Document and Entity Information
Document and Entity Information - USD ($) | 12 Months Ended | ||
Dec. 31, 2020 | Mar. 03, 2021 | Jun. 30, 2020 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Amendment Flag | false | ||
Document Period End Date | Dec. 31, 2020 | ||
Document Fiscal Year Focus | 2020 | ||
Document Fiscal Period Focus | FY | ||
Trading Symbol | TSHA | ||
Entity Registrant Name | Taysha Gene Therapies, Inc. | ||
Entity Central Index Key | 0001806310 | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Current Reporting Status | Yes | ||
Entity Filer Category | Non-accelerated Filer | ||
Entity Small Business | true | ||
Entity Emerging Growth Company | true | ||
Entity Ex Transition Period | false | ||
Entity Shell Company | false | ||
Entity File Number | 001-39536 | ||
Entity Tax Identification Number | 84-3199512 | ||
Entity Address, Address Line One | 2280 Inwood Road | ||
Entity Address, City or Town | Dallas | ||
Entity Address, State or Province | TX | ||
Entity Address, Postal Zip Code | 75235 | ||
City Area Code | 214 | ||
Local Phone Number | 612-0000 | ||
Entity Common Stock, Shares Outstanding | 37,761,435 | ||
Title of 12(b) Security | Common stock, par value $0.00001 per share | ||
Security Exchange Name | NASDAQ | ||
Entity Incorporation, State or Country Code | DE | ||
Entity Interactive Data Current | Yes | ||
Document Annual Report | true | ||
Document Transition Report | false | ||
Entity Well-known Seasoned Issuer | No | ||
Entity Voluntary Filers | No | ||
ICFR Auditor Attestation Flag | false | ||
Entity Public Float | $ 0 |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) | Dec. 31, 2020 | Dec. 31, 2019 |
Current assets: | ||
Cash and cash equivalents | $ 251,253,000 | |
Prepaid expenses and other current assets | 6,626,000 | |
Deferred offering costs | 0 | $ 15,000 |
Total current assets | 257,879,000 | 15,000 |
Deferred lease asset | 715,000 | |
Property and equipment, net | 287,000 | |
Total assets | 258,881,000 | 15,000 |
Current liabilities | ||
Accounts payable | 1,994,000 | |
Accrued expenses and other current liabilities | 5,135,000 | 150,000 |
Total current liabilities | 7,129,000 | 150,000 |
Other non-current liabilities | 450,000 | |
Total liabilities | 7,579,000 | 150,000 |
Commitments and contingencies - Note 11 | ||
Stockholders' equity (deficit) | ||
Preferred stock, $0.00001 par value per share; 10,000,000 shares authorized and no shares issued and outstanding as of December 31, 2020; no shares authorized, issued and outstanding as of December 31, 2019 | ||
Common stock, $0.00001 par value per share; 200,000,000 shares authorized and 37,761,435 issued and outstanding as of December 31, 2020; 10,895,000 shares authorized, 10,894,999 issued and outstanding as of December 31, 2019 | ||
Additional paid-in capital | 312,428,000 | 980,000 |
Accumulated deficit | (61,126,000) | (1,115,000) |
Total stockholders’ equity (deficit) | 251,302,000 | (135,000) |
Total liabilities and stockholders' equity (deficit) | $ 258,881,000 | $ 15,000 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2020 | Dec. 31, 2019 |
Statement Of Financial Position [Abstract] | ||
Preferred stock, par value per share | $ 0.00001 | $ 0.00001 |
Preferred stock, shares authorized | 10,000,000 | 0 |
Preferred stock, shares issued | 0 | 0 |
Preferred stock, shares outstanding | 0 | 0 |
Common stock, par value per share | $ 0.00001 | $ 0.00001 |
Common stock, shares authorized | 200,000,000 | 10,895,000 |
Common stock, shares issued | 37,761,435 | 10,894,999 |
Common stock, shares outstanding | 37,761,435 | 10,894,999 |
Consolidated Statements of Oper
Consolidated Statements of Operations - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended |
Dec. 31, 2019 | Dec. 31, 2020 | |
Operating expenses: | ||
Research and development | $ 987 | $ 31,893 |
General and administrative | 128 | 11,109 |
Total operating expenses | 1,115 | 43,002 |
Loss from operations | (1,115) | (43,002) |
Other expense: | ||
Change in fair value of preferred stock tranche liability | 0 | (17,030) |
Interest income | 49 | |
Interest expense | (28) | |
Total other expense, net | (17,009) | |
Net loss | $ (1,115) | $ (60,011) |
Net loss per common share, basic and diluted | $ (0.12) | $ (3.40) |
Weighted average common shares outstanding, basic and diluted | 9,625,679 | 17,665,683 |
Consolidated Statements of Conv
Consolidated Statements of Convertible Preferred Stock and Stockholders' Equity (Deficit) - USD ($) $ in Thousands | Total | UT Southwestern License | Series A Convertible Preferred Stock | Series B Convertible Preferred Stock | Common Stock | Common StockUT Southwestern License | Additional Paid-in Capital | Additional Paid-in CapitalUT Southwestern License | Accumulated Deficit |
Balance at Sep. 20, 2019 | $ 0 | $ 0 | $ 0 | $ 0 | |||||
Convertible preferred stock balance, shares at Sep. 20, 2019 | 0 | 0 | |||||||
Convertible preferred stock balance at Sep. 20, 2019 | $ 0 | $ 0 | |||||||
Balance, shares at Sep. 20, 2019 | 0 | ||||||||
Issuance of Founders Shares, shares | 8,715,999 | ||||||||
Issuance of shares of common stock | $ 980 | $ 980 | |||||||
Issuance of shares of common stock, shares | 2,179,000 | ||||||||
Net loss | (1,115) | (1,115) | |||||||
Balance at Dec. 31, 2019 | $ (135) | 980 | (1,115) | ||||||
Balance, shares at Dec. 31, 2019 | 10,894,999 | 10,894,999 | |||||||
Issuance of convertible preferred stock | $ 28,345 | $ 95,815 | |||||||
Issuance of convertible preferred stock, shares | 10,000,000 | 5,647,048 | |||||||
Reclassification of preferred stock tranche liability upon issuance of Series A milestone shares | $ 18,080 | ||||||||
Conversion of Series A and Series B convertible preferred stock to common stock | $ (46,425) | $ (95,815) | |||||||
Conversion of Series A and Series B convertible preferred stock to common stock, shares | (10,000,000) | (5,647,048) | |||||||
Conversion of Series A and Series B convertible preferred stock to common stock | $ 142,240 | 142,240 | |||||||
Conversion of convertible to common stock, shares | 17,047,378 | ||||||||
Issuance of shares of common stock | 165,854 | 165,854 | |||||||
Issuance of shares of common stock, shares | 9,050,000 | ||||||||
Issuance of restricted stock award, shares | 769,058 | ||||||||
Stock-based compensation | 3,354 | 3,354 | |||||||
Net loss | (60,011) | (60,011) | |||||||
Balance at Dec. 31, 2020 | $ 251,302 | $ 312,428 | $ (61,126) | ||||||
Balance, shares at Dec. 31, 2020 | 37,761,435 | 37,761,435 |
Consolidated Statements of Co_2
Consolidated Statements of Convertible Preferred Stock and Stockholders' Equity (Deficit) (Parenthetical) $ in Thousands | 12 Months Ended |
Dec. 31, 2020USD ($) | |
Issuance of shares of common stock in initial public offering, net of offering costs and underwriting discounts and commissions | $ 15,145 |
Issuance of preferred stock tranche liability | 1,050 |
Series A Convertible Preferred Stock | |
Issuance of convertible preferred stock, offering costs | 605 |
Series B Convertible Preferred Stock | |
Issuance of convertible preferred stock, offering costs | $ 185 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) | 3 Months Ended | 12 Months Ended |
Dec. 31, 2019 | Dec. 31, 2020 | |
Cash flows from operating activities | ||
Net loss | $ (1,115,000) | $ (60,011,000) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Depreciation expense | 0 | 9,000 |
Change in fair value of preferred stock tranche liability | 0 | 17,030,000 |
Research and development license expense | 980,000 | 9,000,000 |
Stock-based compensation | 0 | 3,354,000 |
Changes in operating assets and liabilities: | ||
Prepaid expenses and other assets | 0 | (6,585,000) |
Accounts payable | 0 | 1,961,000 |
Accrued expenses and other liabilities | 135,000 | 4,513,000 |
Net cash used in operating activities | 0 | (30,729,000) |
Cash flows from investing activities | ||
Purchase of research and development licenses | 0 | (9,000,000) |
Purchase of property and equipment | 0 | (82,000) |
Net cash used in investing activities | 0 | (9,082,000) |
Cash flows from financing activities | ||
Proceeds from issuance of common stock in initial public offering, net of underwriting discounts and commission and other offering costs | 0 | 165,854,000 |
Proceeds from notes payable to related party | 0 | 1,673,000 |
Repayment of notes payable to related party | 0 | (1,673,000) |
Net cash provided by financing activities | 0 | 291,064,000 |
Net increase in cash and cash equivalents | 0 | 251,253,000 |
Cash at the beginning of the period | 0 | 0 |
Cash at the end of the period | 0 | 251,253,000 |
Supplemental disclosure of cash flow information: | ||
Cash paid for interest | 0 | 28,000 |
Supplemental disclosure of noncash investing and financing activities: | ||
Property and equipment in accounts payable and accrued expenses | 0 | 214,000 |
Reclassification of preferred stock tranche liability upon share issuance | 0 | 18,080,000 |
Allocation of preferred stock tranche liability | 0 | 1,050,000 |
Conversion of Series A and Series B convertible preferred stock to common stock | 0 | 142,240,000 |
Deferred offering costs not yet paid | 15,000 | |
Series A Convertible Preferred Stock | ||
Cash flows from financing activities | ||
Proceeds from issuances of convertible preferred stock, net of issuance costs | 0 | 29,395,000 |
Series B Convertible Preferred Stock | ||
Cash flows from financing activities | ||
Proceeds from issuances of convertible preferred stock, net of issuance costs | $ 0 | $ 95,815,000 |
Organization and Description of
Organization and Description of Business Operations | 12 Months Ended |
Dec. 31, 2020 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Organization and Description of Business Operations | Note 1—Organization and Description of Business Operations Taysha Gene Therapies, Inc. (the “Company” or “Taysha”) was originally formed under the laws of the State of Texas on September 20, 2019 (“Inception”). Taysha converted to a Delaware corporation on February 13, 2020, which had no impact to the Company’s par value or issued and authorized capital structure. Taysha is a patient-centric gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (“CNS”) in both rare and large patient populations. Stock Split On September 16, 2020, the Company effected a 1.0895-for-one stock split of its authorized, issued and outstanding shares of common stock and a proportional adjustment to the existing conversion ratios for each series of the Company’s convertible preferred stock as discussed in Note 7. Accordingly, all share and per share amounts for the periods presented in the accompanying consolidated financial statements and notes thereto have been adjusted retroactively, where applicable, to reflect this stock split and adjustment of the convertible preferred stock conversion ratios. On September 16, 2020, the Company also increased the number of shares of common stock authorized for issuance under the 2020 Equity Incentive Plan (the “Existing Plan”) to 3,845,294. Initial Public Offering On September 23, 2020, the Company’s registration statement on Form S-1 (File No. 333-248559) related to the initial public offering (“IPO”) of its common stock became effective and on September 28, 2020, the IPO closed. Pursuant to the IPO, the Company issued and sold 9,050,000 shares of common stock at a public offering price of $20.00 per share, which included 1,180,434 shares of common stock issued upon the exercise in full of the underwriters’ option to purchase additional shares. The Company received net proceeds of $165.9 million after deducting underwriting discounts and commissions and other offering costs. The shares began trading on the Nasdaq Global Select Market on September 24, 2020. On September 28, 2020, in connection with the closing of the IPO, 10,000,000 shares of Series A and 5,647,048 shares of Series B convertible preferred stock automatically converted into an aggregate of 17,047,378 shares of common stock with a conversion ratio of 1.0895 shares of common stock for each share of Series A and Series B convertible preferred stock. As a result of the IPO, including the underwriters’ exercise in full of their option to purchase additional shares, and the conversions of the Series A and B convertible preferred stock, the Company’s total number of outstanding shares increased by 26,097,378 immediately following the closing of the IPO. Upon the effectiveness of the Company’s registration statement related to the IPO, the Company’s 2020 Stock Incentive Plan (the “New Plan”) and 2020 Employee Stock Purchase Plan became effective. At that time, all shares reserved for issuance under the Existing Plan ceased to be available for issuance under such plan and became available for issuance under the New Plan. Liquidity and Capital Resources The Company has incurred operating losses since Inception and expects to continue to incur significant operating losses for the foreseeable future and may never become profitable. As of December 31, 2020, the Company had an accumulated deficit of $61.1 million. Prior to the closing of the Company’s IPO, between March and July 2020, the Company closed on the sale of an aggregate of 10,000,000 shares of Series A convertible preferred stock for gross proceeds of $30.0 million. Between July and August 2020, the Company closed on the sale of an aggregate of 5,647,048 shares of Series B convertible preferred stock for gross proceeds of $96.0 million. Due to the proceeds received from the IPO and the sale of its Series A and Series B convertible preferred stock, management believes that its existing financial resources are sufficient to continue operating activities at least one year past the issuance date of these consolidated financial statements. Future capital requirements will depend on many factors, including the timing and extent of spending on research and development and the market acceptance of the Company’s products. The Company will need to obtain additional financing in order to complete clinical studies and launch and commercialize any product candidates for which it receives regulatory approval. There can be no assurance that such financing will be available or will be on terms acceptable to the Company. During December 2019, the novel coronavirus (“COVID-19”) emerged and subsequently spread worldwide. The World Health Organization declared COVID-19 a global pandemic in March 2020, resulting in federal, state and local governments and private entities implementing various restrictions, including travel restrictions, restrictions on public gatherings, stay at home orders, and advisories and quarantining people who may have been exposed to the virus. The Company has been actively monitoring COVID-19 and its impact globally. Management believes the financial results for the year ended December 31, 2020 were not significantly impacted by COVID-19. In addition, management believes the remote working arrangements and travel restrictions imposed by various governmental jurisdictions have had limited impact on the Company’s ability to maintain internal operations during the year ended December 31, 2020. The full extent to which the COVID-19 pandemic will directly or indirectly impact the Company’s business, results of operations and financial condition will depend on future developments that are highly uncertain, including as a result of new information that may emerge concerning COVID-19 and the actions taken to contain it or treat COVID-19. |
Significant Accounting Policies
Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2020 | |
Accounting Policies [Abstract] | |
Significant Accounting Policies | Note 2—Significant Accounting Policies Basis of Presentation The Company’s financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America (“U.S. GAAP”) as determined by the Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) and include all adjustments necessary for the fair presentation of the Company’s financial position for the periods presented. Principles of Consolidation The accompanying consolidated financial statements include the accounts of Taysha and its inactive wholly owned U.S. subsidiaries that were incorporated during 2020. All intercompany transactions and balances have been eliminated in consolidation. Emerging Growth Company From time to time, new accounting pronouncements are issued by FASB, or other standard setting bodies and adopted by the Company as of the specified effective date. Unless otherwise discussed, the impact of recently issued standards that are not yet effective will not have a material impact on the Company’s financial statements upon adoption. Under the Jumpstart Our Business Startups Act of 2012, as amended, the Company meets the definition of an emerging growth company, and has elected the extended transition period for complying with new or revised accounting standards, which delays the adoption of these accounting standards until they would apply to private companies. Use of Estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of expenses during the reporting period. The most significant estimates and assumptions in the Company’s financial statements relate to the determination of the fair value of the common stock prior to the IPO (as an input into stock-based compensation), estimating preclinical manufacturing accruals and accrued or prepaid research and development expenses, and the valuation of the preferred stock tranche liability. These estimates and assumptions are based on current facts, historical experience and various other factors believed to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities and the recording of expenses that are not readily apparent from other sources. Actual results may differ materially from these estimates. To the extent there are material differences between the estimates and actual results, the Company’s future results of operations will be affected. Risks and Uncertainties The Company is subject to risks common to companies in the biotechnology industry, including, but not limited to, development by the Company or its competitors of technological innovations, risks of failure of clinical studies, dependence on key personnel, protection of proprietary technology, compliance with government regulations, and ability to transition from preclinical manufacturing to commercial production of products. The Company’s product candidates require approvals from the FDA and comparable foreign regulatory agencies prior to commercial sales in their respective jurisdictions. There can be no assurance that any product candidates will receive the necessary approvals. If the Company was denied approval, approval was delayed or the Company was unable to maintain approval for any product candidate, it could have a materially adverse impact on the Company. Segments Operating segments are identified as components of an enterprise about which separate discrete financial information is available for evaluation by the chief operating decision-maker in making decisions regarding resource allocation and assessing performance. The Company views its operations and manages its business as a single operating segment, which is the business of developing AAV-based gene therapies for the treatment of rare monogenic diseases of the CNS. As