HMI-103

HMI-103 is an investigational, nuclease-free gene editing therapy designed to treat phenylketonuria (PKU) by harnessing the body's natural DNA repair process of homologous recombination. It uses an AAVHSC15 vector to insert a functional copy of the phenylalanine hydroxylase (PAH) gene and a liver-specific promoter into a specific region of the genome in liver cells. This approach aims to provide permanent correction for PKU by replacing at least one disease-causing allele with a normal sequence and maximizing PAH enzyme expression in all transduced cells. The therapy is being developed primarily for adults and children with classical PKU due to PAH deficiency.

Other names

Recombinant adeno-associated viral vector serotype HSC expressing human phenylalanine hydroxylase