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BMN 307 is an investigational AAV5-phenylalanine hydroxylase (PAH) gene therapy designed to normalize blood phenylalanine (Phe) concentration levels in patients with PKU.

BMN 307 represents a potential third PKU treatment option from BioMarin, and our second gene therapy clinical program. Both the FDA and European Medicines Agency have granted BMN 307 Orphan Status. BMN 307 has not been determined to be safe or effective and is not approved for use.

Phase (Preclinical, Phase 1, Phase 2, Phase 3, BLA/NDA/MAA, Approval)
  • Phenylketonuria: BMN 307
    Phase 1
    Target Indication

    BMN 307 is an investigational AAV5-mediated gene therapy designed to address phenylketonuria (PKU)

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