CRISPR Therapy Raises Fetal Hemoglobin in Sickle Cell Disease
An investigational Cas12a-edited stem cell treatment sharply boosted HbF and largely eliminated vaso-occlusive crises in an early trial
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CRISPR therapy renizgamglogene autogedtemcel effectively raised fetal hemoglobin in patients with severe sickle cell disease.
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In a phase 1-2 study, total hemoglobin increased from 9.8 g/dL to 13.8 g/dL within six months after treatment.
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Fetal hemoglobin levels rose significantly from 2.5% to 48.1%, with F-cell levels exceeding 99% by month six.
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Twenty-seven out of 28 patients experienced no severe vaso-occlusive events post-infusion, indicating clinical effectiveness.
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The therapy demonstrated robust engraftment, with neutrophil and platelet recovery occurring within 23 and 25 days, respectively.
