CAN104

CAN104 is a recombinant human enzyme replacement therapy (ERT) targeting alpha-galactosidase A (GLA) for the treatment of Fabry disease. Fabry disease is a rare X-linked lysosomal storage disorder caused by a deficiency in the GLA enzyme, which leads to the systemic accumulation of glycosphingolipids, particularly globotriaosylceramide (Gb3), in various tissues including the heart, kidneys, and nervous system. CAN104 is designed to supplement the missing or deficient enzyme, thereby facilitating the breakdown of accumulated substrates and potentially preventing or mitigating organ damage. The drug is being developed by CANbridge Pharmaceuticals (also known as Beihai Kangcheng) and is currently in early-stage clinical development.