General Information of Drug (ID: DM90XQC)

Drug Name
CTX001 Drug Info
Indication
Disease Entry ICD 11 Status REF
Beta thalassemia 3A50.2 Phase 1/2 [1]
Sickle-cell disorder 3A51 Phase 1/2 [2]
Cross-matching ID
TTD Drug ID
DM90XQC

Molecular Interaction Atlas of This Drug

Molecular Interaction Atlas

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

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.