0000950170-23-060339 | 10-Q | XBRL Viewer | Rocket Pharmaceuticals INC

Business Combination, Consideration Transferred [Abstract]

$ 39,154

Renovacor, Inc.

Business Combination [Abstract]

Exchange ratio

0.1763

5,291,855

$ 8,339

$ 8,300

Cash consideration

^[1]

Renovacor, Inc. | Assumed Warrants [Member]

2,700

Total consideration

$ 72,265

Business Combination, Consideration Transferred [Abstract]

^[2]

1,503,229

Renovacor, Inc. | Warrants, Classified as Equity

^[2]

$ 7,183

Business Combination, Consideration Transferred [Abstract]

Renovacor, Inc. | Warrants Classified as Liabilities

5,671

Business Combination, Consideration Transferred [Abstract]

Renovacor, Inc. | Employee Stock Option

$ 1,512

Business Combination, Consideration Transferred [Abstract]

367,852

Renovacor, Inc. | Time Vesting RSU

$ 2,163

Business Combination, Consideration Transferred [Abstract]

28,798

Renovacor, Inc. | Stock Consideration

$ 512

Business Combination, Consideration Transferred [Abstract]

Share price (in dollars per share) | $ / shares

3,391,976

$ 18.39

Weighted-Average Assumptions for Stock Options

$ 62,378

[1]	Represents consideration paid for cash in lieu of fractional shares.
[2]	Assumed Renovacor Warrants of $7,183 with $5,671 classified as equity and $1,512 classified as liabilities.

Stock-Based Compensation (Tables)

9 Months Ended

Sep. 30, 2023

Stock-Based Compensation [Abstract]

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


	Nine Months Ended September 30,
	2023			2022
Risk-free interest rate		3.98	%		2.40	%
Expected term (in years)		5.84			5.82
Expected volatility		73.35	%		73.21	%
Expected dividend yield		0.00	%		0.00	%
Exercise price	$	20.15		$	15.79
Fair value of common stock	$	20.15		$	15.79

Stock Option Activity

The following table summarizes stock option activity for the nine months ended September 30, 2023:


				Weighted		Weighted
				Average		Average		Aggregate
	Number of			Exercise		Contractual		Intrinsic
	Shares			Price		Term (Years)		Value
Outstanding as of December 31, 2022		13,138,870		$	14.52		5.46	$	118,767
Granted		2,275,212			20.15		6.46
Exercised		(193,328	)		9.52				2,013
Cancelled or forfeited		(331,085	)		32.26
Outstanding as of September 30, 2023		14,889,669		$	15.05		5.43	$	127,116

Options vested and exercisable as of September 30, 2023		11,434,192		$	13.24		4.33	$	122,157
Options unvested as of September 30, 2023		3,455,477		$	21.03		9.08	$	4,959

RSU Activity

The following table summarizes the Company’s RSU activity for the nine months ended September 30, 2023:


				Weighted Average
	Number of			Grant Date
	Shares			Fair Value
Unvested as of December 31, 2022		992,874		$	16.49
Granted		976,015			19.63
Vested (1)		(302,271	)		16.29
Forfeited		(60,514	)		18.24
Unvested as of September 30, 2023		1,606,104		$	18.37

(1) Common stock issued is net of 120 shares related to taxes.

Stock-Based Compensation Expense by Award Type

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


	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Stock options	$	7,482	$	6,694	$	21,922	$	19,332
Restricted stock units		2,834		983		7,554		1,984
Total stock-based compensation expense	$	10,316	$	7,677	$	29,476	$	21,316

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 September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Research and development	$	4,673	$	3,040	$	13,130	$	8,247
General and administrative		5,643		4,637		16,346		13,069
Total stock-based compensation expense	$	10,316	$	7,677	$	29,476	$	21,316

Document and Entity Information - shares	9 Months Ended
Document and Entity Information - shares	Sep. 30, 2023	Nov. 03, 2023
Cover [Abstract]
Document Type	10-Q
Amendment Flag	false
Document Quarterly Report	true
Document Period End Date	Sep. 30, 2023
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2023
Document Fiscal Period Focus	Q3
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	609
Local Phone Number	659-8001
Title of 12(b) Security	Common Stock, $0.01 par value per share
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		90,158,895

Agreements Related to Intellectual Property	9 Months Ended
Agreements Related to Intellectual Property	Sep. 30, 2023
Agreements Related to Intellectual Property [Abstract]
Agreements Related to Intellectual Property	14. Agreements Related to Intellectual Property The Company, directly and through its subsidiary Spacecraft Seven, LLC, has various license and research and collaboration arrangements. The transactions principally resulted in the acquisition of rights to intellectual property which is in the preclinical phase and has not been tested for safety or feasibility. In all cases, the Company did not acquire tangible assets, processes, protocols, or operating systems. The Company expenses the acquired intellectual property rights as of the acquisition date on the basis that the cost of intangible assets purchased from others for use in research and development activities has no alternative future uses.

Renovacor Acquisition (Tables)

9 Months Ended

Sep. 30, 2023

Renovacor Acquisition [Abstract]

Total Consideration for Acquisition

The total consideration for the acquisition of Renovacor of $72.3 million consisted of the following:


	Shares		Value		Total
Stock consideration		3,391,976	$	18.39	$	62,378
Cash consideration(1)						29
Stock options		367,852				2,163
Time-vesting RSUs		28,798				512
Assumed warrants(2)		1,503,229				7,183
Total consideration		5,291,855			$	72,265

(1) Represents consideration paid for cash in lieu of fractional shares.

(2) Assumed Renovacor Warrants of $7,183 with $5,671 classified as equity and $1,512 classified as liabilities.

Purchase Price allocation of Assets Acquired and Liabilities Assumed

The preliminary purchase price allocation resulted in the following amounts being allocated to the assets acquired and liabilities assumed at the acquisition date based on their respective preliminary fair values summarized below:


Working capital(1)	$	(5,210	)
Cash and cash equivalents		42,755
Property and equipment		1,414
Operating lease right-of-use assets		1,161
Other non-current assets		113
IPR&D		25,150
Other intangible asset		574
Operating lease liability		(970	)
Deferred tax liability		(1,061	)
Net assets acquired		63,926
Goodwill		8,339
Purchase consideration	$	72,265

(1) Includes other receivables, prepaid expenses, account payable and accrued liabilities.

