CAN201

CAN201 is a preclinical gene therapy candidate being developed for the treatment of Fabry disease, a rare inherited lysosomal storage disorder caused by deficient or absent activity of the lysosomal enzyme α-galactosidase A (α-Gal A). The therapy uses an adeno-associated virus (AAV) sL65 capsid to deliver a human GLA transgene under the control of a liver-specific promoter, aiming to convert the liver into an efficient producer of α-GAL enzyme. Preclinical studies in mouse models have shown dose-dependent increases in α-GAL levels and reductions in disease-causing Gb3 lipid levels, with good tolerability. CANbridge Pharmaceuticals holds exclusive global rights to develop, manufacture, and commercialize this program based on technology licensed from LogicBio Therapeutics (a subsidiary of Alexion, AstraZeneca Rare Disease)[1][2][7].