AAV9-hPCCB

AAV9-hPCCB is an adeno-associated virus serotype 9 (AAV9) gene therapy designed to treat propionic acidemia (PA) caused by mutations in the PCCB gene. Propionic acidemia is a rare autosomal recessive metabolic disorder resulting from a deficiency in propionyl-CoA carboxylase (PCC), a mitochondrial enzyme composed of alpha (PCCA) and beta (PCCB) subunits. AAV9-hPCCB delivers a functional copy of the human PCCB gene to restore enzyme activity, reduce toxic metabolites, and improve metabolic stability. Developed by the National Institutes of Health (NIH) as part of the Bespoke Gene Therapy Consortium (BGTC), the therapy has demonstrated preclinical efficacy in mouse models, showing improved survival and metabolic correction.

Other names

AAV9-PCCBAAV-9-PCCBAAV 9-PCCB