Details of the Drug-Related molecule(s) Interaction Atlas

General Information of Drug (ID: DMISRRC)

Drug Name

AXO-AAV-GM2 Drug Info

Indication

Disease Entry	ICD 11	Status	REF
Tay-sachs disease	5C56.00	Phase 1	[1]

Cross-matching ID

Molecule-Related Drug Atlas

Molecule Type:

DTT

Drug Status:

Clinical Trial Drug(s)

Download the Complete Related Drug List

Drug(s) Targeting Beta-hexosaminidase subunit alpha (HEXA)

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
TSHA-101	DMFTPCT	GM2 gangliosidosis	5C56.00	Phase 1/2	[2]
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Drug(s) Targeting Beta-hexosaminidase subunit beta (HEXB)

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
TSHA-101	DMFTPCT	GM2 gangliosidosis	5C56.00	Phase 1/2	[2]
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Molecular Interaction Atlas of This Drug

Molecular Interaction Atlas

Download the Complete Interaction Atlas for Visualization in Cytoscape

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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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.
2	ClinicalTrials.gov (NCT04798235) Phase 1/2, Open-Label Clinical Study to Evaluate the Safety and Efficacy of Intrathecal TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis. U.S.National Institutes of Health.