MyoAAV4A

MyoAAV4A is an engineered, myotropic adeno-associated virus (AAV) capsid designed for the targeted delivery of genetic payloads to muscle tissues, with a specific emphasis on cardiomyocytes. Developed as part of a series of synthetic capsids (including MyoAAV2A), MyoAAV4A is optimized to provide high transduction efficiency in human cardiac cells while potentially reducing off-target delivery to the liver. In preclinical evaluations using 3D engineered human heart (EHH) tissues derived from human induced pluripotent stem cells (hiPSCs), MyoAAV4A demonstrated the ability to drive robust and sustained transgene expression specifically within cardiomyocytes, with minimal transduction of fibroblasts or endothelial cells. This vector is being explored as a delivery vehicle for gene therapies targeting various cardiomyopathies and muscular dystrophies.