Details of the Drug

General Information of Drug (ID: DMFBMI7)

• Drug Name: AXO-AAV-GM1

Indication

Disease Entry	ICD 11	Status	REF
GM1 gangliosidosis	5C56.00	Phase 1/2	[1]

• Drug Type: Gene therapy
• Cross-matching ID: TTD ID: DN4O0S

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Beta-galactosidase (GLB1)	TTNGJPH	BGAL_HUMAN	Not Available	[1]

References

• [1] Clinical pipeline report, company report or official report of Axovant Gene Therapies