Manufacture of Active Highly Phosphorylated Human Lysosomal Sulfatase Enzymes and Uses Thereof

摘要

This invention provides compositions of active highly phosphorylated lysosomal sulfatase enzymes, their pharmaceutical compositions, methods of producing and purifying such lysosomal sulfatase enzymes and compositions and their use in the diagnosis, prophylaxis, or treatment of diseases and conditions, including particularly lysosomal storage diseases that are caused by, or associated with, a deficiency in the lysosomal sulfatase enzyme.

主权项

1. A recombinant human lysosomal sulfatase enzyme or biologically active fragment, mutant, variant or derivative thereof produced in a END3 complementation group CHO cell or derivative thereof, wherein said END3 complementation group CHO cell or derivative thereof expresses recombinant human sulfatase modifying factor 1 (SUMF1), and wherein said recombinant lysosomal sulfatase enzyme or biologically active fragment, mutant, variant or derivative thereof has a high level of activation, and a high level of phosphorylation.