AAV5-lcoET3

AAV5-lcoET3 is an experimental adeno-associated virus serotype 5 (AAV5) gene therapy vector encoding a bioengineered high-activity variant of human factor VIII (ET3) under an expression cassette optimized with a long codon-optimized (lco) sequence to enhance hepatic expression and secretion of factor VIII for the treatment of hemophilia A. Preclinical studies of related ET3-LCO constructs have shown markedly increased and durable factor VIII activity in hemophilia A mouse models compared with earlier FVIII transgenes, suggesting that AAV5-lcoET3 aims to achieve clinically meaningful, long-term correction of factor VIII deficiency following a single intravenous administration.[7]