Related Party Transactions - Additional Information (Details) - USD ($)	3 Months Ended	9 Months Ended
	Sep. 30, 2023	Sep. 30, 2023
Consulting Agreement
Related Party Transaction [Abstract]
Related party expense relating to service provided	$ 0	$ 0
Restricted Stock Units (RSU)
Related Party Transaction [Abstract]
Shares granted (in shares)		976,015
Public Offering \| Common Stock [Member]
Related Party Transaction [Abstract]
Number of stock warrants issued to purchase shares		3,100,000

Leases - Future Lease Payments of Operating Lease Liabilities (Details) $ in Thousands	Sep. 30, 2023 USD ($)
Future Lease Payments of Operating Lease Liabilities [Abstract]
2023	$ 322
2024	903
2025	538
2026	545
2027	506
Thereafter	2,941
Total lease payments	5,755
Less: interest	(1,686)
Total operating lease liabilities	$ 4,069

Stockholders' Equity

9 Months Ended

Sep. 30, 2023

Stockholders' Equity [Abstract]

Stockholders' Equity

Stockholders’ Equity

At-the-Market Offering Program

On February 28, 2022, the Company entered into the Sales Agreement with Cowen with respect to an at-the-market offering program pursuant to which the Company may offer and sell, from time to time at its sole discretion, shares through Cowen as its sales agent. The shares to be offered and sold under the Sales Agreement, if any, will be offered and sold pursuant to the Company’s shelf registration statement on Form S-3. The Company filed a prospectus supplement with the SEC on February 28, 2022 in connection with the offer and sale of the shares pursuant to the Sales Agreement. The Company will pay Cowen a cash commission of 3.0% of gross proceeds from the sale of the shares pursuant to the Sales Agreement. The Company has provided Cowen with customary indemnification and contribution rights. The Company reimbursed Cowen for certain expenses incurred in connection with the Sales Agreement. Through September 30, 2023, the Company sold 4.2 million shares under the at-the-market offering program for gross proceeds of $65.8 million, less commissions of $2.0 million for net proceeds of $63.8 million. During the nine months ended September 30, 2023, the Company sold 0.9 million shares under the at-the-market offering program for gross proceeds of $17.8 million, less commission of $0.6 million for net proceeds of $17.2 million. On September 12, 2023, the Company and Cowen entered into the Amended Sales Agreement pursuant to which the aggregate offering amount available under the at-the-market offering program was reduced to $180.0 million.

Public Offering

On September 15, 2023, the Company completed a public offering of approximately 9.5 million shares of its common stock at a public offering price of $16.00 per share and pre-funded warrants to purchase 3.1 million shares of common stock at a price of $15.99 per warrant. The gross proceeds to Rocket from the public offering were approximately $201.3 million, net of $12.4 million of offering costs, underwriting discounts and commissions, legal and other expenses for net proceeds from the offering of $188.9 million.

The entire disclosure for equity.

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Net Loss Per Share - Basic and Diluted Net Loss Per Share (Details) - USD ($) $ / shares in Units, $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Numerator [Abstract]
Net loss attributable to common stockholders	$ (61,899)	$ (57,756)	$ (185,935)	$ (155,140)
Denominator [Abstract]
Weighted-average common shares outstanding - basic (in shares)	82,636,120	66,215,535	80,865,658	65,406,844
Weighted-average common shares outstanding - diluted (in shares)	82,636,120	66,215,535	80,865,658	65,406,844
Net loss per share attributable to common stockholders - basic (in dollars per share)	$ (0.75)	$ (0.87)	$ (2.30)	$ (2.37)
Net loss per share attributable to common stockholders - diluted (in dollars per share)	$ (0.75)	$ (0.87)	$ (2.30)	$ (2.37)

The amount of net income (loss) for the period per each share of common stock or unit outstanding during the reporting period.

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The amount of net income (loss) for the period available to each share of common stock or common unit outstanding during the reporting period and to each share or unit that would have been outstanding assuming the issuance of common shares or units for all dilutive potential common shares or units outstanding during the reporting period.

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Amount, after deduction of tax, noncontrolling interests, dividends on preferred stock and participating securities; of income (loss) available to common shareholders.

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Renovacor Acquisition - Total Consideration for Acquisition (Details) - Renovacor, Inc.
$ / shares in Units, $ in Thousands

Dec. 01, 2022

USD ($)

$ / shares

5,291,855

$ 2,700

Cash consideration

^[1]

Total consideration

$ 72,265

Assumed Warrants

1,503,229

^[2]

$ 7,183

^[2]

Employee Stock Option

367,852

$ 2,163

Time Vesting RSU

28,798

$ 512

Stock Consideration

Share price (in dollars per share) | $ / shares

3,391,976

$ 18.39

Intangible Assets and Goodwill [Abstract]

$ 62,378

[1]	Represents consideration paid for cash in lieu of fractional shares.
[2]	Assumed Renovacor Warrants of $7,183 with $5,671 classified as equity and $1,512 classified as liabilities.

Warrants

9 Months Ended

Sep. 30, 2023

Warrants [Abstract]

Warrants

10.

Warrants

A summary of the warrants outstanding as of September 30, 2023 is as follows:


Exercise Price	Outstanding		Grant/Assumption Date	Expiration Date
$57.11		603,386	December 21, 2020	December 21, 2030
33.63		301,291	August 9, 2021	August 9, 2031
22.51		153,155	December 17, 2021	December 17, 2031
22.51		153,155	December 17, 2021	December 17, 2031
65.23		617,050	December 1, 2022	April 23, 2025
65.23		760,086	December 1, 2022	December 1, 2026
$0.01		3,126,955	September 15, 2023	N/A
Total		5,715,078

The following table is a summary of changes in warrants to purchase common stock for the nine months ended September 30, 2023:


	Number of Warrant Shares
	Outstanding and Exercisable			Exercise Price per Share
Balance as of December 31, 2022		2,721,267
Issued		3,126,955		$	0.01
Exercised		(126,093	)		0.06
Expired		(7,051	)	$	24.42
Balance as of September 30, 2023		5,715,078

Warrants Issued in Public Offering

On September 15, 2023, the Company completed a public offering that included pre-funded warrants to purchase 3,126,955 shares of common shares at a price of $0.01 per share (see Note 8 “Stockholders’ Equity” and Note 17 (“Related Party Transactions”).

Assumed Renovacor Public Warrants

In conjunction with the Renovacor acquisition (see Note 15 “Renovacor Acquisition”), Rocket assumed pre-acquisition public warrants (“Public Warrants”) that were converted into Rocket warrants with a right to purchase 760,086 of Rocket common shares at an exercise price of $65.23 per share.

The Company determined that the Public Warrants met all of the criteria for equity classification. Accordingly, upon closing of the Merger, the Public Warrants were recorded as a component of additional paid-in capital of $3.4 million.

