumulative revenue in the future. The Company excluded any consideration related to sales-based milestones, including royalties, which would be recognized when the related sales occur. The transaction price and estimated period of performance are re-evaluated at each reporting period and adjusted as needed to reflect changes in the scope of the project, reimbursable expenses and other conditions affecting variable consideration. In June 2021, the Company received from Roche notice of termination without cause of the 2017 Roche Agreement. The licenses granted by the Company to Roche under the 2017 Roche Agreement terminated in their entirety on September 16, 2021. Therefore, as of September 16, 2021, Roche no longer has a license for 4D-110 for the treatment of choroideremia or any other product class, and the Company is no longer entitled to receive any further milestones or royalties from Roche. In July 2021, the Company notified Roche of the Company’s election to continue development of choroideremia. As a result, in accordance with the terms of the agreement, all rights to 4D-110 data and intellectual property generated under the collaboration reverted to the Company. Under the 2017 Roche Agreement, if the Company is required to initiate a new Phase 1/2a clinical trial for 4D-110, the Company would not owe any royalty to Roche on net sales of 4D-110. If, however, the Company moves directly to a pivotal trial of 4D-110, without first being required to conduct a new Phase 1/2a clinical trial, the Company must pay Roche a mid-single digits percentage royalty on the net sales of 4D-110. As a result of the termination of the 2017 Roche Agreement, an adjustment of $ 8.7 million was made to the transaction price to reflect the decrease in reimbursable costs for the year ended December 31, 2021. The decrease in the transaction price and total budgeted costs for the remaining performance obligation resulted in an $ 11.1 million increase in revenue recognized in the year ended December 31, 2021 related to the performance obligation partially satisfied prior to January 1, 2021. For the year ended December 31, 2020, an adjustment of $ 17.0 million was made to the transaction price to reflect an increase of $ 7.0 million in the scope of the project and expected reimbursable costs and the addition of $ 10.0 million of variable consideration as the uncertainty associated with two development milestones was resolved. The adjustments to the transaction price and total budgeted costs in 2020 resulted in a $ 4.6 million increase in revenue recognized in the year ended December 31, 2020 related to performance obligations partially satisfied in periods prior to January 1, 2020. During the years ended December 31, 2021 and 2020, the Company recognized revenue of $ 17.2 million and $ 12.9 million from Roche, respectively. As of December 31, 2021 and 2020, deferred revenue relating to the Roche Agreement was $ 0.0 million and $ 14.3 million, respectively. Accounts receivable from Roche under this agreement as of December 31, 2021 and 2020 was $ 0.0 million and $ 1.1 million, respectively. As of December 31, 2021 and 2020, the aggregate amount of the transaction price allocated to the remaining performance obligation was $ 0.0 million and $ 25.8 million, respectively. CFF In September 2016, the Company entered into an award agreement for the Optimized Adeno-Associated Virus for Lung Epithelia Gene Delivery Development Program with CFF, a non-profit organization dedicated to finding a cure for cystic fibrosis, an inherited disorder that causes disease in the pulmonary airways leading to morbidity and mortality. Under this agreement, CFF contributes funding to help advance the Company’s CF research program. The agreement was subsequently amended in September 2017, August 2018 and February 2021 (all four agreements are collectively referred to as the “CFF Agreement”). The total amount of the award under the CFF Agreement is $ 3.5 million. As of December 31, 2021 and 2020, the Company achieved milestones totaling $ 1.8 million and $ 1.3 million under the CFF Agreement, respectively. The remaining award amount will be paid by CFF based on achievement of certain development milestones by the Company. The Company expects to make payments to CFF equal to six times the actual award received by the Company in three installments within the first four years of the first commercial sale of a product developed under this agreement. The Company also has agreed to make future sales-based milestone payments to CFF of up to three times the actual award received upon achieving specified commercialization milestones with respect to the first of any product developed utilizing any compound covered under the CFF Agreement. The CFF Agreement also requires the Company to pay to CFF royalties of a mid-single digit percentage, up to six times the actual award received, on any amounts received by the Company from the sale, license or transfer to a third-party of rights in the technology developed as a result of this collaboration. Any such royalty payments shall be credited against the payments owed by the Company upon first commercial sale. In the event of a change of control of the Company, CFF will receive certain payments, depending on the timing of the change of control and the size of the transaction. To date, the Company has not developed a commercial product in connection with the CFF Agreement, and it has not licensed, sold or otherwise transferred to another party the product developed under the CFF Agreement or the underlying technology. If at any time prior to the first commercial sale of a product developed as a result of the CFF Agreement, the Company ceases to use commercially reasonable efforts to develop or commercialize any product under the CFF Agreement for a continuous period of 180 consecutive days and fails to present a reasonable plan to resume commercially reasonable efforts, the Company will grant to CFF an irrevocable, exclusive worldwide interruption license under all of the Company’s interest in the research plan technology to exploit such product. Any third-party license granted by the Company shall be subject to such interruption license. Under ASC 606, the Company identified one performance obligation within the CFF grant agreement for research activities. The Company’s contract with CFF does not include a significant financing component. The Company concluded that the transaction price should not include the variable consideration related to future research milestones as they were considered to be constrained as it is probable that the inclusion of such variable consideration could result in a significant reversal of cumulative revenue in the future. The Company re-evaluates the transaction price and estimated period of performance at each reporting period. For the year ended December 31, 2021, an adjustment of $ 0.5 million was made to the transaction price to reflect the achievement of the fourth milestone related to demonstrating GFP expression and determining a cell specific expression profile in human and NHP upper airway ALI cultures following apical transduction under the CFF Agreement. During the years ended December 31, 2021 and 2020, the Company recognized revenue of $ 0.1 million and $( 0.1 ) million, respectively. As of December 31, 2021 and 2020, deferred revenue relating to the CFF Agreement was $ 1.4 million and $ 1.1 million, respectively. Accounts receivable from CFF under the CFF Agreement as of December 31, 2021 and 2020 was $ 0.0 million and $ 0.4 million, respectively. As of December 31, 2021 and 2020, the aggregate amount of the transaction price allocated to the remaining performance obligation was $ 1.4 million and $ 1.1 million, respectively. Based on current timelines, the deferred revenue is expected to be recognized as revenue over the next three to four years as the Company performs research services through the completion of IND-enabling studies. The obligation to make payments to CFF upon a change of control meets the definition of an embedded derivative that is required to be bifurcated and separately accounted for as a derivative liability. See Note 16 for further discussion of the embedded derivative. |
License Arrangements
License Arrangements | 12 Months Ended |
Dec. 31, 2021 | |
License Arrangements [Abstract] | |
License Arrangements | 8. License Arrangements The Company has exclusive, worldwide license agreements (the “UC Agreements”) with the Regents of the University of California (the “UC Regents”) relating to the use of certain patents and intellectual property surrounding its core technologies, including Therapeutic Vector Evolution. Pursuant to each of the UC Agreements executed prior to January 2019, the Company was obligated to pay a (i) non-refundable license fee of $ 5,000 upon execution, (ii) a non-refundable license fee of $ 5,000 each year thereafter, until sales of a licensed product are made and royalties are paid to the UC Regents, (iii) reimbursement of domestic and foreign patent filing, prosecution and maintenance fees, and (iv) either $ 50,000 or issuance of a 3 % equity interest in the Company upon the closing of the first qualified financing at the option of the UC Regents. The Company’s first qualified financing occurred in 2015 and at the election of the UC Regents, the Company issued the UC Regents in January 2016 an amount of common stock equal to 6 % of the equity interests in the Company pursuant to the applicable clause in each of the UC Agreements. Pursuant to an agreement with the UC Regents executed in January 2019 the Company paid a non-refundable license fee of $ 50,000 to the UC Regents upon execution of the agreement. The Company is obligated to pay a non-refundable license fee of $ 5,000 on the one-year anniversary of the contract effective date and each year thereafter, until sales of a licensed product are made and royalties are paid to the UC Regents. In addition, the Company is obligated to make certain contingent payments including (i) development milestones up to $ 3.1 million, (ii) low single digit royalties on the net sales of its developed products that consists of a minimum annual royalty of up to $ 0.1 million per year for the term of the Agreement beginning in the first calendar year after the year in which net sales first occurred, and (iii) sublicense consideration in the mid-teens to the mid-twenties-range on any future sublicensing arrangements the Company may enter into with third-party licensees. In July 2021, the Company entered into an exclusive license agreement with the UC Regents and the Trustees of the University Pennsylvania to license intellectual property related to certain vectors. In July 2021, the Company paid a non-refundable license fee of $ 100,000 to the UC Regents upon execution of the agreement. The Company is obligated to pay a non-refundable license maintenance fee of $ 10,000 on the one-year anniversary of the contract effective date and each year thereafter, except years for which the Company has paid royalties on the net sales of a licensed product. In addition, the Company is obligated to make certain contingent payments including (i) development milestones up to $ 3.9 million, (ii) low single digit percentage rate royalties on the net sales of its licensed products that consists of a minimum annual royalty of up to $ 0.1 million per year for the term of the Agreement beginning in the first calendar year after the year in which net sales first occurred and (iii) sublicense consideration in the mid-single digits to the low twenties percentage rate range on any future sublicensing arrangements the Company may enter into with third-party licensees. During the years ended December 31, 2021 and 2020, the Company incurred expenses of $ 0.3 million and $ 0.2 million, respectively, under the provisions of the outstanding license arrangements. |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2021 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 9. Commitments and Contingencies Operating Lease Commitments Prior to the adoption of ASC 842, the Company recognized rent expense for its operating leases on a straight-line basis over the noncancelable lease term and recorded the difference between rent payments and the straight-line rent expense was recorded as a deferred rent liability. As of December 31, 2020, the Company performed an evaluation of its other contracts with customers and suppliers in accordance with ASC 842 and determined that, except for the leases described below, none of the Company’s contracts contain a material lease. 5980 Horton Street Building Lease In May 2015, the Company executed a lease agreement for office and laboratory space in Emeryville, California. In January 2016, the Company executed the first amendment to the lease agreement for additional rentable office and laboratory space which extended the lease term to March 31, 2023 and updated the option to renew the lease at market rent from the prior additional term of three years to five years . In October 2018, the Company executed a second amendment to extend the lease to September 2026 . Additionally, the second amendment provided a tenant improvement allowance of $ 0.2 million, which was paid to the Company in November 2018. The 5980 Horton Street Building Lease is considered an operating lease under ASC 842 as it does not meet the criteria of a finance lease. As of December 31, 2021, the operating lease right-of-use asset and operating lease liability was $ 1.9 million and $ 2.3 million, respectively. The discount rate used to determine the lease liability was 7.5 %. 5858 Horton Street Building Lease and Expansion In October 2018, the Company executed a second lease agreement for additional office and laboratory space in Emeryville, California. The new lease has an initial term of 87 months beginning on the rent commencement date with the option to renew the lease for one additional term of five years at market rent. The Company did not have to pay rent until October 2019. This lease agreement also provided for a tenant improvement allowance of $ 0.4 million, which was paid to the Company in December 2019. The Company amortizes the tenant improvement allowance on a straight-line basis over the remaining term of the lease as a reduction of rent expense. In May 2019, the Company amended the second lease agreement executed in October 2018 to add additional office and laboratory space (the "Expansion"). The amendment extended the term of the lease to December 31, 2029. The Company did not have to pay rent until December 2019. The lease agreement also included a tenant improvement allowance, which was increased to $ 2.3 million in February 2021 pursuant to a second amendment to the second lease agreement. In November 2021, the Company received the first installment of this tenant improvement allowance of $ 0.6 million. The tenant improvement allowance funds improvements to the additional office and laboratory space, which the Company has determined to be lessee owned. The allowance is treated as a reduction of lease payments used to measure the lease liability. The 5858 Horton Street Building Lease and Expansion is considered an operating lease under ASC 842 as it does not meet the criteria of a finance lease. As of December 31, 2021, the operating lease right-of-use asset and operating lease liability was $ 12.6 million and $ 14.2 million, respectively. The discount rate used to determine the lease liability was 7.8 %. Total operating lease expense for the year ended December 31, 2021 was $ 2.6 million. Cash paid for amounts included in the measurement of lease liabilities was $ 2.3 million for the year ended December 31, 2021. Neither of the leases include a general option for the Company to terminate the leases. The following table summarizes the components of lease expense for the year ended December 31, 2021, which are included in operating expenses in the Company’s statements of operations (in thousands): Operating lease cost $ 2,631 Variable lease cost 914 Total $ 3,545 Variable lease payments include amounts relating to common area maintenance and are recognized in the statements of operations as incurred. The following table summarizes supplemental information related to operating leases for the year ended December 31, 2021: Weighted-average remaining lease term (in years): 7.6 Weighted-average discount rate: 7.8 % The following table summarizes the maturities of lease liabilities as of December 31, 2021 (in thousands): 2022 $ 1,230 2023 3,058 2024 3,149 2025 3,244 2026 3,135 Thereafter 8,686 Total future minimum lease payments 22,502 Less: Amount representing interest ( 6,054 ) Present value of future minimum lease payments 16,448 Less: Current portion of operating lease liabilities 1,231 Long-term portion of operating lease liabilities $ 15,217 Indemnification Agreements In the ordinary course of business, the Company enters into agreements that may include indemnification provisions, such as with vendors and other parties. Pursuant to such agreements, the Company may indemnify, hold harmless and defend an indemnified party for losses suffered or incurred by the indemnified party. Some of the provisions will limit losses to those arising from third-party actions. In some cases, the indemnification will continue after the termination of the agreement. The maximum potential amount of future payments the Company could be required to make under these provisions is not determinable. The Company has never incurred material costs to defend lawsuits or settle claims related to these indemnification provisions. The Company has also entered into indemnification agreements with its directors and officers that 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 to the fullest extent permitted by Delaware corporate law. The Company currently maintains directors’ and officers’ liability