Method of analyzing the charge profile of heparan N-sulfatase

摘要

The present invention provides, among other things, methods for the characterization of recombinant Heparan N-Sulfatase (HNS) during manufacture. The present invention uses capillary zone electrophoresis to determine the charge profile, isoform distribution, and/or glycan profile of recombinant HNS; and represents a quality feature for the batch consistency, storage stability, biological half-life, pharmacokinetic, pharmacodynamic and biological activity of the enzyme. In particular, such characterization methods may be beneficial to optimize conditions and ensure consistency for the manufacture of HNS for the treatment of a patient diagnosed with Sanfilippo syndrome using enzyme replacement therapy.

主权项

1. A method of analyzing a charge profile of a heparan N-sulfatase (HNS) protein comprising characterizing the charge profile of the HNS protein by capillary zone electrophoresis (CZE),
wherein the CZE separates peak groups indicative of absence or presence of charge variants and, wherein the charge variants are associated with absence or presence of varying amounts of sialic acid and/or mannose-6-phosphate.