By Business Wire, October 28, 2013, 07:39:00 AM EDT
CAMBRIDGE, Mass.--(BUSINESS WIRE)--
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
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 email@example.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
bluebird bio has operations in Cambridge, Massachusetts and Paris,
France. For more information, please visit www.bluebirdbio.com.
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.