The research collaboration will utilise bluebird bio’s proprietary mRNA-based megaTAL™ technology that has the potential to provide a highly specific and efficient way to silence, edit or insert genetic components. Aligned with Novo Nordisk’s haemophilia portfolio, the research collaboration will initially focus on correcting FVIII-clotting factor deficiency, with the potential to explore additional therapeutic targets.
“We are pleased to announce our collaboration with bluebird whose demonstrated capabilities in gene therapy will enable the next-generation of innovative products to make a significant impact on patients’ lives,” said
“bluebird has made tremendous progress on enabling an in vivo gene editing platform based on our megaTAL technology, including important advances in high-quality mRNA production and purification,” said
MegaTALs are a single-chain fusion enzyme that combines the natural DNA cleaving processes of Homing Endonucleases (HEs) with the DNA binding region of transcription activator-like (TAL) effectors. TALs are easily engineered proteins that recognize specific DNA sequences. This protein fusion architecture allows the generation of extremely active and highly specific and compact nucleases that are compatible with all current viral and non-viral cell delivery methods.
The incidence of haemophilia A is approximately one in 5,000 and of haemophilia B it is approximately one in 25,000 male births respectively1. Over 400,000 males have haemophilia A or B and the disease is severely underdiagnosed in developing countries2. An estimated 304,0003 people live with haemophilia A, which is a result of decreased or defective production of the blood clotting factor VIII. People with haemophilia B, of which there are some 136,0004 have deficiencies in producing clotting factor IX. For people with haemophilia, bleeds often occur in the joints, particularly knees and ankles. Bleeds can also occur in the muscles, soft tissues, gastrointestinal tract or even the brain. Trauma, major surgery, tooth extractions or other minor surgical interventions require medical supervision to manage the associated bleeding. Without treatment, bleeds are painful and can cause lasting damages and lead to impaired mobility.
About bluebird bio, Inc.
bluebird bio is pioneering gene therapy with purpose. From our
bluebird bio is a human company powered by human stories. We’re putting our care and expertise to work across a spectrum of disorders by researching cerebral adrenoleukodystrophy, sickle cell disease, transfusion-dependent β-thalassemia and multiple myeloma using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.
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For more information, visit novonordisk.com,
bluebird bio 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 research and development plans for bluebird bio’s gene therapy products and megaTAL platform. 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 risks that bluebird bio’s megaTAL platform will not be successful or will not be safe or effective in clinical trials, the risks that our collaboration with
- Stonebraker JS; Study of variations in the reported haemophilia A prevalence around the world. Haemophilia 2010; 16(1):20-32.
- Stonebraker JS, A study of variations in the reported haemophilia B prevalence around the world. Haemophilia 2012; 18(3):e91-e94
Source: bluebird bio, Inc.
For bluebird bio
Elizabeth Pingpank, 617-914-8736
Jennifer Snyder, 617-448-0281
For Novo Nordisk A/S
Peter Hugreffe Ankersen, +45 3075 9085
Valdemar Borum Svarrer, +45 3079 0301
Ann Søndermølle Rendbæk, +45 3075 2253
Kristoffer Due Berg (US), +1 609 235 2989
Martin Havtorn Petersen, +45 3075 5246
Ken Inchausti (US), +1 609 240 9429