bluebird bio Announces Presentation at Cooley's Anemia Foundation Symposium

bluebird bio Announces Presentation at Cooley's Anemia Foundation Symposium

Professor Marina Cavazzana presents overview on experience with investigational gene therapy in beta-thalassemia


bluebird bio, Inc. (Nasdaq: BLUE) announced that Marina Cavazzana, M.D., Ph.D., professor of hematology and director of the Department of Biotherapy at Hospital Necker, University Paris Descartes and lead investigator for the HGB-205 clinical study, presented a review of the clinical study experience with lentiviral-based gene therapies for beta-thalassemia at the 10th Annual Cooley's Anemia Foundation Symposium in Chicago today.

Professor Cavazzana summarized results from HGB-205, an ongoing clinical study using LentiGlobin® BB305 for the treatment of beta-thalassemia major and severe sickle cell disease, which were presented earlier this year at the Annual Congress of the European Hematology Association. She also provided an update on subject 1003, a patient with beta-thalassemia major treated by Professor Cavazzana in 2007 in the LG001 study using the first-generation HPV569 lentiviral vector. Following approximately seven years of transfusion independence, subject 1003 has recently required two blood transfusions after experiencing clinical symptoms of anemia. Importantly, both the expression of HbAT87Q and the vector copy number in peripheral blood leukocytes, a measure of the persistence of the gene therapy, have remained largely unchanged. The safety profile for both vectors is consistent with autologous transplantation, with no gene-therapy related serious adverse events.

Development of bluebird bio's approach to treating hemoglobinopathies was guided by the data collected from the LG001 study, a pioneering clinical study that served to highlight both the potential for gene therapy as an intervention in beta-thalassemia as well as the need for an improved lentiviral vector. To this end, Professor Cavazzana also discussed the advances in the design of lentiviral vectors for hemoglobinopathy gene therapies -- specifically the evolution from the first-generation HPV569 lentiviral vector to bluebird bio's current lentiviral vector, BB305, which results in substantially improved vector copy number and HbAT87Q expression.

"The early clinical experience with HPV569 represented a crucial proof of concept for the potential of gene therapy to bring life-changing treatment to patients in need," said David Davidson, M.D., chief medical officer, bluebird bio. "The data from the LG001 study were invaluable to our efforts over the last five years to optimize our gene therapy approach with improvements to the potency, robustness and manufacturing for the next-generation lentiviral vector, BB305. LentiGlobin BB305 drug product is being evaluated in three ongoing clinical trials for the treatment of patients with beta-thalassemia major and severe sickle cell disease, and three abstracts related to these ongoing clinical trials have been accepted for presentation at this year's American Society of Hematology's Annual Meeting in December."

About bluebird bio, Inc.
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 T cell-based immunotherapy. bluebird bio's clinical programs include Lenti-Dproduct candidate currently in a Phase 2/3 study, called the Starbeam Study, for the treatment of childhood cerebral adrenoleukodystrophy, and LentiGlobin® BB305 product candidate, currently in three clinical studies: a global Phase 1/2 study, called the Northstar Study, for the treatment of beta-thalassemia major; a single-center Phase 1/2 study in France (HGB-205) for the treatment of beta-thalassemia major or severe sickle cell disease; and a separate U.S. Phase 1 study for the treatment of severe sickle cell disease (HGB-206). bluebird bio also has ongoing preclinical CAR T immuno-oncology programs, as well as discovery research programs utilizing megaTALs/homing endonuclease gene editing technologies.

bluebird bio has operations in Cambridge, Massachusetts, Seattle, Washington, and Paris, France.

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 potential efficacy and safety of the Company's LentiGlobin BB305 product candidate, in subjects with beta thalassemia major, including statements concerning the reduced or eliminated need for transfusion support for the study subjects, statements concerning the Company's future plans with respect to LentiGlobin and its other product candidates. It should be noted that the data for LentiGlobin BB305 announced from the HGB-205 study at the EHA Congress are preliminary in nature and the Northstar, HGB-205 and HGB-206 studies of LentiGlobin BB305 are not completed. There is limited data concerning long-term safety and efficacy following treatment with LentiGlobin BB305 drug product. These data may not continue for these subjects or be repeated or observed in ongoing or future studies involving our LentiGlobin BB305 product candidate, including the HGB-205 Study, the Northstar Study or the HGB-206 study in severe sickle cell disease. It is possible that subjects for whom transfusion support has been reduced or eliminated may receive transfusion support in the future. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that the preliminary results from our clinical trials will not continue or be repeated in our ongoing clinical trials, the risk that previously conducted studies involving similar product candidates will not be repeated or observed in ongoing or future studies involving current product candidates, the risk of cessation or delay of any of the ongoing or planned clinical studies and/or our development of our product candidates, the risk of a delay in the enrollment of patients in our clinical studies, the risk that our 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 and 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.


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