# CRISPR gene-editing therapy for cholesterol reduction

> Live situation record from CLSTR: https://clstr.news/situations/crispr-gene-editing-therapy-for-cholesterol-reduction
> Updated: 2026-09-11T19:56:00.000Z. Sources: 13. Developments: 3.

Researchers have reported successful results from clinical trials involving CRISPR-Cas9 gene-editing therapy designed to reduce cholesterol levels. 

Initial pilot study data involving 15 patients indicated that a single-dose infusion could lead to an approximately 50% reduction in LDL cholesterol and a substantial decrease in triglycerides. These reductions were observed to persist for more than one year without significant adverse effects. 

Subsequent details regarding the experimental one-time infusion therapy, known as CTX310, specify that the treatment works by delivering the CRISPR-Cas9 system to the liver to disable the ANGPTL3 gene. In the highest dosage group, LDL-C levels decreased by 52.5% and triglycerides fell by 47.8% from baseline after one year. No treatment-related serious adverse effects were reported. Participants are scheduled for long-term safety monitoring for a total of 15 years in accordance with FDA recommendations.

Recent trial data from both the United States and China have expanded on these findings. A study by Shanghai Jiao Tong University involving volunteers with familial hypercholesterolemia utilized a modified enzyme derived from the Hafnia paralvei bacterium to target the PCSK9 gene, resulting in a 52% reduction in LDL cholesterol over six months. 

Concurrently, research from the Cleveland Clinic targeting the ANGPTL3 gene corroborated previous findings, with the highest dosage group showing a 52% reduction in LDL cholesterol and a 48% reduction in triglycerides. While some participants reported mild side effects, such as fever and muscle aches, no dangerous adverse effects were observed.

## Timeline

### 2026-09-11: Gene-editing trials show 52% reduction in LDL cholesterol

Gene-editing trials in China and the US have successfully reduced LDL cholesterol by 52% using single-dose injections targeting the PCSK9 and ANGPTL3 genes.

2 sources. https://clstr.news/cluster/gene-editing-trials-show-52-reduction-in-ldl-cholesterol

### 2026-09-09: CRISPR gene-editing therapy reduces bad cholesterol in human trials

A first-in-human trial shows CRISPR-Cas9 gene editing can significantly reduce bad cholesterol and triglycerides in patients with refractory dyslipidemia.

2 sources. https://clstr.news/cluster/crispr-gene-editing-therapy-reduces-bad-cholesterol-in-human-trials

### 2026-08-28: Gene-editing treatment reduces cholesterol by 50% for over one year

A CRISPR-Cas9 gene-editing treatment has demonstrated the ability to reduce LDL cholesterol and triglycerides by approximately 50% for over a year in a small pilot study.

9 sources. https://clstr.news/cluster/cleveland-clinic-researchers-report-durable-cholesterol-reduction-via-gene-editing

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Cite as: CRISPR gene-editing therapy for cholesterol reduction. CLSTR, https://clstr.news/situations/crispr-gene-editing-therapy-for-cholesterol-reduction