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]
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Drug Type

Gene therapy

Cross-matching ID

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]

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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.