Tenaya Therapeutics receives FDA fast track designation for TN-201

Tenaya Therapeutics announced that the U.S. Food and Drug Administration has granted Fast Track designation for its gene therapy product candidate, TN-201, being developed for the treatment of Myosin Binding Protein C3-associated hypertrophic cardiomyopathy. TN-201 is Tenaya’s potential first-in-class adeno-associated virus-based investigational gene therapy for the treatment of HCM caused by mutations in the MYBPC3 gene, the most common genetic cause of HCM. TN-201 is designed to deliver a fully functional MYBPC3 gene to restore normal levels of myosin-binding protein, which regulates the contraction and relaxation of the heart muscle. In preclinical studies of MYBPC3 knock-out models, TN-201 has been shown to halt disease progression and demonstrated significant and durable disease reversal and survival benefit after a single dose. In January 2023, Tenaya announced that the FDA cleared its Investigational New Drug application for TN-201. Tenaya is initiating the MyPeak-1 Phase 1b clinical trial to assess the safety, tolerability and clinical efficacy of a one-time intravenous infusion of TN-201. The multi-center, open-label study will enroll adults diagnosed with MYBPC3-associated nonobstructive HCM. Tenaya anticipates the first patient will be dosed in this clinical trial during the third quarter of 2023. Tenaya is also conducting two non-interventional studies to support the development of TN-201: a study evaluating seroprevalence to AAV9 antibodies among adults with MYBPC3-associated HCM, and MyClimb, a natural history study of pediatric patients with MYBPC3-associated HCM.