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News Article | April 26, 2017
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CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird 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, today announced that members of the management team will present at the following upcoming investor conferences: To access the live webcast of bluebird bio’s presentations, please visit the “Calendar of Events” page within the Investors and Media section of the bluebird bio website at http://investor.bluebirdbio.com. Replays of the webcast will be available on the bluebird bio website for 90 days following the conference. This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Company’s product candidates and research programs. 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, risks that the preliminary results from our clinical trials will not continue or be repeated in our ongoing clinical trials, 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 our clinical studies, risks that the current or planned clinical trials of the LentiGlobin drug product will be insufficient to support regulatory submissions or marketing approval in the United States and European Union, the risk that our collaborations, including the collaboration with Celgene, will not continue or will not be successful, and the risk that any one or more of our product candidates 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 annual report on Form 10-K and our most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.


CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) today announced that it has entered into a worldwide license agreement around its proprietary lentiviral vector platform with GlaxoSmithKline Intellectual Property Development Limited (GSK). “bluebird bio’s work has been integral to the progress of lentiviral vector-based cell and gene therapy; over the past six years, we have taken the incredible potential of our lentiviral vector platform and successfully applied it to our own clinical gene therapy and oncology programs,” said Philip Gregory, D.Phil., chief scientific officer, bluebird bio. “We are pleased that our agreement with GSK now allows us to facilitate the work of others striving to develop transformational therapies for patients with rare genetic diseases.” Under the terms of the agreement, GSK will non-exclusively license certain bluebird patent rights related to lentiviral vector technology to develop and commercialize gene therapies for Wiscott-Aldrich syndrome and metachromatic leukodystrophy, two rare genetic diseases. Financial terms of the agreement include an upfront payment to bluebird as well as potential development and regulatory milestone payments and low single digit royalties on net sales of covered products. 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™ product candidate, currently in four clinical studies for the treatment of transfusion-dependent β-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 megaTAL/homing endonuclease gene editing technologies with the potential for use across the company’s pipeline. 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 Company’s license agreements with third parties and our licensee’s product candidates, as well as the advancement of, and anticipated development and regulatory milestones and plans related to the Company’s product candidates and clinical studies. 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, risks that the product candidates of our licensees are not successfully developed, approved or commercialized, risks that the preliminary results from our clinical trials will not continue or be repeated in our ongoing clinical trials, the risk of cessation or delay of any of the ongoing or planned clinical studies and/or our development of our product candidates, risks that the current or planned clinical trials of the LentiGlobin drug product will be insufficient to support regulatory submissions or marketing approval in the United States and European Union, and the risk that any one or more of our product candidates 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 annual report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.


CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE) today announced that it has entered into a worldwide license agreement around its proprietary lentiviral vector platform with Novartis Pharma AG. “bluebird bio is a pioneer in the field of lentiviral vector-based cell and gene therapy, and our partnerships and licensing agreements have been crucial to our success since our early days. We have continued to build upon our intellectual property, applying the incredible potential of lentiviral vectors to both our ongoing clinical gene therapy and immuno-oncology programs,” said Jeff Walsh, chief financial and strategy officer, bluebird bio. “Our agreement with Novartis is a testament to our leadership in the field, and allows us to facilitate the efforts of others working to develop transformational therapies for patients.” Under the terms of the agreement with Novartis, Novartis will non-exclusively license certain bluebird patent rights related to lentiviral vector technology to develop and commercialize chimeric antigen receptor T cell (CAR T) therapies for oncology, including CTL019, Novartis’s anti-CD19 CAR T investigational therapy. Financial terms of the agreement include an upfront payment to bluebird as well as milestone and royalty payments. 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™ product candidate, currently in four clinical studies for the treatment of transfusion-dependent β-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 megaTAL/homing endonuclease gene editing technologies with the potential for use across the company’s pipeline. This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Company’s license agreements with third parties and our licensee’s product candidates, as well as the advancement of, and anticipated development and regulatory milestones and plans related to the Company’s product candidates and clinical studies. 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, risks that the product candidates of our licensees are not successfully developed, approved or commercialized, risks that the preliminary results from our clinical trials will not continue or be repeated in our ongoing clinical trials, the risk of cessation or delay of any of the ongoing or planned clinical studies and/or our development of our product candidates, risks that the current or planned clinical trials of the LentiGlobin drug product will be insufficient to support regulatory submissions or marketing approval in the United States and European Union, and the risk that any one or more of our product candidates 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 annual report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.


CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird 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, today reported business highlights and financial results for the first quarter ended March 31, 2017. “In the first quarter of 2017, we have been laser-focused on continuing to develop our commercial and manufacturing infrastructure and ensuring that we are prepared for future MAA and BLA filings,” said Nick Leschly, chief bluebird. “Throughout 2017, we will be presenting data across all four of our clinical programs. On our two most advanced programs in TDT and cerebral adrenoleukodystrophy, these data will dictate the timing and path for future regulatory submissions in the US and Europe. For our Phase 1/2 programs in SCD and multiple myeloma, the 2017 data will dictate the timing and path for our planned Phase 3 trials. I’m enthusiastic about the progress our teams are making, and look forward to continuing to move our programs forward through the second half of 2017.” First Quarter 2017 Financial Results and Financial Guidance 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™ product candidate, currently in four clinical studies for the treatment of transfusion-dependent β-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 megaTAL/homing endonuclease gene editing technologies with the potential for use across the company’s pipeline. 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 Company’s financial condition and results of operations, as well as the advancement of, and anticipated development and regulatory milestones and plans related to the Company’s product candidates and clinical studies. 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, risks that the preliminary results from our clinical trials will not continue or be repeated in our ongoing clinical trials, 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 our clinical studies, the risks that the changes we have made in the LentiGlobin drug product manufacturing process or the HGB-206 clinical study protocol will not result in improved patient outcomes, risks that the current or planned clinical trials of the LentiGlobin drug product will be insufficient to support regulatory submissions or marketing approval in the United States and European Union, the risk that our collaborations, including the collaboration with Celgene, will not continue or will not be successful, and the risk that any one or more of our product candidates 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 Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.


This new report includes an updated discussion of approved and clinical stage agents in immuno-oncology, including recently-approved agents. It also addresses the means by which researchers and companies are attempting to build on prior achievements in immuno-oncology to improve outcomes for more patients. Some researchers and companies refer to this approach as immuno-oncology 2.0. The American Society of Clinical Oncology (ASCO), in its 12th Annual Report on Progress Against Cancer (2017), named Immunotherapy 2.0 as its Advance of the Year. Nevertheless, metastatic melanoma remains incurable. Furthermore, in many studies in advanced melanoma and other cancers, only a minority of patients have benefited from immunotherapy treatments. Researchers and companies are therefore looking for ways to build on the initial successes of the immuno-oncology field to improve outcomes for more patients, hence the need for an immuno-oncology 2.0.  Agents that are intended to improve the results of treatment with agents like checkpoint inhibitors may also be referred to as second-wave immuno-oncology agents. As discussed in this report, researchers have found that checkpoint inhibitors produce tumor responses by reactivating TILs (tumor infiltrating lymphocytes)-especially CD8+ cytotoxic T cells. This key observation is perhaps the most important factor driving development of second-wave immuno-oncology strategies. As a result, researchers have been developing biomarkers that distinguish inflamed (i.e., TIL-containing) tumors-which are susceptible to checkpoint inhibitor therapy-from cold tumors, which are not. They have also been working to develop means to render cold tumors inflamed, via treatment with various conventional therapies and/or development of novel agents. These studies are the major theme of second-wave immuno-oncology, or immuno-oncology 2.0. Highlights of this Report Include: - Approvals of checkpoint inhibitors - Biomarkers for checkpoint inhibitor treatments - Approved and clinical-stage immunotherapy biologics other than checkpoint inhibitors - Immunotherapy with TIL cells - Commercialization of TIL therapy - Adoptive immunotherapy with genetically engineered T cells bearing chimeric antigen receptors (CARs) - Manufacturing issues with CAR T-cell therapies - General conclusions on the progress of cellular immunotherapy - Outlook for cancer immunotherapy Key Topics Covered: 1: Introduction - The early history of cancer immunotherapy - Coley's toxins - Cytokines as immunomodulatory drugs - Interleukin-2 - Alpha-interferons - Interleukin-12 - Interleukin-12 as a bridge between innate and adaptive immunity - Investigation of interleukin-12 as an anticancer therapeutic - Interleukin-10 - Interleukin-15 - Admune/Novartis' heterodimeric IL-15:IL-15Ra (hetIL-15) - Altor's ALT-803 - Conclusions: Cytokine-based immunotherapies for cancer 2: What are immune checkpoints? - CTLA-4 blocking agents - Ipilimumab - Tremelimumab - PD-1 blocking agents - Nivolumab - Combination therapy of nivolumab plus ipilimumab in melanoma - Pembrolizumab - Pembrolizumab as a first-line treatment for advanced NSCLC - Pembrolizumab in colorectal carcinoma with mismatch-repair deficiency - Studies of pembrolizumab in combination immunotherapies - PDR001 - PD-L1 blocking agents - Atezolizumab - Atezolizumab in treatment of urothelial carcinoma - Atezolizumab for the treatment of NSCLC - Atezolizumab in treatment of other solid tumors - Other anti-PD-L1 mAb agents - Durvalumab - Avelumab - Anti-LAG-3 agents - anti-TIM-3 - NewLink Genetics' small-molecule IDO pathway inhibitors and checkpoint inhibition - Infinity's PI3K? inhibitor IPI-549 for modulation of immune suppression in tumors - Biomarkers for checkpoint inhibitor treatments - Target biomarkers - Genetic biomarkers - Immunological biomarkers - Use of biomarker tests in treatment with checkpoint inhibitors - Checkpoint inhibitors plus radiation therapy - Checkpoint inhibitors plus targeted therapies - Checkpoint inhibitors with cytotoxic chemotherapies - Discussion 3: Immune Agonists - Celldex Therapeutics' Varlilumab (CDX-1127) - OX40 agonists - MedImmune/AZ's OX40 agonist program - Roche/Genentech's OX40 agonist program - Nektar Therapeutics/BMS's NKTR-214, a CD122 agonist - Glucocorticoid-induced TNFR-related (GITR) protein agonist (Leap Therapeutics' TRX518) - Conclusions 4: Bispecific antibodies - Marketed bispecific antibody agents - Catumaxomab - Blinatumomab - Bispecific antibodies as an alternative to CAR-T cells - Xencor's cross-linking monoclonal antibody (XmAb) bispecific platform technology - Regeneron's native human immunoglobulin-format bsAb, REGN1979 - Roche/Genentech's full-length bsAbs: Generated using CrossmAb technology - MacroGenics' MGD007: Generated using dual-affinity re-targeting (DART) technology - Conclusions 5: Therapeutic Anticancer Vaccines and Oncolytic viruses - Introduction - Cancer vaccines-a field rife with clinical failures - Why has the cancer vaccine field been so prone to clinical failure? - Marketed therapeutic cancer vaccines and oncolytic virus therapies - Dendreon/Valeant's sipuleucel-T - Amgen's talimogene laherparepvec (T-Vec)/Imlygic - Therapeutic cancer vaccines and oncolytic virus therapies in clinical development - Celldex's CDX-1401 - Bavarian Nordic's PROSTVAC-VF - Argos Therapeutics' AGS-003 - Sydys Corporation's CVac - Aduro Biotech's CRS-207 - TapImmune's TPIV110 HER2/neu and TPIV200 folate receptor alpha multi-epitope vaccines - Genelux's GL-ONC1 oncolytic virus - Conclusions 6: Adoptive Immunotherapy for Cancer - Introduction - Adoptive immunotherapy with tumor infiltrating lymphocytes - A specific immunodominant mutation in a melanoma patient who had a durable complete remission due to TIL therapy - Adoptive immunotherapy based on mutation-specific CD4+ T cells in an epithelial cancer - Successful targeting of KRAS G12D via adoptive immunotherapy in a case of metastatic colorectal cancer - Dr. Rosenberg's recent studies on neoantigen-reactive TILs for use in adoptive cellular immunotherapy - Commercializing TIL therapy - Adoptive immunotherapy with genetically engineered T cells bearing chimeric antigen receptors (CARs) - Leading clinical programs in CAR T-cell based immunotherapy - Kite Pharma's KTE-C19 (axicabtagene ciloleucel) - Novartis' CTL019 - Juno's JCAR015 and other Juno anti-CD19 CARs - Other CAR T-cell therapies that target hematologic malignancies - bluebird bio's bb2121 for multiple myeloma - CAR T-cell therapies that target solid tumors - Novartis/University of Pennsylvania's CARTmeso - EGFRvIII CAR T-cell therapies - Companies developing engineered improvements in CAR T-cell therapy - Bellicum Pharmaceuticals' technologies for modulation of CAR T-cell therapies - Cellectis' technologies for design and manufacture of off-the shelf CAR T-cell therapies - Manufacturing issues with CAR T-cell therapies - Can bispecific antibodies be competitive with CAR T-cell therapies? - Adptimmune recombinant TCR clinical candidates - Kite Pharma recombinant TCR program - Juno Therapeutics' recombinant TCR program - Recombinant TCR studies at the NCI - Conclusions - Market size estimates for the T-cell therapy market 7: General Conclusions - Major theme of this report: Immuno-oncology 2.0 or second-wave immuno-oncology - Approvals of checkpoint inhibitors - Biomarkers for checkpoint inhibitor treatments - Approved and