07:42 AM EDT, 06/30/2025 (MT Newswires) -- Rocket Pharmaceuticals ( RCKT ) said Monday that the US Food and Drug Administration has cleared its investigational new drug application for RP-A701, a gene therapy candidate targeting BAG3-associated dilated cardiomyopathy, a rare and severe form of heart failure.

The company said the phase 1 trial will evaluate the safety, biological activity, and early efficacy of a single dose of RP-A701 in adults with advanced BAG3-DCM.

Initial participants will include patients with implantable cardioverter defibrillators at high risk of heart failure progression or cardiac death, it added.