Editas Medicine's CRISPR-Cas12a Therapy Achieves Functional Cure in 27 of 28 Sickle Cell Patients as RUBY Trial Results Land in NEJM
The RUBY trial of renizgamglogene autogedtemcel (reni-cel), a one-time CRISPR-Cas12a gene-editing therapy, showed that 27 of 28 patients with severe sickle cell disease had no painful crises after treatment, with hemoglobin levels normalizing to 13.8 g/dL by six months.
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