Researchers developed a gene-editing technology that reduces 'bad' cholesterol

CRISPR was used to edit genes in trial participants' livers.

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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. This treatment was tested in individuals with hereditary conditions that made them susceptible to developing clogged arteries and heart attacks. Scientists were able to use CRISPR editing techniques to tweak liver gene cells. The researchers “turned off” a cholesterol-raising gene called PCSK9, which is found in the liver, in order to lower LDL-C — sometimes called "bad" cholesterol — which causes plaque to build up in arteries in the first place.

PCSK9 was lowered by as much as 84 percent in the cohorts that received higher infusion rates of the treatment. At those higher treatment doses, Verve scientists said that the reduction of those LDL-C-related proteins lasted 2.5 years in previous studies on primates.

VERVE-101 explained visual.
VERVE-101 explained visual. (Verve Therapeutics)

From a clinical standpoint, this gene editing therapy has the potential to disrupt the current standard treatment for high cholesterol. The current go-to's include prescription statins and PCSK9 inhibitors, but they require strict adherence and can have bad side effects like muscle pain and memory loss.

CRISPR, while seemingly miraculous, is a long way from replacing daily medications though. According to Nature, two of the 10 participants in the study suffered from a “cardiovascular event” that coincided with the infusion. Verve says one was not related to the treatment at all and the second was “potentially related to treatment due to proximity to dosing.” The use of a gene-editing technology will always carry some risk because the edits could occur elsewhere in the genome.

Before a single infusion therapy for high cholesterol can reach consumers, the FDA mandates that the treatment will need to be studied for up to 15 years. Verve recently received FDA clearance for an Investigational New Drug Application for VERVE-101, meaning that the company can begin to conduct trials in the US. The current trials in New Zealand and the United Kingdom will look for willing clinical trial participants to expand the study.

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