LentiGlobin BB305
LentiGlobin BB305 is an experimental treatment for beta thalassemia, a rare and potentially debilitating blood disorder. It is being developed by Bluebird Bio and was given “breakthrough therapy” designation by the Food and Drug Administration in February, 2015.[1] In early clinical trials several patients with beta thalassemia, who usually require frequent blood transfusions to treat their disease, were able to forgo blood transfusions for extended periods of time.[2][3][4]
Mechanism of action
Beta thalassemia is caused by mutations to or deletions of the HBB gene leading to reduced or absent synthesis of the beta chains of hemoglobin that result in variable outcomes ranging from severe anemia to clinically asymptomatic individuals.[5] LentiGlobin BB305 is a lentiviral vector which inserts a functioning version of the HBB gene into a patient's blood producing hematopoietic stem cells (HSC) ex vivo. The resulting engineered HSC cells are then reintroduced to the patient.[6][7]
See also
References
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- ↑ (8 December 2014) bluebird bio Announces Data Demonstrating First Four Patients with β-Thalassemia Major Treated with LentiGlobin™ are Transfusion-Free Yahoo News, Retrieved 17 May 2015
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External links
- Bluebird Bio product development page for LentiGlobin® BB305
