Details of the Drug

General Information of Drug (ID: DMK9DG5)

Drug Name

SPK-3006

Synonyms

RG6359

Indication

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

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

Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This Drug

References

1	ClinicalTrials.gov (NCT04093349) Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults With Late-onset Pompe Disease. U.S.National Institutes of Health.
2	Clinical pipeline report, company report or official report of Spark Therapeutics