Phase 3 study of in vivo CRISPR therapy for hereditary angioedema successfully completed
Tamara Ceaikovski
Amsterdam University Medical Center Jun 13 2026 Researchers from Amsterdam UMC, in collaboration with other hospitals, have successfully completed the first-ever Phase 3 study of an in vivo CRISPR therapy. In this large-scale, double-blind Phase 3 trial, 80 patients with hereditary angioedema were randomized to receive either the CRISPR therapy or a placebo. CRISPR therapy is a medical technique that allows doctors to precisely modify errors in cellular DNA to treat specific hereditary diseases.
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