Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D95GKC
|
|||
| Drug Name |
BCL11a shRNA
|
|||
| Drug Type |
Short hairpin RNA
|
|||
| Indication | Sickle-cell disorder [ICD-11: 3A51; ICD-10: D57, D57.8; ICD-9: 282.5, 282.6] | Phase 1 | [1] | |
| Company |
Boston Children's Hospital; bluebird bio
|
|||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | BCL11A messenger RNA (BCL11A mRNA) | Target Info | Inhibitor | [2] |
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT03282656) Gene Transfer for Sickle Cell Disease. U.S. National Institutes of Health. | |||
| REF 2 | Clinical pipeline report, company report or official report of bluebird bio. | |||
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