CRANBURY, N.J. — June 30, 2025 — Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) announced today that the U.S. Food and Drug Administration has cleared its Investigational New Drug (IND) application for RP-A701, a first-in-class gene therapy candidate aimed at treating BAG3-associated dilated cardiomyopathy (BAG3-DCM), a rare and often fatal inherited heart disease.
The FDA clearance allows Rocket to proceed with a Phase 1 clinical trial, marking the company’s third gene therapy candidate targeting genetic cardiomyopathies to reach clinical-stage development.
“RP-A701’s IND clearance is a key milestone in our mission to deliver transformative gene therapies for patients with life-threatening cardiovascular diseases,” said Kinnari Patel, President and Chief Operating Officer of Rocket Pharmaceuticals. “With trials now underway or planned for hypertrophic, dilated, and arrhythmogenic cardiomyopathies, we are building a comprehensive pipeline to address unmet needs across the genetic heart disease spectrum.”
RP-A701 is based on the AAVrh.74 vector and is designed to deliver a healthy copy of the BAG3 gene, which plays a critical role in heart muscle cell function. Mutations in BAG3 impair cardiac contractility and lead to early-onset, progressive heart failure with high morbidity and premature mortality. BAG3-DCM affects an estimated 30,000 people in the U.S.
The upcoming Phase 1 trial will be a multi-center, dose-escalation study involving adult patients with advanced BAG3-DCM, including those with implantable cardioverter defibrillators (ICDs). Participants will receive a single dose of RP-A701, and researchers will monitor BAG3 protein expression, cardiac biomarkers, and clinical indicators of disease progression.
Current treatments for BAG3-DCM—such as ICDs, cardiac resynchronization devices, or heart transplants—manage symptoms but do not target the genetic cause of the disease. Rocket’s gene therapy approach aims to correct the underlying mutation, offering a potential one-time treatment that could halt or reverse disease progression.
Start-up activities for the Phase 1 trial are underway, with the first patient expected to be treated soon.
