DK2314699T3 - Lægemiddelpræparater til behandlingen af alfa-galactosidase-a mangel - Google Patents

Lægemiddelpræparater til behandlingen af alfa-galactosidase-a mangel Download PDF

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Publication number
DK2314699T3
DK2314699T3 DK10180768.3T DK10180768T DK2314699T3 DK 2314699 T3 DK2314699 T3 DK 2314699T3 DK 10180768 T DK10180768 T DK 10180768T DK 2314699 T3 DK2314699 T3 DK 2314699T3
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Prior art keywords
gal
cells
preparation
cell
enzyme
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DK10180768.3T
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English (en)
Inventor
Marianne Borowski
Carol M Kinoshita
Douglas A Treco
Melanie D Williams
Thomas J Schuetz
Peter Francis Daniel
Richard F Selden
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Shire Human Genetic Therapies
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575—Hormones
    • C07K14/61—Growth hormones [GH] (Somatotropin)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022—Alpha-galactosidase (3.2.1.22)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian

Claims (6)

1. Humant glycosyleret α-Gal A-præparat med en forøget oligosaccharidladning, hvor mellem 35% og 85% af oligosacchariderne lades ved tilsætningen af: (i) én til fire sialinsyregrupper pi kompleksglycanerne, (ii) én til to phosphatgrupper på høj-mannose-glycanerne, eller (iii) et enkelt phosphat og en enkelt sialinsyre på hybridglycanerne, til anvendelse i behandlingen af en α-Gal A mangel ved en ugentlig eller hver anden ugedosis på mellem 0,05 mg til 5,0 mg af α-Gal A-præparatet pr. kg af et individs legemsvægt.
2. α-Gal A-præparatet til anvendelse ifølge krav 1, hvor mindst 50% af oligosacchariderne er ladede.
3. α-Gal A-præparatet til anvendelse ifølge krav 1 eller 2, hvor α-Gal A-enzymet indeholder omtrent 15% neutrale strukturer (høj-mannose og hybrid), omtrent 16% phosphorylerede glycaner, og omtrent 67% kompleks-glycaner med 2 til 4 sialinsyregrupper.
4. α -Gal A-præparatet til anvendelse ifølge et hvilket som helst af kravene 1-3, hvor a-Gal A-enzymet indeholder tre N-bundne glycosyleringssteder, af hvilke to N-bundne glycosyleringssteder er blevet processeret til kompleks-glycaner og det tredje sted er optaget af en høj-mannose glycan, 50% af hvilke er blevet modificeret ved lysosomal enzym-specifik phosphorylering for at give begge de monophosphorylerede og diphosphorylerede arter.
5. α-Gal A-præparatet til anvendelse ifølge krav 3 eller 4, hvor α-Gal A-præparatet produceres i HT-1080-celler.
6. α-Gal A-præparatet til anvendelse ifølge et hvilket som helst af kravene 1-5, hvor a-Gal A-præparatet er oprenset til mindst 99,5% homogenitet, som målt ved SDS-PAGE eller omvendt-fase-HPLC, hvor præparatet omfatter forskellige α-Gal A glycoformer og har en specifik aktivitet på mindst 3,0 x 105 enheder/mg protein.
DK10180768.3T 1999-03-11 2000-03-09 Lægemiddelpræparater til behandlingen af alfa-galactosidase-a mangel DK2314699T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US09/266,014 US6458574B1 (en) 1996-09-12 1999-03-11 Treatment of a α-galactosidase a deficiency
EP10152432A EP2186902B1 (en) 1999-03-11 2000-03-09 Medical preparations for the treatment of alpha-galactosidase a deficiency

Publications (1)

Publication Number Publication Date
DK2314699T3 true DK2314699T3 (da) 2017-08-07

Family

ID=23012821

Family Applications (3)

Application Number Title Priority Date Filing Date
DK00913825T DK1163349T3 (da) 1999-03-11 2000-03-09 Medicinske præparater til behandling af alfa-galactosidase A-mangel
DK10152432.0T DK2186902T3 (da) 1999-03-11 2000-03-09 Medicinske præparater til behandling af alfa-galactosidase A-mangel
DK10180768.3T DK2314699T3 (da) 1999-03-11 2000-03-09 Lægemiddelpræparater til behandlingen af alfa-galactosidase-a mangel

Family Applications Before (2)

Application Number Title Priority Date Filing Date
DK00913825T DK1163349T3 (da) 1999-03-11 2000-03-09 Medicinske præparater til behandling af alfa-galactosidase A-mangel
DK10152432.0T DK2186902T3 (da) 1999-03-11 2000-03-09 Medicinske præparater til behandling af alfa-galactosidase A-mangel

Country Status (22)

Country Link
US (4) US6458574B1 (da)
EP (6) EP1820862A3 (da)
JP (6) JP2002538183A (da)
KR (2) KR100892334B1 (da)
CN (4) CN100417727C (da)
AT (1) ATE386808T1 (da)
AU (1) AU3519400A (da)
CA (3) CA3012663A1 (da)
CY (3) CY1107951T1 (da)
DE (1) DE60038104T2 (da)
DK (3) DK1163349T3 (da)
ES (3) ES2391221T3 (da)
HK (3) HK1043386B (da)
HU (1) HU228743B1 (da)
IL (2) IL145381A0 (da)
MX (1) MXPA01009222A (da)
NO (2) NO329689B1 (da)
NZ (1) NZ514077A (da)
PL (1) PL210833B1 (da)
PT (3) PT2314699T (da)
RU (1) RU2248213C2 (da)
WO (1) WO2000053730A2 (da)

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