A multicenter clinical program led by University College London and Great Ormond Street Hospital reported that zorevunersen, a gene‑regulation therapeutic for Dravet syndrome, produced large reductions in seizure frequency and improved daily functioning in pediatric patients. In an 81‑patient cohort aged 2–18, seizure counts fell between 59% and 91% over follow‑up; most adverse events were mild to moderate. Results published in the New England Journal of Medicine and accompanying first‑in‑human gene regulation reports indicate the approach may address the SCN1A loss‑of‑function mechanism that underlies most Dravet cases. A randomized phase 3 trial is underway to confirm efficacy and safety.