Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D5WAG1
|
|||
| Drug Name |
DTX401
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | Pompe disease [ICD-11: 5C51.3; ICD-10: E74.0] | Phase 1 | [1] | |
| Company |
Ultragenyx Pharmaceutical
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Glucose-6-phosphatase (G6PC) | Target Info | Replacement | [2] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT03517085) Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa). U.S. National Institutes of Health. | |||
| REF 2 | Clinical pipeline report, company report or official report of Ultragenyx Pharmaceutical. | |||
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