CRISPR gene-editing therapy for cholesterol reduction

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.