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Document and Entity Information - shares	6 Months Ended
Document and Entity Information - shares	Jun. 30, 2020	Aug. 03, 2020
Cover page.
Document Type	10-Q
Amendment Flag	false
Document Quarterly Report	true
Document Period End Date	Jun. 30, 2020
Current Fiscal Year End Date	--12-31
Document Transition Report	false
Entity Registrant Name	Rocket Pharmaceuticals, Inc.
Entity Incorporation, State or Country Code	DE
Entity File Number	001-36829
Entity Tax Identification Number	00-0000000
Entity Address, Address Line One	350 Fifth Avenue
Entity Address, Address Line Two	Suite 7530
Entity Address, City or Town	New York
Entity Address, State or Province	NY
Entity Address, Postal Zip Code	10118
City Area Code	646
Local Phone Number	440-9100
Entity Filer Category	Accelerated Filer
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Emerging Growth Company	true
Entity Ex Transition Period	true
Entity Shell Company	false
Entity Small Business	false
Entity Common Stock, Shares Outstanding		55,187,599
Document Fiscal Year Focus	2020
Document Fiscal Period Focus	Q2
Entity Central Index Key	0001281895
Title of 12(b) Security	Common Stock, $0.01 par value
Trading Symbol	RCKT
Security Exchange Name	NASDAQ

Consolidated Balance Sheets (Unaudited) - USD ($) $ in Thousands	Jun. 30, 2020	Dec. 31, 2019
Current assets:
Cash and cash equivalents	$ 163,203	$ 185,383
Investments	86,600	118,732
Prepaid expenses and other assets	5,633	3,639
Total current assets	255,436	307,754
Property and equipment, net	14,314	29,521
Goodwill	30,815	30,815
Restricted cash	1,525	1,525
Deposits	455	455
Operating lease right-of-use assets	1,270	2,051
Finance lease right-of-use asset	49,250	0
Total assets	353,065	372,121
Current liabilities:
Accounts payable and accrued expenses	18,245	17,352
Operating lease liabilities, current	819	957
Finance lease liabilities, current	1,621	0
Total current liabilities	20,685	18,309
Convertible notes, net of unamortized discount	46,228	45,049
Operating lease liabilities, non-current	706	1,443
Finance lease liabilities, non-current	18,900	0
Other liabilities	23	23
Total liabilities	86,542	64,824
Commitments and contingencies (Note 11)
Stockholders' equity:
Common stock, $0.01 par value, 120,000,000 shares authorized; 55,170,003 and 54,773,061 shares issued and 55,170,003 and 54,769,030 shares outstanding at June 30, 2020 and December 31, 2019, respectively	551	548
Treasury stock, at cost, 0 and 4,031 common shares at June 30, 2020 and December 31, 2019, respectively	0	(53)
Additional paid-in capital	498,590	489,925
Accumulated other comprehensive loss	229	20
Accumulated deficit	(232,847)	(183,143)
Total stockholders' equity	266,523	307,297
Total liabilities and stockholders' equity	353,065	372,121
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 (Unaudited) (Parenthetical) - $ / shares	Jun. 30, 2020	Dec. 31, 2019
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)	55,170,003	54,773,061
Common stock, shares outstanding (in shares)	55,170,003	54,769,030
Treasury stock, at cost (in shares)	0	4,031
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 Operations (Unaudited) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2020	Jun. 30, 2019	Jun. 30, 2020	Jun. 30, 2019
Consolidated Statements of Operations (Unaudited) [Abstract]
Revenue	$ 0	$ 0	$ 0	$ 0
Operating expenses:
Research and development	16,731	13,989	33,687	29,126
General and administrative	6,828	4,403	13,990	8,211
Total operating expenses	23,559	18,392	47,677	37,337
Loss from operations	(23,559)	(18,392)	(47,677)	(37,337)
Research and development incentives	0	0	0	250
Interest expense	(1,786)	(1,544)	(3,360)	(3,148)
Interest and other income net	429	941	1,395	1,542
Accretion of discount on investments	(124)	315	(62)	562
Net loss	$ (25,040)	$ (18,680)	$ (49,704)	$ (38,131)
Net loss per share attributable to common stockholders - basic and diluted (in dollars per share)	$ (0.45)	$ (0.38)	$ (0.90)	$ (0.81)
Weighted-average common shares outstanding - basic and diluted (in shares)	55,158,459	49,267,247	55,020,789	47,206,480

Consolidated Statements of Comprehensive Loss (Unaudited) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2020	Jun. 30, 2019	Jun. 30, 2020	Jun. 30, 2019
Consolidated Statements of Comprehensive Loss (Unaudited) [Abstract]
Net loss	$ (25,040)	$ (18,680)	$ (49,704)	$ (38,131)
Other comprehensive loss
Net unrealized gain on investments	304	233	209	271
Total comprehensive loss	$ (24,736)	$ (18,447)	$ (49,495)	$ (37,860)

- 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 Stockholders' Equity (Unaudited) - 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 pursuant to exercise of stock options	$ 0	0	0	0	0	0
Issuance of common stock pursuant to exercise of stock options (in shares)	19,701
Stock repurchase	$ 0	(725)	(2)	0	0	(727)
Stock repurchase (in shares)	0
Retirement of treasury stock	$ (1)	1,393	(1,392)	0	0	0
Retirement of treasury stock (in shares)	(100,000)
Unrealized comprehensive gain (loss) on investments	$ 0	0	0	38	0	38
Share-based compensation	0	0	3,180	0	0	3,180
Net loss	0	0	0	0	(19,451)	(19,451)
Ending Balance at Mar. 31, 2019	$ 451	0	302,039	(89)	(125,324)	177,077
Ending Balance (in shares) at Mar. 31, 2019	45,114,437
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
Unrealized comprehensive gain (loss) on investments						271
Net loss						(38,131)
Ending Balance at Jun. 30, 2019	$ 503	(344)	392,205	144	(144,004)	248,504
Ending Balance (in shares) at Jun. 30, 2019	50,332,435
Beginning Balance at Mar. 31, 2019	$ 451	0	302,039	(89)	(125,324)	177,077
Beginning Balance (in shares) at Mar. 31, 2019	45,114,437
Issuance of common stock, net of issuance costs	$ 52	0	86,028	0	0	86,080
Issuance of common stock, net of issuance costs (in shares)	5,175,000
Net exercise of options	$ 0	(344)	0	0	0	(344)
Issuance of common stock pursuant to exercise of stock options	$ 0	0	0	0	0	0
Issuance of common stock pursuant to exercise of stock options (in shares)	42,998
Unrealized comprehensive gain (loss) on investments	$ 0	0	0	233	0	233
Share-based compensation	0	0	4,138	0	0	4,138
Net loss	0	0	0	0	(18,680)	(18,680)
Ending Balance at Jun. 30, 2019	$ 503	(344)	392,205	144	(144,004)	248,504
Ending Balance (in shares) at Jun. 30, 2019	50,332,435
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
Stock repurchase	$ 0	0	(72)	0	0	(72)
Stock 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

