Policy & Regulation
bluebird bio partners with Novo Nordisk to develop in vivo genome editing candidates for haemophilia
10 October 2019 -

Gene therapy company bluebird bio Inc (Nasdaq:BLUE) revealed on Wednesday that it plans to develop next-generation in vivo genome editing treatments for genetic diseases, including haemophilia, under a joint a research collaboration with Novo Nordisk A/S (CPH:NOVOB).

Pursuant to a three-year research agreement, the partners will focus on identifying a development gene therapy candidate with the ambition of offering people with haemophilia A a lifetime free of factor replacement therapy.

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.

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 recognise specific DNA sequences, bluebird bio explained.

Aligned with Novo Nordisk's haemophilia portfolio, the research will initially focus on correcting FVIII-clotting factor deficiency, with the potential to explore additional therapeutic targets.

Haemophilia A is a result of decreased or defective production of the blood clotting factor VIII. Haemophilia B is a deficiency in producing clotting factor IX. People with haemophilia bleed in the joints, particularly knees and ankles, as well as the muscles, soft tissues, gastrointestinal tract and even the brain, causing lasting damage and leading to impaired mobility.

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