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Application filed by Amicus Therapeutics IncfiledCriticalAmicus Therapeutics Inc
Publication of AR110743A1publicationCriticalpatent/AR110743A1/en
Medicines That Contain Protein Lipid Enzymes And Other Medicines
(AREA)
Enzymes And Modification Thereof
(AREA)
Abstract
Se describen composiciones que comprenden enzimas a-galactosidasa A con perfiles únicos de carbohidratos, así como métodos para preparar y purificar tales enzimas. También se describen métodos para tratar, prevenir y/o mejorar la Enfermedad de Fabry mediante la administración de tales enzimas a un sujeto que lo necesita. También se describen composiciones que comprenden migalastat en combinación con dichas enzimas a-galactosidasa A. Reivindicación 1: Una a-galactosidasa A humana recombinante (rha-Gal A), caracterizada porque comprende una proteína que tiene por lo menos 98% de identidad de secuencia con la SEQ ID Nº 1 o la SEQ ID Nº 2, en donde dicha proteína tiene por lo menos 25% de oligosacáridos N-ligados totales que son mono-manosa-6-fosfato y por lo menos 12% de oligosacáridos N-ligados totales que son bis-manosa-6-fosfato.Compositions comprising a-galactosidase A enzymes with unique carbohydrate profiles are described, as well as methods for preparing and purifying such enzymes. Methods for treating, preventing and / or improving Fabry's disease by administering such enzymes to a subject in need thereof are also described. Compositions comprising migalastat in combination with said a-galactosidase A enzymes are also described. Claim 1: A recombinant human a-galactosidase A (rha-Gal A), characterized in that it comprises a protein that has at least 98% sequence identity with SEQ ID No. 1 or SEQ ID No. 2, wherein said protein has at least 25% of total N-linked oligosaccharides that are mono-mannose-6-phosphate and at least 12% of total N-linked oligosaccharides which are bis-mannose-6-phosphate.
ARP180100069A2017-01-102018-01-11
a-RECOMBINANT GALACTOSIDASE FOR THE TREATMENT OF FABRY'S DISEASE
AR110743A1
(en)
Method for producing oxalate oxidases having optical activity near physiological pH and use of such recombinant oxalate oxidases in the treatment of oxalate-related diseases
recombinant alpha galactosidase a and / or biologically active recombinant alpha galactosidase fragment, composition, recombinant polynucleotide sequence, expression vector, host cell, methods for producing a variant alpha galactosidase ae for treating and / or preventing symptoms of fabry, pharmaceutical composition, and, use of compositions.