Bluebird bio (BLUE) is a biotech company focused on developing and commercializing gene and cell therapies to treat genetic diseases. Lead asset LentiGlobin (Zynteglo) is approved in US and Europe for the treatment of transfusion-dependent thalassemia. Bluebird's LentiGlobin (lovo-cel) is also in pre-registrational stage for sickle cell disease.
Bluebird is also right behind Crispr and Vertex in pursuit of a sickle cell approval.
June 21st 2023: Lovo-cel is a potentially transformative one-time gene therapy for individuals living with sickle cell disease (SCD) ages 12 and older who have a history of vaso-occlusive events. It is specifically designed to treat the underlying cause of SCD through the addition of a functional gene that enables production of anti-sickling adult hemoglobin and is the most deeply studied gene therapy in development for this disease. The agency has set a Prescription Drug User Fee Act goal date of December 20, 2023.