0000950170-24-021096 | 10-K | XBRL Viewer | Rocket Pharmaceuticals INC

$ 39,154

$ 30,815

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

Total Consideration for Acquisition

$ 8,300

8,339

Purchase consideration

$ 72,265

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

Fair Value of Financial Instruments - Measured on Recurring Basis (Details) - Recurring - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Assets [Abstract]
Cash equivalents	$ 53,224	$ 106,892
Investments	351,591	259,153
Fair value of financial instruments	404,815	366,045
Liabilities [Abstract]
Total liabilities	1,876	1,512
Warrant Liability
Liabilities [Abstract]
Total liabilities	1,876	1,512
Money Market Mutual Funds
Assets [Abstract]
Cash equivalents	50,737	90,527
Commercial Paper
Assets [Abstract]
Cash equivalents		3,899
Investments		1,151
U.S. Treasury Securities
Assets [Abstract]
Cash equivalents	2,487	3,848
Investments	312,696	189,444
Corporate Bonds
Assets [Abstract]
Cash equivalents		8,618
Investments	38,895	60,905
Agency Bonds
Assets [Abstract]
Investments		7,653
Level 1
Assets [Abstract]
Cash equivalents	50,737	90,527
Investments	0	0
Fair value of financial instruments	50,737	90,527
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	50,737	90,527
Level 1 \| Commercial Paper
Assets [Abstract]
Cash equivalents		0
Investments		0
Level 1 \| U.S. Treasury Securities
Assets [Abstract]
Cash equivalents	0	0
Investments	0	0
Level 1 \| Corporate Bonds
Assets [Abstract]
Cash equivalents		0
Investments	0	0
Level 1 \| Agency Bonds
Assets [Abstract]
Investments		0
Level 2
Assets [Abstract]
Cash equivalents	2,487	16,365
Investments	351,591	259,153
Fair value of financial instruments	354,078	275,518
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 \| U.S. Treasury Securities
Assets [Abstract]
Cash equivalents	2,487	3,848
Investments	312,696	189,444
Level 2 \| Corporate Bonds
Assets [Abstract]
Cash equivalents		8,618
Investments	38,895	60,905
Level 2 \| Agency Bonds
Assets [Abstract]
Investments		7,653
Level 3
Assets [Abstract]
Cash equivalents	0	0
Investments	0	0
Fair value of financial instruments	0	0
Liabilities [Abstract]
Total liabilities	1,876	1,512
Level 3 \| Warrant Liability
Liabilities [Abstract]
Total liabilities	1,876	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 \| U.S. Treasury Securities
Assets [Abstract]
Cash equivalents	0	0
Investments	0	0
Level 3 \| Corporate Bonds
Assets [Abstract]
Cash equivalents		0
Investments	$ 0	0
Level 3 \| Agency Bonds
Assets [Abstract]
Investments		$ 0

Agreements Related to Intellectual Property - Additional Information (Details) € in Thousands, $ in Thousands							12 Months Ended
	Nov. 30, 2017 EUR (€)	Nov. 30, 2017 USD ($)	Jul. 31, 2016 EUR (€)	Jul. 31, 2016 USD ($)	Mar. 31, 2016 EUR (€)	Mar. 31, 2016 USD ($)	Dec. 31, 2023 USD ($) Milestone Patent	Dec. 31, 2022 Milestone	Dec. 31, 2021 Milestone	Dec. 31, 2020 USD ($)	Dec. 31, 2019 USD ($)	Nov. 30, 2017 USD ($)	Feb. 28, 2017 USD ($)	Jul. 31, 2016 USD ($)	Mar. 31, 2016 USD ($)
License Agreement for Danon Disease
License Agreement [Abstract]
Research and development costs										$ 50
REGENXBIO, Inc. License
License Agreement [Abstract]
Research and development costs							$ 7,000
Potential future obligation							$ 13,000
Term of agreement							10 years
Termination notice period							6 months
Number of patents \| Patent							2
Options right expiry term							4 years
Royalties payable on fees received							20.00%
Payment and expenses for license fee							$ 2,000				$ 1,000
Number of additional milestones achieved \| Milestone							0	0	0
License Agreements with CIEMAT
License Agreement [Abstract]
Research and development costs			€ 100	$ 100	€ 30	$ 30
Potential future obligation			€ 5,000		€ 1,400									$ 6,000	$ 1,500
Termination notice period							90 days
Number of least uninterrupted years							2 years
License Agreement for LAD-I with CIEMAT and UCLB
License Agreement [Abstract]
Research and development costs	€ 30	$ 40
Potential future obligation	€ 1,400											$ 1,500
Termination notice period							90 days
Number of least uninterrupted years							2 years
License Agreement for LAD-I with CIEMAT and UCLB \| Maximum
License Agreement [Abstract]
Potential future obligation													$ 1,500

Warrants - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands			1 Months Ended	12 Months Ended
	Sep. 15, 2023	Oct. 06, 2022	Sep. 30, 2023	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Warrants [Abstract]
Expense in connection with warrant issue				$ 0	$ 0	$ 12,781
Issuance of common stock pursuant to exercise of warrant (in shares)					0
Class of warrant outstanding (in shares)				5,715,078
Additional paid-in capital				$ 1,450,722	$ 1,203,074
Public Offering
Warrants [Abstract]
Gross proceeds from sale of common stock				$ 188,885	108,138	$ 26,354
Common Stock
Warrants [Abstract]
Issuance of common stock pursuant to exercise of warrant (in shares)				126,093
Number of securities called by each warrant (in shares)				1
Gross proceeds from sale of common stock		$ 108,100
Common Stock \| Public Offering
Warrants [Abstract]
Gross proceeds from sale of common stock	$ 188,900
Renovacor, Inc. \| PIPE Investors
Warrants [Abstract]
Warrant to purchase shares of common stock (in shares)				2,284,776
Warrant to purchase shares of common stock price per share (in dollars per share)				$ 10
Warrants \| Renovacor, Inc.
Warrants [Abstract]
Class of warrant outstanding (in shares)				8,622,644
Public Warrants
Warrants [Abstract]
Warrant to purchase shares of common stock (in shares)				2
Warrant redemption price (in dollars per share)				$ 0.01
Notice period to redeem warrants				30 days
Threshold trading days				10 days
Threshold consecutive trading days				30 days
Redemption period				30 days
Additional paid-in capital					$ 3,400
Public Warrants \| Minimum
Warrants [Abstract]
Share price (in dollars per share)				$ 90.75
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
Public Warrants \| Renovacor, Inc.
Warrants [Abstract]
Number of securities called by each warrant (in shares)				0.50
Warrants expire period				5 years
Warrant to purchase shares of common stock price per share (in dollars per share)				$ 11.5
Private Warrants
Warrants [Abstract]
Warrants expire period				1 year 3 months 21 days	2 years 4 months 20 days
Warrant to purchase shares of common stock price per share (in dollars per share)				$ 3.04	$ 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
Private Warrants \| Renovacor, Inc.
Warrants [Abstract]
Class of warrant outstanding (in shares)				3,500,000
Number of securities called by each warrant (in shares)				1
Warrant to purchase shares of common stock price per share (in dollars per share)				$ 11.5
Private Warrants \| Renovacor, Inc. \| Maximum
Warrants [Abstract]
Warrants expire period				5 years
Pre-Funded Warrants
Warrants [Abstract]
Additional paid-in capital				$ 2,300
Pre-Funded Warrants \| Common Stock
Warrants [Abstract]
Warrant to purchase shares of common stock (in shares)				126,093
Warrant to purchase shares of common stock price per share (in dollars per share)	$ 15.99			$ 0.06
Number of stock warrants issued to purchase shares	3,100,000			3,126,955
Pre-Funded Warrants \| Common Stock \| Public Offering
Warrants [Abstract]
Warrant to purchase shares of common stock price per share (in dollars per share)	$ 15.99
Number of stock warrants issued to purchase shares	3,100,000		3,100,000
Indefinitely exercisable at an exercise price			$ 0.01
Pre-Funded Warrants \| Renovacor, Inc.
Warrants [Abstract]
Warrant to purchase shares of common stock (in shares)				715,224
Warrant to purchase shares of common stock price per share (in dollars per share)				$ 0.01
Share price (in dollars per share)				$ 9.99
Gross proceeds from sale of common stock				$ 30,000
Pre-Funded Warrants \| Renovacor, Inc. \| Minimum
Warrants [Abstract]
Percentage of beneficial ownership limitation				9.99%
Pre-Funded Warrants \| Renovacor, Inc. \| Maximum
Warrants [Abstract]
Percentage of beneficial ownership limitation				19.99%

Agreements Related to Intellectual Property

12 Months Ended

Dec. 31, 2023

15.

Agreements Related to Intellectual Property

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

License Agreements with CIEMAT

In March 2016, the Company entered into a license agreement with CIEMAT, CIBER, and FIISFJD, (collectively, “CIEMAT”), granting Rocket worldwide, exclusive rights to certain patents, know-how and other intellectual property relating to LVs containing the human PKLR gene solely within the field of treating PKD. Under the terms of the agreement, the Company is obligated to use commercially reasonable efforts to (a) develop and obtain regulatory approval for one or more products or processes covered by the licensed intellectual property, introduce such products or processes into the commercial market and then make them reasonably available to the public, (b) develop or commercialize at least one product or process covered by the licensed intellectual property in at least one country for at least two uninterrupted years following regulatory approval, and (c) use the licensed intellectual property in an adequate, ethical and legitimate manner. In exchange for the license, Rocket is obligated to pay CIEMAT an up-front payment, royalty payments based on net sales of products or processes involving any of the licensed intellectual property, developmental and regulatory milestone payments, and sublicense revenue payments. The Company is responsible for prosecuting and maintaining the licensed patents at its expense, in cooperation with CIEMAT. Rocket also has the first responsibility to enforce and defend the licensed patents against infringement and/or challenge, in cooperation with CIEMAT. For five years following the effective date of the license agreement, the Company has a right of first refusal to license any improvements to the licensed intellectual property obtained by CIEMAT at market value. Rocket is obligated to license (without charge) to CIEMAT for non-commercial use any improvements to the licensed intellectual property that it creates.

As consideration for the licensed rights, Rocket paid CIEMAT an initial upfront license fee of €0.03 million (approximately $0.03 million) which was expensed as R&D costs. The Company is obligated to make aggregate milestone payments of up to €1.4 million (approximately $1.5 million) to CIEMAT upon the achievement of specified development and regulatory milestones. With respect to any commercialized products covered by the PKD license, Rocket is obligated to pay a low to mid-single digit percentage royalty on net sales, subject to specified adjustments, by the Company or its sublicensees or affiliates. In the event that Rocket enters into a sublicense agreement with a sublicensee, it will be obligated to pay a portion of any consideration received from such sublicensees in specified circumstances.

Rocket may terminate this agreement at any time by providing CIEMAT with 90 days advance notice. The license is in effect for a duration for each of the countries defined in this agreement for as long as a license right exists that covers the licensed product or process in such country, or until the end of any additional legal protection that should be obtained for the license rights in each country.

In July 2016, Rocket entered into a license agreement with CIEMAT granting it worldwide, exclusive rights to certain patents, know-how, data and other intellectual property relating to LVs containing the FANCA gene solely within the field of human therapeutic uses of VSV-G packaged integration component LVs for FA type-A gene therapy. This license is only sublicensable with the prior consent of CIEMAT, not to be unreasonably withheld. Under the terms of the agreement, Rocket is obligated to use commercially reasonable efforts to (a) develop and obtain regulatory approval for one or more products or processes covered by the licensed intellectual property, introduce such products or processes into the commercial market and then make them reasonably available to the public (b) develop or commercialize at least one product or process covered by the licensed intellectual property in at least one country for at least two uninterrupted years following regulatory approval, and (c) use the licensed intellectual property in an adequate, ethical and legitimate manner. In exchange for the license, the Company is obligated to pay CIEMAT an up-front payment, royalty payments based on net sales of products or processes involving any of the licensed intellectual property, regulatory and financing milestone payments, and sublicense revenue payments. The Company is responsible for prosecuting and maintaining the licensed patents at our expense, in cooperation with CIEMAT. Rocket also has the first responsibility to enforce and defend the licensed patents against infringement and/or challenge, in cooperation with CIEMAT. For five years following the effective date of the license agreement, the Company has a right of first refusal to license any improvements to the licensed intellectual property obtained by CIEMAT at market value. Rocket is obligated to license (without charge) to CIEMAT for non-commercial use any improvements to the licensed intellectual property that it creates.

As consideration for the licensed rights, Rocket paid CIEMAT an initial upfront license fee of €0.1 million (approximately $0.1 million), which was expensed as R&D costs. The Company is obligated to make aggregate milestone payments of up to €5.0 million (approximately $6.0 million) to CIEMAT upon the achievement of specified development and regulatory milestones. With respect to any commercialized products covered by the license, Rocket is obligated to pay a mid-single digit percentage royalty on net sales, subject to specified adjustments, by Rocket or its sublicensees or affiliates. In the event that the Company enters into a sublicense agreement with a sublicensee, the Company will be obligated to pay a portion of any consideration received from such sublicensees in specified circumstances.

Rocket may terminate this agreement at any time by providing CIEMAT with 90 days’ advance notice. The license is in effect for a duration for each of the countries defined in this agreement for as long as a license right exists that covers the licensed product or process in such country, or until the end of any additional legal protection that should be obtained for the license rights in each country.

License Agreement for LAD-I with CIEMAT and UCLB

The Company entered into a license agreement in November 2017, effective September 2017, with CIEMAT and UCL Business PLC (“UCLB”), collectively referred to as (“Licensors”), granting the Company worldwide, exclusive rights to certain patents, know-how and other intellectual property relating to LVs containing the human LAD-I gene solely within the field of treating LAD-I. Under the terms of the agreement, Rocket is obligated to use commercially reasonable efforts to (a) develop and obtain regulatory approval for one or more products or processes covered by the licensed intellectual property, introduce such products or processes into the commercial market and then make them reasonably available to the public, (b) develop or commercialize at least one product or process covered by the licensed intellectual property in at least one country for at least two uninterrupted years following regulatory approval, and (c) use the licensed intellectual property in an adequate, ethical and legitimate manner. In exchange for the license, the Company is obligated to pay Licensors an up-front payment, royalty payments in the mid-single digit percentages based on net sales of products or processes involving any of the licensed intellectual property, developmental and regulatory milestone payments, and sublicense revenue payments. Rocket is responsible for prosecuting and maintaining the licensed patents at its expense, in cooperation with Licensors. The Company also has the first responsibility to enforce and defend the licensed patents against infringement and/or challenge, in cooperation with Licensors. For five years following the effective date of the license agreement, Rocket has a right of first refusal to license any improvements to the licensed intellectual property obtained by Licensors at market value. The Company is obligated to license (without charge) to Licensors for non-commercial use any improvements to the licensed intellectual property that it creates.

