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Tenaya Therapeutics Receives FDA Fast Track Designation for TN-201
TN-201 Being Developed for the Potential Treatment of MYBPC3-associated HCM Dosing in Phase 1 Clinical Trial Expected to Commence in Q3 2023 SOUTH SAN
About this update from Tenaya Therapeutics, Inc.
[{"type":"text","content":"TN-201 Being Developed for the Potential Treatment of MYBPC3-associated HCM Dosing in Phase 1 Clinical Trial Expected to Commence in Q3 2023 SOUTH SAN FRANCISCO, Calif., May 02, 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 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. “Receipt of Fast Track designation for TN-201 reflects the pressing unmet need among HCM patients whose disease is caused by MYBPC3 genetic mutations,” said Whit Tingley, M.D., Ph.D., Chief Medical Officer of Tenaya. “As we prepare to begin dosing patients later this year, we look forward to continued close collaboration with the FDA under this designation in support of TN-201’s development.” The FDA Fast Track program is designed to facilitate the development and expedite the review of drug candidates intended to treat serious conditions and for which nonclinical data demonstrates the potential to address unmet medical need. Companies with therapies that receive the Fast Track designation from the FDA are eligible for increased communication with the agency and may qualify for accelerated approval and priority review if relevant criteria are met. The goal of the program is to deliver approved treatments to patients with a serious or life-threatening condition as quickly as possible. This designation is not an assurance that regulatory approval will be received. TN-201 also has ...