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Tenaya Therapeutics Announces FDA Clearance to Begin Clinical Testing of TN-401 Gene Therapy for the Treatment of PKP2-Associated Arrhythmogenic Right Ventricular Cardiomyopathy
PKP2 Mutations Are the Leading Cause of ARVC, a Dangerous Condition Linked to Sudden Cardiac Arrest in Young People Estimated to Affect 70,000 People in the
About this update from Tenaya Therapeutics, Inc.
[{"type":"text","content":"PKP2 Mutations Are the Leading Cause of ARVC, a Dangerous Condition Linked to Sudden Cardiac Arrest in Young People Estimated to Affect 70,000 People in the US TN-401 Preclinical Studies Demonstrated Robust Reduction of Ventricular Arrhythmias and Extended Survival in Knockout Models of Disease After a Single Dose SOUTH SAN FRANCISCO, Calif., Oct. 26, 2023 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, announced today 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 (AAV9)-based investigational gene therapy product candidate for the treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by mutations in the plakophilin-2 (PKP2) 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. ARVC, also known as arrhythmogenic cardiomyopathy (ACM), is a chronic, progressive, familial disease that typically presents before age 40. People with ARVC experience symptoms related to ventricular arrhythmias, including palpitations, lightheadedness and fainting, and are at increased risk of sudden cardiac death. PKP2 mutations are the most common genetic cause of ARVC and result in a loss of key proteins needed to maintain the structural integrity and cell-to-cell signaling of heart muscle cells. TN-401 is designed to address the underlying cause of disease by delivering a fully functional PKP2 gene to restore normal PKP2 protein levels and thereby slow disease progression and reverse the course of disease after a single dose. “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 c...