As consideration for the licensed rights, Rocket paid Licensors an initial upfront license fee of €0.03 million (approximately $0.04 million), which was expensed as R&D costs. The Company is obligated to make aggregate payments of up to €1.4 million (approximately $1.5 million) to Licensors upon the achievement of specified development and regulatory milestones. With respect to any commercialized products covered by the LAD-I license, Rocket is obligated to pay a mid-single digit percentage royalty on net sales, subject to specified adjustments, by the Company or its sublicensees or affiliates. In the event that the Company enters into a sublicense agreement with a sublicensee, it will be obligated to pay a portion of any consideration received from such sublicensees in specified circumstances.

Rocket may terminate this agreement at any time by providing the Licensors with 90 days advance notice. The license is in effect for a duration for each of the countries defined in this agreement for as long as a license right exists that covers the licensed product or process in such country, or until the end of any additional legal protection that should be obtained for the license rights in each country.

License Agreement for DD with UCSD

In February 2017, the Company entered into a License Agreement with The Regents of the University of California, represented by its San Diego campus (“UCSD”), under which UCSD granted us an exclusive, sublicensable, worldwide license to certain intellectual property rights for the treatment of lysosomal storage diseases, including DD. In exchange for the license, the Company became obligated to make an up-front payment, certain clinical and commercial milestone payments, royalty payments (on net sales of products covered by a valid claim within the licensed intellectual property), maintenance fees and sublicense revenue payments. The upfront license fee of $0.05 million was expensed as research and development costs in 2020. The Company is obligated to make aggregate milestone payments of up to $1.5 million to UCSD upon the achievement of specified development and regulatory milestones for the treatment of DD. A reduced schedule of milestone payments applies to achieving the same milestones for additional indications. With respect to any commercialized products covered by the agreement, the Company is obligated to pay a low single digit percentage royalty on net sales, subject to specified adjustments. If it enters into a sublicense agreement with a sublicensee, it will be obligated to pay a portion of any consideration received from such sublicensees in specified circumstances. The Company is also subject to certain diligence milestones for development of a product using the intellectual property licensed from UCSD under this agreement. The term of the license agreement with UCSD is through the expiration of the licensed patents, some of which are still in the pending application phase.

REGENXBIO, Inc. License

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

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

In consideration for the rights granted to the Company under the license agreement, the Company made an upfront payment to RGNX of $7.0 million. The license agreement provides for royalties payable to RGNX in the high-single digits to low-teens on net sales levels of Licensed Products during the royalty term. If successful, the Company will be required to make milestone payments to RGNX of up to $13.0 million for each Licensed Product upon the achievement of specified clinical development and regulatory milestones in the U.S. and EU. In addition, the Company shall pay RGNX 20% of the payment fees received from a priority review voucher issued in connection with or otherwise related to a Licensed Product. These royalty obligations are subject to specified reductions if additional licenses from third parties are required. The Company must also pay RGNX a portion of all non-royalty sublicense income (if any) received from sublicensees. The Company paid and expensed a $1.0 million license fee payment under the RGNX agreement upon the dosing of the first DD patient in 2019 and a $2.0 million license fee payment upon initiation of a Phase 2 pivotal trial in 2023. There were no additional milestones achieved or related payments made during the years ended December 31, 2023, 2022 and 2021.

Stockholders' Equity - Additional Information (Details) $ / shares in Units, $ in Thousands			12 Months Ended
	Sep. 15, 2023 USD ($) $ / shares shares	Oct. 06, 2022 USD ($) $ / shares shares	Dec. 31, 2023 USD ($) Vote $ / shares shares	Dec. 31, 2022 USD ($) $ / shares shares	Dec. 31, 2021 USD ($) shares	Sep. 12, 2023 USD ($)	Feb. 28, 2022 USD ($) $ / shares
Common Stock [Abstract]
Common stock, shares authorized (in shares) \| shares			120,000,000	120,000,000
Common stock, par value (in dollars per share) \| $ / shares			$ 0.01	$ 0.01
Number of voting right for each share \| Vote			1
Treasury Stock [Abstract]
Treasury stock (in shares) \| shares			0	2,571
Sale of treasury stock			$ 56	$ 0	$ 0
At-the-Market Offering Program
Private Placement At-the-Market Offering Program and Public Offerings [Abstract]
Issuance of common stock, net of issuance costs (in shares) \| shares			900,000	3,300,000
Gross proceeds, offering amount			$ 17,800	$ 48,000
Commissions			600	1,400
Net proceeds from offering			$ 17,222	$ 46,566	$ 0
At-the-Market Offering Program \| Cowen and Company, LLC
Common Stock [Abstract]
Common stock, par value (in dollars per share) \| $ / shares							$ 0.01
Private Placement At-the-Market Offering Program and Public Offerings [Abstract]
Issuance of common stock, net of issuance costs (in shares) \| shares			4,200,000
Percentage of cash commission							3.00%
Gross proceeds, offering amount			$ 65,800
Commissions			2,000
Net proceeds from offering			$ 63,800
At-the-Market Offering Program \| Cowen and Company, LLC \| Maximum
Private Placement At-the-Market Offering Program and Public Offerings [Abstract]
Aggregate offering amount						$ 180,000	$ 200,000
Common Stock
Private Placement At-the-Market Offering Program and Public Offerings [Abstract]
Issuance of common stock, net of issuance costs (in shares) \| shares	9,500,000	7,800,000	9,453,418	7,820,000	812,516
Share price (in dollars per share) \| $ / shares		$ 14.75
Gross proceeds, offering amount		$ 115,300
Commissions		7,200
Net proceeds from offering		$ 108,100
Shares issued price per share \| $ / shares	$ 16
Gross proceeds from sale of stock	$ 201,300
Shares issuance cost	12,400
Net proceeds from issuance or sale of stock	$ 188,900
Common Stock \| Pre-Funded Warrants
Private Placement At-the-Market Offering Program and Public Offerings [Abstract]
Number of stock warrants issued to purchase shares \| shares	3,100,000		3,126,955
Warrant to purchase shares of common stock price per share (in dollars per share) \| $ / shares	$ 15.99		$ 0.06
Common Stock \| At-the-Market Offering Program
Private Placement At-the-Market Offering Program and Public Offerings [Abstract]
Issuance of common stock, net of issuance costs (in shares) \| shares			900,000
Net proceeds from offering			$ 17,200
Common Stock \| At-the-Market Offering Program \| Cowen and Company, LLC
Private Placement At-the-Market Offering Program and Public Offerings [Abstract]
Issuance of common stock, net of issuance costs (in shares) \| shares			4,200,000
Net proceeds from offering			$ 63,800
Treasury Stock, Common
Treasury Stock [Abstract]
Shares withheld to pay the payroll tax liability				$ 50
Treasury stock (in shares) \| shares			0

Warrants - Summary of Changes in Rocket Warrants to Purchase Common Stock (Details) - $ / shares	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022
Warrants
Number of Warrant Shares Outstanding and Exercisable [Abstract]
Beginning balance (in shares)	2,721,267	1,218,038
Potential shares included in the calculation of EPS	3,126,955
Exercised (in shares)	(126,093)
Expired (in shares)	(7,051)
Ending balance (in shares)	5,715,078	2,721,267
Exercise Price Per Share, Warrants [Abstract]
Warrants exercise price per share	$ 0.01
Exercised (in dollars per share)	0.06
Expired (in dollars per share)	$ 24.42
Public Warrants
Number of Warrant Shares Outstanding and Exercisable [Abstract]
Assumed equity (in shares)		760,086
Exercise Price Per Share, Warrants [Abstract]
Assumed equity (in dollars per share)		$ 65.23
Private Warrants
Number of Warrant Shares Outstanding and Exercisable [Abstract]
Assumed liability (in shares)		617,050
Exercise Price Per Share, Warrants [Abstract]
Assumed liability (in dollars per share)		$ 65.23
Pre-Funded Warrants
Number of Warrant Shares Outstanding and Exercisable [Abstract]
Assumed equity (in shares)		126,093
Potential shares included in the calculation of EPS	3,126,955
Exercise Price Per Share, Warrants [Abstract]
Assumed equity (in dollars per share)		$ 0.06
Warrants exercise price per share	$ 0.01

Property and Equipment, Net - Additional Information (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Property and Equipment, Net [Abstract]
Depreciation and amortization	$ 4.9	$ 3.9	$ 3.2

Consolidated Balance Sheets - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Current assets:
Cash and cash equivalents	$ 55,904	$ 140,517
Investments	317,271	215,877
Prepaid expenses and other current assets	5,047	7,666
Total current assets	378,222	364,060
Property and equipment, net	39,172	29,009
Goodwill	39,154	39,154
Intangible assets	25,150	25,724
Restricted cash	1,372	1,340
Deposits	533	608
Investments	34,320	43,276
Operating lease right-of-use assets, net	3,901	1,972
Finance lease right-of-use asset, net	44,517	46,664
Total assets	566,341	551,807
Current liabilities:
Accounts payable and accrued expenses	45,789	36,660
Operating lease liabilities, current	925	773
Finance lease liability, current	1,791	1,736
Total current liabilities	48,505	39,169
Operating lease liabilities, non-current	2,973	1,088
Finance lease liability, non-current	19,353	19,269
Other liabilities	2,936	2,595
Total liabilities	73,767	62,121
Commitments and contingencies (Note 14)
Stockholders' equity:
Common stock, $0.01 par value, 120,000,000 shares authorized; 90,282,267 and 79,123,312 shares issued and 90,282,267 and 79,120,741 shares outstanding at December 31, 2023 and December 31, 2022, respectively	903	791
Treasury stock, at cost, 0 common shares at December 31, 2023 and 2,571 common shares at December 31, 2022	0	(47)
Additional paid-in capital	1,450,722	1,203,074
Accumulated other comprehensive income (loss)	319	(357)
Accumulated deficit	(959,370)	(713,775)
Total stockholders' equity	492,574	489,686
Total liabilities and stockholders' equity	566,341	551,807
Series A Convertible Preferred Shares
Stockholders' equity:
Preferred stock
Series B Convertible Preferred Shares
Stockholders' equity:
Preferred stock

Stock-Based Awards - Stock Option Activity (Details) - Employee Stock Option - USD ($) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding [Roll Forward]
Outstanding at beginning of period (in shares)	13,138,870	11,143,761
Granted (in shares)	2,370,862	2,305,910
Exercised (in shares)	(223,145)	(66,887)
Cancelled (in shares)	(422,591)	(611,766)
Outstanding at end of period (in shares)	14,863,996	13,138,870	11,143,761
Options vested and exercisable at end of period (in shares)	11,657,122
Options unvested at ending of period (in shares)	3,206,874
Weighted Average Exercise Price [Abstract]
Outstanding at beginning of period (in dollars per share)	$ 14.52	$ 14.51
Granted (in dollars per share)	20.24	13.94
Exercised (in dollars per share)	10	9.43
Cancelled (in dollars per share)	29.82	32.55
Outstanding at end of period (in dollars per share)	15.07	$ 14.52	$ 14.51
Options vested and exercisable at end of period (in dollars per share)	13.57
Options unvested at ending (in dollars per share)	$ 20.5
Weighted-Average Remaining Contractual Term [Abstract]
Outstanding	5 years 1 month 28 days	5 years 5 months 15 days	5 years 11 months 12 days
Granted	8 years 9 months 3 days	5 years 6 months 10 days
Options vested and exercisable	4 years 1 month 17 days
Options unvested	8 years 10 months 28 days
Aggregate Intrinsic Value [Abstract]
Outstanding at beginning of period	$ 118,767	$ 128,817
Exercised	2,318	514
Outstanding at end of period	250,602	$ 118,767	$ 128,817
Options vested and exercisable	217,871
Options unvested	$ 32,731
Renovacor, Inc.
Share-Based Compensation Arrangement by Share-Based Payment Award, Options, Outstanding [Roll Forward]
Conversion of awards (in shares)		367,852
Weighted Average Exercise Price [Abstract]
Conversion of awards (in dollars per share)		$ 4.63
Weighted-Average Remaining Contractual Term [Abstract]
Conversion of awards		4 months 24 days

Renovacor Acquisition (Tables)

12 Months Ended

Dec. 31, 2023

Renovacor Acquisition [Abstract]

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 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 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

Leases - Operating and Financing Lease Liabilities and Right-of-use Assets (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Mar. 31, 2023	Dec. 31, 2022
Assets and Liabilities, Lessee [Abstract]
Operating right-of-use assets	$ 3,901	$ 200	$ 1,972
Operating current lease liabilities	925		773
Operating noncurrent lease liabilities	2,973	$ 200	1,088
Total operating lease liabilities	3,898		1,861
Finance right-of-use assets	44,517		46,664
Finance current lease liability	1,791		1,736
Finance noncurrent lease liability	19,353		19,269
Total finance lease liability	$ 21,144		$ 21,005

Intangible Assets and Goodwill - Summary of Carrying Value of Goodwill (Details) $ in Thousands	12 Months Ended
	Dec. 31, 2022 USD ($)
Intangible Assets and Goodwill [Abstract]
Goodwill, Beginning Balance	$ 30,815
Acquisition of Renovacor	8,339
Goodwill, Ending Balance	$ 39,154

Warrants Outstanding and Changes in Warrants to Purchase Common Stock

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

Warrants (Tables)

12 Months Ended

Dec. 31, 2023

Warrants [Abstract]

A summary of the Company’s outstanding warrants at December 31, 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 below is the summary of changes in the Company’s warrants to purchase common stock for the years ended December 31, 2023 and 2022:


	Number of Warrant Shares
	Outstanding and Exercisable			Exercise Price per Share
Balance as of December 31, 2021		1,218,038
Assumed Renovacor Private warrants - liability		617,050		$	65.23
Assumed Renovacor Public warrants - equity		760,086			65.23
Assumed Renovacor Pre-funded warrants - equity		126,093			0.06
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 December 31, 2023		5,715,078

Summary of Significant Accounting Policies, Tax Credits (Details) - USD ($)	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
New York State Life Sciences Research and Development Tax Credit [Member]
Tax Credits [Abstract]
Research and development incentive income	$ 0	$ 500,000	$ 1,000,000

