A groundbreaking gene therapy, VERVE-101, developed by Verve Therapeutics, holds promise in reducing ‘bad’ cholesterol levels by up to 50% with a single dose. The therapy utilizes CRISPR-based gene editing to target the PCSK9 gene, a key regulator of LDL cholesterol in the liver. Andrew M. Bellinger, Chief Scientific Officer at Verve Therapeutics, highlights the transformative potential of this one-time treatment, offering a compelling alternative to daily medication.
In a recent human trial presented at the American Heart Association’s Scientific Sessions 2023, participants diagnosed with heterozygous familial hypercholesterolemia received a single infusion of VERVE-101. Despite pre-existing severe coronary artery disease, the therapy demonstrated significant reductions in LDL-C, with the highest dose achieving a remarkable 55% decrease. The study, conducted in New Zealand and the UK, showcased promising results, emphasizing the potential for sustained LDL-C lowering over the long term.
Importantly, adverse events were generally mild, and serious cardiovascular events occurred in individuals with advanced coronary artery disease. As the study continues its follow-up, VERVE-101 emerges as a groundbreaking advancement in addressing the global health challenge of elevated LDL cholesterol, offering hope for a transformative shift in cholesterol management.
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