TN-401 / Tenaya Therap - LARVOL DELTA

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 (GlobeNewswire) - "Tenaya’s RMAT application included data presented at the American Society for Gene and Cell Therapy (ASGCT) Annual Meeting in May 2026. Interim results from the RIDGE-1 clinical trial of TN-401 showed improvements in multiple electrical stability measures, including reduced rates of premature ventricular contractions (PVCs) and non-sustained ventricular tachycardias (NSVTs), two of the most frequently occurring forms of ventricular arrhythmias that characterize ARVC."

FDA event • Cardiomyopathy

Interim Data from RIDGETM-1: A Phase 1b Interventional Study to Evaluate Safety and Efficacy of TN-401 an AAV9 Investigational Gene Replacement Therapy, in Adults with PKP2-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ASGCT 2026) - P1 | "Conclusion Initial clinical data from RIDGE-1 indicates a promising safety profile, consistent transduction of the gene therapy in cardiomyocytes and PKP2 protein expression, and meaningful reduction in PVC and non- sustained VA burden, well-established risk factors for life-straitening and sustained VAs. Longer-term follow-up is ongoing to further evaluate the effect of PKP2 gene replacement therapy on electrophysiologic and other outcomes for patients with PKP2-ARVC."

Clinical • First-in-human • Late-breaking abstract • P1 data • Cardiomyopathy • Cardiovascular • Gene Therapies • Inflammation • TNNI3

Maintenance of Energy Metabolism Is an Integral Part of Plakophilin-2 and Desmosome Functions. (PubMed, JACC Basic Transl Sci) - "TN-401-mediated PKP2 expression improved mitochondrial and glycolytic energetics and rescued cardiomyocyte functions that are dictated by mechanical and structural integrity of desmosome. Therefore, maintaining energy metabolism of cardiomyocytes is an integral part of PKP2 and desmosome functions, adding a new layer of understanding to ARVC disease mechanism."

Journal • Cardiomyopathy • Cardiovascular • Gene Therapies • Heart Failure

RIDGETM-1: a phase 1b interventional study to evaluate safety and efficacy of TN-401, an adeno-associated virus serotype 9 (AAV9) investigational gene therapy, in adults with PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC) (ESGCT 2025) - No abstract available

Clinical • Gene therapy • P1 data • Cardiomyopathy • Cardiovascular • Gene Therapies

Tenaya Therapeutics Receives $8 Million Clinical Grant from California Institute for Regenerative Medicine (GlobeNewswire) - "Tenaya Therapeutics, Inc...today announced that the California Institute for Regenerative Medicine (CIRM), a state of California Agency that funds regenerative medicine, stem cell, and gene therapy research, has awarded Tenaya Therapeutics an $8.0 million CLIN2 grant. Proceeds from the grant will help fund clinical trial costs for Tenaya’s ongoing Phase 1b RIDGE-1 clinical trial of TN-401 gene therapy...'we look forward to sharing initial data from the low-dose cohort in the second half of this year.'"

Financing • P1 data • Cardiomyopathy • Cardiovascular

RIDGE-1: Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC (clinicaltrials.gov) - P1 | N=15 | Recruiting | Sponsor: Tenaya Therapeutics | Not yet recruiting ➔ Recruiting

Enrollment open • Cardiomyopathy • Cardiovascular

RIDGE-1: Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC (clinicaltrials.gov) - P1 | N=15 | Not yet recruiting | Sponsor: Tenaya Therapeutics

New P1 trial • Cardiomyopathy • Cardiovascular