bevufenogene nofeparvovec

Bevufenogene nofeparvovec is an investigational gene therapy that utilizes a recombinant adeno-associated virus vector, specifically hematopoietic stem cell serotype 15 (AAVHSC15), to deliver a functional copy of the human phenylalanine hydroxylase (PAH) gene. It was developed for the treatment of phenylketonuria (PKU), a rare inherited metabolic disorder caused by mutations in the PAH gene leading to impaired metabolism of phenylalanine. The therapy is administered intravenously and aims to restore PAH enzyme activity through targeted gene transference, potentially correcting the underlying genetic defect in PKU patients. The drug was originally developed by Homology Medicines and later by Q32 Bio following a merger between the two companies. Development for PKU has been discontinued after termination of clinical trials[2][4][5].

Other names

HMI-102HMI102HMI 102AAVHSC15-PAHAAVHSC-15-PAHAAVHSC 15-PAH