Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DYK9H4
|
|||
| Drug Name |
AXO-AAV-GM2
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | Tay-sachs disease [ICD-11: 5C56.00] | Phase 1 | [1] | |
| Company |
Sio Gene Therapies
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Beta-hexosaminidase subunit alpha (HEXA) | Target Info | . | [1] |
| Beta-hexosaminidase subunit beta (HEXB) | Target Info | . | [1] | |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT04669535) A Two-Stage, Dose-Escalation and Safety & Efficacy Study of Bilateral Intraparenchymal Thalamic and Intracisternal/Intrathecal Administration of AXO-AAV-GM2 in Tay-Sachs or Sandhoff Disease. U.S.National Institutes of Health. | |||
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