of December 31, 2020 and 2019, the Company did not have assets located outside of the United States. Cash and Cash Equivalents Cash and cash equivalents consist of funds held in a standard checking account and a standard savings account. The Company considers all highly liquid investments with an original maturity of three months or less at the date of purchase to be cash equivalents. As of December 31, 2020 and 2019, respectively, the Company had no cash equivalents. Concentrations of Credit Risk Financial instruments that potentially subject the Company to significant concentration of credit risk consist primarily of cash and cash equivalents. Periodically, the Company may maintain deposits in financial institutions in excess of government insured limits. Management believes that the Company is not exposed to significant credit risk as the Company’s deposits are held at financial institutions that management believes to be of high credit quality. The Company has not experienced any losses on these deposits. Fair Value of Financial Instruments The Company’s financial assets and liabilities are accounted for in accordance with ASC 820, Fair Value Measurements and Disclosures which defines fair value as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. The fair value hierarchy requires an entity to maximize the use of observable inputs when measuring fair value and classifies those inputs into three levels: Level 1—Observable inputs, such as quoted prices in active markets for identical assets or liabilities. Level 2—Inputs other than Level 1 inputs that are either directly or indirectly observable, 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 instrument’s anticipated life. Level 3—Unobservable inputs in which there is little or no market data, which require the reporting entity to develop its own assumptions. To the extent the valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair values requires more judgement. Accordingly, the degree of judgement exercised by the Company in determining fair value is greatest for instruments categorized as Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement. The carrying values reported in the Company’s consolidated balance sheets for cash and cash equivalents, prepaid expenses and other current assets, accounts payable, accrued expenses and other current liabilities are reasonable estimates of their fair values due to the short-term nature of these items. Deferred Offering Costs The Company capitalizes certain legal, professional, accounting and other third-party fees that are directly associated with in-process equity financings as deferred offering costs until such financings are consummated. After consummation of the equity financing, these costs are recorded in stockholders’ deficit as a reduction of additional paid-in capital generated as a result of an equity financing. Should the in-process equity financing be abandoned, the deferred offering costs will be expensed immediately as a charge to operating expenses in the statements of operations. As of December 31, 2020 and 2019, there was $0 and $15,000, respectively, of deferred offering costs capitalized on the consolidated balance sheets. The $15,000 capitalized as of December 31, 2019 related to the Company’s Series A convertible preferred stock financing that closed in March 2020 (see Note 7). Deferred offering costs, consisting of legal, accounting, and filing fees directly relating to the IPO and the Company’s Series A and Series B preferred stock financings, were capitalized and offset against the related proceeds upon the completion of the offerings in 2020. Upon completion of the IPO in September 2020, approximately $2 . Property and Equipment, net Property and equipment are stated at cost less accumulated depreciation and consist solely of computer equipment and laboratory equipment. Depreciation expense is recognized using the straight-line method over its estimated useful life of three to five years. Upon retirement or sale, the cost of assets disposed of and the related accumulated depreciation are removed from the accounts and any resulting gain or loss is included in loss from operations. Expenditures for repairs and maintenance are charged to expense as incurred. Impairment of Long-Lived Assets The Company evaluates its long-lived assets, which consist of property and equipment, for impairment whenever events or changes in circumstances indicate that the carrying amount of such assets may not be recoverable. Recoverability of assets to be held and used is measured by a comparison of the carrying amount of an asset to the future undiscounted net cash flows expected to be generated by the asset. If such assets are considered to be impaired, the impairment to be recognized is measured by the amount by which the carrying amount of the asset exceeds the fair value of the asset. There were no impairment losses recognized during the year ended December 31, 2020 and the period from Inception to December 31, 2019. Convertible Preferred Stock The Company recorded shares of its convertible preferred stock at their respective fair values on the dates of issuance, net of issuance costs. The Company applied the guidance in ASC 480-10-S99-3A, SEC Staff Announcement: Classification and Measurement of Redeemable Securities and therefore classified the Series A convertible preferred stock as mezzanine equity. The convertible preferred stock was recorded outside of stockholders’ deficit because, in the event of certain deemed liquidation events considered not solely within the Company’s control, such as a merger, acquisition and sale of all or substantially all of the Company’s assets (a “Deemed Liquidation Event”), the convertible preferred stock would become redeemable at the option of the holders. In the event of a change of control of the Company, proceeds received from the sale of such shares would have been distributed in accordance with the liquidation preferences set forth in the Company’s Amended and Restated Certificate of Incorporation. The Company determined not to adjust the carrying values of the convertible preferred stock to the liquidation preferences of such shares because of the uncertainty of whether or when such an event would occur. All shares of the Company’s previously issued and outstanding convertible preferred stock were converted into shares of common stock upon the closing of the IPO (see Note 7). Preferred Stock Tranche Liability The Company determined that its obligation to issue, and the Company’s investors’ right to purchase, additional shares of Series A convertible preferred stock pursuant to the milestone closings (see Note 7) represented a freestanding financial instrument (the “tranche liability”). The tranche liability was initially recorded at fair value. The proceeds from the sale of the convertible preferred stock were first allocated to the fair value of the tranche liability with the remaining proceeds from the sale of the convertible preferred stock allocated to the Series A convertible preferred stock. The tranche liability was remeasured at each reporting period and upon the exercise or expiration of the obligation, with gains and losses arising from subsequent changes in its fair value recognized in other expense in the consolidated statements of operations. At the time of the exercise or expiration of the tranche liability, any remaining value of the tranche liability was reclassified to convertible preferred stock on the consolidated balance sheets. Research and Development The Company has entered into research and development contracts with research institutions and other companies. These agreements are generally cancelable, and related payments are recorded as research and development expenses as incurred. Payments for these activities are based on the terms of the individual agreements, which may differ from the pattern of costs incurred, and are reflected on the consolidated balance sheets as prepaid or accrued expenses. The Company records accruals for estimated ongoing research costs. When evaluating the adequacy of the accrued liabilities, the Company analyzes progress of the studies, including the phase or completion of events, invoices received and contracted costs. Research and development costs primarily consist of payroll, stock-based compensation, certain manufacturing costs, laboratory costs and other supplies, and the cost to acquire licenses. Costs incurred in obtaining technology licenses through asset acquisitions are charged to research and development expense if the licensed technology has not reached technological feasibility and has no alternative future use. Stock-Based Compensation The Company accounts for all stock-based payments to employees and non-employees, including grants of stock options, restricted stock awards, or RSAs, and restricted stock units, or RSUs, based on their respective grant date fair values. The Company estimates the fair value of stock option grants using the Black-Scholes option pricing model, which is affected principally by the estimated fair value of shares of the Company’s common stock and requires management to make a number of other assumptions, including the expected life of the option, the volatility of the underlying shares, the risk-free interest rate and expected dividends. Expected volatility is based on the historical share volatility of a set of comparable publicly traded companies over a period of time equal to the expected term of the options. Due to the lack of historical exercise history, the expected term of the Company’s stock options is determined using the “simplified” method. The risk-free interest rate is determined by reference to the U.S. Treasury yield curve in effect at the time of grant of the award for time periods approximately equal to the expected term of the award. Expected dividend yield is zero based on the fact that the Company has never paid cash dividends and does not expect to pay any cash dividends in the foreseeable future. Prior to September 23, 2020, the fair value of common stock underlying the Company’s stock options, RSAs and RSUs was estimated by the Company’s board of directors considering, among other things, contemporaneous valuations of the Company’s common stock prepared by unrelated third-party valuation firms. After the IPO, the fair value of common stock is based on the closing price of the Company’s common stock on the Nasdaq Global Select Market as reported on the date of the grant. The RSAs and RSUs are valued based on the fair value of the Company’s common stock on the date of grant. The Company expenses stock-based compensation related to stock options, RSAs and RSUs over the requisite service period using the straight-line method. All stock-based compensation costs are recorded in research and development expense or general and administrative expense in the consolidated statements of operations based upon the respective employee’s roles within the Company. Forfeitures are recorded as they occur. Income Taxes Income taxes are recorded in accordance with ASC 740, Income Taxes (“ASC 740”), which provides for deferred taxes using an asset and liability approach. The Company recognizes deferred tax assets and liabilities for the expected future tax consequences of events that have been included in the financial statements or tax returns. Deferred tax assets and liabilities are determined based on the difference between the financial statement and tax bases of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse, and net operating loss (“NOL”) carryforwards and research and development tax credit (“R&D Credit”) carryforwards. Valuation allowances are provided, if based upon the weight of available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized. The Company has recorded a full valuation allowance to reduce its net deferred income tax assets to zero. In the event the Company were to determine that it would be able to realize some or all its deferred income tax assets in the future, an adjustment to the deferred income tax asset valuation allowance would increase income in the period such determination was made. The Company accounts for uncertain tax positions in accordance with the provisions of ASC 740. When uncertain tax positions exist, the Company recognizes the tax benefit of tax positions to the extent that the benefit would more likely than not be realized assuming examination by the taxing authority. The determination as to whether the tax benefit will more likely than not be realized is based upon the technical merits of the tax position as well as consideration of the available facts and circumstances. At December 31, 2019, the Company had no liability for income tax associated with uncertain tax positions. During the year ended December 31, 2020, the Company recorded gross unrecognized tax benefits that were not significant. The unrecognized tax benefits, if recognized, would not affect the effective income tax rate due to the valuation allowance that currently offsets deferred tax assets. The Company does not expect the unrecognized tax benefits to change significantly over the next 12 months. The Company would recognize any corresponding interest and penalties associated with its income tax positions in income tax expense. There was no income tax interest or penalties incurred for the year ended December 31, 2020 or for the period from Inception to December 31, 2019. Comprehensive Loss Comprehensive loss is equal to net loss as presented in the accompanying consolidated statements of operations, as the Company did not have any other comprehensive income or loss for the periods presented. Recently Adopted Accounting Pronouncements In May 2014, the FASB issued ASC 606, Revenue from Contracts with Customers (“ASC 606”). This guidance applies to any entity that either enters into contracts with customers to transfer goods or services or enters into contracts for the transfer of nonfinancial assets unless those contracts are within the scope of other standards. The core principle of this guidance is that an entity should recognize revenue to depict the transfer of promised goods or services to customers in an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services. This guidance supersedes existing revenue recognition guidance, including most industry-specific guidance, as well as certain related guidance on accounting for contract costs. The Company early adopted ASC 606 upon its Inception. As the Company does not have any contracts with customers, the adoption of this guidance did not have any impact on the Company’s consolidated financial statements. In June 2018, the FASB issued Accounting Standards Update (“ASU”) No. 2018-07, Compensation – Stock Compensation (Topic 718) (“ASU 2018-07”). This update is intended to reduce cost and complexity and to improve financial reporting for share-based payments issued to non-employees (for example, service providers, external legal counsel, suppliers, etc.). The ASU expands the scope of Topic 718, Compensation—Stock Compensation, which currently only includes share-based payments issued to employees, to also include share-based payments issued to non-employees for goods and services. Consequently, the accounting for share-based payments to non-employees and employees will be substantially aligned. The Company early adopted ASU 2018-07 upon its Inception. The adoption of this ASU did not have a material effect on the Company’s consolidated financial statements at Inception. The Company applied this accounting pronouncement to the issuance of shares to the Board of Regents of the University of Texas System (see Note 5). In August 2018, the FASB issued ASU No. 2018-13, Fair Value Measurement (ASC Topic 820) (“ASU 2018-13”), which modifies, removes and adds certain disclosure requirements on fair value measurements based on the FASB Concepts Statement, Conceptual Framework for Financial Reporting—Chapter 8: Notes to Financial Statements. The amendments on changes in unrealized gains and losses, the range and weighted-average of significant unobservable inputs used to develop Level 3 fair value measurements and the narrative description of measurement uncertainty should be applied prospectively for only the most recent interim or annual period presented in the initial fiscal year of adoption. All other amendments should be applied retrospectively to all periods presented upon their effective date. The Company adopted ASU 2018-13 as of January 1, 2020 for the annual period. The adoption of the guidance did not have a material impact on the Company’s consolidated financial statements. In November 2018, the FASB issued ASU No. 2018-18 – Collaborative Arrangements, which clarifies that certain transactions between collaborative arrangement participants should be accounted for as revenue when the collaborative arrangement participant is a customer in the context of a unit of account and precludes recognizing as revenue consideration received from a collaborative arrangement participant if the participant is not a customer. The Company early adopted this standard as of January 1, 2020 for the annual period and it will be applied to interim periods after December 31, 2020. This ASU requires retrospective adoption to the date the Company adopted ASC 606, which was upon its Inception, by recognizing a cumulative-effect adjustment to the opening balance of retained earnings of the earliest annual period presented. As the Company does not have any contracts with customers or collaborative arrangements, the adoption of this guidance did not have any impact on the Company’s consolidated financial statements. Recently Issued Accounting Pronouncements In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842), as amended, with guidance regarding the accounting for and disclosure of leases. This update requires lessees to recognize the liabilities related to all leases, including operating leases, with a term greater than 12 months on the balance sheets. This update also requires lessees and lessors to disclose key information about their leasing transactions. This guidance will become effective for the Company for annual reporting periods beginning after December 15, 2021 and interim periods within fiscal years beginning after December 15, 2022. The Company is currently evaluating the impact of this standard on its consolidated financial statements. In December 2019, the FASB issued ASU No. 2019-12, Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes (“ASU 2019-12”), which is intended to simplify various aspects related to accounting for income taxes. ASU 2019-12 removes certain exceptions to the general principles in ASC 740 and also clarifies and amends existing guidance to improve consistent application. This guidance is effective for fiscal years beginning after December 15, 2021, and interim periods within fiscal years beginning after December 15, 2022, with early adoption permitted. The Company is currently evaluating the impact of this standard on its consolidated financial statements. |
Property and Equipment
Property and Equipment | 12 Months Ended |
Dec. 31, 2020 | |
Property Plant And Equipment [Abstract] | |
