Genetic and preliminary intervention studies published in two papers in NEJM indicate that angiopoietin-related protein 3 (ANGPTL3) is a promising therapeutic target in patients with dyslipidaemia who are at risk of atherosclerotic cardiovascular disease. Together, the papers show that naturally occurring loss-of-function variants in ANGPTL3 are associated with reduced serum lipid levels and a lower risk of coronary artery disease, and that ANGPTL3 can be safely and effectively inhibited with the use of monoclonal antibodies or antisense oligonucleotides.

Angiopoietin-related proteins are important regulators of lipoprotein metabolism. ANGPTL3 is an endogenous inhibitor of lipoprotein lipase (LPL), which is the main enzyme involved in hydrolysis of triglyceride-rich lipoproteins.