Details of the Drug

General Information of Drug (ID: DMIX5HF)

Drug Name

AAV2/8-LSPhGAA

Synonyms

ACTUS 101

Indication

Disease Entry	ICD 11	Status	REF
Pompe disease	5C51.3	Phase 1	[1]
------------------------------------------------------------------------------------

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
Alpha-glucosidase (GLA)	TTHCF4J	LYAG_HUMAN ; GANAB_HUMAN ; GANC_HUMAN	Not Available	[1]

------------------------------------------------------------------------------------

Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This Drug

References

1	ClinicalTrials.gov (NCT03533673) AAV2/8-LSPhGAA in Late-Onset Pompe Disease. U.S. National Institutes of Health.