The Danish big pharma Novo Nordisk has joined forces with the US gene therapy company bluebird bio to develop next-generation gene editing treatments for hemophilia.

The collaboration will last for three years and has the goal of developing a gene therapy that can permanently treat hemophilia A, a genetic disease causing uncontrolled bleeding.

Novo Nordisk will use bluebird bio’s gene editing technology to correct mutations in a blood-clotting protein called factor VIII. Instead of using the popular gene editing tool CRISPR/Cas9, bluebird bio’s technology consists of proteins called megaTALs, which are designed to be more precise than CRISPR.

Gene editing treatments for rare diseases are currently being tested in humans. For example, the Swiss company CRISPR Therapeutics and its US partner Vertex Pharmaceuticals are testing a CRISPR-based treatment for the blood condition beta-thalassemia in a phase I/II trial. While their treatment is based on editing cells outside of the body, bluebird bio’s strategy is to gene-edit patients directly, which is largely unexplored territory in the gene editing field.


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