Rocket Pharmaceuticals (RCKT) 10-Q 9 Aug 21 2021 Q2 Quarterly report Financial data

Document and Entity Information

Document and Entity Information - shares	6 Months Ended
	Jun. 30, 2021	Aug. 02, 2021
Cover page [Abstract]
Document Type	10-Q
Amendment Flag	false
Document Quarterly Report	true
Document Period End Date	Jun. 30, 2021
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2021
Document Fiscal Period Focus	Q2
Document Transition Report	false
Entity File Number	001-36829
Entity Registrant Name	Rocket Pharmaceuticals, Inc.
Entity Central Index Key	0001281895
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	04-3475813
Entity Address, Address Line One	9 Cedarbrook Drive
Entity Address, City or Town	Cranbury
Entity Address, State or Province	NJ
Entity Address, Postal Zip Code	08512
City Area Code	646
Local Phone Number	440-9100
Title of 12(b) Security	Common Stock, $0.01 par value
Trading Symbol	RCKT
Security Exchange Name	NASDAQ
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Entity Shell Company	false
Entity Common Stock, Shares Outstanding		63,615,685

Consolidated Balance Sheets

Consolidated Balance Sheets - USD ($) $ in Thousands	Jun. 30, 2021	Dec. 31, 2020
Current assets:
Cash and cash equivalents	$ 264,608	$ 297,098
Investments	162,222	185,621
Prepaid expenses and other current assets	3,596	4,626
Total current assets	430,426	487,345
Property and equipment, net	20,989	19,206
Goodwill	30,815	30,815
Restricted cash	1,334	1,568
Deposits	455	455
Operating lease right-of-use assets	1,628	914
Finance lease right-of-use asset	49,507	50,521
Total assets	535,154	590,824
Current liabilities:
Accounts payable and accrued expenses	17,884	25,472
Convertible notes, net of unamortized discount, current	5,102	4,875
Operating lease liabilities, current	792	626
Finance lease liability, current	1,666	1,644
Total current liabilities	25,444	32,617
Convertible notes, net of unamortized discount, non-current	0	35,066
Operating lease liabilities, non-current	1,096	498
Finance lease liability, non-current	19,070	18,988
Other liabilities	107	136
Total liabilities	45,717	87,305
Commitments and contingencies (Note 11)
Stockholders' equity:
Common stock, $0.01 par value, 120,000,000 shares authorized; 63,448,069 and 60,996,367 shares issued and outstanding at June 30, 2021 and December 31, 2020, respectively	634	610
Additional paid-in capital	886,431	825,794
Accumulated other comprehensive loss	(81)	(42)
Accumulated deficit	(397,547)	(322,843)
Total stockholders' equity	489,437	503,519
Total liabilities and stockholders' equity	535,154	590,824
Series A Convertible Preferred Shares [Member]
Stockholders' equity:
Preferred stock	0	0
Series B Convertible Preferred Shares [Member]
Stockholders' equity:
Preferred stock	$ 0	$ 0

Consolidated Balance Sheets (Pa

Consolidated Balance Sheets (Parenthetical) - $ / shares	Jun. 30, 2021	Dec. 31, 2020
Stockholders' 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)	63,448,069	60,996,367
Common stock, shares outstanding (in shares)	63,448,069	60,996,367
Series A Convertible Preferred Shares [Member]
Stockholders' 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 Shares [Member]
Stockholders' 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 Oper

Consolidated Statements of Operations - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2021	Jun. 30, 2020	Jun. 30, 2021	Jun. 30, 2020
Consolidated Statements of Operations [Abstract]
Revenue	$ 0	$ 0	$ 0	$ 0
Operating expenses:
Research and development	24,798	16,731	53,340	33,687
General and administrative	9,250	6,828	19,930	13,990
Total operating expenses	34,048	23,559	73,270	47,677
Loss from operations	(34,048)	(23,559)	(73,270)	(47,677)
Research and development incentives	0	0	500	0
Interest expense	(251)	(1,786)	(1,980)	(3,360)
Interest and other income - net	501	429	1,412	1,395
Amortization of premium on investments - net	(727)	(124)	(1,366)	(62)
Net loss	$ (34,525)	$ (25,040)	$ (74,704)	$ (49,704)
Net loss per share attributable to common stockholders - basic (in dollars per share)	$ (0.55)	$ (0.45)	$ (1.20)	$ (0.90)
Net loss per share attributable to common stockholders - diluted (in dollars per share)	$ (0.55)	$ (0.45)	$ (1.20)	$ (0.90)
Weighted-average common shares outstanding - basic (in shares)	63,061,232	55,158,459	62,321,926	55,020,789
Weighted-average common shares outstanding - diluted (in shares)	63,061,232	55,158,459	62,321,926	55,020,789

Consolidated Statements of Comp

Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2021	Jun. 30, 2020	Jun. 30, 2021	Jun. 30, 2020
Consolidated Statements of Comprehensive Loss [Abstract]
Net loss	$ (34,525)	$ (25,040)	$ (74,704)	$ (49,704)
Other comprehensive loss
Net unrealized gain (loss) on investments	(6)	304	(39)	209
Total comprehensive loss	$ (34,531)	$ (24,736)	$ (74,743)	$ (49,495)

Consolidated Statements of Stoc

Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands	Common Stock [Member]	Treasury Stock [Member]	Additional Paid-In Capital [Member]	Accumulated Other Comprehensive Loss [Member]	Accumulated Deficit [Member]	Total
Beginning Balance at Dec. 31, 2019	$ 548	$ (53)	$ 489,925	$ 20	$ (183,143)	$ 307,297
Beginning Balance (in shares) at Dec. 31, 2019	54,773,061
Issuance of common stock pursuant to exercise of stock options	$ 3	0	(3)	0	0	0
Issuance of common stock pursuant to exercise of stock options (in shares)	386,974
Share repurchase	$ 0	0	(72)	0	0	(72)
Share repurchase (in shares)	(3,000)
Sale of treasury stock	$ 0	53	0	0	0	53
Sale of treasury stock (in shares)	0
Issuance of treasury stock pursuant to exercise of stock options	$ 0	(429)	0	0	0	(429)
Issuance of treasury stock pursuant to exercise of stock options (in shares)	0
Unrealized comprehensive gain (loss) on investments	$ 0	0	0	(95)	0	(95)
Share-based compensation	0	0	3,961	0	0	3,961
Net loss	0	0	0	0	(24,664)	(24,664)
Ending Balance at Mar. 31, 2020	$ 551	(429)	493,811	(75)	(207,807)	286,051
Ending Balance (in shares) at Mar. 31, 2020	55,157,035
Beginning Balance at Dec. 31, 2019	$ 548	(53)	489,925	20	(183,143)	307,297
Beginning Balance (in shares) at Dec. 31, 2019	54,773,061
Unrealized comprehensive gain (loss) on investments						209
Net loss						(49,704)
Ending Balance at Jun. 30, 2020	$ 551	0	498,590	229	(232,847)	266,523
Ending Balance (in shares) at Jun. 30, 2020	55,170,003
Beginning Balance at Mar. 31, 2020	$ 551	(429)	493,811	(75)	(207,807)	286,051
Beginning Balance (in shares) at Mar. 31, 2020	55,157,035
Issuance of common stock pursuant to exercise of stock options	$ 0	0	290	0	0	290
Issuance of common stock pursuant to exercise of stock options (in shares)	12,968
Sale of treasury stock	$ 0	538	0	0	0	538
Sale of treasury stock (in shares)	0
Issuance of treasury stock pursuant to exercise of stock options	$ 0	(109)	0	0	0	(109)
Issuance of treasury stock pursuant to exercise of stock options (in shares)	0
Unrealized comprehensive gain (loss) on investments	$ 0	0	0	304	0	304
Share-based compensation	0	0	4,489	0	0	4,489
Net loss	0	0	0	0	(25,040)	(25,040)
Ending Balance at Jun. 30, 2020	$ 551	0	498,590	229	(232,847)	266,523
Ending Balance (in shares) at Jun. 30, 2020	55,170,003
Beginning Balance at Dec. 31, 2020	$ 610	0	825,794	(42)	(322,843)	503,519
Beginning Balance (in shares) at Dec. 31, 2020	60,996,367
Issuance of common stock pursuant to exercise of stock options	$ 9	0	8,783	0	0	8,792
Issuance of common stock pursuant to exercise of stock options (in shares)	991,432
Unrealized comprehensive gain (loss) on investments	$ 0	0	0	(33)	0	(33)
Share-based compensation	0	0	7,900	0	0	7,900
Net loss	0	0	0	0	(40,179)	(40,179)
Ending Balance at Mar. 31, 2021	$ 619	0	842,477	(75)	(363,022)	479,999
Ending Balance (in shares) at Mar. 31, 2021	61,987,799
Beginning Balance at Dec. 31, 2020	$ 610	0	825,794	(42)	(322,843)	503,519
Beginning Balance (in shares) at Dec. 31, 2020	60,996,367
Unrealized comprehensive gain (loss) on investments						(39)
Net loss						(74,704)
Ending Balance at Jun. 30, 2021	$ 634	0	886,431	(81)	(397,547)	489,437
Ending Balance (in shares) at Jun. 30, 2021	63,448,069
Beginning Balance at Mar. 31, 2021	$ 619	0	842,477	(75)	(363,022)	479,999
Beginning Balance (in shares) at Mar. 31, 2021	61,987,799
Issuance of common stock pursuant to exercise of stock options	$ 2	0	1,113	0	0	1,115
Issuance of common stock pursuant to exercise of stock options (in shares)	133,838
Conversion of convertible securities into common shares	$ 13	0	35,530	0	0	35,543
Conversion of convertible securities into common shares (in shares)	1,326,432
Unrealized comprehensive gain (loss) on investments	$ 0	0	0	(6)	0	(6)
Share-based compensation	0	0	7,311	0	0	7,311
Net loss	0	0	0	0	(34,525)	(34,525)
Ending Balance at Jun. 30, 2021	$ 634	$ 0	$ 886,431	$ (81)	$ (397,547)	$ 489,437
Ending Balance (in shares) at Jun. 30, 2021	63,448,069

