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.
Heart disease is one of the leading causes of death worldwide, and while lifestyle choices like diet, exercise, and smoking ...
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.
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 ...
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 ...
Familial hypercholesterolemia results from gene mutations approximately halving ... decreased transintestinal cholesterol excretion. PCSK9, which mediates post-translational destruction of LDL ...
It is part of a new form of medicine called gene-silencing. Inclisiran does not modify our DNA, but it controls the instructions the body gets. It does this by "silencing" the PCSK9 gene ...