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.
Precision is also using its ARCUS platform to develop a treatment to edit PCSK9, a gene that regulates the levels of low-density lipoprotein (LDL) cholesterol. In preclinical studies, and three ...
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.
The company has launched a competition to offer grants of up to $100,000 to multiple projects that can uncover "new insights about the role of the PCSK9 protein/gene in health and disease", with a ...