Details of the Drug

General Information of Drug (ID: DM3BK2Y)

Drug Name

OTL-103

Indication

Disease Entry	ICD 11	Status	REF
Wiskott-Aldrich syndrome	3B62.0	Phase 3	[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
Wiskott-Aldrich syndrome protein (WAS)	TTE8T73	WASP_HUMAN	Replacement	[2]

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Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This Drug

References

1	ClinicalTrials.gov (NCT03837483) A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome. U.S. National Institutes of Health.
2	Clinical pipeline report, company report or official report of Orchard Therapeutics.