Rocket Pharma Wins FDA IND Clearance For First-in-Human Trial Of RP-A701 In BAG3-Associated Dilated Cardiomyopathy

Rocket Pharmaceuticals has secured FDA clearance for its Investigational New Drug (IND) application to initiate clinical trials of RP-A701, a gene therapy candidate for treating BAG3-associated dilated cardiomyopathy (BAG3-DCM). BAG3-DCM is a rare, aggressive form of heart failure caused by mutations in the BAG3 gene, leading to progressive ventricular enlargement and reduced heart function. The gene therapy utilizes an AAVrh.74 viral vector to deliver a functional copy of the BAG3 gene.

“The FDA clearance of RP-A701, our third clinical-stage gene therapy candidate from our AAV cardiovascular portfolio, is an important milestone for Rocket. With programs in the clinic for each of the major types of genetic cardiomyopathies – hypertrophic, dilated, and arrhythmogenic – we are advancing our mission to bring potentially curative gene therapies to patients with rare and life-threatening cardiovascular diseases. Phase 1 trial start-up activities are currently underway for RP-A701, and we are working towards treating the first patient,” said Kinnari Patel, PharmD, MBA, President, Head of R&D and Chief Operating Officer of Rocket Pharma. 

The upcoming Phase 1 trial will be the first-in-human study of RP-A701. It is a multi-center, dose-escalation trial that will assess the therapy's safety, biological activity, and early signals of efficacy. Initial participants will include adults with advanced BAG3-DCM who are at high risk of cardiac deterioration and already fitted with implantable cardioverter defibrillators (ICDs).

Participants will receive a single intravenous dose of RP-A701, and researchers will monitor key clinical markers, including BAG3 protein expression and cardiac biomarkers, as well as predictors of disease progression. This milestone marks Rocket’s entry into cardiology and expands its gene therapy pipeline beyond rare pediatric disorders.