Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D6XMS1
|
|||
| Drug Name |
DTX301
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | Ornithine transcarbamylase deficiency [ICD-11: 5C50.AY] | Phase 1/2 | [1] | |
| Company |
Ultragenyx Pharmaceutical
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Ornithine transcarbamylase (OTC) | Target Info | Replacement | [2] |
| References | Top | |||
|---|---|---|---|---|
| REF 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. | |||
| REF 2 | Clinical pipeline report, company report or official report of Ultragenyx Pharmaceutical. | |||
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