RP-A701 / Rocket Pharma - LARVOL DELTA

AAV gene therapies for inherited cardiomyopathies (HEART FAILURE 2026) - " In a Phase 1 clinical study in males with DD, treatment with RP-A501 (AAV9.LAMP2B) resulted in LAMP2B protein expression, stabilization/reduction of left ventricular mass index (LVMI), and improvement in heart failure (HF) symptoms1...Preliminary results from an ongoing Phase 1 study of RP-A601 (AAVrh.74-PKP2a) in patients with PKP2-ACM demonstrated cardiac transduction, increased PKP2 protein expression and stabilization/improvement in arrhythmia burden, heart function and quality of life2...A BAG3 gene therapy (RP-A701; AAVrh.74-BAG3) is entering Phase 1 supported by evidence that BAG3 regulates sarcomere integrity, autophagy, apoptosis, and excitation-contraction coupling in cardiomyocytes3... AAV-mediated gene therapies have the potential to alter the natural history of DD, PKP2-ACM, and BAG3-associated DCM by addressing the underlying causative genetic deficits."

Gene therapy • Cardiomyopathy • Cardiovascular • Congestive Heart Failure • Heart Failure • 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 | Recruiting | Sponsor: Rocket Pharmaceuticals Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • IO biomarker • Cardiomyopathy • Cardiovascular • Gene Therapies

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: [email protected]"

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