Rare Daily Staff

Novo Nordisk is jumping into the race to develop next-generation hemophilia therapies through a deal with Bluebird Bio to jointly develop in vivo genome editing treatments for genetic diseases, including hemophilia.

During the three-year research collaboration, bluebird and Novo Nordisk will focus on identifying a development gene therapy candidate that can replace the lifetime of factor replacement therapy that patients with hemophilia A now face.

Hemophilia A is an X-linked recessive bleeding disorder caused by mutations in the F8 gene, which results in the decreased or defective production of the blood clotting factor VIII, thus slowing the blood clotting process. People with this disorder experience prolonged bleeding or oozing following an injury, surgery, or having a tooth pulled. In severe cases, heavy bleeding can occur after minor trauma or even in the absence of injury. Serious complications can result from bleeding into the joints, muscles, brain, or other internal organs. The incidence of hemophilia A is approximately one in 5,000 male births, and an estimated 304,000 people around the world live with hemophilia A.

The research collaboration will utilize Bluebird Bio’s proprietary mRNA-based technology to potentially provide a highly specific and efficient way to silence, edit or insert genetic components. Aligned with Novo Nordisk’s hemophilia portfolio, the research collaboration will initially focus on correcting FVIII-clotting factor deficiency, with the potential to explore additional therapeutic targets.

“We believe this technology has the potential to create a highly differentiated approach to the treatment of many severe genetic diseases,” said Philip Gregory, chief scientific officer of Bluebird Bio. He believes the collaboration will the two companies towards their shared goal of changing the therapeutic approach to hemophilia and reduce the burden of disease for patients with factor VIII deficiency.

Bluebird’s gene editing platform technology easily engineers proteins that recognize specific DNA sequences. This allows the generation of extremely active and highly specific and compact enzymes for gene editing that are compatible with all current viral and non-viral cell delivery methods.

Other companies developing next-generation therapies targeting hemophilia include BioMarin Pharmaceuticals’ which is moving ahead to file for approval of its gene therapy candidate; Roche, which is hoping to expand its franchise through its acquisition of Spark Therapeutics; and Sangamo and Pfizer, which have recently announced early positive data for their hemophilia candidate.

Photo: Philip Gregory, chief scientific officer of Bluebird Bio

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