RP-A701 / Rocket Pharma - LARVOL DELTA

Natural History and Gene Therapy Clinical Studies for Debilitating Inherited Cardiomyopathies (HFSA 2025) - P | "A Phase 1 clinical trial will evaluate RP-A701 (AAVrh.74-BAG3), an AAV-mediated gene therapy containing the human BAG3 coding sequence, in a multi-center, open-label, dose escalation trial as a single intravenous infusion in adult patients (age ≥18y) with a confirmed diagnosis of BAG3-DCM and an implanted ICD...Together, these approaches underscore the critical importance of early and comprehensive genetic testing to enable accurate diagnosis and targeted therapeutic intervention. For more information about ongoing studies, please contact: clinicaltrials@rocketpharma.com"

Clinical • Gene therapy • Cardiomyopathy • Cardiovascular • Gene Therapies • BAG3 • LAMP2

A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy (clinicaltrials.gov) - P1 | N=8 | Not yet recruiting | Sponsor: Rocket Pharmaceuticals Inc.

IO biomarker • New P1 trial • Cardiomyopathy • Cardiovascular • Gene Therapies