RP-A701

RP-A701 is a first-in-class, investigational gene therapy utilizing an AAVrh.74 vector, being developed for the treatment of BAG3-associated dilated cardiomyopathy (BAG3-DCM)[1][3][5]. This severe, inherited cardiac disorder is caused by mutations in the BAG3 gene, leading to early-onset, progressive heart failure characterized by ventricular enlargement and impaired systolic function. RP-A701 is designed to deliver a functional BAG3 gene to affected cardiomyocytes, potentially restoring BAG3 protein expression and improving cardiac function by addressing the underlying molecular defect. The therapy is administered as a single intravenous dose. The initial phase 1 clinical trial is enrolling adults with advanced BAG3-DCM and implantable cardioverter defibrillators to assess safety, biological activity, and preliminary efficacy[1][3][5]. This indication carries notable unmet need, as current treatments do not correct the genetic cause of disease.