Tenaya Therapeutics Reports Positive Interim Data from Cohort 1 of RIDGE™-1 Phase 1b/2 Clinical Trial of TN-401 Gene Therapy for Adults with PKP2-associated ARVC

Core Insights - Tenaya Therapeutics announced interim data from the RIDGE™-1 Phase 1b/2 clinical trial of TN-401 gene therapy for arrhythmogenic right ventricular cardiomyopathy (ARVC), showing promising safety and efficacy results in early patients [1][3] Group 1: Clinical Trial Results - TN-401 was well tolerated at a dose of 3E13 vg/kg, with no dose-limiting toxicities observed [4] - Initial data indicated a significant increase in PKP2 protein expression from baseline in two of three patients, with a mean increase of 10% by Week 8 [12] - Clinically meaningful reductions in arrhythmia burden were observed, with Patient 1 experiencing a 46% decrease in premature ventricular contractions (PVCs) and Patient 2 showing an 89% decrease, along with a reduction of non-sustained ventricular tachycardia (NSVT) counts from 78 to zero [12] Group 2: Safety and Tolerability - Adverse events were generally mild and asymptomatic, with no serious adverse events (SAEs) related to TN-401 treatment reported in the cohort [6][5] - No incidents of thrombotic microangiopathy (TMA) or cardiotoxicities were observed, and all patients have tapered off immunosuppressive medicines [6] - Biopsies demonstrated robust transduction and expression of TN-401 in all patients within the first eight weeks [6] Group 3: Future Developments - Tenaya management plans to host a webcast conference call to discuss the TN-401 data on December 11, 2025 [9] - The RIDGE-1 trial aims to enroll up to fifteen adults diagnosed with PKP2-associated ARVC, assessing the safety, tolerability, and preliminary clinical efficacy of TN-401 [14] - TN-401 has received Orphan Drug and Fast Track Designations from the U.S. Food and Drug Administration, indicating its potential significance in treating ARVC [13]