HMI-204
Metachromatic Leukodystrophy (MLD)
PreclinicalDiscontinued
Key Facts
Indication
Metachromatic Leukodystrophy (MLD)
Phase
Preclinical
Status
Discontinued
Company
About Homology Medicines
Homology Medicines was a biotech company developing genetic medicines for rare diseases using its proprietary nuclease-free gene editing and human AAV-derived gene therapy platforms. Its lead programs targeted phenylketonuria (PKU), with HMI-102 in gene therapy and HMI-103 in gene editing. Facing clinical and financial challenges, the company halted all clinical development in March 2024, reduced its workforce, and initiated a process to seek strategic alternatives for its intellectual property and technology platforms, effectively winding down its former operations.
View full company profileTherapeutic Areas
Other Metachromatic Leukodystrophy (MLD) Drugs
| Drug | Company | Phase |
|---|---|---|
| Libmeldy (atidarsagene autotemcel) | Kyowa Hakko Kirin | Approved |
| Atidarsagene autotemcel | Pfizer | Phase 3 |