Consolidated Statements of Cash

Consolidated Statements of Cash Flows - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2021	Jun. 30, 2020
Operating Activities:
Net loss	$ (74,704)	$ (49,704)
Adjustments to reconcile net loss to net cash used in operating activities:
Accretion of discount on convertible notes	705	1,416
Depreciation and amortization expense	1,380	242
Write down of property and equipment	0	62
Stock-based compensation	15,211	8,450
Accretion of discount on investments, net	1,345	63
Unrealized loss on marketable securities	1	0
Changes in operating assets and liabilities:
Prepaid expenses and other assets	1,030	(2,522)
Accounts payable and accrued expenses	(8,982)	(3,188)
Operating lease liabilities	50	(94)
Finance lease liability	1,184	997
Other long term liabilities	(29)	0
Net cash used in operating activities	(62,809)	(44,278)
Investing activities:
Purchases of investments	(158,571)	(56,436)
Proceeds from maturities of investments	180,584	88,714
Payments made to acquire right of use asset	(61)	(2,731)
Purchases of property and equipment	(1,449)	(7,115)
Purchases of internal use software	(325)	(368)
Net cash provided by investing activities	20,178	22,064
Financing activities:
Issuance of common stock, pursuant to exercise of stock options	9,907	3
Issuance of common stock, net of issuance costs	0	287
Common stock repurchase	0	(72)
Proceeds from sale of treasury stock	0	591
Payment of withholding tax on option exercise	0	(538)
Convertible notes refinancing costs to the lender	0	(237)
Net cash provided by financing activities	9,907	34
Net change in cash, cash equivalents and restricted cash	(32,724)	(22,180)
Cash, cash equivalents and restricted cash at beginning of period	298,666	186,908
Cash, cash equivalents and restricted cash at end of period	265,942	164,728
Supplemental disclosure of non-cash financing and investing activities:
Accrued purchases of property and equipment	1,272	3,958
Accrued purchases of internal use software	122	122
Unrealized (loss) gain on investments	(39)	209
Conversion of convertible notes into common stock	35,544	0
Finance lease right of use asset and lease liability	0	20,179
Reclassification of construction in process (from) to finance right of use asset	(5)	26,465
Supplemental cash flow information:
Cash paid for interest	$ 1,347	$ 1,495

Nature of Business

Nature of Business	6 Months Ended
	Jun. 30, 2021
Nature of Business [Abstract]
Nature of Business	1. Nature of Business Rocket Pharmaceuticals, Inc. (“Rocket” or the “Company”) is a clinical-stage, multi-platform biotechnology company focused on the development of first, only and best-in-class gene therapies, with direct on-target mechanism of action and clear clinical endpoints, for rare and devastating diseases. Rocket has four ex vivo ” in vivo

Risks and Liquidity

Risks and Liquidity	6 Months Ended
	Jun. 30, 2021
Risks and Liquidity [Abstract]
Risks and Liquidity	2. Risks and Liquidity T he Company has not generated any revenue and has incurred losses since inception. The Company’s operations are subject to certain risks and uncertainties, including, among others, uncertainty of drug candidate development, technological uncertainty, uncertainty regarding patents and proprietary rights, the absence of 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 companie 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 as of June 30, 2021. As of June 30, 2021, the Company had of cash, cash equivalents and investments . During the year ended December 31, 2020, the Company received net proceeds of $ million from a public offering of shares of common stock. The Company expects such resources will be sufficient to fund its operating expenses and capital expenditure requirements into the second half of 2023. In April 2021, the Company called for the redemption of the remaining $ million principal balance of its , 2022 Convertible Senior Notes due 2022 which were converted into common stock (see Note 7). On August 2, 2021, holders of $ million of the 2021 Convertible Notes converted the $ million remaining balance of the 2021 Convertible Notes into common stock. As of August 2, 2021, ne 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.

Basis of Presentation, Principl

Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies	6 Months Ended
	Jun. 30, 2021
Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies [Abstract]
Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies	3. Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies Basis of Presentation The accompanying unaudited interim consolidated financial statements should be read in conjunction with the Company’s consolidated financial statements for the year ended December included in the Annual Report on Form -K filed with the Securities and Exchange Commission (“SEC”) on March (“ Form -K”). The unaudited interim consolidated financial statements have been prepared on the same basis as the audited annual financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for the fair statement of the Company’s consolidated financial position as of June and the results of its operations and its cash flows for the and six months ended June The financial data and other information disclosed in these consolidated notes related to the and six months ended June and are unaudited. The results for the and six months ended June are not necessarily indicative of results to be expected for the year ending December and any other interim periods or any future year or period. 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 (“U.S. GAAP”). All intercompany accounts have been eliminated in consolidation. Use of Estimates The preparation of the consolidated financial statements in conformity with U.S. 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 (“R&D”) 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 months or less from the date of purchase to be cash equivalents. The Company’s cash and cash equivalent accounts, at times, exceeds 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 finance and operating leases (see Note “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: June 30, 2021 December 31 2020 Cash and cash equivalents $ 264,608 $ 297,098 Restricted cash 1,334 1,568 $ 265,942 $ 298,666 Significant Accounting Policies The significant accounting policies used in the preparation of these consolidated financial statements for the months ended June are consistent with those disclosed in Note to the consolidated financial statements in the Form -K, except as noted below. 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 three and six months ended June 30, 2021, the Company recorded research and development incentive income of $0 and $0.5 million, respectively. During the three and six months ended June 30, 2020, the Company recorded research and development incentive income of $ . 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 Instrum

Fair Value of Financial Instruments	6 Months Ended
	Jun. 30, 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 June 30 2021 Level 1 Level 2 Level 3 Total Assets: Cash and cash equivalents: Money market mutual funds $ 221,680 $ - $ - $ 221,680 221,680 - - 221,680 Investments: United States Treasury securities 74,468 - - 74,468 Corporate Bonds - 72,435 - 72,435 Municipal Bonds - 6,000 - 6,000 Agency Bonds - 9,319 - 9,319 74,468 87,754 - 162,222 $ 296,148 $ 87,754 $ - $ 383,902 Fair Value Measurements as of December 31, 2020 Using: Level 1 Level 2 Level 3 Total Assets: Cash and 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 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 assets as these assets are not traded in an active market and have been valued through a -party pricing service based on quoted prices for similar assets. The fair value of the Convertible Notes and Convertible Notes as of June was and respectively (see Note The fair value of the 2021 Convertible Notes and 2022 Convertible Notes as of December 31, 2020 was $ million and $ million, respectively (see Note 7 .

