Rocket Pharmaceuticals Receives FDA Clearance to Begin Human Trials of Gene Therapy for BAG3-associated Dilated Cardiomyopathy
Tuesday, July 01, 2025
Rocket Pharmaceuticals has announced that the US Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for RP-A701, a gene therapy candidate for the treatment of BAG3-associated Dilated Cardiomyopathy (BAG3-DCM).
BAG3-associated Dilated Cardiomyopathy is a rare genetic condition caused by mutations in the BAG3 gene. These mutations disrupt the production and function of the BAG3 protein, which plays a critical role in cardiac contractility, structural integrity, protein regulation, and cell survival processes. The resulting protein malfunction leads to the accumulation of damaged proteins in heart cells, which impairs cardiac function and can lead to early-onset heart failure.
Initial participants will include individuals with implantable cardioverter defibrillators (ICDs) who are at high risk of worsening heart failure and cardiac-related death. The trial will monitor BAG3 protein expression, changes in cardiac biomarkers, and key indicators of disease progression.
The therapy is based on the AAVrh.74 viral vector and targets a severe inherited form of heart failure.
RP-A701 becomes the company’s third clinical-stage gene therapy within its cardiovascular portfolio. The upcoming first-in-human Phase 1 clinical trial will focus on adults with advanced BAG3-DCM. The trial, set to begin soon, will be conducted across multiple sites and will use a dose-escalation approach. The primary aim is to assess the safety, biological activity, and early signs of efficacy following a single administration of RP-A701.
It is estimated that up to 30,000 individuals in the United States may be affected by this condition. Current treatments, including ICDs, resynchronisation devices, and heart transplants, may help manage symptoms but often fail to halt disease progression or address the root genetic cause.
Rocket’s investigational therapy aims to target the underlying mutation and provide a long-term therapeutic solution.
Source: rocketpharma.com
