New One-Shot Gene Editing Therapy May Reduce Bad Cholesterol By 62%
A single infusion cut LDL cholesterol by up to 62% and showed no dose-limiting toxicities, though researchers said longer follow-up is needed.
- A landmark study published in the New England Journal of Medicine revealed that an experimental gene-editing therapy successfully lowered harmful LDL cholesterol by up to 62% in high-risk patients.
- The one-time intravenous treatment, known as VERVE-102 and developed by Eli Lilly and Verve Therapeutics, uses precise base-editing technology to permanently deactivate the PCSK9 gene in the liver.
- Data from the Phase 1b clinical trial involving 35 adults showed that the treatment was generally well-tolerated and that its cholesterol-lowering effects remained durable for up to 18 months in some participants.
- While cardiologists view this "one-and-done" approach as a historic breakthrough for people with inherited high cholesterol, experts caution that larger, long-term trials are still required to fully verify its permanent safety profile.
23 Articles
23 Articles
A One-Time Experimental Treatment Might Control Cholesterol for Life
—Ruslanas Baranauskas/Science Photo Library—Getty ImagesHeart disease kills more people worldwide than any other malady, and even though doctors use cholesterol-lowering medications and recommend lifestyle habits to control major risk factors. Making these changes, however, is not easy for people, and sticking with them is even harder. The hope is that one day soon, people may not have to rely solely on these interventions. In a study conducted …
SCIENCE & TECH: ‘Revolutionary’ single-dose treatment cuts cholesterol by 62%
One and done? Pharma giant Eli Lilly announced Monday that its gene-editing therapy can reduce LDL (“bad”) cholesterol by up to 62% with a single infusion. Not only was the experimental treatment successful, but the results were still apparent in some participants 18 months after they received the dose. VERVE-102 may be a revolutionary treatment for bad cholesterol before it leads to heart disease. wutzkoh – stock.adobe.com While most gene thera…
Experimental One-Time Gene Editing Treatment VERVE-102 Lowers LDL Cholesterol by Up to 62% in Early Trial
NEW YORK CITY (VINnews) – A single intravenous infusion of an experimental gene-editing treatment has produced substantial and durable reductions in “bad” LDL cholesterol levels in patients with high cardiovascular risk, according to interim results from a Phase 1b clinical trial. VERVE-102, developed by Verve Therapeutics and now associated with Eli Lilly, uses in vivo […]
Largest Rare Variant–Lipid Association Study Finds Heart Disease Targets
A landmark week for cardiovascular genetics opened with huge news from the gene-editing field: Eli Lilly’s experimental therapy VERVE-102, a single-dose PCSK9 base editor designed for patients with heterozygous familial hypercholesterolemia (FH) and premature coronary artery disease (CAD), demonstrated durable cholesterol-lowering effects in early studies. While the result marks one of the strongest signs yet that in vivo gene editing could beco…
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