Overview

AAV Gene Therapy Study for Subjects With PKU

Status:
Active, not recruiting
Trial end date:
2027-12-01
Target enrollment:
Participant gender:
Summary
This is a Phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of BMN 307 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of BMN 307 and will be followed for safety and efficacy.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
BioMarin Pharmaceutical