First two patients in the HGB-205 Study achieved transfusion independence within two weeks of an autologous transplant with bluebird’s lentiviral gene therapy
“We are gratified that the improvements we introduced into the BB305
lentiviral vector design and manufacturing process appear to have
translated into clinical results that we believe support the potential
for our LentiGlobin BB305 gene therapy to transform the lives of
patients with beta-thalassemia major,” stated
The principal investigator of the HGB-205 Study, Marina Cavazzana, M.D
delivered an oral presentation at the
Summary of the clinical data presented at EHA were:
LG001 Clinical Study
- Clinical update provided on two subjects treated in the prior LG001 Study (subjects 3 and 4) using the prior lentiviral HPV569 product candidate
- Subject 3 remains blood transfusion independent 72 months after being transplanted with the lentiviral HPV569 product candidate
- Subjects 3 and 4 are producing 2.7 g/dL and 0.4 g/dL of therapeutic betaA-T87Q-globin post-transplant, respectively
- No drug product related adverse events were reported in the LG001 Study.
HGB-205 Clinical Study
- Clinical data were presented on two subjects (subjects 1 and 2), both with beta-thalassemia major and the Beta E/Beta 0 genotype who were treated using the new lentiviral vector BB305
- At 4.5 months following autologous transplant subject 1 had a total hemoglobin of 10.1 g/dL of which 6.6 g/dL was therapeutic betaAT87Q-globin, and at 2 months post-transplant subject 2 had a total hemoglobin of 11.6 g/dL of which 4.2 g/dL was betaAT87Q-globin
- Subjects 1 and 2 received their last blood transfusion on day 10 and 12, respectively, post-transplant and both subjects remain blood transfusion independent
- Vector copy number in the drug product for subjects 1 and 2 were 1.5 and 2.1, respectively; multiple times higher than the drug product vector copy numbers reported in the prior LG001 Study (VCN 0.6 and 0.3 for Subjects 3 and 4, respectively)
- No drug product related adverse events were reported, and the integration site analysis performed on subject 1 at the 3-month time point showed polyclonal reconstitution.
We anticipate reporting additional data from the HGB-205 Study and from our ongoing Northstar Study in late 2014.
Conference Call and Webcast
bluebird bio will host a conference call at
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 and
splenomegaly. It is estimated that about 288,000 patients with
beta-thalassemia are alive, of which an estimated 15,000 live in
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 blood 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 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
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 product candidate, the Company’s plans with respect to
LentiGlobin and its other product candidates and anticipated clinical
and business milestones and announcements for 2014. In addition it
should be noted that the data for LentiGlobin announced from the HGB-205
study at the
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.
bluebird bio, Inc.
Richard E. T. Smith, Ph.D., 339-499-9382
Pure Communications, Inc.
Dan Budwick, 973-271-6085