Assumed Renovacor Private Warrants

In conjunction with the Renovacor acquisition (see Note 15 “Renovacor Acquisition”), Rocket assumed pre-acquisition private warrants (“Private Warrants”) that were converted into Rocket warrants with a right to purchase 617,050 of Rocket common shares at an exercise price of $65.23 per share.

The Company determined that the Private Warrants did not meet all of the criteria for equity classification. Accordingly, the Company classifies the Private Warrants as derivative liabilities in its consolidated balance sheets. The Company measures the fair value of the warrants at the end of each reporting period and recognizes changes in the fair value from the prior period in the Company’s operating results for the current period. See Note 4 “Fair Value of Financial Instruments” for discussion of fair value measurement of the warrant liability.

Intangible Assets and Goodwill (Tables)

9 Months Ended

Sep. 30, 2023

Summary of Intangible Assets

Intangible assets as of September 30, 2023 and December 31, 2022 are summarized as follows:


September 30, 2023	Gross Carrying Value		Accumulated Amortization		Intangible Assets, Net
In process research & development	$	25,150	$	-	$	25,150
Total intangible assets	$	25,150	$	-	$	25,150


December 31, 2022	Gross Carrying Value		Accumulated Amortization		Intangible Assets, Net
In process research & development	$	25,150	$	-	$	25,150
Mice colony model		574		-		574
Total intangible assets	$	25,724	$	-	$	25,724

CIRM Grants

9 Months Ended

Sep. 30, 2023

CIRM Grants [Abstract]

CIRM Grants

16.

CIRM Grants

LAD-1 CIRM Grant

On April 30, 2019, the California Institute for Regenerative Medicine (“CIRM”) awarded the Company up to $7.5 million under a CLIN2 grant award to support the clinical development of its LV-based gene therapy for RP-L201. Proceeds from the grant will help fund clinical trial costs as well as manufactured drug product for Phase 1/2 patients enrolled at the U.S. clinical site, University of California, Los Angeles (“UCLA”) Mattel Children’s Hospital, led by principal investigator Donald Kohn, M.D., UCLA Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology and member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA. Through September 30, 2023, the Company has received $5.9 million in total RP-L201 grants from CIRM. No additional milestones were achieved during the three and nine months ended September 30, 2023.

Related Party Transactions

9 Months Ended

Sep. 30, 2023

Related Party Transactions

17.

Related Party Transactions

In June 2023, the Company entered into a consulting agreement with the spouse of one of the Company’s executive officers for information technology advisory services. The Company did not incur any expense for the three and nine months ended September 30, 2023, relating to services provided under this agreement.

In September 2023, in connection with the Company’s public offering, the Company sold approximately 3.1 million pre-funded warrants to purchase shares of the Company’s common stock to funds affiliated with RTW Investments, LP, the Company’s largest shareholder (see Note 8 “Stockholders’ Equity”).

The entire disclosure for related party transactions. Examples of related party transactions include transactions between (a) a parent company and its subsidiary; (b) subsidiaries of a common parent; (c) and entity and its principal owners; and (d) affiliates.

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Intangible Assets and Goodwill [Abstract]

Intangible Assets and Goodwill

9 Months Ended

Sep. 30, 2023

Intangible Assets and Goodwill

The Company’s indefinite lived intangible assets consist of an acquired IPR&D asset and a mice colony model received in the acquisition of Renovacor. Intangible assets as of September 30, 2023 and December 31, 2022 are summarized as follows:


September 30, 2023	Gross Carrying Value		Accumulated Amortization		Intangible Assets, Net
In process research & development	$	25,150	$	-	$	25,150
Total intangible assets	$	25,150	$	-	$	25,150


December 31, 2022	Gross Carrying Value		Accumulated Amortization		Intangible Assets, Net
In process research & development	$	25,150	$	-	$	25,150
Mice colony model		574		-		574
Total intangible assets	$	25,724	$	-	$	25,724

The gross carrying value of intangible assets is due to the acquisition of Renovacor (see Note 15 “Renovacor Acquisition”). The decrease in gross carrying value of intangibles at September 30, 2023 compared to December 31, 2022 was due to an impairment charge related to a reduction in the estimated fair value of the mice colony model to reflect the limited benefit of the model.

The carrying value of Goodwill as of September 30, 2023 and December 31, 2022 was $39.2 million and included $8.3 million as a result of the acquisition of Renovacor (see Note 15 “Renovacor Acquisition”).

Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended						9 Months Ended
Pay vs Performance Disclosure - USD ($) $ in Thousands	Sep. 30, 2023	Jun. 30, 2023	Mar. 31, 2023	Sep. 30, 2022	Jun. 30, 2022	Mar. 31, 2022	Sep. 30, 2023	Sep. 30, 2022
Pay vs Performance Disclosure
Net Income (Loss)	$ (61,899)	$ (65,701)	$ (58,335)	$ (57,756)	$ (54,402)	$ (42,982)	$ (185,935)	$ (155,140)

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

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-Name Accounting Standards Codification
-Section S99
-Paragraph 1B
-Subparagraph (SX 210.13-02(a)(4)(iii)(B))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480097/470-10-S99-1B

Reference 27: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1B
-Subparagraph (SX 210.13-02(a)(4)(iv))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480097/470-10-S99-1B

Reference 28: http://www.xbrl.org/2003/role/disclosureRef
-Topic 470
-SubTopic 10
-Name Accounting Standards Codification
-Section S99
-Paragraph 1B
-Subparagraph (SX 210.13-02(a)(5))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147480097/470-10-S99-1B

Reference 29: http://www.xbrl.org/2003/role/disclosureRef
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-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 30
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-30

Reference 30: http://www.xbrl.org/2003/role/disclosureRef
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-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 32
-Subparagraph (f)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482810/280-10-50-32

Reference 31: http://www.xbrl.org/2003/role/disclosureRef
-Topic 260
-SubTopic 10
-Name Accounting Standards Codification
-Section 45
-Paragraph 60B
-Subparagraph (a)
-Publisher FASB
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Reference 32: http://www.xbrl.org/2003/role/exampleRef
-Topic 280
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-Name Accounting Standards Codification
-Section 50
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-Publisher FASB
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Reference 33: http://www.xbrl.org/2003/role/disclosureRef
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-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 32
-Subparagraph (c)
-Publisher FASB
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Reference 34: http://www.xbrl.org/2003/role/disclosureRef
-Topic 205
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
-Paragraph 7
-Publisher FASB
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-SubTopic 10
-Name Accounting Standards Codification
-Section 45
-Paragraph 28
-Publisher FASB
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Reference 36: http://www.xbrl.org/2003/role/disclosureRef
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-SubTopic 10
-Name Accounting Standards Codification
-Section 45
-Paragraph 1A
-Subparagraph (a)
-Publisher FASB
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Reference 37: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
-Section 45
-Paragraph 1B
-Subparagraph (a)
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-Name Accounting Standards Codification
-Section S99
-Paragraph 2
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-Topic 942
-SubTopic 220
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.9-04(22))
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147483589/942-220-S99-1