insurance that would generally enable it to recover a portion of any future amounts paid. The Company believes the estimated fair value of its indemnification agreements in excess of applicable insurance coverage is not material. Legal Proceedings From time to time, the Company may become involved in legal proceedings arising from the ordinary course of its business. If applicable, the Company records a legal liability when it believes that it is both probable that a liability may be imputed, and the amount of the liability can be reasonably estimated. Significant judgment by the Company is required to determine both probability and the estimated amount. There are no material legal proceedings outstanding at December 31, 2021. See Note 14 for details of a settlement with a former employee in April 2021. |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2021 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 10. Income Taxes The Company did no t record any income tax expense during the years ended December 31, 2021 and 2020. The Company has a net operating loss and has provided a valuation allowance against net deferred tax assets due to uncertainties regarding the Company’s ability to realize these assets. All losses before income taxes arose in the United States. The effective tax rate of the Company’s income tax expense (benefit) differs from the federal statutory rate as follows: December 31, 2021 2020 Federal statutory income tax rate 21.0 % 21.0 % Research tax credit 2.1 % 2.8 % Permanent differences 0.4 % ( 1.7 )% Valuation allowance ( 23.5 )% ( 22.1 )% Provision for income taxes 0.0 % 0.0 % The tax effects of temporary differences that give rise to significant components of the deferred taxes are as follows (in thousands): December 31, 2021 2020 Deferred Tax Assets Net operating loss carryforwards $ 37,295 $ 21,132 Other accrued liabilities 937 422 Deferred revenue 993 3,111 Research tax credits 8,078 6,018 Stock-based compensation expense 2,618 1,151 Intangible asset basis 907 977 Operating lease liabilities 3,460 — Total deferred tax assets $ 54,288 $ 32,811 Deferred Tax Liabilities Operating lease right-of-use assets $ ( 3,064 ) $ — Prepaid expenses ( 992 ) — Total deferred tax liabilities $ ( 4,056 ) $ — Less: valuation allowance ( 50,232 ) ( 32,811 ) Total net deferred tax $ — $ — ASC 740, Income Taxes, requires that the tax benefit of net operating losses, temporary differences and credit carryforwards be recorded as an asset to the extent that management assesses that realization is “more likely than not.” Realization of the future tax benefits is dependent on the Company’s ability to generate sufficient taxable income within the carryforward period. Because of the Company’s recent history of operating losses, management believes that recognition of the deferred tax assets arising from future tax benefits is currently not likely to be realized and, accordingly, has provided a full valuation allowance. The valuation allowance increased by $ 17.4 million during 2021 and $ 12.5 million during the year ended December 31, 2020. The increase in the valuation allowance for each of the years ended December 31, 2021 and 2020 was primarily driven by net losses incurred, stock-based compensation expense and tax credits generated within the U.S. The Company had net operating loss (“NOL”) carryforwards of $ 177.5 million and $ 100.6 million as of December 31, 2021 and 2020, respectively, available to reduce future taxable income, if any, for federal income tax purposes. $ 9.5 million of the federal NOL carryforwards expire in 2037 and the remaining $ 168.0 million carryforward indefinitely. As of December 31, 2021 and 2020, the Company had federal research and development credit carryforwards of $ 8.6 million and $ 5.7 million, respectively, and state research and development credit carryforwards of $ 5.9 million and $ 4.3 million, respectively, available to reduce future taxable income, if any, for federal and California state income tax purposes, respectively. The federal credit carryforwards begin expiring in 2035 and the state credits carryforward indefinitely . Utilization of the NOL carryforwards and research credit carryforwards may be subject to an annual limitation due to the ownership percentage change limitations provided by the Internal Revenue Code and similar state provisions. Annual limitations may result in the expiration of the NOL and tax credit carryforwards before they are utilized. The Company has experienced ownership changes in the past as a result of its Series B redeemable convertible preferred stock financing. As a result of the ownership changes, some of the tax attribute carryforwards may be permanently limited as they will expire unused and such amounts are excluded from our NOLs as of December 31, 2021. Subsequent ownership changes may result in additional limitations. The reconciliation of the beginning and ending unrecognized tax benefits amounts is as follows (in thousands): Unrecognized Balance as of December 31, 2019 $ 1,566 Additions for current year tax positions 1,729 Additions for tax positions of prior years 72 Balance as of December 31, 2020 3,367 Additions for current year tax positions 2,219 Additions for tax positions of prior years — Balance as of December 31, 2021 $ 5,586 The entire amount of the unrecognized tax benefits would not impact the Company’s effective tax rate if recognized. During the years ended December 31, 2021 and 2020, the Company did no t recognize accrued interest and penalties related to unrecognized tax benefits. The Company does not anticipate that the amount of existing unrecognized tax benefits will materially increase or decrease during the next 12 months. The Company files income tax returns in the U.S. federal and California tax jurisdictions. In general, the Company is no longer subject to tax examination by the Internal Revenue Service or state taxing authorities for years before 2017. Although the federal and state statutes are closed for purposes of assessing additional income tax in those prior years, the taxing authorities may still make adjustments to the NOL and credit carryforwards used in open years. Therefore, the tax statutes should be considered open as it relates to the NOL and credit carryforwards used in open years. The Company has no ongoing income tax examinations by tax authorities at this time. In March 2020, the Coronavirus Aid, Relief, and Economic Security Act (the “CARES Act”) was signed into law. The CARES Act changed certain provisions of the Tax Act. Under the CARES Act, NOLs arising in taxable years beginning after December 31, 2017 and before January 1, 2021 may be carried back to each of the five taxable years preceding the tax year of such loss, but NOLs arising in taxable years beginning after December 31, 2020 may not be carried back. In addition, the CARES Act eliminated the limitation on the deduction of NOLs to 80% of current year taxable income for taxable years beginning before January 1, 2021 and increased the amount of interest expense that may be deducted to 50% of adjusted taxable income for taxable years beginning in 2019 or 2020. The Company evaluated the impact of the CARES act on its tax provision and concluded that there was not a material impact. On December 21, 2020, the U.S. president signed into law the Consolidated Appropriations Act, 2021 which includes further COVID-19 economic relief and extension of certain expiring tax provisions. The relief package includes a tax provision clarifying that businesses with forgiven PPP loans can deduct regular business expenses that are paid for with the loan proceeds. Additional pandemic relief tax measures include an expansion of the employee retention credit, enhanced charitable contribution deductions, and a temporary full deduction for business expenses for food and beverages provided by a restaurant. The Company evaluated the impact of the Consolidated Appropriations Act, 2021 on its tax provision and concluded that there was not a material impact. On March 11, 2021, the American Rescue Plan Act was signed into law and contained provisions relating to extending the Employee Retention Tax Credit, additional funding for the PPP loan and Economic Injury Disaster Loans, as well as expanding the covered employees under Section 162(m) for tax years beginning after December 31, 2026. The Company does not expect the American Rescue Plan will have a material impact to its current income tax provision. On November 15, 2021, the Infrastructure Investment and Jobs Act was signed into law and contained several tax provisions including changes to the Employee Retention Tax Credit after September 30, 2021. The Company does not expect the Infrastructure Investment and Jobs Act will have a material impact to its income tax provision. |
Redeemable Convertible Preferre
Redeemable Convertible Preferred Stock | 12 Months Ended |
Dec. 31, 2021 | |
Temporary Equity Disclosure [Abstract] | |
Redeemable Convertible Preferred Stock | 11. Redeemable Convertible Preferred Stock As of December 31, 2019, redeemable convertible preferred stock consisted of the following (in thousands, except per share and share amounts): Shares Original Shares Issued Liquidation Proceeds Net of Series A 909,312 $ 7.70 909,312 $ 7,001 $ 6,960 Series A-1 1,311,687 $ 8.84 1,311,687 11,595 11,548 Series B 5,154,639 $ 17.46 5,154,632 90,000 84,472 Total 7,375,638 7,375,631 $ 108,596 $ 102,980 In April and June of 2020, the Company issued a total of 4,200,353 shares of Series C redeemable convertible preferred stock at $ 18.00 per share for net proceeds of $ 72.5 million. Upon the closing of the Company's IPO on December 15, 2020, all outstanding redeemable convertible preferred stock automatically converted into shares of common stock and the related carrying value was reclassified to common stock and additional paid-in capital. There were no shares of redeemable convertible preferred stock outstanding as of December 31, 2021 and 2020. Funding Agreement with CFF — In April 2020, CFF made a $ 10.0 million investment in the Company's Series C redeemable convertible preferred stock financing. In return for the investment, CFF received shares of Series C redeemable convertible preferred stock, and the Company and CFF entered into a Funding Agreement ("the Funding Agreement"). Pursuant to the terms of the Funding Agreement, except in the event of a technical failure, the $ 10.0 million received from CFF will be used to advance the development program for 4D-710, the Company's lead product in cystic fibrosis, or any other therapeutic approved by the Program Advisory Group ("PAG") to alleviate pulmonary complications of cystic fibrosis ("the Funding Agreement Product"). CFF committed to provide an additional $ 4.0 million of funding upon acceptance of an Investigational New Drug ("IND") application or its equivalent to allow for human testing of the Funding Agreement Product ("Acceptance"). The IND was cleared by the U.S. Food and Drug Administration in October 2021, and CFF made the additional investment of $ 4.0 million in cash for the issuance of 125,715 shares of the Company's common stock to CFF. Except in the event of a technical failure, the Company is committed to providing an amount equal to the funding provided by CFF to be used solely to advance the Funding Agreement Product. A technical failure is defined as a determination by the Company, after consultation with and approval of the PAG that (i) the Funding Agreement Product has failed to reach its intended endpoints due to safety issues, lack of sufficient transgene expression and/or efficacy, each despite commercially reasonable efforts and (iii) the exercise of further commercially reasonable efforts is unlikely to correct such failure. Under the terms of the Funding Agreement, neither the $ 10.0 million investment in the Series C redeemable convertible preferred stock, which converted to common stock as of December 31, 2020, nor the $ 4.0 million of funding upon Acceptance are restricted as to withdrawal or usage. |
Common Stock
Common Stock | 12 Months Ended |
Dec. 31, 2021 | |
Equity [Abstract] | |
Common Stock | 12. Common Stock As of each of December 31, 2021 and 2020, the Company’s certificate of incorporation authorized the Company to issue 300,000,000 shares of common stock, at the par value of $ 0.0001 per share. The holder of each share of common stock is entitled to one vote per share . Common stockholders are entitled to dividends if and when declared by the board of directors, subject to the prior rights of the redeemable preferred stockholders. As of December 31, 2021 and 2020, no dividends on common stock had been declared by the board of directors. As of December 31, 2021 and 2020, the Company has reserved common stock, on an as-converted basis, for future issuance as follows: December 31, 2021 2020 Issuance of common stock under the 2020 Equity Incentive Plan 2,690,097 2,606,546 Issuance of common stock under the Employee Stock Share Purchase Plan 498,747 252,337 Exercise of options issued and outstanding 3,881,274 3,194,113 Exercise of common stock warrants 53,669 98,669 Total common stock reserved 7,123,787 6,151,665 |
Stock-based Compensation
Stock-based Compensation | 12 Months Ended |
Dec. 31, 2021 | |
Share-based Payment Arrangement [Abstract] | |
Stock-based Compensation | 13. Stock-based Compensation 2020 Incentive Award Plan In December 2020, the Company adopted the 2020 Incentive Award Plan (“2020 Plan”), which became effective on December 10, 2020. The 2020 Plan initially reserved 2,606,546 shares of common stock for the issuance of stock options, stock appreciation rights, restricted stock awards, restricted stock units, performance bonus awards, performance stock units, dividend equivalents or other stock or cash based award granted to employees, directors and consultants of the Company. The number of shares reserved for future issuance under the 2020 Plan will increase annually on the first day of each fiscal year beginning in 2021 and ending in 2030 by the lesser of (i) 5 % of the shares of common stock outstanding (on an as converted basis) on the last day of the immediately preceding fiscal year and (ii) such number of shares of common stock as determined by the Company's board of directors, provided, however, no more than 18,000,000 shares of the Company's common stock may be issued upon the exercise of incentive stock options. In 2021, an additional 1,334,099 shares of common stock became available for issuance under the 2020 Plan, as a result of the operation of the automatic annual increase provision. All options are exercisable over a period not to exceed the contractual term of ten years from the date the stock options were issued. As of December 31, 2021, there were 2,690,097 shares available for grant under the 2020 Plan. Following the effectiveness of the 2020 Plan, the Company will not make any further grants under the 2015 Equity Incentive Plan (the “2015 Plan”). However, the 2015 Plan continues to govern the terms of options that remain outstanding under the 2015 Plan. 2015 Equity Incentive Plan The 2015 Plan provided for grants of stock options, stock appreciation rights, restricted stock and restricted stock unit awards to employees, directors and consultants of the Company. As of December 31, 2021, options to purchase 2,519,217 shares of common stock were outstanding under the 2015 Plan. All options are exercisable over a period not to exceed the contractual term of ten years from the date the stock options were issued and are granted at prices not less than the estimated fair market value of the Company’s common stock on the grant date as determined by the board of directors. If an individual owns stock representing more than 10 % of the Company’s outstanding shares, the exercise price of each share shall be at least 110 % of the fair market value on the date of grant. No additional grants will be made under the 2015 Plan, and all outstanding grants under the 2015 Plan that are repurchased, forfeited, expire or are cancelled are returned back to the 2015 Plan and are not available for grant under the 2020 Plan. Employee Stock Purchase Plan In December 2020, the Company adopted the 2020 Employee Stock Purchase Plan (the “2020 ESPP”). Under the 2020 ESPP, 252,337 shares of the Company's common stock were initially reserved for employee purchases of the Company's common stock under terms and provisions established by the Company's board of directors and approved by the Company's stockholders. The number of shares reserved for future issuance under the 2020 ESPP will increase annually on the first day of each fiscal year beginning in 2021 and ending in 2030 by the lesser of (i) 1 % of the shares of common stock outstanding (on an as converted basis) on the last day of the immediately preceding fiscal year and (ii) such number of shares of common stock as determined by the Company's board of directors, provided, however, no more than 15,000,000 shares the Company's common stock may be issued under the 2020 ESPP. On March 28, 2022, an additional 25,000 shares of common stock became available for future issuance under the 2020 ESPP, as a result of the operation of this automatic annual increase provision. Under the 2020 ESPP the Company's employees may purchase common stock through payroll deductions at a price equal to 85 % of the lower of the fair market value of the stock at the beginning of the offering period or at the end of each applicable purchase period. The 2020 ESPP provides for a series of overlapping 24-month offering periods comprising four six-month purchase periods. The initial offering period under the 2020 ESPP is longer than 24 months, commencing February 15, 2021 and ending on May 14, 2023. Contributions under the 2020 ESPP are limited to a maximum of 15 % of an employee's eligible compensation. Stock Options The following