Novo Nordisk A/S has signed a research collaboration with bluebird bio Inc. to identify a gene therapy for hemophilia.
The therapy would potentially end the need for injected factor replacement therapy, which replaces the missing blood clotting factors that can cause uncontrolled bleeding in those with hemophilia A.
The three-year deal will use Cambridge, Mass.-based bluebird bio's mRNA-based technology, which can silence, edit or insert genetic components, to develop treatments for inherited disease; the initial focus will be on correcting factor VIII-clotting factor deficiency, with the potential to explore other targets.
"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 hemophilia and other genetic diseases," Marcus Schindler, Novo Nordisk's senior vice president for global drug discovery, said in a news release.
An estimated 304,000 people live with hemophilia A, which is caused by a lack of the blood clotting factor VIII. Hemophilia B, which accounts for some 136,000 people, occurs in those who have deficiencies in producing clotting factor IX. Bagsværd, Denmark-based Novo hired Ludovic Helfgott from AstraZeneca PLC a year ago to run its hemophilia and growth hormone franchise after competition in the hemophilia market intensified following approval of Roche Holding AG's Hemlibra.
"We believe this technology has the potential to create a highly differentiated approach to the treatment of many severe genetic diseases," said bluebird bio's chief scientific officer, Philip Gregory. "We believe this collaboration will move us toward our shared goal of recoding the treatment paradigm and substantially reduce the burden of disease for patients with factor VIII deficiency."
