0001140361-22-007036 | 10-K | XBRL Viewer | Rocket Pharmaceuticals INC

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Consolidated Balance Sheets - USD ($) $ in Thousands	Dec. 31, 2021	Dec. 31, 2020
Current assets:
Cash and cash equivalents	$ 232,694	$ 297,098
Investments	156,046	185,621
Prepaid expenses and other current assets	3,319	4,626
Total current assets	392,059	487,345
Property and equipment, net	22,299	19,206
Goodwill	30,815	30,815
Restricted cash	1,343	1,568
Deposits	455	455
Operating lease right-of-use assets	1,569	914
Finance lease right-of-use asset	48,480	50,521
Total assets	497,020	590,824
Current liabilities:
Accounts payable and accrued expenses	19,615	25,472
Convertible notes, net of unamortized discount, current	0	4,875
Operating lease liabilities, current	863	626
Finance lease liability, current	1,689	1,644
Total current liabilities	22,167	32,617
Convertible notes, net of unamortized discount, non-current	0	35,066
Operating lease liabilities, non-current	905	498
Finance lease liability, non-current	19,144	18,988
Other liabilities	80	136
Total liabilities	42,296	87,305
Commitments and contingencies (Note 12)
Stockholders' equity:
Common stock, $0.01 par value, 120,000,000 shares authorized; 64,505,889 and 60,996,367 shares issued and outstanding at December 31, 2021 and 2020, respectively	645	610
Additional paid-in capital	946,152	825,794
Accumulated other comprehensive loss	(161)	(42)
Accumulated deficit	(491,912)	(322,843)
Total stockholders' equity	454,724	503,519
Total liabilities and stockholders' equity	497,020	590,824
Series A Convertible Preferred Stock [Member]
Stockholders' equity:
Preferred stock	0	0
Series B Convertible Preferred Stock [Member]
Stockholders' equity:
Preferred stock	$ 0	$ 0

Consolidated Balance Sheets (Parenthetical) - $ / shares	Dec. 31, 2021	Dec. 31, 2020
Shareholders' equity:
Preferred stock, par value (in dollars per share)	$ 0.01	$ 0.01
Preferred stock, shares authorized (in shares)	5,000,000	5,000,000
Common stock, par value (in dollars per share)	$ 0.01	$ 0.01
Common stock, shares authorized (in shares)	120,000,000	120,000,000
Common stock, shares issued (in shares)	64,505,889	60,996,367
Common stock, shares outstanding (in shares)	64,505,889	60,996,367
Series A Convertible Preferred Stock [Member]
Shareholders' equity:
Preferred stock, shares authorized (in shares)	300,000	300,000
Preferred stock, shares issued (in shares)	0	0
Preferred stock, shares outstanding (in shares)	0	0
Series B Convertible Preferred Stock [Member]
Shareholders' equity:
Preferred stock, shares authorized (in shares)	300,000	300,000
Preferred stock, shares issued (in shares)	0	0
Preferred stock, shares outstanding (in shares)	0	0

Consolidated Statements of Operations - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019
Consolidated Statements of Operations [Abstract]
Revenue	$ 0	$ 0	$ 0
Operating expenses:
Research and development	125,476	105,438	57,907
General and administrative	41,772	28,865	18,244
Total operating expenses	167,248	134,303	76,151
Loss from operations	(167,248)	(134,303)	(76,151)
Research and development incentives	1,000	0	250
Interest expense	(2,977)	(6,967)	(5,958)
Interest and other income, net	3,068	2,150	3,414
(Amortization of premium) accretion of discount on investments - net	(2,912)	(580)	1,175
Net loss	$ (169,069)	$ (139,700)	$ (77,270)
Net loss per share attributable to common stockholders - basic (in dollars per share)	$ (2.67)	$ (2.52)	$ (1.58)
Net loss per share attributable to common stockholders - diluted (in dollars per share)	$ (2.67)	$ (2.52)	$ (1.58)
Weighted-average common shares outstanding - basic (in shares)	63,235,417	55,380,740	49,010,358
Weighted-average common shares outstanding - diluted (in shares)	63,235,417	55,380,740	49,010,358

Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019
Consolidated Statements of Comprehensive Loss [Abstract]
Net loss	$ (169,069)	$ (139,700)	$ (77,270)
Other comprehensive loss
Net unrealized (loss) gain on investments	(119)	(62)	147
Total comprehensive loss	$ (169,188)	$ (139,762)	$ (77,123)

- Definition

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

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+ Details

Consolidated Statements of Changes in Stockholders' Equity - USD ($) $ in Thousands	Common Stock [Member]	Treasury Stock [Member]	Additional Paid-In Capital [Member]	Accumulated Other Comprehensive Income/(Loss) [Member]	Accumulated Deficit [Member]	Total
Beginning Balance at Dec. 31, 2018	$ 452	$ (668)	$ 300,253	$ (127)	$ (105,873)	$ 194,037
Beginning Balance (in shares) at Dec. 31, 2018	45,194,736
Issuance of common stock, net of issuance costs	$ 96	0	177,664	0	0	177,760
Issuance of common stock, net of issuance costs (in shares)	9,568,000
Issuance of common stock pursuant to exercise of stock options	$ 1	0	30	0	0	31
Issuance of common stock pursuant to exercise of stock options (in shares)	110,325
Treasury stock purchases	$ 0	(725)		0	0	(726)
Treasury stock purchases			(1)
Treasury stock purchases (in shares)	0
Issuance of treasury stock pursuant to exercise of stock options	$ 0	(397)	0	0	0	(397)
Issuance of treasury stock pursuant to exercise of stock options (in shares)	0
Retirement of treasury stock	$ (1)	1,393	(1,392)	0	0	0
Retirement of treasury stock (in shares)	(100,000)
Sale of treasury stock	$ 0	344	0	0	0	344
Sale of treasury stock (in shares)	0
Unrealized comprehensive gain (loss) on investments	$ 0	0	0	147	0	147
Share-based compensation	0	0	13,371	0	0	13,371
Net loss	0	0	0	0	(77,270)	(77,270)
Ending Balance at Dec. 31, 2019	$ 548	(53)	489,925	20	(183,143)	307,297
Ending Balance (in shares) at Dec. 31, 2019	54,773,061
Issuance of common stock, net of issuance costs	$ 53	0	280,710	0	0	280,763
Issuance of common stock, net of issuance costs (in shares)	5,339,286
Issuance of common stock pursuant to exercise of stock options	$ 6	0	2,552	0	0	2,558
Issuance of common stock pursuant to exercise of stock options (in shares)	586,857
Issuance of treasury stock pursuant to exercise of stock options	$ 0	(614)	0	0	0	(614)
Issuance of treasury stock pursuant to exercise of stock options (in shares)	0
Issuance of common stock pursuant to exercise of warrant (in shares)	1,601
Issuance of common stock pursuant to conversion of notes	$ 3	0	7,626	0	0	7,629
Issuance of common stock pursuant to conversion of notes (in shares)	298,562
Issuance of warrants			26,562			26,562
Stock repurchase	$ 0	0	(72)	0	0	(72)
Stock repurchase (in shares)	(3,000)
Sale of treasury stock	$ 0	667	(76)	0	0	591
Sale of treasury stock (in shares)	0
Unrealized comprehensive gain (loss) on investments	$ 0	0	0	(62)	0	(62)
Share-based compensation	0	0	18,567	0	0	18,567
Net loss	0	0	0	0	(139,700)	(139,700)
Ending Balance at Dec. 31, 2020	$ 610	0	825,794	(42)	(322,843)	503,519
Ending Balance (in shares) at Dec. 31, 2020	60,996,367
Issuance of common stock, net of issuance costs	$ 8	0	26,346	0	0	26,354
Issuance of common stock, net of issuance costs (in shares)	812,516
Issuance of common stock pursuant to exercise of stock options	$ 12	0	11,315	0	0	11,327
Issuance of common stock pursuant to exercise of stock options (in shares)	1,209,960
Issuance of common stock pursuant to conversion of notes	$ 15	0	40,679	0	0	40,694
Issuance of common stock pursuant to conversion of notes (in shares)	1,487,046
Issuance of warrants			12,781			12,781
Unrealized comprehensive gain (loss) on investments	$ 0	0	0	(119)	0	(119)
Share-based compensation	0	0	29,237	0	0	29,237
Net loss	0	0	0	0	(169,069)	(169,069)
Ending Balance at Dec. 31, 2021	$ 645	$ 0	$ 946,152	$ (161)	$ (491,912)	$ 454,724
Ending Balance (in shares) at Dec. 31, 2021	64,505,889

Consolidated Statements of Cash Flows - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019
Operating Activities:
Net loss	$ (169,069)	$ (139,700)	$ (77,270)
Adjustments to reconcile net loss to net cash used in operating activities:
Accretion of discount on convertible notes	753	2,758	3,602
Depreciation and amortization of property and equipment	3,240	1,145	426
Amortization of right of use asset	2,133	5,105	0
Write down of property and equipment, net	261	419	0
Stock-based compensation	29,237	18,567	13,371
Expense in connection with warrant issuances	12,781	26,562	0
Accretion of discount (amortization of premium) on investments, net	2,887	580	(1,066)
Changes in operating assets and liabilities:
Prepaid expenses and other assets	1,307	(1,517)	(917)
Accounts payable and accrued expenses	(4,827)	11,015	(2,722)
Operating lease liabilities	(11)	(139)	(87)
Finance lease liability	201	452	0
Other long term liabilities	(56)	113	0
Net cash used in operating activities	(121,163)	(74,640)	(64,663)
Investing activities:
Purchases of investments	(245,875)	(209,343)	(184,298)
Proceeds from maturities of investments	272,443	141,811	168,556
Payments made to acquire right of use asset	(95)	(8,452)	0
Purchases of property and equipment	(7,620)	(20,607)	(23,269)
Net cash provided by (used in) investing activities	18,853	(96,591)	(39,011)
Financing activities:
Issuance of common stock, net of issuance costs	26,354	280,763	177,760
Issuance of common stock, pursuant to exercise of stock options	11,327	2,558	31
Common stock repurchase	0	(72)	0
Proceeds from sale of treasury stock	0	591	344
Payment of withholding tax on option exercises	0	(614)	(344)
Convertible notes refinancing costs to the lender	0	(237)	0
Net cash provided by financing activities	37,681	282,989	177,791
Net change in cash, cash equivalents and restricted cash	(64,629)	111,758	74,117
Cash, cash equivalents and restricted cash at beginning of period	298,666	186,908	112,791
Cash, cash equivalents and restricted cash at end of period	234,037	298,666	186,908
Supplemental disclosure of non-cash financing and investing activities:
Accrued purchases of property and equipment	728	1,756	4,650
Retirement of treasury stock	0	0	1,393
Treasury stock purchases paid in prior year	0	0	726
Withholding tax payable on shares withheld in treasury stock	0	0	53
Unrealized (loss) gain on investments	(119)	(62)	147
Conversion of 2021 and 2022 convertible notes into common stock	40,694	7,629	0
Finance lease right of use asset and lease liability	0	20,179	0
Reclassification of construction in process to finance right of use asset	98	26,465	0
Supplemental cash flow information:
Cash paid for interest	148	2,960	2,990
Cash paid for income taxes	$ 0	$ 0	$ 26

Nature of Business and Basis of Presentation

12 Months Ended

Dec. 31, 2021

Nature of Business and Basis of Presentation [Abstract]

Nature of Business and Basis of Presentation

1.	Nature of Business and Basis of Presentation

Rocket Pharmaceuticals, Inc. (“Rocket” or the “Company”) is a clinical-stage, multi-platform biotechnology company focused on the development of gene therapies, with direct on-target mechanism of action and clear clinical endpoints, for rare and devastating diseases. Rocket has three clinical-stage ex vivo lentiviral vector (“LVV”) programs. These include programs for Fanconi Anemia (“FA”), a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells, Leukocyte Adhesion Deficiency-I (“LAD-I”), a genetic disorder that causes the immune system to malfunction and Pyruvate Kinase Deficiency (“PKD”), a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. Of these, both the Phase 2 FA program and the Phase 1/2 LAD-I program are in registration-enabling studies in the United States (“U.S.”) and Europe (“EU”). In addition, in the U.S., Rocket has a clinical stage in vivo adeno-associated virus (“AAV”) program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure. Additional discovery efforts on a gene therapy program for the less common FA subtypes C and G is ongoing. The Company has global commercialization and development rights to all of these product candidates under royalty-bearing license agreements.

Effective December 2021, the Company made a decision to no longer pursue Rocket-sponsored clinical evaluation of RP-L401; this program is to be returned to academic innovators. The Company has opted to focus available resources towards advancement of RP-A501, RP-L102, RP-L201 and RP-L301, based on the clinical data to date and potential for therapeutic advancement in these severe disorders of childhood and young adulthood.

Risks and Liquidity

12 Months Ended

Dec. 31, 2021

Risks and Liquidity [Abstract]

Risks and Liquidity

2.	Risks and Liquidity

The Company has not generated any revenue and has incurred losses since inception. Operations of the Company are subject to certain risks and uncertainties, including, among others, uncertainty of drug candidate development, technological uncertainty, uncertainty regarding patents and proprietary rights, having no commercial manufacturing experience, marketing or sales capability or experience, dependency on key personnel, compliance with government regulations and the need to obtain additional financing. Drug candidates currently under development will require significant additional research and development efforts, including extensive preclinical and clinical testing and regulatory approval, prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel infrastructure, and extensive compliance-reporting capabilities.

The Company’s product candidates are in the development and clinical stage. There can be no assurance that the Company’s research and development will be successfully completed, that adequate protection for the Company’s intellectual property will be obtained, that any products developed will obtain necessary government approval or that any approved products will be commercially viable. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will generate significant revenue from product sales. The Company operates in an environment of rapid change in technology and substantial competition from pharmaceutical and biotechnology companies.

The Company’s consolidated financial statements have been prepared on the basis of continuity of operations, realization of assets and the satisfaction of liabilities in the ordinary course of business. The Company has experienced negative cash flows from operations and had an accumulated deficit of $491.9 million as of December 31, 2021. As of December 31, 2021, the Company has $388.7 million of cash, cash equivalents and investments. During the year ended December 31, 2021, the Company issued and sold 812,516 shares of its common stock at a purchase price of $32.48 per share for aggregate net proceeds of approximately $26.4 million in a private placement transaction to a fund affiliated with RTW Investments, LP., the Company’s largest shareholder (see Note 15). The Company expects such resources will be sufficient to fund the Company’s operating expenses and capital expenditure requirements into the second half of 2023.

In April 2021, the Company called for the redemption of the remaining $38.4 million principal balance of the Company’s 6.25%, 2022 Convertible Senior Notes due 2022 which were converted into common stock. On August 2, 2021, holders of $5.15 million of the 2021 Convertible Notes converted the remaining $5.15 million remaining balance of the 2021 Convertible Notes into common stock (see Note 7). As of December 31, 2021, none of the 2021 Convertible Notes or 2022 Convertible Notes were outstanding.

In the longer term, the future viability of the Company is dependent on its ability to generate cash from operating activities or to raise additional capital to finance its operations. The Company’s failure to raise capital as and when needed could have a negative impact on its financial condition and ability to pursue its business strategies.

Summary of Significant Accounting Policies

12 Months Ended

Dec. 31, 2021

Summary of Significant Accounting Policies [Abstract]

Summary of Significant Accounting Policies

3.	Summary of Significant Accounting Policies

Principles of Consolidation

The consolidated financial statements represent the consolidation of the accounts of the Company and its subsidiaries in conformity with accounting principles generally accepted in the United States (“US GAAP”). All intercompany accounts have been eliminated in consolidation.