Property and Equipment, Net

Property and Equipment, Net	6 Months Ended
	Jun. 30, 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: June 30, 2021 December 31, 2020 Laboratory equipment $ 10,401 $ 7,807 Machinery and equipment 10,012 9,933 Computer equipment 218 218 Furniture and fixtures 1,914 1,880 Leasehold improvements 39 29 Internal use software 1,832 1,385 24,416 21,252 Less: accumulated depreciation and amortization (3,427 ) (2,046 ) $ 20,989 $ 19,206 During the three and six months ended June 30, 2021, the Company recognized $0.7 million and $1.4 million of depreciation and amortization expense, respectively. During the three and six months ended June 30, 2020, the Company recognized $ million and $ million of depreciation and amortization expense, respectively

Accounts Payable and Accrued Ex

Accounts Payable and Accrued Expenses	6 Months Ended
	Jun. 30, 2021
Accounts Payable and Accrued Expenses [Abstract]
Accounts Payable and Accrued Expenses	6. Accounts Payable and Accrued Expenses At June 30, 2021 and December 31, 2020, the Company’s accounts payable and accrued expenses consisted of the following: June 30, December 31, 2021 2020 Research and development $ 11,251 $ 14,962 Property and equipment 1,272 1,456 Employee compensation 3,115 4,875 Accrued interest 123 1,122 Government grant payable 597 590 Professional fees 735 1,332 Internal use software 122 300 Other 669 835 $ 17,884 $ 25,472

Convertibles Notes

Convertibles Notes	6 Months Ended
	Jun. 30, 2021
Convertibles Notes [Abstract]
Convertibles Notes	7. Convertibles Notes 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 scheduled to mature on August 1, 2021 (the “Maturity Date”). The 2021 Convertible Notes were unsecured and accrue interest at a rate of 5.75% per annum and interest is 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. The Conversion Rate was subject to adjustment from time to time upon the occurrence of certain events, including the issuance of stock dividends and payment of cash dividends. The 2021 Convertible Notes are considered a hybrid financial instrument consisting of a fixed interest rate “host” and various embedded features that required evaluation as potential embedded derivatives under FASB ASC 815, Derivatives and Hedging On , holders of $ million of the Convertible Notes converted the $ million remaining balance of the Convertible Notes into shares. As of , ne of the Convertible Notes were outstanding. 2022 Convertible Notes On February 20, 2020, and , August 2022 Additionally, the Company repurchased shares of its common stock that have been retired for an aggregate amount of $ from certain holders of the Convertible Notes participating in the exchange transactions in privately negotiated, private transactions. Also pursuant to the Exchange Agreements, on , the Company exchanged $ million aggregate principal amount of the Convertible Notes for (a) $ million aggregate principal amount of its newly issued Convertible Senior Notes due (an exchange ratio equal to Convertible Notes per exchanged Convertible Notes) and (b) approximately $ to pay the accrued and unpaid interest on the exchanged Convertible Notes from, and including, , to, but excluding, the closing date of the exchange transaction, adjusted to take into account the unearned accrued interest on the Convertible Notes from, and including, , to, but excluding, the closing date of the exchange transaction. The Convertible Notes were issued in a private placement exempt from registration in reliance on Section (a)( ) of the Securities Act of , as amended (the “Securities Act”). The Convertible Notes issued in the exchange transaction were issued as additional notes pursuant to the Indenture, dated as of , between the Company and Wilmington Trust, National Association, as trustee, as supplemented by the Supplemental Indenture, dated as of , governing the Convertible Notes. 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 and was subject to adjustment under the terms of the 2022 Convertible Notes. 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 (whether or not consecutive) during any 30 consecutive trading day period (including the last trading day of such period) ending within the five 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 balance 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 , the Company redeemed in full the Convertible Notes prior to the redemption date. Holders of approximately $ remaining million principal amount of the Convertible Notes converted such notes in accordance with the terms of the Exchange Agreements into approximately million shares of the Company’s common stock and cash in lieu of fractional shares. In accordance with ASC - Debt , the settlement of the Convertible Notes is accounted for as a conversion since the Convertible Notes did not include a beneficial conversion feature and the carrying amount of the Convertible Notes, including any unamortized premium or discount, was credited to additional paid in capital upon conversion to reflect the common stock issued and no gain or loss is recognized. The principal amount of $ million and any unamortized discount was recorded to additional paid in capital upon redemption. The table below summarizes the carrying value of the 2021 Convertible Notes as of June 30, 2021 and December 31, 2020: 2021 Notes June 30, 2021 December 31, 2020 Principal amount $ 5,150 $ 5,150 Discount (48 ) (275 ) Carrying value $ 5,102 $ 4,875 Accretion of the Convertible Notes discount was $ million and $ million for the and months ended , respectively. Accretion of the Convertible Notes discount was $ million and $ million for the and months ended , respectively. Accretion of the Convertible Notes discount was $ and $ million for the and months ended , respectively. Accretion of the Convertible Notes discount was $ million and $ million for the and months ended , respectively

Stock Based Compensation

Stock Based Compensation	6 Months Ended
	Jun. 30, 2021
Stock Based Compensation [Abstract]
Stock Based Compensation	8. Stock Based Compensation 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: Six June 30 2021 2020 Risk-free interest rate 0.74 % 1.27 % Expected term (in years) 5.84 5.84 Expected volatility 69.19 % 77.39 % Expected dividend yield 0.00 % 0.00 % Exercise price $ 57.18 $ 21.56 Fair value of common stock $ 57.18 $ 21.56 The following table summarizes stock option activity for the six months ended June 30, 2021 under the Second Amended and Restated 2014 Stock Option and Incentive Plan: Number of Shares Weighted Average Exercise Price Weighted Average Contractual Term (Years) Aggregate Intrinsic Value Outstanding as of December 31, 2020 11,050,931 $ 9.10 6.55 $ 504,079 Granted 1,243,509 57.18 9.32 Exercised (1,125,270 ) 8.76 53,345 Cancelled (128,473 ) 34.15 Outstanding as of June 30 2021 11,040,697 $ 14.27 6.40 $ 346,476 Options vested and exercisable as of June 30 2021 8,127,520 $ 6.27 5.43 $ 308,590 Options unvested as of June 30 2021 2,913,177 $ 36.57 9.11 The weighted average grant-date fair value per share of stock options granted during the six months ended June 30, 2021 and 2020 was $34.64 and $14.29, respectively. The total fair value of options vested during the six months ended June 30, 2021 and 2020 was $13.2 million and $30.0 million, respectively. Stock-Based Compensation Stock-based compensation expense recognized by award type was as follows: Three Months Ended June 30, Six June 30 2021 2020 2021 2020 Stock options $ 7,215 $ 4,489 $ 15,041 $ 8,450 Restricted stock units 96 - 170 - Total share based compensation expense $ 7,311 $ 4,489 $ 15,211 $ 8,450 Stock-based compensation expense by classification included within the consolidated statements of operations and comprehensive loss was as follows: Three Months Ended June 30, Six Months Ended June 30, 2021 2020 2021 2020 Research and development $ 3,148 $ 1,679 $ 6,064 $ 3,414 General and administrative 4,163 2,810 9,147 5,036 Total share based compensation expense $ 7,311 $ 4,489 $ 15,211 $ 8,450 Restricted Stock The following table summarizes the Company's RSU activity for the six months ended June 30, 2021: Number of Shares Weighted Average Grant Date Fair Value Unvested as of December 31, 2020 20,000 $ 25.06 Granted 3,500 62.32 Unvested as of June 30, 2021 23,500 30.61 As of June 30, 2021, the Company had an aggregate of $57.0 million of unrecognized stock-based compensation expense related to both stock options and Restricted Stock Unit grants

Warrants

Warrants	6 Months Ended
	Jun. 30, 2021
Warrants [Abstract]
Warrants	9. Warrants As of June 30, 2021 and December 31, 2020, the Company had warrants outstanding convertible into and shares of common stock at exercise price of $ and $ per share, respectively, which warrants will expire on and , respectively.

