Correctsequence Therapeutics Co., Ltd

This early-stage study tests CS-121, a one-time gene-editing treatment given by IV, for people with familial chylomicronemia syndrome (FCS) – a rare condition causing extremely high blood fats and risk of pancreatitis. About 15 adults aged 18-55 will receive the therapy to see if…

This early-stage study tests a single injection of CS-101, made from a patient's own gene-edited blood stem cells, for severe sickle cell disease. The goal is to see if it is safe and can reduce or eliminate painful vaso-occlusive crises. The study involves 5 participants aged 12…