Consolidated Statements of Cash Flows - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Operating activities:
Net loss	$ (245,595)	$ (221,863)	$ (169,069)
Adjustments to reconcile net loss to net cash used in operating activities:
Accretion of discount on convertible notes	0	0	753
Depreciation and amortization of property and equipment	4,944	3,932	3,240
Amortization of finance lease right of use asset	2,154	2,334	2,133
Impairment of acquired intangible asset	574	0	0
Write down of property and equipment	311	236	261
Stock-based compensation	39,406	31,009	29,237
Accretion of discount and amortization of premium on investments, net	(10,168)	134	2,887
Expense in connection with warrant issuance	0	0	12,781
Changes in operating assets and liabilities:
Prepaid expenses and other assets	2,694	(3,593)	1,307
Accounts payable and accrued expenses	10,147	9,674	(4,827)
Operating lease liability and right of use asset, net	137	(120)	(11)
Finance lease liability	139	172	201
Other liabilities	341	(57)	(56)
Net cash used in operating activities	(194,916)	(178,142)	(121,163)
Investing activities:
Purchases of investments	(390,920)	(376,327)	(245,875)
Proceeds from maturities of investments	309,326	272,894	272,443
Cash proceeds from acquisition of business, net of cash paid	0	42,726	0
Payments made to acquire right of use asset	(36)	(261)	(95)
Purchases of property and equipment	(16,436)	(8,358)	(7,620)
Net cash (used in) provided by investing activities	(98,066)	(69,326)	18,853
Financing activities:
Issuance of common stock, pursuant to exercise of stock options	2,231	631	11,327
Treasury stock repurchase	0	(47)	0
Issuance of common stock, pursuant to sale of treasury stock	56	0	0
Issuance of common stock, pursuant to exercise of warrants	7	0	0
Net cash provided by financing activities	208,401	155,288	37,681
Net change in cash, cash equivalents and restricted cash	(84,581)	(92,180)	(64,629)
Cash, cash equivalents and restricted cash at beginning of period	141,857	234,037	298,666
Cash, cash equivalents and restricted cash at end of period	57,276	141,857	234,037
Supplemental disclosure of non-cash financing and investing activities:
Accrued purchases of property and equipment, ending balance	1,077	2,095	728
Operating lease liabilities	2,929	0	0
Unrealized gain (loss) on investments	676	(196)	(119)
Conversion of convertible notes into common stock	0	0	40,694
Issuance of common stock related to acquisition	0	70,724	0
Reclassification of construction in process from finance right of use asset	0	261	98
Supplemental cash flow information:
Cash paid for interest	0	0	148
Public Offering
Financing activities:
Issuance of common stock, net of issuance costs	188,885	108,138	26,354
At-the-Market Offering Program
Financing activities:
Issuance of common stock, net of issuance costs	$ 17,222	$ 46,566	$ 0

Summary of Significant Accounting Policies, Goodwill (Details) $ in Thousands	12 Months Ended
	Dec. 31, 2023 USD ($) Segment	Dec. 31, 2022 USD ($)	Dec. 31, 2021 USD ($)
Goodwill [Abstract]
Number of operating segment	1
Number of reporting unit	1
Goodwill impairment \| $	$ 0	$ 0	$ 0

Renovacor Acquisition

12 Months Ended

Dec. 31, 2023

Renovacor Acquisition [Abstract]

Renovacor Acquisition

17.

Renovacor Acquisition

On September 19, 2022, the Company entered into an Agreement and Plan of Merger (the “Merger Agreement”) with Renovacor, a Delaware corporation pursuant to which, on December 1, 2022, the Company acquired Renovacor (the “Renovacor Acquisition”). On December 1, 2022, pursuant to the terms of the Merger Agreement, (i) Merger Sub I merged with and into the Company (the “First Merger”) and (ii) the Company, as the surviving company of the First Merger merged with and into Merger Sub II (the “Second Merger”), with Merger Sub II surviving the Second Merger. Subject to the terms and conditions of the Merger Agreement, at the closing of the Renovacor Acquisition each share of Renovacor’s common stock outstanding immediately prior to the effective time of the First Merger were canceled and converted into the right to receive 0.1763 (the “Exchange Ratio”) of fully paid and non-assessable shares of the Company common stock, which was determined on the basis of the exchange formula set forth in the Merger Agreement that was subject to adjustment depending on the level of the Renovacor’s net cash at the closing. Prior to the market opening on December 1, 2022, Renovacor shares ceased to trade on NYSE and upon the closing of the acquisition, Renovacor’s outstanding common stock were converted into 3,391,976 shares of Rocket common stock.

Total consideration for the Renovacor Acquisition was $72.3 million, consisting of $62.4 million for common stock outstanding, $2.7 million for the portion of equity compensation attributable to the pre-combination service period, and $7.2 million for assumed warrants. The consideration was based on the estimated fair values on the acquisition date of (i) 3,391,976 common shares issued for shares outstanding for common shares of Renovacor, (ii) estimated fair value of employee stock options to acquire 367,852 common shares of the Company, (iii) 28,798 common shares issued for employee time-vesting RSUs, and (iv) warrants to acquire 1,503,229 common shares (see Note 10 “Warrants”).

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.

The acquisition has been accounted for as a business combination using the acquisition method of accounting which requires that assets acquired and liabilities assumed be recognized at their fair values as of the acquisition date and that the fair value of acquired IPR&D assets are classified as indefinite-life assets until the successful completion or abandonment of the associated research and development efforts.


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

The fair value assigned to acquired IPR&D was based on the present value of expected after-tax cash flows attributable to Renovacor’s most advanced AAV-based gene therapy targeting BAG3-DCM. The present value of expected after-tax cash flows was determined by estimating the after-tax costs to complete development into a commercially viable product, estimating future revenue and ongoing expenses to produce, and discounting the resulting net cash flows to present value. The cost and revenue projections used were reduced based on the assessed probabilities of different stages of development. Acquired IPR&D will be accounted for as an indefinite-lived intangible asset until regulatory approval in a major market or discontinuation of development.

The excess of purchase price over the fair value of amounts assigned to identifiable assets acquired and liabilities assumed represents the goodwill amount of $8.3 million resulting from the acquisition. The goodwill recorded as part of the acquisition is primarily attributable to the broadening of the Company’s portfolio and research capabilities, deferred taxes and the assembled workforce. The goodwill attributable to the acquisition has been recorded as a non-current asset in the Company’s consolidated balance sheet and is not amortized, but subject to review for impairment annually. The Company incurred $3.2 million of acquisition related general and administrative costs during the year ended December 31, 2022.

Pay vs Performance Disclosure - USD ($) $ in Thousands	12 Months Ended
Pay vs Performance Disclosure - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Pay vs Performance Disclosure
Net Income (Loss)	$ (245,595)	$ (221,863)	$ (169,069)

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

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Reference 36: http://www.xbrl.org/2003/role/disclosureRef
-Topic 220
-SubTopic 10
-Name Accounting Standards Codification
-Section 45
-Paragraph 1A
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482790/220-10-45-1A

Reference 37: http://www.xbrl.org/2003/role/disclosureRef
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-URI https://asc.fasb.org//1943274/2147482790/220-10-45-1B

Reference 38: http://fasb.org/us-gaap/role/ref/legacyRef
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-SubTopic 10
-Name Accounting Standards Codification
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-Subparagraph (SX 210.5-03(20))
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-URI https://asc.fasb.org//1943274/2147483621/220-10-S99-2

Reference 39: http://fasb.org/us-gaap/role/ref/legacyRef
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-Subparagraph (SX 210.9-04(22))
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Consolidated Statements of Operations - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Consolidated Statements of Operations [Abstract]
Revenue	$ 0	$ 0	$ 0
Operating expenses:
Research and development	186,342	165,570	125,476
General and administrative	73,317	58,773	41,772
Total operating expenses	259,659	224,343	167,248
Loss from operations	(259,659)	(224,343)	(167,248)
Research and development incentives		500	1,000
Interest expense	(1,875)	(1,862)	(2,977)
Interest and other income, net	5,288	3,889	3,068
Accretion of discount and amortization of premium on investments, net	10,651	(47)	(2,912)
Net loss	$ (245,595)	$ (221,863)	$ (169,069)
Net loss per share attributable to common stockholders - basic (in dollars per share)	$ (2.92)	$ (3.26)	$ (2.67)
Net loss per share attributable to common stockholders - diluted (in dollars per share)	$ (2.92)	$ (3.26)	$ (2.67)
Weighted-average common shares outstanding - basic (in shares)	84,009,004	68,148,925	63,235,417
Weighted-average common shares outstanding - diluted (in shares)	84,009,004	68,148,925	63,235,417

Leases - Maturity of Finance Lease Liability (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Finance Lease, Liability, to be Paid, Fiscal Year Maturity [Abstract]
2024	$ 1,791
2025	1,856
2026	1,912
2027	1,969
2028	2,028
Thereafter	41,003
Total lease payments	50,559
Less: interest	(29,415)
Total finance lease liability	$ 21,144	$ 21,005

401(k) Savings Plan	12 Months Ended
401(k) Savings Plan	Dec. 31, 2023
401(k) Savings Plan [Abstract]
401(k) Savings Plan	19. 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 years ended December 31, 2023, 2022, and 2021 was $1.4 million, $0.9 million, and $0.6 million, respectively.

Insider Trading Arrangements	3 Months Ended
Insider Trading Arrangements	Dec. 31, 2023
Trading Arrangements, by Individual
Rule 10b5-1 Arrangement Adopted	false
Non-Rule 10b5-1 Arrangement Adopted	false
Rule 10b5-1 Arrangement Terminated	false
Non-Rule 10b5-1 Arrangement Terminated	false

Stock-Based Awards - Additional Information (Details) - USD ($) $ / shares in Units, $ in Millions	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Weighted average grant date fair value of shares granted (in dollars per share)	$ 13.37
Employee Stock Option
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Weighted average grant date fair value of shares granted (in dollars per share)		$ 9.88	$ 31.07
Total fair value of options vested	$ 26.5	$ 34.9	$ 22.6
Restricted Stock Units
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Intrinsic value	6.8	$ 0.8	$ 0.4
Stock Options and Restricted Stock Units Grants
Share-based Compensation Arrangement by Share-based Payment Award [Line Items]
Unrecognized stock-based compensation expense	$ 52.5
Weighted average period expected to recognize unrecognized share-based compensation cost	1 year 9 months 29 days

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.

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
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Intangible Assets and Goodwill [Abstract]

Intangible Assets and Goodwill

12 Months Ended

Dec. 31, 2023

Intangible Assets and Goodwill

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


December 31, 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 Company holds intangible assets as a result of the acquisition of Renovacor (see Note 17 “Renovacor Acquisition”). The decrease in gross carrying value of intangibles at December 31, 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 December 31, 2023 and 2022 was $39.2 million and included $8.3 million as a result of the acquisition of Renovacor in 2022 (see Note 17 “Renovacor Acquisition”):


	Carrying Value
Balance, December 31, 2021	$	30,815
Acquisition of Renovacor		8,339
Balance, December 31, 2022 and 2023	$	39,154

Summary of Significant Accounting Policies (Policies)

12 Months Ended

Dec. 31, 2023

Summary of Significant Accounting Policies [Abstract]

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 U.S. (“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 and intangible asset impairments, the accrual of research and development (“R&D”) expenses, the valuation of equity transactions, and stock-based awards. Changes in estimates and assumptions are reflected in reported results in the period in which they become known. Actual results could differ from those estimates.

Cash, Cash Equivalents and Restricted Cash

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

Restricted cash consists of deposits collateralizing letters of credit issued by a bank in connection with the Company’s operating leases (see Note 13 “Leases” for additional disclosures) and a deposit collateralizing a letter of credit issued by a bank supporting the Company’s corporate credit card. Cash, cash equivalents and restricted cash consist of the following:


	December 31, 2023		December 31, 2022
Cash and cash equivalents	$	55,904	$	140,517
Restricted cash		1,372		1,340
Total cash, cash equivalents and restricted cash	$	57,276	$	141,857

Government Grants

Research and development expense was presented net of reimbursements from CIRM (See Note 16 “CIRM Grant” for additional disclosure).

Concentrations of Credit Risk and Off-balance Sheet Risk

Concentrations of credit risk and off-balance sheet risk

Financial instruments that subject the Company to credit risk primarily consist of cash and cash equivalents and available-for-sale securities. The Company maintains its cash and cash equivalent balances with high-quality financial institutions and, consequently, the Company believes that such funds are subject to minimal credit risk. The Company’s marketable securities consist of U.S. Treasury Securities, Commercial Paper and Corporate and Agency Bonds. The Company’s investment policy limits the amounts the Company may invest in any one type of investment and requires all investments held by the Company to be at least AA-/Aa3 rated, thereby reducing credit risk exposure.

Investments

Investments consist of U.S. Treasury Securities and Corporate Bonds. Management determines the appropriate classification of these securities at the time they are acquired and evaluates the appropriateness of such classifications at each balance sheet date. The Company classifies its investments as available-for-sale pursuant to Financial Accounting Standards Board (“FASB”) Accounting Standard Codification (“ASC”) 320, Investments—Debt and Equity Securities. Investments are recorded at fair value, with unrealized gains and losses included as a component of accumulated other comprehensive income (loss) in stockholders’ equity and a component of total comprehensive loss in the consolidated statements of comprehensive loss, until realized. Realized gains and losses are included in investment income on a specific-identification basis. The Company estimates expected credit losses for investments when unrealized losses exist. Unrealized losses that are credit related are recognized in the Company’s consolidated statement of operations and unrealized losses that are not credit related are recognized in accumulated other comprehensive income (loss). There were no realized gains or losses on investments for the years ended December 31, 2023, 2022 and 2021. For the year ended December 31, 2023 there was net unrealized gains on investments of $0.7 million. For the years ended December 31, 2022 and 2021, there were net unrealized losses on investments of $0.2 million and $0.1 million, respectively.

Intangible Assets

Intangible assets consisted of an indefinite lived intangible IPR&D asset and a mice colony model. Intangible assets related to IPR&D projects are considered to be indefinite-lived until the completion or abandonment of the associated R&D efforts. If and when development is complete, which generally occurs if and when regulatory approval to market a product is obtained, the associated assets would be deemed finite-lived and would then be amortized based on their respective estimated useful lives at that point in time. IPR&D intangible assets which are determined to have had a decrease in their fair value are adjusted downward and an expense is recognized in R&D expenses in the Consolidated Statements of Operations. These IPR&D intangible assets are tested at least annually or when a triggering event occurs that could indicate a potential impairment based on indicators including progress of R&D activities, changes in projected development of assets, and changes in regulatory environment and future commercial markets. If a triggering event occurs that would indicate a potential impairment, the Company will perform a quantitative analysis to determine whether it is more likely than not that the fair value is below carrying amount. If a triggering event occurs that would indicate a potential impairment, the Company will perform a quantitative analysis to determine whether it is more likely than not that the fair value is below carrying amount. The annual impairment assessment for the IPR&D asset related to the Renovacor acquisition was performed as of December 1, 2023. No impairment of the IPR&D asset was recorded for the years ended December 31, 2023 and 2022. The mice colony model was impaired and written off during the year ended December 31, 2023.

