drugset / Trial / NCT04480567
AAV Gene Therapy Study for Subjects with PKU
Non-randomizedSequentialOpen-labelTreatment
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.
Timeline
- Start
- 2020-09-24
- Primary completion
- 2027-12
- Completion
- 2027-12
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | BMN 307 | Gene therapy (AAV / viral vector) | — | Other |