FDA Grants RMAT Status to Tenaya's TN-401

• The FDA granted RMAT designation to TN-401.
• TN-401 is an investigational AAV9-based gene therapy for ARVC caused by PKP2 mutations.
• The RIDGE-1 trial, identifier NCT06228924, is enrolling up to fifteen adults.
• Interim RIDGE-1 data showed clinically meaningful reductions in daily rates of PVCs and NSVTs.
• Tenaya Therapeutics trades as NASDAQ: TNYA.
• Tenaya plans to disclose additional RIDGE-1 data in the fourth quarter.

RMAT Designation

The FDA granted RMAT designation to TN-401. TN-401 is an investigational AAV9-based gene therapy being developed for ARVC due to mutations in the PKP2 gene. The RMAT application included data presented at the ASGCT Annual Meeting in May 2026.

FDA Grants RMAT Status to Tenaya's TN-401

Prior Designations

TN-401 already holds Orphan Drug and Fast Track Designations from the FDA, as well as PRIME designation from the European Medicines Agency. RMAT designation is an FDA expedited program intended to facilitate development and review of regenerative medicine therapies for serious conditions where preliminary clinical evidence indicates potential to address unmet medical needs.

Clinical Data

The RIDGE-1 trial, identifier NCT06228924, is a multi-center, open-label, dose escalation study conducted in the U.S. and UK, enrolling up to fifteen adults. Interim results showed clinically meaningful reductions in daily rates of PVCs and NSVTs, along with a favorable tolerability profile. Separate interim results showed improvements in multiple electrical stability measures, including reduced rates of PVCs and NSVTs. Tenaya plans to disclose additional data from RIDGE-1 in the fourth quarter.

Natural History Study

The RIDGE natural history study carries trial identifier NCT06311708.

Disease Burden

PKP2 mutations account for approximately 40 percent of the ARVC population. PKP2-associated ARVC affects more than 70,000 people in the U.S.. ARVC is typically diagnosed before age 40.

Executive Comment

Faraz Ali serves as Chief Executive Officer of Tenaya Therapeutics.

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.

— Faraz Ali, CEO of Tenaya Therapeutics

Pipeline and Funding

Development of TN-401 is supported in part by a grant from the California Institute for Regenerative Medicine. Tenaya's pipeline also includes TN-201, a gene therapy for MYBPC3-associated hypertrophic cardiomyopathy. Tenaya Therapeutics trades on NASDAQ under the ticker TNYA.