table summarizes the stock options activity for the year ended December 31, 2021: Options Outstanding Number of Number of Weighted- Weighted- Aggregate Balances at December 31, 2020 2,606,546 3,194,113 $ 11.05 8.46 $ 97,105 Options authorized 1,334,099 — Options granted ( 1,566,150 ) 1,566,150 34.83 Options exercised — ( 563,387 ) 6.26 Options expired 4,324 ( 4,324 ) 36.33 Options forfeited 311,278 ( 311,278 ) 29.17 Balances at December 31, 2021 2,690,097 3,881,274 $ 19.86 $ 7.97 $ 24,666 Shares exercisable, December 31, 1,592,139 $ 12.75 $ 7.07 $ 17,280 Shares vested and expected to 3,881,274 $ 19.86 $ 7.97 $ 24,666 The following table is a summary of stock compensation expense for employees and nonemployees by function (in thousands): Year Ended December 31, 2021 2020 Research and development $ 6,847 $ 2,670 General and administrative 6,952 2,314 Total stock-based compensation expense $ 13,799 $ 4,984 During the years ended December 31, 2021 and 2020 the Company granted 1,521,150 and 1,267,743 stock options to employees with a weighted-average grant date fair value of $ 34.46 and $ 11.84 per share, respectively and 45,000 and 54,000 stock options to nonemployees with a weighted-average grant date fair value of $ 47.42 and $ 11.05 per share, respectively. The total fair value of options vested during the years ended December 31, 2021 and 2020 was $ 13.8 million and $ 5.0 million, respectively. As of December 31, 2021, the unrecognized stock-based compensation expense of unvested options was $ 37.7 million and is expected to be recognized over a weighted-average period of 2.7 years. Stock-based compensation expense recorded for employee options was $ 13.3 million and $ 4.6 million for the years ended December 31, 2021 and 2020, respectively. Stock-based compensation expense recorded for nonemployee consultants was $ 0.5 million and $ 0.4 million for the years ended December 31, 2021 and 2020, respectively. Prior to the Company’s IPO, the fair value of the shares of common stock underlying the stock options was estimated by the board of directors at various dates considering the Company’s most recently available third-party valuations of common stock as well as a number of objective and subjective factors including valuation of comparable companies, sales of redeemable convertible preferred stock, operating and financial performance and general and industry specific economic outlook, amongst other factors. The fair value was determined in accordance with the guidance provided by the American Institute of Certified Public Accountants’ Practice Guide, Valuation of Privately-Held-Company Equity Securities Issued as Compensation. Subsequent to the Company’s IPO, the fair value of the Company’s common stock is determined based on its closing market price on the respective grant date. The Company estimates the fair value of employee and nonemployee stock options using the Black-Scholes option pricing model. The fair value of employee and nonemployee options is recognized on a straight-line basis over the requisite service period of the awards. The fair value of the Company's stock options was estimated using the following assumptions for the years ended December 31, 2021 and 2020. Year Ended December 31, 2021 Year Ended December 31, 2020 Employee Nonemployee Employee Nonemployee Expected term 5.8 – 6.3 years 6.3 years 5.5 – 6.3 years 6.0 years Expected volatility 80.0 % – 84.3 % 83.5 % 82.1 % – 83.8 % 82.1 % – 83.1 % Risk-free interest rate 0.6 % – 1.3 % 0.6 % 0.4 % – 0.7 % 0.4 % – 0.7 % Expected dividend yield 0 % 0 % 0 % 0 % Expected Term . The expected term for employee options is calculated using the simplified method as the Company does not have sufficient historical information to provide a basis for estimate. The simplified method is based on the average of the vesting tranches and the contractual life of each grant. The expected term for nonemployee options is the contractual term of the options. Expected Volatility . The expected volatility was estimated based on a study of publicly traded peer companies as the Company did not have sufficient trading history for its common stock. The Company selected the peer group based on similarities in industry, stage of development, size and financial leverage with the Company’s principal business operations. For each grant, the Company measured historical volatility over a period equivalent to the expected term. Risk-free Interest Rate . The risk-free interest rate is based on the yield available on U.S. Treasury zero-coupon issues whose term is similar in duration to the expected term of the respective stock option. Expected Dividend Yield . The Company has not paid and does not anticipate paying any dividends on its common stock in the future. Accordingly, the Company has estimated the dividend yield to be zero . |
Common Stock Warrants
Common Stock Warrants | 12 Months Ended |
Dec. 31, 2021 | |
Equity [Abstract] | |
Common Stock Warrants | 14. Common Stock Warrants In 2016, the Company issued a warrant for 45,000 shares of the Company’s common stock to a service provider with an exercise price of $ 1.14 per share, of which 15,000 warrant shares become exercisable upon completion of an offering of securities in a private placement by the Company with net proceeds in excess of $ 25.0 million and 30,000 warrant shares become exercisable upon completion of an IPO by the Company. The warrant expires in 2023 . The fair value of the warrant was determined at the issuance date using the Black-Scholes option pricing model. 15,000 of these warrant shares became exercisable upon the completion of the Series B financing in 2018 and 30,000 of these warrants became exercisable upon completion of the IPO in 2020. 45,000 shares of this warrant were exercised in October 2021. In May 2018, the Company issued a warrant for 23,669 shares of the Company’s common stock to a service provider with an exercise price of $ 3.19 per share. The fair value of the warrant was determined at the issuance date using the Black-Scholes option pricing model, expires in 2025 and upon issuance was fully vested. In December 2020, the Company issued a warrant for 30,000 shares of the Company’s common stock to a service provider with an exercise price of $ 18.00 per share. This warrant vests over a period of four years and expires in 2027 . The fair value of the warrant was determined at the issuance date using the Black-Scholes option pricing model. In April 2021, the Company issued a warrant for 40,000 shares of the Company’s common stock to a former employee with an exercise price of $ 9.41 per share as a result of a settlement agreement with such former employee. The warrant was exercised in May 2021 . The Company previously recorded an accrual within accrued and other current liabilities within the balance sheet at December 31, 2020 to accrue for the settlement and the related expense was recognized in general and administrative expenses for the year ended December 31, 2020. Upon the issuance of the warrant in April 2021 in connection with the settlement, $ 1.1 million was reclassified from accrued and other current liabilities to additional paid-in capital within the balance sheet as of December 31, 2021. The Company recorded less than $ 0.1 million expense for the above warrants, within operating expenses in the statements of operations during each of the years ended December 31, 2021 and 2020. |
Net Loss Per Share, Basic and D
Net Loss Per Share, Basic and Diluted | 12 Months Ended |
Dec. 31, 2021 | |
Earnings Per Share [Abstract] | |
Net Loss Per Share, Basic and Diluted | 15. Net Loss Per Share, Basic and Diluted The following table sets forth the computation of basic and diluted net loss per share (in thousands, except share and per share data): Year Ended December 31, 2021 2020 Numerator Net loss attributable to common stockholders $ ( 71,317 ) $ ( 56,693 ) Denominator Weighted-average shares outstanding 27,730,420 6,430,555 Weighted-average shares outstanding used in computing net loss per share, basic and diluted 27,730,420 6,430,555 Net loss per share, basic and diluted $ ( 2.57 ) $ ( 8.82 ) The following outstanding shares of potentially dilutive securities were excluded from the computation of diluted net loss per share for the periods presented because including them would have been antidilutive: December 31, 2021 2020 Options issued and outstanding 3,881,274 3,194,113 Common stock warrants 53,669 98,669 Total 3,934,943 3,292,782 |
Derivative Liability
Derivative Liability | 12 Months Ended |
Dec. 31, 2021 | |
Derivative Instruments and Hedging Activities Disclosure [Abstract] | |
Derivative Liability | 16. Derivative Liability The Company identified an embedded derivative resulting from the change of control provision in the CFF Agreement. Embedded derivatives that are required to be bifurcated from the underlying host instrument are accounted for and valued as separate financial instruments. At the inception of the derivative in 2017, the Company recognized this derivative as a liability and revenue was reduced by the initial fair value of the derivative liability. The Company remeasures the derivative liability to fair value at each reporting period and records the change in fair value of the derivative liability as other income (expense), net. The Company uses a present value analysis with multiple scenarios, which incorporates assumptions and estimates to value the derivative instrument. The Company assesses these assumptions and estimates on a periodic basis as additional information impacting the assumptions is obtained. Estimates and assumptions impacting the fair value measurement include the change of control payment to CFF (range of $ 0.0 million to $ 10.6 million at December 31, 2021 and December 31, 2020), the probability of a change of control event, the probability of the product achieving development or commercial status at time of change of control (range of 4.8 % and 17.2 % at December 31, 2021 and 3.4 % to 12.3 % at December 31, 2020) and the discount rate ( 14.0 % at December 31, 2021 and December 31, 2020). The Company determined the estimated fair value of this liability as of the inception date of the CFF Agreement and concluded that the amount was immaterial. The Company determined the fair value of this derivative liability was $ 0.2 million and $ 0.1 million as of December 31, 2021 and 2020, respectively. |
Related Party Transactions
Related Party Transactions | 12 Months Ended |
Dec. 31, 2021 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | 17. Related Party Transactions During the years ended December 31, 2021 and 2020, the Company paid $ 85,000 and $ 93,000 , respectively, to David Schaffer, Ph.D., the co-founder, director and Chief Scientific Advisor of the Company for consulting services. In April 2019, the Company entered into two sponsored research agreements (“SRAs”) with the UC Regents to conduct research in a research facility on the U.C. Berkeley campus, under the direction of Dr. Schaffer. The SRAs have a three year term ending in May 2022. Under the SRAs, the Company has an option to license (on a royalty-bearing basis) all intellectual property generated under the SRAs. The total amount the Company is committed to pay to the UC Regents under the SRAs is $ 1.5 million, which was fully paid as of December 31, 2021. In March 2021, the Company entered into another sponsored research agreement with the UC Regents to conduct research in laboratories on the U.C. Berkeley campus that are under the direction of Dr. Schaffer and another U.C. Berkeley professor covering investigations into how machine learning approaches may enhance AAV capsid engineering (the “Machine Learning SRA”). Pursuant to the Machine Learning SRA, the Company has committed to pay the UC Regents a total of $ 1.4 million, of which $ 0.4 million was paid as of December 31, 2021. The Machine Learning SRA has a three-year term ending in 2024. Any patent prosecution costs incurred under the SRAs will also be borne by the Company. The Company can terminate the SRAs for convenience and without cause with 60 days’ notice. While the Machine Learning SRA is between the Company and the UC Regents, the payments under the SRA will be used to fund the lab under the direction of Dr. Schaffer. As of December 31, 2021 and 2020, accounts payable to the UC Regents related to the SRAs and Machine Learning SRA was $ 0.0 million and $ 0.2 million, respectively. In the years ended December 31, 2021 and 2020, the Company recorded $ 0.9 million and $ 0.5 million, respectively, of expense related to SRAs. In August 2019, the Company and uniQure entered into the amended uniQure Agreement and the Second uniQure Agreement. Under these agreements, the Company agreed to transfer incremental rights and services to uniQure in exchange for uniQure eliminating the exclusivity clause in the uniQure Agreement and transferring other rights back to the Company. Further details and the accounting for these agreements is discussed in Note 7. As of June 17, 2020, uniQure was no longer a related party of the Company. |
401(K) Plan
401(K) Plan | 12 Months Ended |
Dec. 31, 2021 | |
Retirement Benefits [Abstract] | |
401(K) Plan | 18. 401(k) Plan In 2014, the Company adopted a 401(k) plan for all employees who have met certain eligibility requirements. The 401(k) plan allows employees to make pre-tax and post-tax contributions up to the maximum allowable amount set by the Internal Revenue Service. The Company made contributions to the Plan for eligible participants, and recorded contribution expenses of $ 0.5 million and $ 0.4 million for the years ended December 31, 2021 and 2020, respectively. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2021 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The accompanying financial statements have been prepared in accordance with United States Generally Accepted Accounting Principles (“U.S. GAAP”). |
Use of Estimates and Judgements | Use of Estimates and Judgements The preparation of financial statements in conformity with U.S. GAAP requires management to make estimates and judgements that affect the reported amounts of assets, liabilities, revenue and expenses; and disclosure of contingent assets and liabilities as of the date of the financial statements. Such estimates include the determination of useful lives for property and equipment, the contract term, transaction price and costs of collaboration agreements, as well as estimates of the fair value of common stock (prior to the IPO), stock options and derivative instruments and income tax uncertainties. Actual results could differ from those estimates. Due to the coronavirus (“COVID-19”) pandemic, there has been uncertainty and disruption in the global economy and financial markets. The Company is not aware of any specific event or circumstance that would require an update to its estimates or judgments or a revision of the carrying value of its assets or liabilities as of December 31, 2021. While there was not a material impact to the Company’s financial statements as of December 31, 2021, these estimates may change, as new events occur and additional information is obtained, as well as other factors related to the COVID-19 pandemic that could result in material impacts to the financial statements in future reporting periods. |
Segment Information | Segment Information The Company operates and manages its business as one reportable and operating segment. The Company’s chief executive officer, who is the chief operating decision maker, reviews financial information on a company-wide basis for purposes of allocating resources and assessing financial performance. As of and for the years ended December 31, 2021 and 2020, all of the Company’s long-lived assets were located in the United States and all revenue was earned in the United States. |
Concentration of Credit Risk | Concentration of Credit Risk Financial instruments that potentially subject the Company to a concentration of credit risk consist of cash and cash equivalents, marketable securities and accounts receivable. The Company’s cash is held at two financial institutions in the United States of America. The Company’s cash equivalents are invested in money market funds. The Company also invests in U.S. Treasuries, commercial paper and corporate bonds. The Company has not experienced any losses on its deposits of cash and cash equivalents. Such deposits may, at times, exceed federally insured limits. The Company’s partners in collaboration and license agreements who represent 10% or more of the Company’s total revenue are as follows: Year Ended December 31, 2021 2020 Customer A 95 % 95 % Customer B * * Total 95 % 95 % * Less than 10 % The Company’s partners in collaboration and license agreements who represent 10% or more of the Company’s total accounts receivable are as follows: December 31, 2021 2020 Customer A 100 % 74 % Customer B 0 % 26 % Total 100 % 100 % The Company’s total revenues by geographic region, based on the location of the customer, are as follows (in thousands): Year Ended December 31, 2021 2020 Netherlands $ 736 $ 742 Switzerland 17,181 12,897 United States 121 ( 27 ) Total revenue $ 18,038 $ 13,612 |
Cash and Cash Equivalents | Cash and Cash Equivalents The Company considers all highly liquid investments with original maturities of three months or less at the date of purchase to be cash equivalents. Cash equivalents consist of money market funds. |
