FDA Clears Rocket Pharmaceuticals’ Gene Therapy IND for Cardiomyopathy

The U.S. FDA has approved Rocket Pharmaceuticals’ investigational new drug application for RP-A701, a gene therapy candidate based on the AAVrh.74 vector targeting BAG3-associated dilated cardiomyopathy (BAG3-DCM). This severe heart failure condition is marked by progressive ventricular enlargement and impaired systolic function, and RP-A701 represents a potential novel treatment avenue in gene therapy for cardiomyopathies.