Gaucher disease (GD), one of the most common lysosomal storage disorders, is caused by a genetic enzyme deficiency leading to an accumulation of the sphingolipid, glucocerebroside, primarily in macrophages, which results in hepatosplenomegaly, anemia, thrombocytopenia, bone pain and fractures, and death. CAN103 is an enzyme replacement therapy (ERT) under development by CANbridge, as part of its rare disease partnership with WuXi Biologics (2269.HK), for the long-term treatment of adults and children with Gaucher disease Types I and III. Most patients with Gaucher disease in China do not have access to approved treatments due to cost barriers.

“We are pleased to be moving into the Phase 2 part of our registrational Chinese trial in Gaucher disease,” said James Xue, Ph.D., CANbridge founder, chairman and CEO. “Gaucher disease was the first rare disease to have an enzyme replacement therapy approved in China. Despite this, most patients still do not have access to treatment. We hope that, building on the foundation from Phase 1, this additional phase of study will further demonstrate the safety and efficacy of CAN103 in a larger Gaucher patient group and ultimately pave the way for a registration filing. We are excited that Peking Union Medical College Hospital, which is the leader at the National Collaborative Network for the Diagnosis and Treatment of Rare Diseases, continues to be the leading site, as we advance this potentially new Gaucher disease treatment for medically underserved patients globally.”

“We look forward to moving into Phase 2 with CAN103, which has demonstrated a good safety profile to date,” said Bing Han MD, PhD, Chief Physician and Professor in the Department of Hematology at Peking Union Medical College Hospital. “CAN103 has the potential to provide a much-needed new treatment for Gaucher’s disease, where there is a high unmet medical need in China.”