Signal CRISPR Clinical Trials 2026: In Vivo Delivery as the Single Most Important Transition

Summary

FDA-approved Casgevy for sickle cell disease and beta-thalassaemia has spread to broad approval across the United States, the European Union and the United Kingdom. CTX310, reported in the New England Journal of Medicine in November 2025, reduced ANGPTL3 levels and lowered LDL by about 50% from a single CRISPR-based dose. Researchers identify in vivo delivery via lipid nanoparticles and viral vectors as the single most important technical transition. Late-stage trials are now open in high cholesterol, inherited blindness, terminal cancer and rare genetic disease.