Tenaya Therapeutics, Inc. announced that it will report new interim data from Cohort 1 and Cohort 2 of the ongoing MyPEAK-1 Phase 1b/2 trial of TN-201 gene therapy for adults with MYBPC3-associated hypertrophic cardiomyopathy (HCM) on June 3, 2026. Variants in the Myosin Binding Protein C3 (MYBPC3) gene are the most common genetic cause of hypertrophic cardiomyopathy (HCM), accounting for approximately 20% of the overall HCM population, or 120,000 patients, in the United States alone. MYBPC3-associated HCM is a severe and progressive condition affecting adults, teens, children and infants.

Mutations of the MYBPC3 gene result in insufficient expression of a protein, called MyBP-C, needed to regulate heart contraction. The heart becomes hypercontractile and the left ventricle thickens, resulting in symptoms such as chest pain, shortness of breath, palpitations and fainting. Patients whose disease is caused by MYBPC3 mutations are more likely than those with non-genetic forms of HCM to experience earlier disease onset and have high rates of serious outcomes, including heart failure symptoms, arrhythmias, stroke and sudden cardiac arrest or death.

There are currently no approved therapeutics that address the underlying genetic cause of HCM. TN-201 is an adeno-associated virus serotype 9 (AAV9)-based gene therapy designed to address the underlying cause of MYBPC3-associated hypertrophic cardiomyopathy (HCM) by delivering a working MYBPC3 gene to heart muscle cells via a single intravenous infusion and thereby increasing insufficient MyBP-C protein levels with the aim of halting or even reversing disease after a single dose. The U.S. Food and Drug Administration has granted TN-201 Fast Track, Orphan Drug and Rare Pediatric Drug Designations.

TN-201 has also received orphan medicinal product from the European Commission.