Verve Therapeutics has started dosing patients in a phase 1b trial of its in vivo gene-editing drug for high cholesterol, designed to permanently switch off the PCSK9 gene with a one-shot treatment.
At the moment, there is an LNP-based CRISPR-Cas9 candidate treatment for people with heterozygous familial hypercholesterolemia in clinical trials, which targets the PCSK9 gene in the liver.