Use of Estimates

The preparation of the consolidated financial statements in conformity with US 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. Significant estimates and assumptions reflected in these consolidated financial statements include but are not limited to goodwill impairment, the accrual of research and development expenses, the valuation of equity transactions, and stock-based awards. Changes in estimates and assumptions are reflected in reported results in the period in which they become known. Actual results could differ from those estimates.

Cash, Cash Equivalents and Restricted Cash

Cash, cash equivalents and restricted cash consists of bank deposits, certificates of deposit and money market accounts with financial institutions. Cash equivalents are carried at cost which approximates fair value due to their short-term nature and which the Company believes do not have a material exposure to credit risk. The Company considers all highly liquid investments with maturities of three months or less from the date of purchase to be cash equivalents. The Company’s cash and cash equivalent accounts, at times, may exceed federally insured limits. The Company has not experienced any losses in such accounts.

Restricted cash consists of deposits collateralizing letters of credit issued by a bank in connection with the Company’s operating leases (see Note 12 “Commitments and Contingencies” for additional disclosures) and a deposit collateralizing a letter of credit issued by a bank supporting the Company’s corporate credit card. Cash, cash equivalents and restricted cash consist of the following:

	December 31, 2021		December 31, 2020

Cash and cash equivalents	$	232,694	$	297,098
Restricted cash		1,343		1,568
	$	234,037	$	298,666

Government Grants

Research and development expense is presented net of reimbursements from the California Institute for Regenerative Medicine (“CIRM”), which are recognized over the period necessary to match the reimbursement with the related costs when it is probable that the Company has complied with the CIRM conditions and will receive the reimbursement. During the years ended December 31, 2021, 2020, and 2019, the Company offset $0.1 million, $3.6 million and $1.2 million of CIRM grant funds against research and development (“R&D”) expenses (See Note 14 “CIRM Grant” for additional disclosure).

Concentrations of credit risk and off-balance sheet risk

Financial instruments that subject the Company to credit risk primarily consist of cash and cash equivalents and available-for-sale securities. The Company maintains its cash and cash equivalent balances with high-quality financial institutions and, consequently, the Company believes that such funds are subject to minimal credit risk. The Company’s marketable securities consist of U.S. Treasury securities, and Corporate, Government Municipal and Agency Bonds. The Company has adopted an investment policy that limits the amounts the Company may invest in any one type of investment and requires all investments held by the Company to be at least AA+/Aa1 rated, thereby reducing credit risk exposure.

Investments

Investments consist of investments in United States Treasury securities and Corporate, Municipal and Agency Bonds. Management determines the appropriate classification of these securities at the time they are acquired and evaluates the appropriateness of such classifications at each balance sheet date. The Company classifies its investments as available-for-sale pursuant to Financial Accounting Standards Board (“FASB”) Accounting Standard Codification (“ASC”) 320, Investments—Debt and Equity Securities. Investments are recorded at fair value, with unrealized gains and losses included as a component of accumulated other comprehensive income (loss) in stockholders’ equity and a component of total comprehensive loss in the consolidated statements of comprehensive loss, until realized. Realized gains and losses are included in investment income on a specific-identification basis. There were no realized gains or losses on investments for the years ended December 31, 2021, 2020 and 2019. For the years ended December 31, 2021, 2020 and 2019, there was $0.1 million of net unrealized losses, $0.1 million of net unrealized losses and $0.1 million of net unrealized gains on investments, respectively.

Goodwill

Business combinations are accounted for under the acquisition method. The total cost of an acquisition is allocated to the underlying identifiable net assets, based on their respective estimated fair values as of the acquisition date. Determining the fair value of assets acquired and liabilities assumed requires management’s judgment and often involves the use of significant estimates and assumptions, including assumptions with respect to future cash inflows and outflows, discount rates, asset lives and market multiples, among other items. Assets acquired and liabilities assumed are recorded at their estimated fair values. The excess of the purchase price over the estimated fair values of the net assets acquired is recorded as goodwill. Goodwill is tested for impairment annually as of December 31, or more frequently when events or changes in circumstances indicate that the asset might be impaired. Examples of such events or circumstances include, but are not limited to, a significant adverse change in legal or business climate, an adverse regulatory action or unanticipated competition. The Company has one segment and one reporting unit and as such review’s goodwill for impairment at the consolidated level.

When testing goodwill, the Company has the option to first assess qualitative factors for reporting units that carry goodwill. The qualitative assessment includes assessing the totality of relevant events and circumstances that affect the fair value or carrying value of the reporting unit. These events and circumstances include macroeconomic conditions, industry and competitive environment conditions, overall financial performance, reporting unit specific events and market considerations. The Company also considers recent valuations of the reporting unit, including the magnitude of the difference between the most recent fair value estimate and the carrying value, as well as both positive and adverse events and circumstances, and the extent to which each of the events and circumstances identified may affect the comparison of a reporting unit’s fair value with its carrying value. If the qualitative assessment results in a conclusion that it is more likely than not that the fair value of a reporting unit exceeds the carrying value, then no further testing is performed for that reporting unit.

The Company performed the qualitative assessment of its goodwill and determined that it is more likely than not that the fair value of a reporting unit exceeds the carrying value of the reporting unit. As a result, the Company has determined there was no goodwill impairment as of and for the years ended December 31, 2021, 2020 and 2019.

Property and Equipment

Property and equipment are stated at cost less accumulated depreciation. Depreciation expense is recognized using the straight-line method over the useful life of the asset. The estimated useful lives are three to fifteen years. The Company capitalizes purchases of laboratory equipment, machinery and equipment, furniture and fixtures and leasehold improvements in relation to the facility at Cranbury, New Jersey, since it has been determined these assets have alternative future uses to the Company. Expenditures for repairs and maintenance of assets are charged to expense as incurred. Upon retirement or sale, the cost and related accumulated depreciation of assets disposed of are removed from the accounts and any resulting gain or loss is included in loss from operations. Costs incurred in connection with development or purchase of internal use software and cloud computing arrangements, including in-substance software licenses, are capitalized. Amortization is computed on a straight-line basis over the estimated useful life of the asset, which is six years. Capitalized software is included in property and equipment in the consolidated balance sheets.

Impairment of Long-Lived Assets

The Company reviews long-lived assets for impairment whenever events or changes in circumstances indicate that the asset’s carrying amount may not be recoverable. The Company conducted our long-lived asset impairment analyses in accordance with ASC 360-10-15, “Impairment or Disposal of Long-Lived Assets.” ASC 360-10-15 requires us to group assets and liabilities at the lowest level for which identifiable cash flows are largely independent of the cash flows of other assets and liabilities and evaluate the asset group against the sum of the undiscounted future cash flows. If the undiscounted cash flows do not indicate the carrying amount of the asset is recoverable, an impairment charge is measured as the amount by which the carrying amount of the asset group exceeds its fair value based on discounted cash flow analysis or appraisals. There is no impairment of long-lived assets as of and for the years ended December 31, 2021, 2020 and 2019.

Fair Value Measurements

The Company is required to disclose information on all assets and liabilities reported at fair value that enables an assessment of the inputs used in determining the reported fair values. FASB ASC 820, Fair Value Measurements and Disclosures (“ASC 820”) establishes a hierarchy of inputs used when available. Observable inputs are inputs that market participants would use in pricing the asset or liability based on market data obtained from sources independent of the Company. Unobservable inputs are inputs that reflect the Company’s assumptions about the inputs that market participants would use in pricing the asset or liability and are developed based on the best information available in the circumstances. The fair value hierarchy applies only to the valuation inputs used in determining the reported fair value of the investments and is not a measure of the investment credit quality. The three levels of the fair value hierarchy are described below:

Level 1—Valuations based on unadjusted quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date.

Level 2—Valuations based on quoted prices for similar assets or liabilities in markets that are not active or for which all significant inputs are observable, either directly or indirectly.

Level 3—Valuations that require inputs that reflect the Company’s own assumptions that are both significant to the fair value measurement and unobservable.

To the extent that valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized in 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 fair value of the Company’s financial instruments, including cash and cash equivalents, restricted cash, deposits, accounts payable and accrued expenses approximate their respective carrying values due to the short-term nature of these instruments.

Research and Development Expenses

R&D costs, which include salaries and staff costs, license costs, manufacturing and development costs, clinical trial expenses, regulatory and scientific consulting fees, as well as contract research, and stock-based compensation expense, are accounted for in accordance with ASC Topic 730, Research and Development (“ASC 730”). The Company does not currently have any commercial biopharmaceutical products and does not expect to have any for several years, if at all. Accordingly, R&D costs are expensed as incurred. While certain of the Company’s R&D costs may have future benefits, the policy of expensing all R&D expenditures is predicated on the fact that the Company has no history of successful commercialization of product candidates to base any estimate of the number of future periods that would be benefited.

Foreign Currency Transactions

Certain transactions during the years ended December 31, 2021, 2020 and 2019 are denominated in Euros and British pounds. Gains and losses on foreign currency transactions were not significant for the years ended December 31, 2021, 2020 and 2019.

Treasury Stock

The Company records treasury stock at cost.

Stock-Based Compensation

The Company issues stock-based awards to employees and non-employees, generally in the form of stock options and restricted stock units. The Company accounts for its stock-based compensation awards in accordance with FASB ASC Topic 718, Compensation—Stock Compensation (“ASC 718”). ASC 718 requires all stock-based payments, including grants of stock options and restricted stock units and modifications to existing stock options, to be recognized in the consolidated statements of operations and comprehensive loss based on their fair values. The Company measures the compensation expense of employee and nonemployee services received in exchange for an award of equity instruments based on the fair value of the award on the grant date. That cost is recognized on a straight-line basis over the period during which the employee and nonemployee is required to provide service in exchange for the award. The fair value of options on the date of grant is calculated using the Black-Scholes option pricing model based on key assumptions such as stock price, expected volatility and expected term. The Company’s estimates of these assumptions are primarily based on the trading price of the Company’s stock, historical data, peer company data and judgment regarding future trends and factors.

The Company classifies stock-based compensation expense in its consolidated statement of operations in the same manner in which the award recipient’s payroll costs and services are classified or in which the award recipient’s service payments are classified. The Company recognizes compensation expense for at least the portion of awards that are vested. Forfeitures are accounted for as they occur.

NYS Life Sciences Research and Development Tax Credit

New York State allows investors and owners of emerging technology companies focused on biotechnology to claim a tax credit against their New York State Tax return for certain expenditures incurred in New York State, including applicable R&D related expenditures. The credit is recognized as research and development incentives when the eligibility and amount has been approved by New York State. During the years ended December 31, 2021, 2020 and 2019, the Company recorded research and development incentive income of $1.0 million, $0, and $0, respectively related to the NYS Life Sciences Research and Development Tax Credit.

NYC Biotechnology Tax Credit Program

New York City allowed investors and owners of emerging technology companies focused on biotechnology to claim a tax credit against the General Corporation Tax and Unincorporated Business Tax for amounts paid or incurred for certain facilities, operations, and employee training in New York City. The credit was recognized as research and development incentives when approved by New York City of the eligibility for the credit and the credit amount. During the years ended December 31, 2021, 2020 and 2019, the Company recorded research and development incentive income of $0 million, $0, and $0.3 million, respectively related to the NYC Biotechnology Program. This program was not renewed by NYC upon its expiration at the end of 2019.

Income Taxes

The Company accounts for income taxes under the asset and liability method. The Company recognizes deferred tax assets and liabilities for the future tax consequences attributable to differences between the financial statement carrying amounts of existing assets and liabilities and their respective tax bases, as well as for operating loss and tax credit carryforwards. The Company measures deferred tax assets and liabilities using enacted tax rates expected to apply to taxable income in the years in which the Company expects to recover or settle those temporary differences. The Company recognizes the effect of a change in tax rates on deferred tax assets and liabilities in the results of operations in the period that includes the enactment date. The Company reduces the measurement of a deferred tax asset, if necessary, by a valuation allowance if it is more likely than not that the Company will not realize some or all of the deferred tax asset.

The Company’s deferred tax assets relate primarily to its net operating loss carryforwards and other balance sheet differences. In accordance with ASC 740 “Income Taxes”, the Company recorded a full valuation allowance to fully offset the net deferred tax asset because it is not more likely than not that the Company will realize future benefits associated with these deferred tax assets at December 31, 2021 and 2020.

The Company accounts for uncertainty in income taxes recognized in the financial statements by applying a two-step process to determine the amount of tax benefit to be recognized. First, the tax position must be evaluated to determine the likelihood that it will be sustained upon external examination by the taxing authorities. If the tax position is deemed more-likely-than-not to be sustained, the tax position is then assessed to determine the amount of benefit to recognize in the financial statements. The amount of the benefit that may be recognized is the largest amount that has a greater than 50% likelihood of being realized upon ultimate settlement. The provision for income taxes includes the effects of any resulting tax reserves, or unrecognized tax benefits, that are considered appropriate as well as the related net interest and penalties.

Net Loss Per Share

The Company calculates net loss per share in accordance with FASB ASC 260, Earnings per Share. Basic net loss per share attributable to common shareholders is computed by dividing the net loss attributable to common shareholders by the weighted average number of common shares outstanding for the period. Diluted net loss attributable to common shareholders is computed by adjusting net loss attributable to common shareholders to reallocate undistributed earnings based on the potential impact of dilutive securities. Diluted net loss per share attributable to common shareholders is computed by dividing the diluted net loss attributable to common shareholders by the weighted average number of common shares outstanding for the period, including potential dilutive common shares. For purposes of this calculation, outstanding options are considered potential dilutive common shares.

Segment Reporting

Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chief operating decision maker, or decision-making group, in deciding how to allocate resources and in assessing performance. The Company views its operations and manages its business in one operating segment.

Comprehensive Loss

Comprehensive loss is defined as the change in equity of a business enterprise during a period from transactions, and other events and circumstances from non-owner sources and consists of net loss and changes in unrealized gains and losses on investments.

Reclassifications

Certain reclassifications have been made to the prior year financial statements in order to conform to the current year’s presentation.

Recent Accounting Pronouncements

In June 2016, the FASB issued ASU 2016-13, Financial Instruments – Credit Losses (Topic 362): Measurement of Credit Losses on Financial Statements (“ASU 2016-13”). The new standard requires that expected credit losses relating to financial assets measured on an amortized cost basis and available-for-sale debt securities be recorded through an allowance for credit losses. It also limits the amount of credit losses to be recognized for available-for-sale debt securities to the amount by which carrying value exceeds fair value and also requires the reversal of previously recognized credit losses if fair value increases. The targeted transition relief standard allows filers an option to irrevocably elect the fair value option of ASC 825-10, Financial Instruments-Overall, applied on an instrument-by-instrument basis for eligible instruments. The new standard was effective beginning January 1, 2021. The adoption of ASU 2016-13, and related updates, did not have a material impact on the Company’s consolidated financial position and results of operations.

There were no other recent accounting pronouncements that impacted the Company or are expected to have a significant effect on the consolidated financial statements.