Stockholders' Equity - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands		3 Months Ended	9 Months Ended
	Sep. 15, 2023	Sep. 30, 2023	Sep. 30, 2023	Sep. 30, 2022	Sep. 12, 2023	Feb. 28, 2022
At-the-Market Offering Program [Abstract]
Net proceeds from offering			$ 17,222	$ 46,540
Common Stock
At-the-Market Offering Program [Abstract]
Issuance of common stock, net of issuance costs (in shares)		9,453,418
Public Offerings [Abstract]
Common stock shares issued and sold (in shares)		9,453,418
Pre-Funded Warrants \| Common Stock
Public Offerings [Abstract]
Number of stock warrants issued to purchase shares	3,126,955
Warrant to purchase shares of common stock price per share (in dollars per share)	$ 0.01
At-the-Market Offering Program
At-the-Market Offering Program [Abstract]
Issuance of common stock, net of issuance costs (in shares)			900,000
Gross proceeds, offering amount			$ 17,800
Commissions			600
Net proceeds from offering			$ 17,200
Public Offerings [Abstract]
Common stock shares issued and sold (in shares)			900,000
At-the-Market Offering Program \| Cowen and Company, LLC [Member]
At-the-Market Offering Program [Abstract]
Percentage of cash commission						3.00%
Issuance of common stock, net of issuance costs (in shares)			4,200,000
Gross proceeds, offering amount			$ 65,800
Commissions			2,000
Net proceeds from offering			$ 63,800
Public Offerings [Abstract]
Common stock shares issued and sold (in shares)			4,200,000
At-the-Market Offering Program \| Cowen and Company, LLC [Member] \| Maximum
At-the-Market Offering Program [Abstract]
Aggregate offering amount					$ 180,000	$ 200,000
Public Offering \| Common Stock
At-the-Market Offering Program [Abstract]
Issuance of common stock, net of issuance costs (in shares)	9,500,000
Net proceeds from offering	$ 188,900
Aggregate offering amount	$ 201,300
Public Offerings [Abstract]
Common stock shares issued and sold (in shares)	9,500,000
Shares issued price per share	$ 16
Number of stock warrants issued to purchase shares			3,100,000
Gross proceeds from sale of stock	$ 201,300
Shares issuance cost	12,400
Net proceeds from issuance or sale of stock	$ 188,900
Public Offering \| Pre-Funded Warrants \| Common Stock
Public Offerings [Abstract]
Number of stock warrants issued to purchase shares	3,100,000
Warrant to purchase shares of common stock price per share (in dollars per share)	$ 15.99

Fair Value of Financial Instruments - Measured on Recurring Basis (Details) - Recurring - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Assets [Abstract]
Cash equivalents	$ 41,363	$ 106,892
Investments	211,514	259,153
Fair value of financial instruments	252,877	366,045
Liabilities [Abstract]
Total liabilities	808	1,512
Warrant Liability
Liabilities [Abstract]
Total liabilities	808	1,512
Money Market Mutual Funds
Assets [Abstract]
Cash equivalents	41,363	90,527
Commercial Paper
Assets [Abstract]
Cash equivalents		3,899
Investments		1,151
United States Treasury Securities
Assets [Abstract]
Cash equivalents		3,848
Investments	154,023	189,444
Corporate Bonds
Assets [Abstract]
Cash equivalents		8,618
Investments	50,441	60,905
Agency Bonds
Assets [Abstract]
Investments	7,050	7,653
Level 1
Assets [Abstract]
Cash equivalents	41,363	94,375
Investments	154,023	189,444
Fair value of financial instruments	195,386	283,819
Liabilities [Abstract]
Total liabilities	0	0
Level 1 \| Warrant Liability
Liabilities [Abstract]
Total liabilities	0	0
Level 1 \| Money Market Mutual Funds
Assets [Abstract]
Cash equivalents	41,363	90,527
Level 1 \| Commercial Paper
Assets [Abstract]
Cash equivalents		0
Investments		0
Level 1 \| United States Treasury Securities
Assets [Abstract]
Cash equivalents		3,848
Investments	154,023	189,444
Level 1 \| Corporate Bonds
Assets [Abstract]
Cash equivalents		0
Investments	0	0
Level 1 \| Agency Bonds
Assets [Abstract]
Investments	0	0
Level 2
Assets [Abstract]
Cash equivalents	0	12,517
Investments	57,491	69,709
Fair value of financial instruments	57,491	82,226
Liabilities [Abstract]
Total liabilities	0	0
Level 2 \| Warrant Liability
Liabilities [Abstract]
Total liabilities	0	0
Level 2 \| Money Market Mutual Funds
Assets [Abstract]
Cash equivalents	0	0
Level 2 \| Commercial Paper
Assets [Abstract]
Cash equivalents		3,899
Investments		1,151
Level 2 \| United States Treasury Securities
Assets [Abstract]
Cash equivalents		0
Investments	0	0
Level 2 \| Corporate Bonds
Assets [Abstract]
Cash equivalents		8,618
Investments	50,441	60,905
Level 2 \| Agency Bonds
Assets [Abstract]
Investments	7,050	7,653
Level 3
Assets [Abstract]
Cash equivalents	0	0
Investments	0	0
Fair value of financial instruments	0	0
Liabilities [Abstract]
Total liabilities	808	1,512
Level 3 \| Warrant Liability
Liabilities [Abstract]
Total liabilities	808	1,512
Level 3 \| Money Market Mutual Funds
Assets [Abstract]
Cash equivalents	0	0
Level 3 \| Commercial Paper
Assets [Abstract]
Cash equivalents		0
Investments		0
Level 3 \| United States Treasury Securities
Assets [Abstract]
Cash equivalents		0
Investments	0	0
Level 3 \| Corporate Bonds
Assets [Abstract]
Cash equivalents		0
Investments	0	0
Level 3 \| Agency Bonds
Assets [Abstract]
Investments	$ 0	$ 0

