China-based CANbridge Pharmaceuticals, Inc. (HKG: 1228) has announced the first patient dosing in the Phase II part of the ongoing CAN103 Phase I/II study. The trial is focused on treatment-naïve patients with Gaucher disease (GD) Types I and III in China. This milestone marks a significant step forward in the development of CAN103, an enzyme replacement therapy (ERT) for the long-term treatment of adults and children with Gaucher disease.
Gaucher Disease Background
Gaucher disease (GD) is a genetic disorder caused by low levels of glucocerebrosidase (GCase), an enzyme that breaks down glucocerebroside. The deficiency of this enzyme leads to the accumulation of fat-laden Gaucher cells in the spleen, liver, and bone marrow. This results in hepatosplenomegaly, anemia, thrombocytopenia, bone pain, fractures, and potentially death. Most patients with Gaucher disease in China currently lack access to approved treatments due to cost barriers.
CAN103 Development
CAN103 is being developed by CANbridge in partnership with WuXi Biologics (HKG: 2269) as part of its rare disease initiative. The therapy aims to address the significant unmet need for effective and accessible treatments for Gaucher disease Types I and III. The ongoing Phase I/II study will evaluate the safety, efficacy, and pharmacokinetics of CAN103 in treatment-naïve patients, positioning it as a potential new standard of care for this rare condition.-Fineline Info & Tech