Consolidated Statements of Cash Flows (Unaudited) - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2020	Jun. 30, 2019
Operating Activities:
Net loss	$ (49,704)	$ (38,131)
Adjustments to reconcile net loss to net cash used in operating activities:
Accretion of discount on convertible notes	1,416	1,733
Depreciation and amortization expense	242	204
Write down of property and equipment	62	0
Stock-based compensation	8,450	7,319
Accretion of discount on investments	63	(503)
Changes in operating assets and liabilities:
Prepaid expenses and other assets	(2,522)	(2,398)
Accounts payable and accrued expenses	(3,188)	(2,265)
Operating lease liabilities	(94)	(40)
Finance lease liability	997	0
Net cash used in operating activities	(44,278)	(34,081)
Investing activities:
Purchases of investments	(56,436)	(140,336)
Proceeds from maturities of investments	88,714	49,936
Payments made to acquire right of use asset	(2,731)	0
Purchases of property and equipment	(7,115)	(7,305)
Purchases of internal use software	(368)	0
Net cash provided by (used in) investing activities	22,064	(97,705)
Financing activities:
Proceeds from sale of treasury stock	591	0
Issuance of common stock, net of issuance costs	287	86,080
Issuance of common stock, pursuant to exercise of stock options	3	0
Common stock repurchase	(72)	(727)
Payment of withholding tax on option exercise	(538)	0
Convertible notes refinancing costs to the lender	(237)	0
Net cash provided by financing activities	34	85,353
Net change in cash, cash equivalents and restricted cash	(22,180)	(46,433)
Cash, cash equivalents and restricted cash at beginning of period	186,908	112,791
Cash, cash equivalents and restricted cash at end of period	164,728	66,358
Supplemental disclosure of non-cash financing and investing activities:
Accrued purchases of property and equipment	3,958	716
Accrued purchases of internal use software	122	0
Retirement of treasury stock	0	1,395
Net exercise of options	0	344
Unrealized gain on investments	209	271
Finance lease right of use asset and lease liability	20,179	0
Reclassification of construction in process of finance right of use asset	26,465	0
Supplemental cash flow information:
Cash paid for interest	$ 1,495	$ 1,495

Nature of Business

6 Months Ended

Jun. 30, 2020

Nature of Business [Abstract]

Nature of Business

1.	Nature of Business

Rocket Pharmaceuticals, Inc., together with its subsidiaries (collectively “Rocket”, the “Company” or “we”) 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. The Company has four clinical-stage ex vivo lentiviral vector (“LVV”) programs currently enrolling patients in the United States (“U.S.”) and Europe 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, Pyruvate Kinase Deficiency (“PKD”), a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia and Infantile Malignant Osteopetrosis (“IMO”), a genetic disorder characterized by increased bone density and bone mass secondary to impaired bone resorption. Of these, both the Phase 2 FA program and the Phase 1/2 LAD-I program are in registration-enabling studies in the U.S. and Europe. In addition, in the U.S. the Company 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. The Company has global commercialization and development rights to all of these product candidates under royalty-bearing license agreements. Additional work in the discovery stage for an FA CRISPR/CAS9 program as well as a gene therapy program for the less common FA subtypes C and G is ongoing.

Risks and Liquidity

6 Months Ended

Jun. 30, 2020

Risks and Liquidity [Abstract]

Risks and Liquidity

2.	Risks and Liquidity

The 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, lack of commercial manufacturing experience, a lack of marketing or sales capability or experience, dependency on key personnel, compliance with government regulations and the need to obtain additional financing. In addition, there are possible additional delays that could result from the COVID-19 situation. 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 five product candidates are in clinical development. 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 $232.8 million as of June 30, 2020. As of June 30, 2020, the Company had $249.8 million of cash, cash equivalents and investments. Rocket expects such resources will be sufficient to fund its operating expenses and capital expenditure requirements into the second quarter of 2022.

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, Principles of Consolidation and Summary of Significant Accounting Policies

6 Months Ended

Jun. 30, 2020

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 31, 2019 included in the Annual Report on Form 10-K filed with the Securities and Exchange Commission (“SEC”) on March 6, 2020 (“2019 Form 10-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 financial position as of June 30, 2020 and the results of its operations and its cash flows for the three and six months ended June 30, 2020 and 2019. The financial data and other information disclosed in these consolidated notes related to the three and six months ended June 30, 2020 and 2019 are unaudited. The results for the three and six months ended June 30, 2020 are not necessarily indicative of results to be expected for the year ending December 31, 2020 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.

Significant Accounting Policies

The significant accounting policies used in the preparation of these consolidated financial statements for the three and six months ended June 30, 2020 are consistent with those disclosed in Note 3 to the consolidated financial statements in the 2019 Form 10-K, except as noted below.

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 finance and operating leases (see Note 11 “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, 2020		December 31, 2019

Cash and cash equivalents	$	163,203	$	185,383
Restricted cash		1,525		1,525
	$	164,728	$	186,908

Recent Accounting Pronouncements

In August 2018, the FASB issued ASU 2018-13 (“ASU 2018-13”), Fair Value Measurement - Disclosure Framework (Topic 820). The updated guidance improves the disclosure requirements on fair value measurements. The updated guidance is effective for fiscal years, and interim periods within those fiscal years, beginning after December 15, 2019. Early adoption is permitted for any removed or modified disclosures. The Company adopted this guidance on January 1, 2020. The adoption of ASU 2018-13 had no impact on the Company as the Company does not have Level 3 investments.

There were no other recent accounting standards that impacted the Company for the three months ended June 30, 2020.

Fair Value of Financial Instruments

6 Months Ended

Jun. 30, 2020

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, 2020 Using:
	Level 1		Level 2		Level 3		Total
Assets:
Money market mutual funds (included in cash and cash equivalents)	$	143,298	$	-	$	-	$	143,298

United States Treasury securities		15,575		-		-		15,575
Corporate Bonds		-		63,069		-		63,069
Municipal Bonds		-		6,000		-		6,000
Agency Bonds		-		1,956		-		1,956
Investments		15,575		71,025		-		86,600

	$	158,873	$	71,025	$	-	$	229,898

	Fair Value Measurements as of December 31, 2019 Using:
	Level 1		Level 2		Level 3		Total
Assets:
Money market mutual funds (included in cash and cash equivalents)	$	72,114	$	-	$	-	$	72,114
Cash		7,542		-		-		7,542
		79,656		-		-		79,656

United States Treasury securities		75,464		-		-		75,464
Government Bonds		-		8,000		-		8,000
Corporate Bonds		-		29,268		-		29,268
Municipal Bonds		-		6,000		-		6,000
Investments		75,464		43,268		-		118,732

	$	155,120	$	43,268	$	-	$	198,388

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 Government, 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.

The fair value of the 2021 Convertible Notes as of June 30, 2020 was $5.4 million. The fair value of the 2022 Convertible Notes as of June 30, 2020 was $47.2 million (see Note 7).

Property and Equipment

6 Months Ended

Jun. 30, 2020

Property and Equipment [Abstract]

Property and Equipment

5.	Property and Equipment

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

	June 30, 2020			December 31, 2019
Laboratory equipment	$	12,691		$	1,910
Computer equipment		176			179
Furniture and fixtures		1,847			273
Leasehold improvements		29			29
Internal use software		712			226
Construction costs in progress		-			27,809
		15,455			30,426
Less: accumulated depreciation		(1,141	)		(905	)
	$	14,314		$	29,521

Construction costs in progress as of December 31, 2019 comprised costs associated with the build out of the Company’s research, manufacturing and office facility in Cranbury, NJ. See Note 11 “Commitments and Contingencies” for the treatment of construction costs as part of the right of use asset as of June 30, 2020. During the three and six months ended June 30, 2020, the Company recognized $0.1 million and $0.2 million of depreciation expense, respectively. During the three and six months ended June 30, 2019, the Company recognized $0.1 million and $0.2 million of depreciation expense, respectively.

