Details of the Drug

General Information of Drug (ID: DM8M71U)

Drug Name

DTX301

Indication

Disease Entry	ICD 11	Status	REF
Ornithine transcarbamylase deficiency	5C50.AY	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
Ornithine transcarbamylase (OTC)	TT5KIO9	OTC_HUMAN	Replacement	[2]

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Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This Drug

References

1	ClinicalTrials.gov (NCT02991144) Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset OTC Deficiency (CAPtivate). U.S. National Institutes of Health.
2	Clinical pipeline report, company report or official report of Ultragenyx Pharmaceutical.