bluebird bio, Inc.

bluebird bio, Inc. is a biotechnology company. The Company is focused on researching, developing, and commercializing potentially curative gene therapies for severe genetic diseases based on its lentiviral vector (LVV) gene addition platform. Its lead gene therapy programs for sickle cell disease, B-thalassemia, and cerebral adrenoleukodystrophy and is advancing research to apply new technologies to these and other diseases.

It has two gene therapies: ZYNTEGLO (betibeglogene autotemcel) and SKYSONA (elivaldogene autotemcel). ZYNTEGLO is the first gene therapy for people with B-thalassemia who require regular red blood cell transfusions. SKYSONA (elivaldogene autotemcel), also known as eli-cel, is used to slow the progression of eurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD). It is also developing (lovotibeglogene autotemcel), also known as lovo-cel, as a one-time treatment for patients with sickle cell disease (SCD). .