Accounts Payable and Accrued Expenses

6 Months Ended

Jun. 30, 2020

Accounts Payable and Accrued Expenses [Abstract]

Accounts Payable and Accrued Expenses

6.	Accounts Payable and Accrued Expenses

At June 30, 2020 and December 31, 2019, the Company’s accounts payable and accrued expenses consisted of the following:

	June 30,		December 31,
	2020		2019
Research and development	$	9,166	$	7,418
Property and equipment		3,958		4,424
Employee compensation		2,072		2,588
Accrued interest		1,252		1,241
Government grant payable		576		562
Professional fees		784		553
Internal use software		122		226
Other		315		340
	$	18,245	$	17,352

Convertible Notes

6 Months Ended

Jun. 30, 2020

Convertible Notes [Abstract]

Convertible Notes

7.	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 principal amount of $52.0 million, (the “2021 Convertible Notes”). The 2021 Convertible Notes were issued in 2016 and mature on August 1, 2021 (the “Maturity Date”). The 2021 Convertible Notes are 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”) has 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 is 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 Company, at its option, may redeem for cash all or any portion of the 2021 Convertible Notes if the last reported sale price of a share of the Company’s common stock is equal to or greater than 200% of the Conversion Rate for the 2021 Convertible Notes 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 trading days immediately preceding the date on which the Company provides notice of redemption, at a redemption price equal to 100% of the principal amount of the 2021 Convertible Notes to be redeemed, plus accrued and unpaid interest to, but excluding, the redemption date.

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 (“ASC 815”). Based on the nature of the host instrument and the embedded features, management concluded that none of the conversion, put and redemption features required bifurcation and separate accounting from the host instrument. The Company determined that the Additional Interest was an embedded derivative that contains non-credit related events of default. As a result, the Additional Interest feature required bifurcation and separate accounting under ASC 815. Based on the amount of Additional Interest that would be owed and the likelihood of occurrence, the Company estimated the fair value of the Additional Interest feature to be insignificant upon issuance and as of June 30, 2020 and December 31, 2019. As of June 30, 2020, the stated interest rate was 5.75%, and the effective interest rate was 15.3%.

Convertible Notes Exchange Agreement

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 (representing approximately 76% of the aggregate outstanding 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”) (an exchange ratio equal to 1.00 2022 Convertible Note per exchanged 2021 Convertible Note) and (b) $119,416 in cash to pay the accrued and unpaid interest on the exchanged 2021 Convertible Notes from February 1, 2020, to February 20, 2020, the closing date of the February 20, 2020 exchange transactions. Additionally, the Company repurchased 3,000 shares of its common stock that have been retired for an aggregate amount of $71,670 from certain holders of the 2021 Convertible Notes participating in the exchange transactions in privately negotiated, private transactions.

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 (an exchange ratio equal to 1.00 2022 Convertible Notes per exchanged 2021 Convertible Notes) and (b) approximately $11,000 in cash to pay the accrued and unpaid interest on the exchanged 2021 Convertible Notes from, and including, February 1, 2020, to, but excluding, the closing date of the exchange transaction, adjusted to take into account the unearned accrued interest on the 2022 Convertible Notes from, and including, February 20, 2020, to, but excluding, the closing date of the exchange transaction.

The 2022 Convertible Notes were issued in a private placement exempt from registration in reliance on Section 4(a)(2) of the Securities Act of 1933, as amended (the “Securities Act”). The 2022 Convertible Notes issued in the exchange transaction were issued as additional notes pursuant to the Indenture, dated as of August 5, 2016, between the Company and Wilmington Trust, National Association, as trustee, as supplemented by the Second Supplemental Indenture, dated as of February 20, 2020, governing the 2022 Convertible Notes. After giving effect to the issuance of the additional 2022 Convertible Notes and the exchange of the 2021 Notes pursuant to the exchange transaction, $46.85 million aggregate principal amount of the 2022 Convertible Notes remains issued and outstanding and $5.15 million aggregate principal amount of the 2021 Convertible Notes remains issued and outstanding.

The conversion rate for the 2022 Convertible Notes is initially 31.1876 shares of the Company’s common stock per 1.00 principal amount of 2022 Convertible Notes, which is equivalent to an initial conversion price of approximately $32.06 per share of common stock, and is subject to adjustment under the terms of the 2022 Convertible Notes. The Company may redeem for cash all or any portion of the 2022 Convertible Notes, at its option, if the last reported sale price of its common stock is 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 trading days immediately preceding the date on which the Company provides written notice of redemption. The 2022 Convertible Notes Indenture contains customary terms and covenants and events of default. As of June 30, 2020, the stated interest rate was 6.25%, and the effective interest rate was 15.3%.

The exchange transactions were accounted for as a modification of the 2021 Convertibles Notes and have been accounted for based on ASC 470-50 Modifications and Extinguishments. The remaining outstanding principal of the 2021 Convertible Notes will be accounted for consistent with the original accounting assessment. The Company incurred $1.6 million of costs in connection with the February 2020 exchange and $0.3 million of costs in connection with the June 2020 exchange transaction which have been included in G&A expenses in the consolidated financial statements for the three and six months ended June 30, 2020. Costs incurred of $0.1 million in connection with the February exchange transaction and $0.1 million in connection with the June exchange transaction that were paid directly to the creditors have been included as a debt discount and is being amortized over the life of the 2022 Convertible Notes.

The table below summarizes the carrying value of the 2021 Convertible Notes as of June 30, 2020:

	June 30, 2020
Principal amount	$	5,150
Discount		(490	)
Carrying value as of June 30, 2020	$	4,660

Accretion of the 2021 Convertible Notes discount was $0.2 million and $0.8 million for the three and six months ended June 30, 2020, respectively. Accretion of the 2021 Convertible Notes discount was $0.8 million and $1.7 million for the three and six months ended June 30, 2019, respectively.

The table below summarizes the carrying value of the 2022 Convertible Notes as of June 30, 2020:

	June 30, 2020
Principal amount	$	46,850
Discount		(5,282	)
Carrying value as of June 30, 2020	$	41,568

Accretion of the 2022 Convertible Notes discount was $0.5 million and $0.6 million for the three and six months ended June 30, 2020, respectively.

Stock Based Compensation

6 Months Ended

Jun. 30, 2020

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 Months Ended June 30,
	2020			2019

Risk-free interest rate		1.27	%		2.57	%
Expected term (in years)		5.84			5.79
Expected volatility		77.39	%		74.99	%
Expected dividend yield		0.00	%		0.00	%
Exercise price	$	21.56		$	15.01
Fair value of common stock	$	21.56		$	15.01

The Company recognizes compensation expense for only the portion of awards that are expected to vest.

The following table summarizes stock option activity for the six months ended June 30, 2020 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, 2019		9,763,541		$	5.95		6.96	$	164
Granted		1,491,296			21.56		9.55
Exercised		(417,512	)		1.71				7
Cancelled		(60,512	)		17.27
Outstanding as of June 30, 2020		10,776,813		$	8.21		6.92	$	139

Options vested and exercisable as of June 30, 2020		7,814,535		$	4.43		6.09	$	129
Options unvested as of June 30, 2020		2,962,278		$	18.18		9.11

The weighted average grant-date fair value per share of stock options granted during the six months ended June 30, 2020 and 2019 was $14.29 and $9.90, respectively.

The total fair value of options vested during the six months ended June 30, 2020 and 2019 was $30.0 million and $35.7 million, respectively.