Risks and Liquidity - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands		3 Months Ended	9 Months Ended
	Sep. 15, 2023	Sep. 30, 2023	Sep. 30, 2023	Sep. 30, 2022	Sep. 12, 2023	Dec. 31, 2022	Feb. 28, 2022
Class of Stock [Line Items]
Accumulated deficit		$ (899,710)	$ (899,710)			$ (713,775)
Cash, cash equivalents and investments		$ 437,200	$ 437,200
Shareholders' Equity Disclosure [Abstract]
Common stock, par value (in dollars per share)		$ 0.01	$ 0.01			$ 0.01
Issuance of common stock pursuant to the at-the-market offering program, net of issuance costs			$ 17,222	$ 46,540
Common Stock
Shareholders' Equity Disclosure [Abstract]
Common stock shares issued and sold (in shares)		9,453,418
Pre-Funded Warrants \| Common Stock
Shareholders' Equity Disclosure [Abstract]
Number of stock warrants issued to purchase shares	3,126,955
Warrant to purchase shares of common stock price per share (in dollars per share)	$ 0.01
At-the-Market Offering Program
Shareholders' Equity Disclosure [Abstract]
Common stock shares issued and sold (in shares)			900,000
Issuance of common stock pursuant to the at-the-market offering program, net of issuance costs			$ 17,200
At-the-Market Offering Program \| Cowen and Company, LLC
Shareholders' Equity Disclosure [Abstract]
Common stock, par value (in dollars per share)							$ 0.01
Common stock shares issued and sold (in shares)			4,200,000
Issuance of common stock pursuant to the at-the-market offering program, net of issuance costs			$ 63,800
At-the-Market Offering Program \| Cowen and Company, LLC \| Maximum
Shareholders' Equity Disclosure [Abstract]
Aggregate offering amount					$ 180,000		$ 200,000
Public Offering \| Common Stock
Shareholders' Equity Disclosure [Abstract]
Aggregate offering amount	$ 201,300
Common stock shares issued and sold (in shares)	9,500,000
Issuance of common stock pursuant to the at-the-market offering program, net of issuance costs	$ 188,900
Shares issued price per share	$ 16
Number of stock warrants issued to purchase shares			3,100,000
Gross proceeds from sale of stock	$ 201,300
Shares issuance cost	12,400
Net proceeds from issuance or sale of stock	$ 188,900
Public Offering \| Pre-Funded Warrants \| Common Stock
Shareholders' Equity Disclosure [Abstract]
Number of stock warrants issued to purchase shares	3,100,000
Warrant to purchase shares of common stock price per share (in dollars per share)	$ 15.99

Intangible Assets and Goodwill - Summary of Intangible Assets (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Intangible Assets, Net [Abstract]
Gross Carrying Value	$ 25,150	$ 25,724
Intangible Assets, Net	25,150	25,724
In Process Research & Development
Intangible Assets, Net [Abstract]
Gross Carrying Value	25,150	25,150
Intangible Assets, Net	$ 25,150	25,150
Mice Colony Model
Intangible Assets, Net [Abstract]
Gross Carrying Value		574
Intangible Assets, Net		$ 574

Intangible Assets and Goodwill - Additional Information (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022	Dec. 01, 2022
Indefinite-Lived Intangible Assets [Line Items]
Goodwill	$ 39,154	$ 39,154
Renovacor, Inc.
Indefinite-Lived Intangible Assets [Line Items]
Goodwill	$ 8,300		$ 8,339

Amount after accumulated impairment loss of an asset representing future economic benefits arising from other assets acquired in a business combination that are not individually identified and separately recognized.

+ References + Details

Consolidated Balance Sheets - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Current assets:
Cash and cash equivalents	$ 225,657	$ 140,517
Investments	211,514	215,877
Prepaid expenses and other current assets	9,694	7,666
Total current assets	446,865	364,060
Property and equipment, net	36,534	29,009
Goodwill	39,154	39,154
Intangible assets	25,150	25,724
Restricted cash	1,372	1,340
Deposits	542	608
Investments	0	43,276
Operating lease right-of-use assets	4,089	1,972
Finance lease right-of-use asset	45,056	46,664
Total assets	598,762	551,807
Current liabilities:
Accounts payable and accrued expenses	30,624	36,660
Operating lease liabilities, current	1,079	773
Finance lease liability, current	1,773	1,736
Total current liabilities	33,476	39,169
Operating lease liabilities, non-current	2,990	1,088
Finance lease liability, non-current	19,341	19,269
Other liabilities	1,869	2,595
Total liabilities	57,676	62,121
Commitments and contingencies (Note 13)
Stockholders' equity:
Common stock, $0.01 par value, 120,000,000 shares authorized; 90,146,602 and 79,123,312 shares issued and 90,146,602 and 79,120,741 shares outstanding at September 30, 2023 and December 31, 2022, respectively	901	791
Treasury stock, at cost, 0 common shares at September 30, 2023 and 2,571 common shares at December 31, 2022 respectively	0	(47)
Additional paid-in capital	1,440,403	1,203,074
Accumulated other comprehensive loss	(508)	(357)
Accumulated deficit	(899,710)	(713,775)
Total stockholders' equity	541,086	489,686
Total liabilities and stockholders' equity	598,762	551,807
Series A Convertible Preferred Shares
Stockholders' equity:
Preferred stock	0	0
Series B Convertible Preferred Shares
Stockholders' equity:
Preferred stock	$ 0	$ 0

Basis of Presentation, Principles of Consolidation and Summary of Significant Accounting Policies - Investments - Additional Information (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Investments [Abstract]
Net unrealized loss on credit investments	$ 0	$ 0	$ 0	$ 0
Net unrealized gains (losses) on investments	$ 200	$ 200	$ (151)	$ (435)

Leases

9 Months Ended

Sep. 30, 2023

Leases [Abstract]

Leases

12.

Leases

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

Finance Lease

The Company has a lease for a facility in Cranbury, New Jersey, consisting of 103,720 square feet of space including areas for offices, process development, research, and development laboratories and 50,000 square feet dedicated to AAV Current Good Manufacturing Practice (“cGMP”) manufacturing facilities to support the Company’s pipeline (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 lease is estimated to be approximately $29.3 million over the 15-year term of the lease. The Company paid a cash security deposit of $0.3 million to the landlord in connection with the NJ Lease Agreement which has been reflected as part of deposits in the consolidated balance sheets as of September 30, 2023 and December 31, 2022.

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 into Amendment No. 1 to the ESB Lease Agreement (“ESB Lease Amendment”) that extended the term of the lease agreement to June 30, 2024, reduced the rent payments going forward, and reduced the Empire LOC to $0.8 million. The Empire LOC serves as the Company’s security deposit on the lease in which the landlord is the beneficiary and expires August 29, 2024.

The Company has a certificate of deposit of $0.8 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 September 30, 2023 and December 31, 2022.

