What if lowering cholesterol didn’t require taking a pill every day? In a Phase 1a trial, a single dose of CRISPR-Cas9 gene editing therapy CTX310 produced sustained reductions in ANGPTL3 and atherogenic lipids that persisted for up to 1 year. No daily medication. No repeated injections. One-time gene editing. The concept is striking: instead of continuously treating a pathway involved in lipid metabolism, CRISPR aims to permanently modify the underlying biology. And importantly, the early safety signal was encouraging—there were no treatment-related dose-limiting toxicities reported in this initial study. But this is still early clinical development. Phase 1 studies are primarily designed to evaluate safety and establish whether the biological effect is achievable. Larger, longer studies will be needed to determine durability, cardiovascular benefit, and long-term safety. Still, this represents a fascinating shift in medicine: From treating disease repeatedly → to potentially editing the biology that drives it. The era of one-time genetic medicines may be getting closer. 🧬 nejm.org/doi/full/10.1056/NE…