Stock-Based Compensation

	Three Months Ended June 30,				Six Months Ended June 30,
	2020		2019		2020		2019

Research and development	$	1,679	$	2,252	$	3,414	$	3,818
General and administrative		2,810		1,886		5,036		3,500
Total share based compensation expense	$	4,489	$	4,138	$	8,450	$	7,318

As of June 30, 2020, the Company had an aggregate of $31.6 million of unrecognized stock-based compensation expense, which is expected to be recognized over the weighted average period of 1.97 years.

Stockholders' Equity

6 Months Ended

Jun. 30, 2020

Stockholders' Equity [Abstract]

Stockholders' Equity

9.	Stockholders’ Equity

The Company has 14,102 warrants outstanding as of June 30, 2020, convertible into 14,102 shares of common stock at an exercise price of $24.82 per share, and expiring on June 28, 2023.

On December 12, 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.

As of March 31, 2020, the Company recorded treasury stock of $0.4 million for the shares withheld to pay the payroll tax liability of an option exercise which was remitted in June 2020.

As of June 30, 2020, the Company recorded treasury stock of $0.1 million for the shares withheld to pay the payroll tax liability of option exercises which were remitted in June 2020.

Net Loss Per Share

6 Months Ended

Jun. 30, 2020

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,
	2020			2019			2020			2019

Numerator:
Net loss attributable to common stockholders	$	(25,040	)	$	(18,680	)	$	(49,704	)	$	(38,131	)
Denominator:
Weighted-average common shares outstanding - basic and diluted		55,158,459			49,267,247			55,020,789			47,206,480

Net loss per share attributable to common stockholders - basic and diluted	$	(0.45	)	$	(0.38	)	$	(0.90	)	$	(0.81	)

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,
	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		1,460,412		-
Warrants exercisable for common shares		14,102		14,102
Options to purchase common shares		10,776,813		8,870,004
		12,411,863		10,505,054

Commitments and Contingencies

6 Months Ended

Jun. 30, 2020

Commitments and Contingencies [Abstract]

Commitments and Contingencies

11.	Commitments and Contingencies

The Company determines if an arrangement is a lease at inception. Operating leases are included in the Company’s consolidated balance sheet as right-of-use assets from operating leases, current operating lease liabilities and long-term operating lease liabilities. Finance leases are included in the consolidated balance sheet as right-of-use assets from finance leases, and finance 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 will be 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 term of 15 years 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, 2020 and December 31, 2019. The Company entered into the lease prior to the building being available for use as the building construction was not complete.

The Company began making lease payments on September 1, 2019. At that time and as of December 31, 2019, the Company determined the lease commencement date had not occurred since the landlord improvements had not been completed. Prior to the lease commencement date, the lease payments were recorded as prepaid rent. 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 three months ended June 30, 2020, the Company reclassified an additional $2.2 million of construction costs in progress as part of the right of use asset.

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 lease and recorded a loss on termination of $76.

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”) with a bank for $0.9 million which expires on June 30, 2020 and is renewed automatically for a one year period until its expiration date of July 31, 2021. The Empire LOC serves as the Company’s security deposit on the lease in which the landlord is the beneficiary. The Company has a certificate of deposit of $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, 2020 and December 31, 2019.

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 $0.1 million and $0.2 million for the three and six months ended June 30, 2020. Rental income under the same agreement was $0.1 million and $0.2 million for the three and six months ended June 30, 2019. These amounts are netted against rent expense in the consolidated statement of operations.

Rent expense was $0.3 million and $0.5 million for the three and six months ended June 30, 2020, respectively. Rent expense was $0.3 million and $0.5 million for the three and six months ended June 30, 2019, respectively.

The total restricted cash balance for the Company’s finance leases at June 30, 2020 and December 31, 2019 was $1.0 million.

Lease cost	June 30, 2020
Operating lease cost	$	407
Finance lease cost
Amortization of right of use assets		655
Interest on lease liablities		607
Total lease cost	$	1,668

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, 2020:

Maturity of operating lease liabilities	June 30, 2020
2020	$	450
2021		682
2022		445
2023		75
Total lease payments	$	1,652
Less: interest		(127	)
Total operating lease liabilities	$	1,525

Maturity of finance lease liability	June 30, 2020
2020	$	805
2021		1,644
2022		1,689
2023		1,736
2024		1,791
Thereafter		48,767
Total lease payments	$	56,432
Less: interest		(35,911	)
Total finance lease liability	$	20,521

Leases	June 30, 2020

Operating right-of-use assets	$	1,270

Operating current lease liabilities		819
Operating noncurrent lease liabilities		706
Total operating lease liabilities	$	1,525

Finance right-of-use assets	$	49,250

Finance current lease liability		1,621
Finance noncurrent lease liability		18,900
Total finance lease liability	$	20,521

Other information
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows from operating leases	$	447
Cash flows from finance lease	$	265
Weighted-average remaining lease term - operating leases	2.2 years
Weighted-average remaining lease term - finance lease	24.2 years
Weighted-average discount rate - operating leases		7.77	%
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 Intellectual Property

6 Months Ended

Jun. 30, 2020

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.

Strategic Research Collaboration

6 Months Ended

Jun. 30, 2020

Strategic Research Collaboration [Abstract]

Strategic Research Collaboration

13.	Strategic Research Collaboration

On May 16, 2018, the Company and the Stanford University School of Medicine (“Stanford University”) entered into a strategic collaboration agreement to support the advancement of FA and PKD gene therapy research. Under the terms of the collaboration agreement, Stanford University is the lead clinical trial research center in the U.S. for the FA registrational trial and the lead U.S. site for PKD clinical trials. The project will also separately evaluate the potential for non-myeloablative, non-genotoxic antibody-based conditioning regimens as a future development possibility that may be applied across bone marrow-derived disorders. In addition, the Company agreed to support expansion of Stanford University’s Laboratory for Cell and Gene Therapy (“LCGM”) in order to further enhance the development of the Company’s internal pipeline.The Company agreed to contribute up to $3.5 million for the LCGM expansion of which $1.4 million was due upon execution of the collaboration agreement and the remaining $2.1 million balance is due upon the achievement of certain milestones. In January 2019, the Company and Stanford University signed a Clinical Trial Agreement for the treatment of FA and upon signing of the Clinical Trial Agreement, the second milestone of $1.4 million for the LGCM became due and was expensed and paid. During the six months ended June 30, 2020, the remaining milestone under this collaboration agreement was met and the Company expensed and paid the final milestone payment of $0.7 million.

CIRM Grant

6 Months Ended

Jun. 30, 2020

CIRM Grant [Abstract]

CIRM Grant

14.	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, we received the first two grants from CIRM in the aggregate of $1.2 million, based on eligible costs incurred under the grant. The CIRM grant reimbursements are accrued as an offset against R&D expenses as reimbursable expenses are incurred. On July 1, 2020, the Company received the next cash advance from CIRM of $1.6 million. As of June 30, 2020, the Company recorded a receivable and reduction of R&D expenses of $1.6 million for the reimbursable expenses incurred for the three months ended June 30, 2020.

Related Party Transactions

6 Months Ended

Jun. 30, 2020

Related Party Transactions

15.	Related Party Transactions

During March 2018, the Company entered into a consulting agreement with a member of the Board of Directors for strategic and corporate consulting services to be provided to the Company. This agreement was terminated in 2019. The Company incurred expenses of $0 and $4 during the three and six months ended June 30, 2020 and 2019, respectively relating to services provided under this consulting agreement.

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 $27.5 per quarter, and the Company may terminate the agreement with 14 days’ notice. The Company incurred expenses of $27.5 during the three months ended June 30, 2020 and 2019, and incurred expenses of $55 during the six months ended June 30, 2020 and 2019, relating to services provided under this agreement.

