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Tenaya Therapeutics Doses First Patient in the MyPeak-1™ Phase 1b Clinical Trial of TN-201 for the Treatment of MYBPC3-Associated Hypertrophic Cardiomyopathy
TN-201 is the First Gene Therapy for the Leading Genetic Cause of HCM to be Assessed in Humans; Adds Working MYBPC3 Gene to Heart Cells to Address Underlying
About this update from Tenaya Therapeutics, Inc.
[{"type":"text","content":"TN-201 is the First Gene Therapy for the Leading Genetic Cause of HCM to be Assessed in Humans; Adds Working MYBPC3 Gene to Heart Cells to Address Underlying Cause of Disease\nSOUTH SAN FRANCISCO, Calif., Oct. 05, 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, today announced that the first patient has been dosed with TN-201 gene therapy for the treatment of Myosin Binding Protein C3 (MYBPC3)-associated HCM in the MyPeak-1 Phase 1b clinical trial at the Cleveland Clinic, Cleveland, Ohio. Tenaya anticipates sharing initial data from the MyPeak-1 trial in 2024. TN-201 is Tenaya’s potential first-in-class adeno-associated virus (AAV)-based gene therapy designed to deliver a working, full-length copy of the human MYBPC3 gene to heart muscle cells. The working MYBPC3 gene is intended to restore normal levels of myosin-binding protein, which regulates the contraction and relaxation of the heart muscle. In preclinical studies, TN-201 halted disease progression and demonstrated significant and durable disease reversal and survival benefit following a single dose. “MYBPC3 gene mutations are the most common genetic cause of HCM and people with MYBPC3-associated HCM are at increased risk for accelerated decline and serious complications associated with their condition,” said Milind Desai, M.D., MBA, Director at the Cleveland Clinic Hypertrophic Cardiomyopathy Center and Vice Chair, Heart Vascular Thoracic Institute, Cleveland Clinic, and an investigator for the MyPeak-1 Phase 1b clinical trial. “TN-201, a gene therapy for MYBPC3-associated HCM, offers the potential of a one-time treatment to correct the underlying genetic cause of disease and improve patient outcomes. We are pleased to participate in the first-in-human clinical trial of TN-201 to explore this new use of gene therapy treatment.” The MyPeak-1 Phase 1b clinical trial is a multi-center, open-label, dose-escalating study designed to assess the safety, tolerability and clinical efficacy of a one-time intravenous infusion of TN-201. The trial will initially seek to enroll at least six symptomatic (New York Heart Association Class II or III) adults who have been diagnosed with MYBPC3-associate...