Net Loss Per Share

Net Loss Per Share	6 Months Ended
	Jun. 30, 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: Three Months Ended June 30, Six Months Ended June 30, 2021 2020 2021 2020 Numerator: Net loss attributable to common stockholders $ (34,525 ) $ (25,040 ) $ (74,704 ) $ (49,704 ) Denominator: Weighted-average common shares outstanding - basic and diluted 63,061,232 55,158,459 62,321,926 55,020,789 Net loss per share attributable to common stockholders $ (0.55 ) $ (0.45 ) $ (1.20 ) $ (0.90 ) 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: Three and Six Months Ended June 30, 2021 2020 Shares issuable upon conversion of the 2021 Convertible Notes 160,536 160,536 Shares issuable upon conversion of the 2022 Convertible Notes - 1,460,412 Warrants exercisable for common shares 610,437 14,102 Restricted stock units exercisable for common shares 23,500 - Options to purchase common shares 11,040,697 10,776,813 11,835,170 12,411,863

Commitments and Contingencies

Commitments and Contingencies	6 Months Ended
	Jun. 30, 2021
Commitments and Contingencies [Abstract]
Commitments and Contingencies	11. 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). 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. 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 NJ Lease Agreement is estimated to be approximately $29.3 million over the 15-year term of the NJ Lease Agreement. 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 June 30, 2021 and December 31, 2020. 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 in March 2020, 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 o f . During the months ended , the Company reclassified an additional $ million bringing the aggregate reclassification to $ million of construction costs in progress as part of the right of use asset Interest associated with the financing lease was $ million and $ million for the three and six months ended June 30, 2021, respectively. Interest associated with the financing lease was $ million and $ million for the three and six months ended June 30, 2020, respectively. This is recorded as interest expense on the consolidated statements of operations. Operating Leases 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 . 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 June 30, 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 Rental income received under the sublease agreement totaled $ million and $ million for the three and six months ended June 30, 2021, respectively. Rental income received under the same agreement totaled $ million and $ million for the three and six months ended June 30, 2020, respectively. Rental income is netted against rent expense in the consolidated statement of operations. Rent expense was $ million and $ million for the three and six months ended June 30, 2021, respectively. Rent expense was $ million and $ million for the three and six months ended June 30, 2020, respectively. The total restricted cash balance for the Company’s operating and finance leases at and was $ million and $ million, respectively. Lease cost June 30, 2021 Operating lease cost $ 275 Finance lease cost Amortization of right of use assets 1,071 Interest on lease liablities 920 Total lease cost $ 2,266 The following table summarizes the maturity of the Company’s operating and finance lease liabilities on an undiscounted cash flow basis and a reconciliation to the operating and finance lease liabilities as of June 30, 2021: Maturity of operating lease liabilities June 30, 2021 2021 $ 428 2022 860 2023 488 2024 207 Total lease payments $ 1,983 Less: interest (95 ) Total operating lease liabilities $ 1,888 Maturity of finance lease liability June 30, 2021 2021 $ 827 2022 1,689 2023 1,736 2024 1,791 2025 1,856 Thereafter 46,913 Total lease payments $ 54,812 Less: interest (34,076 ) Total finance lease liability $ 20,736 Leases June 30, 2021 Operating right-of-use assets $ 1,628 Operating current lease liabilities 792 Operating noncurrent lease liabilities 1,096 Total operating lease liabilities $ 1,888 Finance right-of-use assets $ 49,507 Finance current lease liability 1,666 Finance noncurrent lease liability 19,070 Total finance lease liability $ 20,736 Other information Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows from operating leases $ 225 Cash flows from finance lease $ 816 Weighted-average remaining lease term - operating leases 2.5 Weighted-average remaining lease term - finance lease 23.2 Weighted-average discount rate - operating leases 4.75 % Weighted-average discount rate - finance lease 8.96 % Litigation From time to time, the Company may be subject to other 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 Intellect

Agreements Related to Intellectual Property	6 Months Ended
	Jun. 30, 2021
Agreements Related to Intellectual Property [Abstract]
Agreements Related to Intellectual Property	12. Agreements Related to Intellectual Property The Company 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.

CIRM Grants

CIRM Grants	6 Months Ended
	Jun. 30, 2021
CIRM Grants [Abstract]
CIRM Grants	13. 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 gene therapy for LAD-I. Proceeds from the grant will help fund clinical trial costs as well as manufactured drug product for Phase I/II 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 , 2020 , the Company met the next CIRM milestone and recorded a receivable, included in prepaid and other assets in the consolidated balance sheet, and a reduction of research and development expenses of $ million 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 lentiviral vector (LVV)-based gene therapy, RP-L401, for the treatment of IMO. The Company received a $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 three and six months ended June 30, 2021.

Related Party Transactions

Related Party Transactions	6 Months Ended
	Jun. 30, 2021
Related Party Transactions [Abstract]
Related Party Transactions	14. Related Party Transactions During April 2018 , the Company incurred expenses of $ and $ during the three and six months ended June 30, 2021 and 2020, relating to services provided under this agreement In , 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 term, the Company granted the board member options to purchase options with a fair value of $ million. In , the Company entered into a consulting agreement with the spouse of of the Company’s executive officers for information technology advisory services. In exchange for the services provided under the agreement, the Company granted restricted stock units which vest over a period. In December 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. There were no warrants issued for the six months ended June 30, 2021.

401(k) Savings Plan

401(k) Savings Plan	6 Months Ended
	Jun. 30, 2021
401(k) Savings Plan [Abstract]
401(k) Savings Plan	15. 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 the safe harbor match of of employee contributions to the Plan, subject to certain limitations. The Company’s matching contribution for the three and six months ended June 30, 2021 was $ million and $ million, respectively. The Company’s matching contribution for the three and six months ended June 30, 2020 was $ million and $ million, respectively.

Subsequent Events

Subsequent Events	6 Months Ended
	Jun. 30, 2021
Subsequent Events [Abstract]
Subsequent Events	16. Subsequent Events On August 2, 2021, holders of $ million of the 2021 Convertible Notes converted the $ million remaining balance of the 2021 Convertible Notes into common shares. As of August 2, 2021, of the 2021 Convertible Notes were outstanding. On August 9, 2021, the Company issued a warrant exercisable for shares of common stock to a related party for business development and asset identification consulting services.

Basis of Presentation, Princi_2

Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies (Policies)	6 Months Ended
	Jun. 30, 2021
Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies [Abstract]
Basis of Presentation	Basis of Presentation The accompanying unaudited interim consolidated financial statements should be read in conjunction with the Company’s consolidated financial statements for the year ended December included in the Annual Report on Form -K filed with the Securities and Exchange Commission (“SEC”) on March (“ Form -K”). The unaudited interim consolidated financial statements have been prepared on the same basis as the audited annual financial statements and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary for the fair statement of the Company’s consolidated financial position as of June and the results of its operations and its cash flows for the and six months ended June The financial data and other information disclosed in these consolidated notes related to the and six months ended June and are unaudited. The results for the and six months ended June are not necessarily indicative of results to be expected for the year ending December and any other interim periods or any future year or period.
Principles of Consolidation	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 (“U.S. GAAP”). All intercompany accounts have been eliminated in consolidation.
Use of Estimates	Use of Estimates The preparation of the consolidated financial statements in conformity with U.S. 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 (“R&D”) 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 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 months or less from the date of purchase to be cash equivalents. The Company’s cash and cash equivalent accounts, at times, exceeds 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 finance and operating leases (see Note “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: June 30, 2021 December 31 2020 Cash and cash equivalents $ 264,608 $ 297,098 Restricted cash 1,334 1,568 $ 265,942 $ 298,666
Significant Accounting Policies	Significant Accounting Policies The significant accounting policies used in the preparation of these consolidated financial statements for the months ended June are consistent with those disclosed in Note to the consolidated financial statements in the Form -K, except as noted below.
NYS Life Sciences Research and Development Tax Credit	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 three and six months ended June 30, 2021, the Company recorded research and development incentive income of $0 and $0.5 million, respectively. During the three and six months ended June 30, 2020, the Company recorded research and development incentive income of $ .
Recent Accounting Pronouncements	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

Basis of Presentation, Princi_3

Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies (Tables)	6 Months Ended
	Jun. 30, 2021
Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies [Abstract]
Cash, Cash Equivalents and Restricted Cash	Restricted cash consists of deposits collateralizing letters of credit issued by a bank in connection with the Company’s finance and operating leases (see Note “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: June 30, 2021 December 31 2020 Cash and cash equivalents $ 264,608 $ 297,098 Restricted cash 1,334 1,568 $ 265,942 $ 298,666

Fair Value of Financial Instr_2

Fair Value of Financial Instruments (Tables)	6 Months Ended
	Jun. 30, 2021
Fair Value of Financial Instruments [Abstract]
Fair Value of Financial Instruments Measured on Recurring Basis	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 June 30 2021 Level 1 Level 2 Level 3 Total Assets: Cash and cash equivalents: Money market mutual funds $ 221,680 $ - $ - $ 221,680 221,680 - - 221,680 Investments: United States Treasury securities 74,468 - - 74,468 Corporate Bonds - 72,435 - 72,435 Municipal Bonds - 6,000 - 6,000 Agency Bonds - 9,319 - 9,319 74,468 87,754 - 162,222 $ 296,148 $ 87,754 $ - $ 383,902 Fair Value Measurements as of December 31, 2020 Using: Level 1 Level 2 Level 3 Total Assets: Cash and 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 (Ta

