Rocket Pharmaceuticals Pulls BLA for Fanconi Anemia Gene Therapy RP-L102

Rocket Pharmaceuticals’ has voluntarily withdrawn the biologics license application (BLA) it had previously submitted to the FDA for mozafancogene autotemcel (also known as fana-cel and RP-L102), an investigational gene therapy intended to treat Fanconi anemia (FA).¹

Rocket quietly announced the move in a United States Securities and Exchange Commission (SEC) Form 8-K, citing corporate prioritization as the reason for its decision. The company specifically pointed out that it intends to focus its resources on “programs with the clearest regulatory and commercial pathways” and that it had stopped investing resources into the development of RP-L102 as of July 2025. Notably, in July 2025 Rocket also withdrew a marketing authorization application (MAA) it had submitted to the European Medicines Agency (EMA) for RP-L102. Although, the company stated that it may engage with potential external partners in the future that could carry on development of the gene therapy.

“The Company’s decision to withdraw the BLA is based on business and strategic considerations and does not reflect concerns regarding the safety or efficacy profile of RP-L102,” Rocket wrote in the SEC document.¹ “Data generated to date continue to support that RP-L102 has been generally well tolerated, with no significant safety signals observed, and a risk-benefit profile that appears favorable.”

Rocket started submission of a rolling BLA for RP-L102 in the first quarter of 2025, and according to a May 2025 press release was expecting to finish the submission either late this year or early next year.² The MAA for the gene therapy was accepted by the EMA in April 2024.³ The MAA was supported by data from a global phase 1/2 clinical trial that showed “sustained genetic correction, comprehensive phenotypic correction, and hematologic stabilization.”³ With regard to safety, there were no indications of bone marrow dysplasia, clonal dominance, or insertional mutagenesis related to the gene therapy product, and Rocket characterized the safety profile of RP-L102 as “highly favorable.”³ No cytotoxic conditioning was used during delivery of the gene therapy.

“Currently, there are no existing options to potentially prevent BMF for patients with FA,” Kinnari Patel, PharmD, MBA, the president and head of research & development and chief operating officer at Rocket Pharma, said in an April 2024 statement.³ “We are appreciative of the patients, their families, and researchers who helped reach this meaningful milestone and continue participating in the clinical development program. We look forward to partnering closely with the EMA throughout the review process to make RP-L102 available to patients with FA who are in need of new treatment options.”

Rocket is continuing to develop cardiovascular gene therapy products, including RP-A601, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat plakophilin-2 related arrhythmogenic cardiomyopathy, RP-A501, an investigational AAV9 vector–based gene therapy intended to treat Danon disease, and RP-A701, an investigational AAV vector-based gene therapy intended to treat BAG3-associated dilated cardiomyopathy (BAG3-DCM).^4-6 The company is also developing marnetegragene autotemcel (RP-L201, to be marketed as Kresladi), a genetically modified autologous hematopoietic stem cell therapy intended to treat leukocyte adhesion deficiency-I (LAD-I).⁷ Notably, on July 24, 2025, Rocket announced that as part of a strategic reorganization, it would be prioritizing the aforementioned therapies over several other therapeutic candidates in its pipeline and cutting its staff by around 30%.

REFERENCES
1. Form 8-K, United States Securities and Exchange Commission. Rocket Pharmaceuticals, Inc. October 3, 2025. Accessed October 6, 2025. https://ir.rocketpharma.com/node/12966/html
2. Rocket pharmaceuticalsreports first quarter 2025 financial results and highlights recent progress. News release. Rocket Pharmaceuticals, Inc. May 8,2025. Accessed October 6, 2025. https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-reports-first-quarter-2025-financial
3. Rocket Pharmaceuticals announces european medicines agency acceptance of RP-L102 marketing authorization application for the treatment of fanconi anemia. News release. Rocket Pharmaceuticals, Inc. April 2, 2024. Accessed October 6, 2025. https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-announces-european-medicines-agency
4. Rocket Pharmaceuticals receives FDA regenerative medicine advanced therapy (RMAT) designation for RP-A601 gene therapy for PKP2-arrhythmogenic cardiomyopathy. News release. Rocket Pharmaceuticals Inc. July 17, 2025. Accessed October 6, 2025. https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-receives-fda-regenerative-medicine-3
5. Rocket Pharmaceuticals provides update on phase 2 clinical trial of RP-A501 for Danon disease. News release. Rocket Pharmaceuticals Inc. May 27, 2025. Accessed October 6, 2025. https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-provides-update-phase-2-clinical-trial-rp
6. Rocket Pharmaceuticals announces FDA IND clearance of RP-A701 for the treatment of BAG3-associated dilated cardiomyopathy. News release. Rocket Pharmaceuticals, Inc. June 30, 2025. Accessed October 6, 2025. https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-announces-fda-ind-clearance-rp-a701
7. Rocket Pharmaceuticals announces strategic corporate reorganization and pipeline prioritization of cardiovascular programs. News release. Rocket Pharmaceuticals, Inc. July 17, 2025. Accessed October 6, 2025. https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-receives-fda-regenerative-medicine-3