TL;DR
Some people are born with a broken copy of a liver gene called ANGPTL3, and they go through life with unusually low blood fats. CRISPR Therapeutics is trying to hand that luck to everyone else in one sitting. Its gene editor, CTX310, is a single IV infusion that permanently switches the gene off in the liver, and on Nov. 9 the company reported that it cut average triglycerides, a type of blood fat, by 63% within 30 days in 10 people whose levels were several times the healthy range. No one had a serious side effect tied to the drug, and the drop held in the seven people followed for at least 90 days. These are company-reported results from a small, open-label trial, and they follow an earlier group, reported in August, that showed its cuts lasting a full year. The bigger idea is a heart-risk medicine taken once instead of every day for life. On Monday we covered a one-shot gene treatment that held cholesterol down for two years in monkeys (our story); this is the same lucky-gene idea, tested in people. To see our odds on when a one-time edit like this reaches patients, and the timeline of breakthroughs that leads there, subscribe.
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