On January 4, 2018, in connection with the Reverse Merger with Inotek, the Company assumed an operating lease for Inotek’s former headquarters in Lexington, Massachusetts, with a term which ended on February 28, 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 $0.3 million for the three and nine months ended September 30, 2023 and 2022, respectively. Rental income is netted against rent expense in the consolidated statements of operations. A security deposit of $0.2 million was returned to the Company in April 2023.

On November 15, 2022, the Company entered into a lease agreement with a lease term until October 31, 2024, for laboratory space in Madrid, Spain. The lease commenced on April 1, 2023 and the Company recognized a right-of-use asset and corresponding lease liability of approximately $0.2 million each.

On December 1, 2022, in connection with the Renovacor acquisition (see Note 15 “Renovacor Acquisition”), the Company acquired the Renovacor operating leases for space at facilities in Hopewell, New Jersey and Cambridge, Massachusetts with remaining lease terms of approximately 10.25 and 1.3 years, respectively. As of September 30, 2023, lease commencement dates have occurred for all leases and the Company recognized total right-of-use assets of $3.8 million with corresponding total lease liabilities of $3.6 million at lease commencement dates, which include right-of-use assets of $2.7 million and lease liabilities of $2.6 million for leases that commenced in 2023. The Company intends to sublease both premises through the remainder of their lease terms.

Operating lease cost was $0.3 million and $1.0 million for the three and nine months ended September 30, 2023, respectively. Operating lease cost was $0.2 million and $0.6 million for the three and nine months ended September 30, 2022, respectively.

Rent expense was $0.7 million and $1.7 million for the three and nine months ended September 30, 2023, respectively. Rent expense was $0.5 million and $1.2 million for the three and nine months ended September 30, 2022, respectively.

The total restricted cash balance for the Company’s operating and finance leases as of September 30, 2023 and December 31, 2022 was $0.8 million.


Lease cost	Nine Months Ended September 30,
Operating lease cost	$	1,026
Finance lease cost:
Amortization of right of use assets		1,615
Interest on lease liabilities		1,405
Total lease cost	$	4,046


Fiscal Year Ending December 31,	September 30, 2023
2023 (three months)	$	322
2024		903
2025		538
2026		545
2027		506
Thereafter		2,941
Total lease payments	$	5,755
Less: interest		(1,686	)
Total operating lease liabilities	$	4,069


Fiscal Year Ending December 31,	September 30, 2023
2023 (three months)	$	440
2024		1,791
2025		1,856
2026		1,912
2027		1,969
Thereafter		43,031
Total lease payments	$	50,999
Less: interest		(29,885	)
Total finance lease liability	$	21,114


Leases	September 30, 2023
Operating right-of-use assets	$	4,089

Operating current lease liabilities	$	1,079
Operating noncurrent lease liabilities		2,990
Total operating lease liabilities	$	4,069

Finance right-of-use assets	$	45,056

Finance current lease liability	$	1,773
Finance noncurrent lease liability		19,341
Total finance lease liability	$	21,114


Other information
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows from operating leases	$	894
Cash flows from finance lease	$	1,296
Weighted-average remaining lease term - operating leases		8.0 years
Weighted-average remaining lease term - finance lease		20.9 years
Weighted-average discount rate - operating leases		8.34	%
Weighted-average discount rate - finance lease		8.96	%

Leases - Finance Lease - Additional Information (Details) $ in Thousands	9 Months Ended
	Sep. 30, 2023 USD ($) ft² Leaseagreement	Dec. 31, 2022 USD ($)
Finance Lease [Abstract]
Total lease payments	$ 50,999
Cash security deposit	$ 542	$ 608
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
AAV Current Good Manufacturing Practice (cGMP) [Member]
Finance Lease [Abstract]
Area of lease \| ft²	50,000

Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Consolidated Statements of Comprehensive Loss [Abstract]
Net loss	$ (61,899)	$ (57,756)	$ (185,935)	$ (155,140)
Other comprehensive loss
Net unrealized gain (loss) on investments	216	169	(151)	(435)
Total comprehensive loss	$ (61,683)	$ (57,587)	$ (186,086)	$ (155,575)

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

Commitments and Contingencies [Abstract]

Net Loss Per Share

9 Months Ended

Sep. 30, 2023

Net Loss Per Share [Abstract]

Net Loss Per Share

11.

Net Loss Per Share

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


	Three Months Ended September 30,						Nine Months Ended September 30,
	2023			2022			2023			2022
Numerator:
Net loss attributable to common stockholders	$	(61,899	)	$	(57,756	)	$	(185,935	)	$	(155,140	)
Denominator:
Weighted-average common shares outstanding - basic and diluted		82,636,120			66,215,535			80,865,658			65,406,844
Net loss per share attributable to common stockholders - basic and diluted	$	(0.75	)	$	(0.87	)	$	(2.30	)	$	(2.37	)

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 Nine Months Ended September 30,
	2023		2022
Warrants exercisable for common shares		2,588,123		1,218,038
Restricted stock units convertible for common shares		1,606,104		895,869
Options to purchase common shares		14,889,669		12,730,994
		19,083,896		14,844,901

Net Loss Per Share - Antidilutive Securities Excluded from Computation of Diluted Net Loss Per Share (Details) - shares	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Weighted Average Number of Shares Outstanding, Diluted, Adjustment [Abstract]
Warrants exercisable for common shares	2,588,123	1,218,038	2,588,123	1,218,038
Restricted stock units convertible for common shares	1,606,104	895,869	1,606,104	895,869
Options to purchase common shares	14,889,669	12,730,994	14,889,669	12,730,994
Weighted average number diluted shares outstanding	19,083,896	14,844,901	19,083,896	14,844,901

Accounts Payable and Accrued Expenses - Schedule of Accounts Payable and Accrued Expenses (Details) - USD ($) $ in Thousands	Sep. 30, 2023	Dec. 31, 2022
Accounts Payable and Accrued Expenses [Abstract]
Research and development	$ 14,700	$ 19,100
Employee compensation	8,359	10,006
Property and equipment	1,670	2,095
Professional fees	4,042	1,436
Acquisition related expenses	0	1,153
Government grant payable	0	597
Other	1,853	2,273
Total accounts payable and accrued expenses	$ 30,624	$ 36,660

Commitments and Contingencies

9 Months Ended

Sep. 30, 2023

Commitments and Contingencies

13.

Commitments and Contingencies

Litigation

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

Indemnification Arrangements

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

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

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

9 Months Ended

Sep. 30, 2023

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

Cash, Cash Equivalents and Restricted Cash


	September 30, 2023		December 31, 2022
Cash and cash equivalents	$	225,657	$	140,517
Restricted cash		1,372		1,340
Total cash, cash equivalents and restricted cash	$	227,029	$	141,857

Renovacor Acquisition - Purchase Price allocation of Assets Acquired and Liabilities Assumed (Details) - USD ($)
$ in Thousands

Sep. 30, 2023

Dec. 31, 2022

Dec. 01, 2022

$ 39,154

Renovacor, Inc.

