Tenaya Therapeutics, Inc. (NASDAQ: TNYA) announced that the U.S. Food and Drug Administration has granted Regenerative Medicine Advanced Therapy (RMAT) designation to TN-401, the company's gene therapy candidate for the treatment of PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC).

The designation was supported by interim data from the RIDGE-1 Phase 1b/2 clinical trial, which showed reductions in daily rates of premature ventricular contractions (PVCs) and non-sustained ventricular tachycardias (NSVTs), along with a favorable tolerability profile. That data was previously presented at the American Society for Gene and Cell Therapy Annual Meeting in May 2026.

TN-401 also holds Orphan Drug and Fast Track designations from the FDA, as well as PRIME designation from the European Medicines Agency. The RMAT designation provides enhanced opportunities for interaction with the FDA, including early guidance on clinical development, manufacturing and regulatory pathways, and may confer eligibility for accelerated approval, priority review and rolling review.

"RMAT designation underscores the continued recognition by regulators of the seriousness of PKP2-associated ARVC and the potential of TN-401 gene therapy to change the course of disease by addressing its underlying cause," said Faraz Ali, Chief Executive Officer of Tenaya Therapeutics.

ARVC caused by PKP2 mutations affects an estimated 70,000 people in the U.S. and accounts for approximately 40% of overall ARVC cases. Current treatments, including anti-arrhythmic medications, implantable cardioverter-defibrillators and ablation procedures, do not address the underlying genetic cause.

TN-401 is an investigational AAV9-based gene therapy administered as a one-time intravenous infusion. The RIDGE-1 trial is a multi-center, open-label, dose escalation study enrolling up to 15 adults in the U.S. and UK.

Tenaya said it expects to share additional data from RIDGE-1 and provide an update on regulatory discussions regarding pivotal trial plans in the fourth quarter of 2026.