Marketable Securities | Marketable Securities Marketable securities consist of commercial paper, corporate bonds and U.S. Treasuries and are included in current and noncurrent assets. The Company classifies its marketable securities as available-for-sale and carries them at fair value on its balance sheet. Fair value is estimated using independent pricing sources based on quoted prices in active markets for similar securities. Unrealized gains and losses on the marketable securities are reported as a component of stockholders' equity (deficit) in accumulated other comprehensive loss. The amortized cost of marketable securities is adjusted for amortization of premiums and accretion of discounts to maturity, which is included in interest income on the statements of operations. Realized gains and losses are included in interest income on the statements of operations. The Company periodically evaluates its marketable securities to assess whether those with unrealized loss positions are other than temporarily impaired. The Company considers various factors in determining whether to recognize an impairment charge. If the Company determines that the decline in an investment's fair value is other-than-temporary, the difference is recognized as an impairment loss in the statements of operations. |
Other Risks and Uncertainties | Other Risks and Uncertainties The Company is subject to risks and uncertainties common to early-stage companies in the biotechnology industry, including, but not limited to, development by competitors of new technological innovations, protection of proprietary technology, dependence on key personnel, suppliers for key raw materials, contract manufacturing organizations (“CMOs”) and contract research organizations (“CROs”), compliance with government regulations and the need to obtain additional financing to fund operations. Product candidates currently under development will require significant additional research and development efforts, including extensive preclinical studies, clinical trials and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel and infrastructure and extensive compliance and reporting. There can be no assurance that the Company’s research and development will be successfully completed, that adequate protection for the Company’s intellectual property will be obtained or maintained, that any products developed will obtain necessary government regulatory approval or that any approved products will be commercially viable. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will generate significant revenue from product sales. The Company operates in an environment of rapid change in technology and substantial competition from other pharmaceutical and biotechnology companies. In addition, the Company is dependent upon the services of its employees, consultants and other third parties (including for clinical trials and some aspects of research and preclinical testing). The extent of the impact of the COVID-19 pandemic on the Company’s business will depend upon the duration and spread of the outbreak and the extent and severity of the impact on the Company’s clinical trial activities, research activities and suppliers, all of which are uncertain and cannot be predicted. The extent to which the coronavirus outbreak may materially impact the Company’s financial condition, liquidity or results of operations is uncertain. |
Fair Value Measurements | Fair Value Measurements The Company applies fair value accounting for all financial assets and liabilities and nonfinancial assets and liabilities that are recognized or disclosed at fair value in the financial statements on a recurring basis. Fair value is an exit price, representing the amount that would be received to sell an asset or paid to transfer a liability in an orderly transaction between market participants on the measurement date. As such, fair value is a market-based measurement that should be determined based on assumptions that market participants would use in pricing an asset or liability. As a basis for considering such assumptions, a three-level fair value hierarchy has been established, which prioritizes the inputs used in measuring fair value as follows: • Level 1 — Observable inputs that reflect unadjusted quoted market prices in active markets for identical assets or liabilities that are accessible at the measurement date. • Level 2 — Inputs other than Level 1 that are observable, either directly or indirectly, such as quoted prices for similar assets or liabilities; quoted prices in markets that are not active; or other inputs that are observable or can be corroborated by observable market data for substantially the full term of the assets or liabilities. • Level 3 — Unobservable inputs that are supported by little or no market activity and that are significant to the fair value of the assets or liabilities. The Company accounts for transfers of financial instruments between levels of the fair value hierarchy on the date of the event or change in circumstance that caused the transfer. |
Accounts Receivable—Allowance for Doubtful Accounts | Accounts Receivable—Allowance for Doubtful Accounts The Company regularly reviews accounts receivable for collectability and establishes an allowance for probable credit losses and writes off uncollectible accounts as necessary. The Company has determined that no allowance was required at December 31, 2021 and 2020. The Company did no t have any write-offs relating to uncollectible accounts receivable during the years ended December 31, 2021 and 2020. |
Property and Equipment, Net | Property and Equipment, Net Property and equipment are stated at cost less accumulated depreciation for acquired assets. Depreciation is computed using the straight-line method over the estimated useful lives of assets, ranging from three to five years . Leasehold improvements are amortized over the shorter of the useful life of the assets or the lease term. Upon sale or retirement of assets, the costs and related accumulated depreciation are removed from the balance sheet and the resulting gain or loss is reflected within operating expenses in the statements of operations. Maintenance and repairs are charged to expense as incurred. |
Impairment of Long-Lived Assets | Impairment of Long-Lived Assets The Company reviews long-lived assets for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. Recoverability is measured by comparison of the carrying amount to the future undiscounted net cash flows, which the assets are expected to generate. If such assets are considered to be impaired, the impairment to be recognized is typically measured by the amount by which the carrying amount of the assets exceeds the projected discounted future net cash flows arising from the asset. There have been no such impairments of long-lived assets in the years ended December 31, 2021 and 2020. |
Redeemable Convertible Preferred Stock | Redeemable Convertible Preferred Stock The Company recorded all shares of redeemable convertible preferred stock at their respective fair values on the date of issuance, net of issuance costs. The redeemable convertible preferred stock was recorded outside of permanent equity because while it was not mandatorily redeemable, in certain events considered not solely within the Company's control, such as a merger or consolidation, sale, lease, or license of substantially all of the Company's assets (each, a "deemed liquidation event"), the convertible preferred stock would become redeemable at the option of the holders of a majority of the outstanding series of redeemable convertible preferred stock. The Company did not adjust the carrying values of the redeemable convertible preferred stock to the liquidation preference of such shares because a deemed liquidation event obligating the Company to pay the liquidation preference did not occur. All outstanding shares of the redeemable convertible preferred stock converted into common stock upon the close of the IPO in December 2020. |
Common Stock Warrants | Common Stock Warrants The Company accounts for common stock warrants which meet the definition of a derivative as liabilities if the warrant requires net cash settlement or gives the holder the option of net cash settlement. The Company accounts for common stock warrants as equity if the contract requires physical settlement or net physical settlement or if the Company has the option of physical settlement or net physical settlement. Common stock warrants classified as liabilities are initially recorded at fair value and remeasured at fair value each balance sheet date with the offset adjustments recorded in other income (expense), net within the statements of operations. Common stock warrants classified as equity are initially measured at fair value on the grant date and are not subsequently remeasured. |
Leases | Leases On December 31, 2021, the Company adopted Accounting Standards Update (“ASU”) No. 2016-02, Leases (Topic 842), ("ASC 842") using the alternative modified retrospective approach effective as of January 1, 2021 to recognize a cumulative-effect adjustment on the effective date and to not adjust financial information and disclosures required under the new lease standards for comparative prior periods. The Company has elected to apply the package of practical expedients requiring no reassessment of whether any expired or existing contracts are or contain leases, the lease classification of any expired or existing leases, or the capitalization of initial direct costs for any existing leases. Additionally, the Company has elected not to separate lease and non-lease components and not to recognize leases with an initial term of twelve months or less. Under ASC 842, at contract inception, the Company determines if an arrangement is or contains a lease. A contract is or contains a lease if the contract conveys the right to control the use of an identified asset for a period of time in exchange for consideration. If determined to be or contain a lease, the lease is assessed for classification as either an operating or finance lease at the lease commencement date, defined as the date on which the leased asset is made available for use by the Company, based on the economic characteristics of the lease. A right-of-use asset represents the economic benefit conveyed to the Company by the right to use the underlying asset over the lease term. A lease liability represents the obligation to make lease payments arising from the lease. Operating lease right-of-use assets represent the Company's right to use an underlying asset for the lease term and operating lease liabilities represent the Company's obligation to make payments arising from the lease. Operating right-of-use assets and liabilities are recognized at the commencement date of the lease and are measured at the present value of the fixed payments due over the expected lease term less the present value of any incentives, rebates, or abatements the Company expects to receive from the lessor. The Company records amortization of operating right-of-use assets and accretion of lease liabilities as a single lease cost on a straight-line basis over the lease term. No lease renewal options are recognized as part of the right-of-use assets and lease liabilities. The Company's operating leases are presented in the balance sheet as operating lease right-of-use assets, classified as noncurrent assets, and operating lease liabilities, classified as current and noncurrent based on the discounted lease payments to be made within the proceeding twelve months. As the Company’s leases do not provide an implicit rate , the Company uses its incremental borrowing rate to discount lease payments. The incremental borrowing rate represents an estimated rate of interest that the Company would have to pay to borrow equivalent funds on a collateralized basis at the lease commencement date. Upon adoption of the new lease standard effective January 1, 2021 , the Company recognized operating lease right-of-use assets of $ 16.5 million, and current and noncurrent operating lease liabilities of $ 2.0 million and $ 16.1 million, respectively. In connection with the adoption of this standard, deferred rent of $ 1.0 million and lease incentive liability of $ 0.5 million, which were previously recorded in prepaid expenses and other current assets, accrued and other current liabilities and other liabilities on the balance sheet as of December 31, 2020, were derecognized. The adoption of the standard did no t have an impact on the Company's statements of operations or comprehensive loss, stockholders' equity or cash flows. The impact of adoption of the new lease standard as of January 1, 2021 is summarized as follows: December 31, 2020 ASC 842 Adjustments January 1, 2021 Operating lease right-of-use assets $ — $ 16,537 $ 16,537 Prepaid expenses and other current assets 4,444 ( 214 ) 4,230 Accrued and other current liabilities ( 8,371 ) 68 ( 8,303 ) Other liabilities ( 1,852 ) 1,696 ( 156 ) Operating lease liabilities, current — ( 2,029 ) ( 2,029 ) Operating lease liabilities, long-term — ( 16,058 ) ( 16,058 ) |
Revenue Recognition | Revenue Recognition The Company determines revenue recognition for arrangements within the scope of Accounting Standards Update 2014-09, Revenue from Contracts with Customers (Topic 606) ("ASC 606") by performing the following five steps: (i) identification of the promised goods or services in the contract; (ii) determination of whether the promised goods or services are performance obligations including whether they are distinct in the context of the contract; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations based on estimated selling prices; and (v) recognition of revenue when (or as) the Company satisfies each performance obligation. The Company’s revenue is primarily derived through its license, research, development and commercialization agreements. The terms of these types of agreements may include (i) licenses to the Company’s technology, (ii) research and development services, and (iii) services or obligations in connection with participation in research or steering committees. Payments to the Company under these arrangements typically include one or more of the following: nonrefundable upfront and license fees, research funding, milestone and other contingent payments to the Company for the achievement of defined collaboration objectives and certain preclinical, clinical, regulatory and sales-based events, as well as royalties on sales of any commercialized products. Arrangements that include upfront payments are recorded as deferred revenue upon receipt or when due and are recognized as revenue as performance conditions are met. The event-based milestone payments, royalties and cost reimbursements represent variable consideration, and the Company uses the most likely amount method to estimate this variable consideration. Royalty payments are recognized when earned or as the sales occur. The Company records cost reimbursements as accounts receivable when right to consideration is unconditional. A performance obligation is a promise in a contract to transfer a distinct good or service to the customer and is the unit of account in ASC 606. The Company allocates the total transaction price to each performance obligation based on the estimated standalone selling price and recognizes revenue when, or as, the performance obligation is satisfied. The Company includes the unconstrained amount of estimated variable consideration in the transaction price. At the end of each reporting period, the Company re-evaluates the estimated variable consideration included in the transaction price and any related constraint, and if necessary, adjusts its estimate of the overall transaction price. Significant management judgment is required to determine the level of effort required under an arrangement and the period over which the Company expects to complete its performance obligations under the arrangement. Changes in these estimates can have a material effect on revenue recognized. |
Research and Development Expenses | Research and Development Expenses Costs related to research, design and development of programs are charged to research and development expense as incurred. Research and development costs include, but are not limited to, payroll and personnel expenses including stock-based compensation, materials, laboratory supplies, outside services and allocated overhead, including rent, insurance, repairs and maintenance, depreciation and utilities. The Company expenses all research and development costs in the period in which they are incurred. Costs incurred in obtaining technology licenses are charged to research and development expense as acquired in-process research and development if the technology licensed has not reached technological feasibility and has no alternative future use. |
Accrued Research and Development | Accrued Research and Development The Company has entered into various agreements with CROs and CMOs. The Company’s research and development accruals are estimated based on the level of services performed, progress of the studies, including the phase or completion of events, and contracted costs. The estimated costs of research and development provided, but not yet invoiced, are included in accrued and other current liabilities on the balance sheet. If the actual timing of the performance of services or the level of effort varies from the original estimates, the Company will adjust the accrual accordingly. Payments made to CROs or CMOs under these arrangements in advance of the performance of the related services are recorded as prepaid expenses and other current assets until the services are rendered. |