Fair Value of Financial Instruments

12 Months Ended

Dec. 31, 2021

Fair Value of Financial Instruments [Abstract]

Fair Value of Financial Instruments

4.	Fair Value of Financial Instruments

Items measured at fair value on a recurring basis are the Company’s investments. The following table sets forth the Company’s financial instruments that were measured at fair value on a recurring basis by level within the fair value hierarchy:

	Fair Value Measurements as of December 31, 2021 Using:
	Level 1		Level 2		Level 3		Total
Assets:
Cash equivalents:
Money market mutual funds	$	179,900	$	-	$	-	$	179,900
		179,900		-		-		179,900

Investments:
United States Treasury securities		44,045		-		-		44,045
Corporate Bonds		-		96,696		-		96,696
Municipal Bonds		-		6,000		-		6,000
Agency Bonds		-		9,305		-		9,305
		44,045		112,001		-		156,046

	$	223,945	$	112,001	$	-	$	335,946

	Fair Value Measurements as of December 31, 2020 Using:
	Level 1		Level 2		Level 3		Total
Assets:
Cash equivalents:
Money market mutual funds	$	193,312	$	-	$	-	$	193,312
United States Treasury securities		62,497		-		-		62,497
Corporate Bonds		-		501		-		501
Agency Bonds		-		8,015		-		8,015
		255,809		8,516		-		264,325

Investments:
United States Treasury securities		112,328		-		-		112,328
Corporate Bonds		-		63,710		-		63,710
Municipal Bonds		-		6,000		-		6,000
Agency Bonds		-		3,583		-		3,583
		112,328		73,293		-		185,621

	$	368,137	$	81,809	$	-	$	449,946

The Company classifies its money market mutual funds and U.S. Treasury securities as Level 1 assets under the fair value hierarchy, as these assets have been valued using quoted market prices in active markets without any valuation adjustment. The Company classifies its Corporate, Municipal and Agency Bonds as Level 2 assets as these assets are not traded in an active market and have been valued through a third-party pricing service based on quoted prices for similar assets.

Property and Equipment, Net

12 Months Ended

Dec. 31, 2021

Property and Equipment, Net [Abstract]

Property and Equipment, Net

5.	Property and Equipment, Net

The Company’s property and equipment consisted of the following:

	December 31, 2021			December 31, 2020
Laboratory equipment	$	12,600		$	7,807
Machinery and equipment		10,432			9,933
Computer equipment		218			218
Furniture and fixtures		1,963			1,880
Leasehold improvements		407			29
Internal use software		1,902			1,385
		27,522			21,252
Less: accumulated depreciation and amortization		(5,223	)		(2,046	)
	$	22,299		$	19,206

Depreciation and amortization during the years ended December 31, 2021, 2020, and 2019 was $3.2 million, $1.1 million and $0.4 million, respectively.

Accounts Payable and Accrued Expenses

12 Months Ended

Dec. 31, 2021

Accounts Payable and Accrued Expenses [Abstract]

Accounts Payable and Accrued Expenses

6.	Accounts Payable and Accrued Expenses

At December 31, 2021 and 2020, the Company’s accounts payable and accrued expenses consisted of the following:

	December 31, 2021		December 31, 2020
Research and development	$	12,082	$	14,962
Property and equipment		725		1,756
Employee compensation		4,533		4,875
Accrued interest		-		1,122
Government grant payable		597		590
Professional fees		1,196		1,332
Other		482		835
	$	19,615	$	25,472

Convertible Notes Payable

12 Months Ended

Dec. 31, 2021

Convertible Notes Payable [Abstract]

Convertible Notes Payable

7.	Convertible Notes Payable

2021 Convertible Notes

On January 4, 2018, in connection with its reverse merger with Inotek Pharmaceuticals, Corporation (“Inotek”), the Company assumed the obligations of Inotek under its outstanding convertible notes, with an aggregate original principal amount of $52.0 million, (the “2021 Convertible Notes”). The 2021 Convertible Notes were issued in 2016 and were initially scheduled to mature on August 1, 2021 (the “Maturity Date”). The 2021 Convertible Notes were unsecured and accrued interest at a rate of 5.75% per annum and interest was payable semi-annually on February 1 and August 1 of each year. Each holder of the 2021 Convertible Notes (“Holder”) had the option until the close of business on the second business day immediately preceding the Maturity Date to convert all, or any portion, of the 2021 Convertible Notes held by it at a conversion rate of 31.1876 shares of the Company’s common stock per $1.00 principal amount of 2021 Convertible Notes (the “Conversion Rate”) which is $32.08 per share.

On August 2, 2021, holders of the 2021 Convertible Notes converted the $5.15 million remaining balance of the 2021 Convertible Notes into 160,614 shares of the Company’s common. As of December 31, 2021, none of the 2021 Convertible Notes were outstanding.

2022 Convertible Notes

On February 20, 2020, and June 5, 2020, the Company entered into separate, privately negotiated exchange agreements (the “Exchange Agreements”) with certain holders of the 2021 Convertible Notes. Pursuant to the Exchange Agreements, on February 20, 2020, the Company exchanged approximately $39.35 million aggregate principal amount of the 2021 Convertible Notes for (a) approximately $39.35 million aggregate principal amount of 6.25% Convertible Senior Notes due August 2022 (the “2022 Convertible Notes”). Also pursuant to the Exchange Agreements, on June 12, 2020, the Company exchanged $7.5 million aggregate principal amount of the 2021 Convertible Notes for (a) $7.5 million aggregate principal amount of its newly issued 6.25% Convertible Senior Notes due 2022.

The conversion rate for the 2022 Convertible Notes was initially 31.1876 shares of the Company’s common stock per $1.00 principal amount of 2022 Convertible Notes, which was equivalent to an initial conversion price of approximately $32.06 per share of common stock. The Company may have redeemed for cash all or any portion of the 2022 Convertible Notes, at its option, if the last reported sale price of its common stock was equal to or greater than 130% of the conversion price then in effect for at least 20 trading days during any 30 consecutive trading day period.

In December 2020, $8.5 million principal amount, representing a carrying value of $7.6 million of the 2022 Convertible Notes was converted into 298,562 shares of the Company’s common stock.

On April 26, 2021, the Company called for the redemption of the remaining $38.4 million principal amount of the 2022 Convertible Notes as the Company’s stock price traded above the Conversion Rate for at least 20 trading days during a 30-day consecutive trading period. On April 26, 2021, the Company redeemed in full the 2022 Convertible Notes prior to the redemption date. Holders of approximately $38.4 million remaining principal amount of the 2022 Convertible Notes converted such notes in accordance with the terms of the Exchange Agreements into approximately 1.3 million shares of the Company’s common stock and cash in lieu of fractional shares. In accordance with ASC 470-Debt, the settlement of the 2022 Convertible Notes is accounted for as a conversion since the 2022 Convertible Notes did not include a beneficial conversion feature and the carrying amount of the 2022 Convertible Notes.

The table below summarizes the carrying value of the 2021 and 2022 Convertible Notes as of December 31, 2021 and 2020:

	2021 Notes					2022 Notes
	December 31, 2021		December 31, 2020			December 31, 2021		December 31, 2020

Principal amount	$	-	$	5,150		$	-	$	38,350
Discount		-		(275	)		-		(3,284	)
Carrying value	$	-	$	4,875		$	-	$	35,066

Accretion of the 2021 Convertible Notes discount was $0.3 million, $1.3 million, and $3.6 million for the years ended December 31, 2021, 2020, and 2019, respectively. Accretion of the 2022 Convertible Notes discount was $0.5 million and $1.5 million for the years ended December 31, 2021 and December 31, 2020.

- Definition

The entire disclosure for information about short-term and long-term debt arrangements, which includes amounts of borrowings under each line of credit, note payable, commercial paper issue, bonds indenture, debenture issue, own-share lending arrangements and any other contractual agreement to repay funds, and about the underlying arrangements, rationale for a classification as long-term, including repayment terms, interest rates, collateral provided, restrictions on use of assets and activities, whether or not in compliance with debt covenants, and other matters important to users of the financial statements, such as the effects of refinancing and noncompliance with debt covenants.

+ References

Reference 1: http://fasb.org/us-gaap/role/ref/legacyRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
-URI http://asc.fasb.org/topic&trid=2208564

Reference 2: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
-SubTopic 20
-Section 50
-Paragraph 1B
-Subparagraph (h)
-URI http://asc.fasb.org/extlink&oid=123466505&loc=SL123495323-112611

Reference 3: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
-SubTopic 20
-Section 50
-Paragraph 1C
-Subparagraph (c)
-URI http://asc.fasb.org/extlink&oid=123466505&loc=SL123495334-112611

Reference 4: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
-Topic 470
-SubTopic 20
-Section 50
-Paragraph 1I
-Subparagraph (a)
-URI http://asc.fasb.org/extlink&oid=123466505&loc=SL123495371-112611

Reference 5: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
-SubTopic 20
-Section 50
-Paragraph 1I
-Subparagraph (d)
-URI http://asc.fasb.org/extlink&oid=123466505&loc=SL123495371-112611

Reference 6: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 235
-SubTopic 10
-Section S99
-Paragraph 1
-Subparagraph (SX 210.4-08(c))
-URI http://asc.fasb.org/extlink&oid=120395691&loc=d3e23780-122690

Reference 7: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
-SubTopic 20
-Section 50
-Paragraph 1B
-Subparagraph (g)
-URI http://asc.fasb.org/extlink&oid=123466505&loc=SL123495323-112611

Reference 8: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
-SubTopic 20
-Section 50
-Paragraph 1C
-Subparagraph (b)
-URI http://asc.fasb.org/extlink&oid=123466505&loc=SL123495334-112611

Reference 9: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
-SubTopic 20
-Section 50
-Paragraph 1C
-Subparagraph (a)
-URI http://asc.fasb.org/extlink&oid=123466505&loc=SL123495334-112611

Reference 10: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
-SubTopic 20
-Section 50
-Paragraph 1E
-Subparagraph (b)
-URI http://asc.fasb.org/extlink&oid=123466505&loc=SL123495348-112611

Reference 11: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 470
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+ Details

Shareholders' Equity

12 Months Ended

Dec. 31, 2021

Stockholders' Equity [Abstract]

Shareholders' Equity

8.	Stockholders’ Equity

Common Stock

The Company is currently authorized to issue up to 120,000,000 shares of $0.01 par value common stock. All issued shares of common stock are entitled to vote on a 1 share/1 vote basis.

Second Amended and Restated 2014 Stock Option and Incentive Plan

In March 2018, Rocket’s Board of Directors approved the Second Amended and Restated 2014 Stock Option and Incentive Plan (the “Revised 2014 Plan”) which was approved by the Company’s shareholders at the Annual Meeting held on June 25, 2018.

Treasury Stock

During fiscal 2019, the Company recorded treasury stock of $0.1 million for shares withheld to pay the payroll tax liability of an option exercise which was remitted in January 2020. There was no treasury stock as of December 31, 2021 and 2020.

Private Placement

Public Offerings

On December 14, 2020, the Company completed a public offering of 5,339,286 shares of common stock, which includes the full exercise of the underwriters’ option to purchase an additional 696,428 shares of our common stock, at a public offering price of $56.00 per share. The gross proceeds to Rocket from the public offering were approximately $299.0 million, net of $18.2 million of offering costs, commissions, legal and other expenses for net proceeds from the offering of $280.8 million.

- Definition

The entire disclosure for shareholders' equity comprised of portions attributable to the parent entity and noncontrolling interest, including other comprehensive income. Includes, but is not limited to, balances of common stock, preferred stock, additional paid-in capital, other capital and retained earnings, accumulated balance for each classification of other comprehensive income and amount of comprehensive income.

+ References

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
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Reference 2: http://www.xbrl.org/2003/role/disclosureRef
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Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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+ Details

Stock-Based Awards

12 Months Ended

Dec. 31, 2021

Stock-Based Awards [Abstract]

Stock-Based Awards

9.	Stock-Based Awards

Stock Option Valuation

The weighted average assumptions that the Company used in the Black-Scholes pricing model to determine the fair value of the stock options granted to employees, non-employees and directors were as follows:

	Years Ended December 31,
	2021			2020			2019

Risk-free interest rate		0.83	%		1.00	%		2.26	%
Expected term (in years)		5.84			5.84			5.81
Expected volatility		69.27	%		76.98	%		75.71	%
Expected dividend yield		0.00	%		0.00	%		0.00	%
Exercise price	$	51.20		$	22.96		$	14.47
Fair value of common stock	$	51.20		$	22.96		$	14.47

The following table summarizes stock option activity for the years ended December 31, 2021 and 2020, under the Revised 2014 Plan:

	Number of Shares			Weighted Average Exercise Price		Weighted Average Contractual Term (Years)		Aggregate Intrinsic Value

Outstanding as of December 31, 2019		9,763,541		$	5.95		6.96	$	164,021
Granted		2,193,546			22.96		8.97
Exercised		(586,857	)		4.82				13,494
Cancelled		(319,299	)		15.82
Outstanding as of December 31, 2020		11,050,931		$	9.10		6.55	$	504,079
Granted		1,671,759			51.20		8.58
Exercised		(1,209,960	)		9.32				54,487
Cancelled		(368,969	)		35.87
Outstanding as of December 31, 2021		11,143,761		$	14.51		5.95	$	128,817

Options vested and exercisable as of December 31, 2021		8,692,898		$	7.31		5.11	$	127,571
Options unvested as of December 31, 2021		2,450,863		$	40.01		8.93

The weighted average grant-date fair value per share of stock options granted during the years ended December 31, 2021, 2020 and 2019 was $31.07, $15.10, and $9.58, respectively.

The total fair value of options vested during the years ended December 31, 2021, 2020 and 2019 was $22.6 million, $15.6 million and $63.1 million, respectively.

Stock-Based Compensation

Stock-based compensation expense recognized by award type is as follows:

	Years Ended December 31,
	2021		2020		2019

Stock options	$	28,811	$	18,527	$	13,371
Restricted stock units		426		40		-
Total share based compensation expense	$	29,237	$	18,567	$	13,371

Stock-based compensation expense by classification included within the consolidated statements of operations and comprehensive loss was as follows:

	Years Ended December 31,
	2021		2020		2019

Research and development	$	11,954	$	7,355	$	6,153
General and administrative		17,283		11,212		7,218
Total share based compensation expense	$	29,237	$	18,567	$	13,371

As of December 31, 2021 and 2020, the Company had an aggregate of $46.3 million and $30.4 million of unrecognized stock-based compensation expense, which is expected to be recognized over the weighted average period of 1.8 years and 2.1 years, respectively.

Restricted Stock Units (“RSU”)

The following table summarizes our RSU activity for the years ended December 31, 2021 and 2020:

	Number of Shares		Weighted Average Grant Date Fair Value

Unvested as of December 31, 2019		-	$	-
Granted		20,000		25.06
Exercised		-
Forfeited		-
Unvested as of December 31, 2020		20,000		25.06
Granted		3,500		62.32
Exercised		-
Forfeited		-
Unvested as of December 31, 2021		23,500		30.61

The intrinsic value of RSU’s vested during the years ended December 31, 2021 and 2020 was $0.4 million and $0.5 million.

As of December 31, 2021, there was approximately $0.5 million of unrecognized compensation cost related to RSU’s granted. This amount is expected to be recognized over a weighted average period of 2.8 years.