In June 2020, 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 9,784 options with a fair value of $119.

401(k) Savings Plan

6 Months Ended

Jun. 30, 2020

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 the safe harbor match of 4% of employee contributions to the Plan, subject to certain limitations. The Company’s matching contribution for the three and six months ended June 30, 2020 was $87 and $186, respectively. The Company’s matching contribution for the three and six months ended June 30, 2019 was $44 and $125, respectively.

Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies (Policies)

6 Months Ended

Jun. 30, 2020

Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies [Abstract]

Basis of Presentation

Principles of Consolidation

Use of Estimates

Significant Accounting Policies

Cash, Cash Equivalents and Restricted Cash

	June 30, 2020		December 31, 2019

Cash and cash equivalents	$	163,203	$	185,383
Restricted cash		1,525		1,525
	$	164,728	$	186,908

Recent Accounting Pronouncements

There were no other recent accounting standards that impacted the Company for the three months ended June 30, 2020.

Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies (Tables)

6 Months Ended

Jun. 30, 2020

Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies [Abstract]

Cash, Cash Equivalents and Restricted Cash

	June 30, 2020		December 31, 2019

Cash and cash equivalents	$	163,203	$	185,383
Restricted cash		1,525		1,525
	$	164,728	$	186,908

Fair Value of Financial Instruments (Tables)

6 Months Ended

Jun. 30, 2020

Fair Value of Financial Instruments [Abstract]

Fair Value of Financial Instruments Measured on Recurring Basis

	Fair Value Measurements as of June 30, 2020 Using:
	Level 1		Level 2		Level 3		Total
Assets:
Money market mutual funds (included in cash and cash equivalents)	$	143,298	$	-	$	-	$	143,298

United States Treasury securities		15,575		-		-		15,575
Corporate Bonds		-		63,069		-		63,069
Municipal Bonds		-		6,000		-		6,000
Agency Bonds		-		1,956		-		1,956
Investments		15,575		71,025		-		86,600

	$	158,873	$	71,025	$	-	$	229,898

	Fair Value Measurements as of December 31, 2019 Using:
	Level 1		Level 2		Level 3		Total
Assets:
Money market mutual funds (included in cash and cash equivalents)	$	72,114	$	-	$	-	$	72,114
Cash		7,542		-		-		7,542
		79,656		-		-		79,656

United States Treasury securities		75,464		-		-		75,464
Government Bonds		-		8,000		-		8,000
Corporate Bonds		-		29,268		-		29,268
Municipal Bonds		-		6,000		-		6,000
Investments		75,464		43,268		-		118,732

	$	155,120	$	43,268	$	-	$	198,388

Property and Equipment (Tables)

6 Months Ended

Jun. 30, 2020

Property and Equipment [Abstract]

Property and Equipment

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

	June 30, 2020			December 31, 2019
Laboratory equipment	$	12,691		$	1,910
Computer equipment		176			179
Furniture and fixtures		1,847			273
Leasehold improvements		29			29
Internal use software		712			226
Construction costs in progress		-			27,809
		15,455			30,426
Less: accumulated depreciation		(1,141	)		(905	)
	$	14,314		$	29,521

Accounts Payable and Accrued Expenses (Tables)

6 Months Ended

Jun. 30, 2020

Accounts Payable and Accrued Expenses [Abstract]

Accounts Payable and Accrued Expenses

At June 30, 2020 and December 31, 2019, the Company’s accounts payable and accrued expenses consisted of the following:

	June 30,		December 31,
	2020		2019
Research and development	$	9,166	$	7,418
Property and equipment		3,958		4,424
Employee compensation		2,072		2,588
Accrued interest		1,252		1,241
Government grant payable		576		562
Professional fees		784		553
Internal use software		122		226
Other		315		340
	$	18,245	$	17,352

Convertible Notes (Tables)

6 Months Ended

Jun. 30, 2020

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, 2020:

	June 30, 2020
Principal amount	$	5,150
Discount		(490	)
Carrying value as of June 30, 2020	$	4,660

2022 Convertible Notes [Member]

Debt Instrument [Line Items]

Carrying Value of Convertible Notes

The table below summarizes the carrying value of the 2022 Convertible Notes as of June 30, 2020:

	June 30, 2020
Principal amount	$	46,850
Discount		(5,282	)
Carrying value as of June 30, 2020	$	41,568

Stock Based Compensation (Tables)

6 Months Ended

Jun. 30, 2020

Stock Based Compensation [Abstract]

Weighted-Average Assumptions for Stock Options

	Six Months Ended June 30,
	2020			2019

Risk-free interest rate		1.27	%		2.57	%
Expected term (in years)		5.84			5.79
Expected volatility		77.39	%		74.99	%
Expected dividend yield		0.00	%		0.00	%
Exercise price	$	21.56		$	15.01
Fair value of common stock	$	21.56		$	15.01

Stock Option Activity

The following table summarizes stock option activity for the six months ended June 30, 2020 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, 2019		9,763,541		$	5.95		6.96	$	164
Granted		1,491,296			21.56		9.55
Exercised		(417,512	)		1.71				7
Cancelled		(60,512	)		17.27
Outstanding as of June 30, 2020		10,776,813		$	8.21		6.92	$	139

Options vested and exercisable as of June 30, 2020		7,814,535		$	4.43		6.09	$	129
Options unvested as of June 30, 2020		2,962,278		$	18.18		9.11

Stock-Based Compensation Expense

Stock-Based Compensation

	Three Months Ended June 30,				Six Months Ended June 30,
	2020		2019		2020		2019

Research and development	$	1,679	$	2,252	$	3,414	$	3,818
General and administrative		2,810		1,886		5,036		3,500
Total share based compensation expense	$	4,489	$	4,138	$	8,450	$	7,318

Net Loss Per Share (Tables)

6 Months Ended

Jun. 30, 2020

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,
	2020			2019			2020			2019

Numerator:
Net loss attributable to common stockholders	$	(25,040	)	$	(18,680	)	$	(49,704	)	$	(38,131	)
Denominator:
Weighted-average common shares outstanding - basic and diluted		55,158,459			49,267,247			55,020,789			47,206,480

Net loss per share attributable to common stockholders - basic and diluted	$	(0.45	)	$	(0.38	)	$	(0.90	)	$	(0.81	)

Antidilutive Securities Excluded from Computation of Diluted Net Loss Per Share

	Three and Six Months Ended June 30,
	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		1,460,412		-
Warrants exercisable for common shares		14,102		14,102
Options to purchase common shares		10,776,813		8,870,004
		12,411,863		10,505,054

Commitments and Contingencies (Tables)

6 Months Ended

Jun. 30, 2020

Commitments and Contingencies [Abstract]

Lease Cost

Lease cost	June 30, 2020
Operating lease cost	$	407
Finance lease cost
Amortization of right of use assets		655
Interest on lease liablities		607
Total lease cost	$	1,668

Maturities of Operating Lease Liabilities

Maturity of operating lease liabilities	June 30, 2020
2020	$	450
2021		682
2022		445
2023		75
Total lease payments	$	1,652
Less: interest		(127	)
Total operating lease liabilities	$	1,525

Maturity of Finance Lease Liability

Maturity of finance lease liability	June 30, 2020
2020	$	805
2021		1,644
2022		1,689
2023		1,736
2024		1,791
Thereafter		48,767
Total lease payments	$	56,432
Less: interest		(35,911	)
Total finance lease liability	$	20,521

