October 6, 2023- 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.
Hypertrophic cardiomyopathy is a complex type of heart disease that causes thickening of the heart muscle, left ventricular stiffness and mitral valve changes.
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.