This investigation demonstrates normalization of hemoglobin levels in patients with severe sickle cell disease, suggesting a novel treatment avenue.
Treatment with reni-cel led to normalization of the total hemoglobin level and an increase in the percentage of fetal hemoglobin, with no vaso-occlusive events occurring in 27 of 28 patients after infusion. These results support further investigation of this gene-editing approach in the treatment of severe sickle cell disease. (Funded by Editas Medicine; RUBY ClinicalTrials.gov number, NCT04853576.).
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Hanna et al. (2026) studied this question.
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