Lead / Health & Medicine
A year free of severe sickle cell pain crises: base-edited cells delivered that relief for patients in a trial
Beam Therapeutics reports that its experimental cell therapy, risto-cel, helped patients with sickle cell disease go at least 12 consecutive months without severe pain crises, meeting its Phase 1/2 trial’s primary endpoint. The results were independently adjudicated, but the company has not disclosed the proportion who achieved that outcome or detailed safety data.
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