Tenaya Therapeutics, Inc., a clinical-stage biotechnology company, discovers, develops, and delivers therapies for heart disease in the United States. Its lead product candidate includes TN-201, a gene therapy for myosin binding protein C3-associated hypertrophic cardiomyopathy that is in Phase 1b/2 clinical trial; TN-401, a gene therapy for plakophilin 2-associated arrhythmogenic right ventricular cardiomyopathy, which is in Phase 1b/2 clinical trial; and TN-301, a small molecule histone deacetylase-6 for heart failure with preserved ejection fraction that is in phase 1 clinical trial. The company develops its products through gene addition, gene editing, gene silencing, and cellular regeneration. It has a research collaboration agreement with Alnylam Pharmaceuticals, Inc. to identify and validate novel gene targets for the potential treatment of cardiovascular disease. Tenaya Therapeutics, Inc. was incorporated in 2016 and is headquartered in South San Francisco, California.
Tenaya Therapeutics Granted U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for TN-401 for the Treatment of PKP2-Associated Arrhythmogenic Right Ventricular Cardiomyopathy
Tenaya Therapeutics received U.S. FDA Regenerative Medicine Advanced Therapy (RMAT) designation for TN-401, its AAV9-based gene therapy for PKP2-associated arrhythmogenic right ventricular cardiomyopathy.
The designation is backed by interim Phase 1b/2 RIDGE-1 data showing clinically meaningful reductions in daily PVC and NSVT arrhythmia rates with a favorable tolerability profile; TN-401 already holds FDA Fast Track and Orphan Drug designations plus EMA PRIME status.
RMAT provides enhanced FDA interactions and potential eligibility for accelerated approval, priority review, and rolling review, with a U.S. patient population estimated at more than 70,000.
Additional RIDGE-1 data and an update on pivotal-trial regulatory discussions are expected in the fourth quarter of 2026, making that the next catalyst.