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bluebird bio Announces First Patient Transplanted in Phase 2/3 Starbeam ALD-102 Study for the Treatment of CCALD

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Oct. 28, 2013-- 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 in its phase 2/3 childhood cerebral adrenoleukodystrophy (CCALD) study, Starbeam (ALD-102) has undergone infusion with bluebird bio’s Lenti-D drug product in an autologous hematopoietic stem cell transplantation.

“Treating our first subject in this study reflects the recent advances in the field of gene therapy and is the culmination of years of collaborative effort between the team at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and our colleagues at Massachusetts General Hospital, INSERM in Paris, and bluebird bio,” stated David A. Williams, MD, Chief of hematology/oncology at Boston Children’s Hospital and Associate Chairman of pediatric oncology at Dana-Farber Cancer Institute. “Boys with CCALD face significant risks of mortality and morbidity with allogeneic stem cell transplantation, the current standard of care treatment, if an optimally matched donor cannot be identified. bluebird’s autologous Lenti-D drug product has the potential to circumvent this challenge and address an important unmet medical need for patients with this devastating disease.”

“Successfully initiating treatment in the Starbeam study represents an important step towards improving outcomes for patients with CCALD and is a major milestone for bluebird and its lentiviral gene therapy platform,” stated Dave Davidson, MD, bluebird bio’s Chief Medical Officer.

About the Starbeam study (ALD-102)

The phase 2/3 study is designed to evaluate the safety and efficacy of Lenti-D drug product in the treatment of subjects with childhood cerebral adrenoleukodystrophy, or CCALD, a rare, hereditary neurological disorder affecting young boys that is often fatal. The trial study is planned to enroll up to 15 boys who will be followed for 24 months following a transplant with bluebird bio’s lentiviral transduced stem cells, Lenti-D. During this 24 month period, patients will be assessed for the onset of major functional disabilities, and other key assessments of disease progression.

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About Childhood Cerebral Adrenoleukodystrophy (CCALD)

Childhood Cerebral Adrenoleukodystrophy is an X-linked disease caused by the aberrant expression of the ABCD1 gene in boys leading to the inability of patients to metabolize very long chain fatty acids in cells of the brain. The disease is characterized by progressive destruction of myelin, leading to severe loss of neurological function and eventual death. The worldwide incidence of adrenoleukodystrophy (ALD) is approximately one in 20,000 newborns. CCALD accounts for about 30-40% of patients diagnosed with ALD.

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 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 a phase 1/2 study in France for the treatment of beta-thalassemia major and severe sickle cell disease. A second phase 1/2 study with LentiGlobin in the United States has been initiated for the treatment of beta-thalassemia major.

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 advancement of the Company’s clinical studies, the timing of data announcement and the potential safety and clinical benefits of the Company’s product candidates. 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 quarterly report on Form 10-Q, 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.

Source: bluebird bio, Inc.

Investor Relations:
bluebird bio, Inc.
Richard E. T. Smith, Ph.D., 617-588-3321
Media Contact:
Pure Communications, Inc.
Dan Budwick, 973-271-6085

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