Impairment of Long-Lived Assets

Business combinations are accounted for under the acquisition method. The total cost of an acquisition is allocated to the underlying identifiable net assets, based on their respective estimated fair values as of the acquisition date. Determining the fair value of assets acquired and liabilities assumed requires management’s judgment and often involves the use of significant estimates and assumptions, including assumptions with respect to future cash inflows and outflows, discount rates, asset lives and market multiples, among other items. Assets acquired and liabilities assumed are recorded at their estimated fair values. The excess of the purchase price over the estimated fair values of the net assets acquired is recorded as goodwill. Goodwill is tested for impairment annually as of December 31, or more frequently when events or changes in circumstances indicate that the asset might be impaired. Examples of such events or circumstances include, but are not limited to, a significant adverse change in legal or business climate, an adverse regulatory action or unanticipated competition. The Company has one segment and one reporting unit and as such review’s goodwill for impairment at the consolidated level. When testing goodwill, the Company has the option to first assess qualitative factors for reporting units that carry goodwill. The qualitative assessment includes assessing the totality of relevant events and circumstances that affect the fair value or carrying value of the reporting unit. These events and circumstances include macroeconomic conditions, industry and competitive environment conditions, overall financial performance, reporting unit specific events and market considerations. The Company also considers recent valuations of the reporting unit, including the magnitude of the difference between the most recent fair value estimate and the carrying value, as well as both positive and adverse events and circumstances, and the extent to which each of the events and circumstances identified may affect the comparison of a reporting unit’s fair value with its carrying value. If the qualitative assessment results in a conclusion that it is more likely than not that the fair value of a reporting unit exceeds the carrying value, then no further testing is performed for that reporting unit.

The Company performed the qualitative assessment of its goodwill and determined that it is more likely than not that the fair value of a reporting unit exceeds the carrying value of the reporting unit. As a result, the Company has determined there was no goodwill impairment as of and for the years ended December 31, 2023, 2022 and 2021.

Property and Equipment

Property and Equipment, Net

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

The Company reviews long-lived assets to be held and used for impairment whenever events or changes in circumstances indicate that the asset’s carrying amount may not be recoverable. The Company conducted its impairment analyses of long-lived assets to be held and used in accordance with ASC 360-10-15, Impairment or Disposal of Long-Lived Assets. ASC 360-10-15 requires the Company to group assets and liabilities at the lowest level for which identifiable cash flows are largely independent of the cash flows of other assets and liabilities and evaluate the asset group against the sum of the undiscounted future cash flows. If the undiscounted cash flows do not indicate the carrying amount of the asset is recoverable, an impairment charge is measured as the amount by which the carrying amount of the asset group exceeds its fair value based on discounted cash flow analysis or appraisals. The Company recorded an impairment of the intangible long-lived asset related to the mice colony model of approximately $0.6 million during the year ended December 31, 2023. The Company also recorded write downs of property and equipment in each of the years ended December 31, 2023, 2022 and 2021.

Fair Value Measurements

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

•

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

•

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

•

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

To the extent that valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized in Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement. The fair value of the Company’s financial instruments, including cash and cash equivalents, restricted cash, deposits, accounts payable and accrued expenses approximate their respective carrying values due to the short-term nature of most of these instruments.

Warrants

The Company accounts for stock warrants as either equity instruments, liabilities or derivative liabilities in accordance with ASC Topic 480, Distinguishing Liabilities from Equity and/or ASC Topic 815, Derivatives and Hedging, depending on the specific terms of the warrant agreement. Liability-classified warrants are recorded at their estimated fair values at each reporting period until they are exercised, terminated, reclassified or otherwise settled. Changes in the estimated fair value of liability-classified warrants are included in interest and other income in the Company’s consolidated statements of operations. Equity-classified warrants are recorded within additional paid-in capital at the time of issuance and are not subject to remeasurement.

Research and Development Expenses

R&D expenses, which include salaries and staff costs, license costs, manufacturing and development costs, clinical trial expenses, depreciation and amortization expenses, regulatory and scientific consulting fees, as well as contract research, and stock-based compensation expense, are accounted for in accordance with ASC Topic 730, Research and Development. Accordingly, R&D costs are expensed as incurred.

Foreign Currency Transactions

Certain transactions during the years ended December 31, 2023, 2022 and 2021 are primarily denominated in Euros and British pounds. Gains and losses on foreign currency transactions were not significant for the years ended December 31, 2023, 2022 and 2021.

Treasury Stock

The Company records treasury stock at cost.

Stock-Based Compensation

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

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

Tax Credits and Income Taxes

New York State Life Sciences Research and Development Tax Credit

New York State (“NYS”) allows investors and owners of emerging technology companies focused on biotechnology to claim a tax credit against their NYS Tax return for certain expenditures incurred in NYS, including applicable R&D related expenditures. The credit is recognized as R&D incentives when the eligibility and amount has been approved by NYS. During the years ended December 31, 2023, 2022 and 2021, the Company recorded R&D incentive income of $0, $0.5 million, and $1.0 million, respectively related to the NYS Life Sciences Research and Development Tax Credit.

Income Taxes

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

The Company’s deferred tax assets relate primarily to its net operating loss carryforwards and other balance sheet differences. In accordance with ASC 740, Income Taxes, the Company recorded a full valuation allowance to fully offset the net deferred tax asset because it is not more likely than not that the Company will realize future benefits associated with these deferred tax assets at December 31, 2023 and 2022.

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

Net Loss Per Share

The Company calculates net loss per share in accordance with FASB ASC 260, Earnings per Share. Basic net loss per share attributable to common shareholders is computed by dividing the net loss attributable to common shareholders by the weighted average number of common shares outstanding for the period. Warrants that meet the definition of equity classification and that requires the holder to pay little or no consideration to receive shares upon exercise are considered outstanding in the context of basic earnings per share. Diluted net loss attributable to common shareholders is computed by adjusting net loss attributable to common shareholders to reallocate undistributed earnings based on the potential impact of dilutive securities. Diluted net loss per share attributable to common shareholders is computed by dividing the diluted net loss attributable to common shareholders by the weighted average number of common shares outstanding for the period, including potential dilutive common shares. For purposes of this calculation, outstanding options are considered potential dilutive common shares.

Segment Reporting

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

Comprehensive Loss

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

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.

Recent Accounting Pronouncements

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

Accounting Pronouncements Not Adopted as of December 31, 2023

ASU 2023-09: Income Taxes Topic 740 - Improvements to Income Tax Disclosures. This update standardizes categories for the effective tax rate reconciliation, requires disaggregation of income taxes and additional income tax-related disclosures. This update is required to be effective for the Company for fiscal periods beginning after December 15, 2024. As this accounting standard only impacts disclosures, it will not have a material impact on the Company's Consolidated Financial Statements.

ASU 2023-07: Segment Reporting Topic 280 - Improvements to Reportable Segment Disclosures. This update requires expanded annual and interim disclosures for significant segment expenses that are regularly provided to the chief operating decision maker and included within each reported measure of segment profit or loss. This update will be effective for fiscal years beginning after December 15, 2023, and is to be applied retrospectively to all periods presented in the financial statements. Early adoption is permitted. As the Company does not have segments and this accounting standard only impacts disclosures, it will not have a material impact on the Company’s consolidated financial statements.

Income Taxes - Additional Information (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2017
Operating Loss Carryforwards [Abstract]
Provision for income taxes	$ 0
R&D credits	20,984	$ 20,984	$ 35,766
Increase (decrease) in valuation allowance	17,200	(2,100)
Annual limitations under section 382 of IRC	$ 1,700
Markup interest rate	10.00%
Uncertain Tax Positions [Abstract]
Uncertain tax positions	$ 0	0	0
Income Tax Penalties and Interest Accrued [Abstract]
Accrued interest or penalties	0	0	0
Federal [Member]
Operating Loss Carryforwards [Abstract]
Provision for income taxes		0	0
NOL carryforwards	$ 197,700			$ 127,100
NOL carryforwards, expiration date	Dec. 31, 2026
R&D credits	$ 21,000
Tax credit, expiration date	Dec. 31, 2038
Net operating loss, subject to expiration	$ 197,700			$ 91,200
Cumulative limitation period	20 years
Cumulative net operating loss, subject to expiration	$ 35,800
Federal [Member] \| Renovacor, Inc. [Member]
Operating Loss Carryforwards [Abstract]
NOL carryforwards	46,400
Federal [Member] \| Research and Development [Member]
Tax Credit Carryforward [Abstract]
Tax credit carryforwards, subject to expiration	$ 4,900
State [Member]
Operating Loss Carryforwards [Abstract]
Provision for income taxes		$ 0	$ 0
NOL carryforwards, expiration date	Dec. 31, 2026
Net operating loss, subject to expiration	$ 77,200

Stock-Based Awards (Tables)

12 Months Ended

Dec. 31, 2023

Stock-Based Awards [Abstract]

Weighted-Average Assumptions for Stock Options

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


	For the Years Ended December 31,
	2023			2022			2021
Risk-free interest rate		4.00	%		2.19	%		0.83	%
Expected term (in years)		5.82			5.09			5.84
Expected volatility		73.32	%		64.12	%		69.27	%
Expected dividend yield		0.00	%		0.00	%		0.00	%
Exercise price	$	20.24		$	18.56		$	51.20
Fair value of common stock	$	20.24		$	18.56		$	51.20

Stock Option Activity

The following table summarizes stock option activity for the years ended December 31, 2023 and 2022:


				Weighted		Weighted
				Average		Average		Aggregate
	Number of			Exercise		Contractual		Intrinsic
	Shares			Price		Term (Yrs)		Value
Outstanding as of December 31, 2021		11,143,761		$	14.51		5.95	$	128,817
Conversion of Renovacor awards		367,852			4.63		0.40
Granted		2,305,910			13.94		5.53
Exercised		(66,887	)		9.43				514
Cancelled or forfeited		(611,766	)		32.55
Outstanding as of December 31, 2022		13,138,870		$	14.52		5.46	$	118,767
Granted		2,370,862			20.24		8.76
Exercised		(223,145	)		10.00				2,318
Cancelled or forfeited		(422,591	)		29.82
Outstanding as of December 31, 2023		14,863,996		$	15.07		5.16	$	250,602

Options vested and exercisable as of December 31, 2023		11,657,122		$	13.57		4.13	$	217,871
Options unvested as of December 31, 2023		3,206,874		$	20.50		8.91	$	32,731

RSU Activity

The following table summarizes the RSU activity for the years ended December 31, 2023 and 2022:


				Weighted Average
	Number of			Grant Date
	Shares			Fair Value
Unvested as of December 31, 2021		23,500		$	30.61
Conversion of Renovacor awards		28,798			0.49
Granted		1,047,301			15.91
Vested		(38,966	)		23.15
Forfeited		(67,759	)		15.94
Unvested as of December 31, 2022		992,874		$	16.49
Granted		1,018,322			19.67
Vested (1)		(408,119	)		16.61
Forfeited		(112,720	)		17.92
Unvested as of December 31, 2023		1,490,357		$	18.53

(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 is as follows:


	For the Years Ended December 31,
	2023		2022		2021
Stock options	$	29,091	$	27,620	$	28,811
Restricted stock units		10,315		3,389		426
Total stock-based compensation expense	$	39,406	$	31,009	$	29,237

Stock-Based Compensation Expense

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


	For the Years Ended December 31,
	2023		2022		2021
Research and development	$	17,509	$	12,466	$	11,954
General and administrative		21,897		18,543		17,283
Total stock-based compensation expense	$	39,406	$	31,009	$	29,237

Leases - Leases Cost (Details) - USD ($) $ in Thousands	12 Months Ended
Leases - Leases Cost (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Lease cost [Abstract]
Operating lease cost	$ 1,377	$ 818	$ 645
Finance Lease Costs [Abstract]
Amortization of right of use assets	2,154	2,139	2,140
Interest on lease liabilities	1,875	1,861	1,845
Total lease cost	$ 5,406	$ 4,818	$ 4,630

Stock-Based Awards

12 Months Ended

Dec. 31, 2023

Stock-Based Awards [Abstract]

Stock-Based Awards

Stock Option Valuation


	For the Years Ended December 31,
	2023			2022			2021
Risk-free interest rate		4.00	%		2.19	%		0.83	%
Expected term (in years)		5.82			5.09			5.84
Expected volatility		73.32	%		64.12	%		69.27	%
Expected dividend yield		0.00	%		0.00	%		0.00	%
Exercise price	$	20.24		$	18.56		$	51.20
Fair value of common stock	$	20.24		$	18.56		$	51.20

The following table summarizes stock option activity for the years ended December 31, 2023 and 2022:


				Weighted		Weighted
				Average		Average		Aggregate
	Number of			Exercise		Contractual		Intrinsic
	Shares			Price		Term (Yrs)		Value
Outstanding as of December 31, 2021		11,143,761		$	14.51		5.95	$	128,817
Conversion of Renovacor awards		367,852			4.63		0.40
Granted		2,305,910			13.94		5.53
Exercised		(66,887	)		9.43				514
Cancelled or forfeited		(611,766	)		32.55
Outstanding as of December 31, 2022		13,138,870		$	14.52		5.46	$	118,767
Granted		2,370,862			20.24		8.76
Exercised		(223,145	)		10.00				2,318
Cancelled or forfeited		(422,591	)		29.82
Outstanding as of December 31, 2023		14,863,996		$	15.07		5.16	$	250,602

Options vested and exercisable as of December 31, 2023		11,657,122		$	13.57		4.13	$	217,871
Options unvested as of December 31, 2023		3,206,874		$	20.50		8.91	$	32,731

The weighted average grant-date fair value per share of stock options granted during the years ended December 31, 2023, 2022 and 2021 was $13.37, $9.88, and $31.07, respectively.

The total fair value of options vested during the years ended December 31, 2023, 2022 and 2021 was $26.5 million, $34.9 million and $22.6 million, respectively.

Restricted Stock Units (“RSU”)

The following table summarizes the RSU activity for the years ended December 31, 2023 and 2022:


				Weighted Average
	Number of			Grant Date
	Shares			Fair Value
Unvested as of December 31, 2021		23,500		$	30.61
Conversion of Renovacor awards		28,798			0.49
Granted		1,047,301			15.91
Vested		(38,966	)		23.15
Forfeited		(67,759	)		15.94
Unvested as of December 31, 2022		992,874		$	16.49
Granted		1,018,322			19.67
Vested (1)		(408,119	)		16.61
Forfeited		(112,720	)		17.92
Unvested as of December 31, 2023		1,490,357		$	18.53

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

The total fair value of RSU’s vested during the years ended December 31, 2023, 2022 and 2021 was $6.8 million, $0.8 million, and $0.4 million, respectively.

Stock-Based Compensation

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


	For the Years Ended December 31,
	2023		2022		2021
Stock options	$	29,091	$	27,620	$	28,811
Restricted stock units		10,315		3,389		426
Total stock-based compensation expense	$	39,406	$	31,009	$	29,237

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


	For the Years Ended December 31,
	2023		2022		2021
Research and development	$	17,509	$	12,466	$	11,954
General and administrative		21,897		18,543		17,283
Total stock-based compensation expense	$	39,406	$	31,009	$	29,237

As of December 31, 2023, the Company had an aggregate of $52.5 million of unrecognized stock-based compensation expense, which is expected to be recognized over the weighted average period of 1.83 years.

Related Party Transactions

12 Months Ended

Dec. 31, 2023

Related Party Transactions

18.

