Manufacture of active highly phosphorylated human lysosomal sulfatase enzymes and uses thereof
AssigneeBiomarin Pharmaceutical Inc.
InventorsErno Pungor, Charles Hague, Zhi Chen
Life of this patent
Estimating the term…
Abstract
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.
Claims18
A method of treating a subject suffering from Mucopolysaccharidosis type IVa (MPS IVa) or Morquio A syndrome, comprising intravenously administering to the subject an effective amount of a purified, recombinant human N-acetylgalactosamine-6-sulfatase (GALNS) enzyme comprising an amino acid sequence at least 95% identical to amino acids 27 to 522 of SEQ ID NO:4, wherein said GALNS enzyme:
- (a) has a purity of at least about 90% as determined by Coomassie Blue staining when subjected to SDS-PAGE under non-reducing conditions;
- (b) has at least about 50% conversion of the cysteine residue at position 53 to Cα-formylglycine (FGly);
- and
- (c) is N-linked glycosylated at the asparagine residues at positions 178 and 397, wherein at least about 50% of the oligomannose chains attached to the asparagine residue at position 178 are bis-phosphorylated.
Dependencies are inferred from the claim text.