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bluebird bio Provides Updates on HSC Gene Therapy Programsbluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic diseases and T cell-based immunotherapies for cancer, provided updates across its hematopoietic stem cell (HSC) gene therapy programs, including:
"Our focus is learning, adjusting and implementing to innovate on behalf of the patients we aim to serve. This year we have made tremendous progress against this objective," said Nick Leschly, chief bluebird. "In 2016, we focused on further enhancing our LentiGlobin programs in TDT and SCD by implementing high potential manufacturing and protocol amendments while advancing our lead bb2121 oncology program and commercialization capabilities. We are encouraged by the data presented at ASH and how that has informed our plans for 2017."
TDT Program Updates Interim data from the Northstar study were highlighted today in an oral presentation by Alexis Thompson, M.D., M.P.H., head of the hematology section of the Division of Hematology Oncology Transplantation and Director of the Comprehensive Thalassemia Program at the Ann and Robert H. Lurie Children's Hospital of Chicago, where she also serves as the A. Watson and Sarah Armour Endowed Chair for Childhood Cancer and Blood Disorders. LentiGlobin Gene Therapy for Transfusion-Dependent ß-Thalassemia: Update from the Northstar (HGB-204) Phase 1/2 Clinical Study (Abstract #1175) The Northstar Study is an ongoing, open-label, single-dose, international, multicenter Phase 1/2 study designed to evaluate the safety and efficacy of LentiGlobin drug product for the treatment of subjects with TDT. Results as of September 16, 2016 include:
"The maturing interim data from the Northstar study support the potential for LentiGlobin to provide a transformative treatment option for patients with TDT by reducing or eliminating the burdensome cycle of chronic blood transfusions and iron chelation," said David Davidson, chief medical officer, bluebird bio. "In addition, we are pleased by the robust vector copy number and high proportion of LVV+ CD34+ stem cells in the drug product manufactured using transduction enhancers for the first patient to be treated in the Phase 3 Northstar-2 study. If the clinical correlation between drug product VCN and hemoglobin production observed in the Northstar study continues with drug product manufactured utilizing process 2, we are hopeful that LentiGlobin drug product wth higher VCNs will consistently yield clinically meaningful outcomes for patients with TDT across all genotypes."
Severe Sickle Cell Disease Program Updates Interim data from the HGB-206 study were highlighted today in an oral presentation by Julie Kanter, M.D., Medical University of South Carolina, Charleston, SC. Interim Results from a Phase 1/2 Clinical Study of LentiGlobin™ Gene Therapy for Severe Sickle Cell Disease (Abstract #1176) HGB-206 is an ongoing, open-label study designed to evaluate the safety and efficacy of LentiGlobin drug product in the treatment of subjects with severe SCD. Results, as of November 9, 2016, include:
Cerebral Adrenoleukodystrophy Program Updates Webcast Information bluebird bio will host a live webcast at 8:30 p.m. PT (11:30 p.m. ET) today, December 5, 2016. The live webcast can be accessed under "Calendar of Events" in the Investors and Media section of the company's website at www.bluebirdbio.com. About TDT Transfusion-dependent ß-thalassemia (TDT), also called ß-thalassemia major or Cooley's anemia, is an inherited blood disease that can cause severe anemia and can be fatal within the first few years of life if not treated. TDT is one of the most common genetic diseases in the world, and approximately 60,000 children are born every year with a serious form of the disease. Despite advances in the supportive conventional management of the disease, which consists of frequent and lifelong blood transfusions and iron chelation therapy, there is still a significant unmet medical need, including the risk for significant morbidity and early mortality. Currently, the only advanced treatment option for transfusion-dependent ß-thalassemia is allogeneic hematopoietic stem cell transplant (HSCT). Complications of allogeneic HSCT include a significant risk of treatment-related mortality, graft failure, graft vs. host disease (GvHD) and opportunistic infections, particularly in patients who undergo non-sibling-matched allogeneic HSCT. About SCD Sickle cell disease (SCD) is an inherited disease caused by a mutation in the beta-globin gene that results in sickle-shaped red blood cells. The disease is characterized by anemia, vaso-occlusive crisis, infections, stroke, overall poor quality of life and sometimes, early death. Where adequate medical care is available, common treatments for patients with SCD largely revolve around management and prevention of acute sickling episodes. Chronic management may include hydroxyurea and, in certain cases, chronic transfusions. Given the limitations of these treatments, there is no effective long-term treatment. The only advanced treatment for SCD is allogeneic HSCT. Complications of allogeneic HSCT include a significant risk of treatment-related mortality, graft failure, GvHD and opportunistic infections, particularly in patients who undergo non-sibling-matched allogeneic HSCT. About the Starbeam (ALD-102) Study The Phase 2/3 Starbeam Study is assessing the efficacy and safety of Lenti-D, an investigational gene therapy, in boys up to 17 years of age with CALD. The study involves transplantation with a patient's own stem cells, which are modified to contain a functioning copy of the ABCD1 gene. This gene addition should result in the production of functional adrenoleukodystrophy protein (ALDP), a protein critical for the breakdown of very long chain fatty acids (VLCFAs). Buildup of VLCFAs in the central nervous system contributes to neurodegeneration in CALD. Patients enrolled in the study are:
The primary efficacy endpoint for the Starbeam study is the proportion of subjects who are alive and have none of six major functional disabilities (MFDs) at 24 months post treatment. MFDs are six symptoms captured in the Neurologic Function Score (NFS) that, if present, are expected to severely affect the patient's capacity for independent living: loss of communication, cortical blindness, tube feeding, total incontinence, wheelchair dependence, and complete loss of voluntary movement. About CALD Cerebral adrenoleukodystrophy (CALD) is a rare and commonly fatal, X-linked, genetic, neurodegenerative disease that primarily affects young boys. CALD involves a progressive destruction of myelin, the protective sheath of the nerve cells in the brain that are responsible for thinking and muscle control. Symptoms usually occur in early childhood and progress rapidly if untreated, leading to severe loss of neurological function and eventual death within 2-5 years in most patients. Early diagnosis is critical for boys to receive effective treatment. The worldwide incidence rate for ALD is approximately one in 21,000 male newborns; of those, 30-40% are affected by the cerebral form of the disease. Currently, the only effective treatment option for patients with CALD is allogeneic HSCT. Complications of allogeneic HSCT include a significant risk of treatment-related mortality, graft failure, GvHD and opportunistic infections, particularly in patients who undergo non-sibling-matched allogeneic HSCT. 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 four clinical studies for the treatment of transfusion-dependent ß-thalassemia 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 program, bb2121, is an anti-BCMA CAR T program partnered with Celgene. bb2121 is currently being studied in a Phase 1 trial 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, Washington; and Paris, France.
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