Researchers have created a gene-editing technology that lowers “bad” cholesterol.

Researchers have created a gene-editing technology that lowers “bad” cholesterol.

Malak Saleh

Researchers found that patients ‘ cholesterol levels could be decreased with just one infusion of the gene-editing medication VERVE-101 in a trial conducted by Cambridge-based biotech companyTherapeutics Verve. People with hereditary conditions that made them prone to heart attacks and clogged arteries were tested for this treatment. Researchers were able to modify liver gene cells using CRISPR editing methods. In order to reduce LDL-C, also known as “bad” cholesterol, which is what causes plaque to accumulate in arteries in the first place, the researchers “turned off” a cholesterol-raising gene called PCSK9. This gene is found in liver tissue.

In the cohorts who received higher treatment infusion rates, PCSK9 was reduced by up to 84 percent. According to Verve scientists, the reduction of those LDL-C-related proteins at those higher treatment doses lasted 2.5 years in earlier primate studies.

VERVE-101 explained visual.

Therapeutics Verve

Clinically speaking, this gene editing therapy may change the way that high cholesterol is currently treated. Prescription statins and PCSK9 inhibitors are the current go-tos, but they require strict adherence and may have negative side effects like muscle pain and memory loss.

Despite appearing miraculous, CRISPR is still far from being a replacement for prescription drugs. Two of the study’s ten participants, according to Nature, experienced a” cardiovascular event” at the same time as the infusion. According to Verve, the first had absolutely nothing to do with the treatment, while the second might have had some connection to it because it was close to the dosing. Because the edits may take place elsewhere in the genome, using gene editing technology always carries some risk.

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The FDA requires up to 15 years of study before a single infusion therapy for high cholesterol can be administered to patients. Verve recently received FDA approval for an investigational new drug application for VERVE-101, allowing the company to start US trials. To expand the study, willing clinical trial participants will be sought out for the current trials in New Zealand and the UK.

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