Statements (17)
| Predicate | Object |
|---|---|
| gptkbp:instanceOf |
gene therapy
|
| gptkbp:clinicalTrialPhase |
Phase 1/2
|
| gptkbp:developedBy |
gptkb:Axovant_Gene_Therapies
|
| https://www.w3.org/2000/01/rdf-schema#label |
AXO-AAV-GM2
|
| gptkbp:indication |
gptkb:Sandhoff_disease
gptkb:Tay-Sachs_disease gptkb:GM2_gangliosidosis |
| gptkbp:mechanismOfAction |
delivers functional HEXA and HEXB genes
|
| gptkbp:orphanDrugDesignation |
gptkb:FDA
gptkb:EMA |
| gptkbp:routeOfAdministration |
intrathecal
|
| gptkbp:status |
investigational
|
| gptkbp:targetGene |
gptkb:HEXA
HEXB |
| gptkbp:vectorFor |
gptkb:AAV9
|
| gptkbp:bfsParent |
gptkb:Sio_Gene_Therapies
|
| gptkbp:bfsLayer |
7
|