Details of the Drug

General Information of Drug (ID: DM90XQC)

Drug Name

CTX001

Indication

Disease Entry	ICD 11	Status	REF
Beta thalassemia	3A50.2	Phase 1/2	[1]
Sickle-cell disorder	3A51	Phase 1/2	[2]
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Drug Type

Gene therapy

Cross-matching ID

Repurposed Drugs (RPD)

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
B-cell lymphoma/leukemia 11A (BCL11A)	TTR61MW	BC11A_HUMAN	Not Available	[3]

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Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This Drug

References

1	ClinicalTrials.gov (NCT03655678) A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent beta-Thalassemia. U.S. National Institutes of Health.
2	ClinicalTrials.gov (NCT03745287) A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease. U.S. National Institutes of Health.
3	Off-the-shelf' allogeneic CAR T cells: development and challenges. Nat Rev Drug Discov. 2020 Mar;19(3):185-199.