Property and Equipment | Note 3—Property and Equipment Property and Equipment consisted of the following (in thousands): December 31, December 31, 2020 2019 Computer equipment $ 95 $ — Construction in progress 201 — 296 — Accumulated depreciation (9 ) — Property and equipment, net $ 287 $ — Depreciation expense was $9,000 for the year ended December 31, 2020. There was no depreciation expense in prior periods. |
Supplemental Financial Informat
Supplemental Financial Information | 12 Months Ended |
Dec. 31, 2020 | |
Balance Sheet Related Disclosures [Abstract] | |
Supplemental Financial Information | Note 4—Supplemental Financial Information Accrued expenses and other current liabilities consisted of the following (in thousands): December 31, December 31, 2020 2019 Accrued research and development $ 2,106 $ 7 Accrued compensation 1,766 — Accrued professional fees 999 122 Other 264 21 Total accrued expenses and other current liabilities $ 5,135 $ 150 Prepaid expenses and other current assets consisted of the following (in thousands): December 31, December 31, 2020 2019 Prepaid insurance $ 2,480 $ — Prepaid research and development 2,462 — Prepaid clinical trial 944 — Other 740 — Total prepaid expenses and other current assets $ 6,626 $ — |
Research, Collaboration, Grant
Research, Collaboration, Grant and License Agreements | 12 Months Ended |
Dec. 31, 2020 | |
Research Collaboration And License Agreements [Abstract] | |
Research, Collaboration, Grant and License Agreements | Note 5—Research, Collaboration, Grant and License Agreements UT Southwestern Agreement On November 19, 2019, the Company entered into a research, collaboration and license agreement (“UT Southwestern Agreement”) with the Board of Regents of the University of Texas System (“UT System”) on behalf of The University of Southwestern Medical Center (“UT Southwestern”). Under the UT Southwestern Agreement, UT Southwestern is primarily responsible for preclinical development activities with respect to licensed products for use in certain specified indications (up to IND-enabling studies), and the Company is responsible for all subsequent clinical development and commercialization activities with respect to the licensed products. UT Southwestern will conduct such preclinical activities for a two-year period under mutually agreed upon sponsored research agreements that were entered into beginning in April 2020. During the initial research phase, the Company has the right to expand the scope of specified indications under the UT Southwestern Agreement. In connection with the UT Southwestern Agreement, the Company obtained an exclusive, worldwide, royalty-free license under certain patent rights of UT Southwestern and a non-exclusive, worldwide, royalty-free license under certain know-how of UT Southwestern, in each case to make, have made, use, sell, offer for sale and import licensed products for use in certain specified indications. Additionally, the Company obtained a non-exclusive, worldwide, royalty-free license under certain patents and know-how of UT Southwestern for use in all human uses, with a right of first refusal to obtain an exclusive license under certain of such patent rights and an option to negotiate an exclusive license under other of such patent rights. The Company is required to use commercially reasonable efforts to develop, obtain regulatory approval for, and commercialize at least one licensed product. On April 2, 2020, the Company amended the UT Southwestern Agreement to include the addition of another licensed product and certain indications, and a right of first refusal to the Company over certain patient dosing patents. No additional consideration was transferred in connection with this amendment. The UT Southwestern Agreement expires on a country-by-country and licensed product-by-licensed product basis upon the expiration of the last valid claim of a licensed patent in such country for such licensed product. After the initial research term, the Company may terminate the agreement, on an indication-by-indication and licensed product-by-licensed product basis, at any time upon specified written notice to UT Southwestern. Either party may terminate the agreement upon an uncured material breach of the agreement or insolvency of the other party. In November 2019, as partial consideration for the license rights granted under the UT Southwestern Agreement, the Company issued 2,179,000 shares of its common stock, or 20% of its then outstanding fully-diluted common stock, to UT Southwestern. As additional consideration, UT Southwestern was entitled to receive additional shares if their holdings fell below 10% on a fully-diluted basis before or as a result of the completion of a qualified financing. In March 2020, following the initial closing of the Series A convertible preferred stock agreement, which met the definition of such qualified financing, the anti-dilution feature expired and no additional shares were issued. The Company does not have any future milestone or royalty obligations to UT Southwestern under the UT Southwestern Agreement other than costs related to maintenance of patents. The Company also had the right of first refusal for any shares that UT Southwestern wishes to sell that officially expired upon the closing of the IPO. UT Southwestern did not sell any shares before the closing of the IPO. The fair value of the shares issued was determined to be $980,000 which was recorded as an increase to common stock and additional paid-in capital and a corresponding research and development expense during the period from Inception through December 31, 2019. The cost to acquire this license is recorded within research and development expense in the consolidated statements of operations because the license relates to specific preclinical research and development activities that do not have an alternative future use. As the acquisition of the license was settled through the issuance of shares of the Company’s common stock, this transaction fell within the scope of ASC Topic 718 since equity was issued in exchange for goods (the license). Specifically, the Company recorded the cost of the license as a non-employee share based payment, measured at the grant date fair value of the common stock of $0.45 per share. The common shares were equity-classified. The anti-dilution provision was concluded to represent a performance condition tied to a future liquidity event, which was not considered probable of occurring at December 31, 2019 since it was deemed outside of the Company’s control. Since there was an absence of a public trading market for the Company’s common stock during the period from Inception to December 31, 2019, the valuation of the common stock issued to UT System was performed using methodologies, approaches, and assumptions consistent with the American Institute of Certified Public Accountants Audit and Accounting Practice Aid Series: Valuation of Privately-Held-Company Equity Securities Issued as Compensation. The estimated fair value of the Company’s common stock was determined with the assistance of a third-party specialist using an Option-Pricing Method (“OPM”). Under the OPM, the Company used a backsolve method so that the enterprise value equaled the contemplated value of the Company determined by the Series A convertible preferred stock financing transaction which initially closed on March 4, 2020 (see Note 7), and giving consideration to any value-generating events that may have occurred between March 4, 2020 and the date the common shares were issued to UT System on November 19, 2019. A discount for lack of marketability (“DLOM”) of 20% was applied to derive the valuation price. The DLOM used was derived from the then-current estimates of the time to a liquidity event made by the Company’s board of directors, with input from the Company’s senior management. The estimates are based, in part, on subjective assumptions. Queen’s Agreement In late December 2019, the Company entered into a research grant agreement (“RGA”) with Queen’s University at Kingston (“Queen’s”), for certain research and development activities related to the generation of AAV9 vector. The Company committed to fund $3.8 million under the RGA with Queen’s. The Company agreed to issue Queen’s a promise-to-pay note whereby any amounts paid directly by Queen’s for the manufacture of the vector for use in the funded research activities, to the extent such amounts have not already been funded by the Company to Queen’s, become a loan obligation for the Company (the “Note”), subject to an interest rate of 6%. Any amounts outstanding under the Note were required to be repaid, along with any accrued interest, by or before June 30, 2020. In the event of default, any amount outstanding shall be deemed immediately payable by RA Session II, the Company’s President and Chief Executive Officer, as a personal guarantor (see Note 6). For the period from Inception through December 31, 2019, the Company did not incur any expenses associated with the Queen’s RGA, and no amounts were due or outstanding under the Note as of December 31, 2019. For the year ended December 31, 2020, the Company paid all expenses associated with the Queen’s RGA, thus no amounts were due or outstanding under the Note as of December 31, 2020, and the promise-to-pay has therefore expired. On February 21, 2020, the Company entered into a license agreement with Queen’s (the “Queen’s Agreement”) to obtain the exclusive perpetual, royalty-bearing license, with the right to sublicense through multiple tiers, under certain patent rights and know-how of Queen’s, including certain improvements to such patent rights and know-how, to develop products in any field which use one or more valid claims of the patents licensed under the Queen’s Agreement (the “Licensed Patents”), or the technology, information and intellectual property related to the patents licensed under the Queen’s Agreement (together with the Licensed Patents, the “Licensed Products”), and to make, have made, use, sell, offer for sale, import and export Licensed Products and otherwise exploit such patents and know-how for use in certain specified indications. In exchange for the rights granted to the Company, the Company made a cash payment of $3.0 million in April 2020 which is recorded in research and development expenses in the consolidated statements of operations and included as an investing outflow in the consolidated statements of cash flows since the acquired license does not have an alternative future use. The Company is obligated to make aggregate cash payments of up to $10.0 million upon the completion of a combination of regulatory milestones and up to $10.0 million upon the completion of a combination of commercial milestones. In further consideration of the rights granted, beginning with the Company’s first commercial sale of the Licensed Products, the Company will also pay an annual earned royalty in the low single digits on net sales of Licensed Products, subject to certain customary reductions, and a percentage of non-royalty sublicensing revenue ranging in the low double digits. Royalties are payable, on a Licensed Products-by-Licensed Products and a country-by-country basis, until expiration of the last valid claim of a Licensed Patent covering such Licensed Products in such country and the expiration of any regulatory exclusivity for such Licensed Products in such country. Abeona CLN1 Agreements In August 2020, the Company entered into license and inventory purchase agreements with Abeona Therapeutics Inc. (“Abeona”) for worldwide exclusive rights to certain intellectual property rights and know-how relating to the research, development and manufacture of ABO-202, an AAV-based gene therapy for CLN1 disease (one of the forms of Batten disease). Under the terms of the agreements, the Company made initial cash payments to Abeona of $3.0 million for the upfront license fee and $4.0 million for purchase of clinical materials and reimbursement for previously incurred development costs in October 2020. In exchange for the license rights, the Company recorded an aggregate of $7.0 million within research and development expenses in the consolidated statements of operations and included the license fee of $3.0 million as an investing outflow in the consolidated statements of cash flows since the acquired license does not have an alternative future use. The Company is obligated to make up to $26.0 million in regulatory-related milestones and up to $30.0 million in sales-related milestones per licensed CLN1 product. The Company will also pay an annual earned royalty in the high single digits on net sales of any licensed CLN1 products. The license agreement expires on a country-by-country and licensed product-by-licensed product basis upon the expiration of the last royalty term of a licensed product. Either party may terminate the agreement upon an uncured material breach of the agreement or insolvency of the other party. The Company may terminate the license agreement for convenience upon specified prior written notice to Abeona. Abeona Rett Agreement On October 29, 2020, the Company entered into a license agreement (the “Abeona Rett Agreement”) with Abeona pursuant to which the Company obtained an exclusive, worldwide, royalty-bearing license, with the right to grant sublicenses under certain patents, know-how and materials originally developed by the University of North Carolina at Chapel Hill, the University of Edinburgh and Abeona to research, develop, manufacture, have manufactured, use, and commercialize licensed products for gene therapy and the use of related transgenes for Rett syndrome. Subject to certain obligations of Abeona, the Company is required to use commercially reasonable efforts to develop at least one licensed product and commercialize at least one licensed product in the United States. In connection with the Abeona Rett Agreement, the Company paid Abeona a one-time upfront license fee of $3.0 million which is recorded in research and development expenses in the consolidated statements of operations and included as an investing outflow in the consolidated statements of cash flows since the acquired license does not have an alternative future use. The Company is obligated to pay Abeona up to $26.5 million in regulatory-related milestones and up to $30.0 million in sales-related milestones per licensed Rett product and high single-digit royalties on net sales of licensed Rett products. Royalties are payable on a licensed product-by-licensed product and country-by-country basis until the latest of the expiration or revocation or complete rejection of the last licensed patent covering such licensed product in the country where the licensed product is sold, the loss of market exclusivity in such country where the product is sold, or, if no licensed product exists in such country and no market exclusivity exists in such country, ten years from first commercial sale of such licensed product in such country. The Abeona Rett Agreement expires on a country-by-country and licensed product-by-licensed product basis upon the expiration of the last royalty term of a licensed product. Either party may terminate the agreement upon an uncured material breach of the agreement or insolvency of the other party. The Company may terminate the agreement for convenience upon specified prior written notice to Abeona. Catalent Agreements During October and November 2020, the Company entered into two agreements (the “Catalent Agreements”) with Catalent Maryland, Inc. (“Catalent”) to support the development and manufacturing of the Company’s gene therapies for CLN1 and Rett syndrome at Catalent’s Maryland-based gene therapy facilities. The Company engaged Catalent, which is a Contract Development and Manufacturing Organization (CDMO), to conduct services related to technology transfer, process development, analytical development, qualification, project administration and manufacture of CLN1 and Rett syndrome gene therapies suitable for use in nonclinical, United States and European Union Phase 1/2 clinical trials. In connection with the Catalent Agreements, the Company paid Catalent an upfront project initiation fee of $2.0 million which is recorded as a prepaid expense in the consolidated balance sheets. The fee will be credited toward fees and expenses incurred under the Catalent Agreements. The Company therefore reduces the prepaid expense concurrently with expenses as they are incurred. |
Related Party Transactions
Related Party Transactions | 12 Months Ended |
Dec. 31, 2020 | |
Related Party Transactions [Abstract] | |
Related Party Transactions | Note 6—Related Party Transactions RA Session II, President and Chief Executive Officer and a member of the Company’s board of directors, is a guarantor under the Guaranty and Security Agreement between himself, Queen’s and the Company, and in the event of the Company’s failure to fund its obligations under the RGA with Queen’s, has personally guaranteed payments due by the Company to Queen’s. In addition, the Company entered into two secured promissory notes with Mr. Session in January 2020 for an aggregate of $1.67 million, with 10% interest. The Company secured the notes with a first priority security interest in certain assets of the Company. As of December 31, 2020, the Company has repaid the notes in full. As a result, Mr. Session released his security interest in the collateral. During the year ended December 31, 2020, the Company incurred and paid approximately $28,000 of interest expense related to the notes. In March 2020, the Company entered into a services agreement with PBM Capital Group, LLC (“PBM”), an affiliate of PBM TGT Holdings, LLC whereby PBM provides accounting and other administrative and management services related to payroll administration, human resources, bookkeeping, preparation of financial statements and tax returns, accounts payable and receivable, and other similar functions for a fee of $2,500 per month. Paul B. Manning, a member of the Company’s board of directors and a holder of more than 5% of the Company’s capital stock, is the Chief Executive Officer of PBM Capital Group, LLC |
Stockholders' Equity (Deficit),
Stockholders' Equity (Deficit), Convertible Preferred Stock and Tranche Liability | 12 Months Ended |
Dec. 31, 2020 | |
Equity [Abstract] | |
