Gene Editing Therapy Shows Success Against Severe Sickle Cell Disease

New results from a clinical trial show promising outcomes for a gene-edited treatment for severe sickle cell disease, a genetic blood disorder with few curative options.

Conducted as part of the multicenter RUBY Trial, researchers published their latest findings in the New England Journal of Medicine. Remarkably, 27 out of 28 patients did not have any painful sickle cell crises after treatment, achieving what physicians call a "functional cure."