Phase 2 is a randomized, double-blind, dose comparison study to evaluate the efficacy, safety and pharmacokinetics of CAN103 in newly treated GD patients, with extension period. This trial will serve as a potential registrational trial for CAN103.
CAN103 is the first clinical stage enzyme replacement therapy (ERT) being developed for GD in
About CAN103
CAN103 is a recombinant human glucocerebrosidase ERT that is being developed to treat 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-glucosidase), an enzyme that helps break down a cellular membrane sphingolipid called glucocerebroside (glucosylceramide) inside 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 trials 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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