Restructuring Efforts See Rocket Focus on Cardiovascular Programs
In efforts to position itself for long-term growth, Rocket is reducing its company headcount, undergoing a structural reorganization, and prioritizing late-stage AAV cardiovascular programs.
Rocket Pharmaceuticals, a fully-integrated, late-stage biotechnology company, is undergoing strategic reorganization and prioritizing its adeno-associated viral (AAV) vector-based cardiovascular portfolio to maximize near-term value. The restructuring will also see the company reduce its headcount by about 30% (1).
“We are grateful for the remarkable contributions of our entire team and the significant progress we've made in advancing genetic therapies for rare diseases,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals, in a company press release (1). “Our prioritization reflects a commitment to the programs with the most compelling near-term opportunities for patients and to setting a foundation for Rocket’s long-term growth and success. Together, our cardiovascular programs have demonstrated strong potential for impact in areas of high unmet need in substantial patient populations.”
Through the reorganization, the company will turn its focus to its AAV portfolio, which includes clinical programs in Danon disease, PKP2-associated arrhythmogenic cardiomyopathy (PKP2-ACM), and BAG3-associated dilated cardiomyopathy (BAG3-DCM). Furthermore, the company will be providing the completed responses to the FDA for its treatment for severe leukocyte adhesion deficiency-I (LAD-I), KRESLADI (formerly RP-L201).
In July 2025, the company revealed that it had received FDA Regenerative Medicine Advanced Therapy (RMAT) designation for its gene therapy to treat PKP2-ACM, RP-A601 (2). FDA based its designation decision on positive safety and efficacy data from a Phase I clinical trial.
“The FDA’s RMAT designation for RP-A601 represents a meaningful advancement for Rocket and for patients living with PKP2-ACM, a life-threatening genetic heart disease characterized by ventricular arrhythmias and sudden cardiac death,” commented Kinnari Patel, PharmD, MBA, President, Head of R&D of Rocket Pharmaceuticals, in a press release (2). “This marks the fifth RMAT designation in our history and underscores our commitment to developing potentially curative gene therapies for patients with rare and inherited cardiovascular diseases. The early clinical data for RP-A601 are highly encouraging, and we look forward to continued collaboration with the FDA throughout the program’s development.”
A month earlier, in June 2025, the company also announced that the FDA had cleared its investigational new drug (IND) application for its BAG3-DCM therapy, RP-A701 (3). “The FDA clearance of RP-A701, our third clinical-stage gene therapy candidate from our AAV cardiovascular portfolio, is an important milestone for Rocket,” remarked Patel in a press release about the regulatory clearance (3). “Phase I trial start-up activities are currently underway for RP-A701, and we are working towards treating the first patient.”
In addition to reducing its workforce by 30%, Rocket has planned other cost-saving initiatives to reduce its 12-month operating expenses by approximately 25%. The company expects to fund operations into the second quarter of 2027 through existing cash resources, exclusive of any potential proceeds that it may gain from a Priority Review Voucher granted by FDA for KRESLADI.
As a result of its reprioritization to its late-stage AAV portfolio, the company also anticipates delays to its Fanconi Anemia (FA; RP-L102) and Pyruvate Kinase Deficiency (PKD; RP-L301) programs. Strategic options will be continually evaluated and further information will be provided by the company along with second quarter earnings updates.
References
Rocket Pharma. Rocket Pharmaceuticals Announces Strategic Corporate Reorganization and Pipeline Prioritization of Cardiovascular Programs. Press Release, July 24, 2025.
Rocket Pharma. Rocket Pharmaceuticals Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for RP-A601 Gene Therapy for PKP2-Arrhythmogenic Cardiomyopathy. Press Release, July 17, 2025.
Rocket Pharma. Rocket Pharmaceuticals Announces FDA IND Clearance of RP-A701 for the Treatment of BAG3-Associated Dilated Cardiomyopathy. Press Release, June 30, 2025.