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CRISPR Gene Therapy Cuts Cholesterol by 50% in Single Treatment

CRISPR gene therapy CTX310 reduces LDL cholesterol by 50% and triglycerides by 55% in single treatment. Phase 1 trial shows sustained results for 60+ days in patients with lipid disorders.

This breakthrough represents a potential paradigm shift in cardiovascular disease prevention, offering hope for millions of people who struggle with medication adherence or inadequate cholesterol control despite existing treatments. High cholesterol affects over 86 million American adults and is a leading contributor to heart disease and stroke—the world's primary causes of death. Current cholesterol medications require daily or monthly dosing, and many patients stop taking them within the first year, leaving them vulnerable to cardiovascular events. A one-time treatment that provides durable cholesterol reduction could dramatically reduce cardiovascular risk, improve quality of life by eliminating medication burdens, and potentially prevent thousands of heart attacks and strokes annually. For patients with genetic lipid disorders or those who don't respond adequately to conventional therapies, this CRISPR-based approach could offer life-changing protection against cardiovascular disease.