Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency

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.

Timeline

Start

2022-06-03

Primary completion

2023-09-14

Completion

2023-09-14

Outcome

Outcome not reported

Stopped: “Homology Medicines Inc. has discontinued the development of this program.”

Drugs

Indications