“We are treating a sickle cell patient for the first time with gene
therapy,” stated Marina Cavazzana, MD, PhD, Professor of Medicine at
“Sickle cell disease shortens life expectancy by decades even in
developed countries, so it is exciting to contemplate that LentiGlobin
may offer the curative potential of allogeneic stem cell transplantation
by using a patient’s own cells,” stated
About the HGB-205 Study
The phase 1/2 study is designed to
evaluate the safety and efficacy of LentiGlobin BB305 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, efficacy
will also be measured based on the frequency of vaso-occlusive crises or
acute chest syndrome events.
For more information on the HGB-205 Study, please visit www.clinicaltrials.gov using identifier NCT02151526.
About the HGB-206 Study
The phase 1 study is designed to
evaluate the safety and efficacy of LentiGlobin BB305 drug product in
the treatment of subjects with severe sickle cell disease. The study is
designed to enroll up to eight subjects. Subjects will be followed to
evaluate safety and efficacy will be measured based on changes in red
cell function tests, hemolysis markers and frequency of clinical events
secondary to sickle cell disease (e.g. vaso-occlusive crises or acute
chest syndrome events).
For more information on the HGB-206 Study, please visit www.clinicaltrials.gov using identifier NCT02140554.
About sickle cell disease
Sickle cell disease (SCD) is 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
crises 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, 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
bluebird bio also has an early-stage chimeric antigen receptor-modified
T cell (CAR-T) program for oncology in collaboration with
bluebird bio has operations in
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 potential efficacy and safety of the Company’s LentiGlobin BB305
product candidate, the Company’s plans with respect to LentiGlobin and
its other product candidates and anticipated clinical and business
milestones and announcements for 2015. In addition it should be noted
that the data for LentiGlobin announced from the HGB-205 study in
patients with beta-thalassemia major are preliminary in nature and the
HGB-205 trial is not completed. These data may not continue for these
subjects or be repeated or observed in ongoing or future studies
involving our LentiGlobin product candidate, including the HGB-205
Study, the Northstar Study or the HGB-206 study in severe SCD. Any
forward-looking statements 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
Availability of other information about bluebird bio
Investors
and others should note that we communicate with our investors and the
public using our company website (www.bluebirdbio.com),
our investor relations website (http://www.bluebirdbio.com/investor-splash.html),
including but not limited to investor presentations and FAQs,
Source: bluebird bio, Inc.
Investor Relations:
bluebird bio, Inc
Richard E. T.
Smith, Ph.D., 339-499-9382
or
Media Contact:
Pure
Communications, Inc.
Dan Budwick, 973-271-6085