SOUTH SAN FRANCISCO, Calif., June 2, 2026
Tenaya Therapeutics announced that it will present new interim clinical data from Cohort 1 and Cohort 2 of the ongoing MyPEAK™-1 Phase 1b/2 trial evaluating TN-201, its investigational gene therapy for adults with MYBPC3-associated hypertrophic cardiomyopathy (HCM). The company plans to share the latest findings during a webcast scheduled for June 3, highlighting progress in the development of a potentially disease-modifying treatment designed to address the genetic cause of one of the most common inherited heart diseases. The update is expected to provide investors, clinicians, and researchers with further insight into the therapy’s safety and early efficacy profile.
TN-201 Targets the Root Cause of Inherited Cardiomyopathy
TN-201 is an AAV9-based gene therapy engineered to deliver a functional copy of the MYBPC3 gene directly to heart muscle cells through a single intravenous infusion. Mutations in the MYBPC3 gene are responsible for approximately 20% of all hypertrophic cardiomyopathy cases in the United States, affecting an estimated 120,000 individuals. These mutations result in reduced levels of the MyBP-C protein, causing abnormal heart muscle thickening, impaired cardiac function, and an increased risk of heart failure, arrhythmias, stroke, and sudden cardiac death. By restoring MyBP-C protein production, TN-201 aims to halt disease progression and potentially reverse structural heart abnormalities after a single treatment.
MyPEAK™-1 Trial Evaluates Safety and Clinical Impact
The ongoing MyPEAK™-1 Phase 1b/2 clinical study is assessing the safety, tolerability, and biological activity of TN-201 in adults diagnosed with MYBPC3-associated HCM. Researchers are evaluating whether the therapy can improve cardiac function and reduce disease burden by addressing the underlying genetic defect rather than merely managing symptoms. The upcoming interim analysis from Cohort 1 and Cohort 2 is expected to offer important evidence regarding TN-201’s therapeutic potential. The program has already received FDA Fast Track, Orphan Drug, and Rare Pediatric Disease Designations, reflecting the significant unmet medical need and the promise of gene therapy for inherited cardiovascular diseases.
Advertisement
Expanding a Pipeline of Potentially Curative Heart Disease Therapies
Beyond TN-201, Tenaya Therapeutics is advancing a growing portfolio of innovative cardiovascular therapies aimed at treating both rare genetic disorders and broader heart conditions. The company’s pipeline includes TN-401 for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC) and TN-301, a selective HDAC6 inhibitor being developed for conditions including heart failure with preserved ejection fraction (HFpEF) and Duchenne muscular dystrophy (DMD). Through its integrated research platform and focus on genetic medicine, Tenaya is working to develop potentially curative treatments that directly target the biological drivers of heart disease. The upcoming MyPEAK™-1 data presentation represents an important milestone in advancing gene therapy approaches for patients with inherited cardiomyopathies.
Source: Tenaya Therapeutics press release
