CRISPR Advances: Gene Therapy Significantly Lowers Cholesterol Levels

Recent studies utilizing CRISPR technology have demonstrated significant reductions in cholesterol levels, potentially revolutionizing treatments for cardiovascular health. A study led by Dr. Steven Nissen at the Cleveland Clinic involved 15 patients with severe cholesterol issues. According to Slashdot, the preliminary results showed nearly a 50% reduction in low-density lipoprotein, or LDL, often referred to as 'bad' cholesterol. Additionally, triglyceride levels dropped by an average of 55% among the participants. This research, presented at the American Heart Association Scientific Sessions, suggests that CRISPR-Cas9 gene editing may offer a 'one and done' solution for patients struggling with high cholesterol, particularly those with genetic predispositions to severe forms of the disease.

The study specifically targeted the ANGPTL3 gene, which is linked to cholesterol metabolism. Individuals with a nonfunctioning ANGPTL3 gene, affecting approximately 1 in 250 people in the U.S., naturally maintain low levels of LDL cholesterol and triglycerides without adverse health effects. Dr. Nissen expressed hope that this gene therapy could provide a permanent solution for younger patients, allowing them to manage their cholesterol levels effectively for the rest of their lives. As highlighted by The Straits Times, the gene therapy, named CTX310, demonstrated safety and efficacy in a small group of participants who had not responded well to traditional treatments.

Patients involved in the study received a single infusion that worked by switching off the ANGPTL3 gene. Among the four patients who received the highest dose, reductions in LDL and triglycerides were observed just two weeks post-treatment, and these levels remained low for at least two months. Dr. Luke Laffin, co-leader of the study, noted that this gene therapy could be practice-changing, offering an alternative to daily medications or frequent injections. Unlike Regeneron's Evkeeza, which requires monthly infusions for a rare genetic disorder, the CRISPR therapy could represent a more straightforward approach to managing high cholesterol, potentially making it accessible to millions of Americans.

Crispr Therapeutics plans to advance the therapy into Phase 2 clinical trials, with the hope of gaining regulatory approval in the coming years. The company aims to initially target patients with genetic forms of high cholesterol but hopes to expand to a broader population if the therapy proves effective. The cost of the treatment remains uncertain, but estimates suggest it may be less than $100,000, significantly lower than some existing gene therapies that can exceed $2 million. Overall, these developments underscore the potential of gene editing technologies like CRISPR in addressing common health issues, particularly in cardiovascular medicine.