New gene therapy partnership between bluebird bio and Novo Nordisk

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Emerging US gene therapy company bluebird bio has entered a collaboration with Novo Nordisk focused on the development of in vivo genome editing treatments for a range of diseases, including haemophilia.

They are amongst a range of companies to identify the potential for gene therapy to revolutionise the treatment of haemophilia. However, in vivo genome editing involves directly modifying a patient's DNA.

BioMarin and Pfizer have gene therapies in development. BioMarin recently reported updated data from a study of its gene therapy, valoctocogene roxaparvovec, that enables haemophilia patients to produce their own factor VIII.

In the study, bleeding episodes dropped from a median of 16.5 per year to none, meaning the patients no longer required blood transfusions. This was down from an average of 139 per year.

The new three-year collaboration between bluebird bio and Novo Nordisk will also focus on potential gene therapy for people living with a factor VIII deficiency.

It will utilise bluebird bio’s mRNA-based megaTAL technology that the company says has the potential to provide a highly specific and efficient way to silence, edit or insert genetic components.

“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,” said Marcus Schindler, senior vice president for Global Drug Discovery at Novo Nordisk.

“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.”

“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,” said Philip Gregory, D. Phil., chief scientific officer, bluebird bio.

“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.”