Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DVU2Y5
|
|||
| Drug Name |
CTX001
|
|||
| Drug Type |
Gene therapy
|
|||
| Indication | Beta thalassemia [ICD-11: 3A50.2; ICD-10: D56.1, D56.3] | Phase 1/2 | [1] | |
| Sickle-cell disorder [ICD-11: 3A51; ICD-10: D57, D57.8; ICD-9: 282.5, 282.6] | Phase 1/2 | [2] | ||
| Company |
CRISPR Therapeutics; Vertex Pharmaceuticals
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | B-cell lymphoma/leukemia 11A (BCL11A) | Target Info | . | [3] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT03655678) A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent beta-Thalassemia. U.S. National Institutes of Health. | |||
| REF 2 | ClinicalTrials.gov (NCT03745287) A Safety and Efficacy Study Evaluating CTX001 in Subjects With Severe Sickle Cell Disease. U.S. National Institutes of Health. | |||
| REF 3 | Off-the-shelf' allogeneic CAR T cells: development and challenges. Nat Rev Drug Discov. 2020 Mar;19(3):185-199. | |||
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