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

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

Business Wire

CAMBRIDGE, Mass. -- October 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.

For more information please contact clinicaltrials@bluebirdbio.com.

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 www.bluebirdbio.com.

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.

Contact:

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