Stockholders' Equity (Deficit), Convertible Preferred Stock and Tranche Liability | Note 7— Stockholders’ Equity (Deficit), Convertible Preferred Stock and Tranche Liability At December 31, 2019, the Company was authorized to issue 10,895,000 shares of common stock with a par value of $0.00001 per share. RA Session II and one other individual were issued 8,661,524 and 54,475 shares of the Company’s common stock (“Founders Shares”), respectively, upon the formation of the Company on September 20, 2019. RA Session II and the other individual had not paid the Company for the aggregate par value, which approximated fair value at Inception, for these Founders Shares as of December 31, 2019. All amounts owed for the issuance of these Founders Shares were settled in July 2020. On November 19, 2019, the Company issued 2,179,000 shares of the Company’s common stock to UT System in exchange for a license agreement. The fair value of the shares on the date of issuance was $980,000 in the aggregate. Except for the issuance of common stock to UT System in connection with the UT Southwestern Agreement, the Company did not grant any equity-based awards during the period from its Inception through December 31, 2019. Authorized shares The Company amended its certificate of incorporation on March 4, 2020, July 2, 2020 and again on July 28, 2020 such that the total number of shares of common stock authorized to be issued was increased to 32,685,000, and the total number of shares of preferred stock authorized to be issued was increased to 15,647,052, of which 10,000,000 preferred shares were designated Series A convertible preferred stock and 5,647,052 were designated Series B convertible preferred stock. On September 28, 2020, the Company amended its certificate of incorporation such that the total number of shares of common stock authorized to be issued was increased to 200,000,000, and the total number of shares of new preferred stock authorized to be issued was 10,000,000. As of December 31, 2020, no shares of preferred stock were issued or outstanding. IPO On September 28, 2020, the Company issued an aggregate of 7,869,566 shares of common stock in the IPO, and on September 29, 2020, the Company issued an aggregate of 1,180,434 shares of common stock upon the underwriters’ exercise in full of their option to purchase additional shares, each at the public offering price of $20.00 per share less underwriting discounts and commissions. In connection with the IPO, the Company received gross proceeds of $181.0 million, which was offset by issuance costs, including underwriters’ discounts and commissions, of approximately $15.1 million. In connection with the closing of the IPO, 10,000,000 shares of Series A convertible preferred stock and 5,647,048 shares of Series B convertible preferred stock automatically converted into an aggregate of 17,047,378 shares of common stock with a conversion ratio of 1.0895 shares of common stock for each share of Series A and Series B convertible preferred stock then outstanding. As a result of the IPO, the underwriters’ exercise of their option, and the conversions of the Series A and B convertible preferred stock, the Company’s total number of outstanding shares increased by 26,097,378 immediately following the closing of the IPO. Series A and B convertible preferred stock On March 4, 2020, the Company entered into a purchase agreement (the “Series A Purchase Agreement”) providing for a private placement of up to 10,000,000 shares of Series A convertible preferred stock at an original issuance price of $3.00 per share, subject to separate closings, including: (1) 6,000,000 shares at the initial closing on March 4, 2020, and (2) 2,000,000 shares at each of two subsequent closings triggered by the achievement of specific clinical milestones. The Series A Purchase Agreement obligated the Company to issue and sell and the Series A investors to purchase up to a total of 4,000,000 additional shares of Series A convertible preferred stock (the “Milestone Shares”) at the same price per share upon the achievement of certain defined clinical milestones (the “tranche liability”). The determination as to whether the milestone events had been met was subject to certification by the Board of Directors. Each Series A investor had the right, but not the obligation, to purchase all or any portion of the Milestone Shares at any time in its sole option and in its sole and absolute discretion, whether or not the Company had achieved the applicable clinical milestone. On June 30, 2020, several affiliated Series A investors elected to exercise in full their options to purchase 200,000 shares, representing all of their remaining pro-rata portion of the Milestone Shares, prior to the Company’s achievement of the clinical milestones for gross proceeds of $0.6 million. The remainder of the Series A investors exercised in full their options to purchase 3,800,000 shares, representing all of their remaining pro-rata portion of the Milestone Shares, prior to the Company’s achievement of the clinical milestones, for gross proceeds of $11.4 million between July 1, 2020 and July 2, 2020. As part of this issuance, the Company issued and sold 3,266,667 shares to PBM TGT Holdings, LLC and 400,000 shares to Nolan Capital, LLC, which stockholders are controlled by certain members of the Company’s board of directors. On July 2, 2020, the Company entered into a purchase agreement (the “Series B Purchase Agreement”), as later amended on July 28, 2020, providing for a private placement of up to 5,647,052 shares of Series B convertible preferred stock. The Company sold 5,647,048 shares of Series B convertible preferred stock at a price of $17.00 per share in multiple closings in July and August 2020 for gross proceeds of $96.0 million. The majority of investors that participated in the Series B Purchase Agreement were new investors. As described above, in connection with the closing of the IPO, all shares of Series A and Series B convertible preferred stock were automatically converted into an aggregate of 17,047,378 shares of common stock with a conversion ratio, which was adjusted for the stock split, of 1.0895 shares of common stock for each share of Series A and Series B convertible preferred stock then outstanding. Series A convertible preferred stock tranche liability The Company concluded that the tranche liability met the definition of a freestanding financial instrument, as it was legally detachable and separately exercisable from the initial closing of the Series A convertible preferred stock. The estimated fair value of the tranche liability was determined using a Monte Carlo simulation at the initial issuance date. As of March 4, 2020, the simulations occurred based on the implied aggregate equity value of the Company derived from the Series A convertible preferred stock offering price of $3.00 per share, along with, in part, the following subjective assumptions: risk-free rate of 0.59%, an expected volatility of 80%, the expected term to a liquidity event of 1 year, and a 60% probability of achieving the clinical milestones and timing thereof. Subsequently, the estimated fair value of the tranche liability was determined using a backsolve approach at June 30, 2020, immediately prior to the issuance of the Milestone Shares, which was calculated based on the aggregate equity value of the Company derived from the Series B convertible preferred stock offering price of $17.00 per share. The subsequent remeasurement also considered, in part, a risk-free rate of 0.17%, an expected volatility of 80%, and the expected term to a liquidity event of 0.5 years. Based on the analysis, the Company recorded a preferred stock tranche liability of $1.1 million at the issue date to account for the obligation to issue the Milestone Shares at a predetermined fixed price at a future settlement date. At June 30, 2020, ahead of the anticipated closing of the Series B Purchase Agreement for $17.00 per share that occurred on July 2, 2020, certain investors elected to exercise in full their options to their pro-rata portion of the Milestone Shares prior to the Company’s achievement of the clinical milestones and purchased 200,000 shares of Series A convertible preferred stock. The Company remeasured the fair value of the entire tranche liability at June 30, 2020 and recognized a non-cash expense of $17.0 million in the consolidated statements of operations. Between June 30, 2020 and July 2, 2020, all of the 4,000,000 Milestone Shares were issued and the related tranche liability was extinguished in its entirety, and the Company reclassified the entire tranche liability of $18.1 million to convertible preferred stock on the consolidated balance sheets. The Company concluded that no beneficial conversion feature (“BCF”) existed as the effective conversion price of the Series A convertible preferred stock exceeded the fair value of the Company’s common stock at each of the commitment dates. Specifically, at the commitment date of June 30, 2020, when 200,000 Milestone Shares were issued, the deemed proceeds were equal to the cash proceeds received for the shares of Series A convertible preferred stock and the fair value of the tranche liability that related to the Milestone Shares, or $7.52 per share. As the effective conversion price exceeded the fair value of the Company’s common stock at the commitment date, no BCF existed. The following table provides a reconciliation of the preferred stock tranche liability measured at fair value using Level 3 significant unobservable inputs (in thousands): Balance at December 31, 2019 $ — Fair value at issuance of Series A convertible preferred stock 1,050 Change in fair value 17,030 Settlement of preferred stock tranche liability due to issuance of Milestone Shares (18,080 ) Balance at December 31, 2020 $ — |
Stock-Based Compensation
Stock-Based Compensation | 12 Months Ended |
Dec. 31, 2020 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Stock-Based Compensation | Note 8—Stock-Based Compensation On July 1, 2020, the Company’s board of directors approved the Existing Plan which permits the granting of incentive stock options, non-statutory stock options, stock appreciation rights, RSAs, RSUs and other stock-based awards to employees, directors, officers and consultants. On July 1, 2020, 3,529,412 shares of common stock were authorized for issuance under the Existing Plan. On September 16, 2020, the Company increased the number of shares of common stock authorized for issuance under the Existing Plan to 3,845,294. On September 16, 2020, the Company’s stockholders approved the New Plan, which became effective upon the execution of the underwriting agreement in connection with the IPO. The number of shares available for future issuance under the New Plan is the sum of (1) 3,390,168 new shares of common stock, (2) 209,841 remaining shares of common stock reserved under the Existing Plan that became available for issuance upon the effectiveness of the New Plan and (3) the number of shares of common stock subject to outstanding awards under the Existing Plan when the New Plan became effective that thereafter expire or are forfeited, canceled, withheld to satisfy tax withholding or to purchase or exercise an award, repurchased by the Company or are otherwise terminated. At December 31, 2020, there were 2,941,509, shares available for future grant under the New Plan. The number of shares of common stock reserved for issuance under the New Plan will automatically increase on January 1 of each year, for a period of ten years, from January 1, 2021 continuing through January 1, 2030, by 5% of the total number of shares of common stock outstanding on December 31 of the preceding calendar year, or a lesser number of shares as may be determined by the Company’s board of directors. Furthermore, on September 16, 2020, the Company’s stockholders approved the Employee Stock Purchase Plan (“ESPP”), which became effective upon the execution of the underwriting agreement in connection with the IPO. The maximum number of shares of common stock that may be issued under the ESPP will not exceed 362,000 shares of common stock, plus the number of shares of common stock that are automatically added on January 1st of each year for a period of up to ten years, commencing on the first January 1 following the IPO Date and ending on (and including) January 1, 2030, in an amount equal to the lesser of (i) one percent (1.0%) of the total number of shares of capital stock outstanding on December 31st of the preceding calendar year, and (ii) 724,000 shares of common stock. No issuances have been made under the ESPP as of December 31, 2020. Stock Options On July 1, 2020, options to purchase 2,896,782 shares of common stock under the Existing Plan were awarded to certain employees and consultants of the Company with an exercise price per share of $0.80, which were expected to vest over a four-year period, all of which were subsequently cancelled (the “Cancelled Options”). The grant date fair value of the Cancelled Options was $13.8 million at the original grant date. In exchange, the Company awarded 2,518,932 RSUs on September 2, 2020, which are expected to vest over a four-year term. The Company accounted for the changes in award terms as a modification in accordance with ASC 718 Compensation – Stock Compensation. The modification is accounted for as an exchange of the original award for a new award with total compensation cost equal to the grant-date fair value of the original award plus any incremental value measured on the modification date. The Company determined that there was no incremental value as the fair value of the original award immediately before the modification was greater than the fair value of the new award immediately after the modification. Accordingly, the Company will recognize the remaining compensation cost at the time of the modification of $13.2 million over the vesting period of the RSUs. The estimated fair value of the Company’s common stock at July 1, 2020 was $5.28 per share, as determined using information derived from the issuance price of the Company’s Series B convertible preferred stock of $17.00 per share on July 2, 2020. The valuation of the common stock was determined using an option-pricing model under which shares are valued by creating a series of call options with exercise prices based on the liquidation preferences and conversion terms of each equity class, adjusted for a discount for the lack of marketability to account for a lack of access to an active public market. The following assumptions were used to estimate the fair value of the Cancelled Options that were granted on July 1, 2020: Risk-free interest rate 0.31 % Expected dividend yield — Expected term in years 6.3 Expected volatility 80 % During the year ended December 31, 2020, excluding the Cancelled Options, options to purchase 674,842 shares of common stock were granted with a weighted-average exercise price per share of $20.68. On September 2, 2020, options to purchase 16,342 shares of common stock under the Existing Plan were awarded to certain directors of the Company with an exercise price per share of $14.90, and on various dates beginning at the closing of the IPO through December 31, 2020, 658,500 shares of common stock under the New Plan were awarded with a weighted-average exercise price per share of $20.83. The stock options generally vest over three or four years and have a ten-year contractual term. The following weighted-average assumptions were used to estimate the fair value of stock options, excluding the Cancelled Options, for the year ended December 31, 2020: Risk-free interest rate 0.54 % Expected dividend yield — Expected term in years 6.2 Expected volatility 77 % The following table summarizes stock option activity, excluding the Cancelled Options, during the year ended December 31, 2020: Weighted Weighted Average Aggregate Average Remaining Intrinsic Stock Exercise Contractual Value Options Price Life (in years) (in thousands) Outstanding at December 31, 2019 — $ — — $ — Options granted 674,842 20.68 Outstanding at December 31, 2020 674,842 $ 20.68 9.8 $ 3,953 Vested and expected to vest at December 31, 2020 674,842 $ 20.68 9.8 $ 3,953 Options exercisable at December 31, 2020 12,054 $ 20.25 9.8 $ 76 The aggregate intrinsic value in the above table is calculated as the difference between the fair value of the Company’s common stock as of December 31, 2020 and the exercise price of the stock options. The weighted-average grant date fair value per share for the stock option awards, excluding the Cancelled Options, granted during the year ended December 31, 2020 was $13.73. As of December 31, 2020, the total unrecognized compensation related to unvested stock option awards granted was $8.8 million, which the Company expects to recognize over a weighted-average period of approximately 3.6 years. Restricted Stock Units On September 2, 2020, the Company issued 331,121 RSUs to an employee under the Existing Plan; 25% of the shares of common stock underlying the RSUs vest at each anniversary over a four-year period. The RSUs are subject to a service-based vesting condition. The RSUs were also subject to a liquidity-based performance vesting condition that was met upon the closing of the IPO. The Company at any time may accelerate the vesting of the RSUs. Such shares are not accounted for as outstanding until they vest. As of December 31, 2020, the total unrecognized compensation related to unvested RSUs granted, including the remaining compensation cost associated with the RSUs granted on September 2, 2020 in exchange for the Cancelled Options, was $16.2 million which is expected to be amortized on a straight-line basis over the weighted-average remaining vesting period of approximately 2.1 years. The Company’s RSU activity for the year ended December 31, 2020 was as follows: Weighted Average Grant Date Number Fair Value of Shares per Share Nonvested at January 1, 2020 — $ — Replacement restricted units granted 2,518,932 5.25 Restricted units granted 331,121 14.90 Vested — — Nonvested at December 31, 2020 2,850,053 $ 6.37 Restricted Stock Awards RA Session II, the Company’s President and Chief Executive Officer, was awarded 769,058 RSAs under the Existing Plan on July 1, 2020, which are expected to vest over a three-year term, subject to continuous employment. As of December 31, 2020, the total unrecognized compensation related to unvested RSAs granted was $3.2 million which is expected to be amortized on a straight-line basis over the weighted-average remaining vesting period of approximately 2.3 years. The fair value of these RSAs at the grant date of July 1, 2020 was $5.28 per share. The Company’s RSA activity for the year ended December 31, 2020 was as follows: Weighted Average Grant Date Number Fair Value of Shares per Share Nonvested at December 31, 2019 — $ — Restricted stock granted 769,058 5.28 Vested — — Nonvested at December 31, 2020 769,058 $ 5.28 The following table summarizes the total stock-based compensation expense for the stock options, RSAs and RSUs recorded in the consolidated statements of operations for the year ended December 31, 2020 and for the period from Inception to December 31, 2019 (in thousands): For the Year Ended December 31, 2020 For the period from September 20, 2019 (date of inception) to December 31, 2019 Research and development expense $ 1,167 $ — General and administrative expense 2,187 — Total $ 3,354 $ — |
Net Loss Per Common Share
Net Loss Per Common Share | 12 Months Ended |
Dec. 31, 2020 | |
Earnings Per Share [Abstract] | |
