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News Article | December 7, 2016
Site: www.businesswire.com

CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage biotechnology company committed to developing potentially transformative gene therapies for severe genetic diseases and cancer, today announced the pricing of an underwritten public offering of 3,289,473 shares of its common stock at a public offering price of $76.00 per share, before underwriting discounts. In addition, bluebird bio has granted the underwriters a 30-day option to purchase up to an additional 493,420 shares of common stock. All of the shares in the offering are to be sold by bluebird bio. Goldman, Sachs & Co., BofA Merrill Lynch and Cowen and Company are acting as joint book-running managers of the offering. Wells Fargo Securities and Wedbush PacGrow are acting as co-managers. The offering is expected to close on or about December 12, 2016, subject to customary closing conditions. bluebird bio anticipates the total gross proceeds from the offering (before deducting underwriters’ discounts and commissions and estimated offering expenses) will be approximately $250 million, excluding any exercise of the underwriters’ option to purchase additional shares. The shares are being offered by bluebird bio pursuant to an automatically effective shelf registration statement that was previously filed with the Securities and Exchange Commission (SEC). A preliminary prospectus supplement relating to and describing the terms of the offering was filed with the SEC on December 6, 2016. The final prospectus supplement relating to the offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. When available, copies of the final prospectus supplement and the accompanying prospectus relating to these securities may also be obtained by contacting one of the following: Goldman, Sachs & Co., Attn: Prospectus Department, 200 West Street, New York, NY 10282, telephone: (212) 902-1171, facsimile: (212) 902-9316, email: prospectus-ny@ny.email.gs.com; BofA Merrill Lynch, NC1-004-03-43, 200 North College Street, 3rd floor, Charlotte, NC 28255-0001, Attn: Prospectus Department, email: dg.prospectus_requests@baml.com; or Cowen and Company, LLC, c/o Broadridge Financial Services, 1155 Long Island Avenue, Edgewood, NY, 11717, Attn: Prospectus Department, by telephone at (631) 274-2806 or by fax at (631) 254-7140. This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of, these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state or jurisdiction. With its lentiviral-based gene therapy 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 beta-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. This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the anticipated final terms, timing and completion of the offering, and bluebird bio’s existing 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 and uncertainties related to market conditions and satisfaction of customary closing conditions related to the public offering, that the preliminary positive results from our prior and ongoing clinical trials will not continue or be repeated in our ongoing, planned or expanded clinical trials, the risk 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, the risk that the current or planned clinical trials of LentiGlobin and Lenti-D will be insufficient to support regulatory submissions or marketing approval in the United States and European Union, 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, and the risk that any one or more of our product candidates will not be successfully developed and commercialized. There can be no assurance that bluebird bio will be able to complete the public offering on the anticipated terms, or at all. 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.


