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bluebird bio Announces First Patient Transplanted in Phase 1/2 Northstar Study for the Treatment of Beta-Thalassemia

CAMBRIDGE, MA, March 27, 2014 – bluebird bio, Inc. (Nasdaq: BLUE) a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and orphan diseases, today announced that the first subject with beta-thalassemia major has been enrolled in its phase 1/2 Northstar Study (HGB-204) in the United States and has undergone infusion with bluebird bio’s LentiGlobin drug product in an autologous hematopoietic stem cell transplantation.

“We are very excited to have reached this critical milestone today with the infusion of modified blood stem cells in our patient with thalassemia. We hope this innovation will be a major advancement in thalassemia treatment,” Alexis Thompson, MD, Professor of Pediatrics at Northwestern University Feinberg School of Medicine and Director of the Comprehensive Thalassemia Program at Ann & Robert H. Lurie Children’s Hospital of Chicago. “Patients with beta thalassemia major suffer from complications of their disease as well as its treatment that can take a significant physical and emotional toll. The potential of gene therapy to reestablish more effective hemoglobin production and, therefore, free patients from dependence on blood transfusions and iron chelation therapies is compelling.”

“We welcome the start of the Northstar Study, the second beta thalassemia major trial initiated by bluebird bio with our LentiGlobin gene therapy product candidate,” stated David Davidson, MD, bluebird bio’s Chief Medical Officer. “We are grateful for the tremendous efforts of our clinical investigators and the patients who have volunteered to undertake this pioneering work. Restoring the ability of a patient’s own blood stem cells to produce red blood cells with functional beta-globin offers the potential for a transformative, one-time treatment for this devastating disease.”

About the Northstar (HGB-204) Study
The phase 1/2 study is designed to evaluate the feasibility, safety and efficacy of LentiGlobin (BB305) drug product in the treatment of subjects with beta-thalassemia major. The study is designed to enroll up to fifteen subjects. Subjects will be evaluated for safety and efficacy post-transplant.

For more information on the Northstar Study, please visit or using identifier NCT01745120.

About beta-thalassemia major
Beta-thalassemia major is a rare hereditary blood disorder caused by a genetic abnormality of the beta globin gene resulting in defective red blood cells. Symptoms of beta-thalassemia include severe anemia, splenomegaly and iron overload in major organs. It is estimated that about 288,000 patients with beta-thalassemia major are alive, of which an estimated 15,000 live in the United States and Europe.

About bluebird bio, Inc.
bluebird bio is a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and orphan diseases. bluebird bio has two clinical-stage programs in development. The most advanced product candidate, Lenti-D, is in a recently-initiated phase 2/3 study, the Starbeam Study, for the treatment of childhood cerebral adrenoleukodystrophy (CCALD), a rare, hereditary neurological disorder affecting young boys. The next most advanced product candidate, LentiGlobin, is currently in two phase 1/2 studies, one in the US (the Northstar Study) and one in France (HGB-205), for the treatment of beta-thalassemia major. The phase 1/2 HGB-205 study also allows enrollment of patient(s) with sickle cell disease and bluebird bio is planning to enroll its first sickle cell patient in a separate U.S. trial in 2014.

bluebird bio also has an early-stage chimeric antigen receptor-modified T cell (CAR-T) program for oncology in partnership with Celgene Corporation.

bluebird bio has operations in Cambridge, Massachusetts and Paris, France. For more information, please visit

Forward-Looking Statements
This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the feasibility, safety and efficacy of the Company’s product candidates, in particular its LentiGlobin drug product candidate, the advancement of the Company’s Phase 1/2 Northstar and HGB-205 clinical studies, and the filing of an IND with FDA to enroll patients with sickle cell disease in a separate study. Any forward-looking statements in this press release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk of cessation or delay of any of the ongoing or planned clinical studies and/or our development of our product candidates, the risk of a delay in the enrollment of patients in the Company’s clinical studies, the risk that the results of previously conducted studies involving similar product candidates will not be repeated or observed in ongoing or future studies involving current product candidates, the risk that our collaboration with Celgene will not continue or will not be successful, and the risk that any one or more of our product candidates will not be successfully developed and commercialized. For a discussion of other risks and uncertainties, and other important factors, any of which could cause our actual results to differ from those contained in the forward-looking statements, see the section entitled "Risk Factors" in our most recent annual report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.

Investor Relations:
Richard E. T. Smith, Ph.D.
bluebird bio, Inc
(339) 499-9382

Media Contact:
Dan Budwick
Pure Communications, Inc.
(973) 271-6085

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Jenn Snyder

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Catherine Falcetti

Senior Director, Corporate Communications

(339) 499-9436


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