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Companies Developing Hemophilia Gene Editing Therapy

Mountain bluebird

Mountain bluebird (A. Kotok)

10 October 2019. A partnership between drug maker Novo Nordisk and biotechnology company bluebird bio is designing a treatment for hemophilia with gene-editing proteins. Financial and intellectual property details of the three-year agreement between Novo Nordisk in Bagsvaerd, Denmark and bluebird bio in Cambridge, Massachusetts — the company spells its name in all lower-case letters — were not disclosed.

Hemophilia is an inherited disorder where blood does not clot adequately, resulting in spontaneous or uncontrolled bleeding. In hemophilia, a genetic defect fails to produce proteins needed to mix with platelets allowing blood to coagulate. About 8 in 10 people with the condition have hemophilia type A, where the protein clotting factor 8 is missing. In type B, clotting factor 9 is missing.

bluebird bio develops gene therapies for cancer and genetic disorders, including sickle cell disease, beta-thalassemia, and cerebral adrenoleukodystrophy, a rare metabolic disease causing learning disabilities and behavioral problems in young children. The company developed a type of gene editing with synthetic proteins known as mega-TALs, based on messenger RNA. These proteins combine homing endonucleases, enzymes that cut DNA strands at specific sequence locations, with other enzymes called transcription activator-like or TAL effectors that bind to the targeted DNA sequence. bluebird says mega-TALs are compact and precise gene editing tools.

In the collaboration, bluebird and Novo Nordisk will start with gene editing techniques for correcting clotting factor 8 deficiencies in hemophilia A that would eliminate the need for factor-replacement therapies. However, the partnership is expected to expand to other genetic disorders. Novo Nordisk is a maker of synthetic clotting factor proteins for factor-replacement treatments. The company’s pipeline also has a targeted synthetic antibody in a mid-stage clinical trial to treat hemophilia A and B, as well as a therapy for sickle cell disease in an early-stage trial.

“We believe this technology has the potential to create a highly differentiated approach to the treatment of many severe genetic diseases,” says Philip Gregory, bluebird bio’s chief scientist in a joint statement. Gregory adds, “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 8 deficiency.”

Novo Nordisk and bluebird bio are not alone in developing gene therapies for hemophilia. As reported by Science & Enterprise, Spark Therapeutics reported last year on findings from a clinical trial showing long-term effects of its gene therapy for hemophilia B, and Sangamo Therapeutics earlier received an orphan drug designation for its gene-editing treatment for hemophilia B, now in clinical trials.

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