Details of the Drug-Related molecule(s) Interaction Atlas

General Information of Drug (ID: DMQWI7V)

Drug Name

Hematopoietic stem cell gene therapy Drug Info

Synonyms

Hematopoietic stem cell gene therapy (Wiskott-Aldrich syndrome)

Indication

Disease Entry	ICD 11	Status	REF
Wiskott-Aldrich syndrome	3B62.0	Phase 1/2	[1]

Cross-matching ID

Molecule-Related Drug Atlas

Molecule Type:

DTT

Drug Status:

Clinical Trial Drug(s)

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Drug(s) Targeting Wiskott-Aldrich syndrome protein (WAS)

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
OTL-103	DM3BK2Y	Wiskott-Aldrich syndrome	3B62.0	Phase 3	[3]
WASP gene therapy	DMN62MP	Wiskott-Aldrich syndrome	3B62.0	Phase 1/2	[4]
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Molecular Interaction Atlas of This Drug

Molecular Interaction Atlas

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Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Wiskott-Aldrich syndrome protein (WAS)	TTE8T73	WASP_HUMAN	Modulator	[2]

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References

1	ClinicalTrials.gov (NCT02559830) Autologous Hematopoietic Stem Cell Gene Therapy for Metachromatic Leukodystrophy and Adrenoleukodystrophy.
2	Development of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome. Curr Opin Mol Ther. 2006 Oct;8(5):390-5.
3	Clinical pipeline report, company report or official report of Orchard Therapeutics.
4	ClinicalTrials.gov (NCT01347242) Gene Therapy for Wiskott-Aldrich Syndrome (WAS). U.S. National Institutes of Health.