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Profile: bluebird bio Inc (BLUE.OQ)

BLUE.OQ on NASDAQ Stock Exchange Global Select Market

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bluebird bio, Inc., incorporated on April 16, 1992, is a clinical-stage biotechnology company. The Company is focused on developing gene therapies for severe diseases and cancer. With its lentiviral-based gene therapy and gene editing capabilities, it has built an integrated product platform with various applications in these areas. The Company's clinical programs in severe genetic diseases include its LentiGlobin product candidate to treat transfusion-dependent b-thalassemia (TDT) and to treat severe sickle cell disease (SCD) and its Lenti-D product candidate to treat cerebral adrenoleukodystrophy (CALD). Its programs in oncology are built upon its 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. bb2121, its lead product candidate in oncology, is a CAR T cell product candidate for the treatment of multiple myeloma. It also has discovery research programs utilizing megaTALs/homing endonuclease gene editing technologies for use across its pipeline.

As of December 31, 2016, it had conducting four clinical studies of its LentiGlobin product candidate: a Phase I/II study for the treatment of subjects with TDT, called the Northstar Study (HGB-204); a multi-site, international, Phase III study for the treatment of subjects with TDT and non-b/b genotypes, called the Northstar-2 Study (HGB-207); a single-center Phase I/II study in France for the treatment of subjects with TDT or with severe SCD (HGB-205), and a multi-site Phase I study in the United States for the treatment of subjects with severe SCD (HGB-206). 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 both b-thalassemia and SCD. Its LentiGlobin product candidate was granted Fast-Track designation by the FDA for the treatment of b-thalassemia major and for the treatment of certain patients with severe SCD. The FDA has granted Breakthrough Therapy designation to its LentiGlobin product candidate for the treatment of transfusion-dependent patients with b-thalassemia major.

The Company is conducting a multi-site, international, Phase II/III clinical study of its Lenti-D product candidate, called the Starbeam Study (ALD-102), for the treatment of subjects with CALD, a rare, hereditary neurological disorder that is often fatal. Its Lenti-D product candidate has been granted Orphan Drug status by the FDA and the EMA for the treatment of adrenoleukodystrophy. The Company is conducting a multi-site Phase I clinical study in the United States of its bb2121 product candidate for the treatment of subjects with relapsed/refractory multiple myeloma (CRB-401). bb2121 is the lead product candidate arising from its multi-year collaboration with Celgene Corporation (Celgene, for the discovery, development and commercialization of CAR T cell therapies targeting B-cell maturation antigen, or BCMA. It has exclusively licensed to Celgene the right to develop and commercialize its bb2121 product candidate, and it may exercise its option to co-develop and co-promote this product candidate in the United States. The FDA has granted Orphan Drug status to bb2121 for the treatment of patients with relapsed/refractory multiple myeloma.

The Company's gene therapy platform is based on viral vectors that utilize a non-replicating version of the Human Immunodeficiency Virus Type 1 (HIV-1). Its lentiviral vectors are used to introduce a functional copy of a gene to the patient's own isolated hematopoietic stem cells (HSCs) in the case of its LentiGlobin and Lenti-D product candidates, or the patient's own isolated white blood cells, which include T cells, in the case of its bb2121 product candidate. Additionally, it has developed a cell-based vector manufacturing process that is both reproducible and scalable. The Company also has discovery research programs utilizing its cell signaling technology and gene editing technology platform across its pipeline.

Company Address

bluebird bio Inc

60 Binney St
CAMBRIDGE   MA   02142-1512
P: +1339.4999300
F: +1302.6365454

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