Novo Nordisk enters into haemophilia gene therapy collaboration

pharmafile | October 10, 2019 | News story | Research and Development  

Novo Nordisk and bluebird bio have announced they have entered into a three-year research collaboration to jointly develop next-generation in vivo genome editing treatments for genetic diseases, including haemophilia.

During this three-year research collaboration, bluebird and Novo 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 use 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.

Alongside Novo Nordisk’s haemophilia portfolio, the collaboration will initially focus on correcting FVIII-clotting factor deficiency with the future potential to explore additional therapeutic targets.

Changes in the treatment of standard of haemophilia A, such as Roche’s 2017 launch of Hemlibra have put Novo Nordisk’s revenues which stand around at around $15.8bn in the US market under pressure.

Last year the Danish company lost about 14% in sales of its factor VIII replacement therapy NovoSeven, because Hemlibra disrupted the market with a once monthly dosing frequency versus every four days at best.

Marcus Schindler, senior vice president for Global Drug Discovery in Novo Nordisk said: “We are pleased to announce our collaboration with bluebird whose demonstrated capabilities in gene therapy will enable the next-generation of innovative products to make a significant impact on patients’ lives.

“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.”

Philip Gregory, Chief Scientific Officer at bluebird bio, added: “bluebird has made tremendous progress on enabling an in vivo gene editing platform based on our megaTAL technology, including important advances in high-quality mRNA production and purification.

“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 haemophilia research and therapeutics. 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.”

Nik Kiran

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