Bluebird Bio and Novo Nordisk have entered into a research agreement to develop in vivo genome editing candidates for haemophilia, as well as other severe genetic diseases.
During the three-year collaboration, the companies plan to utilise Bluebird’s proprietary mRNA-based megaTAL technology, which has the potential to "provide a highly specific and efficient way to silence, edit or insert genetic components."
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.
Aligned with Novo Nordisk’s haemophilia portfolio, Bluebird has stated that 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 Marcus Schindler, senior vice president for global drug discovery at Novo Nordisk.
He continued, “This important research collaboration aimed at addressing genetic diseases at the DNA level reflects Novo Nordisk’s enduring commitment and dedication to inventing disease-modifying medicines that can truly change the lives of people living with haemophilia and other genetic diseases.”
Haemophilia A affects approximately one in 5,000 people, and haemophilia B approximately one in 25,000 males.Over 400,000 males have haemophilia A or B and the disease is severely underdiagnosed in developing countries.
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.