Abstract Title: Outcomes of gene therapy for beta-thalassemia major via transplantation of autologous hematopoietic stem cells transduced ex vivo with a lentiviral beta globin vector.
Lead Author: Marina Cavazzana, M.D.
Session Title: Simultaneous Sessions & EHA Advocacy Sessions
Presentation Date/Time:
Summary of Abstract Data and Clinical Data to be Presented at EHA:
- Clinical data will be presented on two subjects with beta-thalassemia major (#1201 and #1202) transplanted with new lentiviral vector BB305 in the HGB-205 Study
- Vector copy number in the drug product for subjects 1201 and 1202 are 1.5 and 2.1 respectively; higher than the drug product vector copy numbers reported in the prior LG001 Study (0.3-0.6), which used the older lentiviral vector HPV569
- A detailed update on the clinical data and transfusion status of subjects 1201 and 1202 will be provided as part of the EHA presentation
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 advancement of, and anticipated milestones
related to the Company’s product candidates and clinical studies, and
anticipated milestones for 2014. In addition it should be noted that the
data expected to be announced later in 2014 are preliminary and interim
in nature; the HGB-205 trial is not completed. These data may not
be not be repeated or observed in ongoing or future studies involving
our LentiGlobin product candidate, including the HGB-205 trial or
Northstar Study. 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