Homology Medicines

Homology Medicines is a gene therapy company dedicated to transforming the lives of patients with rare genetic diseases. Homology’s leading investigational products include HMI-102 and HMI-202. HMI-102 is for adult phenylketonuria (PKU), a metabolic disease caused by mutations in the PAH gene. The accumulation of the amino acid phenylalanine in the body leads to severe developmental and health issues. HMI-202 is for infantile metachromatic leukodystrophy (MLD), a rare lysosomal storage disorder caused by mutations in the ARSA gene. The ARSA gene encodes arylsulfatase A (ARSA), which functions to degrade cellular components. ARSA mutations result in the buildup of undegraded components within cells, damaging myelin-producing cells in the nervous system and causing neurological disorders.