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Twenty-two patients with inherited blood disorders were free of severe pain and the need for transfusions months after receiving an experimental genome-editing medicine, more evidence the CRISPR-based treatment could be a functional cure.

The therapy, jointly developed by Vertex Pharmaceuticals and CRISPR Therapeutics, is designed to treat sickle cell disease and beta-thalassemia by boosting the body’s production of hemoglobin. According to data from two clinical trials presented at the European Hematology Association annual meeting Friday, all patients in both studies have been free from symptoms of the diseases since their treatment and have not needed blood transfusions.


In the beta-thalassemia trial, 15 patients who received the one-time therapy, called CTX001, showed sustained increases in hemoglobin. In the sickle cell disease study, seven patients had increased hemoglobin, and there were no incidences of vaso-occlusive crisis, a bout of extreme pain that can lead to organ damage, stroke, and early death.

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