Details of the Drug

General Information of Drug (ID: DMISRRC)

Drug Name
AXO-AAV-GM2
Indication
Disease Entry ICD 11 Status REF
Tay-sachs disease 5C56.00 Phase 1 [1]
Drug Type
Gene therapy
Cross-matching ID
TTD ID
DYK9H4

Molecular Interaction Atlas of This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Beta-hexosaminidase subunit alpha (HEXA) TTJI5JW HEXA_HUMAN Not Available [1]
Beta-hexosaminidase subunit beta (HEXB) TTKIBKM HEXB_HUMAN Not Available [1]
Molecular Interaction Atlas (MIA) Jump to Detail Molecular Interaction Atlas of This Drug

References

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.