A - Human Necessities – 61 – K
Patent
A - Human Necessities
61
K
A61K 38/46 (2006.01) C07H 21/04 (2006.01) C12N 1/02 (2006.01) C12N 9/14 (2006.01) C12N 9/16 (2006.01) C12N 15/00 (2006.01)
Patent
CA 2502928
The present invention provides a highly purified recombinant human precursor N~-acetylgalactosamine-4-sulfatase and biologically active mutants, fragments and analogs thereof as well as pharmaceutical formulations comprising highly purified recombinant human precursor N-acetylgalactosamine-4-sulfatase. 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 precursor enzyme to a highly purified form.
L'invention concerne un précurseur humain recombinant hautement purifié N-acétylgalactosamine-4-sulfatase et des mutants biologiquement actifs, des fragments et des analogues dudit précurseur ainsi que des préparations pharmaceutiques comprenant ledit précurseur. L'invention concerne des méthodes de traitement de maladies provoquées totalement ou en partie par des carences de <I>N</I>-acétylgalactosamine-4-sulfatase humaine comprenant MPS VI. L'invention concerne en outre des procédés de production et de purification de l'enzyme précurseur recombinant en une forme hautement purifiée.
Chan Wai-Pan
Chen Lin
Fitzpatrick Paul A.
Henstrand John M.
Qin Minmin
Biomarin Pharmaceutical Inc.
Kirby Eades Gale Baker
LandOfFree
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