Stock-Based Compensation | Stock-Based Compensation As of January 1, 2020 , the Company adopted ASU 2018-07, Compensation - Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting , which aligns the measurement and classification guidance for share-based payments to nonemployees with the guidance for share-based payments to employees. As of January 1, 2020, the Company accounts for stock-based compensation for stock options granted to employees, directors and nonemployees as measured at grant date, based on the fair value of the award. The Company measures the fair value of awards granted using the Black-Scholes option pricing model and recognizes the expense in the statements of operations over the requisite service period, generally four years , using the straight-line method. Forfeitures are accounted for as they occur. The Company’s policy for issuing stock upon stock option exercise is to issue new common stock. Prior to the adoption of ASU 2018-07 on January 1, 2020, stock-based compensation expense related to stock options granted to nonemployees was recognized based on the vesting date fair value of options as the stock options were earned. The Company remeasured the stock-based compensation at each reporting period end with the resulting change in fair value being recognized in the statements of operations over the period the related services were rendered. The Company estimated the service period for the options based on the time that would be required to satisfy the service condition, assuming the service condition would be satisfied. Stock-based compensation expense was recognized over the estimated service period but was accelerated if the performance condition was achieved earlier than estimated. |
Income Taxes | Income Taxes The Company accounts for income taxes under the asset and liability method, which requires, among other things, that deferred income taxes be provided for temporary differences between the tax basis of the Company’s assets and liabilities and their financial statement reported amounts. In addition, deferred tax assets are recorded for the future benefit of utilizing net operating losses and research and development credit carryforwards and are measured using the enacted tax rates and laws that will be in effect when such items are expected to reverse. A valuation allowance is provided against deferred tax assets unless it is more likely than not that they will be realized. The Company accounts for uncertain tax positions by assessing all material positions taken in any assessment or challenge by relevant taxing authorities. Assessing an uncertain tax position begins with the initial determination of the position’s sustainability and is measured at the largest amount of benefit that is greater than fifty percent likely of being realized upon ultimate settlement. The Company’s policy is to recognize interest and penalties related to the underpayment of income taxes as a component of income tax expense or benefit. To date, there have been no interest or penalties charged in relation to the unrecognized tax benefits. |
Embedded Derivative | Embedded Derivative Embedded derivatives that are required to be bifurcated from the underlying host instrument are accounted for and valued as a separate financial instrument. An embedded derivative exists in the award agreement with the Cystic Fibrosis Foundation (“CFF”). As described in Note 16, the embedded derivative has been bifurcated and is classified as a liability on the balance sheet and separately accounted for at its fair value. The derivative liability is subject to remeasurement to fair value each reporting period. Changes in the fair value of the derivative liability are recognized as a component of other income (expense), net within the statements of operations. |
Deferred Offering Costs | Deferred Offering Costs The Company capitalizes certain legal, accounting and other third-party fees that are directly related to the Company’s in-process financings, until such financings are consummated. After consummation of the financing, these costs are recorded as a reduction of the proceeds received as a result of the offering. In the event that a planned offering does not occur or is significantly delayed, all related deferred offering costs will be expensed immediately within the Company’s statements of operations. |
Net Loss Per Share, Basic and Diluted | Net Loss Per Share, Basic and Diluted The Company calculates basic and diluted net loss per share in conformity with the two-class method required for companies with participating securities. The Company considers all series of redeemable convertible preferred stock to be participating securities as the holders are entitled to receive non-cumulative dividends on a pari passu basis in the event the dividend is paid on common shares. Under the two-class method, the net loss is not allocated to the redeemable convertible preferred stock as the holders of redeemable convertible preferred stock do not have a contractual obligation to share in losses. Basic net loss per share is calculated by dividing the net loss by the weighted-average number of shares of common stock outstanding during the period, less shares subject to repurchase. Diluted net loss per share is computed by giving effect to all potentially dilutive common shares outstanding for the period. For purposes of this calculation, redeemable convertible preferred shares, stock options to acquire shares of common stock, common stock warrants, and unvested common stock subject to repurchase, are considered potentially dilutive common shares, but have been excluded from the calculation of diluted net loss per share as their effect is antidilutive. |
Recent Accounting Pronouncements | Recent Accounting Pronouncements New Accounting Pronouncements Recently Adopted In June 2018, the Financial Accounting Standards Board ("FASB") issued Accounting Standards Update ("ASU") No. 2018-07, Compensation-Stock Compensation (Topic 718): Improvements to Nonemployee Share-Based Payment Accounting. This ASU aligns the measurement and classification guidance for share-based payments to nonemployees with the guidance for share-based payment to employees. Under this ASU, the measurement of equity-classified nonemployee awards is fixed at the grant date, which may lower their cost and reduce volatility in the statements of operations. The transition method provided by this ASU is on a modified retrospective basis, which recognizes a cumulative-effect adjustment to the opening balance of retained earnings in the period of adoption. This ASU is effective for fiscal years beginning after December 15, 2019, and interim periods within fiscal years beginning after December 15, 2020, with early adoption permitted, but no earlier than a company's adoption date of ASC 606. The Company adopted this guidance effective January 1, 2020 using the modified retrospective method. The Company recorded a less than $ 0.1 million cumulative-effect adjustment reflected as a decrease to the opening balance of accumulated deficit and a decrease to additional paid-in capital In November 2018, the FASB issued ASU No. 2018-18, Collaborative Arrangements (Topic 808): Clarifying the Interaction Between Topic 808 and Topic 606 . This ASU clarifies that certain transactions between participants in a collaborative arrangement should be accounted for under ASC 606, Revenue from Contracts with Customers, when the counterparty is a customer. This ASU also precludes an entity from presenting consideration received from a transaction as revenue from contracts with customers if the counterparty is not a customer for that transaction. This ASU is effective for fiscal years beginning after December 15, 2020, and interim periods within fiscal years beginning after December 15, 2021. Early adoption is permitted for entities that have adopted ASC 606, Revenue from Contracts with Customers. The Company adopted this ASU on January 1, 2021 . The adoption of this ASU did not result in a material impact on the Company’s financial statements and related disclosures. In December 2019, the FASB issued ASU No. 2019-12, Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes . This ASU affects general principles within Topic 740 and is meant to simplify the accounting for income taxes by removing certain exceptions to the general framework. The ASU adds guidance to reduce complexity in certain areas, including recognizing a franchise (or similar) tax that is partially based on income as an income-based tax and incremental amounts incurred as a non-income-based tax and recognizing deferred taxes for tax goodwill. ASU 2019-12 also created an exception to the incremental approach for intraperiod tax allocation when there is a loss from continuing operations and income or a gain from other items (for example, other comprehensive income). The amendments in ASU 2019-12 are effective for the Company for fiscal years beginning after December 15, 2021, and interim periods within fiscal years beginning after December 15, 2022. Early adoption of the amendments is permitted. The Company adopted ASU 2019-12 effective January 1, 2021 on a prospective basis. The adoption of this ASU did not result in a material impact on the Company's financial statements and related disclosures. In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842) , as subsequently amended, which sets out the principles for the recognition, measurement, presentation and disclosure of leases for both parties to a contract (i.e., lessees and lessors), and replaces the existing guidance in ASC 840, Leases. The new standard requires lessees to apply a dual approach, classifying leases as either finance or operating leases based on the principle of whether or not the lease is effectively a financed purchase by the lessee. The classification will determine the recognition pattern of lease expense over the term of the lease. In addition, a lessee is required to record (i) a right-of-use asset and a lease liability on its balance sheet for all leases with accounting lease terms of more than twelve months regardless of whether it is an operating or financing lease and (ii) lease expense in its statements of operations for operating leases and amortization and interest expense in its statements of operations for financing leases. Leases with a term of twelve months or less may be accounted for similar to existing guidance for operating leases under Topic 840. In July 2018, the FASB issued ASU No. 2018-11, Leases (Topic 842) , which added an optional transition method that allows companies to adopt the standard as of the beginning of the year of adoption as opposed to the earliest comparative period presented. See earlier disclosure in this footnote on the impact on the Company of the adoption of this standard in 2021. New Accounting Pronouncements Not Yet Adopted As an “emerging growth company,” the Jumpstart Our Business Startups Act (“JOBS Act”) allows the Company to delay adoption of new or revised accounting pronouncements applicable to public companies until such pronouncements are made applicable to private companies. The Company has elected to use this extended transition period under the JOBS Act. The adoption dates discussed below reflect this election. In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments-Credit Losses (Topic 326), Measurement of Credit Losses on Financial Instruments , as clarified in subsequent amendments. ASU 2016-13 changes the impairment model for certain financial instruments. The new model is a forward-looking expected loss model and will apply to financial assets subject to credit losses and measured at amortized cost and certain off-balance sheet credit exposures. This includes loans, held-to-maturity debt securities, loan commitments, financial guarantees and net investments in leases, as well as trade receivables. For available-for-sale debt securities with unrealized losses, credit losses will be measured in a manner similar to today, except that the losses will be recognized as allowances rather than reductions in the amortized cost of the securities. In October 2019, the FASB voted to delay the effective date of this standard. Topic 326 will be effective for the Company for fiscal years beginning after December 15, 2022. Early adoption is permitted. The Company is in the process of evaluating the impact of this new guidance on its financial statements. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Accounting Policies [Abstract] | |
Schedule of Concentration of Risk | The Company’s partners in collaboration and license agreements who represent 10% or more of the Company’s total revenue are as follows: Year Ended December 31, 2021 2020 Customer A 95 % 95 % Customer B * * Total 95 % 95 % * Less than 10 % The Company’s partners in collaboration and license agreements who represent 10% or more of the Company’s total accounts receivable are as follows: December 31, 2021 2020 Customer A 100 % 74 % Customer B 0 % 26 % Total 100 % 100 % |
Schedule of Revenues by Geographic Region | The Company’s total revenues by geographic region, based on the location of the customer, are as follows (in thousands): Year Ended December 31, 2021 2020 Netherlands $ 736 $ 742 Switzerland 17,181 12,897 United States 121 ( 27 ) Total revenue $ 18,038 $ 13,612 |
Schedule of Impact of Adoption of New Accounting Standard | The impact of adoption of the new lease standard as of January 1, 2021 is summarized as follows: December 31, 2020 ASC 842 Adjustments January 1, 2021 Operating lease right-of-use assets $ — $ 16,537 $ 16,537 Prepaid expenses and other current assets 4,444 ( 214 ) 4,230 Accrued and other current liabilities ( 8,371 ) 68 ( 8,303 ) Other liabilities ( 1,852 ) 1,696 ( 156 ) Operating lease liabilities, current — ( 2,029 ) ( 2,029 ) Operating lease liabilities, long-term — ( 16,058 ) ( 16,058 ) |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Fair Value Disclosures [Abstract] | |
Fair Value Hierarchy for Financial Assets and Financial Liabilities Measured at Fair Value on Recurring Basis | The following tables represent the Company’s fair value hierarchy for financial assets and financial liabilities measured at fair value on a recurring basis as of December 31, 2021 and 2020 (in thousands): Basis for Fair Value Measurements Fair Value as of (Level 1) (Level 2) (Level 3) December 31, 2021 Assets Money market funds $ 36,556 $ — $ — $ 36,556 Commercial paper — 37,535 — 37,535 Corporate bonds — 94,864 — 94,864 US Treasuries — 30,029 — 30,029 Total $ 36,556 $ 162,428 $ — $ 198,984 Liabilities Derivative liability $ — $ — $ 214 $ 214 Total $ — $ — $ 214 $ 214 Basis for Fair Value Measurements Fair Value as of (Level 1) (Level 2) (Level 3) December 31, 2020 Assets Money market funds $ 276,726 $ — $ — $ 276,726 Total $ 276,726 $ — $ — $ 276,726 Liabilities Derivative liability $ — $ — $ 122 $ 122 Total $ — $ — $ 122 $ 122 |
Summary of Changes in Fair Value of Level 3 Derivative Liability | The following table sets forth a summary of the changes in the fair value of the Company’s Level 3 derivative liability (in thousands): Balance as of December 31, 2019 $ 101 Change in fair value included in other income (expense), net 21 Balance as of December 31, 2020 $ 122 Change in fair value included in other income (expense), net 92 Balance as of December 31, 2021 $ 214 |
Marketable Securities (Tables)
Marketable Securities (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Investments, Debt and Equity Securities [Abstract] | |
Summary of Available for Sale Securities | Marketable securities, which are classified as available-for-sale, consisted of the following as of December 31, 2021 (in thousands): Amortized Cost Basis Gross Unrealized Gains Gross Unrealized Losses Fair Value as of December 31, 2021 Short-term marketable securities: Commercial paper $ 32,471 $ — $ ( 4 ) $ 32,467 Corporate bonds 57,315 — ( 79 ) 57,236 US Treasuries 5,082 — ( 9 ) 5,073 Total short-term marketable securities $ 94,868 $ — $ ( 92 ) $ 94,776 Long-term marketable securities: Commercial paper $ 5,089 $ — $ ( 21 ) $ 5,068 Corporate bonds 37,821 — ( 193 ) 37,628 US Treasuries 25,073 — ( 117 ) 24,956 Total long-term marketable securities $ 67,983 $ — $ ( 331 ) $ 67,652 |
Property and Equipment, Net (Ta
Property and Equipment, Net (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Property, Plant and Equipment [Abstract] | |
Schedule of Property and Equipment, Net | Property and equipment, net, consisted of the following (in thousands): December 31, 2021 2020 Machinery and equipment $ 5,971 $ 4,911 Leasehold improvements 2,527 2,527 Furniture and fixtures 566 473 Office equipment 165 101 Computer equipment and software 421 366 Construction in progress 10,066 539 Total property and equipment 19,716 8,917 Less: Accumulated depreciation and amortization ( 5,325 ) ( 3,844 ) Property and equipment, net $ 14,391 $ 5,073 |
Accrued and Other Liabilities_2
Accrued and Other Liabilities, Current and Noncurrent (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Payables and Accruals [Abstract] | |
Summary of Accrued Liabilities | Accrued liabilities consisted of the following (in thousands): December 31, 2021 2020 Payroll and related expenses $ 3,994 $ 3,437 Accrued clinical and preclinical study costs 407 869 Consulting and professional 1,573 3,574 Other accrued expenses 1,770 491 Total accrued and other current liabilities $ 7,744 $ 8,371 |
Summary of Other Liabilities, Noncurrent | Other liabilities, noncurrent consisted of the following (in thousands): December 31, 2021 2020 Deferred rent $ — $ 1,237 Other payable 120 615 Total other liabilities, noncurrent $ 120 $ 1,852 |
Research and Collaboration Ar_2
Research and Collaboration Arrangements (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Research And Collaboration Arrangements [Abstract] | |
Summary of Collaboration and License Revenue | Collaboration and license revenue for each period was as follows (in thousands): Year Ended December 31, 2021 2020 uniQure $ 736 $ 742 Roche 17,181 12,897 CFF 121 ( 27 ) $ 18,038 $ 13,612 |
Summary of Deferred Revenue | Deferred revenue for each period was as follows (in thousands): Deferred Revenue 2021 2020 uniQure $ 3,661 $ 4,396 Roche — 14,318 CFF 1,429 1,098 $ 5,090 $ 19,812 |
Commitments and Contingencies (
Commitments and Contingencies (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Commitments and Contingencies Disclosure [Abstract] | |
