Cambridge, Massachusetts-based bluebird bio and Bagsvaerd, Denmark-based Novo Nordisk announced they have agreed to collaborate to develop next-generation genome editing therapies for genetic diseases, including hemophilia. The deal will last three years, with a top priority to develop a gene therapy for hemophilia A.

The partnership will leverage bluebirds mRNA-based megaTAL technology that is used to silence, editor or insert genetic components. Novo Nordisk has a hemophilia portfolio. The initial focus will be on correcting FVIII-clotting factor deficiency. No financial details were disclosed.

MegaTALs are a single-chain fusion enzyme. It combines the natural DNA cleaving processes of Homing Endonucleases (HEs) with the activity of transcription activator-like (TAL) effectors at the DNA binding region. These proteins are easily engineered to recognize specific DNA sequences.

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, Novo Nordisks senior vice president for Global Drug Discovery.

He went on to say, This important research collaboration aimed at addressing genetic diseases at the DNA level reflects Novo Nordisks 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 Nordisk is better known for its strong presence in the diabetes market and for metabolic diseases. However, the company has been increasing its efforts in hemophilia, with its hemophilia A drug Esperoct receiving approval from both the U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) this year.

Hemophilia A is found in about one in 5,000 people and hemophilia B in about one in 25,000 male births. It is estimated that more than 400,000 males have hemophilia A or B, which is severely underdiagnosed in developing countries. About 304,000 people are diagnosed with hemophilia A, the result of decreased or defective production of the blood clotting factor VIII. Hemophilia B is not as common, but affects about 136,000 people who have deficiencies in clotting factor IX.

Hemophilia patients often have bleeding into the joints, particularly knees and ankles, and can have uncontrolled bleeding from trauma, surgery, tooth extractions or other minor surgical treatments.

Bluebird bio is a pioneer of gene therapy. On June 3, the European Commission (EC) granted the company conditional marketing approval for its LentiGlobin gene therapy for transfusion-dependent beta-thalassemia (TDT) under the brand name Zynteglo. It was approved for patients 12 years or older with transfusion-dependent beta-thalassemia who did not have a 0/0 genotype and for patients where hematopoietic stem cell (HSC) transplantation wasnt appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor isnt available.

The therapy came with a $1.8 million price tag in Europe, although it has offered a variety of pricing schemes, including a five-year payment plan with annual payments contingent on the therapys continued effectiveness, to offset criticism of the price.

Of the deal with Novo Nordisk, Philip Gregory, bluebirds chief scientific officer, stated, 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 Nordisks 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.

Excerpt from:
Novo Nordisk and bluebird bio Enter 3-Year Gene Therapy Collaboration Pact - BioSpace