CTX320

CTX320 is an investigational in vivo CRISPR/Cas9 gene-editing therapy developed to reduce elevated levels of lipoprotein(a) [Lp(a)], a genetically determined risk factor for atherosclerotic cardiovascular disease and aortic valve stenosis. The therapy uses lipid nanoparticle (LNP) delivery of Cas9 mRNA and guide RNA (gRNA) to the liver, where it targets and knocks out the apolipoprotein(a) component of Lp(a), leading to reduced hepatic expression and plasma levels of Lp(a). Preclinical studies have shown potent, dose-dependent editing in human hepatocytes and non-human primates, resulting in significant reductions in plasma Lp(a). Early clinical trials are ongoing to assess safety, tolerability, efficacy, and durability of effect[1][3][6].