LV-PAH

LV-PAH is an investigational lentiviral vector-mediated gene therapy being developed by Castle Creek Biosciences for the treatment of phenylketonuria (PKU). The therapy is designed to deliver a functional copy of the phenylalanine hydroxylase (PAH) gene to the patient's cells, potentially restoring the body's ability to metabolize phenylalanine. PKU is a rare genetic metabolic disorder caused by mutations in the PAH gene, which leads to the toxic accumulation of phenylalanine in the blood and brain, potentially causing intellectual disability and other neurological complications. LV-PAH is currently in the preclinical stage of development.