Statements (17)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
gene therapy
|
| gptkbp:alternativeName |
gptkb:UX701
|
| gptkbp:clinicalTrialPhase |
Phase 3 (as of 2024)
|
| gptkbp:deliveredBy |
gptkb:AAV8_vector
|
| gptkbp:developedBy |
gptkb:Ultragenyx_Pharmaceutical
|
| gptkbp:encodes |
G6Pase (glucose-6-phosphatase)
|
| https://www.w3.org/2000/01/rdf-schema#label |
DTX402
|
| gptkbp:intendedUse |
treatment of GSDIa
|
| gptkbp:mechanismOfAction |
restores glucose-6-phosphatase activity
|
| gptkbp:orphanDrugDesignation |
gptkb:FDA
gptkb:EMA |
| gptkbp:routeOfAdministration |
intravenous
|
| gptkbp:sponsor |
gptkb:Ultragenyx_Pharmaceutical
|
| gptkbp:status |
investigational
|
| gptkbp:target |
gptkb:Glycogen_Storage_Disease_Type_Ia
|
| gptkbp:bfsParent |
gptkb:Yamaha_DTX_Electronic_Drums
|
| gptkbp:bfsLayer |
6
|