Balance Sheet Information Related to Leases

Leases	June 30, 2020

Operating right-of-use assets	$	1,270

Operating current lease liabilities		819
Operating noncurrent lease liabilities		706
Total operating lease liabilities	$	1,525

Finance right-of-use assets	$	49,250

Finance current lease liability		1,621
Finance noncurrent lease liability		18,900
Total finance lease liability	$	20,521

Other information
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows from operating leases	$	447
Cash flows from finance lease	$	265
Weighted-average remaining lease term - operating leases	2.2 years
Weighted-average remaining lease term - finance lease	24.2 years
Weighted-average discount rate - operating leases		7.77	%
Weighted-average discount rate - finance lease		8.96	%

Nature of Business (Details)	6 Months Ended
Nature of Business (Details)	Jun. 30, 2020 Program
Programs in Clinical Testing [Abstract]
Number of clinical-stage programs currently enrolling patients	4

Risks and Liquidity (Details) $ in Thousands	6 Months Ended
Risks and Liquidity (Details) $ in Thousands	Jun. 30, 2020 USD ($) Candidate	Dec. 31, 2019 USD ($)
Risks and Liquidity [Abstract]
Number of product candidates in clinical development \| Candidate	5
Accumulated deficit	$ (232,847)	$ (183,143)
Cash, cash equivalents and investments	$ 249,800

Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies (Details) - USD ($) $ in Thousands	Jun. 30, 2020	Dec. 31, 2019	Jun. 30, 2019	Dec. 31, 2018
Cash, Cash Equivalents and Restricted Cash [Abstract]
Cash and cash equivalents	$ 163,203	$ 185,383
Restricted cash	1,525	1,525
Total	$ 164,728	$ 186,908	$ 66,358	$ 112,791

Fair Value of Financial Instruments (Details) - USD ($) $ in Thousands	Jun. 30, 2020	Dec. 31, 2019
2021 Convertible Notes [Member]
Additional Information in Fair Value Disclosure [Abstract]
Fair value convertible note	$ 5,400
2022 Convertible Notes [Member]
Additional Information in Fair Value Disclosure [Abstract]
Fair value convertible note	47,200
Recurring [Member]
Assets [Abstract]
Cash and cash equivalents		$ 79,656
Recurring [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments	229,898	198,388
Recurring [Member] \| Money Market Mutual Funds [Member]
Assets [Abstract]
Cash and cash equivalents	143,298	72,114
Recurring [Member] \| Cash [Member]
Assets [Abstract]
Cash and cash equivalents		7,542
Recurring [Member] \| United States Treasury Securities [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments	15,575	75,464
Recurring [Member] \| Government Bonds [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments		8,000
Recurring [Member] \| Corporate Bonds [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments	63,069	29,268
Recurring [Member] \| Municipal Bonds [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments	6,000	6,000
Recurring [Member] \| Agency Bonds [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments	1,956
Recurring [Member] \| Investments [Member] \| Short-term Investments [Member]
Assets [Abstract]
Investments	86,600	118,732
Recurring [Member] \| Level 1 [Member]
Assets [Abstract]
Cash and cash equivalents		79,656
Recurring [Member] \| Level 1 [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments	158,873	155,120
Recurring [Member] \| Level 1 [Member] \| Money Market Mutual Funds [Member]
Assets [Abstract]
Cash and cash equivalents	143,298	72,114
Recurring [Member] \| Level 1 [Member] \| Cash [Member]
Assets [Abstract]
Cash and cash equivalents		7,542
Recurring [Member] \| Level 1 [Member] \| United States Treasury Securities [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments	15,575	75,464
Recurring [Member] \| Level 1 [Member] \| Government Bonds [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments		0
Recurring [Member] \| Level 1 [Member] \| Corporate Bonds [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments	0	0
Recurring [Member] \| Level 1 [Member] \| Municipal Bonds [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments	0	0
Recurring [Member] \| Level 1 [Member] \| Agency Bonds [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments	0
Recurring [Member] \| Level 1 [Member] \| Investments [Member] \| Short-term Investments [Member]
Assets [Abstract]
Investments	15,575	75,464
Recurring [Member] \| Level 2 [Member]
Assets [Abstract]
Cash and cash equivalents		0
Recurring [Member] \| Level 2 [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments	71,025	43,268
Recurring [Member] \| Level 2 [Member] \| Money Market Mutual Funds [Member]
Assets [Abstract]
Cash and cash equivalents	0	0
Recurring [Member] \| Level 2 [Member] \| Cash [Member]
Assets [Abstract]
Cash and cash equivalents		0
Recurring [Member] \| Level 2 [Member] \| United States Treasury Securities [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments	0	0
Recurring [Member] \| Level 2 [Member] \| Government Bonds [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments		8,000
Recurring [Member] \| Level 2 [Member] \| Corporate Bonds [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments	63,069	29,268
Recurring [Member] \| Level 2 [Member] \| Municipal Bonds [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments	6,000	6,000
Recurring [Member] \| Level 2 [Member] \| Agency Bonds [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments	1,956
Recurring [Member] \| Level 2 [Member] \| Investments [Member] \| Short-term Investments [Member]
Assets [Abstract]
Investments	71,025	43,268
Recurring [Member] \| Level 3 [Member]
Assets [Abstract]
Cash and cash equivalents		0
Recurring [Member] \| Level 3 [Member] \| Short-term Investments [Member]
Assets [Abstract]
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] \| Cash [Member]
Assets [Abstract]
Cash and cash equivalents		0
Recurring [Member] \| Level 3 [Member] \| United States Treasury Securities [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments	0	0
Recurring [Member] \| Level 3 [Member] \| Government Bonds [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments		0
Recurring [Member] \| Level 3 [Member] \| Corporate Bonds [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments	0	0
Recurring [Member] \| Level 3 [Member] \| Municipal Bonds [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments	0	0
Recurring [Member] \| Level 3 [Member] \| Agency Bonds [Member] \| Short-term Investments [Member]
Assets [Abstract]
Fair value of financial instruments	0
Recurring [Member] \| Level 3 [Member] \| Investments [Member] \| Short-term Investments [Member]
Assets [Abstract]
Investments	$ 0	$ 0

Property and Equipment (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
Property and Equipment (Details) - USD ($) $ in Thousands	Jun. 30, 2020	Jun. 30, 2019	Jun. 30, 2020	Jun. 30, 2019	Dec. 31, 2019
Property and Equipment [Abstract]
Property and equipment, Gross	$ 15,455		$ 15,455		$ 30,426
Less: accumulated depreciation	(1,141)		(1,141)		(905)
Property and equipment, net	14,314		14,314		29,521
Depreciation expense	100	$ 100	200	$ 200
Laboratory Equipment [Member]
Property and Equipment [Abstract]
Property and equipment, Gross	12,691		12,691		1,910
Computer Equipment [Member]
Property and Equipment [Abstract]
Property and equipment, Gross	176		176		179
Furniture and Fixtures [Member]
Property and Equipment [Abstract]
Property and equipment, Gross	1,847		1,847		273
Leasehold Improvements [Member]
Property and Equipment [Abstract]
Property and equipment, Gross	29		29		29
Internal use Software [Member]
Property and Equipment [Abstract]
Property and equipment, Gross	712		712		226
Construction Costs in Progress [Member]
Property and Equipment [Abstract]
Property and equipment, Gross	$ 0		$ 0		$ 27,809

