Correctsequence Therapeutics Co., Ltd

This early-stage trial tests a single infusion of CS-121, a gene editing therapy, in 15 adults with familial chylomicronemia syndrome (FCS), a rare condition causing extremely high blood fat levels. The therapy targets the APOC3 gene to lower triglycerides and reduce disease burd…

This early-phase trial is testing a new treatment called CS-101 for severe sickle cell disease. It uses a person's own blood stem cells, which are gene-edited in a lab to correct the disease, then given back as a one-time infusion. The study will enroll 5 people aged 12 to 35 and…