Summary of Components of Lease Expense | The following table summarizes the components of lease expense for the year ended December 31, 2021, which are included in operating expenses in the Company’s statements of operations (in thousands): Operating lease cost $ 2,631 Variable lease cost 914 Total $ 3,545 |
Summary of Supplemental Information Related to Leases | The following table summarizes supplemental information related to operating leases for the year ended December 31, 2021: Weighted-average remaining lease term (in years): 7.6 Weighted-average discount rate: 7.8 % |
Summary of Maturities of Lease Liabilities | The following table summarizes the maturities of lease liabilities as of December 31, 2021 (in thousands): 2022 $ 1,230 2023 3,058 2024 3,149 2025 3,244 2026 3,135 Thereafter 8,686 Total future minimum lease payments 22,502 Less: Amount representing interest ( 6,054 ) Present value of future minimum lease payments 16,448 Less: Current portion of operating lease liabilities 1,231 Long-term portion of operating lease liabilities $ 15,217 |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Income Tax Disclosure [Abstract] | |
Schedule of Effective Tax Rate of Income Tax Expense (Benefit) Differs from Federal Statutory Rate | The effective tax rate of the Company’s income tax expense (benefit) differs from the federal statutory rate as follows: December 31, 2021 2020 Federal statutory income tax rate 21.0 % 21.0 % Research tax credit 2.1 % 2.8 % Permanent differences 0.4 % ( 1.7 )% Valuation allowance ( 23.5 )% ( 22.1 )% Provision for income taxes 0.0 % 0.0 % |
Schedule of Tax Effects of Temporary Differences to Significant Components of Deferred Taxes | The tax effects of temporary differences that give rise to significant components of the deferred taxes are as follows (in thousands): December 31, 2021 2020 Deferred Tax Assets Net operating loss carryforwards $ 37,295 $ 21,132 Other accrued liabilities 937 422 Deferred revenue 993 3,111 Research tax credits 8,078 6,018 Stock-based compensation expense 2,618 1,151 Intangible asset basis 907 977 Operating lease liabilities 3,460 — Total deferred tax assets $ 54,288 $ 32,811 Deferred Tax Liabilities Operating lease right-of-use assets $ ( 3,064 ) $ — Prepaid expenses ( 992 ) — Total deferred tax liabilities $ ( 4,056 ) $ — Less: valuation allowance ( 50,232 ) ( 32,811 ) Total net deferred tax $ — $ — |
Reconciliation of Beginning and Ending Unrecognized Tax Benefits | The reconciliation of the beginning and ending unrecognized tax benefits amounts is as follows (in thousands): Unrecognized Balance as of December 31, 2019 $ 1,566 Additions for current year tax positions 1,729 Additions for tax positions of prior years 72 Balance as of December 31, 2020 3,367 Additions for current year tax positions 2,219 Additions for tax positions of prior years — Balance as of December 31, 2021 $ 5,586 |
Redeemable Convertible Prefer_2
Redeemable Convertible Preferred Stock (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Temporary Equity Disclosure [Abstract] | |
Summary of Redeemable Convertible Preferred Stock | As of December 31, 2019, redeemable convertible preferred stock consisted of the following (in thousands, except per share and share amounts): Shares Original Shares Issued Liquidation Proceeds Net of Series A 909,312 $ 7.70 909,312 $ 7,001 $ 6,960 Series A-1 1,311,687 $ 8.84 1,311,687 11,595 11,548 Series B 5,154,639 $ 17.46 5,154,632 90,000 84,472 Total 7,375,638 7,375,631 $ 108,596 $ 102,980 |
Common Stock (Tables)
Common Stock (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Equity [Abstract] | |
Schedule of Common Share Reserved for Future Issuance | As of December 31, 2021 and 2020, the Company has reserved common stock, on an as-converted basis, for future issuance as follows: December 31, 2021 2020 Issuance of common stock under the 2020 Equity Incentive Plan 2,690,097 2,606,546 Issuance of common stock under the Employee Stock Share Purchase Plan 498,747 252,337 Exercise of options issued and outstanding 3,881,274 3,194,113 Exercise of common stock warrants 53,669 98,669 Total common stock reserved 7,123,787 6,151,665 |
Stock-based Compensation (Table
Stock-based Compensation (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Share-based Payment Arrangement [Abstract] | |
Summary of Stock Options Activity | The following table summarizes the stock options activity for the year ended December 31, 2021: Options Outstanding Number of Number of Weighted- Weighted- Aggregate Balances at December 31, 2020 2,606,546 3,194,113 $ 11.05 8.46 $ 97,105 Options authorized 1,334,099 — Options granted ( 1,566,150 ) 1,566,150 34.83 Options exercised — ( 563,387 ) 6.26 Options expired 4,324 ( 4,324 ) 36.33 Options forfeited 311,278 ( 311,278 ) 29.17 Balances at December 31, 2021 2,690,097 3,881,274 $ 19.86 $ 7.97 $ 24,666 Shares exercisable, December 31, 1,592,139 $ 12.75 $ 7.07 $ 17,280 Shares vested and expected to 3,881,274 $ 19.86 $ 7.97 $ 24,666 |
Summary of Stock Compensation Expense for Employees and Nonemployees by Function | The following table is a summary of stock compensation expense for employees and nonemployees by function (in thousands): Year Ended December 31, 2021 2020 Research and development $ 6,847 $ 2,670 General and administrative 6,952 2,314 Total stock-based compensation expense $ 13,799 $ 4,984 |
Schedule of Assumptions Used in Black-Scholes Valuation Model to Estimate Fair Value of Stock Options | The Company estimates the fair value of employee and nonemployee stock options using the Black-Scholes option pricing model. The fair value of employee and nonemployee options is recognized on a straight-line basis over the requisite service period of the awards. The fair value of the Company's stock options was estimated using the following assumptions for the years ended December 31, 2021 and 2020. Year Ended December 31, 2021 Year Ended December 31, 2020 Employee Nonemployee Employee Nonemployee Expected term 5.8 – 6.3 years 6.3 years 5.5 – 6.3 years 6.0 years Expected volatility 80.0 % – 84.3 % 83.5 % 82.1 % – 83.8 % 82.1 % – 83.1 % Risk-free interest rate 0.6 % – 1.3 % 0.6 % 0.4 % – 0.7 % 0.4 % – 0.7 % Expected dividend yield 0 % 0 % 0 % 0 % |
Net Loss Per Share, Basic and_2
Net Loss Per Share, Basic and Diluted (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Earnings Per Share [Abstract] | |
Computation of Basic and Diluted Net Loss Per Share | The following table sets forth the computation of basic and diluted net loss per share (in thousands, except share and per share data): Year Ended December 31, 2021 2020 Numerator Net loss attributable to common stockholders $ ( 71,317 ) $ ( 56,693 ) Denominator Weighted-average shares outstanding 27,730,420 6,430,555 Weighted-average shares outstanding used in computing net loss per share, basic and diluted 27,730,420 6,430,555 Net loss per share, basic and diluted $ ( 2.57 ) $ ( 8.82 ) |
Outstanding Shares of Potentially Dilutive Securities Excluded from Computation of Diluted Net Loss Per Share | The following outstanding shares of potentially dilutive securities were excluded from the computation of diluted net loss per share for the periods presented because including them would have been antidilutive: December 31, 2021 2020 Options issued and outstanding 3,881,274 3,194,113 Common stock warrants 53,669 98,669 Total 3,934,943 3,292,782 |
The Company - Additional Inform
The Company - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 12 Months Ended | ||||
Nov. 30, 2021 | Dec. 31, 2020 | Dec. 31, 2021 | Dec. 31, 2020 | Jan. 01, 2021 | Dec. 31, 2019 | |
Organization And Nature Of Business [Line Items] | ||||||
Aggregate net proceeds, after deducting underwriting discounts and commissions and offering costs | $ 114,296 | $ 205,658 | ||||
Outstanding shares of redeemable convertible preferred stock | 0 | 0 | 0 | 0 | 7,375,631 | |
Accumulated deficit | $ 135,679 | $ 206,996 | $ 135,679 | |||
Common Stock | ||||||
Organization And Nature Of Business [Line Items] | ||||||
Number of shares sold and issued | 4,875,715 | 9,660,000 | ||||
Common Stock | Initial Public Offering | ||||||
Organization And Nature Of Business [Line Items] | ||||||
Number of shares sold and issued | 9,660,000 | |||||
Stock sold and issued price per share | $ 23 | $ 23 | ||||
Aggregate net proceeds, after deducting underwriting discounts and commissions and offering costs | $ 204,700 | |||||
Number shares issued upon conversion of redeemable convertible preferred shares | 11,575,984 | 11,575,984 | ||||
Common Stock | Exercise of Overallotment Option | ||||||
Organization And Nature Of Business [Line Items] | ||||||
Number of shares sold and issued | 1,260,000 | |||||
Common Stock | Follow-On Offering | ||||||
Organization And Nature Of Business [Line Items] | ||||||
Stock sold and issued price per share | $ 25 | |||||
Aggregate net proceeds, after deducting underwriting discounts and commissions and offering costs | $ 111,100 | |||||
Number of shares priced under public offering | 4,750,000 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Additional Information (Details) | 12 Months Ended | |||
Dec. 31, 2021USD ($)SegmentFinancial_Institution | Dec. 31, 2020USD ($) | Jan. 01, 2021USD ($) | Dec. 31, 2019USD ($) | |
Summary Of Significant Accounting Policies [Line Items] | ||||
Number of reportable segments | Segment | 1 | |||
Number of operating segments | Segment | 1 | |||
Number of financial institutions cash is held | Financial_Institution | 2 | |||
Allowance for doubtful accounts | $ 0 | $ 0 | ||
Write-offs relating to uncollectible accounts receivable | 0 | 0 | ||
Impairments of long-lived assets | $ 0 | 0 | ||
Award requisite service period | 4 years | |||
Operating lease, right-of-use assets | $ 14,562,000 | 0 | $ 16,537,000 | |
Operating lease liabilities, current | 1,231,000 | 0 | 2,029,000 | |
Operating lease liabilities, long -term portion | 15,217,000 | 0 | 16,058,000 | |
Deferred rent | 1,000,000 | |||
Lease incentive liability | $ 500,000 | |||
Stockholders' equity attributable to parent | 319,107,000 | 256,387,000 | $ (72,970,000) | |
Additional Paid-in Capital | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Stockholders' equity attributable to parent | $ 526,523,000 | 392,063,000 | 6,054,000 | |
Additional Paid-in Capital | Cumulative Effect, Period of Adoption, Adjustment | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Stockholders' equity attributable to parent | $ (39,000) | |||
Accounting Standard Update ("ASU") 2016-02 | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Change in accounting principle accounting standards update, adopted | true | |||
Change in accounting principle accounting standards update, adoption date | Jan. 1, 2021 | |||
Change in accounting principle accounting standards update, immaterial effect | true | |||
Accounting Standard Update ("ASU") 2018-07 | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Change in accounting principle accounting standards update, adopted | true | |||
Change in accounting principle accounting standards update, adoption date | Jan. 1, 2020 | |||
Accounting Standard Update ("ASU") 2018-18 | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Change in accounting principle accounting standards update, adopted | true | |||
Change in accounting principle accounting standards update, adoption date | Jan. 1, 2021 | |||
Change in accounting principle accounting standards update, immaterial effect | true | |||
Accounting Standard Update ("ASU") 2019-12 | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Change in accounting principle accounting standards update, adopted | true | |||
Change in accounting principle accounting standards update, adoption date | Jan. 1, 2021 | |||
Change in accounting principle accounting standards update, immaterial effect | true | |||
Minimum | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Property and equipment, estimated useful life | 3 years | |||
Maximum | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Property and equipment, estimated useful life | 5 years | |||
Maximum | Accounting Standard Update ("ASU") 2018-07 | Additional Paid-in Capital | Cumulative Effect, Period of Adoption, Adjustment | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Stockholders' equity attributable to parent | $ 100,000 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Schedule of Concentration of Risk (Details) | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Revenue | Customer Concentration Risk | Customer A | ||
Concentration Risk [Line Items] | ||
Concentration risk, percentage | 95.00% | 95.00% |
Revenue | Customer Concentration Risk | Customer | ||
Concentration Risk [Line Items] | ||
Concentration risk, percentage | 95.00% | 95.00% |
Accounts Receivable | Credit Concentration Risk | Customer A | ||
Concentration Risk [Line Items] | ||
Concentration risk, percentage | 100.00% | 74.00% |
Accounts Receivable | Credit Concentration Risk | Customer B | ||
Concentration Risk [Line Items] | ||
Concentration risk, percentage | 0.00% | 26.00% |
Accounts Receivable | Credit Concentration Risk | Customer | ||
Concentration Risk [Line Items] | ||
Concentration risk, percentage | 100.00% | 100.00% |
Summary of Significant Accoun_6
Summary of Significant Accounting Policies - Schedule of Concentration of Risk (Details) (Parenthetical) | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Revenue | Customer Concentration Risk | Customer B | Maximum | ||
Concentration Risk [Line Items] | ||
Concentration risk, percentage | 10.00% | 10.00% |
Summary of Significant Accoun_7
Summary of Significant Accounting Policies - Schedule of Revenues by Geographic Region (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Disaggregation Of Revenue [Line Items] | ||
Total revenues | $ 18,038 | $ 13,612 |
Netherlands | ||
Disaggregation Of Revenue [Line Items] | ||
Total revenues | 736 | 742 |
Switzerland | ||
Disaggregation Of Revenue [Line Items] | ||
Total revenues | 17,181 | 12,897 |
United States | ||
Disaggregation Of Revenue [Line Items] | ||
Total revenues | $ 121 | $ (27) |
Summary of Significant Accoun_8
Summary of Significant Accounting Policies - Schedule of Impact of Adoption of New Accounting Standard (Details) - USD ($) $ in Thousands | Dec. 31, 2021 | Jan. 01, 2021 | Dec. 31, 2020 |
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||
Operating lease, right-of-use assets | $ 14,562 | $ 16,537 | $ 0 |
Prepaid expenses and other current assets | 8,456 | 4,230 | 4,444 |
Accrued and other current liabilities | (7,744) | (8,303) | (8,371) |
Other liabilities | (120) | (156) | (1,852) |
Operating lease liabilities, current | (1,231) | (2,029) | 0 |
Operating lease liabilities, long-term | $ (15,217) | (16,058) | 0 |
Previously Reported | |||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||
Prepaid expenses and other current assets | 4,444 | ||
Accrued and other current liabilities | (8,371) | ||
Other liabilities | $ (1,852) | ||
Revision of Prior Period, Accounting Standards Update, Adjustment | ASC 842 Adjustments | |||
New Accounting Pronouncements or Change in Accounting Principle [Line Items] | |||
Operating lease, right-of-use assets | 16,537 | ||
Prepaid expenses and other current assets | (214) | ||
Accrued and other current liabilities | 68 | ||
Other liabilities | 1,696 | ||
Operating lease liabilities, current | (2,029) | ||
Operating lease liabilities, long-term | $ (16,058) |
Fair Value Measurements - Fair
Fair Value Measurements - Fair Value Hierarchy for Financial Assets and Financial Liabilities Measured at Fair Value on Recurring Basis (Details) - Fair Value on Recurring Basis - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | $ 198,984 | $ 276,726 |
Liabilities | 214 | 122 |
Corporate Bonds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 94,864 | |
US Treasuries | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 30,029 | |
Derivative Liability | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Liabilities | 214 | 122 |
Commercial Paper | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 37,535 | |
Money Market Funds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 36,556 | 276,726 |
Level 1 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 36,556 | 276,726 |
Level 1 | Money Market Funds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 36,556 | 276,726 |
Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 162,428 | |
Level 2 | Corporate Bonds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 94,864 | |
Level 2 | US Treasuries | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 30,029 | |
Level 2 | Commercial Paper | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets | 37,535 | |
Level 3 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Liabilities | 214 | 122 |
Level 3 | Derivative Liability | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Liabilities | $ 214 | $ 122 |
Fair Value Measurements - Addit
Fair Value Measurements - Additional Information (Details) - USD ($) | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Fair Value Disclosures [Abstract] | ||
Assets, Level 1 to Level 2 transfers | $ 0 | $ 0 |
Assets, Level 2 to Level 1 Transfers | 0 | 0 |
Assets, Transfers into Level 3 | 0 | 0 |
Assets, Transfers out of Level 3 | 0 | 0 |
Liabilities, Level 1 to Level 2 transfers | 0 | 0 |
Liabilities, Level 2 to Level 1 transfers | 0 | 0 |
Liabilities, Transfers into Level 3 | 0 | 0 |
Liabilities, Transfers out of Level 3 | $ 0 | $ 0 |
Fair Value Measurements - Summa
Fair Value Measurements - Summary of Changes in Fair Value of Level 3 Derivative Liability (Details) - Derivative Liability - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Fair Value Liabilities Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Beginning Balance | $ 122 | $ 101 |
Change in fair value included in other income (expense), net | $ 92 | $ 21 |
Fair Value, Recurring Basis, Unobservable Input Reconciliation, Liability, Gain (Loss), Statement of Income [Extensible List] | Other income (expense), net | Other income (expense), net |
