HMI-104 / HMI-105
Metachromatic Leukodystrophy (MLD)
PreclinicalDiscontinued
Key Facts
Indication
Metachromatic Leukodystrophy (MLD)
Phase
Preclinical
Status
Discontinued
Company
About Homology Medicines
Homology Medicines was a biotechnology company with a mission to develop curative genetic medicines for rare diseases through its dual-platform approach of gene therapy and nuclease-free gene editing. Its core assets were a suite of 15 human-derived AAVHSC vectors, which powered a clinical pipeline led by candidates for phenylketonuria (PKU). In March 2024, the company announced a strategic wind-down of its clinical programs, pivoting to seek partnerships or a sale of its intellectual property and technology platforms, marking a significant transition from drug developer to asset licensor.
View full company profileTherapeutic Areas
Other Metachromatic Leukodystrophy (MLD) Drugs
| Drug | Company | Phase |
|---|---|---|
| Libmeldy (atidarsagene autotemcel) | Kyowa Hakko Kirin | Marketed |
| Atidarsagene autotemcel | Pfizer | Phase 3 |