CANbridge Pharmaceuticals Inc. is a global biopharmaceutical company. It is focused on research, development and commercialization of transformative therapies for rare disease. It has a drug portfolio, with three approved drugs and a pipeline of eight assets, targeting rare disease indications that have unmet needs. These include Hunter syndrome and other lysosomal storage disorders, paroxysmal nocturnal hemoglobinuria and other complement-mediated disorders, hemophilia A, metabolic disorders, rare cholestatic liver diseases and neuromuscular diseases. Its pipeline includes Hunterase, Livmarli, CAN106, and CAN103. Hunterase is an enzyme replacement therapy for the treatment of Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome. Livmarli an oral, minimally absorbed, reversible inhibitor of the ileal bile acid transporter that is under development to treat rare cholestatic liver diseases including Alagille syndrome and progressive familial intrahepatic cholestasis.