World's First Phase 3 Trial of In Vivo CRISPR Therapy Successfully

The world's first Phase 3 trial of an in vivo CRISPR therapy marks a milestone in genetic medicine. Conducted by Amsterdam University Medical Center (Amsterdam UMC) in collaboration with multiple international hospitals, the study tested lonvoguran-ziclumeran, a CRISPR-Cas9–based treatment delivered through a single intravenous infusion. The trial enrolled eighty patients with hereditary angioedema (HAE), a rare genetic disorder characterized by episodic, potentially life-threatening swelling. As the first Phase 3 effort of its kind to edit DNA directly inside the patient, the study used a rigorous randomized, double-blind, placebo-controlled design to assess safety and efficacy.