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.
AstraZeneca has reported the first clinical data with its oral PCSK9 inhibitor AZD0780, which it hopes could offer an alternative to current injectable drugs used to lower cholesterol. The new ...
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.