Rocket Pharma’s RP-A601 AAV Gene Therapy for PKP2-ACM Earns FDA RMAT Designation

CRANBURY, N.J. – July 17, 2025 – Rocket Pharmaceuticals, a late-stage biotechnology company focused on genetic therapies for rare disorders, just announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-A601. This investigational AAV-based gene therapy is designed to treat PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening inherited heart disease marked by ventricular arrhythmias and sudden cardiac death.

This marks Rocket’s fifth RMAT designation and was awarded based on positive safety and efficacy data from the ongoing Phase 1 RP-A601 clinical trial. The RMAT status will expedite the therapy’s development and review, providing key benefits like intensive FDA guidance and potential priority review for its Biologics License Application (BLA).

Kinnari Patel, President and Head of R&D at Rocket Pharmaceuticals, called this a significant step for both the company and PKP2-ACM patients. It underscores Rocket’s dedication to developing potentially curative gene therapies for rare cardiovascular conditions.

Encouraging Phase 1 Clinical Data

Preliminary results from the Phase 1 trial, presented at the 2025 American Society of Gene and Cell Therapy Annual Meeting, showed promising early safety and efficacy. All three adult patients who received a single dose of RP-A601 (AAVrh74.PKP2) demonstrated increased PKP2 protein expression (up to a 398% rise in those with low baseline levels) and improved desmosomal integrity. Clinically meaningful improvements or stabilization were seen across key endpoints, including:

• Right ventricular function
• Reduced ventricular arrhythmias
• Enhanced quality of life (e.g., 34–41 point increases in KCCQ-12 scores and improvement from NYHA Class II to Class I)

The therapy showed a favorable safety profile, was generally well-tolerated with no dose-limiting toxicities, and most adverse events were mild, moderate, and self-limiting.

About RP-A601

RP-A601 is an investigational, one-time, potentially curative gene therapy for PKP2-ACM. It uses a recombinant adeno-associated serotype rh74 capsid (AAVrh74) to deliver a functional human PKP2 transgene via a single intravenous (IV) infusion. This program also holds Fast Track designation in the U.S. and Orphan Drug designation in the U.S. and Europe.

About PKP2-Arrhythmogenic Cardiomyopathy (PKP2-ACM)

PKP2-ACM is an inherited heart disease caused by PKP2 gene mutations, affecting about 50,000 adults and children in the U.S. and Europe. These patients have an urgent unmet medical need, as current treatments often fail to prevent disease progression or arrhythmia recurrence and do not address the underlying genetic cause.