CRISPR Therapeutics has revealed promising but early data from its first clinical study of a gene editing therapy to lower the risk of heart disease, setting the stage for competition with other drugmakers working on similar treatments. The therapy, dubbed CTX310, uses lipid nanoparticles (LNPs) to deliver the CRISPR therapy
into the liver, where it cuts a gene called ANGPTL3. The approach aims to mimic the effects of natural mutations that cause some people to have unusually low levels of cholesterol and triglycerides. The Phase 1 trial tested four dose levels in 10 people, but the most dramatic results came from the sole patient who got the highest dose, which reduced their triglyceride levels by 82% and low-density lipoprotein (LDL) — often called “bad" cholesterol — by 65% a month after the infusion. |