– Study to enroll approximately fifteen adult, adolescent and pediatric patients –
“We are excited to start our second Phase 3 study of LentiGlobin in
patients with TDT,” said
“Ongoing studies of LentiGlobin in patients with TDT have indicated the
potential for a durable effect, with patients seeing consistent
production of HbAT87Q for more than three years after
LentiGlobin is also being investigated in Northstar-2 (HGB-207), a Phase 3 study in patients with TDT and non-β0/β0 genotypes, Northstar (HGB-204), a Phase 1/2 study in patients with TDT and all genotypes, HGB-205, a Phase 1 study in patients with TDT and severe sickle cell disease (SCD), and HGB-206, a Phase 1 study in patients with SCD.
About Northstar-3 (HGB-212)
Northstar-3 is a Phase 3, global, multi-center study designed to evaluate the safety and efficacy of LentiGlobin in patients with transfusion-dependent beta-thalassemia with a β0/β0 genotype. In this study, the manufacturing process by which the patient’s cells are transduced with the LentiGlobin viral vector has been modified, with the intent of increasing the percentage of cells successfully transduced and the average vector copy number per diploid genome.
The study’s primary endpoint is the proportion of patients who meet the
definition of "transfusion reduction" (TR). TR is defined as
demonstration of reduction of at least 60% in volume of red blood cell
Transfusion-dependent β-thalassemia (TDT) is a severe genetic disease characterized by reduced or absent hemoglobin levels that results in severe anemia and ineffective red blood cell production. Supportive care for people with TDT consists of a lifelong regimen of chronic blood transfusions to enable survival and suppress symptoms of the disease, and iron chelation therapy to manage iron overload that results from the transfusions. Despite the availability of supportive care, many people with TDT experience serious complications and organ damage due to underlying disease and iron overload.
Allogeneic hematopoietic stem cell transplant (HSCT) is currently the only available option to address the underlying genetic cause of TDT, though it carries significant risks. Complications of allogeneic HSCT include a risk of treatment-related mortality, graft failure, graft vs. host disease (GvHD) and opportunistic infections, particularly in patients who undergo HSCT from a donor who is not a matched sibling.
About bluebird bio, Inc.
With its lentiviral-based gene therapies, T cell immunotherapy expertise and gene editing capabilities, bluebird bio has built an integrated product platform with broad potential application to severe genetic diseases and cancer. bluebird bio’s gene therapy clinical programs include its Lenti-D™ product candidate, currently in a Phase 2/3 study, called the Starbeam Study, for the treatment of cerebral adrenoleukodystrophy, and its LentiGlobin® BB305 product candidate, currently in three clinical studies for the treatment of transfusion-dependent β-thalassemia, also known as β-thalassemia major, and severe sickle cell disease. bluebird bio’s oncology pipeline is built upon the company’s leadership in lentiviral gene delivery and T cell engineering, with a focus on developing novel T cell-based immunotherapies, including chimeric antigen receptor (CAR T) and T cell receptor (TCR) therapies. bluebird bio’s lead oncology programs, bb2121 and bb21217, are anti-BCMA CAR T programs partnered with Celgene. bb2121 and bb21217 are each currently being studied in Phase 1 trials for the treatment of relapsed/refractory multiple myeloma. bluebird bio also has discovery research programs utilizing megaTALs/homing endonuclease gene editing technologies with the potential for use across the company’s pipeline.
bluebird bio has operations in Cambridge, Massachusetts, Seattle,
LentiGlobin and Lenti-D are trademarks of bluebird bio, Inc.
This release contains “forward-looking statements” within the meaning
of the Private Securities Litigation Reform Act of 1995, including
statements regarding the Company’s research, development, manufacturing
and regulatory approval plans for its LentiGlobin product candidate to
treat transfusion-dependent ß-thalassemia, including statements whether
the manufacturing process changes for LentiGlobin will improve outcomes
of patients with transfusion-dependent ß-thalassemia. 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 risks that the preliminary positive efficacy and safety results
from our prior and ongoing clinical trials of LentiGlobin will not
continue or be repeated in our ongoing, planned or expanded clinical
trials of LentiGlobin, risks that the current or planned clinical trials
of LentiGlobin will be insufficient to support regulatory submissions or
marketing approval in the US and EU, the risk of a delay in the
enrollment of patients in our clinical studies, and the risk that any
one or more of our product candidates, including our bb2121 product
candidate, will not be successfully developed, approved or
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 most recent Form 10-Q, as well as
discussions of potential risks, uncertainties, and other important
factors in our subsequent filings with the
Source: bluebird bio, Inc.
Investors & Media
Elizabeth Pingpank, 617-914-8736