Cash and cash equivalents

Working capital

^[1]

$ (5,210)

42,755

Property and equipment

1,414

Operating lease right-of-use assets

1,161

Other non-current assets

113

IPR&D

25,150

Other intangible asset

574

Operating lease liability

(970)

Deferred tax liability

(1,061)

Net assets acquired

63,926

$ 8,300

8,339

Purchase consideration

$ 72,265

[1]	Includes other receivables, prepaid expenses, account payable and accrued liabilities.

Warrants - Summary of Changes in Warrants to Purchase Common Stock (Details) - Warrants	9 Months Ended
	Sep. 30, 2023 $ / shares shares
Number of Warrant Shares Outstanding and Exercisable [Abstract]
Beginning balance (in shares)	2,721,267
Issued (in shares)	3,126,955
Exercised (in shares)	(126,093)
Expired (in shares)	(7,051)
Ending balance (in shares)	5,715,078
Exercise Price Per Share [Abstract]
Issued (in dollars per share) \| $ / shares	$ 0.01
Exercised (in dollars per share) \| $ / shares	0.06
Expired (in dollars per share) \| $ / shares	$ 24.42

Stock-Based Compensation, Stock-Based Compensation (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
Stock-Based Compensation Expense [Abstract]
Total stock-based compensation expense	$ 10,316	$ 7,677	$ 29,476	$ 21,316
Research and Development
Stock-Based Compensation Expense [Abstract]
Total stock-based compensation expense	4,673	3,040	13,130	8,247
General and Administrative
Stock-Based Compensation Expense [Abstract]
Total stock-based compensation expense	5,643	4,637	16,346	13,069
Employee Stock Option
Stock-Based Compensation Expense [Abstract]
Total stock-based compensation expense	7,482	6,694	21,922	19,332
Restricted Stock Units
Stock-Based Compensation Expense [Abstract]
Total stock-based compensation expense	2,834	$ 983	7,554	$ 1,984
Stock Options and RSU Grants
Stock-Based Compensation Expense [Abstract]
Unrecognized stock-based compensation expense	$ 62,100		$ 62,100
Weighted average period expected to recognize unrecognized stock-based compensation expense			2 years 3 days

Stock-Based Compensation

9 Months Ended

Sep. 30, 2023

Stock-Based Compensation [Abstract]

Stock-Based Compensation

Stock Option Valuation

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


	Nine Months Ended September 30,
	2023			2022
Risk-free interest rate		3.98	%		2.40	%
Expected term (in years)		5.84			5.82
Expected volatility		73.35	%		73.21	%
Expected dividend yield		0.00	%		0.00	%
Exercise price	$	20.15		$	15.79
Fair value of common stock	$	20.15		$	15.79

The following table summarizes stock option activity for the nine months ended September 30, 2023:


				Weighted		Weighted
				Average		Average		Aggregate
	Number of			Exercise		Contractual		Intrinsic
	Shares			Price		Term (Years)		Value
Outstanding as of December 31, 2022		13,138,870		$	14.52		5.46	$	118,767
Granted		2,275,212			20.15		6.46
Exercised		(193,328	)		9.52				2,013
Cancelled or forfeited		(331,085	)		32.26
Outstanding as of September 30, 2023		14,889,669		$	15.05		5.43	$	127,116

Options vested and exercisable as of September 30, 2023		11,434,192		$	13.24		4.33	$	122,157
Options unvested as of September 30, 2023		3,455,477		$	21.03		9.08	$	4,959

The weighted average grant-date fair value per share of stock options granted during the nine months ended September 30, 2023 and 2022 was $13.32 and $15.79, respectively.

The total fair value of options vested during the nine months ended September 30, 2023 and 2022 was $26.9 million and $26.9 million, respectively.

Restricted Stock Units (“RSU”)

The following table summarizes the Company’s RSU activity for the nine months ended September 30, 2023:


				Weighted Average
	Number of			Grant Date
	Shares			Fair Value
Unvested as of December 31, 2022		992,874		$	16.49
Granted		976,015			19.63
Vested (1)		(302,271	)		16.29
Forfeited		(60,514	)		18.24
Unvested as of September 30, 2023		1,606,104		$	18.37

(1) Common stock issued is net of 120 shares related to taxes.

Stock-Based Compensation Expense

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


	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Stock options	$	7,482	$	6,694	$	21,922	$	19,332
Restricted stock units		2,834		983		7,554		1,984
Total stock-based compensation expense	$	10,316	$	7,677	$	29,476	$	21,316

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


	Three Months Ended September 30,				Nine Months Ended September 30,
	2023		2022		2023		2022
Research and development	$	4,673	$	3,040	$	13,130	$	8,247
General and administrative		5,643		4,637		16,346		13,069
Total stock-based compensation expense	$	10,316	$	7,677	$	29,476	$	21,316

As of September 30, 2023, the Company had an aggregate of $62.1 million of unrecognized stock-based compensation expense related to both stock options and RSU grants, which is expected to be recognized over a weighted average period of 2.01 years.

Renovacor Acquisition - Total Consideration for Acquisition (Parenthetical) (Details) - Renovacor, Inc.
$ in Thousands

Dec. 01, 2022

USD ($)

$ 2,700

Assumed Warrants

Warrants, Classified as Equity

7,183

^[1]

Warrants Classified as Liabilities

5,671

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

$ 1,512

[1]	Assumed Renovacor Warrants of $7,183 with $5,671 classified as equity and $1,512 classified as liabilities.

401(k) Savings Plan - Additional Information (Details) - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022	Sep. 30, 2023	Sep. 30, 2022
401(k) Savings Plan [Abstract]
Percentage of matching employee contributions			4.00%
Matching employee contributions	$ 400	$ 200	$ 1,100	$ 700

CIRM Grants - Additional Information (Details) - LAD-1 CIRM Grant $ in Millions	3 Months Ended	9 Months Ended
	Sep. 30, 2023 USD ($) Milestone	Sep. 30, 2023 USD ($) Milestone	Apr. 30, 2019 USD ($)
CIRM Grants [Abstract]
Grant award for clinical development support			$ 7.5
Milestone payments received	$ 5.9	$ 5.9
Number of milestones achieved \| Milestone	0	0

401(k) Savings Plan	9 Months Ended
401(k) Savings Plan	Sep. 30, 2023
401(k) Savings Plan [Abstract]
401(k) Savings Plan	18. 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 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 nine months ended September 30, 2023, was $0.4 million and $1.1 million, respectively. The Company’s matching contribution for the three and nine months ended September 30, 2022, was $0.2 million and $0.7 million, respectively.