Related Party Transactions

On December 21, 2020, the Company entered into a consulting agreement with a related party. Pursuant to the consulting agreement, the related party provides certain business development and asset identification consulting services to the Company. On August 9, 2021, the Company issued a warrant exercisable for 301,291 shares of common stock to the same related party for business development and asset identification consulting services (“August 2021 Warrant”). The Company recorded a non-cash R&D expense of $7.6 million during year ended December 31, 2021, related to the issuance of the August 2021 warrant. On December 17, 2021, the Company issued warrants exercisable for 153,155 and 153,155 shares of common stock, respectively to the same related party for business development and asset identification consulting services (“December 2021 Warrants”). The Company recorded a non-cash R&D expense of $5.2 million during year ended December 31, 2021, related to the issuance of the December 2021 warrant. Total non-cash R&D expense of $12.8 million during the year ended December 31, 2021, related to the issuance of the August 2021 and December 2021 warrants.

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

In 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 incurred expenses of approximately $0.02 million for the year ended December 31, 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.

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
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Reference 2: http://www.xbrl.org/2003/role/disclosureRef
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Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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Reference 4: http://www.xbrl.org/2003/role/disclosureRef
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Reference 5: http://www.xbrl.org/2003/role/disclosureRef
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Reference 6: http://www.xbrl.org/2003/role/disclosureRef
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Reference 9: http://www.xbrl.org/2003/role/disclosureRef
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Reference 10: http://www.xbrl.org/2003/role/disclosureRef
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Reference 11: http://www.xbrl.org/2003/role/disclosureRef
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Reference 13: http://www.xbrl.org/2003/role/disclosureRef
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Commitments and Contingencies [Abstract]

CIRM Grants (Details) - LAD-I CIRM Grant [Member] - USD ($) $ in Thousands	Jan. 02, 2024	Dec. 31, 2023	Apr. 30, 2019
CIRM Grant [Abstract]
Grant award for clinical development support			$ 7,500
Milestone payments received		$ 5,800
Grant receivable included in prepaid and other assets		$ 50
Subsequent Event
CIRM Grant [Abstract]
Milestone payments received	$ 50

Stock-Based Awards - Stock-Based Compensation (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Stock-Based Compensation Expense [Abstract]
Total share based compensation expense	$ 39,406	$ 31,009	$ 29,237
Stock options
Stock-Based Compensation Expense [Abstract]
Total share based compensation expense	29,091	27,620	28,811
Restricted Stock Units
Stock-Based Compensation Expense [Abstract]
Total share based compensation expense	10,315	3,389	426
Research and Development
Stock-Based Compensation Expense [Abstract]
Total share based compensation expense	17,509	12,466	11,954
General and Administrative
Stock-Based Compensation Expense [Abstract]
Total share based compensation expense	$ 21,897	$ 18,543	$ 17,283

Commitments and Contingencies

12 Months Ended

Dec. 31, 2023

Commitments and Contingencies

14.

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.

Income Taxes - Deferred Tax Assets and Liabilities (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Deferred income tax assets (liabilities) [Abstract]
R&D credits	$ 20,984	$ 20,984	$ 35,766
Net operating losses and credit carryforwards	45,313	33,718	26,789
Capitalized research and development costs	17,205	19,085	19,753
Stock-based compensation	28,499	19,781	11,552
Warrants	9,283	8,390	8,382
Intangible assets	(5,787)	(5,424)	0
Other	(8,086)	(6,332)	(8,881)
Valuation allowance	(108,472)	(91,263)	(93,361)
Net deferred income tax asset (liability)	$ (1,061)	$ (1,061)	$ 0

Accounts Payable and Accrued Expenses

12 Months Ended

Dec. 31, 2023

Accounts Payable and Accrued Expenses [Abstract]

Accounts Payable and Accrued Expenses

The Company’s accounts payable and accrued expenses consisted of the following:


	December 31, 2023		December 31, 2022
Research and development	$	13,867	$	19,100
Investment payable		13,137		-
Employee compensation		9,930		10,006
Property and equipment		1,077		2,095
Professional fees		6,006		1,436
Acquisition related expenses		-		1,153
Government grant payable		-		597
Other		1,772		2,273
Total accounts payable and accrued expenses	$	45,789	$	36,660

The $13.1 million investment payable was related to investment purchases of available-for-sale securities in 2023 that settled in 2024.

Related Party Transactions - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands			1 Months Ended	12 Months Ended
	Sep. 15, 2023	Oct. 06, 2022	Sep. 30, 2023	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021	Dec. 17, 2021	Aug. 09, 2021
Related Party Transaction [Abstract]
Related party expenses				$ 20
Expense in connection with warrant issue				$ 0	$ 0	$ 12,781
Common Stock
Related Party Transaction [Abstract]
Issuance of common stock, net of issuance costs (in shares)	9,500,000	7,800,000		9,453,418	7,820,000	812,516
Share price (in dollars per share)		$ 14.75
Common Stock \| Public Offering
Related Party Transaction [Abstract]
Issuance of common stock, net of issuance costs (in shares)	9,500,000
Restricted Stock Units
Related Party Transaction [Abstract]
Shares granted (in shares)				1,018,322	1,047,301
Warrants
Related Party Transaction [Abstract]
Shares granted (in shares)				3,126,955
August 2021 Warrant \| Common Stock
Related Party Transaction [Abstract]
Expense in connection with warrant issue						$ 7,600
August 2021 Warrant \| Consulting Agreement, Business Development Services \| Common Stock
Related Party Transaction [Abstract]
Warrant issued (in shares)								301,291
December 2021 Warrants \| Common Stock
Related Party Transaction [Abstract]
Expense in connection with warrant issue						$ 5,200
December 2021 Warrants \| Consulting Agreement, Business Development Services \| Common Stock
Related Party Transaction [Abstract]
Warrant issued (in shares)							153,155
December 2021 Warrants \| Consulting Agreement, Asset Identification Services \| Common Stock
Related Party Transaction [Abstract]
Warrant issued (in shares)							153,155
Pre-Funded Warrants \| Common Stock
Related Party Transaction [Abstract]
Warrant issued (in shares)				126,093
Number of stock warrants issued to purchase shares	3,100,000			3,126,955
Pre-Funded Warrants \| Common Stock \| Public Offering
Related Party Transaction [Abstract]
Number of stock warrants issued to purchase shares	3,100,000		3,100,000
Member of the Board of Directors - Two
Related Party Transaction [Abstract]
Term of consulting agreement				1 year
Number of options granted (in shares)				20,000
Fair value amount of options granted				$ 400

Nature of Business and Basis of Presentation - Additional Information (Details)	12 Months Ended
	Dec. 31, 2023 Program
LV Member
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items]
Number of clinical-stage programs	3
AAV Member
Collaborative Arrangements and Non-collaborative Arrangement Transactions [Line Items]
Number of clinical-stage programs	2
Number of pre clinical-stage programs	1

Consolidated Statements of Changes in Stockholders' Equity - USD ($) $ in Thousands	Total	Common Stock	Treasury Stock, Common	Additional Paid In Capital	Accumulated Other Comprehensive Income / (Loss)	Accumulated Deficit
Beginning Balance at Dec. 31, 2020	$ 503,519	$ 610		$ 825,794	$ (42)	$ (322,843)
Beginning Balance (in shares) at Dec. 31, 2020		60,996,367
Issuance of common stock, net of issuance costs	26,354	$ 8		26,346
Issuance of common stock, net of issuance costs (in shares)		812,516
Issuance of common stock pursuant to exercise of stock options	11,327	$ 12		11,315
Issuance of common stock pursuant to exercise of stock options (in shares)		1,209,960
Issuance of common stock pursuant to conversion of notes	40,694	$ 15		40,679
Issuance of common stock pursuant to conversion of notes (in shares)		1,487,046
Issuance of common stock related to acquisition	0
Issuance of warrants	12,781			12,781
Unrealized comprehensive gain (loss) on investments	(119)				(119)
Stock-based compensation	29,237			29,237
Net loss	(169,069)					(169,069)
Ending Balance at Dec. 31, 2021	454,724	$ 645		946,152	(161)	(491,912)
Ending Balance (in shares) at Dec. 31, 2021		64,505,889
Issuance of common stock, net of issuance costs	108,138	$ 78		108,060
Issuance of common stock, net of issuance costs (in shares)		7,820,000
Issuance of common stock pursuant to exercise of stock options	$ 631	$ 1		630
Issuance of common stock pursuant to exercise of stock options (in shares)		66,887
Issuance of common stock pursuant to exercise of warrant (in shares)	0
Issuance of common stock pursuant to vesting of restricted stock units (in shares)		10,168
Issuance of common stock related to acquisition	$ 70,724	$ 34		70,690
Issuance of common stock related to acquisition (in shares)		3,420,774
Issuance of common stock related to earnout restricted stock units settlement (in shares)		5,101
Issuance of common stock pursuant to the at-the-market offering program, net of issuance costs	46,566	$ 33		46,533
Issuance of common stock pursuant to the at-the-market offering program, net of issuance costs (in shares)		3,291,922
Treasury stock purchases	(47)		$ (47)
Treasury stock purchases (in shares)		2,571
Unrealized comprehensive gain (loss) on investments	(196)				(196)
Stock-based compensation	31,009			31,009
Net loss	(221,863)					(221,863)
Ending Balance at Dec. 31, 2022	489,686	$ 791	(47)	1,203,074	(357)	(713,775)
Ending Balance (in shares) at Dec. 31, 2022		79,123,312
Issuance of common stock, net of issuance costs	188,885	$ 95		188,790
Issuance of common stock, net of issuance costs (in shares)		9,453,418
Issuance of common stock pursuant to exercise of stock options	2,231	$ 2		2,229
Issuance of common stock pursuant to exercise of stock options (in shares)		223,145
Issuance of common stock pursuant to exercise of warrants	7	$ 1		6
Issuance of common stock pursuant to exercise of warrant (in shares)		126,093
Issuance of common stock pursuant to vesting of restricted stock units		$ 4		(4)
Issuance of common stock pursuant to vesting of restricted stock units (in shares)		407,999
Issuance of common stock related to acquisition	0
Issuance of common stock pursuant to the at-the-market offering program, net of issuance costs	17,222	$ 10		17,212
Issuance of common stock pursuant to the at-the-market offering program, net of issuance costs (in shares)		948,300
Sale of treasury stock	56		47	9
Unrealized comprehensive gain (loss) on investments	676				676
Stock-based compensation	39,406			39,406
Net loss	(245,595)					(245,595)
Ending Balance at Dec. 31, 2023	$ 492,574	$ 903		$ 1,450,722	$ 319	$ (959,370)
Ending Balance (in shares) at Dec. 31, 2023		90,282,267

Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Consolidated Statements of Comprehensive Loss [Abstract]
Net Income (Loss)	$ (245,595)	$ (221,863)	$ (169,069)
Other comprehensive loss
Net unrealized gain (loss) on investments	676	(196)	(119)
Total comprehensive loss	$ (244,919)	$ (222,059)	$ (169,188)

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

Fair Value of Financial Instruments [Abstract]

Property and Equipment, Net - Summary of Property and Equipment (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Property and Equipment [Abstract]
Property and equipment, gross	$ 53,204	$ 38,102
Less: accumulated depreciation and amortization	(14,032)	(9,093)
Total property, plant and equipment, net	39,172	29,009
Laboratory Equipment
Property and Equipment [Abstract]
Property and equipment, gross	29,232	21,905
Machinery and Equipment
Property and Equipment [Abstract]
Property and equipment, gross	12,325	11,326
Computer Equipment
Property and Equipment [Abstract]
Property and equipment, gross	244	244
Furniture and Fixtures
Property and Equipment [Abstract]
Property and equipment, gross	2,777	2,135
Leasehold Improvements
Property and Equipment [Abstract]
Property and equipment, gross	6,723	589
Internal use Software
Property and Equipment [Abstract]
Property and equipment, gross	$ 1,903	$ 1,903

Consolidated Balance Sheets (Parenthetical) - $ / shares	Dec. 31, 2023	Dec. 31, 2022
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)	90,282,267	79,123,312
Common stock, shares outstanding (in shares)	90,282,267	79,120,741
Treasury stock, at cost (in shares)	0	2,571
Series A Convertible Preferred Shares
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
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

Fair Value of Financial Instruments

12 Months Ended

Dec. 31, 2023

Fair Value of Financial Instruments

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


	Fair Value Measurements as of December 31, 2023 Using:
	Level 1		Level 2		Level 3		Total
Assets:
Cash equivalents:
Money market mutual funds	$	50,737	$	-	$	-	$	50,737
U.S. Treasury Securities		-		2,487		-		2,487
		50,737		2,487		-		53,224

Investments:
U.S. Treasury Securities		-		312,696		-		312,696
Corporate Bonds		-		38,895		-		38,895
		-		351,591		-		351,591

Total assets	$	50,737	$	354,078	$	-	$	404,815

Liabilities:
Warrant liability	$	-	$	-	$	1,876	$	1,876
Total liabilities	$	-	$	-	$	1,876	$	1,876


	Fair Value Measurements as of December 31, 2022 Using:
	Level 1		Level 2		Level 3		Total
Assets:
Cash equivalents:
Money market mutual funds	$	90,527	$	-	$	-	$	90,527
Commercial Paper		-		3,899		-		3,899
U.S. Treasury Securities		-		3,848		-		3,848
Corporate Bonds		-		8,618		-		8,618
		90,527		16,365		-		106,892

Investments:
Commercial Paper		-		1,151		-		1,151
U.S. Treasury Securities		-		189,444		-		189,444
Corporate Bonds		-		60,905		-		60,905
Agency Bonds		-		7,653		-		7,653
		-		259,153		-		259,153

Total assets	$	90,527	$	275,518	$	-	$	366,045

Liabilities:
Warrant liability	$	-	$	-	$	1,512	$	1,512
Total liabilities	$	-	$	-	$	1,512	$	1,512

The Company classifies its money market mutual funds 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 U.S. Treasury Securities, Commercial Paper and Corporate 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 reconciliation of the Company’s warrant liability, which is recorded as part of Other Liabilities in the consolidated balance sheets, measured at fair value on a recurring basis using unobservable inputs (Level 3) is as follows:


	Warrant Liability
Balance, December 31, 2021	$	-
Acquisition of Renovacor		1,512
Balance, December 31, 2022	$	1,512
Fair value adjustments		364
Balance, December 31, 2023	$	1,876

The Company utilizes a Black-Scholes model to value the Private Warrants (see Note 10 “Warrants”) at each reporting period, with changes in fair value recognized in the consolidated statements of operations. The estimated fair value of the warrant liability is determined using Level 3 inputs. Inherent in an options pricing model are assumptions related to expected share-price volatility, expected life, risk-free interest rate and dividend yield. The Company estimates the expected volatility of its common stock based on historical volatility of a peer group, considering the expected remaining life of the Private Warrants. The risk-free interest rate is based on the U.S. Treasury zero-coupon yield curve on the valuation date for a maturity similar to the expected remaining life of the Private Warrants. The expected life of the Private Warrants is assumed to be equivalent to their remaining contractual term. The dividend rate is based on the historical rate, which the Company anticipates will remain at zero.

