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