In a trial run by Verve Therapeutics, a Cambridge–based biotech company, researchers discovered that a single infusion of a gene-editing treatment called VERVE-101 was able to reduce cholesterol levels in patients with hereditary conditions. By using gene-editing techniques to tweak liver gene cells, scientists lowered PCSK9, a cholesterol-raising gene found in the liver, leading to lowered levels of “bad” cholesterol which causes plaque to build up in arteries.
At higher doses, the reduction of LDL-C-related proteins lasted in previous studies on primates. However, two out of 10 participants suffered from a cardiovascular event that may have been related to the treatment, highlighting the potential risks of gene-editing technology.
While this gene editing therapy has the potential to disrupt the current standard treatment for high cholesterol, it is a long way from replacing daily medications. Regulatory approval and extensive studies will be required before the treatment can reach consumers. Trials are currently being conducted in New Zealand and the United Kingdom, and Verve recently received FDA clearance to conduct trials in the US.