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Tenaya Therapeutics Doses First Hypertrophic Cardiomyopathy Patient in Gene Therapy Trial

NEW YORK – Tenaya Therapeutics on Thursday said the first hypertrophic cardiomyopathy (HCM) patient has received its investigational gene therapy TN-201 in a Phase Ib trial.

In the study, dubbed MyPeak-1, Tenaya is testing TN-201's ability to treat HCM associated with mutations in the MYBPC3 gene. TN-201 uses an adeno-associated virus vector to deliver a full-length functional copy of the same gene to a patient's heart muscle cells and is designed to restore normal levels of myosin-binding protein, which regulates heart muscle contractions and relaxations. The MYBPC3-linked form of disease accounts for about 20 percent of HCM cases.

Tenaya's aim in the open-label, dose-escalating study is to assess the safety, tolerability, and efficacy of a one-time intravenous infusion of TN-201. Investigators hope to enroll at least six symptomatic adults diagnosed with MYBPC3-associated nonobstructive HCM who have an implantable cardioverter defibrillator.

If the South San Francisco, California-based firm is able to establish safety and dosing for TN-201 in this subset of patients, Tenaya said it will study the drug in all HCM patients with a pathogenic MYBPC3 mutation.

The first trial site recruiting patients in MyPeak-1 is the hypertrophic cardiomyopathy center at the Cleveland Clinic. Tenaya plans to open up to 12 sites in the US and share initial data next year.