Drug development for sickle-cell disease, largely neglected for decades, is becoming a crowded field: two papers published on Saturday in the New England Journal of Medicine give promising results from a study of experimental therapies, including Crisper gene editing for the disease.
In addition, Beam Therapeutics Inc. The American Society of Hematology presents lab and mouse data at its annual meeting on Saturday to support the safety of other approaches to using chrysoprine gene acquisition for sickle-cell disease. The company said it hopes to open the trial next year.
More than a dozen companies are competing to develop experimental treatments for sickle-cell disease, a legacy of anemia that affects 100,000 predominantly black Americans.
The surge of interest led to the long-running question: What good is a new treatment for a disease if many patients with the disease are unable or unwilling to get admission?
In the papers of the New England Journal of Medicine, two partners in developing Crisper gene-editing therapy – Crisper Therapeutics and Vertex Pharmaceuticals Inc. Reports that a patient with sickle-cell disease and another inherited blood disorder, transfusion-dependent beta thalassemia, did not need a blood transfusion even after every acquired cell and more than a year. They said they have treated a total of 10 patients with beta thalassemia today through Cecil-Cell disease or crystroph therapy.
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