Gene editing results positive in sickle-cell disease
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Sickle-cell
Drug development for sickle-cell disease, largely overlooked for decades, is becoming a crowded 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.
In addition, Beam Therapeutics Inc. on Saturday presented lab and mouse data at the American Society of Hematology annual meeting to support the safety of another approach to using Crispr gene editing for sickle-cell disease. The company said it 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 mainly Black Americans.
Both sickle-cell disease and beta thalassemia are caused by errors in the gene for hemoglobin, the protein in red blood cells that carries oxygen from the lungs to other parts of the body. People with the diseases inherit two copies of the faulty gene, one from each parent.
