4D-C102

**4D-C102** is a proprietary adeno-associated virus (AAV) serotype 2 capsid variant engineered by 4D Molecular Therapeutics using their Therapeutic Vector Evolution platform for superior tropism to cardiac and skeletal muscle tissues. It enables efficient intravenous gene delivery with high transduction efficiency in human pluripotent stem cell-derived cardiomyocytes and primary skeletal muscle cells in vitro, outperforming wild-type AAV serotypes like AAV1, AAV8, and AAV9. In non-human primates, systemic administration demonstrated robust genome delivery and protein expression primarily in heart and skeletal muscle with minimal off-target distribution to liver or spleen and low inflammation. 4D-C102 is the capsid component of the investigational gene therapy **4D-310**, which carries a codon-optimized human alpha-galactosidase A (GLA) transgene for the treatment of Fabry disease cardiomyopathy, targeting cell-autonomous enzyme production in cardiomyocytes and kidney glomeruli.[1][3][7][11]