China-based rare disease firm CANbridge Pharmaceuticals announced the dosing of the first patient in a Phase I/II clinical trial for CAN103, its enzyme replacement therapy (ERT) candidate, in treatment-naïve patients with type I and III Gaucher disease. The trial marks progress in CANbridge’s partnership with WuXi Biologics (HKG: 2269) to develop therapies for rare diseases.
Drug Profile
CAN103 is an ERT developed by CANbridge to address long-term management of type I and III Gaucher disease. The therapy aims to replenish glucocerebrosidase enzyme activity, targeting lysosomal storage accumulation that causes organ enlargement, blood disorders, and bone pain.
Gaucher Disease Overview
Gaucher disease, an autosomal recessive lysosomal storage disorder, results from mutations in the glucocerebrosidase gene. Symptoms include splenomegaly, hepatomegaly, anemia, thrombocytopenia, and skeletal complications. ERT is the standard of care globally, with Takeda’s Vpriv (velaglucerase alfa) approved in China for type I disease.
Clinical Trial Details
The Phase I/II study evaluates CAN103’s safety and efficacy in patients previously untreated for Gaucher disease. The trial’s initiation follows strategic collaboration between CANbridge and WuXi Biologics to advance rare disease therapies.-Fineline Info & Tech