Net Loss Per Common Share | Note 9—Net Loss Per Common Share Basic net loss per common share is computed by dividing net loss attributable to common stockholders by the weighted-average number of shares of common stock outstanding for the period. Since the Company had a net loss in the periods presented, basic and diluted net loss per common share are the same. The following table represents the calculation of basic and diluted net loss per common share for the year ended December 31, 2020 and the period from Inception to December 31, 2019 (in thousands, except share and per share data): For the Year Ended December 31, 2020 For the Period from September 20, 2019 (date of inception) to December 31, 2019 Net loss $ (60,011 ) $ (1,115 ) Weighted-average shares of common stock outstanding used to compute net loss per common share, basic and diluted 17,665,683 9,625,679 Net loss per common share, basic and diluted $ (3.40 ) $ (0.12 ) The following common stock equivalents outstanding as of December 31, 2020 and 2019, respectively, were excluded from the computation of diluted net loss per share attributable to common stockholders for the periods presented because including them would have been anti‑dilutive: For the Year Ended December 31, 2020 For the Period from September 20, 2019 (date of inception) to December 31, 2019 Unvested RSUs 2,850,053 — Unvested RSAs 769,058 — Stock options 674,842 — Total 4,293,953 — |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2020 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | Note 10—Income Taxes Provision for income taxes There is no provision for income taxes because the Company has incurred operating losses and capitalized certain items for income tax purposes since its inception and maintains a full valuation allowance against its net deferred tax assets. The reported amount of income tax expense for the period differs from the amount that would result from applying the federal statutory tax rate to net loss before taxes primarily because of the change in valuation allowance. On March 27, 2020, the CARES Act, an economic relief package in response to the COVID-19 pandemic, was signed into law. The CARES Act contains several corporate income tax provisions, including making remaining alternative minimum tax credits immediately refundable; providing a 5-year carryback of NOL carryforwards generated in tax years 2018, 2019, and 2020, and removing the 80% taxable income limitation on utilization of those NOLs if carried back to prior tax years or utilized in tax years beginning before 2021; and temporarily liberalizing the interest deductibility rules under Section 163(j) of the Tax Cuts and Jobs Act, by raising the adjusted taxable income limitation from 30% to 50% for tax years 2019 and 2020 and giving taxpayers the election of using 2019 adjusted taxable income for purposes of computing 2020 interest deductibility. The CARES Act did not have a material effect on the realizability of deferred income tax assets or tax expense in 2020. The effective tax rate of the Company's provision (benefit) for income taxes differs from the federal statutory rate as follows: For the Year Ended December 31, 2020 For the Period from September 20, 2019 (date of inception) to December 31, 2019 Statutory rate 21.00 % 21.00 % State tax 0.06 % — General business credits 1.06 % — Other permanent differences (0.25 )% — Change in valuation allowance (15.91 )% (21.00 )% Change in fair value of tranche liability (5.96 )% — Income tax provision (benefit) 0.00 % 0.00 % Deferred tax assets and valuation allowance Deferred tax assets reflect the tax effects of NOLs, tax credit carryovers, and temporary differences between the carrying amounts of assets and liabilities for financial reporting purposes and the amounts used for income tax purposes. At December 31, 2020 and December 31, 2019, the significant components of the Company’s net deferred tax asset are as follows (in thousands): For the Year Ended December 31, 2020 For the Period from September 20, 2019 (date of inception) to December 31, 2019 Net operating loss carryforwards $ 5,810 $ — Tax credit carryforwards 672 — Accruals and reserves 334 — Other 21 — Intangibles 2,390 206 Organizational and start-up costs — 28 Non-qualified stock options 555 — Total deferred tax assets 9,782 234 Valuation allowance (9,782 ) (234 ) Deferred tax asset, net of allowance $ — $ — There are no deferred tax liabilities as of December 31, 2020 and December 31, 2019, respectively. The valuation allowance is equal to the total deferred tax asset amounts as of December 31, 2020 and December 31, 2019 as there are no deferred tax liabilities in the respective periods. ASC 740 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 the above-mentioned future tax benefits is currently not likely to be realized and, accordingly, has provided a valuation allowance. The valuation allowance increased by $9.5 million during the year ended December 31, 2020 and by $0.2 million for the period from Inception to December 31, 2019. There were no net operating loss and tax credit carryforwards as of December 31, 2019. As of December 31, 2020, there were net federal operating losses of $27.7 million, federal tax credit carryforward of $0.9 million, and state tax credit carryforward of less than $0.1 million. The net operating losses and state tax credit carryforward do not expire. The federal tax credit carryforward will expire in 2040. The Company files federal and state income tax returns and, in the normal course of business, the Company is subject to examination by these taxing authorities. Pursuant to Section 382 of the Internal Revenue Code, certain substantial changes in the Company's ownership may result in a limitation on the amount of NOL carryforwards and tax credit carryforwards that may be used in future years. Utilization of the NOL and tax credit carryforwards may be subject to a substantial annual limitation under Section 382 of the Internal Revenue Code of 1986 due to ownership change limitations that have occurred previously or that could occur in the future. These ownership changes may limit the amount of NOL and tax credit carryforwards that can be utilized annually to offset future taxable income and tax, respectively. The Company has not completed a study to assess whether an ownership change has occurred, or whether there have been multiple ownership changes since its formation. There could also be additional ownership changes in the future which may result in additional limitations on the utilization of NOL carryforwards and credits. |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2020 | |
Commitments And Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | Note 11—Commitments and Contingencies Litigation The Company is not a party to any material legal proceedings and is not aware of any pending or threatened claims. From time to time, the Company may be subject to various legal proceedings and claims that arise in the ordinary course of its business activities. Commitments In the normal course of business, the Company enters into contracts that contain a variety of indemnifications with its employees, licensors, suppliers and service providers. The Company’s maximum exposure under these arrangements is unknown at December 31, 2020. The Company does not anticipate recognizing any significant losses relating to these arrangements. As of December 31, 2019, the Company was not a party to any leasing agreements. On December 17, 2020, the Company entered into a lease agreement (the “Durham Lease”) with Patriot Park Partners II, LLC, a Delaware limited liability company (the “Landlord”), pursuant to which the Company agreed to lease approximately 187,500 square feet of a manufacturing facility located at 5 National Way, Durham, North Carolina (the “Facility”). The Durham Lease commences on April 1, 2021 and is expected to have a term of approximately fifteen years and six months. The Company has two options to extend the term of the Durham Lease, each for a period of an additional five years. The Company was not required to provide a security deposit in connection with its entry into the Durham Lease. The Company will be responsible for constructing interior improvements within the Facility. The Company also has a one-time right of first offer during the term of the Durham Lease to purchase the Facility. The Landlord has the right to terminate the Durham Lease upon specified events of default, including the Company’s failure to pay rent in a timely manner and upon the occurrence of certain events of insolvency with respect to the Company. The Company may terminate the Durham Lease if construction of the base building shell of the Facility is not complete by May 16, 2021. As of December 31, 2020, the Company incurred initial direct costs to enter into the Durham Lease of approximately $0.8 million. The costs have been recorded on the consolidated balance sheets as a deferred lease asset and will be amortized into earnings over the term of the Durham Lease. The following table summarizes lease commitments as of December 31, 2020 (in thousands): Year Ending December 31, 2021 $ 314 2022 1,282 2023 1,317 2024 1,354 2025 1,391 Thereafter 17,599 Total Lease Commitments $ 23,257 |
Subsequent Events
Subsequent Events | 12 Months Ended |
Dec. 31, 2020 | |
Subsequent Events [Abstract] | |
Subsequent Events | Note 12—Subsequent Events Lease Agreement On January 11, 2021, the Company entered into a lease agreement (the “Dallas Lease”) with Pegasus Park, LLC, a Delaware limited liability company (the “Dallas Landlord”), pursuant to which the Company will lease approximately 15,000 square feet of office space at 3000 Pegasus Park Drive, Dallas, Texas 75247 (the “Office Space”). The Dallas Lease commences on the date on which certain improvements to the Office Space have been made and the Office Space is tendered to the Company for possession, which the Company and the Dallas Landlord presently anticipate to be delivered on or about April 15, 2021, and will have a term of approximately ten years. The Company has an option to extend the term of the Dallas Lease for one additional period of five years. Total future minimum lease payments under the Dallas Lease over the initial 10 year term are approximately $4.9 million. The Company may terminate the Dallas Lease if the Office Space is not delivered with all improvements to be made by the Dallas Landlord pursuant to the Dallas Lease if not substantially completed by May 31, 2021. Equity Grants In January, February, and March 2021, the Company granted options to purchase an aggregate of 1,621,900 shares of its common stock at a weighted-average exercise price of $28.81 per share. |
Significant Accounting Polici_2
Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2020 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The Company’s financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America (“U.S. GAAP”) as determined by the Financial Accounting Standards Board (“FASB”) Accounting Standards Codification (“ASC”) and include all adjustments necessary for the fair presentation of the Company’s financial position for the periods presented. |
Principles of Consolidation | Principles of Consolidation The accompanying consolidated financial statements include the accounts of Taysha and its inactive wholly owned U.S. subsidiaries that were incorporated during 2020. All intercompany transactions and balances have been eliminated in consolidation. |
Emerging Growth Company | Emerging Growth Company From time to time, new accounting pronouncements are issued by FASB, or other standard setting bodies and adopted by the Company as of the specified effective date. Unless otherwise discussed, the impact of recently issued standards that are not yet effective will not have a material impact on the Company’s financial statements upon adoption. Under the Jumpstart Our Business Startups Act of 2012, as amended, the Company meets the definition of an emerging growth company, and has elected the extended transition period for complying with new or revised accounting standards, which delays the adoption of these accounting standards until they would apply to private companies. |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets and liabilities and disclosure of contingent assets and liabilities at the date of the financial statements and the reported amounts of expenses during the reporting period. The most significant estimates and assumptions in the Company’s financial statements relate to the determination of the fair value of the common stock prior to the IPO (as an input into stock-based compensation), estimating preclinical manufacturing accruals and accrued or prepaid research and development expenses, and the valuation of the preferred stock tranche liability. These estimates and assumptions are based on current facts, historical experience and various other factors believed to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities and the recording of expenses that are not readily apparent from other sources. Actual results may differ materially from these estimates. To the extent there are material differences between the estimates and actual results, the Company’s future results of operations will be affected. |
Risks and Uncertainties | Risks and Uncertainties The Company is subject to risks common to companies in the biotechnology industry, including, but not limited to, development by the Company or its competitors of technological innovations, risks of failure of clinical studies, dependence on key personnel, protection of proprietary technology, compliance with government regulations, and ability to transition from preclinical manufacturing to commercial production of products. The Company’s product candidates require approvals from the FDA and comparable foreign regulatory agencies prior to commercial sales in their respective jurisdictions. There can be no assurance that any product candidates will receive the necessary approvals. If the Company was denied approval, approval was delayed or the Company was unable to maintain approval for any product candidate, it could have a materially adverse impact on the Company. |
Segments | Segments Operating segments are identified as components of an enterprise about which separate discrete financial information is available for evaluation by the chief operating decision-maker in making decisions regarding resource allocation and assessing performance. The Company views its operations and manages its business as a single operating segment, which is the business of developing AAV-based gene therapies for the treatment of rare monogenic diseases of the CNS. As of December 31, 2020 and 2019, the Company did not have assets located outside of the United States. |
Cash and Cash Equivalents | Cash and Cash Equivalents Cash and cash equivalents consist of funds held in a standard checking account and a standard savings account. The Company considers all highly liquid investments with an original maturity of three months or less at the date of purchase to be cash equivalents. As of December 31, 2020 and 2019, respectively, the Company had no cash equivalents. |
Concentrations of Credit Risk | Concentrations of Credit Risk Financial instruments that potentially subject the Company to significant concentration of credit risk consist primarily of cash and cash equivalents. Periodically, the Company may maintain deposits in financial institutions in excess of government insured limits. Management believes that the Company is not exposed to significant credit risk as the Company’s deposits are held at financial institutions that management believes to be of high credit quality. The Company has not experienced any losses on these deposits. |
Fair Value of Financial Instruments | Fair Value of Financial Instruments The Company’s financial assets and liabilities are accounted for in accordance with ASC 820, Fair Value Measurements and Disclosures which defines fair value as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. The fair value hierarchy requires an entity to maximize the use of observable inputs when measuring fair value and classifies those inputs into three levels: Level 1—Observable inputs, such as quoted prices in active markets for identical assets or liabilities. Level 2—Inputs other than Level 1 inputs that are either directly or indirectly observable, 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 instrument’s anticipated life. Level 3—Unobservable inputs in which there is little or no market data, which require the reporting entity to develop its own assumptions. To the extent the valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair values requires more judgement. Accordingly, the degree of judgement exercised by the Company in determining fair value is greatest for instruments categorized as Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement. The carrying values reported in the Company’s consolidated balance sheets for cash and cash equivalents, prepaid expenses and other current assets, accounts payable, accrued expenses and other current liabilities are reasonable estimates of their fair values due to the short-term nature of these items. |
Deferred Offering Costs | Deferred Offering Costs The Company capitalizes certain legal, professional, accounting and other third-party fees that are directly associated with in-process equity financings as deferred offering costs until such financings are consummated. After consummation of the equity financing, these costs are recorded in stockholders’ deficit as a reduction of additional paid-in capital generated as a result of an equity financing. Should the in-process equity financing be abandoned, the deferred offering costs will be expensed immediately as a charge to operating expenses in the statements of operations. As of December 31, 2020 and 2019, there was $0 and $15,000, respectively, of deferred offering costs capitalized on the consolidated balance sheets. The $15,000 capitalized as of December 31, 2019 related to the Company’s Series A convertible preferred stock financing that closed in March 2020 (see Note 7). Deferred offering costs, consisting of legal, accounting, and filing fees directly relating to the IPO and the Company’s Series A and Series B preferred stock financings, were capitalized and offset against the related proceeds upon the completion of the offerings in 2020. Upon completion of the IPO in September 2020, approximately $2 . |
Property and Equipment, net | Property and Equipment, net Property and equipment are stated at cost less accumulated depreciation and consist solely of computer equipment and laboratory equipment. Depreciation expense is recognized using the straight-line method over its estimated useful life of three to five years. Upon retirement or sale, the cost of assets disposed of and the related accumulated depreciation are removed from the accounts and any resulting gain or loss is included in loss from operations. Expenditures for repairs and maintenance are charged to expense as incurred. |
Impairment of Long-Lived Assets | Impairment of Long-Lived Assets The Company evaluates its long-lived assets, which consist of property and equipment, for impairment whenever events or changes in circumstances indicate that the carrying amount of such assets may not be recoverable. Recoverability of assets to be held and used is measured by a comparison of the carrying amount of an asset to the future undiscounted net cash flows expected to be generated by the asset. If such assets are considered to be impaired, the impairment to be recognized is measured by the amount by which the carrying amount of the asset exceeds the fair value of the asset. There were no impairment losses recognized during the year ended December 31, 2020 and the period from Inception to December 31, 2019. |
