CS-121

CS-121 is an in vivo base editing therapy designed for the treatment of hyperchylomicronemia, including Familial Chylomicronemia Syndrome (FCS). Developed by CorrectSequence Therapeutics in collaboration with Shanghai Jiao Tong University School of Medicine, the therapy utilizes lipid nanoparticles (LNPs) to deliver base editing components specifically to hepatocytes. CS-121 targets the APOC3 gene, which encodes Apolipoprotein C-III, a critical regulator of triglyceride metabolism that inhibits lipoprotein lipase. By permanently disrupting the APOC3 gene through base editing, CS-121 aims to reduce Apolipoprotein C-III levels, thereby enhancing the clearance of triglyceride-rich lipoproteins and lowering serum triglyceride levels. It is currently being evaluated in a Phase 1 clinical trial focusing on children and adolescents.