Genetix Biotherapeutics

bluebird bio, Inc. is a clinical-stage biotechnology company, which engages in researching, developing and commercializing potentially transformative gene therapies for severe genetic diseases. The company is headquartered in Somerville, Massachusetts and currently employs 248 full-time employees. The firm is focused on researching, developing and commercializing potentially curative gene therapies for severe genetic diseases based on its proprietary lentiviral vector (LVV) gene addition platform. The company markets three gene therapies: ZYNTEGLO (betibeglogene autotemcel, also known as beti-cel), SKYSONA (elivaldogene autotemcel, also known as eli-cel), and LYFGENIA (lovotibeglogene autotemcel, also known as lovo-cel). ZYNTEGLO is the first gene therapy for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell transfusions. SKYSONA is used to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD). LYFGENIA is used for the treatment of sickle cell disease in patients ages 12 and older who have a history of vaso-occlusive events (VOEs).