The fair value of the Private Warrants has been estimated with the following assumptions:


	December 31, 2023			December 1, 2022
Stock price	$	29.50		$	18.39
Exercise price	$	65.23		$	65.23
Expected volatility		68.83	%		71.25	%
Risk-free interest rate		4.70	%		4.14	%
Expected dividend yield		-			-
Expected life (years)		1.31			2.39
Fair value per warrant	$	3.04		$	2.45

The fair value change from December 1, 2022, to December 31, 2022, was immaterial.

Income Taxes

12 Months Ended

Dec. 31, 2023

Income Taxes [Abstract]

Income Taxes

12.

Income Taxes

No provision for federal or state income taxes was recorded during the years ended December 31, 2023, 2022 and 2021, as the Company incurred operating losses and maintains a full valuation allowance against its net deferred tax assets.

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


	For the Years Ended December 31,
	2023			2022			2021
U.S. federal tax at statutory rate		21.0	%		21.0	%		21.0	%
Foreign rate differential		(17.3	%)		(16.9	%)		(13.0	%)
Change in state tax apportionment		1.4	%		(0.1	%)		0.1	%
Stock compensation		0.1	%		0.1	%		1.5	%
Return to provision		1.7	%		—	%		—	%
Transfer pricing adjustments		—	%		—	%		(22.8	%)
Valuation allowance		(7.0	)%		6.3	%		4.6	%
Federal NOL true-up		0.3	%		(2.7	%)		—	%
Tax credits		0.0	%		(6.7	%)		8.7	%
Other		(0.2	%)		(1.0	%)		(0.1	%)
Effective tax rate		—	%		—	%		—	%

On October 4, 2023, the Governor of Massachusetts signed into law a bill that included the adoption of a single sales apportionment factor effective on January 1, 2025. As required under ASC 740, the Company has accounted for the deferred tax impacts of this tax law change in the period the tax law was enacted, which has the impact of reducing its state deferred tax assets. The impact of the tax law change is offset by a change in valuation allowance.

Intellectual property rights in different jurisdictions are reflected in the Company’s effective tax rate.

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


	As of December 31,
	2023			2022			2021
Deferred income tax assets (liabilities)
R&D credits	$	20,984		$	20,984		$	35,766
Net operating losses and credit carryforwards		45,313			33,718			26,789
Capitalized research and development costs		17,205			19,085			19,753
Stock-based compensation		28,499			19,781			11,552
Warrants		9,283			8,390			8,382
Intangible assets		(5,787	)		(5,424	)		—
Other		(8,086	)		(6,332	)		(8,881	)
Valuation allowance		(108,472	)		(91,263	)		(93,361	)
Net deferred income tax asset (liability)	$	(1,061	)	$	(1,061	)	$	-

As of December 31, 2023, the Company had federal and state net operating loss (“NOL”) carryforwards of approximately $197.7 million and $77.2 million, respectively. The state NOL begins to expire in 2026. Additionally, $197.7 million of the federal NOL can be carried forward indefinitely. The Company has federal R&D credits of $21.0 million which will begin to expire in 2038.

As required by ASC 740, Income Taxes, the Company has evaluated the positive and negative evidence bearing upon the realizability of its deferred tax assets, which are comprised principally of NOL carryforwards and capitalized research and development costs. As a result of the fact that the Company has incurred tax losses from inception, management has determined that it is more likely than not that the Company will not recognize the benefits of federal and state net deferred tax assets and, as a result, a full valuation allowance has been established against its net deferred tax assets as of December 31, 2023, 2022 and 2021. The Company has offset certain deferred tax liabilities with deferred tax assets that are expected to generate offsetting deductions within the same period. During the years ended December 31, 2023 and 2022, the valuation allowance increased by $17.2 million and decreased by $2.1 million, respectively. Realization of deferred tax assets is dependent upon the generation of future taxable income.

Under Internal Revenue Code Section 382, if a corporation undergoes an “ownership change,” the corporation’s ability to use its pre-change NOL carryforwards and other pre-change tax attributes to offset its post-change income may be limited. The Company has completed a study to assess whether an ownership change has occurred or whether there have been multiple ownership changes since the Company became a “loss corporation” as defined in Section 382. The Company experienced multiple ownership changes occurring in 2005, 2007, 2015, and 2018. The ownership change has and will continue to subject our pre-ownership change net operating loss carryforwards to an annual limitation, which will significantly restrict our ability to use them to offset taxable income in periods following the ownership change. In general, the annual use limitation equals the aggregate value of our stock at the time of the ownership change multiplied by a specified tax-exempt interest rate. As a result of the ownership change, the Company is limited to an approximate $1.7 million annual limitation on our ability to utilize our pre-merger NOL’s and R&D Credits. Due to this limitation, approximately $91.2 million of the $127.1 million pre-merger Federal NOL will expire unutilized as the cumulative limitation amount over a 20-year carryforward period is $35.8 million. Additionally, $4.9 million of federal R&D credits will expire unutilized. As a result, the Company has reduced its deferred tax assets related to the Federal NOL and federal R&D credits by an aggregate of $4.9 million which is offset by the corresponding decrease in the valuation allowance. In conjunction with the Renovacor Acquisition (see Note 17 “Renovacor Acquisition”), the Company acquired Renovacor’s federal NOL’s of $46.4 million. The Company has completed a study to assess whether an ownership change has occurred as a result of the transaction. The Company experienced ownership changes occurring in 2021 and 2022. As a result of the ownership changes, the Company is limited on our ability to utilize our Renovacor NOL’s. As a result of the Company’s study, any limitation under IRC Sec. 382 would not result in NOLs expiring unused.

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

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

The Company records uncertain tax positions as liabilities in accordance with ASC 740 and adjusts these liabilities when our judgment changes as a result of the evaluation of new information not previously available. Because of the complexity of some of these uncertainties, the ultimate resolution may result in a payment that is materially different from the Company’s current estimate of the unrecognized tax benefit liabilities. These differences will be reflected as increases or decreases to income tax expense in the period in which new information is available. As of December 31, 2023, 2022 and 2021, the Company has not recorded any uncertain tax positions in its financial statements.

The Company recognizes interest and penalties related to unrecognized tax benefits on the income tax expense line in the accompanying consolidated statements of operations. As of December 31, 2023 and 2022, no accrued interest or penalties are included on the related tax liability line in the consolidated balance sheets.

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

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
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Stock-Based Awards - RSU Activity (Parenthetical) (Details)	12 Months Ended
	Dec. 31, 2023 shares
Restricted Stock Units
Share-based Compensation Arrangement by Share-based Payment Award, Compensation Cost [Line Items]
Common stock shares issued related to tax (in shares)	120

Document and Entity Information - USD ($) $ in Billions	12 Months Ended
Document and Entity Information - USD ($) $ in Billions	Dec. 31, 2023	Feb. 22, 2024	Jun. 30, 2023
Cover [Abstract]
Document Type	10-K
Amendment Flag	false
Document Annual Report	true
Document Period End Date	Dec. 31, 2023
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2023
Document Fiscal Period Focus	FY
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
Trading Symbol	RCKT
Security Exchange Name	NASDAQ
Entity Well-known Seasoned Issuer	Yes
Entity Voluntary Filers	No
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
ICFR Auditor Attestation Flag	true
Document Financial Statement Error Correction [Flag]	false
Entity Shell Company	false
Entity Public Float			$ 1.2
Entity Common Stock, Shares Outstanding		90,504,248
Documents Incorporated by Reference	Documents Incorporated by Reference Part III of this annual report on Form 10-K incorporates by reference information (to the extent specific sections are referred to herein) from the registrant’s definitive proxy statement for its 2024 Annual Meeting of Stockholders (the “Proxy Statement”). The Proxy Statement will be filed with the United States Securities and Exchange Commission within 120 days of the end of the period covered by this Annual Report on Form 10-K.
Auditor Firm ID	274
Auditor Name	EISNERAMPER LLP
Auditor Location	Iselin, New Jersey

Stock-Based Awards - Weighted-Average Assumptions for Stock Options (Details) - Employees, Non-employees and Directors - $ / shares	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Weighted-Average Assumptions [Abstract]
Risk-free interest rate	4.00%	2.19%	0.83%
Expected term	5 years 9 months 25 days	5 years 1 month 2 days	5 years 10 months 2 days
Expected volatility	73.32%	64.12%	69.27%
Expected dividend yield	0.00%	0.00%	0.00%
Exercise price (in dollars per share)	$ 20.24	$ 18.56	$ 51.2
Fair value of common stock (in dollars per share)	$ 20.24	$ 18.56	$ 51.2

Warrants - Summary of Rocket Warrants Outstanding (Details)	12 Months Ended
	Dec. 31, 2023 $ / shares shares
Warrants [Abstract]
Outstanding (in shares)	5,715,078
57.11
Warrants [Abstract]
Exercise price per share (in dollars per share) \| $ / shares	$ 57.11
Outstanding (in shares)	603,386
Grant date	Dec. 21, 2020
Expiration date	Dec. 21, 2030
33.63
Warrants [Abstract]
Exercise price per share (in dollars per share) \| $ / shares	$ 33.63
Outstanding (in shares)	301,291
Grant date	Aug. 09, 2021
Expiration date	Aug. 09, 2031
22.51
Warrants [Abstract]
Exercise price per share (in dollars per share) \| $ / shares	$ 22.51
Outstanding (in shares)	153,155
Grant date	Dec. 17, 2021
Expiration date	Dec. 17, 2031
22.51
Warrants [Abstract]
Exercise price per share (in dollars per share) \| $ / shares	$ 22.51
Outstanding (in shares)	153,155
Grant date	Dec. 17, 2021
Expiration date	Dec. 17, 2031
65.23
Warrants [Abstract]
Exercise price per share (in dollars per share) \| $ / shares	$ 65.23
Outstanding (in shares)	617,050
Grant date	Dec. 01, 2022
Expiration date	Apr. 23, 2025
65.23
Warrants [Abstract]
Exercise price per share (in dollars per share) \| $ / shares	$ 65.23
Outstanding (in shares)	760,086
Grant date	Dec. 01, 2022
Expiration date	Dec. 01, 2026
0.01
Warrants [Abstract]
Exercise price per share (in dollars per share) \| $ / shares	$ 0.01
Outstanding (in shares)	3,126,955
Grant date	Sep. 15, 2023

Summary of Significant Accounting Policies, Cash, Cash Equivalents and Restricted Cash (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Cash, Cash Equivalents and Restricted Cash [Abstract]
Cash and cash equivalents	$ 55,904	$ 140,517
Restricted cash	1,372	1,340
Total cash, cash equivalents and restricted cash	$ 57,276	$ 141,857	$ 234,037	$ 298,666

Leases - Operating Leases - Additional Information (Details) - USD ($) $ in Thousands		12 Months Ended
	Mar. 26, 2021	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021	Mar. 31, 2023	Dec. 01, 2022	Jun. 07, 2018
Operating Leases [Abstract]
Lease expiration date		Oct. 31, 2024
Total right-of-use asset		$ 3,901	$ 1,972		$ 200
Total lease liabilities		2,973	1,088		$ 200
Operating lease cost		1,377	818	$ 645
Rent expense		2,300	1,200	1,100
Restricted cash		1,372	1,340
Operating and Finance Leases [Member]
Operating Leases [Abstract]
Restricted cash		$ 800	800
Renovacor, Inc. [Member]
Operating Leases [Abstract]
Remaining lease term		1 year 3 months 18 days
Total right-of-use asset		$ 3,800				$ 2,700
Total lease liabilities		3,600				$ 2,600
Other Current Assets [Member]
Operating Leases [Abstract]
Security deposit		$ 200
ESB Lease Agreement [Member]
Operating Leases [Abstract]
Term of lease agreement		3 years
Letter of credit							$ 900
Lease expiration date		Aug. 29, 2024
Certificate of deposit		$ 800	800
ESB Lease Agreement Amendment [Member]
Operating Leases [Abstract]
Letter of credit		$ 800
Lease expiration date	Jun. 30, 2024
Inotek Lexington Massachusetts Lease Agreement [Member]
Operating Leases [Abstract]
Lease expiration date		Feb. 28, 2023
Rental income received under sublease agreements		$ 300	$ 400	$ 400
Hopewell, New Jersey and Cambridge [Member] \| Renovacor, Inc. [Member]
Operating Leases [Abstract]
Remaining lease term		10 years 3 months

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]

Stock options

367,852

$ 2,163

Time Vesting RSU

28,798

$ 512

Stock Consideration

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

3,391,976

$ 18.39

Summary of Significant Accounting Policies [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.

Summary of Significant Accounting Policies (Tables)

12 Months Ended

Dec. 31, 2023

Cash, Cash Equivalents and Restricted Cash


	December 31, 2023		December 31, 2022
Cash and cash equivalents	$	55,904	$	140,517
Restricted cash		1,372		1,340
Total cash, cash equivalents and restricted cash	$	57,276	$	141,857

Leases (Tables)

12 Months Ended

Dec. 31, 2023

Leases [Abstract]

Lease Cost

The following table summarizes lease cost for the years ended December 31, 2023, 2022 and 2021:


	For the Years Ended December 31,
Lease cost	2023		2022		2021
Operating lease cost	$	1,377	$	818	$	645
Finance lease cost:
Amortization of right of use assets		2,154		2,139		2,140
Interest on lease liabilities		1,875		1,861		1,845
Total lease cost	$	5,406	$	4,818	$	4,630

Maturity of Operating Lease Liabilities

The following table summarizes the maturity of the Company’s lease liabilities on an undiscounted cash flow basis:


Fiscal Year Ending December 31,	December 31, 2023
2024	$	931
2025		567
2026		571
2027		506
2028		522
Thereafter		2,419
Total lease payments	$	5,516
Less: interest		(1,618	)
Total operating lease liabilities	$	3,898

Maturity of Finance Lease Liability


Fiscal Year Ending December 31,	December 31, 2023
2024	$	1,791
2025		1,856
2026		1,912
2027		1,969
2028		2,028
Thereafter		41,003
Total lease payments	$	50,559
Less: interest		(29,415	)
Total finance lease liability	$	21,144

Operating and Financing Lease Liabilities and Right-of-use Assets

The following table summarizes the operating and financing lease liabilities and right-of-use assets as of December 31, 2023 and 2022:


	December 31, 2023		December 31, 2022
Operating right-of-use assets	$	3,901	$	1,972

Operating current lease liabilities	$	925	$	773
Operating noncurrent lease liabilities		2,973		1,088
Total operating lease liabilities	$	3,898	$	1,861

Finance right-of-use assets	$	44,517	$	46,664

Finance current lease liability	$	1,791	$	1,736
Finance noncurrent lease liability		19,353		19,269
Total finance lease liability	$	21,144	$	21,005


	For the Years Ended December 31,
Other information	2023			2022			2021
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows from operating leases	$	1,229		$	938		$	655
Cash flows from finance lease	$	1,736		$	1,689		$	1,644
Weighted-average remaining lease term - operating leases	8.0 years			4.8 years			2.5 years
Weighted-average remaining lease term - finance lease	20.7 years			21.7 years			22.7 years
Weighted-average discount rate - operating leases		8.32	%		6.44	%		4.46	%
Weighted-average discount rate - finance lease		8.96	%		8.69	%		8.96	%

Summary of Significant Accounting Policies, Intangible Assets (Details) - USD ($)	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022
Intangible Assets [Abstract]
Impairment of intangible assets	$ 0	$ 0

Accounts Payable and Accrued Expenses (Tables)

12 Months Ended

Dec. 31, 2023

Accounts Payable and Accrued Expenses [Abstract]

Schedule of Accounts Payable and Accrued Expenses

The Company’s accounts payable and accrued expenses consisted of the following:


	December 31, 2023		December 31, 2022
Research and development	$	13,867	$	19,100
Investment payable		13,137		-
Employee compensation		9,930		10,006
Property and equipment		1,077		2,095
Professional fees		6,006		1,436
Acquisition related expenses		-		1,153
Government grant payable		-		597
Other		1,772		2,273
Total accounts payable and accrued expenses	$	45,789	$	36,660

The $13.1 million investment payable was related to investment purchases of available-for-sale securities in 2023 that settled in 2024.

