Profile: bluebird bio Inc (BLUE.OQ)
bluebird bio, Inc., incorporated on April 16, 1992, is a biotechnology company. The Company is focused on developing transformative gene therapies for severe genetic and rare diseases and in the field of T cell-based immunotherapy. With its lentiviral-based gene therapies, T cell immunotherapy expertise and gene editing capabilities, it offers an integrated product platform with application in severe genetic and rare diseases, and in oncology. Its gene therapy clinical programs include LentiGlobin product candidate to treat transfusion-dependent B-thalassemia (TDT) and severe sickle cell disease (SCD), and Lenti-D product candidate to treat cerebral adrenoleukodystrophy (CALD), a rare hereditary neurological disorder. Its oncology programs are built using lentiviral gene delivery and T cell engineering, with a focus on developing T cell-based immunotherapies, including chimeric antigen receptor (CAR) and T cell receptor (TCR) T cell therapies. Its oncology program, bb2121, is a CAR T cell product candidate targeting B-cell maturation antigen, or BCMA, in multiple myeloma. It also offers discovery research programs utilizing megaTALs/homing endonuclease gene editing technologies for use across its pipeline.
The Company is conducting approximately three clinical studies of its LentiGlobin product candidate in various rare, hereditary blood disorders that often lead to severe anemia and shortened lifespans: a global Phase I/II study, called the Northstar Study, for the treatment of TDT; a single-center Phase I/II study in France (HGB-205) for the treatment of TDT and severe SCD, and a Phase I study in the United States (HGB-206) for the treatment of severe SCD. Its LentiGlobin product candidate has been granted Orphan Drug status by the United States Food and Drug Administration (FDA), and the European Medicines Agency (EMA), for the treatment of both B-thalassemia and SCD. It is conducting a Phase II/III clinical study, called the Starbeam Study, of its Lenti-D product candidate, to evaluate its safety and efficacy in pediatric and adolescent subjects with CALD. It has initiated a Phase I clinical study in the United States (CRB-401) of its bb2121 product candidate for the treatment of relapsed/refractory multiple myeloma.
The Company's gene therapy platform is based on viral vectors that utilize a modified, non-replicating version of the Human Immunodeficiency Virus Type 1, or HIV-1, that has been stripped of all of the components required for it to self-replicate and infect additional cells. HIV-1 is part of the lentivirus family of viruses, and it refers to its vectors as lentiviral vectors. Its lentiviral vectors are used to introduce a functional copy of a gene to the patient's own isolated blood stem cells, called hematopoietic stem cells, or HSCs, which reside in a patient's bone marrow and are capable of differentiating into a range of cell types. Additionally, it has developed a cell-based vector manufacturing process that is both reproducible and scalable.
bluebird bio Inc
150 2nd St
CAMBRIDGE MA 02141-2115