AAV-MYBPC3

AAV-MYBPC3 is an adeno-associated virus (AAV)-based gene therapy designed to treat hypertrophic cardiomyopathy (HCM) caused by mutations in the MYBPC3 gene, which leads to haploinsufficiency of cardiac myosin-binding protein C (cMyBP-C). The therapy involves the delivery of a functional MYBPC3 expression cassette using a cardiotropic AAV capsid to restore physiological levels of cMyBP-C in cardiomyocytes. This restoration aims to normalize cardiac contractility and improve heart function. Alexion Pharmaceuticals is developing a version of AAV-MYBPC3 that utilizes a novel cardiotropic capsid, which has demonstrated significantly higher functional cardiac transduction compared to AAV9 in non-human primate models. Other companies, such as Tenaya Therapeutics (TN-201) and Rocket Pharmaceuticals (RP-A601), also have AAV-MYBPC3 programs in development.