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 in earlier primate studies.
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 , 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.
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.