Statements (15)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
gene therapy
|
| gptkbp:administeredBy |
intravenous injection
|
| gptkbp:clinicalTrialPhase |
Phase 1/2
|
| gptkbp:deliveredBy |
AAV9 vector
|
| gptkbp:developedBy |
gptkb:Axovant_Gene_Therapies
|
| gptkbp:encodes |
GLB1 gene
|
| https://www.w3.org/2000/01/rdf-schema#label |
AXO-AAV-GM1
|
| gptkbp:intendedUse |
gptkb:GM1_gangliosidosis
|
| gptkbp:mechanismOfAction |
restores beta-galactosidase activity
|
| gptkbp:orphanDrugDesignation |
gptkb:FDA
gptkb:EMA |
| gptkbp:routeOfAdministration |
systemic
|
| gptkbp:targetAudience |
pediatric patients
|
| gptkbp:bfsParent |
gptkb:Sio_Gene_Therapies
|
| gptkbp:bfsLayer |
7
|