Drug Profile
BCL11a shRNA(miR)
Alternative Names: shRNA targeting BCL11a; shRNAmiRLatest Information Update: 28 Aug 2023
Price :
$50
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At a glance
- Originator bluebird bio; Boston Childrens Hospital
- Class Antianaemics; Gene therapies
- Mechanism of Action Haemoglobin stimulants; RNA interference
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity No
Highest Development Phases
- Phase II Sickle cell anaemia
Most Recent Events
- 28 Aug 2023 No recent reports of development identified for phase-I development in Sickle-cell-anaemia(In adolescents, In children, In adults) in USA (IV, Infusion)
- 10 Dec 2022 Efficacy and adverse event data from a phase I trial in Sickle Cell anaemia presented at the 64rd American Society of Hematology Annual Meeting and Exposition (ASH-Hem-2022)
- 12 Jul 2022 Phase-II clinical trials in Sickle cell anaemia (In adults, In adolescents) in USA (IV) (NCT05353647)