FAQ: CRISPR Gene-Editing Therapy for Cholesterol and Triglyceride Reduction
TL;DR
CTX310's one-time CRISPR therapy offers patients a lasting advantage by eliminating daily medication needs while cutting cholesterol and triglycerides by nearly 50%.
CTX310 uses CRISPR-Cas9 delivered via fat-based particles to edit liver genes and durably turn off ANGPTL3 expression, reducing lipid levels within two weeks.
This one-time treatment could transform cardiovascular care globally by preventing heart disease through sustained cholesterol reduction and improving medication adherence challenges.
A groundbreaking CRISPR therapy achieved unprecedented simultaneous reductions in both LDL cholesterol and triglycerides, potentially revolutionizing lifelong lipid disorder treatment.
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CTX310 is an investigational CRISPR-Cas9 gene-editing therapy delivered as a one-time infusion that targets and turns off the ANGPTL3 gene in the liver, resulting in significant reductions in LDL cholesterol and triglycerides.
The therapy reduced LDL cholesterol by nearly 50% and triglycerides by about 55% on average at the highest dose, with effects beginning within two weeks and sustained for at least 60 days of follow-up.
This is the first therapy to achieve large simultaneous reductions in both LDL cholesterol and triglycerides, representing a major advance for patients with mixed lipid disorders who often have elevations in both.
CTX310 uses tiny fat-based particles to carry the CRISPR editing mechanism into the liver, where it durably switches off the ANGPTL3 gene, mimicking the natural mutations that some people are born with that provide lifelong low cholesterol and triglyceride levels.
The treatment was found to be safe, with three participants experiencing minor infusion-related reactions that resolved with medication, and one participant had a temporary rise in liver enzymes that returned to normal without treatment.
The research was led by Dr. Luke J. Laffin from the Cleveland Clinic and was presented at the American Heart Association's Scientific Sessions 2025 in New Orleans, with simultaneous publication in The New England Journal of Medicine.
If confirmed in larger and longer-term studies, this one-time treatment could transform care for people with lifelong lipid disorders by eliminating the need for daily or monthly cholesterol-lowering medications and dramatically reducing cardiovascular risk.
This was a Phase 1, first-in-human trial involving 15 patients, which is the earliest stage of human testing focused primarily on safety and initial effectiveness.
Unlike current medications that require daily or monthly dosing, this is a one-time treatment that provides durable gene editing effects, potentially offering lifelong benefits from a single infusion.
The therapy requires larger and longer-term studies to confirm these initial findings, and the FDA recommends long-term safety monitoring for up to 15 years for all CRISPR-based therapies due to their durable DNA changes.
Curated from NewMediaWire