Convertible Preferred Stock | Convertible Preferred Stock The Company recorded shares of its convertible preferred stock at their respective fair values on the dates of issuance, net of issuance costs. The Company applied the guidance in ASC 480-10-S99-3A, SEC Staff Announcement: Classification and Measurement of Redeemable Securities and therefore classified the Series A convertible preferred stock as mezzanine equity. The convertible preferred stock was recorded outside of stockholders’ deficit because, in the event of certain deemed liquidation events considered not solely within the Company’s control, such as a merger, acquisition and sale of all or substantially all of the Company’s assets (a “Deemed Liquidation Event”), the convertible preferred stock would become redeemable at the option of the holders. In the event of a change of control of the Company, proceeds received from the sale of such shares would have been distributed in accordance with the liquidation preferences set forth in the Company’s Amended and Restated Certificate of Incorporation. The Company determined not to adjust the carrying values of the convertible preferred stock to the liquidation preferences of such shares because of the uncertainty of whether or when such an event would occur. All shares of the Company’s previously issued and outstanding convertible preferred stock were converted into shares of common stock upon the closing of the IPO (see Note 7). |
Preferred Stock Tranche Liability | Preferred Stock Tranche Liability The Company determined that its obligation to issue, and the Company’s investors’ right to purchase, additional shares of Series A convertible preferred stock pursuant to the milestone closings (see Note 7) represented a freestanding financial instrument (the “tranche liability”). The tranche liability was initially recorded at fair value. The proceeds from the sale of the convertible preferred stock were first allocated to the fair value of the tranche liability with the remaining proceeds from the sale of the convertible preferred stock allocated to the Series A convertible preferred stock. The tranche liability was remeasured at each reporting period and upon the exercise or expiration of the obligation, with gains and losses arising from subsequent changes in its fair value recognized in other expense in the consolidated statements of operations. At the time of the exercise or expiration of the tranche liability, any remaining value of the tranche liability was reclassified to convertible preferred stock on the consolidated balance sheets. |
Research and Development | Research and Development The Company has entered into research and development contracts with research institutions and other companies. These agreements are generally cancelable, and related payments are recorded as research and development expenses as incurred. Payments for these activities are based on the terms of the individual agreements, which may differ from the pattern of costs incurred, and are reflected on the consolidated balance sheets as prepaid or accrued expenses. The Company records accruals for estimated ongoing research costs. When evaluating the adequacy of the accrued liabilities, the Company analyzes progress of the studies, including the phase or completion of events, invoices received and contracted costs. Research and development costs primarily consist of payroll, stock-based compensation, certain manufacturing costs, laboratory costs and other supplies, and the cost to acquire licenses. Costs incurred in obtaining technology licenses through asset acquisitions are charged to research and development expense if the licensed technology has not reached technological feasibility and has no alternative future use. |
Stock-Based Compensation | Stock-Based Compensation The Company accounts for all stock-based payments to employees and non-employees, including grants of stock options, restricted stock awards, or RSAs, and restricted stock units, or RSUs, based on their respective grant date fair values. The Company estimates the fair value of stock option grants using the Black-Scholes option pricing model, which is affected principally by the estimated fair value of shares of the Company’s common stock and requires management to make a number of other assumptions, including the expected life of the option, the volatility of the underlying shares, the risk-free interest rate and expected dividends. Expected volatility is based on the historical share volatility of a set of comparable publicly traded companies over a period of time equal to the expected term of the options. Due to the lack of historical exercise history, the expected term of the Company’s stock options is determined using the “simplified” method. The risk-free interest rate is determined by reference to the U.S. Treasury yield curve in effect at the time of grant of the award for time periods approximately equal to the expected term of the award. Expected dividend yield is zero based on the fact that the Company has never paid cash dividends and does not expect to pay any cash dividends in the foreseeable future. Prior to September 23, 2020, the fair value of common stock underlying the Company’s stock options, RSAs and RSUs was estimated by the Company’s board of directors considering, among other things, contemporaneous valuations of the Company’s common stock prepared by unrelated third-party valuation firms. After the IPO, the fair value of common stock is based on the closing price of the Company’s common stock on the Nasdaq Global Select Market as reported on the date of the grant. The RSAs and RSUs are valued based on the fair value of the Company’s common stock on the date of grant. The Company expenses stock-based compensation related to stock options, RSAs and RSUs over the requisite service period using the straight-line method. All stock-based compensation costs are recorded in research and development expense or general and administrative expense in the consolidated statements of operations based upon the respective employee’s roles within the Company. Forfeitures are recorded as they occur. |
Income Taxes | Income Taxes Income taxes are recorded in accordance with ASC 740, Income Taxes (“ASC 740”), which provides for deferred taxes using an asset and liability approach. The Company recognizes deferred tax assets and liabilities for the expected future tax consequences of events that have been included in the financial statements or tax returns. Deferred tax assets and liabilities are determined based on the difference between the financial statement and tax bases of assets and liabilities using enacted tax rates in effect for the year in which the differences are expected to reverse, and net operating loss (“NOL”) carryforwards and research and development tax credit (“R&D Credit”) carryforwards. Valuation allowances are provided, if based upon the weight of available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized. The Company has recorded a full valuation allowance to reduce its net deferred income tax assets to zero. In the event the Company were to determine that it would be able to realize some or all its deferred income tax assets in the future, an adjustment to the deferred income tax asset valuation allowance would increase income in the period such determination was made. The Company accounts for uncertain tax positions in accordance with the provisions of ASC 740. When uncertain tax positions exist, the Company recognizes the tax benefit of tax positions to the extent that the benefit would more likely than not be realized assuming examination by the taxing authority. The determination as to whether the tax benefit will more likely than not be realized is based upon the technical merits of the tax position as well as consideration of the available facts and circumstances. At December 31, 2019, the Company had no liability for income tax associated with uncertain tax positions. During the year ended December 31, 2020, the Company recorded gross unrecognized tax benefits that were not significant. The unrecognized tax benefits, if recognized, would not affect the effective income tax rate due to the valuation allowance that currently offsets deferred tax assets. The Company does not expect the unrecognized tax benefits to change significantly over the next 12 months. The Company would recognize any corresponding interest and penalties associated with its income tax positions in income tax expense. There was no income tax interest or penalties incurred for the year ended December 31, 2020 or for the period from Inception to December 31, 2019. |
Comprehensive Loss | Comprehensive Loss Comprehensive loss is equal to net loss as presented in the accompanying consolidated statements of operations, as the Company did not have any other comprehensive income or loss for the periods presented. |
Recently Adopted Accounting Pronouncements | Recently Adopted Accounting Pronouncements In May 2014, the FASB issued ASC 606, Revenue from Contracts with Customers (“ASC 606”). This guidance applies to any entity that either enters into contracts with customers to transfer goods or services or enters into contracts for the transfer of nonfinancial assets unless those contracts are within the scope of other standards. The core principle of this guidance is that an entity should recognize revenue to depict the transfer of promised goods or services to customers in an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services. This guidance supersedes existing revenue recognition guidance, including most industry-specific guidance, as well as certain related guidance on accounting for contract costs. The Company early adopted ASC 606 upon its Inception. As the Company does not have any contracts with customers, the adoption of this guidance did not have any impact on the Company’s consolidated financial statements. In June 2018, the FASB issued Accounting Standards Update (“ASU”) No. 2018-07, Compensation – Stock Compensation (Topic 718) (“ASU 2018-07”). This update is intended to reduce cost and complexity and to improve financial reporting for share-based payments issued to non-employees (for example, service providers, external legal counsel, suppliers, etc.). The ASU expands the scope of Topic 718, Compensation—Stock Compensation, which currently only includes share-based payments issued to employees, to also include share-based payments issued to non-employees for goods and services. Consequently, the accounting for share-based payments to non-employees and employees will be substantially aligned. The Company early adopted ASU 2018-07 upon its Inception. The adoption of this ASU did not have a material effect on the Company’s consolidated financial statements at Inception. The Company applied this accounting pronouncement to the issuance of shares to the Board of Regents of the University of Texas System (see Note 5). In August 2018, the FASB issued ASU No. 2018-13, Fair Value Measurement (ASC Topic 820) (“ASU 2018-13”), which modifies, removes and adds certain disclosure requirements on fair value measurements based on the FASB Concepts Statement, Conceptual Framework for Financial Reporting—Chapter 8: Notes to Financial Statements. The amendments on changes in unrealized gains and losses, the range and weighted-average of significant unobservable inputs used to develop Level 3 fair value measurements and the narrative description of measurement uncertainty should be applied prospectively for only the most recent interim or annual period presented in the initial fiscal year of adoption. All other amendments should be applied retrospectively to all periods presented upon their effective date. The Company adopted ASU 2018-13 as of January 1, 2020 for the annual period. The adoption of the guidance did not have a material impact on the Company’s consolidated financial statements. In November 2018, the FASB issued ASU No. 2018-18 – Collaborative Arrangements, which clarifies that certain transactions between collaborative arrangement participants should be accounted for as revenue when the collaborative arrangement participant is a customer in the context of a unit of account and precludes recognizing as revenue consideration received from a collaborative arrangement participant if the participant is not a customer. The Company early adopted this standard as of January 1, 2020 for the annual period and it will be applied to interim periods after December 31, 2020. This ASU requires retrospective adoption to the date the Company adopted ASC 606, which was upon its Inception, by recognizing a cumulative-effect adjustment to the opening balance of retained earnings of the earliest annual period presented. As the Company does not have any contracts with customers or collaborative arrangements, the adoption of this guidance did not have any impact on the Company’s consolidated financial statements. |
Recently Issued Accounting Pronouncements Policy | Recently Issued Accounting Pronouncements In February 2016, the FASB issued ASU No. 2016-02, Leases (Topic 842), as amended, with guidance regarding the accounting for and disclosure of leases. This update requires lessees to recognize the liabilities related to all leases, including operating leases, with a term greater than 12 months on the balance sheets. This update also requires lessees and lessors to disclose key information about their leasing transactions. This guidance will become effective for the Company for annual reporting periods beginning after December 15, 2021 and interim periods within fiscal years beginning after December 15, 2022. The Company is currently evaluating the impact of this standard on its consolidated financial statements. In December 2019, the FASB issued ASU No. 2019-12, Income Taxes (Topic 740): Simplifying the Accounting for Income Taxes (“ASU 2019-12”), which is intended to simplify various aspects related to accounting for income taxes. ASU 2019-12 removes certain exceptions to the general principles in ASC 740 and also clarifies and amends existing guidance to improve consistent application. This guidance is effective for fiscal years beginning after December 15, 2021, and interim periods within fiscal years beginning after December 15, 2022, with early adoption permitted. The Company is currently evaluating the impact of this standard on its consolidated financial statements. |
Property and Equipment (Tables)
Property and Equipment (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Property Plant And Equipment [Abstract] | |
Schedule of Property and Equipment | Property and Equipment consisted of the following (in thousands): December 31, December 31, 2020 2019 Computer equipment $ 95 $ — Construction in progress 201 — 296 — Accumulated depreciation (9 ) — Property and equipment, net $ 287 $ — |
Supplemental Financial Inform_2
Supplemental Financial Information (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Balance Sheet Related Disclosures [Abstract] | |
Schedule of Accrued Expenses and Other Current Labilities | Accrued expenses and other current liabilities consisted of the following (in thousands): December 31, December 31, 2020 2019 Accrued research and development $ 2,106 $ 7 Accrued compensation 1,766 — Accrued professional fees 999 122 Other 264 21 Total accrued expenses and other current liabilities $ 5,135 $ 150 |
Schedule of Prepaid Expenses and Other Current Assets | Prepaid expenses and other current assets consisted of the following (in thousands): December 31, December 31, 2020 2019 Prepaid insurance $ 2,480 $ — Prepaid research and development 2,462 — Prepaid clinical trial 944 — Other 740 — Total prepaid expenses and other current assets $ 6,626 $ — |
Stockholders' Equity (Deficit_2
Stockholders' Equity (Deficit), Convertible Preferred Stock and Tranche Liability (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Equity [Abstract] | |
Reconciliation of Preferred Stock Tranche Liability Measured at Fair Value | The following table provides a reconciliation of the preferred stock tranche liability measured at fair value using Level 3 significant unobservable inputs (in thousands): Balance at December 31, 2019 $ — Fair value at issuance of Series A convertible preferred stock 1,050 Change in fair value 17,030 Settlement of preferred stock tranche liability due to issuance of Milestone Shares (18,080 ) Balance at December 31, 2020 $ — |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Schedule of Restricted Stock Units Activity | The Company’s RSU activity for the year ended December 31, 2020 was as follows: Weighted Average Grant Date Number Fair Value of Shares per Share Nonvested at January 1, 2020 — $ — Replacement restricted units granted 2,518,932 5.25 Restricted units granted 331,121 14.90 Vested — — Nonvested at December 31, 2020 2,850,053 $ 6.37 |
Schedule of Restricted Stock Awards Activity | The Company’s RSA activity for the year ended December 31, 2020 was as follows: Weighted Average Grant Date Number Fair Value of Shares per Share Nonvested at December 31, 2019 — $ — Restricted stock granted 769,058 5.28 Vested — — Nonvested at December 31, 2020 769,058 $ 5.28 |
Schedule of Total Stock-Based Compensation Expense for Stock Options, RSAs and RSUs (Detail) | The following table summarizes the total stock-based compensation expense for the stock options, RSAs and RSUs recorded in the consolidated statements of operations for the year ended December 31, 2020 and for the period from Inception to December 31, 2019 (in thousands): For the Year Ended December 31, 2020 For the period from September 20, 2019 (date of inception) to December 31, 2019 Research and development expense $ 1,167 $ — General and administrative expense 2,187 — Total $ 3,354 $ — |
Cancelled Options | |
Schedule of Assumptions Used to Estimate Fair Value of Stock Options and Cancelled Options | The following assumptions were used to estimate the fair value of the Cancelled Options that were granted on July 1, 2020: Risk-free interest rate 0.31 % Expected dividend yield — Expected term in years 6.3 Expected volatility 80 % |
Stock Options | |
Schedule of Assumptions Used to Estimate Fair Value of Stock Options and Cancelled Options | The following weighted-average assumptions were used to estimate the fair value of stock options, excluding the Cancelled Options, for the year ended December 31, 2020: Risk-free interest rate 0.54 % Expected dividend yield — Expected term in years 6.2 Expected volatility 77 % |
Schedule of Stock Option Activity, Excluding Cancelled Options | The following table summarizes stock option activity, excluding the Cancelled Options, during the year ended December 31, 2020: Weighted Weighted Average Aggregate Average Remaining Intrinsic Stock Exercise Contractual Value Options Price Life (in years) (in thousands) Outstanding at December 31, 2019 — $ — — $ — Options granted 674,842 20.68 Outstanding at December 31, 2020 674,842 $ 20.68 9.8 $ 3,953 Vested and expected to vest at December 31, 2020 674,842 $ 20.68 9.8 $ 3,953 Options exercisable at December 31, 2020 12,054 $ 20.25 9.8 $ 76 |
Net Loss Per Common Share (Tabl
Net Loss Per Common Share (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Earnings Per Share [Abstract] | |