Accounts Payable and Accrued Expenses (Details) - USD ($) $ in Thousands	Jun. 30, 2020	Dec. 31, 2019
Accounts Payable and Accrued Expenses [Abstract]
Research and development	$ 9,166	$ 7,418
Property and equipment	3,958	4,424
Employee compensation	2,072	2,588
Accrued interest	1,252	1,241
Government grant payable	576	562
Professional fees	784	553
Internal use software	122	226
Other	315	340
Total	$ 18,245	$ 17,352

Convertible Notes (Details)			3 Months Ended				6 Months Ended
Convertible Notes (Details)	Jun. 12, 2020 USD ($)	Feb. 20, 2020 USD ($) $ / shares shares	Jun. 30, 2020 USD ($) $ / shares	Mar. 31, 2020 USD ($)	Jun. 30, 2019 USD ($)	Mar. 31, 2019 USD ($)	Jun. 30, 2020 USD ($) $ / shares	Jun. 30, 2019 USD ($)	Dec. 31, 2019 USD ($)	Jan. 04, 2018 USD ($)
Debt [Abstract]
Aggregate amount of common stock shares repurchased				$ 72,000		$ 727,000
Convertible notes refinancing costs to the lender							$ 237,000	$ 0
Carrying value of convertible notes [Abstract]
Carrying value as of June 30, 2020			$ 46,228,000				46,228,000		$ 45,049,000
Accretion of discount on convertible notes							$ 1,416,000	1,733,000
2021 Convertible Notes [Member]
Debt [Abstract]
Debt instrument maturity date							Aug. 01, 2021
Convertible senior notes, interest rate, stated percentage	6.25%		5.75%				5.75%
Debt conversion ratio							31.1876
Conversion per principal amount of debt			$ 1,000.00				$ 1,000.00
Convertible Note exchange	1.00
Conversion price (in dollars per share) \| $ / shares			$ 32.08				$ 32.08
Percentage of common stock conversion price							200.00%
Number of trading days immediately preceding notice of redemption							5 days
Redemption price percentage							100.00%
Effective interest rate			15.30%				15.30%
Debt instrument, aggregate principal amount exchanged	$ 7,500,000	$ 39,350,000
Debt instrument, percentage of aggregate outstanding principal		76.00%
Accrued and unpaid interest amount to pay in cash	11,000,000	$ 119,416,000
Number of common stock shares repurchased (in shares) \| shares		3,000
Aggregate amount of common stock shares repurchased		$ 71,670,000
Cost associated with exchange transactions	300,000	$ 1,600,000
Carrying value of convertible notes [Abstract]
Principal amount	7,500,000		$ 5,150,000				$ 5,150,000			$ 52,000,000.0
Discount			(490,000)				(490,000)
Carrying value as of June 30, 2020	5,150,000		4,660,000				4,660,000
Accretion of discount on convertible notes			$ 200,000		$ 800,000		$ 800,000	$ 1,700,000
2022 Convertible Notes [Member]
Debt [Abstract]
Debt instrument maturity date							Aug. 31, 2022
Convertible senior notes, interest rate, stated percentage		6.25%	6.25%				6.25%
Debt conversion ratio		31.1876
Conversion per principal amount of debt		$ 1,000.00
Convertible Note exchange		1.00
Conversion price (in dollars per share) \| $ / shares		$ 32.06
Percentage of common stock conversion price		130.00%
Number of trading days immediately preceding notice of redemption							5 days
Effective interest rate			15.30%				15.30%
Convertible notes refinancing costs to the lender	100,000	$ 100,000
Carrying value of convertible notes [Abstract]
Principal amount		$ 39,350,000	$ 46,850,000				$ 46,850,000
Discount			(5,282,000)				(5,282,000)
Carrying value as of June 30, 2020	$ 46,850,000		41,568,000				41,568,000
Accretion of discount on convertible notes			$ 500,000				$ 600,000

Stock Based Compensation, Share Option Valuation (Details) - Stock Options [Member] - USD ($) $ / shares in Units, $ in Millions	6 Months Ended		12 Months Ended
	Jun. 30, 2020	Jun. 30, 2019	Dec. 31, 2019
Stock Option Activity [Roll Forward]
Outstanding at beginning of period (in shares)	9,763,541
Granted (in shares)	1,491,296
Exercised (in shares)	(417,512)
Cancelled (in shares)	(60,512)
Outstanding at end of period (in shares)	10,776,813		9,763,541
Options vested and exercisable at end of period (in shares)	7,814,535
Options unvested at ending of period (in shares)	2,962,278
Weighted Average Exercise Price [Abstract]
Outstanding at beginning of period (in dollars per share)	$ 5.95
Granted (in dollars per share)	21.56
Exercised (in dollars per share)	1.71
Cancelled (in dollars per share)	17.27
Outstanding at end of period (in dollars per share)	8.21		$ 5.95
Options vested and exercisable at end of period (in dollars per share)	4.43
Options unvested at ending (in dollars per share)	$ 18.18
Weighted-Average Remaining Contractual Term [Abstract]
Outstanding	6 years 11 months 1 day		6 years 11 months 15 days
Granted	9 years 6 months 18 days
Options vested and exercisable	6 years 1 month 2 days
Options unvested	9 years 1 month 9 days
Aggregate Intrinsic Value [Abstract]
Outstanding at beginning of period	$ 164.0
Exercised	7.0
Outstanding at end of period	139.0		$ 164.0
Options vested and exercisable	$ 129.0
Weighted average grant date fair value of shares granted (in dollars per share)	$ 14.29	$ 9.90
Total fair value of options vested	$ 30.0	$ 35.7
Employees and Directors [Member]
Weighted-Average Assumptions [Abstract]
Risk-free interest rate	1.27%	2.57%
Expected term	5 years 10 months 2 days	5 years 9 months 14 days
Expected volatility	77.39%	74.99%
Expected dividend yield	0.00%	0.00%
Exercise price (in dollars per share)	$ 21.56	$ 15.01
Fair value of common stock (in dollars per share)	$ 21.56	$ 15.01

Stock Based Compensation, Share-Based Compensation (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2020	Jun. 30, 2019	Jun. 30, 2020	Jun. 30, 2019
Stock-Based Compensation Expense [Abstract]
Total share based compensation expense	$ 4,489	$ 4,138	$ 8,450	$ 7,318
Unrecognized share-based compensation cost	31,600		$ 31,600
Weighted average period expected to recognize unrecognized share-based compensation cost			1 year 11 months 19 days
Research and Development [Member]
Stock-Based Compensation Expense [Abstract]
Total share based compensation expense	1,679	2,252	$ 3,414	3,818
General and Administrative [Member]
Stock-Based Compensation Expense [Abstract]
Total share based compensation expense	$ 2,810	$ 1,886	$ 5,036	$ 3,500

Stockholders' Equity (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended
	Jun. 30, 2020	Mar. 31, 2020	Dec. 31, 2019
Shareholders' Equity Disclosure [Abstract]
Warrants outstanding (in shares)	14,102
Exercise price of warrants (in dollars per share)	$ 24.82
Warrants expiry date	Jun. 28, 2023
Issuance of treasury stock pursuant to exercise of stock options	$ (109)	$ (429)	$ (100)
Warrants Exercisable for Common Shares [Member]
Shareholders' Equity Disclosure [Abstract]
Number of shares issuable on exercise of warrants outstanding (in shares)	14,102
Common Stock [Member]
Shareholders' Equity Disclosure [Abstract]
Issuance of treasury stock pursuant to exercise of stock options	$ 0	0
Treasury Stock [Member]
Shareholders' Equity Disclosure [Abstract]
Issuance of treasury stock pursuant to exercise of stock options	$ (109)	$ (429)

