Details of the Drug-Related molecule(s) Interaction Atlas

General Information of Drug (ID: DM8M71U)

Drug Name

DTX301 Drug Info

Indication

Disease Entry	ICD 11	Status	REF
Ornithine transcarbamylase deficiency	5C50.AY	Phase 1/2	[1]

Cross-matching ID

Molecule-Related Drug Atlas

Molecule Type:

DTT

Drug Status:

Clinical Trial Drug(s)

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Drug(s) Targeting Ornithine transcarbamylase (OTC)

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
Avalotcagene ontaparvovec	DMAOHT4	Ornithine transcarbamylase deficiency	5C50.AY	Phase 3	[3]
ARCT-810	DM91F9I	Ornithine transcarbamylase deficiency	5C50.AY	Phase 2	[4]
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Molecular Interaction Atlas of This Drug

Molecular Interaction Atlas

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Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Ornithine transcarbamylase (OTC)	TT5KIO9	OTC_HUMAN	Replacement	[2]

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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.
3	Prednisolone reduces the interferon response to AAV in cynomolgus macaques and may increase liver gene expression. Mol Ther Methods Clin Dev. 2022 Jan 19;24:292-305.
4	Clinical pipeline report, company report or official report of Arcturus Therapeutics