Clinical Trial Update: AAV Gene Therapy Study for Subjects with PKU [Phase 1, Phase 2]

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Summary

BioMarin's AAV-based gene therapy for phenylketonuria (PKU) is being studied in a Phase 1/Phase 2 trial and is currently active but not recruiting.

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Diseases / indications

• Phenylketonuria

Drugs mentioned

• BMN307 (AAV PKU gene therapy)

Snippet

Sponsor: BioMarin Pharmaceutical | Conditions: Phenylketonuria (PKU) | Phase 1, Phase 2 | Status: ACTIVE_NOT_RECRUITING