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Vertex, Crispr Gene-Editing Treatment Shows Lasting Impact

June 11, 2022, 7:00 AM

Vertex Pharmaceuticals Inc. and Crispr Therapeutics AG said their gene editing treatment for two rare blood disorders delivered potentially long-lasting benefits in a study.

The treatment, called exa-cel, aims to correct genetic mutations that cause beta thalassemia and sickle cell disease, which can cause painful symptoms and early death. None of 31 sickle cell patients who received the treatment, also called CTX001, experienced a painful symptom that occurs when tissues lose blood flow, the companies said at a scientific meeting. All but two of 42 patients beta thalassemia patients no longer needed blood transfusions following the therapy.

The findings, presented ...