AAV9-hPCCA

AAV9-hPCCA is an adeno-associated virus (AAV) serotype 9-based gene therapy designed for the treatment of propionic acidemia (PA), a rare organic acidemia. It delivers a functional copy of the human propionyl-CoA carboxylase alpha subunit (PCCA) gene to restore the activity of the propionyl-CoA carboxylase (PCC) enzyme, which is deficient in patients with PCCA mutations. Developed as the lead candidate of the NIH's Platform Vector Gene Therapy (PaVe-GT) program, the therapy utilizes a platform approach to streamline preclinical and clinical development, including standardized manufacturing and regulatory strategies. The program aims to communicate efficiencies that could benefit drug developers in the rare disease space and reduce development costs.

Other names

AAV serotype 9 carrying the human propionyl-CoA carboxylase Alpha subunit gene