Property and Equipment, Net (Tables)	6 Months Ended
	Jun. 30, 2021
Property and Equipment, Net [Abstract]
Property and Equipment	The Company’s property and equipment consisted of the following: June 30, 2021 December 31, 2020 Laboratory equipment $ 10,401 $ 7,807 Machinery and equipment 10,012 9,933 Computer equipment 218 218 Furniture and fixtures 1,914 1,880 Leasehold improvements 39 29 Internal use software 1,832 1,385 24,416 21,252 Less: accumulated depreciation and amortization (3,427 ) (2,046 ) $ 20,989 $ 19,206

Accounts Payable and Accrued _2

Accounts Payable and Accrued Expenses (Tables)	6 Months Ended
	Jun. 30, 2021
Accounts Payable and Accrued Expenses [Abstract]
Accounts Payable and Accrued Expenses	At June 30, 2021 and December 31, 2020, the Company’s accounts payable and accrued expenses consisted of the following: June 30, December 31, 2021 2020 Research and development $ 11,251 $ 14,962 Property and equipment 1,272 1,456 Employee compensation 3,115 4,875 Accrued interest 123 1,122 Government grant payable 597 590 Professional fees 735 1,332 Internal use software 122 300 Other 669 835 $ 17,884 $ 25,472

Convertibles Notes (Tables)

Convertibles Notes (Tables)	6 Months Ended
	Jun. 30, 2021
2021 Convertible Notes [Member]
Debt Instrument [Line Items]
Carrying Value of Convertible Notes	The table below summarizes the carrying value of the 2021 Convertible Notes as of June 30, 2021 and December 31, 2020: 2021 Notes June 30, 2021 December 31, 2020 Principal amount $ 5,150 $ 5,150 Discount (48 ) (275 ) Carrying value $ 5,102 $ 4,875

Stock Based Compensation (Table

Stock Based Compensation (Tables)	6 Months Ended
	Jun. 30, 2021
Stock Based Compensation [Abstract]
Weighted-Average Assumptions for Stock Options	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: Six June 30 2021 2020 Risk-free interest rate 0.74 % 1.27 % Expected term (in years) 5.84 5.84 Expected volatility 69.19 % 77.39 % Expected dividend yield 0.00 % 0.00 % Exercise price $ 57.18 $ 21.56 Fair value of common stock $ 57.18 $ 21.56
Stock Option Activity	The following table summarizes stock option activity for the six months ended June 30, 2021 under the Second Amended and Restated 2014 Stock Option and Incentive Plan: Number of Shares Weighted Average Exercise Price Weighted Average Contractual Term (Years) Aggregate Intrinsic Value Outstanding as of December 31, 2020 11,050,931 $ 9.10 6.55 $ 504,079 Granted 1,243,509 57.18 9.32 Exercised (1,125,270 ) 8.76 53,345 Cancelled (128,473 ) 34.15 Outstanding as of June 30 2021 11,040,697 $ 14.27 6.40 $ 346,476 Options vested and exercisable as of June 30 2021 8,127,520 $ 6.27 5.43 $ 308,590 Options unvested as of June 30 2021 2,913,177 $ 36.57 9.11
Stock-Based Compensation Expense by Award Type	Stock-based compensation expense recognized by award type was as follows: Three Months Ended June 30, Six June 30 2021 2020 2021 2020 Stock options $ 7,215 $ 4,489 $ 15,041 $ 8,450 Restricted stock units 96 - 170 - Total share based compensation expense $ 7,311 $ 4,489 $ 15,211 $ 8,450
Stock-Based Compensation Expense	Stock-based compensation expense by classification included within the consolidated statements of operations and comprehensive loss was as follows: Three Months Ended June 30, Six Months Ended June 30, 2021 2020 2021 2020 Research and development $ 3,148 $ 1,679 $ 6,064 $ 3,414 General and administrative 4,163 2,810 9,147 5,036 Total share based compensation expense $ 7,311 $ 4,489 $ 15,211 $ 8,450
RSU Activity	The following table summarizes the Company's RSU activity for the six months ended June 30, 2021: Number of Shares Weighted Average Grant Date Fair Value Unvested as of December 31, 2020 20,000 $ 25.06 Granted 3,500 62.32 Unvested as of June 30, 2021 23,500 30.61

Net Loss Per Share (Tables)

Net Loss Per Share (Tables)	6 Months Ended
	Jun. 30, 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: Three Months Ended June 30, Six Months Ended June 30, 2021 2020 2021 2020 Numerator: Net loss attributable to common stockholders $ (34,525 ) $ (25,040 ) $ (74,704 ) $ (49,704 ) Denominator: Weighted-average common shares outstanding - basic and diluted 63,061,232 55,158,459 62,321,926 55,020,789 Net loss per share attributable to common stockholders $ (0.55 ) $ (0.45 ) $ (1.20 ) $ (0.90 )
Antidilutive Securities Excluded from Computation of Diluted Net Loss Per Share	The Company excluded the following potential shares of common stock, presented based on amounts outstanding at each period end, from the computation of diluted net loss per share attributable to common stockholders for the periods indicated because including them would have had an anti-dilutive effect: Three and Six Months Ended June 30, 2021 2020 Shares issuable upon conversion of the 2021 Convertible Notes 160,536 160,536 Shares issuable upon conversion of the 2022 Convertible Notes - 1,460,412 Warrants exercisable for common shares 610,437 14,102 Restricted stock units exercisable for common shares 23,500 - Options to purchase common shares 11,040,697 10,776,813 11,835,170 12,411,863

Commitments and Contingencies (

Commitments and Contingencies (Tables)	6 Months Ended
	Jun. 30, 2021
Commitments and Contingencies [Abstract]
Lease Cost	Lease cost June 30, 2021 Operating lease cost $ 275 Finance lease cost Amortization of right of use assets 1,071 Interest on lease liablities 920 Total lease cost $ 2,266
Maturities of Operating Lease Liabilities	The following table summarizes the maturity of the Company’s operating and finance lease liabilities on an undiscounted cash flow basis and a reconciliation to the operating and finance lease liabilities as of June 30, 2021: Maturity of operating lease liabilities June 30, 2021 2021 $ 428 2022 860 2023 488 2024 207 Total lease payments $ 1,983 Less: interest (95 ) Total operating lease liabilities $ 1,888
Maturity of Finance Lease Liability	Maturity of finance lease liability June 30, 2021 2021 $ 827 2022 1,689 2023 1,736 2024 1,791 2025 1,856 Thereafter 46,913 Total lease payments $ 54,812 Less: interest (34,076 ) Total finance lease liability $ 20,736
Balance Sheet Information Related to Leases	Leases June 30, 2021 Operating right-of-use assets $ 1,628 Operating current lease liabilities 792 Operating noncurrent lease liabilities 1,096 Total operating lease liabilities $ 1,888 Finance right-of-use assets $ 49,507 Finance current lease liability 1,666 Finance noncurrent lease liability 19,070 Total finance lease liability $ 20,736
Lease Related to Cash Flow Information, Lease Term and Discount Rate	Other information Cash paid for amounts included in the measurement of lease liabilities: Operating cash flows from operating leases $ 225 Cash flows from finance lease $ 816 Weighted-average remaining lease term - operating leases 2.5 Weighted-average remaining lease term - finance lease 23.2 Weighted-average discount rate - operating leases 4.75 % Weighted-average discount rate - finance lease 8.96 %

Nature of Business (Details)

Nature of Business (Details)	6 Months Ended
	Jun. 30, 2021Program
Nature of Business [Abstract]
Number of clinical-stage programs	4

Risks and Liquidity (Details)

Risks and Liquidity (Details) - USD ($) $ in Thousands	Aug. 02, 2021	Apr. 26, 2021	Jun. 30, 2021	Jun. 30, 2020	Dec. 31, 2020	Mar. 31, 2021	Jun. 12, 2020	Feb. 20, 2020	Jan. 04, 2018
Risks and Liquidity [Abstract]
Accumulated deficit			$ (397,547)		$ (322,843)
Cash, cash equivalents and investments			426,800
Shareholders' Equity Disclosure [Abstract]
Conversion of convertible notes into common stock			$ 35,544	$ 0
2022 Convertible Notes [Member]
Shareholders' Equity Disclosure [Abstract]
Redemption of principal balance						$ 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
2022 Convertible Notes [Member] | Subsequent Event [Member]
Shareholders' Equity Disclosure [Abstract]
Outstanding amount	$ 0
2021 Convertible Notes [Member]
Shareholders' Equity Disclosure [Abstract]
Redemption of principal balance			$ 5,150		5,150		$ 7,500		$ 52,000
Convertible senior notes, interest rate, stated percentage			5.75%				6.25%
Debt instrument maturity date			Aug. 1, 2021
2021 Convertible Notes [Member] | Subsequent Event [Member]
Shareholders' Equity Disclosure [Abstract]
Conversion of convertible notes into common stock	5,150
Outstanding amount	$ 0
IPO [Member] | Common Stock [Member]
Shareholders' Equity Disclosure [Abstract]
Net proceeds, offering amount					$ 280,800
Common stock shares issued (in shares)					5,339,286