Warrants

A summary of the warrants outstanding at December 31, 2021 is as follows:

Price	Outstanding		Grant Date	Expiration Date
24.42		7,051	June 28, 2013	June 28, 2023
57.11		603,386	December 21, 2020	December 21, 2030
33.63		301,291	August 9, 2021	August 9, 2031
22.51		153,155	December 17, 2021	December 17, 2031
22.51		153,155	December 17, 2021	December 17, 2031
Total		1,218,038

The following table below is the summary of changes in warrants to purchase common stock for the years ended December 31, 2021 and 2020:

	Number of Warrant Shares Outstanding and Exercisable			Exercise Price per Share

Balance as of December 31, 2019		14,102		$	24.42
Granted		603,386		$	57.11
Exercised		(7,051	)	$	-
Balance as of December 31, 2020		610,437
Granted August 2021		301,291		$	33.63
Granted December 2021		306,310		$	22.51
Exercised		-		$	-
Balance as of December 31, 2021		1,218,038

The Company issued warrants to a related party during the years ended December 31, 2021 and 2020 and incurred a non-cash R&D expense of $12.8 million and $26.6 million respectively (see Note 15- Related Party Transactions). During the year ended December 31, 2020, 7,051 of warrants were exercised into 1,601 shares of common stock.

The fair value of the 2021 and 2020 warrants was calculated using the Black-Scholes fair value pricing model with the following inputs:

	Years Ended December 31,
	2021			2020

Risk-free interest rate		1.37	%		0.95	%
Expected term (in years)		10.00			10.00
Expected volatility		70.25	%		74.20	%
Expected dividend yield		0.00	%		0.00	%
Exercise price	$	28.07		$	57.11
Fair value of common stock	$	28.07		$	57.11

Net Loss Per Share

12 Months Ended

Dec. 31, 2021

Net Loss Per Share [Abstract]

Net Loss Per Share

10.	Net Loss Per Share

Basic and diluted net loss per share attributable to common stockholders was calculated as follows:

	For the Years Ended December 31,
	2021			2020			2019

Numerator:
Net loss attributable to common stockholders	$	(169,069	)	$	(139,700	)	$	(77,270	)
Denominator:
Weighted-average common shares outstanding - basic and diluted		63,235,417			55,380,740			49,010,358
Net loss per share attributable to common stockholders - basic and diluted	$	(2.67	)	$	(2.52	)	$	(1.58	)

The Company excluded the following potential shares of common stock, presented based on amounts outstanding at each period end, from the computation of diluted net loss per share attributable to common stockholders for the periods indicated because including them would have had an anti-dilutive effect:

	For the Years Ended December 31,
	2021		2020		2019
Shares issuable upon conversion of the 2021 Convertible Notes		-		160,536		1,620,948
Shares issuable upon conversion of the 2022 Convertible Notes		-		1,195,449		-
Warrants exercisable for common shares		1,218,038		610,437		14,102
Restricted stock units exercisable for common shares		23,500		-		-
Options to purchase common shares		11,143,761		11,050,931		9,608,537
		12,385,299		13,017,353		11,243,587

Income Taxes

12 Months Ended

Dec. 31, 2021

Income Taxes [Abstract]

Income Taxes

11.	Income Taxes

No provision for federal or state income taxes was recorded during the years ended December 31, 2021, 2020 and 2019, as the Company incurred operating losses and maintains a full valuation allowance against its net deferred tax assets.

A reconciliation of income tax benefit computed at the statutory federal income tax rate to income taxes as reflected in the financial statements is as follows:

	For the years ended December 31,
	2021			2020			2019
U.S. federal tax at statutory rate		21.0	%		21.0	%		21.0	%
Foreign rate differential		(13.0	)%		0.0	%		0.0	%
Change in state tax apportionment		0.1	%		0.1	%		(17.4	)%
Stock compensation		1.5	%		0.9	%		(0.7	)%
Transfer pricing adjustments		(22.8	)%		0.0	%		0.0	%
Valuation allowance		4.6	%		(35.0	)%		(5.1	)%
Tax credits		8.7	%		13.5	%		0.0	%
Other		(0.2	)%		(0.5	)%		2.1	%
Effective tax rate		0	%		0	%		0	%

The significant components of the Company’s deferred income tax assets and liabilities after applying the enacted corporate tax rates are as follows:

	At December 31,
	2021			2020			2019
Deferred income tax assets (liabilities)
R&D Credits	$	35,766		$	42,613		$	18,471
Net operating losses ("NOL") and credit carryforwards		26,789			21,359			2,517
Capitalized research and development costs		19,753			23,179			27,652
Stock-based compensation		11,552			7,406			3,896
Debt discount		-			(748	)		(1,460	)
Warrants		8,382			5,585			-
Other		(8,881	)		1,189			643
Valuation allowance		(93,361	)		(100,583	)		(51,719	)
Net deferred income tax asset	$	-		$	-		$	-

As of December 31, 2021, the Company had federal and state net operating loss carryforwards of approximately $118.2 million and $30.9 million, respectively, which begin to expire in 2026. Additionally, $85.7 million of the Federal NOLs can be carried forward indefinitely. The Company has federal R&D credits of $35.8 million which will begin to expire in 2038.

As required by ASC 740, Income Taxes, management of Rocket has evaluated the positive and negative evidence bearing upon the realizability of its deferred tax assets, which are comprised principally of NOL carryforwards and capitalized research and development costs. As a result of the fact that Rocket has incurred tax losses from inception, management has determined that it is more likely than not that Rocket will not recognize the benefits of federal and state net deferred tax assets and, as a result, a full valuation allowance has been established against its net deferred tax assets as of December 31, 2021, 2020 and 2019. Rocket has offset certain deferred tax liabilities with deferred tax assets that are expected to generate offsetting deductions within the same period. During the years ended December 31, 2021 and 2020, the valuation allowance decreased by $7.2 million and increased by $48.9 million, respectively. Realization of deferred tax assets is dependent upon the generation of future taxable income.

Under Internal Revenue Code Section 382, if a corporation undergoes an “ownership change,” the corporation’s ability to use its pre-change NOL carryforwards and other pre-change tax attributes to offset its post-change income may be limited. The Company has completed a study to assess whether an ownership change has occurred or whether there have been multiple ownership changes since the Company became a “loss corporation” as defined in Section 382. The Company experienced multiple ownership changes occurring in 2005, 2007, 2015, and 2018. The ownership change has and will continue to subject our pre-ownership change net operating loss carryforwards to an annual limitation, which will significantly restrict our ability to use them to offset taxable income in periods following the ownership change. In general, the annual use limitation equals the aggregate value of our stock at the time of the ownership change multiplied by a specified tax-exempt interest rate. As a result of the ownership change, the Company is limited to an approximate $1.7 million annual limitation on our ability to utilize our pre-merger NOL’s and R&D Credits. Due to this limitation, approximately $91.2 million of the $127.1 million pre-merger Federal NOL will expire unutilized as the cumulative limitation amount over a 20-year carryforward period is $35.8 million. Additionally, $4.9 million of Federal R&D Credits will expire unutilized. As a result, the Company has reduced its deferred tax assets related to the Federal NOL and Federal R&D Credits by an aggregate of $4.9 million which is offset by the corresponding decrease in the valuation allowance.

The company evaluated intercompany transfer pricing agreements. Based on a review of the 2018, 2019 and 2020 tax years, the Company determined that a markup of 10% should have been applied to R&D expenses paid for on behalf of Rocket Pharmaceuticals, Ltd by Rocket Pharmaceuticals, Inc. The net impact was to reduce the Company’s net operating losses for 2018, 2019 and 2020. No income tax expense was recorded as a result of these adjustments.

The calculation of our tax liabilities involves dealing with uncertainties in the application of complex tax laws and regulations for both federal taxes and the many states in which Rocket operates or does business in. ASC 740 states that a tax benefit from an uncertain tax position may be recognized when it is more likely than not that the position will be sustained upon examination, including resolutions of any related appeals or litigation processes, on the basis of the technical merits.

The Company records uncertain tax positions as liabilities in accordance with ASC 740 and adjusts these liabilities when our judgment changes as a result of the evaluation of new information not previously available. Because of the complexity of some of these uncertainties, the ultimate resolution may result in a payment that is materially different from our current estimate of the unrecognized tax benefit liabilities. These differences will be reflected as increases or decreases to income tax expense in the period in which new information is available. As of December 31, 2021, 2020 and 2019, the Company has not recorded any uncertain tax positions in its financial statements.

The Company recognizes interest and penalties related to unrecognized tax benefits on the income tax expense line in the accompanying consolidated statement of operations. As of December 31, 2021, 2020 and 2019, no accrued interest or penalties are included on the related tax liability line in the consolidated balance sheets. The Company was audited by New York City for the 2017 tax year and the audit was completed with no change to the 2017 New York City tax return.

- Definition

The entire disclosure for income taxes. Disclosures may include net deferred tax liability or asset recognized in an enterprise's statement of financial position, net change during the year in the total valuation allowance, approximate tax effect of each type of temporary difference and carryforward that gives rise to a significant portion of deferred tax liabilities and deferred tax assets, utilization of a tax carryback, and tax uncertainties information.

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+ Details

Commitments and Contingencies

12 Months Ended

Dec. 31, 2021

Commitments and Contingencies [Abstract]

Commitments and Contingencies

12.	Commitments and Contingencies

The Company determines if an arrangement is a lease at inception. Operating and finance leases are presented in the Company’s consolidated balance sheet as right-of-use assets from leases, current lease liabilities and long-term lease liabilities. Certain of the Company’s lease agreements contain renewal options; however, the Company does not recognize right-of-use assets or lease liabilities for renewal periods unless it is determined that the Company is reasonably certain of renewing the lease at inception or when a triggering event occurs. As the Company’s leases do not provide an implicit rate, the Company estimated the incremental borrowing rate in calculating the present value of the lease payments using an estimate of the Company’s collateralized borrowing rate for debt with a similar term. The Company has utilized its incremental borrowing rate based on the long-term borrowing costs of comparable companies in the biotechnology industry. Since the Company elected to account for each lease component and its associated non-lease components as a single combined lease component, all contract consideration was allocated to the combined lease component. Some of the Company’s lease agreements contain rent escalation clauses (including index-based escalations). For operating leases, the Company recognizes the minimum rental expense on a straight-line basis based on the fixed components of a lease arrangement. The Company will amortize this expense over the term of the lease beginning with the lease commencement date. Variable lease components represent amounts that are not fixed in nature and are not tied to an index or rate and are recognized as incurred.

Finance Lease

The Company has a lease for a facility in Cranbury, New Jersey, consisting of 103,720 square feet of space including areas for offices, process development, research and development laboratories and 50,000 square feet dedicated to AAV Current Good Manufacturing Practice (“cGMP”) manufacturing facilities to support the Company’s pipeline. A smaller area within this facility was originally leased in August 2018, and the lease was amended in June 2019 to include the full building (such lease, as amended, the “NJ Lease Agreement”). The NJ Lease Agreement has a 15-year term from September 1, 2019, with an option to renew for two consecutive five-year renewal terms.

The Company determined the lease commencement date was reached on March 15, 2020, when the construction of all landlord owned improvements had been substantially completed and when the Company began including its leasehold improvements on the balance sheet and move equipment into the space. Upon commencement of the NJ Lease Agreement, the Company recognized total right-of-use assets of $47.7 million, with a corresponding lease liability of $20.2 million. The Company reclassified $26.5 million of construction costs in progress and $1.1 million of prepaid rent as part of the right-of-use asset upon the lease commencement date of March 15, 2020. During the year ended December 31, 2021, the Company reclassified an additional $0.1 million of construction costs in progress bringing the aggregate reclassification through December 31, 2021, of $32.1 million of construction costs in progress as part of the right of use asset.

Estimated rent payments for the NJ Lease Agreement are $1.2 million per annum, payable in monthly installments, depending upon the nature of the leased space, and subject to annual base rent increases of 3%. The total commitment under the lease is estimated to be approximately $29.3 million over the 15-year term of the lease. The Company paid a cash security deposit of $0.3 million to the landlord in connection with the NJ Lease Agreement which has been reflected in deposits in the consolidated balance sheets as of December 31, 2021 and 2020.

The total restricted cash balance for the Company’s operating and finance leases at December 31, 2021 and 2020 was $0.8 million and $1.1 million, respectively.

Operating Leases

On March 31, 2016, the Company entered into a lease agreement for its office and laboratory space at the Alexandria Center for Life Sciences in New York, New York with an initial term ending on July 31, 2022 (the “NY Lease Agreement”). Effective May 31, 2020, the Company terminated the NY Lease Agreement and recorded a loss on lease termination of $76 for the year ended December 31, 2020.

On June 7, 2018, the Company entered into a three-year lease agreement for office space in the Empire State Building in New York, NY (the “ESB Lease Agreement”). In connection with the ESB Lease Agreement, the Company established an irrevocable standby letter of credit (the “Empire LOC”) for $0.9 million. On March 26, 2021, the Company entered in Amendment No. 1 to the ESB Lease Agreement (“ESB Lease Amendment”) that extended the term of the lease agreement to June 30, 2024, reduced the rent payments going forward, and reduced the Empire LOC to $0.8 million. The Empire LOC serves as the Company’s security deposit on the lease in which the landlord is the beneficiary and expires August 29, 2024. The Company has accounted for the ESB Lease Amendment as a modification to the ESB Lease Agreement and remeasured the lease liability and adjusted the operating lease right of use asset by $1.1 million.

The Company has a certificate of deposit of $0.8 million and $0.9 million with a bank as collateral for the Empire LOC which is classified as part of restricted cash in the consolidated balance sheets as of December 31, 2021 and December 31, 2020, respectively.

On January 4, 2018, in connection with the Reverse Merger, the Company assumed an operating lease for Inotek’s former headquarters in Lexington, Massachusetts, with a term ending in February 2023. In July 2018, the Company signed an agreement to sublease a portion of the Lexington, Massachusetts space and in September 2018, the Company signed an agreement to sublease the remaining portion of the Lexington, Massachusetts space. Rental income received under the sublease agreement totaled $0.4 million, $0.4 million, and $0.3 million for the years ended December 31, 2021, 2020 and 2019, respectively. These amounts are netted against rent expense in the consolidated statement of operations for the years ended December 31, 2021, 2020 and 2019.

Rent expense was $1.2 million, $1.1 million, and $0.9 million for the years ended December 31, 2021, 2020 and 2019, respectively.

Lease cost	December 31, 2021
Operating lease cost	$	645
Finance lease cost
Amortization of right of use assets		2,140
Interest on lease liablities		1,845
Total lease cost	$	4,630

The following table summarizes the maturity of the Company’s lease liabilities on an undiscounted cash flow basis and a reconciliation to the operating lease liabilities recognized on the Company’s balance sheet as of December 31, 2021:

Maturity of operating lease liabilities	December 31, 2021
2022		917
2023		548
2024		269
2025		64
2026		54
Total lease payments	$	1,852
Less: interest		(84	)
Total operating lease liabilities	$	1,768

Maturity of finance lease liability	December 31, 2021
2022		1,689
2023		1,736
2024		1,791
2025		1,856
2026		1,912
Thereafter		45,000
Total lease payments	$	53,984
Less: interest		(33,151	)
Total finance lease liability	$	20,833

Leases	December 31, 2021

Operating right-of-use assets	$	1,569

Operating current lease liabilities		863
Operating noncurrent lease liabilities		905
Total operating lease liabilities	$	1,768

Finance right-of-use assets	$	48,480

Finance current lease liability		1,689
Finance noncurrent lease liability		19,144
Total finance lease liability	$	20,833

Other information
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows from operating leases	$	655
Cash flows from finance lease	$	1,644
Weighted-average remaining lease term - operating leases	2.5 years
Weighted-average remaining lease term - finance lease	22.7 years
Weighted-average discount rate - operating leases		4.46	%
Weighted-average discount rate - finance lease		8.96	%

Litigation

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. Although the results of litigation and claims cannot be predicted with certainty, the Company does not believe it is party to any other claim or litigation the outcome of which, if determined adversely to the Company, would individually or in the aggregate be reasonably expected to have a material adverse effect on its business. Regardless of the outcome, litigation can have an adverse impact on the Company because of defense and settlement costs, diversion of management resources and other factors.

