Shares of Rocket Pharmaceuticals Inc (NASDAQ:RCKT) advanced 2.4% after the company announced that the U.S. Food and Drug Administration has approved its Investigational New Drug (IND) application for RP-A701, a gene therapy aimed at treating BAG3-associated Dilated Cardiomyopathy (DCM).
The regulatory green light represents a major development for Rocket’s pipeline of cardiovascular gene therapies. RP-A701 is now the third candidate in the company’s portfolio to reach clinical-stage evaluation, focused specifically on genetic forms of cardiomyopathy. The therapy is designed to address a particularly severe type of heart failure characterized by progressive enlargement of the heart’s ventricles and reduced ability to pump blood effectively.
“The FDA clearance of RP-A701, our third clinical-stage gene therapy candidate from our AAV cardiovascular portfolio, is an important milestone for Rocket,” said Kinnari Patel, PharmD, MBA, President, Head of R&D, and Chief Operating Officer at Rocket Pharmaceuticals.
Following the FDA’s decision, Rocket is moving forward with preparations to launch a Phase 1 clinical trial. The planned study will be the first to test RP-A701 in humans and will be conducted across multiple sites. It will feature a dose-escalation format to evaluate the gene therapy’s safety, biological activity, and initial signs of efficacy in adults diagnosed with BAG3-associated DCM.
The trial will initially enroll patients with advanced disease who are at high risk of rapid heart failure progression or sudden cardiac death. These individuals will typically already have implantable cardioverter defibrillators. Researchers will monitor key outcomes such as BAG3 protein levels, shifts in cardiac biomarkers, and clinical indicators of disease trajectory following a one-time administration of RP-A701.