Tenaya fast tracked for gene therapy candidate

TN-201 Being Developed for the Potential Treatment of MYBPC3-associated HCM

Dosing in Phase 1 Clinical Trial Expected to Commence in Q3 2023

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 U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its gene therapy product candidate, TN-201, being developed for the treatment of Myosin Binding Protein C3 (MYBPC3)-associated hypertrophic cardiomyopathy (HCM).

TN-201 is Tenaya’s potential first-in-class adeno-associated virus (AAV)-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.

https://finance.yahoo.com/news/tenaya-therapeutics-receives-fda-fast-115000395.html