CRISPR Therapy Raises Fetal Hemoglobin in Sickle Cell Disease - Takeaways - MDSpire

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

  • April 13, 2026

  • 2 min

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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.

Original Source(s)

CRISPR Therapy Raises Fetal Hemoglobin in Sickle Cell Disease

  • April 13, 2026

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