Gene editing shows promise in sickle cell disease

Drug development for sickle cell disease, largely overlooked for decades, becomes a busy field: Two papers published Saturday in the New England Journal of Medicine report promising results from studies of experimental therapies, including Crispr gene editing, for the disease .

Additionally, Beam Therapeutics Inc. Lab and mouse data Saturday at the American Society of Hematology annual meeting to support the safety of a different approach to using Crispr gene editing for sickle cell anemia. The company hopes to open a trial next year.

More than a dozen companies are competing to develop experimental treatments for sickle cell disease, an inherited form of anemia that affects 100,000 mostly black Americans.

The great interest has raised a long-standing question: What good is new therapies for a disease if many patients who suffer from it are unable or choose not to access them?

In one of the New England Journal of Medicine articles, two partners in the development of a Crispr gene editing therapy – Crispr Therapeutics and Vertex Pharmaceuticals Inc. that a patient with sickle cell disease and a patient with another inherited blood disease transfusion dependent beta thalassemia each received processed cells and more than a year later they did not require blood transfusions. They said they have treated a total of 10 patients with sickle cell disease or beta thalassemia so far with the Crispr therapy.

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