Fair Value of Financial Instruments - Level 3 Fair Value of the Private Warrants (Details) - Private Warrants	Sep. 30, 2023 USD ($) $ / shares	Dec. 31, 2022 USD ($) $ / shares
Fair Value of the Private Warrants, Assumptions [Abstract]
Term	1 year 6 months 21 days	2 years 4 months 20 days
Fair value per warrant (in dollars per share) \| $ / shares	$ 1.31	$ 2.45
Stock Price
Fair Value of the Private Warrants, Assumptions [Abstract]
Measurement input	20.49	18.39
Exercise Price
Fair Value of the Private Warrants, Assumptions [Abstract]
Measurement input	65.23	65.23
Expected Volatility
Fair Value of the Private Warrants, Assumptions [Abstract]
Measurement input	0.6708	0.7125
Risk-Free Interest Rate
Fair Value of the Private Warrants, Assumptions [Abstract]
Measurement input	0.0522	0.0414
Expected Dividend Yield
Fair Value of the Private Warrants, Assumptions [Abstract]
Measurement input	0	0

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

9 Months Ended

Sep. 30, 2023

Basis of Presentation

Significant Accounting Policies

Principles of Consolidation

Use of Estimates

Cash, Cash Equivalents and Restricted Cash


	September 30, 2023		December 31, 2022
Cash and cash equivalents	$	225,657	$	140,517
Restricted cash		1,372		1,340
Total cash, cash equivalents and restricted cash	$	227,029	$	141,857

Concentrations of Credit Risk and Off-balance Sheet Risk

Concentrations of credit risk and off-balance sheet risk

Investments

Intangible Assets

Fair Value Measurements

•

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

•

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

•

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

To the extent that valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment.

Warrants

Stock-Based Compensation

Income Taxes

Recent Accounting Pronouncements

There were no recent accounting pronouncements that impacted the Company or which had a significant effect on the consolidated financial statements.

Stock-Based Compensation - Stock Option Activity (Details) - Employee Stock Option $ / shares in Units, $ in Thousands	9 Months Ended	12 Months Ended
	Sep. 30, 2023 USD ($) $ / shares shares	Dec. 31, 2022 USD ($) $ / shares shares
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding [Roll Forward]
Outstanding at beginning of period (in shares) \| shares	13,138,870
Granted (in shares) \| shares	2,275,212
Exercised (in shares) \| shares	(193,328)
Cancelled or forfeited (in shares) \| shares	(331,085)
Outstanding at end of period (in shares) \| shares	14,889,669	13,138,870
Options vested and exercisable at end of period (in shares) \| shares	11,434,192
Options unvested at ending of period (in shares) \| shares	3,455,477
Weighted Average Exercise Price [Abstract]
Outstanding at beginning of period (in dollars per share) \| $ / shares	$ 14.52
Granted (in dollars per share) \| $ / shares	20.15
Exercised (in dollars per share) \| $ / shares	9.52
Cancelled or forfeited (in dollars per share) \| $ / shares	32.26
Outstanding at end of period (in dollars per share) \| $ / shares	15.05	$ 14.52
Options vested and exercisable at end of period (in dollars per share) \| $ / shares	13.24
Options unvested at ending (in dollars per share) \| $ / shares	$ 21.03
Weighted-Average Remaining Contractual Term [Abstract]
Outstanding	5 years 5 months 4 days	5 years 5 months 15 days
Granted	6 years 5 months 15 days
Options vested and exercisable	4 years 3 months 29 days
Options unvested	9 years 29 days
Aggregate Intrinsic Value [Abstract]
Outstanding at beginning of period \| $	$ 118,767
Exercised \| $	2,013
Outstanding at end of period \| $	127,116	$ 118,767
Options vested and exercisable \| $	122,157
Options unvested \| $	$ 4,959

Net Loss Per Share (Tables)

9 Months Ended

Sep. 30, 2023

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 September 30,						Nine Months Ended September 30,
	2023			2022			2023			2022
Numerator:
Net loss attributable to common stockholders	$	(61,899	)	$	(57,756	)	$	(185,935	)	$	(155,140	)
Denominator:
Weighted-average common shares outstanding - basic and diluted		82,636,120			66,215,535			80,865,658			65,406,844
Net loss per share attributable to common stockholders - basic and diluted	$	(0.75	)	$	(0.87	)	$	(2.30	)	$	(2.37	)

Antidilutive Securities Excluded from Computation of Diluted Net Loss Per Share


	Three and Nine Months Ended September 30,
	2023		2022
Warrants exercisable for common shares		2,588,123		1,218,038
Restricted stock units convertible for common shares		1,606,104		895,869
Options to purchase common shares		14,889,669		12,730,994
		19,083,896		14,844,901

Warrants - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands	Sep. 15, 2023	Sep. 30, 2023	Dec. 31, 2022
Warrants [Abstract]
Additional paid-in capital		$ 1,440,403	$ 1,203,074
Public Warrants
Warrants [Abstract]
Additional paid-in capital		$ 3,400
Public Warrants \| Common Stock
Warrants [Abstract]
Warrant to purchase shares of common stock (in shares)		760,086
Warrant to purchase shares of common stock price per share (in dollars per share)		$ 65.23
Private Warrants
Warrants [Abstract]
Warrant to purchase shares of common stock price per share (in dollars per share)		$ 1.31	$ 2.45
Private Warrants \| Common Stock
Warrants [Abstract]
Warrant to purchase shares of common stock (in shares)		617,050
Warrant to purchase shares of common stock price per share (in dollars per share)		$ 65.23
Pre-Funded Warrants \| Common Stock
Warrants [Abstract]
Number of stock warrants issued to purchase shares	3,126,955
Warrant to purchase shares of common stock price per share (in dollars per share)	$ 0.01

Stock-Based Compensation - Additional Information (Details) - Employee Stock Option - USD ($) $ / shares in Units, $ in Millions	9 Months Ended
	Sep. 30, 2023	Sep. 30, 2022
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Weighted average grant date fair value of shares granted (in dollars per share)	$ 13.32	$ 15.79
Total fair value of options vested	$ 26.9	$ 26.9

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

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Renovacor Acquisition - Purchase Price allocation of Assets Acquired and Liabilities Assumed (Parenthetical) (Details) - Renovacor, Inc.
$ in Thousands

Dec. 01, 2022

USD ($)

Warrants Classified as Liabilities

$ 2,700

Warrants, Classified as Equity

1,512

5,671

Assumed Warrants