MyoAAV4 adenine base editor

MyoAAV4 adenine base editor is an experimental gene therapy candidate designed for the treatment of Duchenne muscular dystrophy (DMD). It utilizes the MyoAAV4 capsid, an engineered adeno-associated virus (AAV) variant with high tropism for skeletal and cardiac muscle, to deliver an adenine base editor (ABE) payload. The ABE is a CRISPR-derived tool that enables precise A•T to G•C base pair conversions without requiring double-strand DNA breaks, specifically targeting nonsense mutations in the dystrophin gene to restore functional protein expression. Research conducted at Indiana University has evaluated this asset in preclinical models (such as mdx4cv mice) to assess its efficacy and safety, including its interaction with lymphodepletion regimens and CAR-T cell-mediated B-cell depletion intended to facilitate AAV redosing by managing neutralizing antibodies.

Other names

MyoAAV4A-adenine base editorMyoAAV-4A-adenine base editorMyoAAV 4A-adenine base editor