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

General Information of Drug (ID: DM3BK2Y)

Drug Name

OTL-103 Drug Info

Indication

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

Cross-matching ID

Molecule-Related Drug Atlas

Molecule Type:

DTT

Drug Status:

Clinical Trial Drug(s)

Download the Complete Related Drug List

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

Drug Name	Drug ID	Indication	ICD 11	Highest Status	REF
Hematopoietic stem cell gene therapy	DMQWI7V	Wiskott-Aldrich syndrome	3B62.0	Phase 1/2	[3]
WASP gene therapy	DMN62MP	Wiskott-Aldrich syndrome	3B62.0	Phase 1/2	[4]
------------------------------------------------------------------------------------

Molecular Interaction Atlas of This Drug

Molecular Interaction Atlas

Download the Complete Interaction Atlas for Visualization in Cytoscape

Drug Therapeutic Target (DTT)

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

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

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.
3	Development of hematopoietic stem cell gene therapy for Wiskott-Aldrich syndrome. Curr Opin Mol Ther. 2006 Oct;8(5):390-5.
4	ClinicalTrials.gov (NCT01347242) Gene Therapy for Wiskott-Aldrich Syndrome (WAS). U.S. National Institutes of Health.