HEALTH & SCIENCE
ZakGT Science
The CLIMB-SCD-121 trial's 18-month follow-up data, published in the New England Journal of Medicine, confirms that all 12 patients treated with the CTX001 CRISPR base-editing therapy have maintained complete resolution of vaso-occlusive crises โ the debilitating pain episodes that define severe sickle cell disease. Hematologists not involved in the study describe the results as "the most compelling gene therapy outcome published in any blood disorder to date," while noting that 18-month follow-up cannot yet establish whether the benefit is permanent.
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