Schedule of Calculation of Basic and Diluted Net Loss Per Common Share | The following table represents the calculation of basic and diluted net loss per common share for the year ended December 31, 2020 and the period from Inception to December 31, 2019 (in thousands, except share and per share data): For the Year Ended December 31, 2020 For the Period from September 20, 2019 (date of inception) to December 31, 2019 Net loss $ (60,011 ) $ (1,115 ) Weighted-average shares of common stock outstanding used to compute net loss per common share, basic and diluted 17,665,683 9,625,679 Net loss per common share, basic and diluted $ (3.40 ) $ (0.12 ) |
Schedule of Common Stock Equivalents Outstanding Excluded from Computation of Diluted Net Loss Per Share Attributable to Common Stockholders | The following common stock equivalents outstanding as of December 31, 2020 and 2019, respectively, were excluded from the computation of diluted net loss per share attributable to common stockholders for the periods presented because including them would have been anti‑dilutive: For the Year Ended December 31, 2020 For the Period from September 20, 2019 (date of inception) to December 31, 2019 Unvested RSUs 2,850,053 — Unvested RSAs 769,058 — Stock options 674,842 — Total 4,293,953 — |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Income Tax Disclosure [Abstract] | |
Schedule of Reconciliation of Federal Statutory Income Tax Rate | The effective tax rate of the Company's provision (benefit) for income taxes differs from the federal statutory rate as follows: For the Year Ended December 31, 2020 For the Period from September 20, 2019 (date of inception) to December 31, 2019 Statutory rate 21.00 % 21.00 % State tax 0.06 % — General business credits 1.06 % — Other permanent differences (0.25 )% — Change in valuation allowance (15.91 )% (21.00 )% Change in fair value of tranche liability (5.96 )% — Income tax provision (benefit) 0.00 % 0.00 % |
Schedule of Significant Components of Net Deferred Tax Asset | At December 31, 2020 and December 31, 2019, the significant components of the Company’s net deferred tax asset are as follows (in thousands): For the Year Ended December 31, 2020 For the Period from September 20, 2019 (date of inception) to December 31, 2019 Net operating loss carryforwards $ 5,810 $ — Tax credit carryforwards 672 — Accruals and reserves 334 — Other 21 — Intangibles 2,390 206 Organizational and start-up costs — 28 Non-qualified stock options 555 — Total deferred tax assets 9,782 234 Valuation allowance (9,782 ) (234 ) Deferred tax asset, net of allowance $ — $ — |
Commitments and Contingencies (
Commitments and Contingencies (Tables) | 12 Months Ended |
Dec. 31, 2020 | |
Commitments And Contingencies Disclosure [Abstract] | |
Schedule of Lease Commitments | The following table summarizes lease commitments as of December 31, 2020 (in thousands): Year Ending December 31, 2021 $ 314 2022 1,282 2023 1,317 2024 1,354 2025 1,391 Thereafter 17,599 Total Lease Commitments $ 23,257 |
Organization and Description _2
Organization and Description of Business Operations - Additional Information (Details) $ / shares in Units, $ in Thousands | Sep. 29, 2020shares | Sep. 28, 2020USD ($)$ / sharesshares | Sep. 16, 2020shares | Aug. 31, 2020USD ($)shares | Dec. 31, 2019USD ($) | Jul. 31, 2020USD ($)shares | Dec. 31, 2020USD ($)$ / sharesshares | Jul. 01, 2020shares | Jun. 30, 2020$ / shares |
Organization and Description of Business Operations [Line Items] | |||||||||
Date of incorporation | Feb. 13, 2020 | ||||||||
Share issue price per share | $ / shares | $ 7.52 | ||||||||
Accumulated deficit | $ | $ (1,115) | $ (61,126) | |||||||
Series A Convertible Preferred Stock | |||||||||
Organization and Description of Business Operations [Line Items] | |||||||||
Conversion of convertible preferred stock to common stock, shares | 10,000,000 | 10,000,000 | |||||||
Aggregate shares sold | 10,000,000 | ||||||||
Proceeds from issuances of convertible preferred stock, net of issuance costs | $ | 0 | $ 30,000 | $ 29,395 | ||||||
Series B Convertible Preferred Stock | |||||||||
Organization and Description of Business Operations [Line Items] | |||||||||
Conversion of convertible preferred stock to common stock, shares | 5,647,048 | 5,647,048 | |||||||
Aggregate shares sold | 5,647,048 | ||||||||
Proceeds from issuances of convertible preferred stock, net of issuance costs | $ | $ 96,000 | $ 0 | $ 95,815 | ||||||
IPO | |||||||||
Organization and Description of Business Operations [Line Items] | |||||||||
Issuance of shares of common stock, shares | 7,869,566 | 9,050,000 | |||||||
Share issue price per share | $ / shares | $ 20 | $ 20 | |||||||
Net proceeds from issuance of common stock | $ | $ 165,900 | ||||||||
Increase in outstanding shares | 26,097,378 | ||||||||
Underwriters’ Option to Purchase Additional Shares | |||||||||
Organization and Description of Business Operations [Line Items] | |||||||||
Issuance of shares of common stock, shares | 1,180,434 | 1,180,434 | |||||||
2020 Equity Incentive Plan | |||||||||
Organization and Description of Business Operations [Line Items] | |||||||||
Number of common stock authorized for issuance | 3,845,294 | ||||||||
Common Stock | |||||||||
Organization and Description of Business Operations [Line Items] | |||||||||
Conversion ratio | 1.0895 | 1.0895 | |||||||
Issuance of shares of common stock, shares | 9,050,000 | ||||||||
Conversion of convertible to common stock, shares | 17,047,378 | 17,047,378 | |||||||
Common Stock | 2020 Equity Incentive Plan | |||||||||
Organization and Description of Business Operations [Line Items] | |||||||||
Number of common stock authorized for issuance | 3,845,294 | 3,529,412 |
Significant Accounting Polici_3
Significant Accounting Policies - Additional Information (Details) - USD ($) | 3 Months Ended | 12 Months Ended | |
Dec. 31, 2019 | Dec. 31, 2020 | Sep. 30, 2020 | |
Significant Accounting Policies [Line Items] | |||
Assets | $ 15,000 | $ 258,881,000 | |
Cash equivalents | 0 | 0 | |
Deferred offering costs | 15,000 | 0 | |
Impairment losses | 0 | 0 | |
Liability for income tax associated with uncertain tax positions | 0 | ||
Unrecognized tax benefits to change significantly over next 12 months | 0 | ||
Interest and penalties associated with income tax positions | 0 | $ 0 | |
Minimum | |||
Significant Accounting Policies [Line Items] | |||
Property and equipment, useful life | 3 years | ||
Maximum | |||
Significant Accounting Policies [Line Items] | |||
Property and equipment, useful life | 5 years | ||
Series A Convertible Preferred Stock | |||
Significant Accounting Policies [Line Items] | |||
Deferred offering costs | 15,000 | ||
IPO | |||
Significant Accounting Policies [Line Items] | |||
Deferred offering costs | $ 2,500,000 | ||
United States | |||
Significant Accounting Policies [Line Items] | |||
Assets | $ 0 | $ 0 |
Property and Equipment - Schedu
Property and Equipment - Schedule of Property and Equipment (Details) $ in Thousands | Dec. 31, 2020USD ($) |
Property, Plant and Equipment [Line Items] | |
Property and equipment, gross | $ 296 |
Accumulated depreciation | (9) |
Property and equipment, net | 287 |
Computer Equipment | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, gross | 95 |
Construction in Progress | |
Property, Plant and Equipment [Line Items] | |
Property and equipment, gross | $ 201 |
Property and Equipment - Additi
Property and Equipment - Additional Information (Details) - USD ($) | 3 Months Ended | 12 Months Ended |
Dec. 31, 2019 | Dec. 31, 2020 | |
Property Plant And Equipment [Abstract] | ||
Depreciation expense | $ 0 | $ 9,000 |
Supplemental Financial Inform_3
Supplemental Financial Information - Schedule of Accrued Expenses And Other Current Liabilities (Details) - USD ($) $ in Thousands | Dec. 31, 2020 | Dec. 31, 2019 |
Balance Sheet Related Disclosures [Abstract] | ||
Accrued research and development | $ 2,106 | $ 7 |
Accrued compensation | 1,766 | |
Accrued professional fees | 999 | 122 |
Other | 264 | 21 |
Total accrued expenses and other current liabilities | $ 5,135 | $ 150 |
Supplemental Financial Inform_4
Supplemental Financial Information - Schedule of Prepaid Expenses And Other Current Assets (Details) $ in Thousands | Dec. 31, 2020USD ($) |
Balance Sheet Related Disclosures [Abstract] | |
Prepaid insurance | $ 2,480 |
Prepaid research and development | 2,462 |
Prepaid clinical trial | 944 |
Other | 740 |
Total prepaid expenses and other current assets | $ 6,626 |
Research, Collaboration, Gran_2
Research, Collaboration, Grant and License Agreements - Additional Information (Details) | Oct. 29, 2020USD ($) | Oct. 31, 2020USD ($) | Aug. 31, 2020USD ($) | Apr. 30, 2020USD ($)Product | Dec. 31, 2019USD ($)$ / sharesshares | Nov. 30, 2020USD ($) | Dec. 31, 2019USD ($)$ / sharesshares | Dec. 31, 2020USD ($)shares | Jun. 30, 2020$ / shares | Mar. 31, 2020shares | Feb. 21, 2020USD ($) | Nov. 30, 2019shares |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Common stock, shares issued | shares | 10,894,999 | 10,894,999 | 37,761,435 | |||||||||
Stock price per share | $ / shares | $ 7.52 | |||||||||||
Research and development | $ 987,000 | $ 31,893,000 | ||||||||||
UT Southwestern Agreement | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Preclinical activities period under sponsored research agreements | 2 years | |||||||||||
Minimum licensed product to develop obtain regulatory approval for and commercialize | Product | 1 | |||||||||||
Common stock, shares issued | shares | 0 | 2,179,000 | ||||||||||
Percentage of fully diluted common stock shares outstanding | 20.00% | |||||||||||
Percentage of entitled to receive additional shares on fully diluted basis | 10.00% | |||||||||||
UT Southwestern Agreement | Discount for Lack of Marketability | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Measurement input | 20 | 20 | ||||||||||
UT Southwestern Agreement | Common Stock | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Share issued an increase to common stock and additional paid in capital | $ 980,000 | |||||||||||
Stock price per share | $ / shares | $ 0.45 | $ 0.45 | ||||||||||
Queen's Agreement | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Research grant of committed fund amount | $ 3,800,000 | |||||||||||
Percentage of interest rate | 6.00% | 6.00% | ||||||||||
Research grant outstanding fund amount | 0 | |||||||||||
Queen's Agreement | Research and Development Expense | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Cash payment to acquire license agreement | $ 3,000,000 | |||||||||||
Queen's Agreement | Maximum | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
License agreement regulatory milestones payment | $ 10,000,000 | |||||||||||
License agreement commercial milestones payment | $ 10,000,000 | |||||||||||
Abeona CLN1 Agreements | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
License fee payment | $ 3,000,000 | |||||||||||
Purchase of clinical materials and reimbursement incurred development costs | $ 4,000,000 | |||||||||||
Research and development | 7,000,000 | |||||||||||
License fee | $ 3,000,000 | |||||||||||
Abeona CLN1 Agreements | Maximum | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
License and inventory purchase agreement regulatory related milestones payment | 26,000,000 | |||||||||||
License and inventory purchase agreement sales related milestones payment | $ 30,000,000 | |||||||||||
Abeona | Abeona Rett Agreement | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Payment of one-time upfront license fee | $ 3,000,000 | |||||||||||
Abeona | Maximum | Abeona Rett Agreement | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Regulatory related milestones obligation payable | 26,500,000 | |||||||||||
Sale-related milestones per licensed product and royalties on net sales of licensed products payable | $ 30,000,000 | |||||||||||
Catalent Agreements | ||||||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||||||
Payment of upfront project initiation fee | $ 2,000,000 |
Related Party Transactions - Ad
Related Party Transactions - Additional Information (Details) | 1 Months Ended | 12 Months Ended | ||
Mar. 31, 2020USD ($) | Jan. 31, 2020USD ($)PromissoryNote | Dec. 31, 2020USD ($) | Sep. 30, 2020USD ($) | |
PBM Capital Group, LLC | Services Agreement | ||||
Related Party Transaction [Line Items] | ||||
Accounting and other administrative and management services fee per month | $ 2,500 | |||
Due to related party | $ 9,800 | |||
PBM TGT Holdings, LLC | ||||
Related Party Transaction [Line Items] | ||||
Additional consideration payable under operating agreement | $ 0 | |||
RA Session II | ||||
Related Party Transaction [Line Items] | ||||
Number of secured promissory notes | PromissoryNote | 2 | |||
Paul B. Manning | Services Agreement | ||||
Related Party Transaction [Line Items] | ||||
Ownership percentage more than capital stock | 5.00% | |||
Secured Promissory Notes | RA Session II | ||||
Related Party Transaction [Line Items] | ||||
Aggregate amount, related party transaction | $ 1,670,000 | |||
Percentage of interest rate | 10.00% | |||
Interest expense related party | $ 28,000 |
Stockholders' Equity (Deficit_3
Stockholders' Equity (Deficit), Convertible Preferred Stock and Tranche Liability - Additional Information (Details) | Sep. 29, 2020shares | Sep. 28, 2020USD ($)$ / sharesshares | Sep. 16, 2020 | Jul. 28, 2020$ / sharesshares | Jul. 02, 2020USD ($)$ / sharesshares | Jun. 30, 2020USD ($)$ / sharesshares | Mar. 05, 2020shares | Mar. 04, 2020USD ($)$ / sharesshares | Nov. 19, 2019USD ($)shares | Aug. 31, 2020USD ($) | Dec. 31, 2019USD ($)$ / sharesshares | Jul. 31, 2020USD ($) | Dec. 31, 2020USD ($)$ / sharesshares |
Class Of Stock [Line Items] | |||||||||||||
Common stock, shares authorized | 200,000,000 | 32,685,000 | 10,895,000 | 200,000,000 | |||||||||
Common stock, par value per share | $ / shares | $ 0.00001 | $ 0.00001 | |||||||||||
Issuance of shares of common stock | $ | $ 165,854,000 | ||||||||||||
Equity-based awards | $ | $ 3,354,000 | ||||||||||||
Convertible preferred stock, shares authorized | 15,647,052 | ||||||||||||
Convertible preferred stock, shares authorized | 10,000,000 | 0 | 10,000,000 | ||||||||||
Preferred stock, shares issued | 0 | 0 | |||||||||||
Preferred stock, shares outstanding | 0 | 0 | |||||||||||
Share issue price per share | $ / shares | $ 7.52 | ||||||||||||
Preferred stock tranche liability | $ | $ 1,100,000 | ||||||||||||
Non cash expenses due to remeasured fair value of tranche liability | $ | $ 17,000,000 | $ 0 | $ (17,030,000) | ||||||||||
Mile stone shares issued during the period | 4,000,000 | 200,000 | |||||||||||
Tranche liability amount reclassified to convertible preferred stock | $ | $ 18,100,000 | ||||||||||||
IPO | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Shares issued during period | 7,869,566 | 9,050,000 | |||||||||||
Share issue price per share | $ / shares | $ 20 | $ 20 | |||||||||||
Gross proceeds from issuance of common stock | $ | $ 181,000,000 | ||||||||||||
Stock issuance costs, including underwriters’ discounts and commissions | $ | $ 15,100,000 | ||||||||||||
Increase in outstanding shares | 26,097,378 | ||||||||||||
Underwriters’ Option to Purchase Additional Shares | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Shares issued during period | 1,180,434 | 1,180,434 | |||||||||||
Series A Convertible Preferred Stock | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Convertible preferred stock, shares authorized | 10,000,000 | ||||||||||||
Conversion of convertible preferred stock to common stock, shares | 10,000,000 | 10,000,000 | |||||||||||
Number of share options exercised | 3,800,000 | 200,000 | |||||||||||
Clinical milestones for gross proceeds | $ | $ 11,400,000 | $ 600,000 | |||||||||||
Proceeds from issuances of convertible preferred stock, net of issuance costs | $ | 0 | $ 30,000,000 | $ 29,395,000 | ||||||||||
Convertible preferred stock tranche liability, offering price | $ / shares | $ 3 | ||||||||||||
Convertible preferred stock tranche liability, term | 1 year | ||||||||||||
Milestone pro-rate shares issued during the period | 200,000 | ||||||||||||
Series A Convertible Preferred Stock | Risk Free Rate | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Convertible preferred stock tranche liability, measurement input | 0.0059 | ||||||||||||
Series A Convertible Preferred Stock | Expected Volatility | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Convertible preferred stock tranche liability, measurement input | 0.80 | ||||||||||||
Series A Convertible Preferred Stock | Probability of Achieving Clinical Milestones and Timing | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Convertible preferred stock tranche liability, measurement input | 0.60 | ||||||||||||
Series A Convertible Preferred Stock | Private Placement | Series A Purchase Agreement | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Share issue price per share | $ / shares | $ 3 | ||||||||||||
Series A Convertible Preferred Stock | Private Placement | Series A Purchase Agreement | Maximum | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Shares issued during period | 10,000,000 | ||||||||||||
Series A Convertible Preferred Stock | Private Placement | Initial Closing | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Shares issued during period | 6,000,000 | ||||||||||||
Series A Convertible Preferred Stock | Private Placement | Achievement of Specific Clinical Milestone One | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Shares issued during period | 2,000,000 | ||||||||||||
Series A Convertible Preferred Stock | Private Placement | Achievement of Specific Clinical Milestone Two | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Shares issued during period | 2,000,000 | ||||||||||||
Series A Convertible Preferred Stock | Private Placement | Upon Achievement of Certain Defined Clinical Milestones | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Shares issued during period | 4,000,000 | ||||||||||||
Series B Convertible Preferred Stock | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Convertible preferred stock, shares authorized | 5,647,052 | ||||||||||||
Conversion of convertible preferred stock to common stock, shares | 5,647,048 | 5,647,048 | |||||||||||
Proceeds from issuances of convertible preferred stock, net of issuance costs | $ | $ 96,000,000 | 0 | $ 95,815,000 | ||||||||||
Convertible preferred stock tranche liability, offering price | $ / shares | $ 17 | ||||||||||||
Convertible preferred stock tranche liability, term | 6 months | ||||||||||||
Series B Convertible Preferred Stock | Risk Free Rate | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Convertible preferred stock tranche liability, measurement input | 0.0017 | ||||||||||||
Series B Convertible Preferred Stock | Expected Volatility | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Convertible preferred stock tranche liability, measurement input | 0.80 | ||||||||||||
Series B Convertible Preferred Stock | Series B Purchase Agreement | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Share issue price per share | $ / shares | $ 17 | ||||||||||||
