“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
"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
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
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
bluebird bio also has an early-stage chimeric antigen receptor-modified
T cell (CAR-T) program for oncology in partnership with
bluebird bio has operations in
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
Source: bluebird bio, Inc.
bluebird bio, Inc.
Richard E. T. Smith, Ph.D., 617-588-3321
Pure Communications, Inc.
Dan Budwick, 973-271-6085