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bluebird bio Presents megaTAL Genome Editing Data at American Society of Hematology (ASH) Workshop on Genome Editing

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jul. 18, 2016-- 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 presentation of pre-clinical data from its megaTAL genome editing platform at the ASH Workshop on Genome Editing, held July 14-15 in Washington, D.C.

In an invited presentation entitled “Gene Editing with megaTALs: Advantages, Challenges and Future Prospects,” Jordan Jarjour, Ph.D., director of nuclease research and development, bluebird bio, detailed the anatomy and physiology of megaTALs and discussed how to expand their DNA targeting capacity and refine their specificity.

The therapeutic application of genome editing demands technology that is capable of both high on-target activity and exquisite genome-wide specificity. The data reported at the ASH workshop highlight recent progress bluebird has made in:

  • Expanding the number of megaTAL targetable sites in the genome to permit the fine placement of an editing event within a target gene
  • Refining the specificity of megaTALs to eliminate undesirable off-target activity
  • Combining megaTALs targeting different target genes to achieve the knockout of multiple genes simultaneously

In addition, the presentation discussed the unique manner in which megaTALs engage DNA, resulting in both extra layers of specificity as well as efficient gene editing outcomes, such as gene addition via homology-directed insertion.

“Our work with our proprietary megaTAL genome editing platform has shown that we can engineer the protein interface to target genes or genetic elements at very high resolution, and also that we have validated a suite of protein engineering tools to reduce or eliminate off-target activity,” said Dr. Jarjour. “Additionally, the orthogonal nature of the enzymes used allows us to independently edit multiple targets simultaneously. We believe multiplex gene editing has particularly exciting potential for use in development of T cell-based oncology therapies, offering the possibility of addressing multiple immunoregulatory pathways.”

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 three clinical studies for the treatment of transfusion-dependent ß-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.

Forward-Looking Statements

This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding 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 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.

Source: bluebird bio, Inc.

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