HMI-103
Phenylketonuria (PKU) in pediatric patients
Key Facts
Indication
Phenylketonuria (PKU) in pediatric patients
Phase
Phase 1
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.
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