Basis of Presentation, Princi_4

Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2021	Jun. 30, 2020	Jun. 30, 2021	Jun. 30, 2020	Dec. 31, 2020	Dec. 31, 2019
Cash, Cash Equivalents and Restricted Cash [Abstract]
Cash and cash equivalents	$ 264,608		$ 264,608		$ 297,098
Restricted cash	1,334		1,334		1,568
Total	265,942	$ 164,728	265,942	$ 164,728	$ 298,666	$ 186,908
NYS Life Sciences Research and Development Tax Credit [Abstract]
Research and development incentive income	$ 0	$ 0	$ 500	$ 0

Fair Value of Financial Instr_3

Fair Value of Financial Instruments (Details) - USD ($) $ in Thousands	Jun. 30, 2021	Dec. 31, 2020
2021 Convertible Notes [Member]
Additional Information in Fair Value Disclosure [Abstract]
Fair value convertible note	$ 7,500	$ 9,500
2022 Convertible Notes [Member]
Additional Information in Fair Value Disclosure [Abstract]
Fair value convertible note	0	65,600
Recurring [Member]
Assets [Abstract]
Cash and cash equivalents	221,680	264,325
Investments	162,222	185,621
Fair value of financial instruments	383,902	449,946
Recurring [Member] | Money Market Mutual Funds [Member]
Assets [Abstract]
Cash and cash equivalents	221,680	193,312
Recurring [Member] | United States Treasury Securities [Member]
Assets [Abstract]
Cash and cash equivalents		62,497
Investments	74,468	112,328
Recurring [Member] | Corporate Bonds [Member]
Assets [Abstract]
Cash and cash equivalents		501
Investments	72,435	63,710
Recurring [Member] | Municipal Bonds [Member]
Assets [Abstract]
Investments	6,000	6,000
Recurring [Member] | Agency Bonds [Member]
Assets [Abstract]
Cash and cash equivalents		8,015
Investments	9,319	3,583
Recurring [Member] | Level 1 [Member]
Assets [Abstract]
Cash and cash equivalents	221,680	255,809
Investments	74,468	112,328
Fair value of financial instruments	296,148	368,137
Recurring [Member] | Level 1 [Member] | Money Market Mutual Funds [Member]
Assets [Abstract]
Cash and cash equivalents	221,680	193,312
Recurring [Member] | Level 1 [Member] | United States Treasury Securities [Member]
Assets [Abstract]
Cash and cash equivalents		62,497
Investments	74,468	112,328
Recurring [Member] | Level 1 [Member] | Corporate Bonds [Member]
Assets [Abstract]
Cash and cash equivalents		0
Investments	0	0
Recurring [Member] | Level 1 [Member] | Municipal Bonds [Member]
Assets [Abstract]
Investments	0	0
Recurring [Member] | Level 1 [Member] | Agency Bonds [Member]
Assets [Abstract]
Cash and cash equivalents		0
Investments	0	0
Recurring [Member] | Level 2 [Member]
Assets [Abstract]
Cash and cash equivalents	0	8,516
Investments	87,754	73,293
Fair value of financial instruments	87,754	81,809
Recurring [Member] | Level 2 [Member] | Money Market Mutual Funds [Member]
Assets [Abstract]
Cash and cash equivalents	0	0
Recurring [Member] | Level 2 [Member] | United States Treasury Securities [Member]
Assets [Abstract]
Cash and cash equivalents		0
Investments	0	0
Recurring [Member] | Level 2 [Member] | Corporate Bonds [Member]
Assets [Abstract]
Cash and cash equivalents		501
Investments	72,435	63,710
Recurring [Member] | Level 2 [Member] | Municipal Bonds [Member]
Assets [Abstract]
Investments	6,000	6,000
Recurring [Member] | Level 2 [Member] | Agency Bonds [Member]
Assets [Abstract]
Cash and cash equivalents		8,015
Investments	9,319	3,583
Recurring [Member] | Level 3 [Member]
Assets [Abstract]
Cash and cash equivalents	0	0
Investments	0	0
Fair value of financial instruments	0	0
Recurring [Member] | Level 3 [Member] | Money Market Mutual Funds [Member]
Assets [Abstract]
Cash and cash equivalents	0	0
Recurring [Member] | Level 3 [Member] | United States Treasury Securities [Member]
Assets [Abstract]
Cash and cash equivalents		0
Investments	0	0
Recurring [Member] | Level 3 [Member] | Corporate Bonds [Member]
Assets [Abstract]
Cash and cash equivalents		0
Investments	0	0
Recurring [Member] | Level 3 [Member] | Municipal Bonds [Member]
Assets [Abstract]
Investments	0	0
Recurring [Member] | Level 3 [Member] | Agency Bonds [Member]
Assets [Abstract]
Cash and cash equivalents		0
Investments	$ 0	$ 0

Property and Equipment, Net (De

Property and Equipment, Net (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2021	Jun. 30, 2020	Jun. 30, 2021	Jun. 30, 2020	Dec. 31, 2020
Property and Equipment [Abstract]
Property and equipment, gross	$ 24,416		$ 24,416		$ 21,252
Less: accumulated depreciation and amortization	(3,427)		(3,427)		(2,046)
Property and equipment, net	20,989		20,989		19,206
Depreciation and amortization expense	700	$ 100	1,380	$ 242
Laboratory Equipment [Member]
Property and Equipment [Abstract]
Property and equipment, gross	10,401		10,401		7,807
Machinery and Equipment [Member]
Property and Equipment [Abstract]
Property and equipment, gross	10,012		10,012		9,933
Computer Equipment [Member]
Property and Equipment [Abstract]
Property and equipment, gross	218		218		218
Furniture and Fixtures [Member]
Property and Equipment [Abstract]
Property and equipment, gross	1,914		1,914		1,880
Leasehold Improvements [Member]
Property and Equipment [Abstract]
Property and equipment, gross	39		39		29
Internal use Software [Member]
Property and Equipment [Abstract]
Property and equipment, gross	$ 1,832		$ 1,832		$ 1,385

Accounts Payable and Accrued _3

Accounts Payable and Accrued Expenses (Details) - USD ($) $ in Thousands	Jun. 30, 2021	Dec. 31, 2020
Accounts Payable and Accrued Expenses [Abstract]
Research and development	$ 11,251	$ 14,962
Property and equipment	1,272	1,456
Employee compensation	3,115	4,875
Accrued interest	123	1,122
Government grant payable	597	590
Professional fees	735	1,332
Internal use software	122	300
Other	669	835
Total	$ 17,884	$ 25,472

Convertibles Notes (Details)

