AGT324

AGT324 is an investigational **lentiviral vector gene therapy** being developed by American Gene Technologies for **phenylketonuria**. The program is designed to modify liver cells to restore **phenylalanine hydroxylase** activity, thereby lowering toxic phenylalanine levels and potentially providing a durable or single-dose corrective treatment for this inherited metabolic disorder. Publicly available company materials indicate a proprietary lentiviral platform, and associated patent material describes vectors expressing **wild-type PAH** and, in some embodiments, incorporating **shRNA directed against defective endogenous PAH transcripts**. The asset appears to have received FDA orphan drug designation for PKU, but publicly visible evidence of clinical-stage testing specific to AGT324 was not identified.