Photo by Dalle-E OpenAI

Tenaya Therapeutics Initiates First-in-Human Clinical Trial for Gene Therapy in Heart Disease Treatment

Tenaya Therapeutics, a clinical-stage biotechnology company, has announced the initiation of its first-in-human clinical trial for TN-201 gene therapy. The trial aims to evaluate the potential of TN-201 in treating Myosin Binding Protein C3 (MYBPC3)-associated Hypertrophic Cardiomyopathy (HCM). The first patient has been dosed with TN-201 at the Cleveland Clinic in Ohio.

HCM is the most common inherited cardiac disorder, and MYBPC3 gene mutations are the leading genetic cause of HCM. MYBPC3-associated HCM affects approximately 115,000 patients in the United States alone. This chronic condition can lead to serious complications, including heart failure and sudden cardiac death.

TN-201 is an adeno-associated virus (AAV)-based gene therapy designed to deliver a functional MYBPC3 gene to heart muscle cells. By restoring normal levels of myosin-binding protein, TN-201 aims to regulate the contraction and relaxation of the heart muscle. Preclinical studies have shown promising results, with TN-201 halting disease progression and demonstrating significant disease reversal and survival benefits.

The MyPeak-1 Phase 1b clinical trial is a multi-center study that will assess the safety, tolerability, and clinical efficacy of TN-201. The trial aims to enroll at least six symptomatic adults with MYBPC3-associated nonobstructive HCM. The initial dose of TN-201 being evaluated is associated with near-maximal efficacy in preclinical studies.

Dr. Milind Desai, an investigator for the MyPeak-1 trial at the Cleveland Clinic, highlighted the potential of TN-201 as a one-time treatment to correct the underlying genetic cause of MYBPC3-associated HCM and improve patient outcomes. The trial’s initial data is expected to be shared in 2024.

Tenaya Therapeutics expressed gratitude for the support of study sites, patient advocacy organizations, and patients and families involved in their efforts to explore the potential of TN-201 as a novel treatment for MYBPC3-associated HCM. The company plans to enroll additional patients in the MyPeak-1 study and subsequent studies.

The initiation of the MyPeak-1 clinical trial marks a significant milestone for Tenaya Therapeutics in their mission to develop new treatments that target the genetic underpinnings of heart disease. With their integrated Gene Therapy, Cellular Regeneration, and Precision Medicine platforms, Tenaya is advancing a pipeline of therapies for rare genetic cardiovascular disorders and more prevalent heart conditions.

For more information about the gene therapy for HCM and participation in the MyPeak-1 study, interested individuals can visit or (NCT05836259).

Tenaya Therapeutics is dedicated to discovering, developing, and delivering potentially curative therapies that address the underlying drivers of heart disease. Their pipeline includes TN-201 for MYBPC3-associated HCM, TN-401 for PKP2-associated arrhythmogenic right ventricular cardiomyopathy (ARVC), and TN-301 for heart failure with preserved ejection fraction (HFpEF).

Leave a comment