News Article | December 6, 2016
Site: www.businesswire.com

CAMBRIDGE, Mass.--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage biotechnology company committed to developing potentially transformative gene therapies for severe genetic diseases and cancer, today announced that it has commenced an underwritten public offering of $200 million of its common stock. bluebird bio also intends to grant the underwriters a 30-day option to purchase up to an additional fifteen percent (15%) of the shares of common stock offered in the public offering. All of the shares in the proposed offering are to be sold by bluebird bio. Goldman, Sachs & Co., BofA Merrill Lynch and Cowen and Company are acting as joint book-running managers of the proposed offering. Wells Fargo Securities and Wedbush PacGrow are acting as co-managers. The offering is subject to market and other conditions, and there can be no assurance as to whether or when the offering may be completed, or as to the actual size or terms of the offering. The shares are being offered by bluebird bio pursuant to an automatically effective shelf registration statement that was previously filed with the Securities and Exchange Commission (SEC). A preliminary prospectus supplement relating to and describing the terms of the offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. When available, copies of the preliminary prospectus supplement and the accompanying prospectus relating to these securities may also be obtained by contacting one of the following: Goldman, Sachs & Co., Attn: Prospectus Department, 200 West Street, New York, NY 10282, telephone: (212) 902-1171, facsimile: (212) 902-9316, email: prospectus-ny@ny.email.gs.com; BofA Merrill Lynch, NC1-004-03-43, 200 North College Street, 3rd floor, Charlotte, NC 28255-0001, Attn: Prospectus Department, email: dg.prospectus_requests@baml.com; or Cowen and Company, LLC, c/o Broadridge Financial Services, 1155 Long Island Avenue, Edgewood, NY, 11717, Attn: Prospectus Department, by telephone at (631) 274-2806 or by fax at (631) 254-7140. The final terms of the offering will be disclosed in a final prospectus supplement to be filed with the SEC. This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of, these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of such state or jurisdiction. With its lentiviral-based gene therapy 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 beta-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. This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the anticipated final terms, timing and completion of the proposed offering, and bluebird bio’s existing 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 and uncertainties related to market conditions and satisfaction of customary closing conditions related to the proposed public offering, that the preliminary positive results from our prior and ongoing clinical trials will not continue or be repeated in our ongoing, planned or expanded clinical trials, the risk 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, the risk that the current or planned clinical trials of LentiGlobin and Lenti-D will be insufficient to support regulatory submissions or marketing approval in the United States and European Union, 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, and the risk that any one or more of our product candidates will not be successfully developed and commercialized. There can be no assurance that bluebird bio will be able to complete the proposed public offering on the anticipated terms, or at all. 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, announced treatment of the first patient in Northstar-2, the Phase 3 study of its LentiGlobin drug product in patients with transfusion-dependent β-thalassemia (TDT) and non-β0/β0 genotypes. This study will use LentiGlobin drug product manufactured with the addition of transduction enhancers intended to increase the drug product vector copy number and percent of cells transduced. The study’s primary endpoint is the proportion of treated subjects who meet the definition of "transfusion independence," defined as total hemoglobin levels of at least 9g/dL without any RBC transfusions for a continuous period of at least 12 months at any time during the study. “The opening of our first Phase 3 trial in TDT is an exciting milestone for bluebird,” said David Davidson, chief medical officer. “At ASH we presented interim LentiGlobin data from the Northstar study showing substantial and durable treatment effects, with all patients with non-β0/β0 genotypes and at least twelve months of follow-up achieving freedom from transfusions. We are hopeful that our new manufacturing process incorporating transduction enhancers will build upon these results and may provide even better outcomes for patients. It is an encouraging start for Northstar-2 to have achieved a robust drug product VCN of 2.9 c/dg and 77% LVV+ for our first treated patient. We are tremendously grateful to the patients, families, study investigators, and site staff who have participated in our LentiGlobin program and enabled progress toward our goal of improving the lives of patients with TDT.” “The current results of the HGB-204 and HGB-205 LentiGlobin studies show a reduction, and in some cases, elimination of RBC transfusions in patients with TDT,” said Mark C. Walters, M.D., UCSF Benioff Children’s Hospital Oakland, a principal investigator on the study. “The current study, Northstar-2, will test a new manufacturing method to increase the VCN in the drug product. If it is successful, this new enhanced transduction method could expand the number of patients who no longer need transfusions after the gene therapy treatment.” bluebird bio is also moving forward with plans to initiate Northstar-3 (HGB-212), a Phase 3 trial of LentiGlobin drug product in patients with transfusion-dependent β-thalassemia with the β0/β0 genotype. This study will also be conducted under the new manufacturing process, and is expected to begin enrolling patients in 2017. The primary endpoint of this planned study is transfusion reduction. Northstar-2 is a Phase 3, global, multi-center study designed to evaluate the safety and efficacy of LentiGlobin drug product in patients with transfusion-dependent beta-thalassemia with non-β0/β0 genotypes. In this study, the manufacturing process by which the patient’s cells are transduced with the LentiGlobin viral vector is modified, with the intent of increasing vector copy number and the percentage of cells successfully transduced. The target enrollment of the study is 15 adult and adolescent patients and 8 pediatric patients. The study’s primary endpoint is the proportion of treated subjects who meet the definition of "transfusion independence," defined as total hemoglobin levels of at least 9g/dL without any RBC transfusions for a continuous period of at least 12 months at any time during the study. Transfusion-dependent β-thalassemia (TDT), also called β-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 TDT 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 and opportunistic infections, particularly in patients who undergo non-sibling-matched allogeneic HSCT. 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™ drug product, 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 megaTALs/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 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, 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 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-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.


