Geruining (Velaglucerase Beta for Injection) is a lysosomal enzyme. It exerts its therapeutic effect following cellular uptake via mannose receptor-mediated intracellular endocytosis. Geruining's molecular design utilizes CRISPR-Cas9 technology to achieve targeted knockout of the GnT1 gene. This gene is pivotal in protein glycosylation, catalyzing the conversion of high-mannose type glycans to complex-type or hybrid-type glycans. Knockout of this gene blocks further protein glycosylation, resulting in exposure of mannose residues. Consequently, Geruining achieves autonomous mannose exposure without requiring any additives or exogenous enzymes. Geruining is classified as a Class 1 innovative therapeutic
Hunterase®, (iduronidase-2-sulfatase) is a recombinant human Enzyme Replacement Therapy (ERT)