Overview

Safety and Efficacy of HMI-103, a Gene Editing Development Candidate in Adults With Classical PKU Due to PAH Deficiency

Status:
Not yet recruiting
Trial end date:
2029-05-01
Target enrollment:
Participant gender:
Summary
This is an open-label, sequential ascending dose-escalation, Phase 1 study to evaluate the safety and efficacy of a single intravenous (I.V.) administration of HMI-103, a gene editing development candidate, in adult participants aged 18 to 55 years, inclusive, with classical PKU due to PAH deficiency who have uncontrolled disease despite Phe restricted dietary management.
Phase:
Phase 1
Details
Lead Sponsor:
Homology Medicines, Inc