- ByInvesting.com
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bluebird bio, Inc. is a biotechnology company. The Company is principally focused on researching, developing and commercializing gene therapies for genetic diseases. The Company’s gene therapy programs in genetic diseases include programs for B-thalassemia; LentiGlobin product candidate as a treatment for sickle cell disease (SCD), and elivaldogene autotemcel (eli-cel) as a treatment for cerebral adrenoleukodystrophy (CALD). It is using a lentiviral vector (LVV) platform, it custom designs each of its therapies to address the underlying cause of disease. The Company is developing lovotibeglogene autotemcel (lovo-cel) as a one-time treatment for patients with SCD, a genetic disease caused by a single mutation in the B-globin gene that leads to the production of abnormal sickle hemoglobin (HbS). The BLA for betibeglogene autotemcel (beti-cel) is for B-thalassemia in patients who receive regular red blood cell (RBC) transfusions.