CTX310

CTX310 is an investigational in vivo CRISPR/Cas9 gene editing therapy developed by CRISPR Therapeutics. It is designed to permanently knock out hepatic expression of angiopoietin-like protein 3 (ANGPTL3), a key regulator of lipid metabolism. The therapy consists of Cas9 mRNA and guide RNA (gRNA) targeting ANGPTL3, both encapsulated in lipid nanoparticles for intravenous administration. By disrupting ANGPTL3 expression in the liver, CTX310 aims to significantly reduce serum triglycerides and low-density lipoprotein cholesterol (LDL-C). Early clinical data show dose-dependent reductions in triglycerides and LDL-C, with up to 82% reduction in triglycerides and 81% reduction in LDL-C at higher doses. The drug has demonstrated a favorable safety profile with no severe adverse events reported so far. Primary indications under investigation include mixed dyslipidemia, severe hypertriglyceridemia, homozygous familial hypercholesterolemia (HoFH), heterozygous familial hypercholesterolemia (HeFH), and related cardiovascular risk conditions[2][4][5][6][9].

Brand names

CTX310CTX-310CTX 310

Other names

CTX310CTX-310CTX 310