Details of the Drug

General Information of Drug (ID: DMZJIIB)

Drug Name

AT845

Indication

Disease Entry	ICD 11	Status	REF
Pompe disease	5C51.3	Phase 2	[1]
Glycogen storage disease type II	5C51.3	Phase 1/2	[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
Lysosomal alpha-glucosidase (GAA)	TTLPC70	LYAG_HUMAN	Not Available	[2]

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Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This Drug

References

1	ClinicalTrials.gov (NCT04174105) A Phase 1/2, Open-Label, Ascending-Dose Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT845, an AAV8-Delivered Gene Transfer Therapy in Patients With Late Onset Pompe Disease. U.S.National Institutes of Health.
2	Muscle-directed gene therapy corrects Pompe disease and uncovers species-specific GAA immunogenicity. EMBO Mol Med. 2022 Jan 11;14(1):e13968.