Ending Balance | $ 214 | $ 122 |
Marketable Securities - Summary
Marketable Securities - Summary of Available for Sale Securities (Details) $ in Thousands | Dec. 31, 2021USD ($) |
Short-term Marketable Securities | |
Debt Securities, Available-for-sale [Line Items] | |
Amortized Cost Basis | $ 94,868 |
Gross Unrealized Gains | 0 |
Gross Unrealized Losses | (92) |
Fair Value | 94,776 |
Short-term Marketable Securities | Commercial Paper | |
Debt Securities, Available-for-sale [Line Items] | |
Amortized Cost Basis | 32,471 |
Gross Unrealized Gains | 0 |
Gross Unrealized Losses | (4) |
Fair Value | 32,467 |
Short-term Marketable Securities | Corporate Bonds | |
Debt Securities, Available-for-sale [Line Items] | |
Amortized Cost Basis | 57,315 |
Gross Unrealized Gains | 0 |
Gross Unrealized Losses | (79) |
Fair Value | 57,236 |
Short-term Marketable Securities | US Treasuries | |
Debt Securities, Available-for-sale [Line Items] | |
Amortized Cost Basis | 5,082 |
Gross Unrealized Gains | 0 |
Gross Unrealized Losses | (9) |
Fair Value | 5,073 |
Long-term Marketable Securities | |
Debt Securities, Available-for-sale [Line Items] | |
Amortized Cost Basis | 67,983 |
Gross Unrealized Gains | 0 |
Gross Unrealized Losses | (331) |
Fair Value | 67,652 |
Long-term Marketable Securities | Commercial Paper | |
Debt Securities, Available-for-sale [Line Items] | |
Amortized Cost Basis | 5,089 |
Gross Unrealized Gains | 0 |
Gross Unrealized Losses | (21) |
Fair Value | 5,068 |
Long-term Marketable Securities | Corporate Bonds | |
Debt Securities, Available-for-sale [Line Items] | |
Amortized Cost Basis | 37,821 |
Gross Unrealized Gains | 0 |
Gross Unrealized Losses | (193) |
Fair Value | 37,628 |
Long-term Marketable Securities | US Treasuries | |
Debt Securities, Available-for-sale [Line Items] | |
Amortized Cost Basis | 25,073 |
Gross Unrealized Gains | 0 |
Gross Unrealized Losses | (117) |
Fair Value | $ 24,956 |
Marketable Securities (Addition
Marketable Securities (Additional Information) (Details) | 12 Months Ended | |
Dec. 31, 2021USD ($)Security | Dec. 31, 2020USD ($) | |
Debt Securities, Available-for-sale [Line Items] | ||
Realized gains on marketable securities | $ 0 | |
Realized losses on marketable securities | $ 0 | |
Number of marketable securities unrealized loss position more than one year | Security | 0 | |
Other-than-temporary impairment on investment in debt securities available-for-sale | $ 0 | |
Maximum Maturity Period of Marketable Securities | 2 years | |
Marketable securities | $ 0 |
Property and Equipment, Net - S
Property and Equipment, Net - Schedule of Property and Equipment, Net (Details) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Property Plant And Equipment [Line Items] | ||
Total property and equipment | $ 19,716 | $ 8,917 |
Less: Accumulated depreciation and amortization | (5,325) | (3,844) |
Property and equipment, net | 14,391 | 5,073 |
Machinery and Equipment | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | 5,971 | 4,911 |
Leasehold Improvements | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | 2,527 | 2,527 |
Furniture and Fixtures | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | 566 | 473 |
Office Equipment | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | 165 | 101 |
Computer Equipment and Software | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | 421 | 366 |
Construction in Progress | ||
Property Plant And Equipment [Line Items] | ||
Total property and equipment | $ 10,066 | $ 539 |
Property and Equipment, Net - A
Property and Equipment, Net - Additional Information (Details) - USD ($) $ in Millions | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Property, Plant and Equipment [Abstract] | ||
Depreciation expense | $ 1.5 | $ 1.4 |
Accrued and Other Liabilities_3
Accrued and Other Liabilities, Current and Noncurrent - Summary of Accrued Liabilities (Details) - USD ($) $ in Thousands | Dec. 31, 2021 | Jan. 01, 2021 | Dec. 31, 2020 |
Payables and Accruals [Abstract] | |||
Payroll and related expenses | $ 3,994 | $ 3,437 | |
Accrued clinical and preclinical study costs | 407 | 869 | |
Consulting and professional | 1,573 | 3,574 | |
Other accrued expenses | 1,770 | 491 | |
Total accrued and other current liabilities | $ 7,744 | $ 8,303 | $ 8,371 |
Accrued and Other Liabilities_4
Accrued and Other Liabilities, Current and Noncurrent - Summary of Other Liabilities, Noncurrent (Details) - USD ($) $ in Thousands | Dec. 31, 2021 | Jan. 01, 2021 | Dec. 31, 2020 |
Payables and Accruals [Abstract] | |||
Deferred rent | $ 1,237 | ||
Other payable | $ 120 | 615 | |
Total other liabilities, noncurrent | $ 120 | $ 156 | $ 1,852 |
Research and Collaboration Ar_3
Research and Collaboration Arrangements - Summary of Collaboration and License Revenue (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Collaboration and license revenue | $ 18,038 | $ 13,612 |
Collaboration and License Revenue | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Collaboration and license revenue | 18,038 | 13,612 |
Collaboration and License Revenue | uniQure | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Collaboration and license revenue | 736 | 742 |
Collaboration and License Revenue | Roche | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Collaboration and license revenue | 17,181 | 12,897 |
Collaboration and License Revenue | CFF | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Collaboration and license revenue | $ 121 | $ (27) |
Research and Collaboration Ar_4
Research and Collaboration Arrangements - Summary of Deferred Revenue (Details) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Total deferred revenue | $ 5,090 | $ 19,812 |
uniQure | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Total deferred revenue | 3,661 | 4,396 |
Roche | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Total deferred revenue | 14,318 | |
CFF | ||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||
Total deferred revenue | $ 1,429 | $ 1,098 |
Research and Collaboration Ar_5
Research and Collaboration Arrangements - Additional Information (Details) - USD ($) | 1 Months Ended | 12 Months Ended | ||
Jan. 31, 2014 | Dec. 31, 2021 | Dec. 31, 2020 | Aug. 31, 2019 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Deferred revenue | $ 5,090,000 | $ 19,812,000 | ||
Revenue recognized | 15,100,000 | 3,200,000 | ||
uniQure | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Deferred revenue | 3,661,000 | 4,396,000 | ||
Due from related parties | $ 0 | 0 | ||
uniQure | uniQure Agreement | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Collaborative arrangement, initial research term | 3 years | |||
Upfront payments received | $ 200,000 | |||
Options to purchase ordinary shares | 609,744 | |||
Ordinary shares vesting period | 3 years | |||
Fair value of options | $ 10,600,000 | |||
Fair value of non monetary consideration of intellectual property right | $ 5,100,000 | |||
uniQure | uniQure Agreement | Minimum | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Deferred revenue, estimated performance period | 1 year | |||
uniQure | uniQure Agreement | Maximum | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Contingent payments entitled to receive for achievement of research and development milestones | $ 5,000,000 | |||
Deferred revenue, estimated performance period | 2 years | |||
uniQure | Amended uniQure Agreement | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Revenue recognized | $ 700,000 | 700,000 | ||
Remaining performance obligations | $ 3,700,000 | $ 4,400,000 | ||
uniQure | Amended uniQure Agreement | Maximum | ||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||
Contingent payments eliminated | $ 5,000,000 |
Research and Collaboration Ar_6
Research and Collaboration Arrangements - Additional Information (Details 1) | Jan. 31, 2014 |
uniQure | uniQure Agreement | Revenue, Remaining Performance Obligation, Expected Timing of Satisfaction, Start Date: 2022-01-01 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |
Deferred revenue, estimated performance period | 4 years |
Research and Collaboration Ar_7
Research and Collaboration Arrangements - CRF Agreement - Additional Information (Details) - CRF - CRF Agreement - USD ($) | 1 Months Ended | 12 Months Ended | |
Nov. 30, 2015 | Dec. 31, 2021 | Dec. 31, 2020 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Collaborative arrangement, initial research term | 2 years | ||
Collaborative arrangement contribution upon development milestone | $ 2,500,000 | ||
Collaborative arrangement additional research term | 3 years | ||
Collaborative arrangement achieved milestones | $ 0 | ||
Deferred revenue | 0 | $ 0 | |
Due from related parties | 0 | 0 | |
Amount due to related party | $ 0 | $ 0 |
Research and Collaboration Ar_8
Research and Collaboration Arrangements - Roche Agreement - Additional Information (Details) - USD ($) $ in Thousands | 1 Months Ended | 12 Months Ended | |
Nov. 30, 2017 | Dec. 31, 2021 | Dec. 31, 2020 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Revenues | $ 18,038 | $ 13,612 | |
Roche | 2017 Roche Agreement | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Non-refundable upfront payments received | $ 21,000 | ||
Contingent payment for each nominated product beyond the first three | 1,000 | ||
Maximum contingent payment upon exercise of the option to convert a product | 30,000 | ||
Maximum development milestone payments | 223,000 | ||
Development milestone payments relating to Choroideremia | 86,000 | ||
Maximum sales-based milestone payments | $ 123,000 | ||
Non-refundable development milestone payments | 10,000 | ||
Collaborative arrangement adjustment to transaction price | 8,700 | 17,000 | |
Collaborative arrangement adjustment to transaction price due to increase in project scope and expected reimbursable costs | 7,000 | ||
Collaborative arrangement adjustment to transaction price due to variable consideration | 10,000 | ||
Increase in revenue recognized related to performance obligations partially satisfied in the beginning of the current year | 11,100 | 4,600 | |
Revenues | 17,200 | 12,900 | |
Deferred revenue | 0 | 14,300 | |
Accounts receivable | 0 | 1,100 | |
Aggregate amount of transaction price allocated to remaining performance obligation | $ 0 | $ 25,800 |
Research and Collaboration Ar_9
Research and Collaboration Arrangements - CFF - Additional Information (Details) $ in Thousands | 1 Months Ended | 12 Months Ended | |
Sep. 30, 2016USD ($)Installment | Dec. 31, 2021USD ($) | Dec. 31, 2020USD ($) | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Revenue recognized | $ 18,038 | $ 13,363 | |
Estimated fair value of derivative liability | 200 | 100 | |
CFF | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Collaborative arrangement total amount of award | $ 3,500 | ||
Collaborative arrangement achieved milestones | 1,800 | 1,300 | |
Expected payments percentage to equal actual award received | 600.00% | ||
Number of installments to pay actual award | Installment | 3 | ||
Actual award payment period upon first commercial sale of product | 4 years | ||
Royalties percentage on actual award received | 600.00% | ||
Continuous period where the Company ceases to develop or commercialize any product | 180 days | ||
Collaborative arrangement transaction price adjustment | 500 | ||
Revenue recognized | 100 | 100 | |
Deferred revenue | 1,400 | 1,100 | |
Accounts receivable | 0 | 400 | |
Remaining performance obligations | $ 1,400 | $ 1,100 | |
CFF | Maximum | |||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||
Future sales-based milestone payments percentage | 300.00% |
Research and Collaboration A_10
Research and Collaboration Arrangements - CFF - Additional Information (Details 1) - CFF - Revenue, Remaining Performance Obligation, Expected Timing of Satisfaction, Start Date: 2022-01-01 | Dec. 31, 2021 |
Minimum | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |
Deferred revenue, estimated performance period | 3 years |
Maximum | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |
Deferred revenue, estimated performance period | 4 years |
License Arrangements - Addition
License Arrangements - Additional Information (Details) - UC Agreements - USD ($) | 1 Months Ended | 12 Months Ended | ||||
Jul. 31, 2021 | Jan. 31, 2016 | Dec. 31, 2021 | Dec. 31, 2020 | Jan. 31, 2019 | Dec. 31, 2018 | |
Research And Development Arrangement Contract To Perform For Others [Line Items] | ||||||
Non-refundable license fee payable upon execution | $ 5,000 | |||||
Non-refundable license fee payable upon execution every year thereafter until sales of a licensed product and payment of royalties | 5,000 | |||||
Amount obligated to pay upon closing first qualified financing at option of UC Regents | $ 50,000 | |||||
Percentage of equity interest upon closing of first qualified financing at option of UC Regents | 3.00% | |||||
Percentage of equity interests issued as common stock | 6.00% | |||||
Amount paid non-refundable license fees | $ 100,000 | $ 50,000 | ||||
Non-refundable license fee payable annually | 10,000 | $ 5,000 | ||||
Incurred expenses | $ 300,000 | $ 200,000 | ||||
Maximum | ||||||
Research And Development Arrangement Contract To Perform For Others [Line Items] | ||||||
Development milestones | 3,900,000 | 3,100,000 | ||||
Minimum | ||||||
Research And Development Arrangement Contract To Perform For Others [Line Items] | ||||||
Annual royalty | $ 100,000 | $ 100,000 |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Details) - USD ($) $ in Thousands | 1 Months Ended | 12 Months Ended | |||||||
Nov. 30, 2021 | Feb. 28, 2021 | Dec. 31, 2019 | Nov. 30, 2018 | Oct. 31, 2018 | Jan. 31, 2016 | Dec. 31, 2021 | Jan. 01, 2021 | Dec. 31, 2020 | |
Commitment And Contingencies [Line Items] | |||||||||
Operating lease right-of-use ("ROU") assets | $ 14,562 | $ 16,537 | $ 0 | ||||||
Operating lease liability | $ 16,448 | ||||||||
Operating lease, discount rate | 7.80% | ||||||||
Operating lease expense | $ 2,600 | ||||||||
Cash paid for amounts included in the measurement of lease liabilities | 2,300 | ||||||||
5980 Horton Street Building Lease | |||||||||
Commitment And Contingencies [Line Items] | |||||||||
Lease extended term date | Mar. 31, 2023 | ||||||||
Tenant improvement allowance | $ 200 | ||||||||
Lease extended term period | 2026-09 | ||||||||
Operating lease right-of-use ("ROU") assets | 1,900 | ||||||||
Operating lease liability | $ 2,300 | ||||||||
Operating lease, discount rate | 7.50% | ||||||||
5858 Horton Street Building Lease and Expansion | |||||||||
Commitment And Contingencies [Line Items] | |||||||||
Tenant improvement allowance | $ 600 | $ 2,300 | $ 400 | ||||||
Operating lease right-of-use ("ROU") assets | $ 12,600 | ||||||||
Operating lease liability | $ 14,200 | ||||||||
Operating lease, discount rate | 7.80% | ||||||||
Lease initial term | 87 months | ||||||||
Lease option to renew term description | The new lease has an initial term of 87 months beginning on the rent commencement date with the option to renew the lease for one additional term of five years at market rent. | ||||||||
Lease existence of option to renew term | true | ||||||||
Lease renewal term | 5 years | ||||||||
Minimum | 5980 Horton Street Building Lease | |||||||||
Commitment And Contingencies [Line Items] | |||||||||
Lease renewal term | 3 years | ||||||||
Maximum | 5980 Horton Street Building Lease | |||||||||
Commitment And Contingencies [Line Items] | |||||||||
Lease renewal term | 5 years |
Commitments and Contingencies_2
Commitments and Contingencies - summary of Components of Lease Expense (Details) $ in Thousands | 12 Months Ended |
Dec. 31, 2021USD ($) | |
Commitments and Contingencies Disclosure [Abstract] | |
Operating lease cost | $ 2,631 |
Variable lease cost | 914 |
Total | $ 3,545 |
Commitments and Contingencies_3
Commitments and Contingencies - Summary of Supplemental Information Related to Leases (Details) | Dec. 31, 2021 |
Commitments and Contingencies Disclosure [Abstract] | |
Weighted-average remaining lease term (in years) | 7 years 7 months 6 days |
Weighted-average discount rate | 7.80% |
Commitments and Contingencies_4
Commitments and Contingencies - Summary of Maturities of Lease Liabilities (Details) - USD ($) $ in Thousands | Dec. 31, 2021 | Jan. 01, 2021 | Dec. 31, 2020 |
Commitments and Contingencies Disclosure [Abstract] | |||
2022 | $ 1,230 | ||
2023 | 3,058 | ||
2024 | 3,149 | ||
2025 | 3,244 | ||
2026 | 3,135 | ||
Thereafter | 8,686 | ||
Total future minimum lease payments | 22,502 | ||
Less: Amount representing interest | (6,054) | ||
Present value of future minimum lease payments | 16,448 | ||
Less: Current portion of operating lease liabilities | 1,231 | $ 2,029 | $ 0 |
Long-term portion of operating lease liabilities | $ 15,217 | $ 16,058 | $ 0 |
Income Taxes - Schedule of Effe
Income Taxes - Schedule of Effective Tax Rate of Income Tax Expense (Benefit) Differs from Federal Statutory Rate (Details) | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Income Tax Disclosure [Abstract] | ||
Federal statutory income tax rate | 21.00% | 21.00% |
Research tax credit | 2.10% | 2.80% |
Permanent differences | 0.40% | (1.70%) |
Valuation allowance | (23.50%) | (22.10%) |
Provision for income taxes | 0.00% | 0.00% |
Income Taxes - Schedule of Tax
Income Taxes - Schedule of Tax Effects of Temporary Differences to Significant Components of Deferred Taxes (Details) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Deferred Tax Assets | ||
Net operating loss carryforwards | $ 37,295 | $ 21,132 |
Other accrued liabilities | 937 | 422 |
Deferred revenue | 993 | 3,111 |
Research tax credits | 8,078 | 6,018 |
Stock-based compensation expense | 2,618 | 1,151 |
Intangible asset basis | 907 | 977 |
Operating lease liabilities | 3,460 | |
Total deferred tax assets | 54,288 | 32,811 |
Deferred Tax Liabilities, Net [Abstract] | ||
