C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 9/16 (2006.01) A61K 38/46 (2006.01) A61P 3/00 (2006.01) C12N 5/10 (2006.01) C12N 15/55 (2006.01) A61K 38/00 (2006.01) A61K 48/00 (2006.01)
Patent
CA 2443555
The present invention provides a recombinant human N-acetylgalactosamine-4- sulfatase (rhASB) and biologically active mutants, fragments and analogs thereof as well as pharmaceutical formulations comprising recombinant human N- acetylgalactosamine-4-sulfatase (rhASB). The invention also provides methods for treating diseases caused all or in part by deficiencies in human N- acetylgalactosamine-4-sulfatase including MPS VI and methods for producing and purifying the recombinant enzyme.
L'invention porte sur une <i>N</i>-acétylgalactosamine-4-sulfatase (rhASB) humaine de recombinaison, et ses mutants biologiquement actifs, sur ses fragments et analogues, ainsi que sur des préparations pharmaceutiques la comprenant. L'invention porte également sur des méthodes de traitement de maladies causées totalement ou partiellement par une carence en <i>N</i>-acétylgalactosamine-4-sulfatase (rhASB) humaine, dont la MPS VI, et sur des procédés de production et de purification de ladite enzyme.
Biomarin Pharmaceuticals Inc.
Kirby Eades Gale Baker
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