Convertibles Notes (Details)	Aug. 02, 2021USD ($)shares	Apr. 26, 2021USD ($)dshares	Feb. 20, 2020USD ($)$ / sharesshares	Jun. 30, 2021USD ($)$ / shares	Jun. 30, 2020USD ($)	Mar. 31, 2020USD ($)shares	Jun. 30, 2021USD ($)d$ / shares	Jun. 30, 2020USD ($)	Dec. 31, 2020USD ($)shares	Mar. 31, 2021USD ($)	Jun. 12, 2020USD ($)	Jan. 04, 2018USD ($)
Debt [Abstract]
Aggregate amount of common stock shares repurchased						$ 72,000
Conversion of convertible notes into common stock							$ 35,544,000	$ 0
Convertible notes converted							35,544,000	0
Principal amount recorded to additional paid in capital				$ 886,431,000			886,431,000		$ 825,794,000
Carrying value of convertible notes [Abstract]
Carrying value				$ 0			0		35,066,000
Accretion of debt discount							$ 705,000	1,416,000
Common Stock [Member]
Debt [Abstract]
Number of common stock shares repurchased (in shares) | shares						3,000
Aggregate amount of common stock shares repurchased						$ 0
2021 Convertible Notes [Member]
Debt [Abstract]
Debt instrument maturity date							Aug. 1, 2021
Convertible senior notes, interest rate, stated percentage				5.75%			5.75%				6.25%
Debt conversion ratio							31.1876
Conversion per principal amount of debt				$ 1,000			$ 1,000
Convertible Note exchange											1
Conversion price (in dollars per share) | $ / shares				$ 32.08			$ 32.08
Effective interest rate				15.30%			15.30%
Debt instrument, aggregate principal amount exchanged			$ 39,350,000								$ 7,500,000
Debt instrument, percentage of aggregate outstanding principal			76.00%
Accrued and unpaid interest			$ 100,000								11,000,000
Number of common stock shares repurchased (in shares) | shares			3,000
Aggregate amount of common stock shares repurchased			$ 71,670,000
Redemption of principal balance				$ 5,150,000			$ 5,150,000		5,150,000		7,500,000	$ 52,000,000
Carrying value of convertible notes [Abstract]
Principal amount				5,150,000			5,150,000		5,150,000		$ 7,500,000	$ 52,000,000
Discount				(48,000)			(48,000)		(275,000)
Carrying value				5,102,000			5,102,000		4,875,000
Accretion of debt discount				100,000	$ 200,000		$ 200,000	800,000
2021 Convertible Notes [Member] | Subsequent Event [Member]
Debt [Abstract]
Conversion of convertible notes into common stock	$ 5,150,000
Convertible notes converted	5,150,000
Convertible notes, outstanding	$ 0
2021 Convertible Notes [Member] | Subsequent Event [Member] | Common Stock [Member]
Debt [Abstract]
Convertible Notes converted into shares of common stock (in shares) | shares	160,614
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
Convertible Note exchange			1
Conversion price (in dollars per share) | $ / shares			$ 32.06
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
Number of trading days immediately preceding notice of redemption							5 days
Conversion of convertible notes into common stock, principal amount									8,500,000
Conversion of convertible notes into common stock		$ 38,400,000							7,600,000
Convertible notes converted		38,400,000							$ 7,600,000
Redemption of principal balance			$ 39,350,000							$ 38,400,000
Principal amount recorded to additional paid in capital		$ 38,400,000
Carrying value of convertible notes [Abstract]
Principal amount			$ 39,350,000							$ 38,400,000
Accretion of debt discount				$ 0	$ 500,000		$ 500,000	$ 600,000
2022 Convertible Notes [Member] | Common Stock [Member]
Debt [Abstract]
Convertible Notes converted into shares of common stock (in shares) | shares		1,300,000							298,562
2022 Convertible Notes [Member] | Subsequent Event [Member]
Debt [Abstract]
Convertible notes, outstanding	$ 0

Stock Based Compensation, Share

Stock Based Compensation, Share Option Valuation (Details) - Stock Options [Member] - USD ($) $ / shares in Units, $ in Thousands	6 Months Ended		12 Months Ended
	Jun. 30, 2021	Jun. 30, 2020	Dec. 31, 2020
Stock Option Activity [Roll Forward]
Outstanding at beginning of period (in shares)	11,050,931
Granted (in shares)	1,243,509
Exercised (in shares)	(1,125,270)
Cancelled (in shares)	(128,473)
Outstanding at end of period (in shares)	11,040,697		11,050,931
Options vested and exercisable at end of period (in shares)	8,127,520
Options unvested at ending of period (in shares)	2,913,177
Weighted Average Exercise Price [Abstract]
Outstanding at beginning of period (in dollars per share)	$ 9.10
Granted (in dollars per share)	57.18
Exercised (in dollars per share)	8.76
Cancelled (in dollars per share)	34.15
Outstanding at end of period (in dollars per share)	14.27		$ 9.10
Options vested and exercisable at end of period (in dollars per share)	6.27
Options unvested at ending (in dollars per share)	$ 36.57
Weighted-Average Remaining Contractual Term [Abstract]
Outstanding	6 years 4 months 24 days		6 years 6 months 18 days
Granted	9 years 3 months 25 days
Options vested and exercisable	5 years 5 months 4 days
Options unvested	9 years 1 month 9 days
Aggregate Intrinsic Value [Abstract]
Outstanding at beginning of period	$ 504,079
Exercised	53,345
Outstanding at end of period	346,476		$ 504,079
Options vested and exercisable	$ 308,590
Weighted average grant date fair value of shares granted (in dollars per share)	$ 34.64	$ 14.29
Total fair value of options vested	$ 13,200	$ 30,000
Employees and Directors [Member]
Weighted-Average Assumptions [Abstract]
Risk-free interest rate	0.74%	1.27%
Expected term	5 years 10 months 2 days	5 years 10 months 2 days
Expected volatility	69.19%	77.39%
Expected dividend yield	0.00%	0.00%
Exercise price (in dollars per share)	$ 57.18	$ 21.56
Fair value of common stock (in dollars per share)	$ 57.18	$ 21.56

Stock Based Compensation, Stock

Stock Based Compensation, Stock-Based Compensation (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2021	Jun. 30, 2020	Jun. 30, 2021	Jun. 30, 2020
Stock-Based Compensation Expense [Abstract]
Total share based compensation expense	$ 7,311	$ 4,489	$ 15,211	$ 8,450
Research and Development [Member]
Stock-Based Compensation Expense [Abstract]
Total share based compensation expense	3,148	1,679	6,064	3,414
General and Administrative [Member]
Stock-Based Compensation Expense [Abstract]
Total share based compensation expense	4,163	2,810	9,147	5,036
Stock Options [Member]
Stock-Based Compensation Expense [Abstract]
Total share based compensation expense	7,215	4,489	15,041	8,450
Restricted Stock Units [Member]
Stock-Based Compensation Expense [Abstract]
Total share based compensation expense	$ 96	$ 0	$ 170	$ 0

Stock Based Compensation, Restr

Stock Based Compensation, Restricted Stock Units (Details) $ / shares in Units, $ in Millions	6 Months Ended
	Jun. 30, 2021USD ($)$ / sharesshares
Stock-Based Compensation Expense [Abstract]
Weighted average period expected to recognize unrecognized share-based compensation cost	1 year 11 months 19 days
Stock Options [Member]
Stock-Based Compensation Expense [Abstract]
Unrecognized share-based compensation cost | $	$ 57
Restricted Stock Units (RSU) [Member]
Number of Shares [Roll Forward]
Unvested, beginning balance (in shares) | shares	20,000
Granted (in shares) | shares	3,500
Unvested, ending balance (in shares) | shares	23,500
Weighted Average Grant Date Fair Value [Abstract]
Unvested, beginning balance (in dollars per share) | $ / shares	$ 25.06
Granted (in dollars per share) | $ / shares	62.32
Unvested, ending balance (in dollars per share) | $ / shares	$ 30.61
Stock-Based Compensation Expense [Abstract]
Unrecognized share-based compensation cost | $	$ 57

Warrants (Details)

Warrants (Details) - $ / shares	Jun. 30, 2021	Dec. 31, 2020
Warrant One [Member]
Warrants [Abstract]
Exercise price of warrants (in dollars per share)	$ 24.82
Warrants expiry date	Jun. 28, 2023
Warrant One [Member] | Common Stock [Member]
Warrants [Abstract]
Number of common stock shares called by warrants (in shares)	7,051
Warrant Two [Member]
Warrants [Abstract]
Exercise price of warrants (in dollars per share)		$ 57.11
Warrants expiry date	Dec. 21, 2030
Warrant Two [Member] | Common Stock [Member]
Warrants [Abstract]
Number of common stock shares called by warrants (in shares)		603,386

Net Loss Per Share (Details)

Net Loss Per Share (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2021	Jun. 30, 2020	Jun. 30, 2021	Jun. 30, 2020
Numerator [Abstract]
Net loss attributable to common stockholders	$ (34,525)	$ (25,040)	$ (74,704)	$ (49,704)
Denominator [Abstract]
Weighted-average common shares outstanding - basic (in shares)	63,061,232	55,158,459	62,321,926	55,020,789
Weighted-average common shares outstanding - diluted (in shares)	63,061,232	55,158,459	62,321,926	55,020,789
Net loss per share attributable to common stockholders - basic (in dollars per share)	$ (0.55)	$ (0.45)	$ (1.20)	$ (0.90)
Net loss per share attributable to common stockholders - diluted (in dollars per share)	$ (0.55)	$ (0.45)	$ (1.20)	$ (0.90)
Weighted Average Number of Shares Outstanding Reconciliation [Abstract]
Warrants exercisable for common shares (in shares)	610,437	14,102	610,437	14,102
Restricted stock units exercisable for common shares (in shares)	23,500	0	23,500	0
Options to purchase common shares (in shares)	11,040,697	10,776,813	11,040,697	10,776,813
Weighted average number diluted shares outstanding (in shares)	11,835,170	12,411,863	11,835,170	12,411,863
Convertible Notes 2021 [Member]
Weighted Average Number of Shares Outstanding Reconciliation [Abstract]
Shares issuable upon conversion of Convertible Notes (in shares)	160,536	160,536	160,536	160,536
Convertible Notes 2022 [Member]
Weighted Average Number of Shares Outstanding Reconciliation [Abstract]
Shares issuable upon conversion of Convertible Notes (in shares)	0	1,460,412	0	1,460,412

