NO329689B1 - Medisinske preparater til behandling av <alfa>-galaktosidase-A-defisiens - Google Patents

Medisinske preparater til behandling av <alfa>-galaktosidase-A-defisiens Download PDF

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Publication number
NO329689B1
NO329689B1 NO20014415A NO20014415A NO329689B1 NO 329689 B1 NO329689 B1 NO 329689B1 NO 20014415 A NO20014415 A NO 20014415A NO 20014415 A NO20014415 A NO 20014415A NO 329689 B1 NO329689 B1 NO 329689B1
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Norway
Prior art keywords
gal
use according
cells
preparation
glycoforms
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NO20014415A
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English (en)
Norwegian (no)
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NO20014415D0 (no
NO20014415L (no
Inventor
Douglas A Treco
Richard F Selden
Carol M Kinoshita
Melanie D Williams
Thomas J Schuetz
Peter Francis Daniel
Marianne Borowski
Original Assignee
Shire Human Genetic Therapies
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Application filed by Shire Human Genetic Therapies filed Critical Shire Human Genetic Therapies
Publication of NO20014415D0 publication Critical patent/NO20014415D0/no
Publication of NO20014415L publication Critical patent/NO20014415L/no
Publication of NO329689B1 publication Critical patent/NO329689B1/no

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575Hormones
    • C07K14/61Growth hormone [GH], i.e. somatotropin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00Drugs for disorders of the urinary system
    • A61P13/12Drugs for disorders of the urinary system of the kidneys
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/02Drugs for disorders of the nervous system for peripheral neuropathies
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465Hydrolases (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)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/01022Alpha-galactosidase (3.2.1.22)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/10Plasmid DNA
    • C12N2800/106Plasmid DNA for vertebrates
    • C12N2800/107Plasmid DNA for vertebrates for mammalian

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Endocrinology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Diabetes (AREA)
  • Immunology (AREA)
  • Obesity (AREA)
  • Toxicology (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Plant Pathology (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Neurology (AREA)
  • Physics & Mathematics (AREA)
  • Emergency Medicine (AREA)
NO20014415A 1999-03-11 2001-09-11 Medisinske preparater til behandling av <alfa>-galaktosidase-A-defisiens NO329689B1 (no)

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
PCT/US2000/006118 WO2000053730A2 (en) 1999-03-11 2000-03-09 Medical preparations for the treatment of alpha-galactosidase a deficiency

Publications (3)

Publication Number Publication Date
NO20014415D0 NO20014415D0 (no) 2001-09-11
NO20014415L NO20014415L (no) 2001-11-12
NO329689B1 true NO329689B1 (no) 2010-11-29

Family

ID=23012821

Family Applications (2)

Application Number Title Priority Date Filing Date
NO20014415A NO329689B1 (no) 1999-03-11 2001-09-11 Medisinske preparater til behandling av <alfa>-galaktosidase-A-defisiens
NO20101493A NO20101493L (no) 1999-03-11 2010-10-22 Medisinske preparater til behandling av alfa-galaktosidase-A-defisiens

Family Applications After (1)

Application Number Title Priority Date Filing Date
NO20101493A NO20101493L (no) 1999-03-11 2010-10-22 Medisinske preparater til behandling av alfa-galaktosidase-A-defisiens

Country Status (22)

Country Link
US (4) US6458574B1 (ko)
EP (6) EP2287319A1 (ko)
JP (6) JP2002538183A (ko)
KR (2) KR100961740B1 (ko)
CN (4) CN101219213A (ko)
AT (1) ATE386808T1 (ko)
AU (1) AU3519400A (ko)
CA (3) CA2365923A1 (ko)
CY (3) CY1107951T1 (ko)
DE (1) DE60038104T2 (ko)
DK (3) DK2314699T3 (ko)
ES (3) ES2391221T3 (ko)
HK (3) HK1043386B (ko)
HU (1) HU228743B1 (ko)
IL (2) IL145381A0 (ko)
MX (1) MXPA01009222A (ko)
NO (2) NO329689B1 (ko)
NZ (1) NZ514077A (ko)
PL (1) PL210833B1 (ko)
PT (3) PT1163349E (ko)
RU (1) RU2248213C2 (ko)
WO (1) WO2000053730A2 (ko)

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