Net Loss Per Share (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended				6 Months Ended
	Jun. 30, 2020	Mar. 31, 2020	Jun. 30, 2019	Mar. 31, 2019	Jun. 30, 2020	Jun. 30, 2019
Numerator:
Net loss attributable to common stockholders	$ (25,040)	$ (24,664)	$ (18,680)	$ (19,451)	$ (49,704)	$ (38,131)
Denominator:
Weighted-average common shares outstanding - basic and diluted (in shares)	55,158,459		49,267,247		55,020,789	47,206,480
Net loss per share attributable to common stockholders - basic and diluted (in dollars per share)	$ (0.45)		$ (0.38)		$ (0.90)	$ (0.81)
Weighted Average Number of Shares Outstanding Reconciliation [Abstract]
Securities excluded from computation of diluted net loss per share (in shares)	12,411,863		10,505,054		12,411,863	10,505,054
Shares Issuable upon Conversion of the 2021 Convertible Notes [Member]
Weighted Average Number of Shares Outstanding Reconciliation [Abstract]
Securities excluded from computation of diluted net loss per share (in shares)	160,536		1,620,948		160,536	1,620,948
Shares issuable upon conversion of the 2022 Convertible Notes [Member]
Weighted Average Number of Shares Outstanding Reconciliation [Abstract]
Securities excluded from computation of diluted net loss per share (in shares)	1,460,412		0		1,460,412	0
Warrants Exercisable for Common Shares [Member]
Weighted Average Number of Shares Outstanding Reconciliation [Abstract]
Securities excluded from computation of diluted net loss per share (in shares)	14,102		14,102		14,102	14,102
Options to Purchase Common Shares [Member]
Weighted Average Number of Shares Outstanding Reconciliation [Abstract]
Securities excluded from computation of diluted net loss per share (in shares)	10,776,813		8,870,004		10,776,813	8,870,004

Commitments and Contingencies, Finance Lease (Details) $ in Thousands	6 Months Ended
	Jun. 30, 2020 USD ($) ft² LeaseAgreement	Mar. 15, 2020 USD ($)	Dec. 31, 2019 USD ($)
Finance Lease [Abstract]
Total lease payments	$ 56,432
Finance right-of-use assets	49,250		$ 0
Finance lease, liability	$ 20,521
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 right-of-use assets		$ 47,700
Finance lease, liability		20,200
Reclassification of construction costs in progress to right-of-use assets	$ 2,200	26,500
Reclassification of prepaid rent to right-of-use assets		$ 1,100
AAV Current Good Manufacturing Practice (cGMP) [Member]
Finance Lease [Abstract]
Area of lease \| ft²	50,000

Commitments and Contingencies, Operating Leases (Details) - USD ($) $ in Thousands		3 Months Ended		6 Months Ended
	May 31, 2020	Jun. 30, 2020	Jun. 30, 2019	Jun. 30, 2020	Jun. 30, 2019	Dec. 31, 2019	Jun. 07, 2018
Operating Leases [Abstract]
Rent expense		$ 300	$ 300	$ 500	$ 500
Restricted cash		$ 1,525		$ 1,525		$ 1,525
NY Lease Agreement [Member]
Operating Leases [Abstract]
Loss on termination of lease	$ (76)
Lease expiration date				Jul. 31, 2022
ESB Lease Agreement [Member]
Operating Leases [Abstract]
Term of lease agreement		3 years		3 years
Letter of credit							$ 900
Letter of credit expiration date				Jun. 30, 2020
LOC automatic renewal period				1 year
Lease expiration date				Jul. 31, 2021
Certificate of deposit		$ 900		$ 900		900
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 Leases [Member]
Operating Leases [Abstract]
Restricted cash		$ 1,000		$ 1,000		$ 1,000

Commitments and Contingencies, Details of Operating and Finance Lease (Details) - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2020	Dec. 31, 2019
Lease cost [Abstract]
Operating lease cost	$ 407
Finance lease cost [Abstract]
Amortization of right of use assets	655
Interest on lease liabilities	607
Total lease cost	1,668
Maturities of operating lease liabilities [Abstract]
2020	450
2021	682
2022	445
2023	75
Total lease payments	1,652
Less: interest	(127)
Total operating lease liabilities	1,525
Maturity of finance lease liability [Abstract]
2020	805
2021	1,644
2022	1,689
2023	1,736
2024	1,791
Thereafter	48,767
Total lease payments	56,432
Less: interest	(35,911)
Total finance lease liability	20,521
Lease assets and liabilities [Abstract]
Operating right-of-use assets	1,270	$ 2,051
Operating current lease liabilities	819	957
Operating noncurrent lease liabilities	706	1,443
Total operating lease liabilities	1,525
Finance right-of-use assets	49,250	0
Finance current lease liability	1,621	0
Finance noncurrent lease liability	18,900	$ 0
Total finance lease liability	20,521
Cash paid for amounts included in the measurement of lease liabilities [Abstract]
Operating cash flows from operating leases	447
Cash flows from finance lease	$ 265
Weighted-average remaining lease term - operating leases	2 years 2 months 12 days
Weighted-average remaining lease term - finance lease	24 years 2 months 12 days
Weighted-average discount rate - operating leases	7.77%
Weighted-average discount rate - finance lease	8.96%

Strategic Research Collaboration (Details) - USD ($) $ in Millions		1 Months Ended	6 Months Ended
	May 16, 2018	Jan. 31, 2019	Jun. 30, 2020
Collaboration Agreement [Abstract]
Funding in strategic research collaboration	$ 3.5
Funding in strategic research collaboration	1.4	$ 1.4
Remaining funding due upon the execution of the agreement	$ 2.1
Milestone payment made during period			$ 0.7

CIRM Grant (Details) - USD ($) $ in Millions		12 Months Ended
CIRM Grant (Details) - USD ($) $ in Millions	Jul. 01, 2020	Dec. 31, 2019	Jun. 30, 2020	Apr. 30, 2019
CIRM Grant [Abstract]
Grant award for clinical development support				$ 6.5
Amount received from grant for eligible costs incurred		$ 1.2
Grant receivable			$ 1.6
Subsequent Event [Member]
CIRM Grant [Abstract]
Amount received from grant for eligible costs incurred	$ 1.6

Related Party Transactions (Details) - USD ($)	3 Months Ended		6 Months Ended
Related Party Transactions (Details) - USD ($)	Jun. 30, 2020	Jun. 30, 2019	Jun. 30, 2020	Jun. 30, 2019
Member of the Board of Directors - One [Member]
Related Party Transaction [Abstract]
Related party expenses	$ 0	$ 4,000	$ 0	$ 4,000
Member of the Board of Directors - Two [Member]
Related Party Transaction [Abstract]
Related party expenses	$ 27,500	$ 27,500	55,000	$ 55,000
Business development consulting services expense (per quarter)			$ 27,500
Termination notice period for business development consulting services agreement			14 days
Member of the Board of Directors - Three [Member]
Related Party Transaction [Abstract]
Term of Consulting Agreement			1 year
Number of options granted for purchase (in shares)			9,784
Fair value amount of options granted			$ 119,000

401(k) Savings Plan (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
401(k) Savings Plan (Details) - USD ($) $ in Thousands	Jun. 30, 2020	Jun. 30, 2019	Jun. 30, 2020	Jun. 30, 2019
401(k) Savings Plan [Abstract]
Percentage of matching employee contributions			4.00%
Matching employee contributions	$ 87	$ 44	$ 186	$ 125