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Novo Nordisk, bluebird team up to develop haemophilia A therapies

Companies enter into three-year research collaboration


Novo Nordisk has joined forces with bluebird bio to develop in vivo genome editing treatments for haemophilia and other genetic diseases.

The three-year research collaboration will leverage the ability of bluebird’s mRNA-based megaTAL technology to silence, edit or insert genetic components. Novo is contributing its haemophilia expertise.

Together, bluebird and Novo will initially work on therapies intended to correct FVIII-clotting factor deficiency. The collaboration may expand to cover other therapeutic targets later.

“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,” Philip Gregory, chief scientific officer at bluebird, said in a statement.

Multiple companies are developing therapies designed to address the genetic underpinnings of FVIII deficiency, a disease also known as haemophilia A.

Spark Therapeutics, the subject of a protracted takeover attempt by Roche, has a haemophilia A gene therapy in late-phase development. BioMarin and uniQure are also working on haemophilia A gene therapies.

The nature of bluebird’s technology means it and Novo will take a different approach to treating the disease. Whereas gene therapies use vectors such as adeno-associated viruses to insert genetic material into target cells, bluebird’s technology uses molecular scissors to cut DNA, triggering the body’s repair pathways.

Other gene editing technologies such as zinc finger nucleases, CRISPR/Cas9 and TALENs use similar mechanisms but bluebird thinks its hybrid nucleases have an edge over the competition.

“Since these hybrid nucleases still cut DNA using homing endonuclease cleavage biochemistry, they engage DNA repair pathways in a manner distinct from all other gene editing nucleases,” bluebird said.

By accessing the technology, Novo hopes to gain the ability to compete with companies that want to claim its share of the haemophilia A market.

Novo sells a recombinant FVIII under the brand NovoEight and recently won FDA approval for Esperoct, an extended half-life FVIII molecule. The Danish company also has a monoclonal antibody against Tissue Factor Pathway Inhibitor in midphase development.

However, genetic therapies that target the root cause of haemophilia A could lessen the need for Novo’s existing treatments, threatening a source of revenues at a time when the company’s key diabetes franchise is also under pressure.

Article by
Nick Taylor

10th October 2019

From: Research



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