Conference Roundup

Gene editing reduces cholesterol levels in monkeys

Posted on

The use of base editing technology to turn of a gene in monkeys, safely edited the primate genome, resulting in significantly lower blood LDL cholesterol and triglycerides, according to a presentation by Sekar Kathiresan, co-founder and CEO of Verve Therapeutics, at the International Society for Stem Cell Research (ISSCR) 2020 Virtual Annual Meeting.

The animals’ blood levels of LDL cholesterol were reduced by 59% and triglycerides by 64%.

“At Verve, our goal is to develop medicines, given once in life, that precisely edit targeted genes in the liver to permanently reduce LDL cholesterol and triglyceride levels in adults with coronary heart disease, the leading cause of death in the U.S. and worldwide,” said Dr Kathiresan, in a press release. “These proof-of-concept data, which to the best of our knowledge represent the first successful application of the base editing technology in non-human primates, show that we can safely edit the primate genome at highly efficacious levels to significantly lower blood LDL cholesterol and triglycerides. The findings are very encouraging and add to our growing body of evidence in using both base editing and CRISPR-Cas9 in vivo against various gene targets. We expect to choose a lead program by year-end 2020 with the goal of initiating human clinical studies within the next three years.”

“These data are exciting and demonstrate our ability to turn off PCSK9 or ANGPTL3 in the liver to safely and effectively lower LDL cholesterol and triglyceride levels in non-human primate models using adenine base editing,” said Andrew Bellinger, MD, PhD, chief scientific officer of Verve Therapeutics in the press release. “Very importantly, we do not find evidence of off-target editing using adenine base editing with carefully selected guide RNAs. These findings support Verve’s transformative idea to develop once-and-done gene-editing treatments for adults with coronary heart disease and we look forward to presenting additional data in the future.”

Read the full press release here.