Indemnification Arrangements

Pursuant to its bylaws and as permitted under Delaware law, the Company has indemnification obligations to directors, officers, employees or agents of the Company or anyone serving in these capacities. The maximum potential amount of future payments the Company could be required to pay is unlimited. The Company has insurance that reduces its monetary exposure and would enable it to recover a portion of any future amounts paid. As a result, the Company believes that the estimated fair value of these indemnification commitments is minimal.

Throughout the normal course of business, the Company has agreements with vendors that provide goods and services required by the Company to run its business. In some instances, vendor agreements include language that requires the Company to indemnify the vendor from certain damages caused by the Company’s use of the vendor’s goods and/or services. The Company has insurance that would allow it to recover a portion of any future amounts that could arise from these indemnifications. As a result, the Company believes that the estimated fair value of these indemnification commitments is minimal.

Agreements Related to Intellectual Property

12 Months Ended

Dec. 31, 2021

13.	Agreements Related to Intellectual Property

The Company, directly and through its subsidiary Spacecraft Seven, LLC, has various license and research and collaboration arrangements. The transactions principally resulted in the acquisition of rights to intellectual property which is in the preclinical phase and has not been tested for safety or feasibility. In all cases, the Company did not acquire tangible assets, processes, protocols or operating systems. The Company expenses the acquired intellectual property rights as of the acquisition date on the basis that the cost of intangible assets purchased from others for use in research and development activities, has no alternative future uses.

License Agreement for Danon Disease with UCSD

In February 2017, the Company entered into a License Agreement with The Regents of the University of California, represented by its San Diego campus (“UCSD”), under which UCSD granted us an exclusive, sublicensable, worldwide license to certain intellectual property rights for the treatment of lysosomal storage diseases, including Danon disease. In exchange for the license, the Company became obligated to make an up-front payment, certain clinical and commercial milestone payments, royalty payments (on net sales of products covered by a valid claim within the licensed intellectual property), maintenance fees and sublicense revenue payments. The upfront license fee of $0.05 million was expensed as research and development costs. The Company is obligated to make aggregate milestone payments of up to $1.5 million to UCSD upon the achievement of specified development and regulatory milestones for the treatment of Danon disease. A reduced schedule of milestone payments applies to achieving the same milestones for additional indications. With respect to any commercialized products covered by the agreement, the Company is obligated to pay a low single digit percentage royalty on net sales, subject to specified adjustments. If it enters into a sublicense agreement with a sublicensee, it will be obligated to pay a portion of any consideration received from such sublicensees in specified circumstances. The Company is also subject to certain diligence milestones for development of a product using the intellectual property licensed from UCSD under this agreement. The term of the license agreement with UCSD is through the expiration of the licensed patents, some of which are still in the pending application phase.

REGENXBIO, Inc. License

On November 19, 2018, the Company entered into a license agreement with REGENXBIO Inc. (“RGNX”), pursuant to which the Company obtained an exclusive license for all U.S. patents and patent applications related to RGNX’s NAV AAV-9 vector for the treatment of Danon disease in humans by in vivo gene therapy using AAV-9 to deliver any known LAMP2 transgene isoforms and all possible combinations of LAMP2 transgene isoforms (the “Field”), as well as an exclusive option to license (the “Option Right”) all U.S. patents and patent applications for two additional NAV AAV vectors in the Field (each, a “Licensed Patent” and collectively, the “Licensed Patents”).

Under the terms of the license agreement, the Company is obligated to use commercially reasonable efforts to develop, commercialize, market, promote and sell products incorporating the Licensed Patents (“Licensed Products”). Unless the license agreement is terminated earlier as provided below, the license from RGNX expires on a country-by-country, Licensed Product-by-Licensed Product basis until the later of the expiration date of the last to expire of the last valid claim of the applicable Licensed Patent or ten years after the first commercial sale of a Licensed Product in such country. The license agreement provides that RGNX may terminate the agreement upon a material breach by the Company if the Company does not cure such breach within a specified notice period if the Company commences a challenge against RGNX or certain of its licensors to declare or render invalid or unenforceable the licensed patents or upon the Company’s bankruptcy or insolvency. The Company may terminate the agreement in its entirety or terminate one or more of the licensed vectors at any time upon six months’ notice. The Company’s Option Right expires four years from the date of the license agreement.

In consideration for the rights granted to the Company under the license agreement, the Company made an upfront payment to RGNX of $7.0 million included as research and development expenses. A fee of $2.0 million per additional vector would be due if the Company exercises its Option Right to purchase additional vectors. The license agreement provides for royalties payable to RGNX in the high-single digits to low-teens on net sales levels of Licensed Products during the royalty term. If successful, the Company will be required to make milestone payments to RGNX of up to $13.0 million for each Licensed Product upon the achievement of specified clinical development and regulatory milestones in the U.S. and European Union. In addition, the Company shall pay RGNX 20% of the payment fees received from a priority review voucher issued in connection with or otherwise related to a Licensed Product. These royalty obligations are subject to specified reductions if additional licenses from third parties are required. The Company must also pay RGNX a portion of all non-royalty sublicense income (if any) received from sublicensees. The Company paid a $1.0 million license fee payment under the RGNX agreement upon the dosing of the first Danon patient in 2019. There were no additional milestones achieved or related payments made during the years ended December 31, 2021 and 2020.

CIRM Grants

12 Months Ended

Dec. 31, 2021

CIRM Grant [Abstract]

CIRM Grants

14.	CIRM Grants

LAD-I CIRM Grant

On April 30, 2019, the California Institute for Regenerative Medicine (“CIRM”) awarded the Company up to $6.5 million under a CLIN2 grant award to support the clinical development of its LVV-based gene therapy for RP-L201. Proceeds from the grant will help fund clinical trial costs as well as manufactured drug product for Phase 1/2 patients enrolled at the U.S. clinical site, University of California, Los Angeles (“UCLA”) Mattel Children’s Hospital, led by principal investigator Donald Kohn, M.D., UCLA Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. In 2019, the Company received the first two grants from CIRM in the aggregate of $1.2 million which were included as an offset against R&D expenses. In 2020, the Company met additional CIRM milestones and received an additional $1.1 million milestone which was recorded as a reduction of R&D expenses in 2020. The Company received the additional milestone payments of $1.1 million and $1.0 million in January and April of 2021, respectively. As of December 31, 2021, the Company did not meet the next milestone and therefore no receivable has been recorded.

IMO CIRM Grant

On November 12, 2020, the CIRM awarded the Company up to $3.7 million under a CLIN2 grant award to support the clinical development of its LVV-based gene therapy, RP-L401, for the treatment of IMO. The Company received $1.0 million pursuant to the grant on January 4, 2021, related to the CIRM IMO award and recorded a receivable, included in prepaid and other assets in the consolidated balance sheet, and a reduction of research and development expenses of $0.9 million as of December 31, 2020. The Company recorded a reduction of research and development expense of $0.1 million for the year ended December 31, 2021. As of December 31, 2021, the Company did not meet the next milestone and no receivable has been recorded. Effective December 2021, a decision was made to no longer pursue Rocket-sponsored clinical evaluation of RP-L401; this program is to be returned to academic innovators.

Related Party Transactions

12 Months Ended

Dec. 31, 2021

Related Party Transactions

15.	Related Party Transactions

During April 2018, the Company entered into an agreement with a member of the Board of Directors for business development consulting services. Payments for the services under the agreement are $28 per quarter, and the Company may terminate the agreement with 14 days’ notice. The Company incurred expenses of $0.1 million for each of the years ended December 31, 2021, 2020 and 2019, relating to services provided under this agreement.

In September 2021, the Company entered into a consulting agreement with a member of the Board of Directors for pipeline development, new asset evaluation, and corporate strategy. In lieu of cash for services to be provided under the consulting agreement during its one-year term, the Company granted the board member options to purchase 20,000 shares of the Company’s common stock with a fair value of $0.4 million.

In October 2020, the Company entered into a consulting agreement with the spouse of one of the Company’s executive officers for information technology advisory services. In exchange for the services provided under the agreement, the Company granted 10,000 restricted stock units which vest over a three-year period.

On August 27, 2021, the Company entered into a Securities Purchase Agreement (the “Purchase Agreement”) with a fund affiliated with RTW Investments, LP, the Company’s largest shareholder (the “Purchaser”), pursuant to which it agreed to sell and issue to the Purchaser, in a private placement (the “Private Placement”), 812,516 shares of the Company’s common stock at a purchase price of $32.48 per share for aggregate net proceeds of approximately $26.4 million to the Company before deducting estimated offering expenses payable by the Company. The Private Placement closed on August 31, 2021. In addition, concurrently with the execution of the Purchase Agreement, the Company entered into a registration rights agreement with the Purchaser, pursuant to which the Company agreed, following demand by the Purchaser, to file with the Securities and Exchange Commission a Registration Statement on Form S-3 covering the resale of shares of common stock held by the Purchaser as promptly as reasonably practicable following such demand, and in any event within 60 days of such demand.

On December 21, 2020, the Company entered into a consulting agreement with a related party. Pursuant to the consulting agreement, the related party provides certain business development and asset identification consulting services to the Company. The term of the consulting agreement is three years and may be terminated with 60 days’ notice by either party. In exchange for the business development services to be provided under the agreement, the Company issued a warrant exercisable for 603,386 shares of Common Stock. Pursuant to the consulting agreement, the related party is entitled to receive additional warrants exercisable for common stock upon identification of new assets for the Company to in-license. The Company recorded a non-cash R&D expense of $26.6 million for the year ended December 31, 2020, related to the issuance of the Common Stock warrant.

On August 9, 2021, the Company issued a warrant exercisable for 301,291 shares of common stock to the same related party for business development and asset identification consulting services ("August 2021 Warrant"). The Company recorded a non-cash R&D expense of $7.6 million during year ended December 31, 2021, related to the issuance of the August 2021 warrant. On December 17, 2021, the Company issued warrants exercisable for 153,155 and 153,155 shares of common stock, respectively to the same related party for business development and asset identification consulting services (“December 2021 Warrants”). The Company recorded a non-cash R&D expense of $5.2 million during year ended December 31, 2021, related to the issuance of the December 2021 warrant. Total non-cash R&D expense of $12.8 million during the year ended December 31, 2021, related to the issuance of the August 2021 and December 2021 warrants.

401(k) Savings Plan

12 Months Ended

Dec. 31, 2021

401(k) Savings Plan [Abstract]

401(k) Savings Plan

16.	401(k) Savings Plan

The Company has a defined contribution savings plan (the “Plan”) under Section 401(k) of the Internal Revenue Code of 1986. This Plan covers substantially all employees who meet minimum age and service requirements and allows participants to defer a portion of their annual compensation on a pre-tax basis. Company contributions to the Plan may be made at the discretion of the Company’s Board of Directors. The Company has elected to match 4% of employee contributions to the Plan, subject to certain limitations. The Company’s matching contribution for the years ended December 31, 2021, 2020, and 2019 was $0.6 million, $0.3 million, and $0.2 million, respectively.

Summary of Significant Accounting Policies (Policies)

12 Months Ended

Dec. 31, 2021

Summary of Significant Accounting Policies [Abstract]

Principles of Consolidation

Use of Estimates

Cash, Cash Equivalents and Restricted Cash

	December 31, 2021		December 31, 2020

Cash and cash equivalents	$	232,694	$	297,098
Restricted cash		1,343		1,568
	$	234,037	$	298,666

Government Grants

Concentrations of Credit Risk and Off-balance Sheet Risk

Concentrations of credit risk and off-balance sheet risk

Investments

Goodwill

Property and Equipment

Impairment of Long-Lived Assets

Fair Value Measurements

Level 1—Valuations based on unadjusted quoted prices in active markets for identical assets or liabilities that the Company has the ability to access at the measurement date.

Level 2—Valuations based on quoted prices for similar assets or liabilities in markets that are not active or for which all significant inputs are observable, either directly or indirectly.

Level 3—Valuations that require inputs that reflect the Company’s own assumptions that are both significant to the fair value measurement and unobservable.

Research and Development Expenses

Foreign Currency Transactions

Treasury Stock

The Company records treasury stock at cost.

Stock-Based Compensation

Tax Credits and Income Taxes

NYS Life Sciences Research and Development Tax Credit

NYC Biotechnology Tax Credit Program

Income Taxes

Reclassifications

Certain reclassifications have been made to the prior year financial statements in order to conform to the current year’s presentation.

Recent Accounting Pronouncements

There were no other recent accounting pronouncements that impacted the Company or are expected to have a significant effect on the consolidated financial statements.

Summary of Significant Accounting Policies (Tables)

12 Months Ended

Dec. 31, 2021

Summary of Significant Accounting Policies [Abstract]

Cash, Cash Equivalents and Restricted Cash

	December 31, 2021		December 31, 2020

Cash and cash equivalents	$	232,694	$	297,098
Restricted cash		1,343		1,568
	$	234,037	$	298,666

Fair Value of Financial Instruments (Tables)

12 Months Ended

Dec. 31, 2021

Fair Value of Financial Instruments [Abstract]

Fair Value of Financial Instruments Measured on Recurring Basis

	Fair Value Measurements as of December 31, 2021 Using:
	Level 1		Level 2		Level 3		Total
Assets:
Cash equivalents:
Money market mutual funds	$	179,900	$	-	$	-	$	179,900
		179,900		-		-		179,900

Investments:
United States Treasury securities		44,045		-		-		44,045
Corporate Bonds		-		96,696		-		96,696
Municipal Bonds		-		6,000		-		6,000
Agency Bonds		-		9,305		-		9,305
		44,045		112,001		-		156,046

	$	223,945	$	112,001	$	-	$	335,946

	Fair Value Measurements as of December 31, 2020 Using:
	Level 1		Level 2		Level 3		Total
Assets:
Cash equivalents:
Money market mutual funds	$	193,312	$	-	$	-	$	193,312
United States Treasury securities		62,497		-		-		62,497
Corporate Bonds		-		501		-		501
Agency Bonds		-		8,015		-		8,015
		255,809		8,516		-		264,325

Investments:
United States Treasury securities		112,328		-		-		112,328
Corporate Bonds		-		63,710		-		63,710
Municipal Bonds		-		6,000		-		6,000
Agency Bonds		-		3,583		-		3,583
		112,328		73,293		-		185,621

	$	368,137	$	81,809	$	-	$	449,946

Property and Equipment, Net (Tables)

12 Months Ended

Dec. 31, 2021

Property and Equipment, Net [Abstract]

Property and Equipment

The Company’s property and equipment consisted of the following:

	December 31, 2021			December 31, 2020
Laboratory equipment	$	12,600		$	7,807
Machinery and equipment		10,432			9,933
Computer equipment		218			218
Furniture and fixtures		1,963			1,880
Leasehold improvements		407			29
Internal use software		1,902			1,385
		27,522			21,252
Less: accumulated depreciation and amortization		(5,223	)		(2,046	)
	$	22,299		$	19,206

Accounts Payable and Accrued Expenses (Tables)

12 Months Ended

Dec. 31, 2021

Accounts Payable and Accrued Expenses [Abstract]

Accounts Payable and Accrued Expenses

At December 31, 2021 and 2020, the Company’s accounts payable and accrued expenses consisted of the following:

	December 31, 2021		December 31, 2020
Research and development	$	12,082	$	14,962
Property and equipment		725		1,756
Employee compensation		4,533		4,875
Accrued interest		-		1,122
Government grant payable		597		590
Professional fees		1,196		1,332
Other		482		835
	$	19,615	$	25,472