Summary of Significant Accounting Policies, Property and Equipment (Details)	Dec. 31, 2023
Internal use Software [Member]
Property and Equipment [Abstract]
Estimated useful lives	6 years
Minimum [Member]
Property and Equipment [Abstract]
Estimated useful lives	3 years
Maximum [Member]
Property and Equipment [Abstract]
Estimated useful lives	15 years

Accounts Payable and Accrued Expenses - Schedule of Accounts Payable and Accrued Expenses (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Accounts Payable and Accrued Expenses [Abstract]
Research and development	$ 13,867	$ 19,100
Investment payable	13,137	0
Employee compensation	9,930	10,006
Property and equipment	1,077	2,095
Professional fees	6,006	1,436
Acquisition related expenses	0	1,153
Government grant payable	0	597
Other	1,772	2,273
Total accounts payable and accrued expenses	$ 45,789	$ 36,660

Stockholders' Equity

12 Months Ended

Dec. 31, 2023

Stockholders' Equity [Abstract]

Stockholders' Equity

Stockholders’ Equity

Common Stock

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

Second Amended and Restated 2014 Stock Option and Incentive Plan

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

Treasury Stock

During the year ended December 31, 2023, the Company recorded sale of treasury stock of $0.06 million. During the year ended December 31, 2022, the Company recorded a repurchase of treasury stock of $0.05 million for shares withheld to pay the payroll tax liability of the vesting of RSUs. There was no treasury stock as of December 31, 2023.

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 December 31, 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 year ended December 31, 2023, the Company sold 0.9 million shares under the at-the-market offering program for gross proceeds of $17.8 million, less commissions of approximately $0.6 million for net proceeds of $17.2 million. During the year ended December 31, 2022, the Company sold 3.3 million shares under the at-the-market offering program for gross proceeds of $48.0 million, less commissions of $1.4 million for net proceeds of $46.6 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 Offerings

On October 6, 2022, the Company completed a public offering of approximately 7.8 million shares of our common stock at a public offering price of $14.75 per share. The gross proceeds from the public offering were approximately $115.3 million, net of $7.2 million of offering costs, commissions, legal and other expenses for net proceeds from the offering of $108.1 million.

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 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.

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
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Reference 2: http://www.xbrl.org/2003/role/disclosureRef
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Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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Reference 4: http://www.xbrl.org/2003/role/disclosureRef
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Reference 5: http://www.xbrl.org/2003/role/disclosureRef
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Reference 6: http://www.xbrl.org/2003/role/disclosureRef
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Reference 7: http://www.xbrl.org/2003/role/disclosureRef
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Reference 8: http://www.xbrl.org/2003/role/disclosureRef
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Reference 9: http://www.xbrl.org/2003/role/disclosureRef
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Reference 10: http://fasb.org/us-gaap/role/ref/legacyRef
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Reference 11: http://www.xbrl.org/2003/role/disclosureRef
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-Name Accounting Standards Codification
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Reference 12: http://www.xbrl.org/2003/role/disclosureRef
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Reference 13: http://www.xbrl.org/2003/role/disclosureRef
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-URI https://asc.fasb.org//1943274/2147481112/505-10-50-13

Reference 14: http://www.xbrl.org/2003/role/disclosureRef
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Reference 15: http://www.xbrl.org/2003/role/disclosureRef
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Reference 16: http://www.xbrl.org/2003/role/disclosureRef
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Reference 17: http://www.xbrl.org/2003/role/disclosureRef
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-Subparagraph (a)
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Reference 18: http://www.xbrl.org/2003/role/disclosureRef
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Reference 19: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
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-URI https://asc.fasb.org//1943274/2147481112/505-10-50-18

Fair Value of Financial Instruments [Abstract]

Fair Value of Financial Instruments (Tables)

12 Months Ended

Dec. 31, 2023

Measured on Recurring Basis


	Fair Value Measurements as of December 31, 2023 Using:
	Level 1		Level 2		Level 3		Total
Assets:
Cash equivalents:
Money market mutual funds	$	50,737	$	-	$	-	$	50,737
U.S. Treasury Securities		-		2,487		-		2,487
		50,737		2,487		-		53,224

Investments:
U.S. Treasury Securities		-		312,696		-		312,696
Corporate Bonds		-		38,895		-		38,895
		-		351,591		-		351,591

Total assets	$	50,737	$	354,078	$	-	$	404,815

Liabilities:
Warrant liability	$	-	$	-	$	1,876	$	1,876
Total liabilities	$	-	$	-	$	1,876	$	1,876


	Fair Value Measurements as of December 31, 2022 Using:
	Level 1		Level 2		Level 3		Total
Assets:
Cash equivalents:
Money market mutual funds	$	90,527	$	-	$	-	$	90,527
Commercial Paper		-		3,899		-		3,899
U.S. Treasury Securities		-		3,848		-		3,848
Corporate Bonds		-		8,618		-		8,618
		90,527		16,365		-		106,892

Investments:
Commercial Paper		-		1,151		-		1,151
U.S. Treasury Securities		-		189,444		-		189,444
Corporate Bonds		-		60,905		-		60,905
Agency Bonds		-		7,653		-		7,653
		-		259,153		-		259,153

Total assets	$	90,527	$	275,518	$	-	$	366,045

Liabilities:
Warrant liability	$	-	$	-	$	1,512	$	1,512
Total liabilities	$	-	$	-	$	1,512	$	1,512

Changes in Level 3 Liabilities Measured at Fair Value


	Warrant Liability
Balance, December 31, 2021	$	-
Acquisition of Renovacor		1,512
Balance, December 31, 2022	$	1,512
Fair value adjustments		364
Balance, December 31, 2023	$	1,876

Level 3 Fair Value of the Private Warrants

The fair value of the Private Warrants has been estimated with the following assumptions:


	December 31, 2023			December 1, 2022
Stock price	$	29.50		$	18.39
Exercise price	$	65.23		$	65.23
Expected volatility		68.83	%		71.25	%
Risk-free interest rate		4.70	%		4.14	%
Expected dividend yield		-			-
Expected life (years)		1.31			2.39
Fair value per warrant	$	3.04		$	2.45

Intangible Assets and Goodwill - Summary of Intangible Assets (Details) - USD ($) $ in Thousands	Dec. 31, 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

Leases - Maturity of Operating Lease Liabilities (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Lessee, Operating Lease, Liability, to be Paid, Fiscal Year Maturity [Abstract]
2024	$ 931
2025	567
2026	571
2027	506
2028	522
Thereafter	2,419
Total lease payments	5,516
Less: interest	(1,618)
Total operating lease liabilities	$ 3,898	$ 1,861

Property and Equipment, Net

12 Months Ended

Dec. 31, 2023

Property and Equipment, Net [Abstract]

Property and Equipment, Net

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


	December 31, 2023			December 31, 2022
Laboratory equipment	$	29,232		$	21,905
Machinery and equipment		12,325			11,326
Computer equipment		244			244
Furniture and fixtures		2,777			2,135
Leasehold improvements		6,723			589
Internal use software		1,903			1,903
		53,204			38,102
Less: accumulated depreciation and amortization		(14,032	)		(9,093	)
Total property, plant and equipment, net	$	39,172		$	29,009

Depreciation and amortization during the years ended December 31, 2023, 2022, and 2021 was $4.9 million, $3.9 million and $3.2 million, respectively.

Net Loss Per Share - Basic and Diluted Net Loss Per Share (Details) - USD ($) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Numerator [Abstract]
Net loss attributable to common stockholders	$ (245,595)	$ (221,863)	$ (169,069)
Denominator [Abstract]
Weighted-average common shares outstanding - basic (in shares)	84,009,004	68,148,925	63,235,417
Weighted-average common shares outstanding - diluted (in shares)	84,009,004	68,148,925	63,235,417
Net loss per share attributable to common stockholders - basic (in dollars per share)	$ (2.92)	$ (3.26)	$ (2.67)
Net loss per share attributable to common stockholders - diluted (in dollars per share)	$ (2.92)	$ (3.26)	$ (2.67)

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

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Topic 250
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Reference 8: http://www.xbrl.org/2003/role/disclosureRef
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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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-URI https://asc.fasb.org//1943274/2147482689/260-10-45-60B

CIRM Grants

12 Months Ended

Dec. 31, 2023

CIRM Grants [Abstract]

CIRM Grants

16.

CIRM Grants

LAD-I CIRM Grant

On April 30, 2019, the 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, 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 December 31, 2023, the Company has received $5.8 million in total RP-L201 grants from CIRM. As of December 31, 2023, the Company met the final CIRM milestone and recorded a receivable of $0.05 million, included in prepaid and other current assets in the consolidated balance sheet, recorded as a reduction of research and development expenses. The Company received the $0.05 million final milestone payment on January 2, 2024.

Intangible Assets and Goodwill - Additional Information (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022	Dec. 01, 2022	Dec. 31, 2021
Indefinite-Lived Intangible Assets [Line Items]
Goodwill	$ 39,154	$ 39,154		$ 30,815
Renovacor, Inc.
Indefinite-Lived Intangible Assets [Line Items]
Goodwill	$ 8,300	$ 8,300	$ 8,339

Basic and Diluted Net Loss Per Share

+ References + Details

Net Loss Per Share (Tables)

12 Months Ended

Dec. 31, 2023

Net Loss Per Share [Abstract]

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


	For the Years Ended December 31,
	2023			2022			2021
Numerator:
Net loss attributable to common stockholders	$	(245,595	)	$	(221,863	)	$	(169,069	)
Denominator:
Weighted-average common shares outstanding - basic and diluted		84,009,004			68,148,925			63,235,417
Net loss per share attributable to common stockholders - basic and diluted	$	(2.92	)	$	(3.26	)	$	(2.67	)

Antidilutive Securities Excluded from Computation of Diluted Net Loss Per Share

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


	For the Years Ended December 31,
	2023		2022		2021
Warrants exercisable for common shares		2,588,123		2,721,267		1,218,038
Restricted stock units convertible for common shares		1,490,357		992,874		23,500
Options to purchase common shares		14,863,996		13,138,870		11,143,761
		18,942,476		16,853,011		12,385,299

Summary of Significant Accounting Policies, Investments (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Investments [Abstract]
Realized gains (losses) on investment	$ 0	$ 0	$ 0
Unrealized gain (loss) on investments	$ 676	$ (196)	$ (119)

Income Taxes (Tables)

12 Months Ended

Dec. 31, 2023

Income Taxes [Abstract]

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

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


	For the Years Ended December 31,
	2023			2022			2021
U.S. federal tax at statutory rate		21.0	%		21.0	%		21.0	%
Foreign rate differential		(17.3	%)		(16.9	%)		(13.0	%)
Change in state tax apportionment		1.4	%		(0.1	%)		0.1	%
Stock compensation		0.1	%		0.1	%		1.5	%
Return to provision		1.7	%		—	%		—	%
Transfer pricing adjustments		—	%		—	%		(22.8	%)
Valuation allowance		(7.0	)%		6.3	%		4.6	%
Federal NOL true-up		0.3	%		(2.7	%)		—	%
Tax credits		0.0	%		(6.7	%)		8.7	%
Other		(0.2	%)		(1.0	%)		(0.1	%)
Effective tax rate		—	%		—	%		—	%

Intellectual property rights in different jurisdictions are reflected in the Company’s effective tax rate.

Deferred Tax Assets and Liabilities

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


	As of December 31,
	2023			2022			2021
Deferred income tax assets (liabilities)
R&D credits	$	20,984		$	20,984		$	35,766
Net operating losses and credit carryforwards		45,313			33,718			26,789
Capitalized research and development costs		17,205			19,085			19,753
Stock-based compensation		28,499			19,781			11,552
Warrants		9,283			8,390			8,382
Intangible assets		(5,787	)		(5,424	)		—
Other		(8,086	)		(6,332	)		(8,881	)
Valuation allowance		(108,472	)		(91,263	)		(93,361	)
Net deferred income tax asset (liability)	$	(1,061	)	$	(1,061	)	$	-

Income Taxes - Reconciliation Between Effective Tax Rates and Statutory Rates (Details)	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Reconciliation of Income Tax Benefit Computed at Statutory Federal Income Tax Rate to Income Taxes [Abstract]
U.S. federal tax at statutory rate	21.00%	21.00%	21.00%
Foreign rate differential	(17.30%)	(16.90%)	(13.00%)
Change in state tax apportionment	1.40%	(0.10%)	0.10%
Stock compensation	0.10%	0.10%	1.50%
Return to provision	1.70%	0.00%	0.00%
Transfer pricing adjustments	0.00%	0.00%	(22.80%)
Valuation allowance	(7.00%)	6.30%	4.60%
Federal NOL true-up	0.30%	(2.70%)	0.00%
Tax credits	0.00%	(6.70%)	8.70%
Other	(0.20%)	(1.00%)	(0.10%)
Effective tax rate	0.00%	0.00%	0.00%

Leases - Lease Related to Cash Flow Information, Lease Term and Discount Rate (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Cash Flow, Operating and Financing Activities, Lessee [Abstract]
Operating cash flows from operating leases	$ 1,229	$ 938	$ 655
Cash flows from finance lease	$ 1,736	$ 1,689	$ 1,644
Weighted-average remaining lease term - operating leases	8 years	4 years 9 months 18 days	2 years 6 months
Weighted-average remaining lease term - finance lease	20 years 8 months 12 days	21 years 8 months 12 days	22 years 8 months 12 days
Weighted-average discount rate - operating leases	8.32%	6.44%	4.46%
Weighted-average discount rate - finance lease	8.96%	8.69%	8.96%

Stock-Based Awards - RSU Activity (Details) - Restricted Stock Units - USD ($) $ / shares in Units, $ in Millions	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Number of Shares [Roll Forward]
Beginning balance (in shares)	992,874	23,500
Granted (in shares)	1,018,322	1,047,301
Vested (in shares)	(408,119)	(38,966)
Forfeited (in shares)	(112,720)	(67,759)
Ending balance (in shares)	1,490,357	992,874	23,500
Weighted Average Grant Date Fair Value [Abstract]
Beginning balance (in dollars per share)	$ 16.49	$ 30.61
Granted (in dollars per share)	19.67	15.91
Vested (in dollars per share)	16.61	23.15
Forfeited (in dollars per share)	17.92	15.94
Ending balance (in dollars per share)	$ 18.53	$ 16.49	$ 30.61
Intrinsic value	$ 6.8	$ 0.8	$ 0.4
Renovacor, Inc.
Number of Shares [Roll Forward]
Conversion of awards (in shares)		28,798
Weighted Average Grant Date Fair Value [Abstract]
Conversion of awards (in dollars per share)		$ 0.49

Summary of Significant Accounting Policies

12 Months Ended

Dec. 31, 2023

Summary of Significant Accounting Policies [Abstract]

Summary of Significant Accounting Policies

Principles of Consolidation

Use of Estimates

Cash, Cash Equivalents and Restricted Cash


	December 31, 2023		December 31, 2022
Cash and cash equivalents	$	55,904	$	140,517
Restricted cash		1,372		1,340
Total cash, cash equivalents and restricted cash	$	57,276	$	141,857

Government Grants

Research and development expense was presented net of reimbursements from CIRM (See Note 16 “CIRM Grant” for additional disclosure).

