Specialists at Cleveland Clinic have treated a hypertrophic cardiomyopathy (HCM) patient with a new gene therapy designed to reverse disease progression.
This represents the first patient to receive treatment as part of the MyPeak-1 Phase Ib clinical trial, a first-in-human study sponsored by Tenaya Therapeutics. The trial intends to recruit at least six adult patients diagnosed with MYBPC3-associated nonobstructive HCM.
Myosin binding protein C3 (MYBPC3) gene mutations are a common cause of HCM. The group hopes this new-look gene therapy, known as TN-201, can restore the patient’s MYBPC3 levels and eventually lead to disease reversal. The only other HCM treatments at this time are mavacamten, which the U.S. Food and Drug Administration (FDA) approved in 2022, and more invasive procedures such as septal myectomy and alcohol ablation.
Cardiologist and cardiac imaging specialist Milind Desai, MD, director of Cleveland Clinic’s Hypertrophic Cardiomyopathy Center and vice chair of its Heart, Vascular and Thoracic Institute, is one of the trial’s primary investigators. Desai has years of experience studying potential HCM treatments and even presented data at ACC.22 on mavacamten’s safety and effectiveness before it had received FDA approval. He is also a paid consultant of Tenaya Therapeutics.