bluebird bio Announces First Patient Transplanted in Phase 1/2 HGB-205 Study for the Treatment of Beta-Thalassemia and Sickle

  bluebird bio Announces First Patient Transplanted in Phase 1/2 HGB-205 Study
  for the Treatment of Beta-Thalassemia and Sickle Cell Anemia

Business Wire

CAMBRIDGE, Mass. -- December 2, 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 with beta-thalassemia
major has been enrolled in its phase 1/2 HGB-205 study in France and has
undergone infusion with bluebird bio’s LentiGlobin drug product in an
autologous hematopoietic stem cell transplantation.

“We believe gene therapy represents a potentially new and exciting treatment
option for patients with severe forms of beta-thalassemia and sickle cell
disease,” stated Marina Cavazzana, MD, Professor of Medicine at Paris
Descartes University and Research Director at the Centre for Clinical Research
in Biotherapy, Necker Hospital, Paris France. “The beta-hemoglobinopathies are
the most prevalent inherited disorders worldwide, and they result in
substantial morbidity and mortality. The HGB-205 study builds on early and
encouraging clinical data from the LG001 study based on the pioneering work of
Professor Philippe Leboulch and colleagues at the Universities of Paris and
Harvard, Inserm and the French Atomic Energy and Alternative Energies
Commission (CEA) research center in Paris.”

"It is very gratifying for our research, manufacturing and development teams
to see their efforts to improve the potency and scalability of our product
platform finally reach the clinic for patients with this life threatening
disease. This milestone brings us closer towards realizing our vision of
making hope a reality for patients with limited therapeutic options,” stated
Dave Davidson, MD, bluebird bio’s Chief Medical Officer."

About the HGB-205 Study

The phase 1/2 study is designed to evaluate the safety and efficacy of
LentiGlobin drug product in the treatment of subjects with beta-thalassemia
major and severe sickle cell disease. The study is designed to enroll up to
seven subjects. Subjects will be followed to evaluate safety and transfusion
requirements post-transplant. In sickle cell disease patients only, efficacy
will also be measured based on the number of vaso-occlusive crises or acute
chest syndrome events.

About beta-thalassemia major and sickle cell disease

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.

Sickle cell disease (SCD) is also a hereditary blood disorder resulting from a
mutation in the beta globin gene that causes polymerization of hemoglobin
proteins and abnormal red blood cell function. The symptoms of SCD include
anemia, vaso-occlusive crisis and strokes. The global incidence of SCD is
estimated to be 250,000 to 300,000 births annually, and the global prevalence
of the disease is estimated to be about 20 to 25 million.

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 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 most recent 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:
Pure Communications, Inc.
Dan Budwick, 973-271-6085
 
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