Details of the Drug

General Information of Drug (ID: DMY0SKM)

Drug Name

BMN-307

Indication

Disease Entry	ICD 11	Status	REF
Phenylketonuria	5C50.0	Phase 1/2	[1]
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Drug Type

Gene therapy

Cross-matching ID

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Phenylalanine hydroxylase (PAH)	TTGSVH2	PH4H_HUMAN	Not Available	[1]

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Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This Drug

References

1	ClinicalTrials.gov (NCT04480567) 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. U.S.National Institutes of Health.