Convertible Notes Payable (Tables)

12 Months Ended

Dec. 31, 2021

Convertible Notes Payable [Abstract]

Carrying Value of Convertible Notes

The table below summarizes the carrying value of the 2021 and 2022 Convertible Notes as of December 31, 2021 and 2020:

	2021 Notes					2022 Notes
	December 31, 2021		December 31, 2020			December 31, 2021		December 31, 2020

Principal amount	$	-	$	5,150		$	-	$	38,350
Discount		-		(275	)		-		(3,284	)
Carrying value	$	-	$	4,875		$	-	$	35,066

Stock-Based Awards (Tables)

12 Months Ended

Dec. 31, 2021

Stock-Based Awards [Abstract]

Weighted-Average Assumptions for Stock Options

	Years Ended December 31,
	2021			2020			2019

Risk-free interest rate		0.83	%		1.00	%		2.26	%
Expected term (in years)		5.84			5.84			5.81
Expected volatility		69.27	%		76.98	%		75.71	%
Expected dividend yield		0.00	%		0.00	%		0.00	%
Exercise price	$	51.20		$	22.96		$	14.47
Fair value of common stock	$	51.20		$	22.96		$	14.47

Stock Option Activity

The following table summarizes stock option activity for the years ended December 31, 2021 and 2020, under the Revised 2014 Plan:

	Number of Shares			Weighted Average Exercise Price		Weighted Average Contractual Term (Years)		Aggregate Intrinsic Value

Outstanding as of December 31, 2019		9,763,541		$	5.95		6.96	$	164,021
Granted		2,193,546			22.96		8.97
Exercised		(586,857	)		4.82				13,494
Cancelled		(319,299	)		15.82
Outstanding as of December 31, 2020		11,050,931		$	9.10		6.55	$	504,079
Granted		1,671,759			51.20		8.58
Exercised		(1,209,960	)		9.32				54,487
Cancelled		(368,969	)		35.87
Outstanding as of December 31, 2021		11,143,761		$	14.51		5.95	$	128,817

Options vested and exercisable as of December 31, 2021		8,692,898		$	7.31		5.11	$	127,571
Options unvested as of December 31, 2021		2,450,863		$	40.01		8.93

Stock-Based Compensation Expense by Award Type

Stock-based compensation expense recognized by award type is as follows:

	Years Ended December 31,
	2021		2020		2019

Stock options	$	28,811	$	18,527	$	13,371
Restricted stock units		426		40		-
Total share based compensation expense	$	29,237	$	18,567	$	13,371

Stock-Based Compensation Expense

Stock-based compensation expense by classification included within the consolidated statements of operations and comprehensive loss was as follows:

	Years Ended December 31,
	2021		2020		2019

Research and development	$	11,954	$	7,355	$	6,153
General and administrative		17,283		11,212		7,218
Total share based compensation expense	$	29,237	$	18,567	$	13,371

RSU Activity

The following table summarizes our RSU activity for the years ended December 31, 2021 and 2020:

	Number of Shares		Weighted Average Grant Date Fair Value

Unvested as of December 31, 2019		-	$	-
Granted		20,000		25.06
Exercised		-
Forfeited		-
Unvested as of December 31, 2020		20,000		25.06
Granted		3,500		62.32
Exercised		-
Forfeited		-
Unvested as of December 31, 2021		23,500		30.61

Warrants Outstanding and Changes in Warrants to Purchase Common Stock

A summary of the warrants outstanding at December 31, 2021 is as follows:

Price	Outstanding		Grant Date	Expiration Date
24.42		7,051	June 28, 2013	June 28, 2023
57.11		603,386	December 21, 2020	December 21, 2030
33.63		301,291	August 9, 2021	August 9, 2031
22.51		153,155	December 17, 2021	December 17, 2031
22.51		153,155	December 17, 2021	December 17, 2031
Total		1,218,038

The following table below is the summary of changes in warrants to purchase common stock for the years ended December 31, 2021 and 2020:

	Number of Warrant Shares Outstanding and Exercisable			Exercise Price per Share

Balance as of December 31, 2019		14,102		$	24.42
Granted		603,386		$	57.11
Exercised		(7,051	)	$	-
Balance as of December 31, 2020		610,437
Granted August 2021		301,291		$	33.63
Granted December 2021		306,310		$	22.51
Exercised		-		$	-
Balance as of December 31, 2021		1,218,038

Fair Value Pricing Model of Warrants

The fair value of the 2021 and 2020 warrants was calculated using the Black-Scholes fair value pricing model with the following inputs:

	Years Ended December 31,
	2021			2020

Risk-free interest rate		1.37	%		0.95	%
Expected term (in years)		10.00			10.00
Expected volatility		70.25	%		74.20	%
Expected dividend yield		0.00	%		0.00	%
Exercise price	$	28.07		$	57.11
Fair value of common stock	$	28.07		$	57.11

Net Loss Per Share (Tables)

12 Months Ended

Dec. 31, 2021

Net Loss Per Share [Abstract]

Basic and Diluted Net Loss Per Share

Basic and diluted net loss per share attributable to common stockholders was calculated as follows:

	For the Years Ended December 31,
	2021			2020			2019

Numerator:
Net loss attributable to common stockholders	$	(169,069	)	$	(139,700	)	$	(77,270	)
Denominator:
Weighted-average common shares outstanding - basic and diluted		63,235,417			55,380,740			49,010,358
Net loss per share attributable to common stockholders - basic and diluted	$	(2.67	)	$	(2.52	)	$	(1.58	)

Antidilutive Securities Excluded from Computation of Diluted Net Loss Per Share

	For the Years Ended December 31,
	2021		2020		2019
Shares issuable upon conversion of the 2021 Convertible Notes		-		160,536		1,620,948
Shares issuable upon conversion of the 2022 Convertible Notes		-		1,195,449		-
Warrants exercisable for common shares		1,218,038		610,437		14,102
Restricted stock units exercisable for common shares		23,500		-		-
Options to purchase common shares		11,143,761		11,050,931		9,608,537
		12,385,299		13,017,353		11,243,587

Income Taxes (Tables)

12 Months Ended

Dec. 31, 2021

Income Taxes [Abstract]

Reconciliation of Income Tax Benefit Computed at Statutory Federal Income Tax Rate to Income Taxes

A reconciliation of income tax benefit computed at the statutory federal income tax rate to income taxes as reflected in the financial statements is as follows:

	For the years ended December 31,
	2021			2020			2019
U.S. federal tax at statutory rate		21.0	%		21.0	%		21.0	%
Foreign rate differential		(13.0	)%		0.0	%		0.0	%
Change in state tax apportionment		0.1	%		0.1	%		(17.4	)%
Stock compensation		1.5	%		0.9	%		(0.7	)%
Transfer pricing adjustments		(22.8	)%		0.0	%		0.0	%
Valuation allowance		4.6	%		(35.0	)%		(5.1	)%
Tax credits		8.7	%		13.5	%		0.0	%
Other		(0.2	)%		(0.5	)%		2.1	%
Effective tax rate		0	%		0	%		0	%

Deferred Tax Assets and Liabilities

The significant components of the Company’s deferred income tax assets and liabilities after applying the enacted corporate tax rates are as follows:

	At December 31,
	2021			2020			2019
Deferred income tax assets (liabilities)
R&D Credits	$	35,766		$	42,613		$	18,471
Net operating losses ("NOL") and credit carryforwards		26,789			21,359			2,517
Capitalized research and development costs		19,753			23,179			27,652
Stock-based compensation		11,552			7,406			3,896
Debt discount		-			(748	)		(1,460	)
Warrants		8,382			5,585			-
Other		(8,881	)		1,189			643
Valuation allowance		(93,361	)		(100,583	)		(51,719	)
Net deferred income tax asset	$	-		$	-		$	-

Commitments and Contingencies (Tables)

12 Months Ended

Dec. 31, 2021

Commitments and Contingencies [Abstract]

Lease Cost

Rent expense was $1.2 million, $1.1 million, and $0.9 million for the years ended December 31, 2021, 2020 and 2019, respectively.

Lease cost	December 31, 2021
Operating lease cost	$	645
Finance lease cost
Amortization of right of use assets		2,140
Interest on lease liablities		1,845
Total lease cost	$	4,630

Maturities of Operating Lease Lease Liabilities

Maturity of operating lease liabilities	December 31, 2021
2022		917
2023		548
2024		269
2025		64
2026		54
Total lease payments	$	1,852
Less: interest		(84	)
Total operating lease liabilities	$	1,768

Maturity of Finance Lease Liability

Maturity of finance lease liability	December 31, 2021
2022		1,689
2023		1,736
2024		1,791
2025		1,856
2026		1,912
Thereafter		45,000
Total lease payments	$	53,984
Less: interest		(33,151	)
Total finance lease liability	$	20,833

Balance Sheet Information Related to Leases

Leases	December 31, 2021

Operating right-of-use assets	$	1,569

Operating current lease liabilities		863
Operating noncurrent lease liabilities		905
Total operating lease liabilities	$	1,768

Finance right-of-use assets	$	48,480

Finance current lease liability		1,689
Finance noncurrent lease liability		19,144
Total finance lease liability	$	20,833

Other information
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows from operating leases	$	655
Cash flows from finance lease	$	1,644
Weighted-average remaining lease term - operating leases	2.5 years
Weighted-average remaining lease term - finance lease	22.7 years
Weighted-average discount rate - operating leases		4.46	%
Weighted-average discount rate - finance lease		8.96	%

Nature of Business and Basis of Presentation (Details)	12 Months Ended
Nature of Business and Basis of Presentation (Details)	Dec. 31, 2021 Program
Nature of Business and Basis of Presentation [Abstract]
Number of clinical-stage programs	3

Risks and Liquidity (Details) - USD ($) $ / shares in Units, $ in Thousands				1 Months Ended	12 Months Ended
	Aug. 27, 2021	Aug. 02, 2021	Apr. 26, 2021	Dec. 31, 2020	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019	Jun. 30, 2021	Mar. 31, 2021	Jun. 12, 2020	Feb. 20, 2020	Jan. 04, 2018
Risks and Liquidity [Abstract]
Accumulated deficit				$ (322,843)	$ (491,912)	$ (322,843)
Cash, cash equivalents and investments					388,700
Shareholders' Equity Disclosure [Abstract]
Issuance of common stock, net of issuance costs					26,354	280,763	$ 177,760
Conversion of convertible notes into common stock					40,694	7,629	$ 0
2022 Convertible Notes [Member]
Shareholders' Equity Disclosure [Abstract]
Redemption of principal balance			$ 38,400	38,350	$ 0	38,350			$ 38,400		$ 39,350
Convertible senior notes, interest rate, stated percentage			6.25%								6.25%
Debt instrument maturity date					Aug. 31, 2022
Conversion of convertible notes into common stock			$ 38,400	7,600
Outstanding amount					$ 0
2021 Convertible Notes [Member]
Shareholders' Equity Disclosure [Abstract]
Redemption of principal balance				$ 5,150	$ 0	$ 5,150		$ 5,150		$ 7,500		$ 52,000
Convertible senior notes, interest rate, stated percentage					5.75%					6.25%
Debt instrument maturity date					Aug. 01, 2021
Conversion of convertible notes into common stock		$ 5,150
Outstanding amount					$ 0
Common Stock [Member]
Shareholders' Equity Disclosure [Abstract]
Common stock shares issued and sold (in shares)					812,516	5,339,286	9,568,000
Private Placement [Member]
Shareholders' Equity Disclosure [Abstract]
Issuance of common stock, net of issuance costs	$ 26,400
Private Placement [Member] \| Common Stock [Member]
Shareholders' Equity Disclosure [Abstract]
Common stock shares issued and sold (in shares)	812,516				812,516
Share price (in dollars per share)	$ 32.48				$ 32.48
Issuance of common stock, net of issuance costs	$ 26,400				$ 26,400

Summary of Significant Accounting Policies, Cash, Cash Equivalents and Restricted Cash (Details) - USD ($) $ in Thousands	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019	Dec. 31, 2018
Cash, Cash Equivalents and Restricted Cash [Abstract]
Cash and cash equivalents	$ 232,694	$ 297,098
Restricted cash	1,343	1,568
Total	$ 234,037	$ 298,666	$ 186,908	$ 112,791

- Definition

Amount of cash equivalents restricted as to withdrawal or usage, classified as noncurrent. Cash equivalents include, but are not limited to, short-term, highly liquid investments that are both readily convertible to known amounts of cash and so near their maturity that they present insignificant risk of changes in value because of changes in interest rates.

+ References + Details

Summary of Significant Accounting Policies, Government Grants (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019
Government Grants [Abstract]
Receivable of offset grants against expenses	$ 0.1	$ 3.6	$ 1.2

Summary of Significant Accounting Policies, Investments (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019
Investments [Abstract]
Realized gains (losses) on investment	$ 0	$ 0	$ 0
Unrealized gain on investments	$ (119)	$ (62)	$ 147

Summary of Significant Accounting Policies, Goodwill (Details) $ in Millions	12 Months Ended
	Dec. 31, 2021 USD ($) Segment	Dec. 31, 2020 USD ($)	Dec. 31, 2019 USD ($)
Goodwill [Abstract]
Number of operating segment	1
Number of reporting unit	1
Goodwill impairment \| $	$ 0	$ 0	$ 0

Summary of Significant Accounting Policies, Property and Equipment (Details)	12 Months Ended
	Dec. 31, 2021
Internal use Software [Member]
Property and Equipment [Abstract]
Estimated useful lives	6 years
Minimum [Member]
Property and Equipment [Abstract]
Estimated useful lives	3 years
Maximum [Member]
Property and Equipment [Abstract]
Estimated useful lives	15 years

Summary of Significant Accounting Policies, Impairment of Long-Lived Assets (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019
Impairment of Long-Lived Assets [Abstract]
Impairment of long-lived assets	$ 0	$ 0	$ 0

Summary of Significant Accounting Policies, Tax Credits (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019
NYS Life Sciences Research and Development Tax Credit [Member]
Tax Credits [Abstract]
Research and development incentive income	$ 1.0	$ 0.0	$ 0.0
NYC Biotechnology Tax Credit Program [Member]
Tax Credits [Abstract]
Research and development incentive income	$ 0.0	$ 0.0	$ 0.3

