Novo Nordisk looks to bluebird for gene therapy targets

By Ben Hargreaves

- Last updated on GMT

(Image: Getty/Ismagilov)
(Image: Getty/Ismagilov)

Related tags Novo nordisk bluebird bio Gene therapy Hemophilia

Novo Nordisk partners with bluebird on a three-year research arrangement looking to discover gene therapy candidates for the treatment of genetic diseases, including hemophilia.

The two companies will work collaboratively to develop in-vivo​ genome editing treatments for genetic diseases. Specifically, the companies aim to develop a gene therapy for hemophilia A that could end patients’ reliance on factor replacement therapy.

Both companies will look to leverage bluebird bio’s mRNA-based ‘megaTAL’ technology, which the companies state can silence, edit or insert genetic components.

MegaTals are a single-chain fusion enzyme that combines the DNA cleaving processes of homing endonucleases with the DNA binding region of transcription activator-like (TAL) effectors.

The partners will look to target FVIII-clotting factor deficiency, before potentially moving to research additional therapeutic targets.

Philip Gregory, CSO at bluebird bio, said, “We believe this technology has the potential to create a highly differentiated approach to the treatment of many severe genetic diseases. Moreover, we are thrilled to be able to combine this new platform technology with Novo Nordisk’s deep expertise in hemophilia research and therapeutics.”

For Novo Nordisk, the partnership falls in-line with its hemophilia product portfolio and its efforts to expand into the advanced therapeutics areas, such as through its recent investment in bolstering its stem cell therapy capabilities​.

The company announced last year that it would be cutting 3% of its overall employee count​ in order to free up capital to reinvest in its pipeline of products, specifically within biologics and ‘technological innovation’.

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