Tenaya Therapeutics Receives RMAT Designation for TN-401 in ARVC Treatment

Tenaya Therapeutics, a clinical-stage biotechnology company, has received a significant regulatory endorsement for its gene therapy candidate TN-401. The U.S. Food and Drug Administration (FDA) has granted the company Regenerative Medicine Advanced Therapy (RMAT) designation for TN-401, which is being developed to treat PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC).

According to Faraz Ali, the CEO of Tenaya Therapeutics, the RMAT designation underscores the seriousness of PKP2-associated ARVC and the potential of TN-401 to address the underlying cause of the disease.

The RMAT designation is intended to facilitate the development and review of regenerative medicine therapies for serious conditions where preliminary clinical evidence indicates the potential to address unmet medical needs. It provides enhanced opportunities for interaction with the FDA, including early and ongoing guidance regarding clinical development, manufacturing, and potential regulatory pathways. RMAT designation may also provide eligibility for accelerated approval, priority review, and rolling review.

The interim data from the RIDGE-1 clinical trial, presented at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting in May 2026, showed improvements in multiple electrical stability measures. TN-401, an investigational AAV9-based gene therapy, is being developed for the treatment of ARVC due to mutations in the PKP2 gene. AAV9 was selected as the vector for delivery of Tenaya’s PKP2 gene therapy based on its extensive clinical and commercial safety record and demonstrated ability to target heart muscle cells.

Tenaya’s pipeline includes other clinical-stage candidates, such as TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy (HCM), and TN-301, a highly specific small molecule HDAC6 inhibitor with broad potential clinical utility in cardiac, metabolic, and muscular conditions, including heart failure with preserved ejection fraction (HFpEF) and Duchenne muscular dystrophy (DMD).

The RIDGE-1 Phase 1b/2 clinical trial, which is being conducted in the U.S. and UK, is a multi-center, open-label, dose escalation study. It is designed to assess the safety, tolerability, and preliminary clinical efficacy of a one-time intravenous infusion of TN-401. The trial seeks to enroll up to fifteen adults who have been diagnosed with PKP2-associated ARVC, have an implantable cardioverter-defibrillator (ICD), and are at increased risk for arrhythmias as determined by premature ventricular count (PVC) during screening.

Tenaya Therapeutics is committed to discovering, developing, and delivering potentially curative therapies that address the underlying drivers of heart disease. The company’s mission is to bring potentially transformative treatments to patients as quickly as possible. The RMAT designation is expected to enhance Tenaya’s ability to engage with the FDA as it advances TN-401 toward a pivotal trial. The company anticipates sharing additional data from the RIDGE-1 trial in the fourth quarter of 2026 and providing an update on ongoing discussions with regulators regarding pivotal trial plans for TN-401.