News Article | December 15, 2016
Site: www.businesswire.com

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Surface Oncology, an immuno-oncology company developing next-generation immunotherapies that target the tumor microenvironment, today announced the appointment of Robert W. Ross, MD as its Chief Medical Officer. Dr. Ross will be responsible for advancing Surface Oncology’s development programs, including SRF231, a fully human CD47 antibody, into and through the clinic. Dr. Ross joins Surface from bluebird bio, where he was most recently head of the Oncology franchise and Senior Vice President of Clinical Development and pharmacovigilance. “Immune therapy is the future of oncology treatment. In the last five years, with the introduction of immune system modulation, the lives of patients have been fundamentally changed, but there is clearly more work to do to bring more cures to patients,” said Dr. Ross. “Surface Oncology’s strategy targeting the various components of the tumor microenvironment has an excellent chance of achieving that goal. I look forward to working closely with this outstanding team to move their programs to the clinic as rapidly as possible.” Dr. Ross has diverse clinical development experience, including cancer patient care at Dana Farber Cancer Center, and clinical development roles at Genentech and Infinity Pharmaceuticals. Most recently he was at bluebird bio, leading the clinical development of genetically modified cellular therapies in sickle cell disease and β thalassemia. He was also the head of oncology at bluebird bio, building a multifaceted oncology program, led by an anti-BCMA chimeric antigen T cell therapy currently in clinical trials in patients with multiple myeloma. “The addition of such a well-respected and experienced professional to our team positions us well as we continue to expand our pipeline and advance novel programs into the clinic,” said Detlev Biniszkiewicz, PhD, President and CEO of Surface Oncology. “Rob has extensive experience building strong teams and leading early stage development programs into late stage clinical development. We are excited he will be applying these skills at Surface to help achieve our goal of bringing cures to patients.” In order to re-activate a patient’s immune response to cancer, Surface Oncology is developing next-generation immunotherapies targeting the tumor microenvironment. Our broad attack has the potential to convert patients’ non-responsive ‘cold’ tumors into immune-active ones. We are committed to advancing our diverse pipeline of immunotherapies including our fully human CD47 antibody program, to bring more cures to patients who do not benefit from existing cancer treatments. To accomplish this, Surface’s world-class team is working with our network of leading academic advisors and a robust scientific platform integrating tumor immunology, antibody characterization, and translational science. Headquartered in the heart of Cambridge’s Kendall Square, Surface was founded by Atlas Venture and financed by an industry-leading syndicate of investors. In 2016, Surface entered into a global strategic collaboration with Novartis to bring four next-generation immunotherapies to patients. CD47 is an important immune escape mechanism exploited by multiple tumor types, making it a target with broad therapeutic potential. CD47 acts as a macrophage checkpoint or “don’t eat me” signal that prevents cells from being eliminated by a macrophage-mediated process called phagocytosis. Surface presented data on SRF231, which demonstrated it binds with high affinity to CD47, stimulates phagocytosis of cancer cells in vitro, and has potent in vivo anti-tumor activity, both as monotherapy and in combination with standard of care. These data also demonstrated that SRF231 does not induce detectable hemagglutination or phagocytosis of red blood cells in vitro, a potentially important safety advantage.