Operating lease right-of-use assets | (3,064) | |
Prepaid expenses | (992) | |
Total deferred tax liabilities | (4,056) | |
Less: valuation allowance | $ (50,232) | $ (32,811) |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Details) - USD ($) | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Operating Loss Carryforwards [Line Items] | ||
Income tax expense | $ 0 | $ 0 |
Increase in valuation allowance, deferred tax asset | 17,400,000 | 12,500,000 |
Net operating loss carryforwards | 177,500,000 | 100,600,000 |
Accrued interest and penalties related to unrecognized tax benefits | $ 0 | 0 |
CARES Act, relief description | The CARES Act changed certain provisions of the Tax Act. Under the CARES Act, NOLs arising in taxable years beginning after December 31, 2017 and before January 1, 2021 may be carried back to each of the five taxable years preceding the tax year of such loss, but NOLs arising in taxable years beginning after December 31, 2020 may not be carried back. In addition, the CARES Act eliminated the limitation on the deduction of NOLs to 80% of current year taxable income for taxable years beginning before January 1, 2021 and increased the amount of interest expense that may be deducted to 50% of adjusted taxable income for taxable years beginning in 2019 or 2020. | |
Federal | ||
Operating Loss Carryforwards [Line Items] | ||
Net operating loss carryforwards | $ 9,500,000 | |
Net operating loss carryforwards, expiration year | 2037 | |
Net operating loss carryforward indefinitely | $ 168,000,000 | |
Research and development credit carryforward | $ 8,600,000 | 5,700,000 |
Tax credit carryforward beginning expiration year | 2035 | |
State | ||
Operating Loss Carryforwards [Line Items] | ||
Research and development credit carryforward | $ 5,900,000 | $ 4,300,000 |
Tax credit carryforward, expiration description | indefinitely |
Income Taxes - Reconciliation o
Income Taxes - Reconciliation of Beginning and Ending Unrecognized Tax Benefits (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Income Tax Disclosure [Abstract] | ||
Balance at beginning of year | $ 3,367 | $ 1,566 |
Additions for current year tax positions | 2,219 | 1,729 |
Additions for tax positions of prior years | 0 | 72 |
Balance at end of year | $ 5,586 | $ 3,367 |
Redeemable Convertible Prefer_3
Redeemable Convertible Preferred Stock - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 6 Months Ended | 12 Months Ended | ||||
Oct. 31, 2021 | Apr. 30, 2020 | Jun. 30, 2020 | Dec. 31, 2021 | Dec. 31, 2020 | Jan. 01, 2021 | Dec. 31, 2019 | |
Temporary Equity [Line Items] | |||||||
Temporary equity, shares issued | 7,375,631 | ||||||
Net proceeds from issuance of convertible preferred stock | $ 0 | $ 72,468 | |||||
Outstanding shares of redeemable convertible preferred stock | 0 | 0 | 0 | 7,375,631 | |||
Common Stock | |||||||
Temporary Equity [Line Items] | |||||||
Purchase shares of common stock | 4,875,715 | 9,660,000 | |||||
Series C Redeemable Convertible Preferred Stock | |||||||
Temporary Equity [Line Items] | |||||||
Temporary equity, shares issued | 4,200,353 | ||||||
Temporary equity, shares issued price per share | $ 18 | ||||||
Net proceeds from issuance of convertible preferred stock | $ 72,500 | ||||||
Funding Agreement Product | CFF | |||||||
Temporary Equity [Line Items] | |||||||
Additional funding upon acceptance of investigational New Drug application | $ 4,000 | ||||||
Additional investment in cash | $ 4,000 | ||||||
Funding Agreement Product | CFF | Common Stock | |||||||
Temporary Equity [Line Items] | |||||||
Purchase shares of common stock | 125,715 | ||||||
Funding Agreement Product | CFF | Series C Redeemable Convertible Preferred Stock | |||||||
Temporary Equity [Line Items] | |||||||
Investment in redeemable convertible preferred stock financing | $ 10,000 |
Redeemable Convertible Prefer_4
Redeemable Convertible Preferred Stock - Summary of Redeemable Convertible Preferred Stock (Details) - USD ($) $ / shares in Units, $ in Thousands | Dec. 31, 2021 | Jan. 01, 2021 | Dec. 31, 2020 | Dec. 31, 2019 |
Temporary Equity [Line Items] | ||||
Shares Authorized | 7,375,638 | |||
Shares Issued | 7,375,631 | |||
Shares Outstanding | 0 | 0 | 0 | 7,375,631 |
Liquidation Value | $ 108,596 | |||
Proceeds Net of Issuance Cost | $ 102,980 | |||
Series A Redeemable Convertible Preferred Stock | ||||
Temporary Equity [Line Items] | ||||
Shares Authorized | 909,312 | |||
Original Issuance Price | $ 7.70 | |||
Shares Issued | 909,312 | |||
Shares Outstanding | 909,312 | |||
Liquidation Value | $ 7,001 | |||
Proceeds Net of Issuance Cost | $ 6,960 | |||
Series A-1 Redeemable Convertible Preferred Stock | ||||
Temporary Equity [Line Items] | ||||
Shares Authorized | 1,311,687 | |||
Original Issuance Price | $ 8.84 | |||
Shares Issued | 1,311,687 | |||
Shares Outstanding | 1,311,687 | |||
Liquidation Value | $ 11,595 | |||
Proceeds Net of Issuance Cost | $ 11,548 | |||
Series B Redeemable Convertible Preferred Stock | ||||
Temporary Equity [Line Items] | ||||
Shares Authorized | 5,154,639 | |||
Original Issuance Price | $ 17.46 | |||
Shares Issued | 5,154,632 | |||
Shares Outstanding | 5,154,632 | |||
Liquidation Value | $ 90,000 | |||
Proceeds Net of Issuance Cost | $ 84,472 |
Common Stock - Additional Infor
Common Stock - Additional Information (Details) - USD ($) | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Equity [Abstract] | ||
Common stock, shares authorized | 300,000,000 | 300,000,000 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock voting rights | one vote per share | |
Common stock dividend | $ 0 | $ 0 |
Common Stock - Schedule of Comm
Common Stock - Schedule of Common Share Reserved for Future Issuance (Details) - shares | Dec. 31, 2021 | Dec. 31, 2020 |
Class Of Stock [Line Items] | ||
Common stock reserved | 7,123,787 | 6,151,665 |
2020 Equity Incentive Plan | ||
Class Of Stock [Line Items] | ||
Common stock reserved | 2,690,097 | 2,606,546 |
Employee Stock Share Purchase Plan | ||
Class Of Stock [Line Items] | ||
Common stock reserved | 498,747 | 252,337 |
Options Issued and Outstanding | ||
Class Of Stock [Line Items] | ||
Common stock reserved | 3,881,274 | 3,194,113 |
Common Stock Warrants | ||
Class Of Stock [Line Items] | ||
Common stock reserved | 53,669 | 98,669 |
Stock-based Compensation - Addi
Stock-based Compensation - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands | Mar. 25, 2021 | Dec. 31, 2020 | Dec. 31, 2021 | Dec. 31, 2020 | Mar. 24, 2022 |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Number of shares reserved for issuance | 6,151,665 | 7,123,787 | 6,151,665 | ||
Number of shares available for grant | 2,606,546 | 2,690,097 | 2,606,546 | ||
Number of options outstanding | 3,194,113 | 3,881,274 | 3,194,113 | ||
Number of stock options granted | 1,566,150 | ||||
Fair value of options vested | $ 13,800 | $ 5,000 | |||
Unrecognized stock-based compensation of unvested options | $ 37,700 | ||||
Unrecognized stock-based compensation, expected to be recognized over weighted-average period | 2 years 8 months 12 days | ||||
Stock-based compensation expense | $ 13,799 | $ 4,984 | |||
Expected dividend yield | 0.00% | 0.00% | |||
Employee | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Number of stock options granted | 1,521,150 | 1,267,743 | |||
Weighted-average grant date fair value | $ 34.46 | $ 11.84 | |||
Stock-based compensation expense | $ 13,300 | $ 4,600 | |||
Expected dividend yield | 0.00% | 0.00% | |||
Nonemployee | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Number of stock options granted | 45,000 | 54,000 | |||
Weighted-average grant date fair value | $ 47.42 | $ 11.05 | |||
Stock-based compensation expense | $ 500 | $ 400 | |||
Expected dividend yield | 0.00% | 0.00% | |||
2020 Incentive Award Plan | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Number of shares reserved for issuance | 2,606,546 | 2,606,546 | |||
Percentage of common stock outstanding increased annually for future issuance | 5.00% | ||||
Number of shares available for grant | 2,690,097 | ||||
Number of shares authorized and reserved for issuance | 1,334,099 | ||||
2020 Incentive Award Plan | Maximum | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Options exercisable period | 10 years | ||||
Number of shares available for grant | 18,000,000 | ||||
2015 Equity Incentive Plan | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Options exercisable period | 10 years | ||||
Number of shares available for grant | 0 | ||||
Number of options outstanding | 2,519,217 | ||||
Minimum percentage of outstanding shares held by individual | 10.00% | ||||
Exercise price as percentage of fair market value, minimum | 110.00% | ||||
2020 ESPP | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Percentage of aggregate number of shares of common stock outstanding reserved for future issuance | 1.00% | ||||
Number of shares authorized and reserved for issuance | 252,337 | 252,337 | 25,000 | ||
Employee purchase price of common stock as percentage of fair market value | 85.00% | ||||
Common stock overlapping offering period | 24 months | ||||
Duration of purchase period | 6 months | ||||
Percentage of maximum contributions of employee's eligible compensation | 15.00% | 15.00% | |||
2020 ESPP | Maximum | |||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |||||
Number of shares available for grant | 15,000,000 |
Stock-based Compensation - Summ
Stock-based Compensation - Summary of Stock Options Activity (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Share-based Payment Arrangement [Abstract] | ||
Number of Shares Available for Grant, Beginning Balance | 2,606,546 | |
Number of Shares Available for Grant, Options authorized | 1,334,099 | |
Number of Shares Available for Grant, Options granted | (1,566,150) | |
Number of Shares Available for Grant, Options expired | 4,324 | |
Number of Shares Available for Grant, Options forfeited | 311,278 | |
Number of Shares Available for Grant, Ending Balance | 2,690,097 | 2,606,546 |
Number of Shares Underlying Outstanding Options, Beginning Balance | 3,194,113 | |
Number of Shares Underlying Outstanding Options, granted | 1,566,150 | |
Number of Shares Underlying Outstanding Options, exercised | (563,387) | |
Number of Shares Underlying Outstanding Options, expired | (4,324) | |
Number of Shares Underlying Outstanding Options, forfeited | (311,278) | |
Number of Shares Underlying Outstanding Options, Ending Balance | 3,881,274 | 3,194,113 |
Number of Shares Underlying Outstanding Options, Shares exercisable, December 31, 2021 | 1,592,139 | |
Number of Shares Underlying Outstanding Options, Shares vested and expected to vest, December 31, 2021 | 3,881,274 | |
Weighted-Average Exercise Price, Beginning Balance | $ 11.05 | |
Options granted | 34.83 | |
Options exercised | 6.26 | |
Options expired | 36.33 | |
Options forfeited | 29.17 | |
Weighted-Average Exercise Price, Ending Balance | 19.86 | $ 11.05 |
Weighted-Average Exercise Price, Shares exercisable, December 31,2021 | 12.75 | |
Weighted-Average Exercise Price, Shares vested and expected to vest, December 31,2021 | $ 19.86 | |
Weighted-Average Remaining Contractual Term (in years), Outstanding | 7 years 11 months 19 days | 8 years 5 months 15 days |
Weighted-Average Remaining Contractual Term (in years), Shares exercisable, December 31,2021 | 7 years 25 days | |
Weighted-Average Remaining Contractual Term (in years), Shares vested and expected to vest, December 31,2021 | 7 years 11 months 19 days | |
Aggregate Intrinsic Value, Outstanding Balance | $ 24,666 | $ 97,105 |
Aggregate Intrinsic Value, Shares exercisable, December 31,2021 | 17,280 | |
Aggregate Intrinsic Value, Shares vested and expected to vest, December 31,2021 | $ 24,666 |
Stock-based Compensation - Su_2
Stock-based Compensation - Summary of Stock Compensation Expense for Employees and Nonemployees by Function (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||
Total stock-based compensation expense | $ 13,799 | $ 4,984 |
Research and Development | ||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||
Total stock-based compensation expense | 6,847 | 2,670 |
General and Administrative | ||
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | ||
Total stock-based compensation expense | $ 6,952 | $ 2,314 |
Stock-based Compensation - Sche
Stock-based Compensation - Schedule of Assumptions Used in Black-Scholes Valuation Model to Estimate Fair Value of Stock Options (Details) | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Expected dividend yield | 0.00% | 0.00% |
Employee | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Expected volatility, minimum | 80.00% | 82.10% |
Expected volatility, maximum | 84.30% | 83.80% |
Risk-free interest rate, minimum | 0.60% | 0.40% |
Risk-free interest rate, maximum | 1.30% | 0.70% |
Expected dividend yield | 0.00% | 0.00% |
Nonemployee | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Expected term | 6 years 3 months 18 days | 6 years |
Expected volatility, minimum | 82.10% | |
Expected volatility, maximum | 83.10% | |
Expected volatility | 83.50% | |
Risk-free interest rate, minimum | 0.40% | |
Risk-free interest rate, maximum | 0.70% | |
Risk-free interest rate | 0.60% | |
Expected dividend yield | 0.00% | 0.00% |
Minimum | Employee | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Expected term | 5 years 9 months 18 days | 5 years 6 months |
Maximum | Employee | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Expected term | 6 years 3 months 18 days | 6 years 3 months 18 days |
Common Stock Warrants - Additio
Common Stock Warrants - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 12 Months Ended | |||||
Apr. 30, 2021 | May 31, 2018 | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2016 | Oct. 31, 2021 | Dec. 31, 2018 | |
Class Of Warrant Or Right [Line Items] | |||||||
Number of warrants issued | 40,000 | 23,669 | 30,000 | 45,000 | |||
Exercise price | $ 9.41 | $ 3.19 | $ 18 | $ 1.14 | |||
Warrant shares exercisable | 45,000 | ||||||
Issuance of common stock warrant in connection with settlement | $ 1,146 | ||||||
Proceeds from offering of securities in a private placement | $ 25,000 | ||||||
Warrant expiration year | 2025 | 2027 | 2023 | ||||
Warrant vesting period | 4 years | ||||||
Warrant exercised month and year | 2021-05 | ||||||
Additional Paid-in Capital | |||||||
Class Of Warrant Or Right [Line Items] | |||||||
Issuance of common stock warrant in connection with settlement | $ 1,100 | ||||||
Maximum | |||||||
Class Of Warrant Or Right [Line Items] | |||||||
Fair value of warrant shares | $ 100 | $ 100 | |||||
Series B Financing | |||||||
Class Of Warrant Or Right [Line Items] | |||||||
Warrant shares exercisable | 15,000 | ||||||
Private Placement | |||||||
Class Of Warrant Or Right [Line Items] | |||||||
Warrant shares exercisable | 15,000 | ||||||
Initial Public Offering | |||||||
Class Of Warrant Or Right [Line Items] | |||||||
Warrant shares exercisable | 30,000 |
Net Loss Per Share, Basic and_3
Net Loss Per Share, Basic and Diluted - Computation of Basic and Diluted Net Loss Per Share (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Numerator | ||
Net loss attributable to common stockholders | $ (71,317) | $ (56,693) |
Denominator | ||
Weighted-average shares outstanding | 27,730,420 | 6,430,555 |
Weighted-average shares outstanding used in computing net loss per share, basic and diluted | 27,730,420 | 6,430,555 |
Net loss per share, basic and diluted | $ (2.57) | $ (8.82) |
Net Loss Per Share, Basic and_4
Net Loss Per Share, Basic and Diluted - Outstanding Potentially Dilutive Securities Excluded From Computation of Diluted Net Loss Per Share (Details) - shares | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Antidilutive securities | 3,934,943 | 3,292,782 |
Options Issued and Outstanding | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Antidilutive securities | 3,881,274 | 3,194,113 |
Common Stock Warrants | ||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||
Antidilutive securities | 53,669 | 98,669 |
Derivative Liability - Addition
Derivative Liability - Additional Information (Details) - USD ($) $ in Millions | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Derivative [Line Items] | ||
Change in control discount rate | 14.00% | 14.00% |
Estimated fair value of derivative liability | $ 0.2 | $ 0.1 |
Minimum | ||
Derivative [Line Items] | ||
Change of control payment | $ 0 | $ 0 |
Change in control event percentage | 4.80% | 3.40% |
Maximum | ||
Derivative [Line Items] | ||
Change of control payment | $ 10.6 | $ 10.6 |
Change in control event percentage | 17.20% | 12.30% |
Related Party Transactions - Ad
Related Party Transactions - Additional Information (Details) | 1 Months Ended | 12 Months Ended | ||
Mar. 31, 2021USD ($) | Apr. 30, 2019USD ($)Agreement | Dec. 31, 2021USD ($) | Dec. 31, 2020USD ($) | |
David Schaffer, PhD, | Consulting Services and Services as Board of Director | ||||
Related Party Transaction [Line Items] | ||||
Related party expenses | $ 85,000 | $ 93,000 | ||
UC Regents | SRAs | ||||
Related Party Transaction [Line Items] | ||||
Related party expenses | 900,000 | 500,000 | ||
Number of sponsored research agreements entered | Agreement | 2 | |||
Agreement term | 3 years | |||
Amount due to related party | $ 1,500,000 | |||
UC Regents | Machine Learning SRA | ||||
Related Party Transaction [Line Items] | ||||
Agreement term | 3 years | |||
Amount due to related party | $ 1,400,000 | |||
Payment to related party | 400,000 | |||
UC Regents | SRAs and Machine Learning SRA | ||||
Related Party Transaction [Line Items] | ||||
Accounts payable, related party | $ 0 | $ 200,000 |
401(K) Plan - Additional Inform
401(K) Plan - Additional Information (Details) - USD ($) $ in Millions | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
2014 401(k) Plan | ||
Defined Contribution Plan Disclosure [Line Items] | ||
Employer contribution | $ 0.5 | $ 0.4 |