On bluebird's heels, CRISPR Therapeutics and Vertex take the air with interim CRISPR blood cell therapy data
Patients with severe forms of sickle cell disease or beta-thalassemia lack enough functioning red blood cells to shuttle ample oxygen through their systems. As a result, they may need recurring blood transfusions throughout their entire lives, on top of a wide range of other serious symptoms.
However, a handful of biotechs are touting one-time treatments that could enable these patients to make more healthy red blood cells and do away with the need for regular transfusions. Among them are CRISPR Therapeutics and Vertex, who at an EHA late-breaking presentation Saturday gave an early glimpse into the longer-term results of their gene editing therapy, known as CTX001 and recently dubbed exa-cel.
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