ROCKET PHARMACEUTICALS, INC.
For the period ended Dec 31, 2025. Filed Feb 26, 2026.
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FY2025 · Filed Feb 26, 2026
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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, DC 20549
FORM 10-K
(Mark One)
For the fiscal year ended December 31,2025
OR
| □ | TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 |
|---|
For the transition period from to
Commission File Number:001-36829
(Exact Name of Registrant as Specified in Its Charter)
| Delaware | 04-3475813 |
|---|---|
| (State or Other Jurisdiction of Incorporation or Organization) | (IRS Employer Identification No.) |
| 9 Cedarbrook Drive, Cranbury, NJ | 08512 |
|---|---|
| (Address of Principal Executive Offices) | (Zip Code) |
(609)659-8001
(Registrant’s Telephone Number, including Area Code)
Securities registered pursuant to Section 12(b) of the Act:
| Title of each class | Trading Symbol(s) | Name of each exchange on which registered |
|---|---|---|
| Common Stock, $0.01 par value | RCKT | NASDAQ Global Market |
Securities registered pursuant to Section 12(g) of the Act: None
Indicate by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act. Yes□No□
Indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or Section 15(d) of the Exchange Act. Yes□No☒
Indicate by check mark whether the registrant: (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ☒No□
Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§ 232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ☒No□
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company or an emerging growth company. See the definitions of“large accelerated filer,” “accelerated filer,” “smaller reporting company” and“emerging growth company” in Rule 12b-2 of the Exchange Act.
| Large accelerated filer | □ | Accelerated filer | □ |
|---|---|---|---|
| Non-accelerated filer | ☒ | Smaller reporting company | ☒ |
| Emerging growth company | □ | ||
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.□
Indicate by check mark whether the registrant has filed a report on and attestation to its management’s assessment of the effectiveness of its internal control over financial reporting under Section 404(b) of the Sarbanes-Oxley Act (15 U.S.C. 7262(b)) by the registered public accounting firm that prepared or issued its audit report.□
If securities are registered pursuant to Section 12(b) of the Act, indicate by check mark whether the financial statements of the registrant included in the filing reflect the correction of an error to previously issued financial statements.□
Indicate by check mark whether any of those error corrections are restatements that required a recovery analysis of incentive-based compensation received by any of the registrant’s executive officers during the relevant recovery period pursuant to §240.10D-1(b).□
Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes□No☒
The aggregate market value of the common stock held by non-affiliates of the registrant as of June 30, 2025 (the last business day of the registrant’s most recently completed second fiscal quarter) was approximately $212.5 million, based upon the closing price on the NASDAQ Global Market reported for such date.
As of February 20, 2026, there were 108,569,143 shares of common stock, $0.01 par value per share, outstanding.
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 2026 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.
Table of Contents
| Page | ||
|---|---|---|
| PART I. | ||
| Item 1. | Business | 9 |
| Item 1A. | Risk Factors | 37 |
| Item 1B. | Unresolved SEC Comments | 64 |
| Item 1C. | Cybersecurity | 64 |
| Item 2. | Properties | 65 |
| Item 3. | Legal Proceedings | 65 |
| Item 4. | Mine Safety Disclosures | 66 |
| PART II. | ||
| Item 5. | Market for Registrant’s Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities | 67 |
| Item 6. | Reserved | 67 |
| Item 7. | Management’s Discussion and Analysis of Financial Condition and Results of Operations | 67 |
| Item 7A. | Quantitative and Qualitative Disclosures About Market Risk | 76 |
| Item 8. | Financial Statements and Supplementary Data | 76 |
| Item 9. | Changes in and Disagreements with Accountants on Accounting and Financial Disclosure | 76 |
| Item 9A. | Controls and Procedures | 76 |
| Item 9B. | Other Information | 77 |