Concentrations of credit risk and off-balance sheet risk

Investments

Intangible Assets

Goodwill

Property and Equipment, Net

Impairment of Long-Lived Assets

Fair Value Measurements

•

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

•

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

•

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

Warrants

Research and Development Expenses

Foreign Currency Transactions

Treasury Stock

The Company records treasury stock at cost.

Stock-Based Compensation

New York State Life Sciences Research and Development Tax Credit

Income Taxes

Net Loss Per Share

Segment Reporting

Comprehensive Loss

Leases

Recent Accounting Pronouncements

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

Accounting Pronouncements Not Adopted as of December 31, 2023

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

Nature of Business and Basis of Presentation [Abstract]

$ 1,512

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

Net Loss Per Share

12 Months Ended

Dec. 31, 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:


	For the Years Ended December 31,
	2023			2022			2021
Numerator:
Net loss attributable to common stockholders	$	(245,595	)	$	(221,863	)	$	(169,069	)
Denominator:
Weighted-average common shares outstanding - basic and diluted		84,009,004			68,148,925			63,235,417
Net loss per share attributable to common stockholders - basic and diluted	$	(2.92	)	$	(3.26	)	$	(2.67	)

In 2023, the Company included the 3,126,955 potential shares from the pre-funded warrants acquired by RTW as it was determined that these met the definition for equity classification and only requires the holder to pay $0.01 per share upon exercise.


	For the Years Ended December 31,
	2023		2022		2021
Warrants exercisable for common shares		2,588,123		2,721,267		1,218,038
Restricted stock units convertible for common shares		1,490,357		992,874		23,500
Options to purchase common shares		14,863,996		13,138,870		11,143,761
		18,942,476		16,853,011		12,385,299

Leases

12 Months Ended

Dec. 31, 2023

Leases [Abstract]

Leases

13.

Leases

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 December 31, 2023 and 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 December 31, 2023, and 2022, respectively.

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 $0.3 million for the year ended December 31, 2023, and $0.4 million for the years ended December 31, 2022 and 2021. These amounts were 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 17 “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 December 31, 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 the facilities in Hopewell, New Jersey and signed an agreement to sublease one of these facilities in January 2024.

Rent expense was $2.3 million, $1.2 million, and $1.1 million for the years ended December 31, 2023, 2022 and 2021, respectively.

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

Operating lease cost was $1.4 million, $0.8 million, and $0.6 million for the years ended December 31, 2023, 2022 and 2021, respectively.

The following table summarizes lease cost for the years ended December 31, 2023, 2022 and 2021:


	For the Years Ended December 31,
Lease cost	2023		2022		2021
Operating lease cost	$	1,377	$	818	$	645
Finance lease cost:
Amortization of right of use assets		2,154		2,139		2,140
Interest on lease liabilities		1,875		1,861		1,845
Total lease cost	$	5,406	$	4,818	$	4,630

The following table summarizes the maturity of the Company’s lease liabilities on an undiscounted cash flow basis:


Fiscal Year Ending December 31,	December 31, 2023
2024	$	931
2025		567
2026		571
2027		506
2028		522
Thereafter		2,419
Total lease payments	$	5,516
Less: interest		(1,618	)
Total operating lease liabilities	$	3,898


Fiscal Year Ending December 31,	December 31, 2023
2024	$	1,791
2025		1,856
2026		1,912
2027		1,969
2028		2,028
Thereafter		41,003
Total lease payments	$	50,559
Less: interest		(29,415	)
Total finance lease liability	$	21,144

The following table summarizes the operating and financing lease liabilities and right-of-use assets as of December 31, 2023 and 2022:


	December 31, 2023		December 31, 2022
Operating right-of-use assets	$	3,901	$	1,972

Operating current lease liabilities	$	925	$	773
Operating noncurrent lease liabilities		2,973		1,088
Total operating lease liabilities	$	3,898	$	1,861

Finance right-of-use assets	$	44,517	$	46,664

Finance current lease liability	$	1,791	$	1,736
Finance noncurrent lease liability		19,353		19,269
Total finance lease liability	$	21,144	$	21,005


	For the Years Ended December 31,
Other information	2023			2022			2021
Cash paid for amounts included in the measurement of lease liabilities:
Operating cash flows from operating leases	$	1,229		$	938		$	655
Cash flows from finance lease	$	1,736		$	1,689		$	1,644
Weighted-average remaining lease term - operating leases	8.0 years			4.8 years			2.5 years
Weighted-average remaining lease term - finance lease	20.7 years			21.7 years			22.7 years
Weighted-average discount rate - operating leases		8.32	%		6.44	%		4.46	%
Weighted-average discount rate - finance lease		8.96	%		8.69	%		8.96	%

401(k) Savings Plan - Additional Information (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
401(k) Savings Plan [Abstract]
Percentage of matching employee contributions	4.00%
Matching employee contributions	$ 1.4	$ 0.9	$ 0.6

Net Loss Per Share - Additional Information (Details) - Pre-Funded Warrants [Member]	12 Months Ended
	Dec. 31, 2023 $ / shares shares
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items]
Potential shares included in the calculation of EPS \| shares	3,126,955
Warrants exercise price per share \| $ / shares	$ 0.01

Leases - Finance Lease - Additional Information (Details) $ in Thousands	12 Months Ended
	Dec. 31, 2023 USD ($) ft² Leaseagreement	Dec. 31, 2022 USD ($)
Finance Lease [Abstract]
Total lease payments	$ 50,559
Cash security deposit	$ 533	$ 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

Risks and Liquidity - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands			1 Months Ended	12 Months Ended
	Sep. 15, 2023	Oct. 06, 2022	Sep. 30, 2023	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021	Sep. 12, 2023	Feb. 28, 2022
Risks and Liquidity [Abstract]
Accumulated deficit				$ (959,370)	$ (713,775)
Cash, cash equivalents and investments				407,500
Cash				$ 394,400
Shareholders' Equity Disclosure [Abstract]
Common stock, par value (in dollars per share)				$ 0.01	$ 0.01
Investments in Securities
Risks and Liquidity [Abstract]
Cash, cash equivalents and investments				$ 13,100
Common Stock
Shareholders' Equity Disclosure [Abstract]
Common stock shares issued and sold (in shares)	9,500,000	7,800,000		9,453,418	7,820,000	812,516
Gross proceeds from sale of common stock		$ 108,100
Shares issued price per share	$ 16
Shares issuance cost	$ 12,400
Net proceeds from offering		$ 108,100
Common Stock \| Pre-Funded Warrants
Shareholders' Equity Disclosure [Abstract]
Number of stock warrants issued to purchase shares	3,100,000			3,126,955
Warrant to purchase shares of common stock price per share (in dollars per share)	$ 15.99			$ 0.06
At-the-Market Offering
Shareholders' Equity Disclosure [Abstract]
Common stock shares issued and sold (in shares)				900,000	3,300,000
Gross proceeds from sale of common stock				$ 17,222	$ 46,566	$ 0
Net proceeds from offering				$ 17,222	46,566	0
At-the-Market Offering \| Common Stock
Shareholders' Equity Disclosure [Abstract]
Common stock shares issued and sold (in shares)				900,000
Gross proceeds from sale of common stock				$ 17,200
Net proceeds from offering				$ 17,200
At-the-Market Offering \| 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
Gross proceeds from sale of common stock				$ 63,800
Net proceeds from offering				$ 63,800
At-the-Market Offering \| Cowen and Company, LLC \| Maximum
Shareholders' Equity Disclosure [Abstract]
Aggregate offering price							$ 180,000	$ 200,000
At-the-Market Offering \| Cowen and Company, LLC \| Common Stock
Shareholders' Equity Disclosure [Abstract]
Common stock shares issued and sold (in shares)				4,200,000
Gross proceeds from sale of common stock				$ 63,800
Net proceeds from offering				63,800
Public Offering
Shareholders' Equity Disclosure [Abstract]
Gross proceeds from sale of common stock				188,885	108,138	26,354
Net proceeds from offering				$ 188,885	$ 108,138	$ 26,354
Public Offering \| Common Stock
Shareholders' Equity Disclosure [Abstract]
Gross proceeds from sale of shares	$ 201,300
Common stock shares issued and sold (in shares)	9,500,000
Gross proceeds from sale of common stock	$ 188,900
Shares issued price per share	$ 16
Shares issuance cost	$ 12,400
Net proceeds from offering	$ 188,900
Public Offering \| Common Stock \| Pre-Funded Warrants
Shareholders' Equity Disclosure [Abstract]
Number of stock warrants issued to purchase shares	3,100,000		3,100,000
Warrant to purchase shares of common stock price per share (in dollars per share)	$ 15.99

Nature of Business and Basis of Presentation

12 Months Ended

Dec. 31, 2023

Nature of Business and Basis of Presentation

Rocket Pharmaceuticals, Inc. (“Rocket” or the “Company”) is a fully integrated, late-stage 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 three clinical-stage ex vivo lentiviral vector (“LV”) programs, which include programs for:

•

Fanconi Anemia (“FA”), a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells;

•

Leukocyte Adhesion Deficiency-I (“LAD-I”), a genetic disorder that causes the immune system to malfunction; and

•

Pyruvate Kinase Deficiency (“PKD”), a red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia.

In September 2023, the FDA accepted the Biologics License Application (BLA) and granted priority review for RP-L201 for the treatment of severe LAD-I. Treatments in the FA Phase 2 studies were completed in 2023 with regulatory filings in the United States (“U.S.”) and Europe (“EU”) for FA anticipated in 2024. Additional work on a gene therapy program for the less common FA subtypes C and G is ongoing.

In the U.S., the Company also has two clinical stage and one pre-clinical stage in vivo adeno-associated virus (“AAV”) programs, which include programs for:

•

Danon disease (“DD”), a multi-organ lysosomal-associated disorder leading to early death due to heart failure. The DD program is currently in an ongoing Phase 2 trial.

•

Plakophilin-2 Arrhythmogenic Cardiomyopathy (“PKP2-ACM”), an inheritable cardiac disorder that is characterized by a progressive loss of cardiac muscle mass, severe right ventricular dilation, dysplasia, fibrofatty replacement of the myocardium and a high propensity to arrhythmias and sudden death. This program received FDA clearance of an Investigational New Drug (“IND”) application and the Company has initiated a Phase 1 study.

•

BAG3 Dilated Cardiomyopathy (“DCM”), which is the most common form of cardiomyopathy and is characterized by progressive thinning of the walls of the heart resulting in enlarged heart chambers that are unable to pump blood. The Company utilizes recombinant AAV9-based gene therapy designed to slow or halt progression of BAG3-DCM.

The Company has global commercialization and development rights to all of these product candidates under royalty-bearing license agreements.

Fair Value of Financial Instruments - Changes in Level 3 Liabilities Measured at Fair Value (Details) - Recurring - Level 3 - Warrant Liability - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022
Changes in Fair Value of Warrant Liabilities [Roll Forward]
Fair value, beginning of period	$ 1,512	$ 0
Acquisition of Renovacor		1,512
Fair value adjustments	364
Fair value, end of period	$ 1,876	$ 1,512

Intangible Assets and Goodwill (Tables)

12 Months Ended

Dec. 31, 2023

Intangible Assets and Goodwill [Abstract]

Summary of Intangible Assets

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


December 31, 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

Summary of Carrying Value of Goodwill


	Carrying Value
Balance, December 31, 2021	$	30,815
Acquisition of Renovacor		8,339
Balance, December 31, 2022 and 2023	$	39,154

Tabular disclosure of goodwill by reportable segment and in total which includes a rollforward schedule.

Reference 1: http://www.xbrl.org/2003/role/exampleRef
-Topic 350
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Reference 2: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 350
-SubTopic 20
-Name Accounting Standards Codification
-Section 50
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Reference 3: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 350
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-Name Accounting Standards Codification
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Reference 4: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 350
-SubTopic 20
-Name Accounting Standards Codification
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Reference 5: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 350
-SubTopic 20
-Name Accounting Standards Codification
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Reference 6: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 7: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 350
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Reference 8: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 350
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-Name Accounting Standards Codification
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Reference 9: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 10: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 11: http://www.xbrl.org/2009/role/commonPracticeRef
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Reference 12: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 350
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-Publisher FASB
-URI https://asc.fasb.org//1943274/2147482573/350-20-50-2

Business Combination, Consideration Transferred [Abstract]

Accounts Payable and Accrued Expenses - Additional Information (Details) - USD ($) $ in Thousands	Dec. 31, 2023	Dec. 31, 2022
Accounts Payable and Accrued Expenses [Abstract]
Investment payable	$ 13,137	$ 0

Renovacor Acquisition - Additional Information (Details)
$ / shares in Units, $ in Thousands

12 Months Ended

Dec. 01, 2022

USD ($)

$ / shares

Sep. 19, 2022

Dec. 31, 2022

USD ($)

Dec. 31, 2023

USD ($)

Dec. 31, 2021

USD ($)

Business Combination, Consideration Transferred [Abstract]

$ 39,154

$ 30,815

Renovacor, Inc.

Business Combination [Abstract]

Exchange ratio

0.1763

5,291,855

Cash consideration

^[1]

$ 29

2,700

Total consideration

72,265

Renovacor, Inc. | Assumed Warrants

$ 8,339

8,300

$ 8,300

$ 3,200

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