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News Article | May 8, 2017
Site: www.businesswire.com

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Agilis Biotherapeutics, Inc. (Agilis), a biotechnology company advancing innovative gene therapy products for the treatment of rare genetic diseases that affect the central nervous system (CNS), announced today that the Company has expanded its commercial and medical teams, hiring Markus Peters, Ph.D., as Chief Commercial Officer, Kirsten Gruis, M.D. as Chief Medical Officer, and Anne Marie Conway, M.H.A, R.N., as Vice President Clinical Operations. “We are pleased to welcome these three talented individuals to our leadership team. Each brings a wealth of experience to Agilis that is directly aligned with our mission to help patients with rare CNS diseases, advance our clinical pipeline, and lay the foundation for future approval and commercialization of our promising gene therapy product candidates,” said Dr. Mark Pykett, President and CEO of Agilis. Dr. Markus Peters will lead Agilis’ commercial, business development and business analytics activities, and will spearhead market efforts for the company’s aromatic L-amino acid decarboxylase (AADC) deficiency gene therapy globally. He brings a significant background to Agilis in the commercialization of rare disease therapeutics and specialty pharmaceuticals. Most recently, he was an Associate Partner with the consulting group Alacrita. Dr. Peters was previously Vice President, Global Marketing/Commercial with Synageva, where he led the cross-functional global launch team for first-in-class enzyme replacement therapy Kanuma to develop and implement the global strategy and launch plan for the product in ultra-rare LAL deficiency. He was also responsible for the commercial assessment of the Synageva pipeline. Before Synageva, he was Head of Global Marketing Nephrology and Transplant Therapeutic Area at Alexion, leading the global launch of the ultra-orphan Soliris aHUS (atypical hemolytic uremic syndrome) franchise. Dr. Peters previously worked at Merck where he led the global launch of recombinant biologic Elonva, and at Sepracor, Wyeth, Bayer and Boehringer Ingelheim in the US, Japan and Europe in business and commercial roles of increasing responsibility. He holds a Ph.D. in Biochemistry from Heinrich-Heine Universität. Kirsten Gruis, M.D., is an accomplished physician scientist, board certified neurologist, and rare disease specialist, with a broad background in the development of innovative therapeutics. She has worked in Friedreich’s ataxia, Spinal Muscular Atrophy (SMA), Amyotrophic Lateral Sclerosis (ALS) and Duchenne Muscular Dystrophy (DMD), among others, across a range of development stages, including pre-clinical, Phase I, Phase II and Phase III programs. Dr. Gruis was most recently at WAVE Life Sciences leading their clinical development plans in DMD. Previously, she was at Idera Pharmaceuticals where she lead the team to initiate a global, phase II trial in dermatomyositis and, before that, she was at Alnylam Pharmaceuticals as the clinical lead of a global, phase III program of patisiran in the rare disease familial amyloidotic polyneuropathy and, before that, was with Pfizer managing pre-clinical and phase I-II assets in the Rare Disease Research Unit focused on Friedreich’s ataxia, SMA, ALS, and DMD. Dr. Gruis held academic appointments as an Associate Professor of Neurology at both the University of Michigan and SUNY Upstate Medical University. She received her MD from the University of Iowa and did her residency training at the University of Michigan, where she subsequently joined the faculty. While at SUNY Upstate Medical University, she served as the Director of the MDA clinic, Co-Director of the ALS Clinic, and prior to that was Director of the Motor Neuron Disease Center/ALS Clinic at the University of Michigan. Dr. Gruis is a member of the American Academy of Neurology and World Muscle Society, as well as a Fellow American Association of Neuromuscular & Electrodiagnostic Medicine with additional board certification in Neuromuscular Disorders. She has served on multiple NIH Scientific Review Panels for the NINDS and Neurotechnology study groups as well as a principal investigator of several clinical studies for ALS. Anne Marie Conway, M.H.A., R.N., brings extensive experience in clinical operations to Agilis’ clinical development programs. Most recently, she was Principal at AMC Consulting, providing clinical operations services to a range of drug development organizations including Rhythm Pharmaceuticals, bluebird bio and Lantheus Medical Imaging, among others. Before that, she worked at Ziopharm Oncology as head of Clinical Operations and Data Management. While there, she managed the start-up of the gene therapy program for high grade gliomas. Prior to that, she was at Shire Human Genetic Therapies (now integrated into Shire, plc) as Vice President, Development Operations, providing management oversight for the global filing and approval of VPRIV™ for Gaucher disease, running a global Phase III trial and subsequent approval of Firazyr™ for hereditary angioedema and, leading global clinical operations, data management and registry group for eight rare disease pipeline products in Phases I through IV studies. Prior to its acquisition by Shire, Ms. Conway worked at Transkaryotic Therapies and led the integrated development team for the clinical sections of the BLA/MAA filing and subsequent approval in the United States, European Union, and Japan for elaprase™. Before moving into industry, Ms. Conway worked at Tufts Medical Center as a Clinical Trials Manager, Outpatient Nurse Coordinator, and Staff Nurse. She has an M.H.A. from Suffolk University and a B.S. from Boston University, is a licensed nurse, and holds an adjunct faculty position at Suffolk University. Agilis is advancing innovative gene therapies designed to provide long-term efficacy for patients with debilitating, often fatal, rare genetic diseases that affect the central nervous system. Agilis’ gene therapies are engineered to impart sustainable clinical benefits by inducing persistent expression of a therapeutic gene through precise targeting and restoration of lost gene function to achieve long-term efficacy. Agilis’ rare disease programs are focused on gene therapy for AADC deficiency, Friedreich’s ataxia, and Angelman syndrome, all rare genetic diseases that include neurological deficits and result in physically debilitating conditions. We invite you to visit our website at www.agilisbio.com Some of the statements made in this press release are forward-looking statements. These forward-looking statements are based upon our current expectations and projections about future events and generally relate to our plans, objectives and expectations for the development of our business. Although management believes that the plans and objectives reflected in or suggested by these forward-looking statements are reasonable, all forward-looking statements involve risks and uncertainties and actual future results may be materially different from the plans, objectives and expectations expressed in this press release.


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


News Article | April 26, 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 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), 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.


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.

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