Fair Value of Financial Instruments (Details) - Recurring [Member] - USD ($) $ in Thousands	Dec. 31, 2021	Dec. 31, 2020
Assets [Abstract]
Cash equivalents	$ 179,900	$ 264,325
Investments	156,046	185,621
Fair value of financial instruments	335,946	449,946
Money Market Mutual Funds [Member]
Assets [Abstract]
Cash equivalents	179,900	193,312
United States Treasury Securities [Member]
Assets [Abstract]
Cash equivalents		62,497
Investments	44,045	112,328
Corporate Bonds [Member]
Assets [Abstract]
Cash equivalents		501
Investments	96,696	63,710
Municipal Bonds [Member]
Assets [Abstract]
Investments	6,000	6,000
Agency Bonds [Member]
Assets [Abstract]
Cash equivalents		8,015
Investments	9,305	3,583
Level 1 [Member]
Assets [Abstract]
Cash equivalents	179,900	255,809
Investments	44,045	112,328
Fair value of financial instruments	223,945	368,137
Level 1 [Member] \| Money Market Mutual Funds [Member]
Assets [Abstract]
Cash equivalents	179,900	193,312
Level 1 [Member] \| United States Treasury Securities [Member]
Assets [Abstract]
Cash equivalents		62,497
Investments	44,045	112,328
Level 1 [Member] \| Corporate Bonds [Member]
Assets [Abstract]
Cash equivalents		0
Investments	0	0
Level 1 [Member] \| Municipal Bonds [Member]
Assets [Abstract]
Investments	0	0
Level 1 [Member] \| Agency Bonds [Member]
Assets [Abstract]
Cash equivalents		0
Investments	0	0
Level 2 [Member]
Assets [Abstract]
Cash equivalents	0	8,516
Investments	112,001	73,293
Fair value of financial instruments	112,001	81,809
Level 2 [Member] \| Money Market Mutual Funds [Member]
Assets [Abstract]
Cash equivalents	0	0
Level 2 [Member] \| United States Treasury Securities [Member]
Assets [Abstract]
Cash equivalents		0
Investments	0	0
Level 2 [Member] \| Corporate Bonds [Member]
Assets [Abstract]
Cash equivalents		501
Investments	96,696	63,710
Level 2 [Member] \| Municipal Bonds [Member]
Assets [Abstract]
Investments	6,000	6,000
Level 2 [Member] \| Agency Bonds [Member]
Assets [Abstract]
Cash equivalents		8,015
Investments	9,305	3,583
Level 3 [Member]
Assets [Abstract]
Cash equivalents	0	0
Investments	0	0
Fair value of financial instruments	0	0
Level 3 [Member] \| Money Market Mutual Funds [Member]
Assets [Abstract]
Cash equivalents	0	0
Level 3 [Member] \| United States Treasury Securities [Member]
Assets [Abstract]
Cash equivalents		0
Investments	0	0
Level 3 [Member] \| Corporate Bonds [Member]
Assets [Abstract]
Cash equivalents		0
Investments	0	0
Level 3 [Member] \| Municipal Bonds [Member]
Assets [Abstract]
Investments	0	0
Level 3 [Member] \| Agency Bonds [Member]
Assets [Abstract]
Cash equivalents		0
Investments	$ 0	$ 0

Property and Equipment, Net (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019
Property and Equipment [Abstract]
Property and equipment, gross	$ 27,522	$ 21,252
Less: accumulated depreciation and amortization	(5,223)	(2,046)
Property and equipment, net	22,299	19,206
Depreciation and amortization	3,240	1,145	$ 426
Laboratory Equipment [Member]
Property and Equipment [Abstract]
Property and equipment, gross	12,600	7,807
Machinery and Equipment [Member]
Property and Equipment [Abstract]
Property and equipment, gross	10,432	9,933
Computer Equipment [Member]
Property and Equipment [Abstract]
Property and equipment, gross	218	218
Furniture and Fixtures [Member]
Property and Equipment [Abstract]
Property and equipment, gross	1,963	1,880
Leasehold Improvements [Member]
Property and Equipment [Abstract]
Property and equipment, gross	407	29
Internal use Software [Member]
Property and Equipment [Abstract]
Property and equipment, gross	$ 1,902	$ 1,385

Accounts Payable and Accrued Expenses (Details) - USD ($) $ in Thousands	Dec. 31, 2021	Dec. 31, 2020
Accounts Payable and Accrued Expenses [Abstract]
Research and development	$ 12,082	$ 14,962
Property and equipment	725	1,756
Employee compensation	4,533	4,875
Accrued interest	0	1,122
Government grant payable	597	590
Professional fees	1,196	1,332
Other	482	835
Total	$ 19,615	$ 25,472

Convertible Notes Payable (Details)				1 Months Ended	12 Months Ended
Convertible Notes Payable (Details)	Aug. 02, 2021 USD ($) shares	Apr. 26, 2021 USD ($) d shares	Feb. 20, 2020 USD ($) $ / shares	Dec. 31, 2020 USD ($) shares	Dec. 31, 2021 USD ($) d $ / shares	Dec. 31, 2020 USD ($)	Dec. 31, 2019 USD ($)	Jun. 30, 2021 USD ($)	Mar. 31, 2021 USD ($)	Jun. 12, 2020 USD ($)	Jan. 04, 2018 USD ($)
Debt [Abstract]
Conversion of convertible notes into common stock					$ 40,694,000	$ 7,629,000	$ 0
Carrying value of convertible notes [Abstract]
Carrying value				$ 35,066,000	0	35,066,000
Accretion of debt discount					$ 753,000	2,758,000	3,602,000
2021 Convertible Notes [Member]
Debt [Abstract]
Debt instrument maturity date					Aug. 01, 2021
Convertible senior notes, interest rate, stated percentage					5.75%					6.25%
Debt conversion ratio					31.1876
Conversion per principal amount of debt					$ 1,000.00
Conversion price (in dollars per share) \| $ / shares					$ 32.08
Conversion of convertible notes into common stock	$ 5,150,000
Convertible Notes converted into shares of common stock (in shares) \| shares	160,614
Convertible notes, outstanding					$ 0
Debt instrument, aggregate principal amount exchanged			$ 39,350,000							$ 7,500,000
Carrying value of convertible notes [Abstract]
Principal amount				5,150,000	0	5,150,000		$ 5,150,000		$ 7,500,000	$ 52,000,000.0
Discount				(275,000)	0	(275,000)
Carrying value				4,875,000	0	4,875,000
Accretion of debt discount					$ 300,000	1,300,000	$ 3,600,000
2022 Convertible Notes [Member]
Debt [Abstract]
Debt instrument maturity date					Aug. 31, 2022
Convertible senior notes, interest rate, stated percentage		6.25%	6.25%
Debt conversion ratio			31.1876
Conversion per principal amount of debt			$ 1,000.00
Conversion price (in dollars per share) \| $ / shares			$ 32.06
Conversion of convertible notes into common stock		$ 38,400,000		$ 7,600,000
Convertible Notes converted into shares of common stock (in shares) \| shares		1,300,000		298,562
Convertible notes, outstanding					$ 0
Percentage of common stock conversion price			130.00%
Number of trading days, not consecutive \| d		20			20
Number of consecutive trading days \| d		30			30
Conversion of convertible notes into common stock, principal amount				$ 8,500,000
Carrying value of convertible notes [Abstract]
Principal amount		$ 38,400,000	$ 39,350,000	38,350,000	$ 0	38,350,000			$ 38,400,000
Discount				(3,284,000)	0	(3,284,000)
Carrying value				$ 35,066,000	0	35,066,000
Accretion of debt discount					$ 500,000	$ 1,500,000

Shareholders' Equity (Details) $ / shares in Units, $ in Thousands				12 Months Ended
	Aug. 27, 2021 USD ($) $ / shares shares	Dec. 14, 2020 USD ($) $ / shares shares	Dec. 12, 2019 USD ($)	Dec. 31, 2021 USD ($) Vote $ / shares shares	Dec. 31, 2020 USD ($) $ / shares shares	Dec. 31, 2019 USD ($) shares
Common Stock [Abstract]
Common stock, shares authorized (in shares) \| shares				120,000,000	120,000,000
Common stock, par value (in dollars per share) \| $ / shares				$ 0.01	$ 0.01
Number of voting right for each share \| Vote				1
Treasury Stock [Abstract]
Shares withheld to pay the payroll tax liability				$ 0	$ 614	$ 344
Private Placement and Public Offerings [Abstract]
Issuance of common stock, net of issuance costs				26,354	280,763	177,760
Net proceeds from offering				$ 11,327	$ 2,558	$ 31
Private Placement [Member]
Private Placement and Public Offerings [Abstract]
Issuance of common stock, net of issuance costs	$ 26,400
Common Stock [Member]
Private Placement and Public Offerings [Abstract]
Common stock shares issued (in shares) \| shares				812,516	5,339,286	9,568,000
Common Stock [Member] \| Private Placement [Member]
Private Placement and Public Offerings [Abstract]
Common stock shares issued (in shares) \| shares	812,516			812,516
Share price (in dollars per share) \| $ / shares	$ 32.48			$ 32.48
Issuance of common stock, net of issuance costs	$ 26,400			$ 26,400
Common Stock [Member] \| Public Offering [Member]
Private Placement and Public Offerings [Abstract]
Common stock shares issued (in shares) \| shares		5,339,286
Share price (in dollars per share) \| $ / shares		$ 56.00
Gross proceeds, offering amount		$ 299,000
Offering costs		18,200
Net proceeds from offering		$ 280,800
Common Stock [Member] \| Exercise of Underwriters' Option [Member]
Private Placement and Public Offerings [Abstract]
Common stock shares issued (in shares) \| shares		696,428
Treasury Stock [Member]
Treasury Stock [Abstract]
Shares withheld to pay the payroll tax liability			$ 100
Treasury stock (in shares) \| shares				0	0

Stock-Based Awards, Stock Option Valuation (Details) - Stock Options [Member] - USD ($) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019
Stock Option Activity [Roll Forward]
Outstanding at beginning of period (in shares)	11,050,931	9,763,541
Granted (in shares)	1,671,759	2,193,546
Exercised (in shares)	(1,209,960)	(586,857)
Cancelled (in shares)	(368,969)	(319,299)
Outstanding at end of period (in shares)	11,143,761	11,050,931	9,763,541
Options vested and exercisable at end of period (in shares)	8,692,898
Options unvested at end of period (in shares)	2,450,863
Weighted Average Exercise Price [Abstract]
Outstanding at beginning of period (in dollars per share)	$ 9.10	$ 5.95
Granted (in dollars per share)	51.20	22.96
Exercised (in dollars per share)	9.32	4.82
Cancelled (in dollars per share)	35.87	15.82
Outstanding at end of period (in dollars per share)	14.51	$ 9.10	$ 5.95
Options vested and exercisable at end of period (in dollars per share)	7.31
Options unvested at end of period (in dollars per share)	$ 40.01
Weighted-Average Remaining Contractual Term [Abstract]
Outstanding	5 years 11 months 12 days	6 years 6 months 18 days	6 years 11 months 15 days
Granted	8 years 6 months 29 days	8 years 11 months 19 days
Options vested and exercisable	5 years 1 month 9 days
Options unvested	8 years 11 months 4 days
Aggregate Intrinsic Value [Abstract]
Outstanding at beginning of period	$ 504,079	$ 164,021
Exercised	54,487	13,494
Outstanding at end of period	128,817	$ 504,079	$ 164,021
Options vested and exercisable	$ 127,571
Weighted average grant date fair value of shares granted (in dollars per share)	$ 31.07	$ 15.10	$ 9.58
Total fair value of options vested	$ 22,600	$ 15,600	$ 63,100
Employees and Directors [Member]
Fair Value Assumptions [Abstract]
Risk-free interest rate	0.83%	1.00%	2.26%
Expected term (in years)	5 years 10 months 2 days	5 years 10 months 2 days	5 years 9 months 21 days
Expected volatility	69.27%	76.98%	75.71%
Expected dividend yield	0.00%	0.00%	0.00%
Exercise price (in dollars per share)	$ 51.20	$ 22.96	$ 14.47
Fair value of common stock (in dollars per share)	$ 51.20	$ 22.96	$ 14.47

Stock-Based Awards, Stock-Based Compensation (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019
Stock-Based Compensation [Abstract]
Share based compensation expense	$ 29,237	$ 18,567	$ 13,371
Unrecognized share-based compensation cost	$ 46,300	$ 30,400
Weighted average period expected to recognize unrecognized share-based compensation cost	1 year 9 months 18 days	2 years 1 month 6 days
Research and Development [Member]
Stock-Based Compensation [Abstract]
Share based compensation expense	$ 11,954	$ 7,355	6,153
General and Administrative [Member]
Stock-Based Compensation [Abstract]
Share based compensation expense	17,283	11,212	7,218
Stock Options [Member]
Stock-Based Compensation [Abstract]
Share based compensation expense	28,811	18,527	13,371
Restricted Stock Units [Member]
Stock-Based Compensation [Abstract]
Share based compensation expense	426	$ 40	$ 0
Unrecognized share-based compensation cost	$ 500
Weighted average period expected to recognize unrecognized share-based compensation cost	2 years 9 months 18 days

Stock-Based Awards, Restricted Stock Units (Details) $ / shares in Units, $ in Millions	12 Months Ended
	Dec. 31, 2021 USD ($) $ / shares shares	Dec. 31, 2020 USD ($) $ / shares shares
Number of Shares [Roll Forward]
Exercised (in shares)		7,051
Weighted Average Grant Date Fair Value [Abstract]
Unrecognized share-based compensation cost \| $	$ 46.3	$ 30.4
Weighted average period expected to recognize unrecognized share-based compensation cost	1 year 9 months 18 days	2 years 1 month 6 days
Restricted Stock Units (RSU) [Member]
Number of Shares [Roll Forward]
Beginning balance (in shares)	20,000	0
Granted (in shares)	3,500	20,000
Exercised (in shares)	0	0
Forfeited (in shares)	0	0
Ending balance (in shares)	23,500	20,000
Weighted Average Grant Date Fair Value [Abstract]
Beginning balance (in dollars per share) \| $ / shares	$ 25.06	$ 0
Granted (in dollars per share) \| $ / shares	62.32	25.06
Ending balance (in dollars per share) \| $ / shares	$ 30.61	$ 25.06
Intrinsic value \| $	$ 0.4	$ 0.5
Unrecognized share-based compensation cost \| $	$ 0.5
Weighted average period expected to recognize unrecognized share-based compensation cost	2 years 9 months 18 days

Stock-Based Awards, Warrants (Details) - USD ($) $ / shares in Units, $ in Thousands	1 Months Ended		12 Months Ended
	Dec. 31, 2021	Aug. 31, 2021	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019	Dec. 17, 2021	Aug. 09, 2021	Dec. 21, 2020
Warrants [Abstract]
Outstanding (in shares)	1,218,038		1,218,038
Number of Warrant Shares Outstanding and Exercisable [Abstract]
Exercised (in shares)				(7,051)
Expense in connection with warrant issue			$ 12,781	$ 26,562	$ 0
Warrants [Member]
Warrants [Abstract]
Exercise price per share (in dollars per share)					$ 24.42	$ 22.51	$ 33.63	$ 57.11
Number of Warrant Shares Outstanding and Exercisable [Abstract]
Beginning balance (in shares)			610,437	14,102
Granted (in shares)	306,310	301,291		603,386
Exercised (in shares)			0	(7,051)
Ending balance (in shares)	1,218,038		1,218,038	610,437	14,102
Fair Value Assumptions [Abstract]
Risk-free interest rate			1.37%	0.95%
Expected term (in years)			10 years	10 years
Expected volatility			70.25%	74.20%
Expected dividend yield			0.00%	0.00%
Exercise price (in dollars per share)	$ 28.07		$ 28.07	$ 57.11
Fair value of common stock (in dollars per share)	28.07		28.07	$ 57.11
Common Stock [Member]
Number of Warrant Shares Outstanding and Exercisable [Abstract]
Issuance of common stock pursuant to exercise of warrant (in shares)				1,601
22.42 [Member]
Warrants [Abstract]
Exercise price per share (in dollars per share)	$ 24.42		$ 24.42
Outstanding (in shares)	7,051		7,051
Grant date			Jun. 28, 2013
Expiration date	Jun. 28, 2023		Jun. 28, 2023
57.11 [Member]
Warrants [Abstract]
Exercise price per share (in dollars per share)	$ 57.11		$ 57.11
Outstanding (in shares)	603,386		603,386
Grant date			Dec. 21, 2020
Expiration date	Dec. 21, 2030		Dec. 21, 2030
33.63 [Member]
Warrants [Abstract]
Exercise price per share (in dollars per share)	$ 33.63		$ 33.63
Outstanding (in shares)	301,291		301,291
Grant date			Aug. 09, 2021
Expiration date	Aug. 09, 2031		Aug. 09, 2031
22.51 [Member]
Warrants [Abstract]
Exercise price per share (in dollars per share)	$ 22.51		$ 22.51
Outstanding (in shares)	153,155		153,155
Grant date			Dec. 17, 2021
Expiration date	Dec. 17, 2031		Dec. 17, 2031
22.51 [Member]
Warrants [Abstract]
Exercise price per share (in dollars per share)	$ 22.51		$ 22.51
Outstanding (in shares)	153,155		153,155
Grant date			Dec. 17, 2021
Expiration date	Dec. 17, 2031		Dec. 17, 2031

