CANbridge Pharmaceuticals said that it has dosed the first patient in a phase 1/2 trial of CAN103 in treatment-naïve patients in China having Gaucher disease (GD) Types I and III. The clinical trial of the recombinant human glucocerebrosidase enzyme replacement therapy is likely to enroll nearly 40 Gaucher disease patients. Gaucher disease is a lysosomal […]
US gene therapy company Avrobio has secured orphan-drug designation for AVR-RD-02, its investigational gene therapy, for the treatment of Gaucher disease, from the US Food and Drug Administration (FDA). AVR-RD-02 is made up of the patient’s own hematopoietic stem cells, which are modified genetically to express glucocerebrosidase (GCase), an enzyme that is deficient in Gaucher […]
