HMI-204

HMI-204 is an investigational gene therapy candidate developed by Homology Medicines for the treatment of metachromatic leukodystrophy (MLD), a rare and fatal lysosomal storage disorder. It utilizes an adeno-associated virus (AAV) vector to deliver a functional copy of the ARSA gene, which encodes the arylsulfatase A enzyme. The therapy is designed for systemic administration via a single intravenous infusion and is engineered to cross the blood-brain barrier to address both central and peripheral nervous system manifestations of MLD. Following the reverse merger of Homology Medicines with Q32 Bio in 2024, HMI-204 was designated as a legacy asset intended for monetization through a contingent value right (CVR) structure, with active development by the parent company halted.