Net Loss Per Share (Details) - USD ($) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019
Numerator [Abstract]
Net loss attributable to common stockholders	$ (169,069)	$ (139,700)	$ (77,270)
Denominator [Abstract]
Weighted-average common shares outstanding - basic (in shares)	63,235,417	55,380,740	49,010,358
Weighted-average common shares outstanding - diluted (in shares)	63,235,417	55,380,740	49,010,358
Net loss per share attributable to common stockholders - basic (in dollars per share)	$ (2.67)	$ (2.52)	$ (1.58)
Net loss per share attributable to common stockholders - diluted (in dollars per share)	$ (2.67)	$ (2.52)	$ (1.58)
Weighted Average Number of Shares Outstanding Reconciliation [Abstract]
Warrants exercisable for common shares (in shares)	1,218,038	610,437	14,102
Restricted stock units exercisable for common shares (in shares)	23,500	0	0
Options to purchase common shares (in shares)	11,143,761	11,050,931	9,608,537
Weighted Average Number Diluted Shares Outstanding (in shares)	12,385,299	13,017,353	11,243,587
Convertible Notes 2021 [Member]
Weighted Average Number of Shares Outstanding Reconciliation [Abstract]
Shares issuable upon conversion of Convertible Notes (in shares)	0	160,536	1,620,948
Convertible Notes 2022 [Member]
Weighted Average Number of Shares Outstanding Reconciliation [Abstract]
Shares issuable upon conversion of Convertible Notes (in shares)	0	1,195,449	0

Income Taxes, Net Loss by Jurisdiction (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019
Federal [Member]
Details of Income Taxes [Abstract]
Provision for income taxes	$ 0	$ 0	$ 0
State [Member]
Details of Income Taxes [Abstract]
Provision for income taxes	$ 0	$ 0	$ 0

- Definition

Amount of current income tax expense (benefit) and deferred income tax expense (benefit) pertaining to continuing operations.

+ References + Details

Income Taxes, Reconciliation Between Effective Tax Rates and Statutory Rates (Details)	12 Months Ended
	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019
Reconciliation of Income Tax Benefit Computed at Statutory Federal Income Tax Rate to Income Taxes [Abstract]
U.S. federal tax at statutory rate	21.00%	21.00%	21.00%
Foreign rate differential	(13.00%)	0.00%	0.00%
Change in state tax apportionment	0.10%	0.10%	(17.40%)
Stock compensation	1.50%	0.90%	(0.70%)
Transfer pricing adjustments	(22.80%)	0.00%	0.00%
Valuation allowance	4.60%	(35.00%)	(5.10%)
Tax credits	8.70%	13.50%	0.00%
Other	(0.20%)	(0.50%)	2.10%
Effective tax rate	0.00%	0.00%	0.00%

Income Taxes, Deferred Tax Assets and Liabilities (Details) - USD ($) $ in Thousands	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019
Deferred income tax assets (liabilities) [Abstract]
R&D Credits	$ 35,766	$ 42,613	$ 18,471
Net operating losses ("NOL") and credit carryforwards	26,789	21,359	2,517
Capitalized research and development costs	19,753	23,179	27,652
Stock-based compensation	11,552	7,406	3,896
Debt discount	0	(748)	(1,460)
Warrants	8,382	5,585	0
Other		1,189	643
Other	(8,881)
Valuation allowance	(93,361)	(100,583)	(51,719)
Net deferred income tax asset	$ 0	$ 0	$ 0

Income Taxes, NOL Carryforwards and Tax Credits (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019	Dec. 31, 2017
Operating Loss Carryforwards [Abstract]
R&D Credits	$ 35,766	$ 42,613	$ 18,471
Increase in valuation allowance	(7,200)	$ 48,900
Annual limitations under section 382 of IRC	$ 1,700
Tax Credit Carryforward [Abstract]
Transfer pricing adjustments	(22.80%)	0.00%	0.00%
Uncertain Tax Positions [Abstract]
Uncertain tax positions	$ 0	$ 0	$ 0
Income Tax Penalties and Interest Accrued [Abstract]
Accrued interest or penalties	0	0	0
Income tax adjustments from prior periods	$ 0
Research and Development [Member]
Tax Credit Carryforward [Abstract]
Transfer pricing adjustments	10.00%
Income tax expense	$ 0
Federal [Member]
Operating Loss Carryforwards [Abstract]
NOL carryforwards	$ 118,200			$ 127,100
NOL carryforwards, expiration date	Dec. 31, 2026
Net operating loss not subject to expiration	$ 85,700
R&D Credits	$ 35,800
Tax credit, expiration date	Dec. 31, 2038
Net operating loss, subject to expiration	$ 35,800			$ 91,200
Cumulative limitation period	20 years
Tax Credit Carryforward [Abstract]
Income tax expense	$ 0	0	0
Federal [Member] \| Research and Development [Member]
Tax Credit Carryforward [Abstract]
Tax credit carryforwards, subject to expiration	4,900
State [Member]
Operating Loss Carryforwards [Abstract]
NOL carryforwards	$ 30,900
NOL carryforwards, expiration date	Dec. 31, 2026
Tax Credit Carryforward [Abstract]
Income tax expense	$ 0	$ 0	$ 0

Commitments and Contingencies, Finance Lease (Details) $ in Thousands	12 Months Ended
	Dec. 31, 2021 USD ($) ft² LeaseAgreement	Dec. 31, 2020 USD ($)	Mar. 15, 2020 USD ($)
Finance Lease [Abstract]
Total lease payments	$ 53,984
Cash security deposit	455	$ 455
Restricted cash	1,343	1,568
Finance lease, right-of-use assets	48,480	50,521
Finance lease, liability	20,833
Operating and Finance Leases [Member]
Finance Lease [Abstract]
Restricted cash	$ 800	1,100
NJ Lease Agreement [Member]
Finance Lease [Abstract]
Area of lease \| ft²	103,720
Term of finance lease agreement	15 years
Number of options to renew lease agreement \| LeaseAgreement	2
Term of renewal of finance lease agreement	5 years
Estimated rent payments	$ 1,200
Percentage of annual increase in base rent	3.00%
Total lease payments	$ 29,300
Cash security deposit	300	$ 300
Finance lease, right-of-use assets			$ 47,700
Finance lease, liability			20,200
Reclassification of construction costs in progress to right-of-use assets	100		26,500
Reclassification of prepaid rent to right-of-use assets			$ 1,100
Aggregate amount for reclassification of construction costs in progress to right-of-use assets	$ 32,100
AAV Current Good Manufacturing Practice (cGMP) [Member]
Finance Lease [Abstract]
Area of lease \| ft²	50,000

Commitments and Contingencies, Operating Leases (Details) - USD ($) $ in Millions			12 Months Ended
	Mar. 26, 2021	May 31, 2020	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019	Jun. 07, 2018
Operating Leases [Abstract]
Rent expense			$ 1.2	$ 1.1	$ 0.9
NY Lease Agreement [Member]
Operating Leases [Abstract]
Loss on lease termination		$ (76.0)
Lease expiration date			Jul. 31, 2022
ESB Lease Agreement [Member]
Operating Leases [Abstract]
Term of lease agreement			3 years
Letter of credit						$ 0.9
Certificate of deposit			$ 0.8	0.9
Lease expiration date			Aug. 29, 2024
ESB Lease Agreement Amendment [Member]
Operating Leases [Abstract]
Letter of credit			$ 0.8
Operating lease right of use asset, adjustment	$ 1.1
Lease expiration date			Jun. 30, 2024
Inotek Lexington Massachusetts Lease Agreement [Member]
Operating Leases [Abstract]
Lease expiration date			Feb. 28, 2023
Rental income received under sublease agreement			$ 0.4	$ 0.4	$ 0.3

Commitments and Contingencies, Details of Operating and Finance Lease (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2021	Dec. 31, 2020
Lease cost [Abstract]
Operating lease cost	$ 645
Finance lease cost [Abstract]
Amortization of right of use assets	2,140
Interest on lease liabilities	1,845
Total lease cost	4,630
Maturities of operating lease liabilities [Abstract]
2022	917
2023	548
2024	269
2025	64
2026	54
Total lease payments	1,852
Less: interest	(84)
Total operating lease liabilities	1,768
Maturity of finance lease liability [Abstract]
2022	1,689
2023	1,736
2024	1,791
2025	1,856
2026	1,912
Thereafter	45,000
Total lease payments	53,984
Less: interest	(33,151)
Total finance lease liability	20,833
Lease assets and liabilities [Abstract]
Operating right-of-use assets	1,569	$ 914
Operating current lease liabilities	863	626
Operating noncurrent lease liabilities	905	498
Total operating lease liabilities	1,768
Finance lease, right-of-use assets	48,480	50,521
Finance current lease liability	1,689	1,644
Finance noncurrent lease liability	19,144	$ 18,988
Total finance lease liability	20,833
Cash paid for amounts included in the measurement of lease liabilities [Abstract]
Operating cash flows from operating leases	655
Cash flows from finance lease	$ 1,644
Weighted-average remaining lease term - operating leases	2 years 6 months
Weighted-average remaining lease term - finance lease	22 years 8 months 12 days
Weighted-average discount rate - operating leases	4.46%
Weighted-average discount rate - finance lease	8.96%

Agreements Related to Intellectual Property (Details) $ in Thousands, $ / Vector in Millions	1 Months Ended	12 Months Ended
	Feb. 28, 2017 USD ($)	Dec. 31, 2021 USD ($) Patent Milestone $ / Vector	Dec. 31, 2020 Milestone
License Agreement for Danon Disease [Member]
License Agreement [Abstract]
Research and development costs	$ 50
License Agreement for Danon Disease [Member] \| Maximum [Member]
License Agreement [Abstract]
Potential future obligation	$ 1,500
REGENXBIO, Inc. License [Member]
License Agreement [Abstract]
Research and development costs		$ 7,000
Potential future obligation		$ 13,000
Term of agreement		10 years
Termination notice period		6 months
Number of patents \| Patent		2
Options right expiry term		4 years
Fee payable (per additional vector) \| $ / Vector		2.0
Royalties payable on fees received		20.00%
Payment for License Fee		$ 1,000
Number of additional milestones achieved \| Milestone		0	0

CIRM Grants (Details) $ in Millions			12 Months Ended
CIRM Grants (Details) $ in Millions	Apr. 01, 2021 USD ($)	Jan. 04, 2021 USD ($)	Dec. 31, 2021 USD ($) Grant	Dec. 31, 2019 USD ($)	Dec. 31, 2020 USD ($)	Nov. 12, 2020 USD ($)	Apr. 30, 2019 USD ($)
LAD-I CIRM Grant [Member]
CIRM Grant [Abstract]
Grant award for clinical development support							$ 6.5
Amount received from grant for eligible costs incurred				$ 1.2
Grant receivable included in prepaid and other assets					$ 1.1
Number of grants \| Grant			2
Milestone payments received	$ 1.0	$ 1.1
IMO CIRM Grant [Member]
CIRM Grant [Abstract]
Grant award for clinical development support						$ 3.7
Amount received from grant for eligible costs incurred		$ 1.0
Grant receivable included in prepaid and other assets					$ 0.9
Decrease in research and development expense			$ 0.1

Related Party Transactions (Details) - USD ($) $ / shares in Units, $ in Thousands		1 Months Ended			12 Months Ended
	Aug. 27, 2021	Dec. 31, 2021	Aug. 31, 2021	Oct. 31, 2020	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019	Dec. 17, 2021	Aug. 09, 2021	Dec. 21, 2020
Related Party Transaction [Abstract]
Expense in connection with warrant issue					$ 12,781	$ 26,562	$ 0
Issuance of common stock, net of issuance costs					$ 26,354	$ 280,763	$ 177,760
Private Placement [Member]
Related Party Transaction [Abstract]
Issuance of common stock, net of issuance costs	$ 26,400
Period to file S-3 following demand by purchaser	60 days
Common Stock [Member]
Related Party Transaction [Abstract]
Issuance of common stock, net of issuance costs (in shares)					812,516	5,339,286	9,568,000
Common Stock [Member] \| Private Placement [Member]
Related Party Transaction [Abstract]
Issuance of common stock, net of issuance costs (in shares)	812,516				812,516
Share price (in dollars per share)	$ 32.48	$ 32.48			$ 32.48
Issuance of common stock, net of issuance costs	$ 26,400				$ 26,400
Restricted Stock Units (RSU) [Member]
Related Party Transaction [Abstract]
Shares granted (in shares)					3,500	20,000
Warrants [Member]
Related Party Transaction [Abstract]
Shares granted (in shares)		306,310	301,291			603,386
Warrant issued (in shares)		12,800,000			12,800,000
Consulting Agreement, Business Development Services [Member]
Related Party Transaction [Abstract]
Termination notice period for business development consulting services agreement					60 days
Term of consulting agreement					3 years
Consulting Agreement, Business Development Services [Member] \| Warrants [Member]
Related Party Transaction [Abstract]
Warrant issued (in shares)										603,386
Expense in connection with warrant issue						$ 26,600
August 2021 Warrant [Member] \| Common Stock [Member]
Related Party Transaction [Abstract]
Expense in connection with warrant issue					$ 7,600
August 2021 Warrant [Member] \| Consulting Agreement, Business Development Services [Member] \| Common Stock [Member]
Related Party Transaction [Abstract]
Warrant issued (in shares)									301,291
December 2021 Warrants [Member] \| Common Stock [Member]
Related Party Transaction [Abstract]
Expense in connection with warrant issue					5,200
December 2021 Warrants [Member] \| Consulting Agreement, Business Development Services [Member] \| Common Stock [Member]
Related Party Transaction [Abstract]
Warrant issued (in shares)								153,155
December 2021 Warrants [Member] \| Consulting Agreement, Asset Identification Services [Member] \| Common Stock [Member]
Related Party Transaction [Abstract]
Warrant issued (in shares)								153,155
Member of the Board of Directors - One [Member]
Related Party Transaction [Abstract]
Business development consulting services expense (per quarter)					$ 28
Termination notice period for business development consulting services agreement					14 days
Related party expenses					$ 100	$ 100	$ 100
Member of the Board of Directors - Two [Member]
Related Party Transaction [Abstract]
Term of consulting agreement					1 year
Number of options granted (in shares)					20,000
Fair value amount of options granted					$ 400
Spouse of Executive officer [Member] \| Restricted Stock Units (RSU) [Member]
Related Party Transaction [Abstract]
Shares granted (in shares)				10,000
Vesting period					3 years

401(k) Savings Plan (Details) - USD ($) $ in Millions	12 Months Ended
401(k) Savings Plan (Details) - USD ($) $ in Millions	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019
401(k) Savings Plan [Abstract]
Percentage of matching employee contributions	4.00%
Matching employee contributions	$ 0.6	$ 0.3	$ 0.2