Cleveland Clinic has infused a new gene therapy to deliver a working gene to address the leading cause of hypertrophic cardiomyopathy (HCM) in the first patient in the world as part of a clinical trial.
Myosin binding protein C3 (MYBPC3) gene mutations are the most common genetic cause of HCM. The gene therapy is designed to deliver a working MYBPC3 to the heart muscle through a one-time infusion of TN-201, which is a first-in-class adeno-associated virus-based gene therapy.
The hope is that this new gene will restore normal levels of the protein, which regulates the contraction and relaxation of the heart muscle. In preclinical studies, a single dose restored normal levels of the protein which led to disease reversal.
Hypertrophic cardiomyopathy is a complex type of heart disease that causes thickening of the heart muscle, left ventricular stiffness and mitral valve changes. It affects an estimated 600,000 to 1.5 million Americans, or one in 500 people, but many of those patients go undiagnosed until the disease has progressed.
The cause of hypertrophic cardiomyopathy may be unknown or attributed to genetics, high blood pressure or aging, making it difficult to identify a high-risk population. Symptoms include chest pain, palpitations, shortness of breath, fatigue and syncope (fainting). Most people with hypertrophic cardiomyopathy have a low risk for sudden cardiac death. However, the condition is the most common cause of sudden cardiac death in people under age 30.
“Our understanding of HCM has progressed and we are excited to be a part of combining that knowledge with technology like gene therapy to further it even more” said Milind Desai, MD, MBA, director of the Hypertrophic Cardiomyopathy Center at Cleveland Clinic and vice chair, Heart Vascular Thoracic Institute, Cleveland Clinic. Dr. Desai is an investigator for the MyPeak-1 Phase 1b clinical trial. “We look forward to continuing to study this therapy that involves a patient population that is often underdiagnosed and can suffer debilitating symptoms and sudden death.”
👉 Cleveland Clinic Performs World’s First In-Human Gene Therapy for Hypertrophic Cardiomyopathy – Cleveland Clinic Newsroom
