Details of the Drug

General Information of Drug (ID: DMAOHT4)

Drug Name

Avalotcagene ontaparvovec

Synonyms

DTX301

Indication

Disease Entry	ICD 11	Status	REF
Ornithine transcarbamylase deficiency	5C50.AY	Phase 3	[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	Not Available	[2]

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Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This Drug

References

1	ClinicalTrials.gov (NCT05345171) A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients With Late-onset OTC Deficiency. U.S.National Institutes of Health.
2	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.