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
We are pleased to be moving into the Phase 2 part of our registrational Chinese trial in Gaucher disease,' said
We look forward to moving into Phase 2 with CAN103, which has demonstrated a good safety profile to date,' said
About CAN103
CAN103 is a recombinant human glucocerebrosidase enzyme replacement therapy that is being developed to treat Gaucher disease (GD) Types I and III, which are the chronic non-neuronopathic and neuronopathic forms of the disease that constitute the majority of patients. CAN103 is delivered intravenously and is intended to supplement the lack of glucocerebrosidase in the lysosomes of GD patients.
About Gaucher disease (GD)
Gaucher disease, one of the most common lysosomal storage disorders, is a rare inherited genetic metabolic disease caused by autosomal recessive mutations in the GBA gene located on chromosome 1 and affects both males and females equally. Gaucher disease is a clinical spectrum that comprises perinatal-lethal, Type I (chronic non-neuronopathic), Type II (acute neuronopathic), and Type III (chronic neuronopathic) forms, with Types I and III surviving into adulthood. Gaucher disease is caused by a deficiency of glucocerebrosidase (acid b-glucosidase), an enzyme that helps break down a cellular membrane sphingolipid called glucocerebroside (glucosylceramide) within lysosomes. As a result, glucocerebroside accumulates primarily in cells of the monocyte-macrophage lineage (Gaucher cells) within certain organs, leading to splenomegaly, hepatomegaly, anemia, thrombocytopenia, bone pain and fractures, and in the most severe forms (perinatal-lethal, Types II and III), early neurological symptoms. For more than 25 years, recombinant human glucocerebrosidase enzyme replacement therapy (ERT) has been the standard of care for Gaucher disease, with clinical trial and real-world data demonstrating significant improvement in the major non-neurological signs and symptoms of disease and quality of life. There were 3,000 patients with Gaucher disease in
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