Tenaya Therapeutics announces FDA clearance to begin testing of TN-401

Tenaya Therapeutics announced that the U.S. Food and Drug Administration, FDA, has provided clearance of the company’s Investigational New Drug, IND, application to initiate clinical testing of TN-401. TN-401 is Tenaya’s adeno-associated virus serotype 9-based investigational gene therapy product candidate for the treatment of arrhythmogenic right ventricular cardiomyopathy caused by mutations in the plakophilin-2 gene. Based on this IND clearance, the company plans to initiate the RIDGE-1 Phase 1b clinical trial of TN-401, a multi-center, open-label study to assess the safety, tolerability and clinical efficacy of a one-time intravenous infusion of TN-401. Tenaya is currently conducting the RIDGE global non-interventional natural history and serotype study of PKP2-associated ARVC. “People with arrhythmogenic cardiomyopathy report high levels of fear and stress and must withstand burdensome physical and lifestyle restrictions in an effort to manage the frequent abnormal heart rhythms and constant risk of sudden cardiac arrest associated with their disease,” said Whit Tingley, M.D., Ph.D., Tenaya’s Chief Medical Officer. “TN-401 is intended to address the genetic mutation most frequently underlying ARVC. The initial dose for TN-401 in the RIDGE-1 study was associated with near maximal efficacy in our preclinical studies. With clinical site and patient community engagement well underway, we look forward to rapidly advancing TN-401 into the clinic.”