MyoAAV4A-CK8e-ABE

MyoAAV4A-CK8e-ABE is an experimental gene therapy designed for the treatment of Duchenne muscular dystrophy (DMD) caused by nonsense mutations. It utilizes a single-vector approach to deliver an adenine base editor (ABE) comprising a muscle-specific CK8e promoter, a compact Cas9 nickase, and a bespoke TadA8e deaminase. The therapy is packaged in the MyoAAV4A capsid, a highly myotropic AAV variant engineered for efficient delivery to skeletal and cardiac muscle. By performing precise A•T-to-G•C transitions, the drug aims to correct the DMD c.9445C>T (p.Q3149X) variant, thereby restoring the production of full-length, functional dystrophin protein. Preclinical studies in humanized mouse models have demonstrated significant restoration of dystrophin expression and improvement in cardiac and skeletal muscle function.