In vivo CRISPR therapy successfully reduces hereditary angioedema attacks in first Phase III trial

Article excerpt

Researchers at Amsterdam UMC have completed the first Phase III trial of an in vivo CRISPR therapy, marking a significant milestone in gene-editing medicine. In the double-blind study, 80 patients with hereditary angioedema, a rare genetic disorder causing unpredictable swelling attacks, received either the CRISPR treatment or placebo. The therapy successfully reduced attack frequency in patients who received the gene-editing intervention. Hereditary angioedema, which can be life-threatening when swelling obstructs airways, has limited treatment options; this trial represents the first large-scale proof that directly editing DNA inside the body can control the condition.