Commitments and Contingencies,

Commitments and Contingencies, Finance Lease (Details) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2021USD ($)	Jun. 30, 2020USD ($)	Jun. 30, 2021USD ($)ft²LeaseAgreement	Jun. 30, 2020USD ($)	Dec. 31, 2020USD ($)	Mar. 15, 2020USD ($)
Finance Lease [Abstract]
Total lease payments	$ 54,812		$ 54,812
Cash security deposit	455		455		$ 455
Finance right-of-use assets	49,507		49,507		50,521
Finance lease, liability	$ 20,736		20,736
Finance lease, interest expense			$ 920
NJ Lease Agreement [Member]
Finance Lease [Abstract]
Area of lease | ft²			103,720
Term of finance lease agreement	15 years		15 years
Number of options to renew lease agreement | LeaseAgreement			2
Term of renewal of finance lease agreement	5 years		5 years
Estimated rent payments			$ 1,200
Percentage of annual increase in base rent			3.00%
Total lease payments	$ 29,300		$ 29,300
Cash security deposit	300		300		$ 300
Finance right-of-use assets						$ 47,700
Finance lease, liability						20,200
Reclassification of construction costs in progress to right-of-use assets	100		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	32,200		32,200
Finance lease, interest expense	$ 500	$ 500	$ 900	$ 600
AAV Current Good Manufacturing Practice (cGMP) [Member]
Finance Lease [Abstract]
Area of lease | ft²			50,000

Commitments and Contingencies_2

Commitments and Contingencies, Operating Leases (Details) - USD ($) $ in Thousands	Mar. 26, 2021	Jun. 30, 2021	Jun. 30, 2020	Jun. 30, 2021	Jun. 30, 2020	Dec. 31, 2020	Jun. 07, 2018
Operating Leases [Abstract]
Rent expense		$ 300	$ 300	$ 600	$ 500
Restricted cash		$ 1,334		$ 1,334		$ 1,568
ESB Lease Agreement [Member]
Operating Leases [Abstract]
Term of lease agreement		3 years		3 years
Letter of credit							$ 900
Lease expiration date				Aug. 29, 2024
Certificate of deposit		$ 800		$ 800		900
ESB Lease Agreement Amendment [Member]
Operating Leases [Abstract]
Letter of credit		800		$ 800
Lease expiration date	Jun. 30, 2024
Operating lease right of use asset, adjustment	$ 1,100
Inotek Lexington Massachusetts Lease Agreement [Member]
Operating Leases [Abstract]
Lease expiration date				Feb. 28, 2023
Rental income received under sublease agreement		100	$ 100	$ 200	$ 200
Operating and Finance Leases [Member]
Operating Leases [Abstract]
Restricted cash		$ 1,000		$ 1,000		$ 1,100

Commitments and Contingencies_3

Commitments and Contingencies, Details of Operating and Finance Lease (Details) - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2021	Dec. 31, 2020
Lease cost [Abstract]
Operating lease cost	$ 275
Finance lease cost [Abstract]
Amortization of right of use assets	1,071
Interest on lease liabilities	920
Total lease cost	2,266
Maturity of operating lease liabilities [Abstract]
2021	428
2022	860
2023	488
2024	207
Total lease payments	1,983
Less: interest	(95)
Total operating lease liabilities	1,888
Maturity of finance lease liability [Abstract]
2021	827
2022	1,689
2023	1,736
2024	1,791
2025	1,856
Thereafter	46,913
Total lease payments	54,812
Less: interest	(34,076)
Total finance lease liability	20,736
Lease assets and liabilities [Abstract]
Operating right-of-use assets	1,628	$ 914
Operating current lease liabilities	792	626
Operating noncurrent lease liabilities	1,096	498
Total operating lease liabilities	1,888
Finance right-of-use assets	49,507	50,521
Finance current lease liability	1,666	1,644
Finance noncurrent lease liability	19,070	$ 18,988
Total finance lease liability	20,736
Cash paid for amounts included in the measurement of lease liabilities [Abstract]
Operating cash flows from operating leases	225
Cash flows from finance lease	$ 816
Weighted-average remaining lease term - operating leases	2 years 6 months
Weighted-average remaining lease term - finance lease	23 years 2 months 12 days
Weighted-average discount rate - operating leases	4.75%
Weighted-average discount rate - finance lease	8.96%

CIRM Grants (Details)

CIRM Grants (Details) $ in Millions	Apr. 01, 2021USD ($)	Jan. 04, 2021USD ($)	Dec. 31, 2019USD ($)Grant	Jun. 30, 2021USD ($)	Dec. 31, 2020USD ($)	Nov. 12, 2020USD ($)	Apr. 30, 2019USD ($)
LAD-I CIRM Grant [Member]
CIRM Grants [Abstract]
Grant award for clinical development support							$ 6.5
Number of grants | Grant			2
Amount received from grant for eligible costs incurred			$ 1.2
Grant receivable included in prepaid and other assets				$ 1	$ 1.1
Milestone payments received	$ 1	$ 1.1
IMO CIRM Grant [Member]
CIRM Grants [Abstract]
Grant award for clinical development support						$ 3.7
Amount received from grant for eligible costs incurred		$ 1
Grant receivable included in prepaid and other assets				$ 0.1	$ 0.9

Related Party Transactions (Det

Related Party Transactions (Details) - USD ($)	1 Months Ended	3 Months Ended		6 Months Ended
	Oct. 31, 2020	Jun. 30, 2021	Jun. 30, 2020	Jun. 30, 2021	Jun. 30, 2020	Dec. 21, 2020
Restricted Stock Units [Member]
Related Party Transaction [Abstract]
Shares granted (in shares)				3,500
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
Warrants Issue, Shares		0		0
Consulting Agreement, Business Development Services [Member] | Common Stock [Member]
Related Party Transaction [Abstract]
Number of common stock shares called by warrants (in shares)						603,386
Member of the Board of Directors - One [Member]
Related Party Transaction [Abstract]
Business development consulting services expense (per quarter)				$ 27,500
Termination notice period for business development consulting services agreement				14 days
Related party expenses		$ 27,500	$ 27,500	$ 55,000	$ 55,000
Member of the Board of Directors - Two [Member]
Related Party Transaction [Abstract]
Term of consulting agreement				1 year
Number of options granted (in shares)				9,784
Fair value amount of options granted				$ 100,000
Spouse of Executive officer [Member] | Restricted Stock Units [Member]
Related Party Transaction [Abstract]
Shares granted (in shares)	10,000
Vesting period				3 years

401(k) Savings Plan (Details)

401(k) Savings Plan (Details) - USD ($) $ in Millions	3 Months Ended		6 Months Ended
	Jun. 30, 2021	Jun. 30, 2020	Jun. 30, 2021	Jun. 30, 2020
401(k) Savings Plan [Abstract]
Percentage of matching employee contributions			4.00%
Matching employee contributions	$ 0.1	$ 0.1	$ 0.3	$ 0.2

Subsequent Events (Details)

Subsequent Events (Details) - USD ($) $ in Thousands	Aug. 02, 2021	Jun. 30, 2021	Jun. 30, 2020	Aug. 09, 2021	Dec. 31, 2020	Jun. 12, 2020	Jan. 04, 2018
Convertible Notes [Abstract]
Convertible notes converted		$ 35,544	$ 0
2021 Convertible Notes [Member]
Convertible Notes [Abstract]
Redemption of principal balance		$ 5,150			$ 5,150	$ 7,500	$ 52,000
Subsequent Event [Member] | Common Stock [Member]
Convertible Notes [Abstract]
Number of common shares issuable pursuant to exercise of warrant (in shares)				301,291
Subsequent Event [Member] | 2021 Convertible Notes [Member]
Convertible Notes [Abstract]
Convertible notes converted	$ 5,150
Convertible notes, outstanding	$ 0
Subsequent Event [Member] | 2021 Convertible Notes [Member] | Common Stock [Member]
Convertible Notes [Abstract]
Common stock issued for convertible notes (in shares)	160,614