HMI-106
Hunter Syndrome (MPS II)
PreclinicalDiscontinued
Key Facts
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 Hunter Syndrome (MPS II) Drugs
| Drug | Company | Phase |
|---|---|---|
| MPS II (Hunter Syndrome) Program | NeuroGT | Pre-clinical |
| RG6356 (DNL310) | Roche | Phase II/III |
| JR-141 (pabinafusp alfa) | JCR Pharmaceuticals | Approved |
| DNL310 | Denali Therapeutics | Phase 2/3 |