Researchers discover single-infusion treatment to slash cholesterol levels
Jakarta, CNBC Indonesia - Researchers have discovered a new method to ‘eliminate’ cholesterol, a common condition in modern society, using experimental gene-editing therapy called VERVE-102.
This approach reportedly reduces LDL (‘bad’) cholesterol by up to 62% with a single infusion, based on preliminary Phase 1 clinical trial results.
Preliminary findings published in the medical journal The New England Journal of Medicine indicate the therapy is safe for the 35 participants in the trial, with no serious side effects reported, even among those receiving the highest dose.
Beyond safety, initial data shows promising effectiveness. In the highest-dose group, average LDL levels fell by 62% to 78 mg per decilitre.
If the effect persists long-term, this reduction could potentially cut the risk of heart and vascular diseases caused by plaque buildup by up to 50%.
According to Arstechnica, VERVE-102 works using mRNA-based gene-editing technology. The therapy targets the PCSK9 gene, which regulates LDL cholesterol levels in the blood.
Unlike cholesterol-lowering drugs like statins, which require regular dosing, this treatment is designed to permanently disable the PCSK9 gene, stopping the body from producing the protein that drives high LDL.
During the trial, researchers administered doses ranging from 0.3 mg per kilogram of body weight up to 1 mg/kg.
Results showed a consistent pattern: higher doses led to greater cholesterol reduction. At the highest dose, PCSK9 levels dropped by 88% and LDL by 62%.
Trial participants were patients with early-onset cardiovascular disease or those with genetically inherited high cholesterol.
VERVE-102 was developed by Verve Therapeutics, a biotech company acquired by Eli Lilly last year in a $1.3 billion transaction.
One of Verve’s co-founders, now Eli Lilly’s Senior Vice President Sekar Kathiresan, hopes the therapy could provide an alternative for high-cholesterol patients who no longer need lifelong medication like statins.
Cardiologist Riyaz Patel also welcomed the preliminary data, stating VERVE-102 has potential as a new approach to significantly and durably lower LDL with just one treatment.
However, researchers emphasised the data is still very early stage. Further trials with larger participant numbers and longer monitoring periods are needed to confirm safety and effectiveness. If results remain positive, VERVE-102 has already received Fast Track status from the US Food and Drug Administration.