Evinacumab, a recombinant monoclonal antibody against human ANGPTL3, has been recently approved by the FDA for the treatment of homozygous familial hypercholesterolemia (HoFH), and other ANGPTL3 inhibitors (including antisense oligonucleotide, ASO) are currently at different stages of clinical trials. The gene discussed is ANGPTL3; the disease is homozygous familial hypercholesterolemia.