CRISPR Geneโ Editing Considerably Lowers Cholesterol in First Human Trial
New Orleans, LA – In a landmark achievement for gene editing, a single dose of CRISPR-Cas9 therapy has demonstrated a significant โand sustained reduction in LDL cholesterol levelsโฃ in patients with a genetic predispositionโค to high cholesterol, according to results presented saturday at the American Heart Association’s annual scientific meeting andโ concurrently published in the New England Journal of Medicine. The trialโฃ marks โขthe first time CRISPR gene editing has โขbeen used to directly alter a gene inside the human body to treat a disease.
The experimental therapy, developed by Verve Therapeutics, targets the PCSK9 gene, whichโ regulates cholesterol โlevels. By โคdisabling this gene,the treatment aims to mimic the effects of naturally occurring genetic mutations that confer โlifelong protection against heart disease. โฃThe findings offer aโฃ potential one-time curative โคapproach for millions burdenedโฃ by high cholesterol and at risk of cardiovascular events, movingโข beyond the need โคfor lifelong medication.
The Phase 1 trial involved sixโ patients with heterozygous familial โhypercholesterolemia,โ a common genetic condition causing dangerouslyโฃ high LDL cholesterol despite standard treatments like statins. Participants receivedโ varying doses of the CRISPR therapy via intravenous infusion. Results showedโฃ a dose-dependent reduction โin PCSK9 protein levels, leading to a corresponding decrease in LDL cholesterol.
The highest dose tested resulted in a 55% reduction in LDL cholesterol โlevels after six months, with โขlevels remaining significantly lower than baseline. Researchers reported no serious adverseโ events related to theโ treatment, though continued โคmonitoringโฃ is underway to assess long-term safety and efficacy.
“Thisโ is a watershed moment for theโฃ field of gene editing,” saidโข Dr. โSekar Kathiresan, CEO of Verve Therapeutics and lead author of the study. “To beโ able to permanently lower cholesterolโ with a single treatment, rather than relying on daily pills, has the potential to transform the prevention of heart disease.”
Familial hypercholesterolemia affects an estimated 1 in 250 people โworldwide. Even among those without the genetic condition, high LDL cholesterolโฃ is a โฃmajor risk factor for heart attack and stroke, impacting hundreds of millions globally. While statins are effective for many, a significant portion of patients cannot tolerate them or do not โฃachieve adequate cholesterol control.
The success โof this trial paves the way for larger, Phase 2 andโข 3 studies to confirm the findings and โคevaluate the therapy’s impact on cardiovascularโ outcomes. Verve Therapeutics is also exploring the potential of using CRISPR gene editing to target othre genes involvedโ in cardiovascular disease and other genetic conditions.
