bluebird bio Presents megaTAL Genome Editing Data at American Society of Hematology (ASH) Workshop on Genome
Editing
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.
Investors and Media
bluebird bio, Inc.
Manisha Pai, 617-245-2107
mpai@bluebirdbio.com
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