Novo taps bluebird bio for gene-editing tech

Novo Nordisk
Novo Nordisk is looking to follow up its bleeding disorder franchise with next-gen treatments. (Novo Nordisk)

Looking to further broaden its reach outside of diabetes, Novo Nordisk has teamed up with bluebird bio to treat a series of genetic diseases.

The pair will over a three-year R&D pact develop next-gen in vivo genome-editing treatments for genetic diseases, with hemophilia being an early focus.

Financial details of the pact were not disclosed.

The Danish pharma, more known for its metabolic disease products in diabetes and obesity (certainly in sales terms), has been ramping up work in hemophilia over the past few years, and earlier this year earned FDA and EMA nods for hemophilia A drug Esperoct, its latest antihemophilic factor.

Now, however, it’s looking to the future of R&D and will alongside bluebird find a “development gene therapy candidate with the ambition of offering people with hemophilia A a lifetime free of factor replacement therapy.”

This is in line with efforts from across hemophilia R&D to focus on gene therapies as potential long-term fixes to the bleeding disorder, including from biotechs BioMarin Pharmaceutical and Spark Therapeutics (which is in a protracted takeover bid from Roche, also working separately on hemophilia) as well as Pfizer and Sangamo Therapeutics. Traditional treatments in this space are locked in a fierce competitive battle for market share, and Novo is now in the game to look for hemophilia treatment 2.0.

Novo’s collab will tap into bluebird’s mRNA-based megaTAL tech that is set up to edit or insert genetic components. The pact will initially focus on correcting factor VIII clotting deficiency with “the potential to explore additional therapeutic targets,” the partners said in a joint statement.

RELATED: BioMarin drops lower dose of its hemophilia gene therapy as it eyes submissions by year-end

“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 of 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 hemophilia and other genetic diseases.”

Novo also has its own antibody in development for the bleeding disorder, known as concizumab. The drug recently completed a phase 2 trial in hemophilia A and B patients with inhibitors and showed a reduced annual bleeding rate on prophylaxis compared with its on-demand NovoSeven.

While working on hemophilia, bluebird’s biggest internal work is on other blood disorders and cancers, using its LentiGlobin gene therapy in transfusion-dependent beta thalassemia as well as work anti-BCMA therapy bb21217 in multiple myeloma.

“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,” added Philip Gregory, D. Phil., chief scientific officer at 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 hemophilia 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.”