SAN DIEGO--(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, announced the presentation of new data from the ongoing HGB-205 clinical study evaluating its LentiGlobin product candidate in patients with transfusion-dependent β-thalassemia (TDT) and severe sickle cell disease (SCD) at the 58th American Society of Hematology Annual Meeting. The data from the HGB-205 study were highlighted today in a poster presentation by Marina Cavazzana, M.D., Ph.D., lead investigator of the HGB-205 study conducted in Necker Hospital, AP-HP 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 Hospitals, AP-HP and Inserm) and the Lymphohematopoiesis Laboratory, Institute of Genetic Diseases, Imagine, Paris, France. “We believe the enduring responses seen in this study - in the patients with TDT as well as the patient with SCD - demonstrate the continued promise of LentiGlobin gene therapy in both of these patient populations. We have seen nearly three years of transfusion independence in TDT in certain patients, providing important data on the long-term safety and durability of this therapy,” said David Davidson, M.D., chief medical officer, bluebird bio. “In addition, it is encouraging that the patient with SCD has remained free of acute SCD-related clinical events in the 21 months since treatment, when he previously required monthly blood transfusions to help control his SCD symptoms. This patient’s successful outcome not only offers hope for the potential of LentiGlobin to benefit other patients with SCD, but also provides important insights into this complex disease that we are applying to our ongoing HGB-206 study.” Abstract #2311: Update from the HGB-205 Phase 1/2 Clinical Study of LentiGlobin Gene Therapy: Sustained Clinical Benefit in Severe Hemoglobinopathies HGB-205 is an ongoing, open-label, single-center Phase 1/2 study designed to evaluate the safety and efficacy of LentiGlobin drug product in the treatment of patients with TDT and severe SCD. Four patients with TDT and one patient with severe SCD have undergone infusion with LentiGlobin drug product in this study as of September 9, 2016. The patients with TDT have between 11.6 and 33.5 months of follow-up, and the patient with SCD has 22.9 months of follow-up. “These data show a stable clinical and biological effect in patients with TDT or severe SCD who have received a one-time treatment with LentiGlobin,” said Professor Cavazzana. “We are now seeing the benefit of gene therapy with LentiGlobin beyond two years in TDT in certain patients, and clinical benefit continues to be realized in the patient with severe SCD after almost 24 months of follow-up. We are encouraged by these results and the potential benefit treatment with LentiGlobin can have on patients living with these debilitating diseases and without an HLA compatible sibling donor.” bluebird bio will host a live webcast at 8:30 p.m. PT (11:30 p.m. ET) on Monday, 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. 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 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. 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 and 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 results from our prior and ongoing clinical trials of LentiGlobin, including HGB-205, will not continue or be repeated in our ongoing or planned 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-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. About AP-HP: AP-HP - Greater Paris University hospitals - is a European world-renowned European 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 grzeat source of pride. The AP-HP is the leading employer un 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


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 | November 16, 2016
Site: marketersmedia.com

