Tenaya Therapeutics’ MYPEAK-1 Trial Demonstrates Improved Outcomes with AAV Gene Therapy for Heart Disease
Tenaya Therapeutics’ gene therapy, TN-201, has shown promise in improving physical activity limitations in patients with severe heart disease, according to interim data from their Phase Ib/2 MYPEAK-1 trial (NCT05836259). Presented at the 2025 American College of Cardiology (ACC) conference in Chicago, the data revealed that a single dose of TN-201 was safe and well tolerated in the first three patients dosed in the first cohort.
This California-based company’s therapy for hypertrophic cardiomyopathy (HCM), caused by a deficiency of myosin-binding protein C (MyBP-C), led to an increase in MyBP-C protein levels in two of the first three patients after 52 weeks (from 56% to 59% and from 62% to 64%). Notably, all three patients experienced a decrease in their New York Heart Association classification level (a scale for measuring heart failure severity) from Class two or three to one. TN-201 is an investigational AAV9-based gene therapy and it aims to restore normal cardiac function by delivering a functional MYBPC3 gene to cardiomyocytes, enabling the production of cardiac MyBP-C. Administered systemically via intravenous injection, TN-201 leverages AAV9’s strong cardiac tropism to achieve targeted gene delivery.
Researchers also observed improvements in heart enlargement (hypertrophy) and a decrease in left ventricular posterior wall thickness by up to 40% and into a normal range in two patients. “For patients with HCM caused by mutations to the MYBPC3 gene, a population often at higher risk of serious and fatal complications, TN-201 is the first gene therapy to be tested,” said MyPEAK-1 trial investigator Milind Desai. “We need to continue studying this to evaluate whether this patient population may benefit from gene therapy, as has been seen in other diseases.”
https://www.clinicaltrialsarena.com/news/tenaya-highlights-hcm-gene-therapy-interim-data-at-acc-2025/?cf-view
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