HMI-202

**HMI-202** is an investigational in vivo gene therapy developed by Homology Medicines for metachromatic leukodystrophy (MLD), a rare lysosomal storage disorder caused by mutations in the ARSA gene leading to arylsulfatase A (ARSA) enzyme deficiency and sulfatide accumulation in the central and peripheral nervous systems. It uses an AAVHSC vector to deliver the human ARSA gene via single intravenous administration, crossing the blood-brain and blood-nerve barriers to achieve therapeutic ARSA expression and activity in brain regions, lysosomes, and other MLD-relevant tissues, modulating biomarkers like sulfatides, LAMP-1, and myelin transcripts in preclinical murine models over 48 weeks and confirming barrier penetration in non-human primates.[1][2][3][4][5]