Rocket Pharmaceuticals, Inc, a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that it has received clearance from the U.S. Food and Drug Administration (FDA) for the Company’s Investigational New Drug (IND) application for RP-A701, an AAVrh.74-based gene therapy candidate for the treatment of BAG3-associated Dilated Cardiomyopathy (BAG3-DCM), a severe form of heart failure characterized by progressive ventricular enlargement and impaired systolic function.

“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 of Rocket Pharma. “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.”

The first-in-human Phase 1 clinical trial will be a multi-center, dose-escalation study designed to evaluate the safety, biological activity, and preliminary efficacy of RP-A701 in adults with BAG3-DCM. Initial study participants will include adults with implantable cardioverter defibrillators (ICDs) and advanced disease at high risk for heart failure progression and cardiac death. Participants will receive a single dose of RP-A701, and the trial will assess BAG3 protein expression, changes in cardiac biomarkers, and clinical predictors of disease progression.

Source: businesswire.com