Series B Convertible Preferred Stock | Private Placement | Series B Purchase Agreement | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Shares issued during period | 5,647,048 | ||||||||||||
Share issue price per share | $ / shares | $ 17 | ||||||||||||
Proceeds from issuances of convertible preferred stock, net of issuance costs | $ | $ 96,000,000 | ||||||||||||
Series B Convertible Preferred Stock | Private Placement | Series B Purchase Agreement | Maximum | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Shares issued during period | 5,647,052 | ||||||||||||
UT System in Connection with UT Southwestern Agreement | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Shares issued during period | 2,179,000 | ||||||||||||
Issuance of shares of common stock | $ | $ 980,000 | ||||||||||||
Equity-based awards | $ | $ 0 | ||||||||||||
PBM TGT Holdings, LLC | Series A Convertible Preferred Stock | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Shares issued during period | 3,266,667 | ||||||||||||
Nolan Capital, LLC | Series A Convertible Preferred Stock | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Shares issued during period | 400,000 | ||||||||||||
Common Stock | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Shares issued during period | 9,050,000 | ||||||||||||
Amount owed for issuance settled month and year | 2020-07 | ||||||||||||
Conversion of stock, shares issued | 17,047,378 | 17,047,378 | |||||||||||
Conversion ratio | 1.0895 | 1.0895 | |||||||||||
RA Session II | Common Stock | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Shares issued during period | 8,661,524 | ||||||||||||
One Other Individual | Common Stock | |||||||||||||
Class Of Stock [Line Items] | |||||||||||||
Shares issued during period | 54,475 |
Stockholders' Equity (Deficit_4
Stockholders' Equity (Deficit), Convertible Preferred Stock and Tranche Liability - Reconciliation of Preferred Stock Tranche Liability Measured at Fair Value (Details) - Preferred Stock Tranche Liability $ in Thousands | 12 Months Ended |
Dec. 31, 2020USD ($) | |
Fair Value Liabilities Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | |
Fair value at issuance of Series A convertible preferred stock | $ 1,050 |
Change in fair value | 17,030 |
Settlement of preferred stock tranche liability due to issuance of Milestone Shares | $ (18,080) |
Stock-Based Compensation - Addi
Stock-Based Compensation - Additional Information (Details) - USD ($) | Sep. 16, 2020 | Sep. 02, 2020 | Jul. 01, 2020 | Dec. 31, 2020 | Dec. 31, 2020 | Jul. 02, 2020 |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||
Fair value of common stock per share | $ 5.28 | |||||
Series B Convertible Preferred Stock | ||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||
Preferred stock, issuing price per share | $ 17 | |||||
Employee Stock Purchase Plan (“ESPP”) | ||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||
Common stock reserved for future issuance | 724,000 | |||||
Share-based payment award, expiration period | 10 years | |||||
Share-based payment award, expiration date | Jan. 1, 2030 | |||||
Percentage of automatic increase every year in common stock shares reserved for future issuance | 1.00% | |||||
Share-based payment award, terms of award issuance | The number of shares of common stock that are automatically added on January 1st of each year for a period of up to ten years, commencing on the first January 1 following the IPO Date and ending on (and including) January 1, 2030, in an amount equal to the lesser of (i) one percent (1.0%) of the total number of shares of capital stock outstanding on December 31st of the preceding calendar year, and (ii) 724,000 shares of common stock | |||||
Number of shares of common stock issued under ESPP | 0 | |||||
Restricted Stock Units | ||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||
Number of shares awarded | 2,518,932 | |||||
Share-based payment award, compensation cost not yet recognized, weighted-average period of recognition | 2 years 1 month 6 days | |||||
Share-based payment award, RSU/RSA granted | 331,121 | |||||
Share-based payment award, unvested RSU/RSA granted, compensation cost not yet recognized | $ 16,200,000 | $ 16,200,000 | ||||
Share-based payment award, RSU/RSA granted, weighted average grant date fair value | $ 14.90 | |||||
Stock Options | ||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||
Share-based payment award, expiration period | 10 years | |||||
Share-based payment award, options granted | 674,842 | |||||
Share-based payment award, options granted, exercise price per share | $ 20.68 | |||||
Share-based payment award, options, weighted-average grant date fair value | $ 13.73 | |||||
Share-based payment award, options, compensation cost not yet recognized | 8,800,000 | $ 8,800,000 | ||||
Share-based payment award, compensation cost not yet recognized, weighted-average period of recognition | 3 years 7 months 6 days | |||||
Restricted Stock Awards | ||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||
Share-based payment award, compensation cost not yet recognized, weighted-average period of recognition | 2 years 3 months 18 days | |||||
Share-based payment award, RSU/RSA granted | 769,058 | |||||
Share-based payment award, unvested RSU/RSA granted, compensation cost not yet recognized | 3,200,000 | $ 3,200,000 | ||||
Share-based payment award, RSU/RSA granted, weighted average grant date fair value | $ 5.28 | $ 5.28 | ||||
Maximum | Employee Stock Purchase Plan (“ESPP”) | ||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||
Number of shares authorized for issuance | 362,000 | |||||
Maximum | Stock Options | ||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||
Award vesting period | 4 years | |||||
Minimum | Stock Options | ||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||
Award vesting period | 3 years | |||||
2020 Equity Incentive Plan | ||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||
Number of shares authorized for issuance | 3,845,294 | |||||
Common stock reserved for future issuance | 209,841 | |||||
Share-based payment award, plan modification, incremental cost | $ 0 | |||||
Share-based payment award, compensation cost not yet recognized | $ 13,200,000 | $ 13,200,000 | ||||
2020 Equity Incentive Plan | Cancelled Options | Certain Employees and Consultants | ||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||
Share-based payment award, options granted | 2,896,782 | |||||
Share-based payment award, options granted, exercise price per share | $ 0.80 | |||||
Award vesting period | 4 years | |||||
Share-based payment award, options grant date fair value | $ 13,800,000 | |||||
2020 Equity Incentive Plan | Restricted Stock Units | ||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||
Award vesting period | 4 years | |||||
Number of shares awarded | 2,518,932 | |||||
Share-based payment award, RSU/RSA granted | 331,121 | |||||
2020 Equity Incentive Plan | Restricted Stock Units | Share-based Payment Arrangement, Tranche One | ||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||
Share-based payment award, vesting rights percentage | 25.00% | |||||
2020 Equity Incentive Plan | Restricted Stock Units | Share-based Payment Arrangement, Tranche Two | ||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||
Share-based payment award, vesting rights percentage | 25.00% | |||||
2020 Equity Incentive Plan | Restricted Stock Units | Share-based Payment Arrangement, Tranche Three | ||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||
Share-based payment award, vesting rights percentage | 25.00% | |||||
2020 Equity Incentive Plan | Restricted Stock Units | Share-based Payment Arrangement, Tranche Four | ||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||
Share-based payment award, vesting rights percentage | 25.00% | |||||
2020 Equity Incentive Plan | Stock Options | Directors | ||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||
Share-based payment award, options granted | 16,342 | |||||
Share-based payment award, options granted, exercise price per share | $ 14.90 | |||||
2020 Equity Incentive Plan | Restricted Stock Awards | RA Session II | ||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||
Share-based payment award, RSU/RSA granted | 769,058 | |||||
Award requisite service vesting period | 3 years | |||||
2020 Equity Incentive Plan | Common Stock | ||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||
Number of shares authorized for issuance | 3,845,294 | 3,529,412 | ||||
New Plan | ||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||
Common stock reserved for future issuance | 3,390,168 | 2,941,509 | 2,941,509 | |||
Share-based payment award, expiration period | 10 years | |||||
Share-based payment award, commencement date | Jan. 1, 2021 | |||||
Share-based payment award, expiration date | Jan. 1, 2030 | |||||
Percentage of automatic increase every year in common stock shares reserved for future issuance | 5.00% | |||||
Share-based payment award, terms of award issuance | At December 31, 2020, there were 2,941,509, shares available for future grant under the New Plan. The number of shares of common stock reserved for issuance under the New Plan will automatically increase on January 1 of each year, for a period of ten years, from January 1, 2021 continuing through January 1, 2030, by 5% of the total number of shares of common stock outstanding on December 31 of the preceding calendar year, or a lesser number of shares as may be determined by the Company’s board of directors. | |||||
New Plan | Stock Options | ||||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||||
Share-based payment award, options granted | 658,500 | |||||
Share-based payment award, options granted, exercise price per share | $ 20.83 |
Stock-Based Compensation - Sche
Stock-Based Compensation - Schedule of Assumptions Used to Estimate Fair Value of Stock Options and Cancelled Options (Details) | Jul. 01, 2020 | Dec. 31, 2020 |
Cancelled Options | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Risk-free interest rate | 0.31% | |
Expected term in years | 6 years 3 months 18 days | |
Expected volatility | 80.00% | |
Stock Options | ||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Risk-free interest rate | 0.54% | |
Expected term in years | 6 years 2 months 12 days | |
Expected volatility | 77.00% |
Stock-Based Compensation - Sc_2
Stock-Based Compensation - Schedule of Stock Option Activity, Excluding Cancelled Options (Details) - Stock Options - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended |
Dec. 31, 2020 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Outstanding. Stock Options, Beginning Balance | |
Options granted, Stock Options | 674,842 |
Outstanding. Stock Options, Ending Balance | 674,842 |
Vested and expected to vest at December 31, 2020, Stock Options | 674,842 |
Options exercisable at December 31, 2020, Stock Options | 12,054 |
Outstanding, Weighted Average Exercise Price, Beginning balance | |
Options granted, Weighted Average Exercise Price | $ 20.68 |
Outstanding, Weighted Average Exercise Price, Ending balance | 20.68 |
Vested and expected to vest at December 31, 2020, Weighted Average Exercise Price | 20.68 |
Options exercisable at December 31, 2020, Weighted Average Exercise Price | $ 20.25 |
Outstanding, Weighted Average Remaining Contractual Life | 9 years 9 months 18 days |
Vested and expected to vest at December 31, 2020, Weighted Average Remaining Contractual Life | 9 years 9 months 18 days |
Weighted Average Remaining Contractual Life, Options exercisable at December 31, 2020 | 9 years 9 months 18 days |
Outstanding, Aggregate Intrinsic Value, Beginning Balance | |
Outstanding, Aggregate Intrinsic Value, Ending Balance | $ 3,953 |
Vested and expected to vest at December 31, 2020, Aggregate Intrinsic Value | 3,953 |
Options exercisable at December 31, 2020, Aggregate Intrinsic Value | $ 76 |
Stock-Based Compensation - Sc_3
Stock-Based Compensation - Schedule of Restricted Stock Units Activity (Details) - Restricted Stock Units | 12 Months Ended |
Dec. 31, 2020$ / sharesshares | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Replacement restricted units granted, Number of Shares | shares | 2,518,932 |
Restricted units/stock granted, Number of Shares | shares | 331,121 |
Nonvested, Number of Shares, Ending Balance | shares | 2,850,053 |
Replacement restricted units granted, Weighted Average Grant Date Fair Value per Share | $ / shares | $ 5.25 |
Restricted units/stock granted, Weighted Average Grant Date Fair Value per Share | $ / shares | 14.90 |
Nonvested, Weighted Average Grant Date Fair Value per Share, Ending Balance | $ / shares | $ 6.37 |
Stock-Based Compensation - Sc_4
Stock-Based Compensation - Schedule of Restricted Stock Awards Activity (Details) - Restricted Stock Awards - $ / shares | Jul. 01, 2020 | Dec. 31, 2020 |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||
Restricted units/stock granted, Number of Shares | 769,058 | |
Nonvested, Number of Shares, Ending Balance | 769,058 | |
Restricted units/stock granted, Weighted Average Grant Date Fair Value per Share | $ 5.28 | $ 5.28 |
Nonvested, Weighted Average Grant Date Fair Value per Share, Ending Balance | $ 5.28 |
Stock-Based Compensation - Sc_5
Stock-Based Compensation - Schedule of Total Stock-Based Compensation Expense for Stock Options, RSAs and RSUs (Details) $ in Thousands | 12 Months Ended |
Dec. 31, 2020USD ($) | |
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | |
Stock-based compensation expense | $ 3,354 |
Research and Development Expense | |
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | |
Stock-based compensation expense | 1,167 |
General and Administrative Expense | |
Employee Service Share Based Compensation Allocation Of Recognized Period Costs [Line Items] | |
Stock-based compensation expense | $ 2,187 |
Net Loss Per Common Share - Sch
Net Loss Per Common Share - Schedule of Calculation of Basic and Diluted Net Loss Per Common Share (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended |
Dec. 31, 2019 | Dec. 31, 2020 | |
Earnings Per Share [Abstract] | ||
Net loss | $ (1,115) | $ (60,011) |
Weighted average common shares outstanding, basic and diluted | 9,625,679 | 17,665,683 |
Net loss per common share, basic and diluted | $ (0.12) | $ (3.40) |
Net Loss Per Common Share - S_2
Net Loss Per Common Share - Schedule of Common Stock Equivalents Outstanding Excluded from Computation of Diluted Net Loss Per Share Attributable to Common Stockholders (Details) | 12 Months Ended |
Dec. 31, 2020shares | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | |
Anti-dilutive securities excluded from computation of diluted net loss per share | 4,293,953 |
Unvested RSUs | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | |
Anti-dilutive securities excluded from computation of diluted net loss per share | 2,850,053 |
Unvested RSAs | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | |
Anti-dilutive securities excluded from computation of diluted net loss per share | 769,058 |
Stock Options | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | |
Anti-dilutive securities excluded from computation of diluted net loss per share | 674,842 |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Details) - USD ($) | 3 Months Ended | 12 Months Ended | ||
Dec. 31, 2019 | Dec. 31, 2020 | Dec. 31, 2019 | Dec. 31, 2018 | |
Income Tax Disclosure [Line Items] | ||||
Provision for income taxes | $ 0 | |||
Net operating loss carryforward percentage | 80.00% | 80.00% | 80.00% | |
Deferred tax liabilities | $ 0 | $ 0 | $ 0 | |
Valuation allowance increased | 200,000 | 9,500,000 | ||
Net operating loss carryforward | 0 | 0 | ||
Tax credit carryforwards | $ 0 | 672,000 | $ 0 | |
Federal | ||||
Income Tax Disclosure [Line Items] | ||||
Net operating loss carryforward | 27,700,000 | |||
Tax credit carryforwards | $ 900,000 | |||
Tax credit carryforward expiration or due date year | 2040 | |||
Minimum | ||||
Income Tax Disclosure [Line Items] | ||||
Tax Cuts and Jobs Act, adjusted taxable income limitation percentage | 0.30 | 0.30 | ||
Maximum | ||||
Income Tax Disclosure [Line Items] | ||||
Tax Cuts and Jobs Act, adjusted taxable income limitation percentage | 0.50 | 0.50 | ||
Maximum | State | ||||
Income Tax Disclosure [Line Items] | ||||
Tax credit carryforwards | $ 100,000 |
Income Taxes - Schedule of Reco
Income Taxes - Schedule of Reconciliation of Federal Statutory Income Tax Rate (Details) | 3 Months Ended | 12 Months Ended |
Dec. 31, 2019 | Dec. 31, 2020 | |
Income Tax Disclosure [Abstract] | ||
Statutory rate | 21.00% | 21.00% |
State tax | 0.06% | |
General business credits | 1.06% | |
Other permanent differences | (0.25%) | |
Change in valuation allowance | (21.00%) | (15.91%) |
Change in fair value of tranche liability | (5.96%) | |
Income tax provision (benefit) | 0.00% | 0.00% |
Income Taxes - Schedule of Sign
Income Taxes - Schedule of Significant Components of Net Deferred Tax Asset (Details) - USD ($) | Dec. 31, 2020 | Dec. 31, 2019 |
Components Of Deferred Tax Assets [Abstract] | ||
Net operating loss carryforwards | $ 5,810,000 | |
Tax credit carryforwards | 672,000 | $ 0 |
Accruals and reserves | 334,000 | |
Other | 21,000 | |
Intangibles | 2,390,000 | 206,000 |
Organizational and start-up costs | 28,000 | |
Non-qualified stock options | 555,000 | |
Total deferred tax assets | 9,782,000 | 234,000 |
Valuation allowance | (9,782,000) | (234,000) |
Deferred tax asset, net of allowance |
Commitments and Contingencies -
Commitments and Contingencies - Additional Information (Details) $ in Millions | Dec. 17, 2020ft² | Dec. 31, 2020USD ($) |
Commitments And Contingencies Disclosure [Abstract] | ||
Number of operating lease landlord square feet manufacturing facility | ft² | 187,500 | |
Operating lease commencement date | Apr. 1, 2021 | |
Operating lease term of contract | 15 years 6 months | |
Operating lease extend term of contract | 5 years | |
Operating leases initial direct costs | $ | $ 0.8 |
Commitments and Contingencies_2
Commitments and Contingencies - Schedule of Lease Commitments (Details) $ in Thousands | Dec. 31, 2020USD ($) |
Commitments And Contingencies Disclosure [Abstract] | |
2021 | $ 314 |
2022 | 1,282 |
2023 | 1,317 |
2024 | 1,354 |
2025 | 1,391 |
Thereafter | 17,599 |
Total Lease Commitments | $ 23,257 |
Subsequent Events - Additional
Subsequent Events - Additional Information (Details) $ / shares in Units, $ in Thousands | Jan. 11, 2021USD ($)ft² | Mar. 31, 2021$ / sharesshares | Dec. 31, 2020USD ($) | Dec. 17, 2020 |
Subsequent Event [Line Items] | ||||
Term of lease period | 15 years 6 months | |||
Total future minimum lease payments | $ 23,257 | |||
Subsequent Event | ||||
Subsequent Event [Line Items] | ||||
Options granted for purchase an aggregate share of common stock | shares | 1,621,900 | |||
Weighted-average exercise price | $ / shares | $ 28.81 | |||
Subsequent Event | Dallas Lease | Pegasus Park, LLC | ||||
Subsequent Event [Line Items] | ||||
Office space area under lease agreement | ft² | 15,000 | |||
Term of lease period | 10 years | |||
Lease, option to extend description | Company has an option to extend the term of the Dallas Lease for one additional period of five years. | |||
Lease, option to extend | true | |||
Extending term of lease | 5 years | |||
Total future minimum lease payments | $ 4,900 |