clinical-stage immunotherapy biologics other than checkpoint inhibitors - Immunotherapy with TIL cells - Commercialization of TIL therapy - Adoptive immunotherapy with genetically engineered T cells bearing chimeric antigen receptors (CARs) - Manufacturing issues with CAR T-cell therapies - Adoptive immunotherapy via autologous recombinant TCR technology - General conclusions on the progress of cellular immunotherapy - Insight Pharma Reports survey on cancer immunotherapy - Outlook for cancer immunotherapy For more information about this report visit http://www.researchandmarkets.com/research/rkf8pp/cancer Research and Markets Laura Wood, Senior Manager press@researchandmarkets.com For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716 To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/global-cancer-immunotherapy-report-2017-building-on-initial-successes-to-improve-clinical-outcomes---research-and-markets-300441006.html


CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird 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, today announced that it has appointed John O. Agwunobi, M.D. and Douglas A. Melton, Ph.D. to its Board of Directors. “We are delighted to welcome Dr. Agwunobi and Dr. Melton to our Board. John’s experience in the government and payor realm will be invaluable as we work to identify and create paths for access to one-time transformative therapies. Doug’s deep scientific expertise in regenerative medicine and stem cell biology will be critical as we continue to innovate to deliver a pipeline of therapies for the long term,” said Nick Leschly, chief bluebird. “With these two esteemed leaders, we are adding to the expertise of our seasoned Board of Directors to prepare us for continued progress and growth at bluebird.” Since February 2016, John O. Agwunobi, M.D. has served as Chief Health and Nutrition Officer at Herbalife Nutrition Institute (Nasdaq: HLF), responsible for training, education, science strategy and product development. Prior to joining Herbalife, Dr. Agwunobi advised a number of privately-held health-related companies and served as Senior Vice President and President of Health and Wellness for Wal-Mart in the United States, where he grew the business and provided insight and advice on the company’s health reform position. From December 2005 to September 2007, he served as the Assistant Secretary of Health for the U.S. Department of Health and Human Services, where he was responsible for disease prevention and health promotion. Dr. Agwunobi has served on numerous boards, and is currently a director at Magellan Health, Inc. (NASDAQ: MGLN), and at the U.S. African Development Foundation. Dr. Agwunobi is a licensed physician in Florida, Maryland and Washington, D.C. “I’m excited to be joining the board of bluebird bio, a company with a singular focus on changing the lives of patients,” said Dr. Agwunobi. “In my career, I have had the opportunity to work on some of the most challenging issues in healthcare. I look forward to now working with bluebird as they strive to deliver on the hope of one-time transformative therapies for patients.” Douglas A. Melton, Ph.D. is the Xander University Professor at Harvard University, where he has been a professor for over thirty years. He has served as the Co-Director of the Harvard Stem Cell Institute since 2004, and also as the Co-Chair of the Department of Stem Cell and Regenerative Biology since 2007. Since 1994, Dr. Melton has been an Investigator of the Howard Hughes Medical Institute. He was a scientific co-founder of Gilead Sciences, Inc., Curis, Inc. and Semma Therapeutics, Inc. “bluebird bio is leading the development of rare disease and cancer therapies derived from patients’ own cells, and is uniquely positioned to deliver on the scientific promise of gene therapy,” said Dr. Melton. “I’ve had the privilege to be part of several other companies breaking new ground in science, and I look forward to bringing that experience to bluebird as they continue their pioneering work.” 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™ product candidate, currently in four clinical studies for the treatment of transfusion-dependent β-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 megaTAL/homing endonuclease gene editing technologies with the potential for use across the company’s pipeline. 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 Company’s product candidates and research programs. 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, risks that the preliminary results from our clinical trials will not continue or be repeated in our ongoing clinical trials, 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 our clinical studies, the risk that our collaborations, including the collaboration with Celgene, will not continue or will not be successful, and the risk that any one or more of our product candidates 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 quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.


CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird 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, today announced the publication in the New England Journal of Medicine of a case study on Patient 1204, the first patient with severe sickle cell disease (SCD) to be treated with gene therapy. This patient, who was 13 years old at the time of treatment, was treated with LentiGlobin drug product in the HGB-205 clinical study conducted in Necker Hospital, Assistance Publique-Hôpitaux de Paris, Paris, France. The data in the publication reflect 15 months of follow-up, and a brief summary of this patient’s outcomes with 21 months of follow-up was presented at the 58th American Society of Hematology Annual Meeting in December 2016. “We have managed this patient at Necker for more than 10 years, and standard treatments were not able to control his SCD symptoms. He had to receive blood transfusions every month to prevent severe pain crises,” said Professor Marina Cavazzana, M.D., Ph.D., principal investigator of this study and professor of hematology at Paris Descartes University, head of the department of Biotherapy Hospital, the clinical research center of Biotherapy at Necker Enfants Malades - Greater Paris University Hospital, AP-HP and INSERM, and of the Lymphohematopoiesis Laboratory, Imagine Institute of Genetic Diseases, Paris, France. “Since receiving the autologous stem cell transplant with LentiGlobin, he has been free from severe symptoms and has resumed normal activities, without the need for further transfusions.” “Since our initial publication of this therapeutic approach in mouse models in 2001, we are delighted to obtain such a clear proof-of-principle of its efficacy in a patient,” said Philippe Leboulch, M.D. Dr. Leboulch is professor of medicine at the University Paris-Sud and High Counselor and International Scientific Director at France’s CEA. He was a scientific founder of bluebird bio and serves as the co-chairman of its Scientific Advisory Board. Dr. Leboulch led the development of the anti-sickling T87Q globin vector used in LentiGlobin. “We are pleased to see this case study published in NEJM and shared with the broader research community. The successful outcome in Patient 1204 demonstrates the promise of treatment with LentiGlobin gene therapy in patients with severe SCD and serves as a guide for our efforts to optimize outcomes in future patients,” said David Davidson, M.D., chief medical officer, bluebird bio. “By analyzing this patient’s experience, we have identified key variables to optimize in our ongoing HGB-206 study of LentiGlobin gene therapy in severe SCD, and we are hopeful that these protocol changes will enable subsequent patients to achieve the transformative benefit seen in Patient 1204.” Clinical and Biological Outcomes for the First Patient with Sickle Cell Disease Treated with Gene Therapy Patient 1204, a male patient with βS/βS genotype, was enrolled in May 2014 at 13 years of age into the HGB-205 clinical study. The patient underwent a regular transfusion regimen for 4 years prior to this study. He had an average of 1.6 SCD-related events annually in the 9 years prior to initiating transfusions, and his complications from SCD included vaso-occlusive crises, acute-chest syndrome, bilateral hip osteonecrosis, and cerebral vasculopathy. The patient underwent two bone marrow harvests to collect hematopoietic stem cells (HSCs) for gene transfer and back-up (6.2×108 and 5.4×108 total nucleated cells/kg harvested). CD34+ cells were enriched from the harvested marrow and then transduced with LentiGlobin BB305 lentiviral vector. The vector copy numbers (VCN; vector copies per diploid genome) for the drug product lots manufactured were 1.0 and 1.2. The patient underwent myeloablation with intravenous busulfan (2.3 to 4.8 mg/kg per day for 4 days) with daily pharmacokinetic studies and dose adjustment. Total busulfan area under the curve (AUC) was 19,363 μmol*min. After a 2-day washout, Patient 1204 was infused with LentiGlobin drug product in October 2014 at a post-thaw total dose of 5.6×106 CD34+ cells/kg. RBC transfusions were to be continued after transplantation until a sufficient proportion of HbAT87Q (25-30% of total Hb) was detected. Neutrophil and platelet engraftment were achieved on Day +38 and Day +91 post-transplantation, respectively. HbAT87Q levels increased steadily and RBC transfusions were discontinued after the last transfusion on Day +88. HbAT87Q reached 5.5 g/dL (46% of total Hb) at Month 9 and continued to increase to 5.7 g/dL at Month 15 (48%), with a reciprocal decrease in HbS levels to 5.5 g/dL (46%) at Month 9, and 5.8 g/dL (49%) at Month 15. Total Hb levels have been