Tenaya Receives Positive Safety Reviews from Independent DSMBs to Advance Both TN-201 and TN-401 Gene Therapy Clinical Trials as Designed

Core Insights - Tenaya Therapeutics has received positive endorsements from the Data Safety and Monitoring Boards (DSMB) for its two cardiovascular gene therapy clinical trials, MyPEAK-1 for TN-201 and RIDGE-1 for TN-401, allowing the trials to proceed as planned [1][2] MyPEAK-1 Phase 1b/2 Clinical Trial - The MyPEAK-1 trial is focused on TN-201 for MYBPC3-associated hypertrophic cardiomyopathy (HCM) and has completed enrollment in both dose cohorts [1] - The DSMB has reviewed data from the first three patients in Cohort 2 and determined that TN-201 has an acceptable safety profile, allowing for the enrollment of expansion cohorts at both 3E13 vg/kg and 6E13 vg/kg dose levels [3][4] - Initial data from Cohort 1 showed that TN-201 reached cardiomyocytes, resulting in increased MyBP-C protein levels and significant improvement in heart failure symptoms for patients [4][5] RIDGE-1 Phase 1b Clinical Trial - The RIDGE-1 trial is investigating TN-401 for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC) and has also completed initial patient enrollment [6] - The DSMB has endorsed dose escalation to 6E13 vg/kg and expansion of Cohort 1, with the first patient in Cohort 2 already dosed [6][7] - Initial data from Cohort 1 is expected to focus on safety and tolerability, with results anticipated in the fourth quarter of 2025 [7] Future Expectations - Tenaya plans to report longer-term follow-up data from Cohort 1 and initial data from Cohort 2 of both trials in the fourth quarter of 2025, which will inform dose selection and pivotal study design for both pediatric and adult populations [5][7] Company Overview - Tenaya Therapeutics is a clinical-stage biotechnology company focused on developing potentially curative therapies for heart disease, with a pipeline that includes TN-201 and TN-401 [13] - TN-201 is designed to deliver a functional MYBPC3 gene to heart muscle cells, while TN-401 targets the PKP2 gene to restore healthy protein levels in ARVC [10][12] - Both therapies have received Fast Track and Orphan Drug designations from the FDA, indicating their potential significance in treating rare diseases [10][12]