— Burkitt lymphoma is a form of non-Hodgkin's lymphoma in which cancer starts in immune cells called B-cells. Symptoms include fever, night sweats, swollen lymph nodes and weight loss. The disease is more common in males and people with compromised immune systems, such as those with HIV/AIDS. Treatment includes chemotherapy and radiation therapy. Report Highlights Pharmaceutical and Healthcare latest pipeline guide Burkitt Lymphoma – Pipeline Review, H2 2016, provides comprehensive information on the therapeutics under development for Burkitt Lymphoma (Oncology), complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The guide covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. The Burkitt Lymphoma (Oncology) pipeline guide also reviews of key players involved in therapeutic development for Burkitt Lymphoma and features dormant and discontinued projects. The guide covers therapeutics under Development by Companies /Universities /Institutes, the molecules developed by Companies in Phase II, Phase I and Preclinical stages are 1, 3 and 13 respectively. Similarly, the Universities portfolio in Phase II and Preclinical stages comprises 1 and 2 molecules, respectively.Burkitt Lymphoma. Burkitt Lymphoma (Oncology) pipeline guide helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis. Scope - The pipeline guide provides a snapshot of the global therapeutic landscape of Burkitt Lymphoma (Oncology). - The pipeline guide reviews pipeline therapeutics for Burkitt Lymphoma (Oncology) by companies and universities/research institutes based on information derived from company and industry-specific sources. - The pipeline guide covers pipeline products based on several stages of development ranging from pre-registration till discovery and undisclosed stages. - The pipeline guide features descriptive drug profiles for the pipeline products which comprise, product description, descriptive licensing and collaboration details, R&D brief, MoA & other developmental activities. - The pipeline guide reviews key companies involved in Burkitt Lymphoma (Oncology) therapeutics and enlists all their major and minor projects. - The pipeline guide evaluates Burkitt Lymphoma (Oncology) therapeutics based on mechanism of action (MoA), drug target, route of administration (RoA) and molecule type. - The pipeline guide encapsulates all the dormant and discontinued pipeline projects. - The pipeline guide reviews latest news related to pipeline therapeutics for Burkitt Lymphoma (Oncology) Reasons to buy - Procure strategically important competitor information, analysis, and insights to formulate effective R&D strategies. - Recognize emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage. - Find and recognize significant and varied types of therapeutics under development for Burkitt Lymphoma (Oncology). - Classify potential new clients or partners in the target demographic. - Develop tactical initiatives by understanding the focus areas of leading companies. - Plan mergers and acquisitions meritoriously by identifying key players and it’s most promising pipeline therapeutics. - Formulate corrective measures for pipeline projects by understanding Burkitt Lymphoma (Oncology) pipeline depth and focus of Indication therapeutics. - Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope. - Adjust the therapeutic portfolio by recognizing discontinued projects and understand from the know-how what drove them from pipeline. Table of Contents Table of Contents 2 List of Tables 5 List of Figures 6 Introduction 7 Burkitt Lymphoma Overview 8 Therapeutics Development 9 Pipeline Products for Burkitt Lymphoma - Overview 9 Pipeline Products for Burkitt Lymphoma - Comparative Analysis 10 Burkitt Lymphoma - Therapeutics under Development by Companies 11 Burkitt Lymphoma - Therapeutics under Investigation by Universities/Institutes 13 Burkitt Lymphoma - Pipeline Products Glance 14 Clinical Stage Products 14 Early Stage Products 15 Burkitt Lymphoma - Products under Development by Companies 16 Burkitt Lymphoma - Products under Investigation by Universities/Institutes 17 Burkitt Lymphoma - Companies Involved in Therapeutics Development 18 AbbVie Inc 18 Arvinas, Inc. 19 bluebird bio, Inc. 20 Boehringer Ingelheim GmbH 21 Constellation Pharmaceuticals, Inc. 22 Immunomedics, Inc. 23 Karyopharm Therapeutics, Inc. 24 Millennium Pharmaceuticals Inc 25 Patrys Limited 26 Seattle Genetics, Inc. 27 Takeda Pharmaceutical Company Limited 28 Theravectys SA 29 Burkitt Lymphoma - Therapeutics Assessment 30 Assessment by Monotherapy Products 30 Assessment by Target 31 Assessment by Mechanism of Action 33 Assessment by Route of Administration 35 Assessment by Molecule Type 37 Drug Profiles 39 19-3s - Drug Profile 39 Product Description 39 Mechanism Of Action 39 R&D Progress 39 20-3s - Drug Profile 40 Product Description 40 Mechanism Of Action 40 R&D Progress 40 alisertib - Drug Profile 41 Product Description 41 Mechanism Of Action 41 R&D Progress 41 ARV-825 - Drug Profile 46 Product Description 46 Mechanism Of Action 46 R&D Progress 46 bb-2121 - Drug Profile 47 Product Description 47 Mechanism Of Action 47 R&D Progress 47 Cellular Immunotherapy to Target CD19 for ALL - Drug Profile 49 Product Description 49 Mechanism Of Action 49 R&D Progress 49 Cellular Immunotherapy to Target CD30 for Oncology - Drug Profile 50 Product Description 50 Mechanism Of Action 50 R&D Progress 50 denintuzumab mafodotin - Drug Profile 51 Product Description 51 Mechanism Of Action 51 R&D Progress 51 E1-3s - Drug Profile 54 Product Description 54 For more information, please visit http://www.wiseguyreports.com


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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