| Item 9C. | Disclosure Regarding Foreign Jurisdictions that Prevent Inspections | 77 |
| PART III | ||
| Item 10. | Directors, Executive Officers, and Corporate Governance | 78 |
| Item 11. | Executive Compensation | 78 |
| Item 12. | Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters | 78 |
| Item 13. | Certain Relationships and Related Transactions, and Director Independence | 78 |
| Item 14. | Principal Accountant Fees and Services | 78 |
| PART IV | ||
| Item 15. | Exhibits and Financial Statement Schedules | 79 |
| Signatures | 82 | |
Summary of Abbreviated Terms
Rocket Pharmaceuticals, Inc. may be referred to as Rocket, the Company, we, our or us, in this Annual Report on Form 10-K (the “Annual Report”), unless the context otherwise indicates. Throughout this Annual Report, we have used terms which are defined below:
| AAV | Adeno-associated virus | HNJ | Hospital Infantil de Niño Jesús |
|---|---|---|---|
| AAV9 | Adeno-associated serotype 9 | HSCT | Hematopoietic stem cell transplant |
| ACM | Arrhythmogenic cardiomyopathy | ICD | Internal Classification of Diseases |
| ASC | Accounting Standard Codification | IND | Investigational New Drug application |
| ASGCT | American Society of Gene & Cell Therapy | IV | Intravenous |
| ATMP | Advanced Therapy Medical Product | IPR&D | In process research and development |
| BAG3 | BCL2-associated athanogene 3 | KCCQ | Kansas City Cardiovascular Questionnaire |
| BAG3-DCM | BCL2-associated athanogene 3 mutations associated with dilated cardiomyopathy | LAD-I | Leukocyte Adhesion Deficiency-I |
| BLA | Biologics License Application | LAMP2 | Lysosome-associated membrane protein 2 |
| BNP | Brain natriuretic peptide | Lexeo | Lexeo Therapeutics, Inc |
| cGMP | Current Good Manufacturing Practice | LV | Lentiviral vector |
| cGTP | Current Good Tissue Practice | LVMI | Left ventricular mass index |
| cKO | conditional knockout | MAA | Marketing Authorization Application |
| CIBER | Centro de Investigación Biomédica En Red | MHRA | Medicines and Healthcare Products Regulatory Agency |
| CIEMAT | Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas | NIH | National Institutes of Health |
| CIEMAT Group | CIEMAT, CIBER and FIISFJD | NYHA | New York Heart Association |
| CIRM | California Institute for Regenerative Medicine | Offering | Equity offering of shares of common stock, which closed December 12, 2024 |
| CISO | Chief Information Security Officer | PDUFA | Prescription Drug User Fee Act |
| CMC | Chemistry Manufacturing Controls | PKD | Pyruvate Kinase Deficiency |
| CODM | Chief Operating Decision Maker | PKP2 | Plakophilin-2 |
| CCPA | California Consumer Privacy Act | PKP2-ACM | Plakophilin-2 Arrhythmogenic Cardiomyopathy |
| CRL | Complete Response Letter | PSU | Performance-Based Restricted Stock Unit |
| CRO | Contract research organization | PRIME | Priority Medicines |
| Cowen | Cowen and Company, LLC | Private Placement | Issuance of pre-funded warrants on December 12, 2024 |
| CTIS | Clinical Trials Information Systems | R&D | Research and development |
| DCM | Dilated cardiomyopathy | Renovacor | Renovacor, Inc. acquired on December 1, 2022 |
| DD | Danon Disease | RIF | Reduction in workforce |
| DNA | Deoxyribonucleic acid | RMAT | Regenerative Medicine Advanced Therapy |
| EMA | European Medicines Agency | RSU | Restricted Stock Unit |
| ESB Lease Agreement | Office Lease agreement for office space in the Empire State Building in New York City | RTW | RTW Investments, L.P |
| EU | European Union | SAE | Serious Adverse Event |
| FA | Fanconi Anemia | SCD | Sudden cardiac death |
| FDA | U.S. Food and Drug Administration | SEC | Securities and Exchange Commission |
| FDCA | Federal Food, Drug and Cosmetic Act | Stanford | Center for Definitive and Curative Medicine at Stanford University School of Medicine |
| FIBHNJS | Fundación para la Investigacion Biomédica del Hospital del Nino Jesus | UCLA | University of California, Los Angeles |
| FIISFJD | Fundacion Instituto de Investigación Sanitaria Fundacion Jiménez Díaz | UCLB | UCL Business PLC |
| GCP | Good Clinical Practices | UCSD | The Regents of the University of California, San Diego |
| GDPR | General Data Protection Regulation | UPC | Unitary Patent Court |
| GLP | Good Laboratory Practice | U.K. | United Kingdom |
| GMP | Good Manufacturing Practice | U.S. | United States |
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