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bluebird bio Announces Collaboration with Duke University’s Robert J. Margolis, MD, Center for Health Policy on Value-Based Payment Framework for Gene Therapy

– Collaboration includes industry leaders; aims to develop strategies for sustainable, value-based approach to access transformative gene therapy treatments –

CAMBRIDGE, Mass.--(BUSINESS WIRE)--May 23, 2017-- 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 that it will participate in a consortium led by Duke University’s Robert J. Margolis, MD, Center for Health Policy to develop a broadly-supported path for value-based payment reform models for gene therapies and other innovative treatments.

“bluebird bio is focused on creating gene therapy products that have a dramatic impact on the lives of patients. Understanding the potential value of these treatments across the healthcare system is not easy, but it is critical that we all work together to develop new and effective payment models that work for all stakeholders, especially patients,” said Nick Leschly, chief bluebird. “Duke-Margolis is a national leader in health policy, and this consortium will bring together key stakeholders across healthcare sectors to create pathways for value-based payment. We look forward to the consortium’s ability to not only drive engagement but also help explore more creative, forward looking, and perhaps most importantly, concrete solutions that brings value first and foremost to the patient.”

The consortium – composed of patient advocates, payers, manufacturers, and providers, as well as experts in regulatory science, law, and policy – will collaborate to outline a path forward for payment reform involving innovative therapies, including genomic medicines for rare diseases.

“Moving from payment arrangements based on volume to those based on value is not easy,” said Duke-Margolis director Mark McClellan, MD, PhD. “Our consortium members represent a wide range of perspectives but share a common goal of payment models that encourage innovation, resulting in better outcomes for patients at a sustainable cost.”

The initial phase of the project is expected to be completed in 2017. For more information, visit

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.

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

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 collaboration with the Duke-Margolis center and the potential of its gene therapy product candidates. 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 our product candidates will not continue or be repeated in our ongoing, planned or expanded clinical trials 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, 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.

Source: bluebird bio, Inc.

bluebird bio, Inc.
Manisha Pai, 617-245-2107
Elizabeth Pingpank, 617-914-8736