A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects with Phenylketonuria and Plasma Phe Levels > 600umol/L

Grants and Contracts Details

StatusFinished
Effective start/end date3/13/23 → 6/30/24

Funding

  • BioMarin Pharma Inc: $34,425.00