New One-Shot Gene Editing Therapy May Reduce Bad Cholesterol By 62%
A single infusion cut PCSK9 by up to 88% and LDL cholesterol by up to 62%, with no treatment-related serious adverse events reported.
- 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.
14 Articles
14 Articles
New One-Shot Gene Editing Therapy May Reduce Bad Cholesterol By 62%
A groundbreaking gene-editing therapy developed by Eli Lilly and Verve Therapeutics reduced "bad" LDL cholesterol by up to 62% after a single infusion in an early trial, raising hopes for a long-lasting treatment to reduce heart attack risk
Could Gene Therapy Become The ‘Ozempic’ For Bad Cholesterol? 6 Things To Know About Eli Lilly’s Breakthrough Research
Eli Lilly and Verve Therapeutics’ experimental gene-editing therapy VERVE-102 is showing promising results in lowering bad cholesterol with a single dose.
One-time gene editing cuts LDL cholesterol in early hypercholesterolemia trial
A phase 1 NEJM interim analysis found that a single infusion of the investigational base-editing therapy VERVE-102 produced dose-dependent reductions in circulating PCSK9 protein and LDL cholesterol in adults with heterozygous familial hypercholesterolemia or premature coronary artery disease.
So Far, So Good for PCSK9-Targeting Gene Therapy in Hypercholesterolemia
(MedPage Today) -- An investigational gene therapy passed muster for reducing PCSK9 and LDL cholesterol levels, with reassuring safety, among patients with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease ...
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