Rocket Pharmaceuticals Restructures To Prioritize Cardiovascular Gene Therapies And Delays FA And PKD Programs

Rocket Pharmaceuticals has announced a strategic reorganization to focus on its high-priority adeno-associated virus (AAV) cardiovascular gene therapy programs. This pipeline reprioritization aims to extend the company’s cash runway into Q2 2027, drive near-term value creation, and strengthen its position in delivering one-time genetic therapies for rare cardiac disorders. Resources will now be concentrated on advanced programs targeting Danon disease, PKP2-associated arrhythmogenic cardiomyopathy, and BAG3-associated dilated cardiomyopathy, alongside progressing the regulatory response for KRESLADI™ for LAD-I.

“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. 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,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. 

As part of the restructuring, Rocket will reduce its workforce by approximately 30%. The combination of this headcount reduction and other cost-saving measures is expected to lower 12-month operating expenses by around 25%. The company emphasized that its existing cash reserves, excluding any proceeds from a potential Priority Review Voucher for KRESLADI™, will be sufficient to fund operations into the second quarter of 2027.

In alignment with this streamlined focus, Rocket will delay its Fanconi Anemia (RP-L102) and Pyruvate Kinase Deficiency (RP-L301) programs. Approval for RP-L102 is no longer anticipated in 2026. These adjustments highlight Rocket’s disciplined capital allocation and sharpened strategy around gene therapies for inherited cardiomyopathies, which affect more than 100,000 patients across the U.S. and EU.