Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D6V9FN
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| Drug Name |
Avalotcagene ontaparvovec
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| Synonyms |
DTX301
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| Drug Type |
Gene therapy
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| Indication | Ornithine transcarbamylase deficiency [ICD-11: 5C50.AY] | Phase 3 | [1] | |
| Company |
Ultragenyx Pharmaceutical
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Ornithine transcarbamylase (OTC) | Target Info | . | [2] |
| References | Top | |||
|---|---|---|---|---|
| REF 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. | |||
| REF 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. | |||
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