Novo Nordisk aims to add gene therapy to haemophilia portfolio via development deal with bluebird bio

Novo Nordisk entered a partnership with bluebird bio to jointly develop next-generation in vivo genome editing treatments for use in patients with haemophilia and other genetic diseases, the companies announced Wednesday. Financial details of the agreement were not disclosed.

The drugmakers noted that the three-year collaboration will initially aim to provide patients with haemophilia A with a treatment that can correct FVIII-clotting factor deficiency, in line with Novo Nordisk's current haemophilia portfolio. The companies will use bluebird bio's mRNA-based megaTAL technology, which they stated has the potential to provide a highly specific and efficient method for silencing, editing or inserting genetic components.

Marcus Schindler, senior vice president for global drug discovery at Novo Nordisk, commented "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."

There are a number of companies developing gene therapies for patients with haemophilia A, with BioMarin expected to seek US and EU approval of valoctocogene roxaparvovec by the end of the year. For related analysis, see ViewPoints: Fresh concern about BioMarin prioritising speed over quality with val-rox.

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