Tenaya Therapeutics Publishes Preclinical Data Demonstrating TN-201 Enhances Cardiac Function and Survival in MYBPC3 Cardiomyopathy Models

Core Insights - Tenaya Therapeutics has published positive preclinical data for its gene therapy candidate TN-201, aimed at treating MYBPC3-associated hypertrophic cardiomyopathy (HCM) [1][2][3] Company Overview - Tenaya Therapeutics is a clinical-stage biotechnology company focused on developing potentially curative therapies for heart disease [1][13] - The company is currently evaluating TN-201 in the MyPEAK-1 Phase 1b/2 clinical trial, which is designed to assess the safety and efficacy of the therapy [11] Preclinical Findings - TN-201, an AAV9-based gene therapy, has shown dose-dependent increases in MyBP-C protein levels, improving cardiac function in preclinical models [2][5] - Treatment with TN-201 reversed left ventricular hypertrophy, a key feature of HCM, demonstrating significant improvements in cardiac parameters [2][8] - The therapy achieved near-maximal efficacy at doses of 3x10 vg/kg, with durable effects lasting up to 20 months post-treatment [9][15] Clinical Development - The MyPEAK-1 trial is enrolling symptomatic adults diagnosed with MYBPC3-associated HCM, testing doses of 3E13 vg/kg and 6E13 vg/kg [11] - Initial findings from the first cohort of patients in the MyPEAK-1 trial are expected to be presented at the upcoming American College of Cardiology Scientific Sessions [4] Disease Context - MYBPC3 mutations are the most common genetic cause of HCM, affecting approximately 120,000 patients in the U.S. [10] - The condition leads to severe symptoms and high rates of serious outcomes, with no approved therapies targeting the underlying genetic cause [10]