First U.S. CRISPR trial nearly 100% effective three years later

Incredible new data presented at the European Hematology Association Congress shows that an experimental CRISPR gene-editing therapy remains safe and effective three years after treatment. The results come from human trials using CRISPR technology to treat two rare inherited blood disorders, one of the longest such trials. The human trial, which began in 2019, is the first in the United States. The trial targets two rare blood disorders: beta-thalassemia and sickle cell disease. The therapy starts by collecting stem cells from the patient’s blood. A single genetic change was then made using CRISPR technology aimed at increasing levels of fetal heme in red blood cells. These stem cells are then reinjected into the patient. Preliminary results are very promising. The first two patients treated were largely cured within a few months, but questions about long-term efficacy persist. Follow-up announcements from last year continued the impressive results, with 22 patients treated, all showing 100% success. Seven of these patients showed no reduction in efficacy 12 months after initial treatment. Now a new data release provides results from 75 patients treated with this breakthrough CRISPR therapy, now known as exa-cel (exagamglogene autotemcel). Of the 75 treated patients, 44 had transfusion-dependent beta-thalassemia (TDT) and 31 had severe sickle cell disease (SCD). All but two of the 44 TDT patients were largely cured of their disease and no longer required blood transfusions. The two TDT patients who still required blood transfusions had 75% and 89% less blood transfusions, respectively. All 31 SCD patients were also free of disease symptoms at subsequent long-term follow-up.

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