stable between 10.6 and 12.0 g/dL since Month 6 post-transplant. HbF levels have remained below 1.0 g/dL. Adverse events (AEs) were consistent with busulfan conditioning, and no AEs related to LentiGlobin drug product have been observed to date. Over the 15 months since transplantation, no SCD-related clinical events or hospitalizations have occurred, contrasting favorably with the period before the patient began regular transfusions. All medications have been discontinued, including pain medication. The patient has resumed regular school attendance and reports full participation in normal physical activities. About SCD Sickle cell disease (SCD) is an inherited disease caused by a mutation in the β-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 therapy for SCD is allogeneic hematopoietic stem cell transplantation (HSCT). Complications of allogeneic HSCT include a significant risk of treatment-related mortality, graft failure, graft-versus-host disease, 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 β-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) 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 megaTAL/homing endonuclease gene editing technologies with the potential for use across the company’s pipeline. About AP-HP AP-HP - Greater Paris University hospitals - is a European world-renowned university hospital. Its 39 hospitals treat 8 million people every year: in consultation, emergency, during scheduled or home hospitalizations. The AP-HP provides a public health service for everyone, 24 hours a day. This mission is a duty as well as a great source of pride. The AP-HP is the leading employer in the Greater Paris area: 100,000 staff members – doctors, researchers, paramedical staff, administrative personnel and workers – work there. http://www.aphp.fr About the Imagine Institute As the leading European center for research, care and teaching in genetic diseases, the Imagine Institute's primary aim is to understand and cure. The Institute's staff includes 850 of the best physicians, scientists and healthcare professionals housed in an innovative new building designed to realize synergies. This unprecedented continuum of expertise available in close proximity to patients allows Imagine to accelerate discoveries and their application at the bedside. www.institutimagine.org 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 Company’s research and development plans for its LentiGlobin product candidate to treat severe sickle cell disease, including statements whether the manufacturing process changes for LentiGlobin will improve outcomes of patients with severe sickle cell disease and whether the planned changes to the HGB-206 clinical trial protocol will improve outcomes in patients with severe sickle cell disease. 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, 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, the risks that the changes we have made in the LentiGlobin manufacturing process or the HGB-206 clinical trial protocol will not result in improved patient outcomes, 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 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 quarterly report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.


News Article | February 28, 2017
Site: www.businesswire.com

CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird 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, today announced that members of the management team will present at the following upcoming investor conferences: To access the live webcast of bluebird bio’s presentations, please visit the “Calendar of Events” page within the Investors and Media section of the bluebird bio website at http://investor.bluebirdbio.com. Replays of the webcast will be available on the bluebird bio website for 90 days following the conference. This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the Company’s product candidates and research programs. 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, risks that the preliminary results from our clinical trials will not continue or be repeated in our ongoing clinical trials, 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 our clinical studies, risks that the current or planned clinical trials of the LentiGlobin drug product will be insufficient to support regulatory submissions or marketing approval in the United States and European Union, the risk that our collaborations, including the collaboration with Celgene, will not continue or will not be successful, and the risk that any one or more of our product candidates 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 quarterly report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.


CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird 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, today reported business highlights and financial results for the fourth quarter and full year ended December 31, 2016. “We ended 2016 with momentum to drive progress in 2017 and cash to fund the business well into 2019,” said Nick Leschly, chief bluebird. “2017 is a critical year for bluebird, with data readouts across all four of our clinical programs, including proof-of-concept data on manufacturing improvements for LentiGlobin; proof-of-concept data for the changes to the HGB-206 study protocol; additional data from our anti-BCMA CAR T program, bb2121; and full data from the first 17 patients in the Starbeam study of Lenti-D. Execution will also be a key theme for 2017, with a focus on laying the groundwork for future MAA and BLA filings and engagement with payors. All of these activities are building to the 2022 vision we laid out in January: to have multiple products on the market with dramatic patient impact and a deep pipeline driven by a sustainable innovation engine.” Fourth Quarter and Full Year 2016 Financial Results and Financial Guidance 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™ product candidate, currently in four clinical studies for the treatment of transfusion-dependent β-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 megaTAL/homing endonuclease gene editing technologies with the potential for use across the company’s pipeline. 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 Company’s financial condition and results of operations, the sufficiency of its cash, cash equivalents and marketable securities, as well as the advancement of, and anticipated development and regulatory milestones and plans related to the Company’s product candidates and clinical studies, including statements regarding whether the planned manufacturing process changes for the LentiGlobin drug product will improve outcomes in patients with transfusion-dependent β-thalassemia and severe sickle cell disease, whether the planned changes to the HGB-206 clinical study protocol will improve outcomes in patients with severe sickle cell disease and plans for future clinical data disclosures. 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, risks that the preliminary results from our clinical trials will not continue or be repeated in our ongoing clinical trials, 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 our clinical studies, the risks that the changes we have made in the LentiGlobin drug product manufacturing process or the HGB-206 clinical study protocol will not result in improved patient outcomes, risks that the current or planned clinical trials of the LentiGlobin drug product will be insufficient to support regulatory submissions or marketing approval in the United States and European Union, the risk that our collaborations, including the collaboration with Celgene, will not continue or will not be successful, and the risk that any one or more of our product candidates 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 annual report on Form 10-K, as well as discussions of potential risks, uncertainties, and other important factors in our subsequent filings with the Securities and Exchange Commission. All information in this press release is as of the date of the release, and bluebird bio undertakes no duty to update this information unless required by law.


News Article | March 3, 2017
Site: news.yahoo.com

Sickle Cell Disease (SCD) affects around five million people and sufferers often have anaemia, run a higher risk of infections and experience bouts of severe body pain (AFP Photo/Junior D. Kannah) Paris (AFP) - Scientists have used gene therapy to relieve the symptoms of a teenager suffering from sickle cell disease (SCD) in a world-first breakthrough, they reported on Thursday. SCD is an inherited disease caused by a gene mutation that results in red blood cells losing their usual donut-like appearance and taking on a sickle or crescent moon shape. Sufferers -- around five million worldwide -- often have anaemia and get tired easily, run a higher risk of infections and stroke, and experience bouts of severe body pain. But a team from the AP-HP university hospital group in Paris, the Imagine Institute of Genetic Diseases and gene therapy company bluebird bio said they managed to get a teenager off transfusions. The boy was the first person to be treated, in Paris in October 2014, for sickle cell disease in a clinical trial with gene therapy. Others have been tested since, but no official results published. The team collected so-called haematopoietic stem cells, which give rise to red blood cells, from the bone marrow of the youngster, then aged 13. The immature cells were treated with a therapeutic gene, carried in a deactivated virus, which recoded their DNA to correct blood cell production. The treated cells were then reinjected into the boy's body. Thursday's results, published in the New England Journal of Medicine, report on the child's health 15 months after treatment. He was still doing well after this point, but an official, updated status has yet to be published in a peer-reviewed journal. "He is well, he no longer needs monthly (blood) transfusions, anti-pain medication, or hospitalisation," study leader Marina Cavazzana told AFP. SCD is common in Africa, where up to 40 percent of a country's population can carry the mutated gene, though most never get sick. Last month, French researchers reported progress in developing a rapid, on-the-spot diagnosis for the disease. Early results from a trial in Togo, Mali and the Democratic Republic of Congo suggested the Sickle Scan was a faster, cheaper blood test than existing ones relying on lab equipment, its makers said. Rapid diagnosis is crucial to start SCD sufferers, especially young children, on potentially life-saving treatment.

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