Gene Therapy Clinical Study in Adult PKU

Summary

This is a Phase 1/2, open-label, randomized, concurrently-controlled, dose escalation study to evaluate the safety and efficacy of HMI-102 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of HMI-102 and will be followed for safety and efficacy for 1 year.

Timeline

Start

2019-06-10

Primary completion

2023-01-10

Completion

2023-08-01

Outcome

Outcome not reported

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

Drugs

Indications