DK2374876T3 - Fremgangsmåde til oprensning af alfa-galactosidase A - Google Patents

Fremgangsmåde til oprensning af alfa-galactosidase A Download PDF

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Publication number
DK2374876T3
DK2374876T3 DK10181859.9T DK10181859T DK2374876T3 DK 2374876 T3 DK2374876 T3 DK 2374876T3 DK 10181859 T DK10181859 T DK 10181859T DK 2374876 T3 DK2374876 T3 DK 2374876T3
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gal
cell
human
cells
seq
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DK10181859.9T
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Richard F Selden
Marianne Borowski
Frances P Gillespie
Carol M Kinoshita
Douglas A Treco
Melanie D Williams
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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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/14—Prodigestives, e.g. acids, enzymes, appetite stimulants, antidyspeptics, tonics, antiflatulents
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/04—Centrally acting analgesics, e.g. opioids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • 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
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • 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
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • 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 hormone [GH], i.e. somatotropin
    • 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)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Cardiology (AREA)
  • Microbiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Neurosurgery (AREA)
  • General Engineering & Computer Science (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Endocrinology (AREA)
  • Biotechnology (AREA)
  • Pain & Pain Management (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Obesity (AREA)
  • Neurology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Immunology (AREA)
  • Urology & Nephrology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Toxicology (AREA)

Claims (9)

1. Fremgangsmåde til oprensning af humant α-galactosidase A (α-Gal A) fra en prøve, omfattende at lade prøven passere over en hydrofob interaktionsresin omfattende en butylgruppe som en funktionel enhed.
2. Fremgangsmåden ifølge krav 1, hvor prøven der lades passere over en hydrofob interaktionsresin omfattende en butylgruppe som en funktionel enhed er det første kromatografitrin af fremgangsmåden.
3. Fremgangsmåden ifølge krav 1 eller 2, hvor fremgangsmåden omfatter at lade prøven passere over en hydrofob interaktionsresin og efterfølgende over en immobiliseret heparinresin, en hydroxyapatitsøjle, en anionbytterresin, eller en størrelseseksklusionsresin.
4. Fremgangsmåden ifølge krav 1 eller 2, hvor fremgangsmåden omfatter at lade prøven passere over en hydrofob interaktionsresin og efterfølgende over en immobiliseret heparinresin, en hydroxyapatitsøjle, en anionbytterresin, og en størrelseseksklusionsresin
5. Fremgangsmåden ifølge et hvilket som helst af kravene 1 til 4, hvor prøven opnås ved at dyrke en human celle der er blevet genetisk modificeret til at overudtrykke humant α-Gal A under betingelser der tillader overekspression og sekretion af humant α-Gal A og isolere humant α-Gal A fra de dyrkede-celler ved at samle mediet i hvilket-cellerne vokser og/eller ved at lysere cellerne til at frigive deres indhold.
6. Fremgangsmåden ifølge krav 5, hvor de humane celler vælges fra gruppen bestående af en fibroblastcelle, en epitelcelle, en endotelcelle, en lymfocyt, en knoglemarvcelle, en gliacelle, en hepatocyt, en keratinocyt, en muskelcelle, en nervecelle, eller en prækursor af en hvilken som helst af disse celletyper.
7. Fremgangsmåden ifølge krav 5, hvor den humane celle er fra en immortaliseret cellelinje valgt fra gruppen bestående afen Bowes-melanomcelle, en Daudi-celle, en HeLa-celle, en HL-60-celle, en HT1080-celle, en Jurkat-celle, en KB-carcinoma- celle, en K-562-leukæmi-celle, en MCF-7-brystcancer-celle, en MOLT-4-celle, en Namalwa-celle, en Raji-celle, en RPMI 8226-celle, en U-937-celle, en WI-38VA13-underlinje 2R4-celle, og en 2780AD-ovarie-carcinoma-celle.
8. Fremgangsmåden ifølge et hvilket som helst af kravene 5-7, hvor den dyrkede human celle er blevet genetisk modificeret til at indeholde et DNA-molekyle der koder for humant α-Gal A-bundet ved N-terminus til signalpeptidet af humant væksthormon (hGH) med aminosyresekvensen SEQ ID NO:21.
9. Fremgangsmåden ifølge krav 8, hvor humant α-Gal A uden signalpeptidet har aminosyresekvensen SEQ ID NO:26.
DK10181859.9T 1996-09-13 1997-09-12 Fremgangsmåde til oprensning af alfa-galactosidase A DK2374876T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US71261496A 1996-09-13 1996-09-13
EP04030107.9A EP1538202B1 (en) 1996-09-13 1997-09-12 Production of human alpha-galactosidase A

Publications (1)

Publication Number Publication Date
DK2374876T3 true DK2374876T3 (da) 2016-05-30

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Family Applications (2)

Application Number Title Priority Date Filing Date
DK10181859.9T DK2374876T3 (da) 1996-09-13 1997-09-12 Fremgangsmåde til oprensning af alfa-galactosidase A
DK04030107.9T DK1538202T3 (da) 1996-09-13 1997-09-12 Fremstilling af humant alpha-galactosidase a

Family Applications After (1)

Application Number Title Priority Date Filing Date
DK04030107.9T DK1538202T3 (da) 1996-09-13 1997-09-12 Fremstilling af humant alpha-galactosidase a

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EP (4) EP2374876B1 (da)
JP (7) JP4001925B2 (da)
KR (2) KR100547402B1 (da)
CN (1) CN1268741C (da)
AT (1) ATE286120T1 (da)
AU (1) AU4424497A (da)
CA (1) CA2265464C (da)
DE (1) DE69732129T2 (da)
DK (2) DK2374876T3 (da)
ES (3) ES2458292T3 (da)
HU (2) HU230275B1 (da)
IL (2) IL128960A (da)
MX (1) MX340738B (da)
NO (4) NO328443B1 (da)
NZ (2) NZ506214A (da)
PL (1) PL190041B1 (da)
PT (1) PT1538202E (da)
RU (1) RU2179034C2 (da)
TW (1) TW585919B (da)
WO (1) WO1998011206A2 (da)

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WO1998011206A3 (en) 1998-08-13
TW585919B (en) 2004-05-01
NO991225D0 (no) 1999-03-12
IL184637A (en) 2015-07-30
PT1538202E (pt) 2014-04-22
JP4313381B2 (ja) 2009-08-12
HU9904666A (en) 2000-05-28
JP4313405B2 (ja) 2009-08-12
JP2009060918A (ja) 2009-03-26
IL128960A (en) 2007-10-31
ATE286120T1 (de) 2005-01-15
CN1268741C (zh) 2006-08-09
IL184637A0 (en) 2009-09-22
HK1162580A1 (en) 2012-08-31
ES2581828T3 (es) 2016-09-07
CA2265464A1 (en) 1998-03-19
EP2327775A3 (en) 2012-10-24
HU1000445D0 (en) 2010-11-29
KR20050084473A (ko) 2005-08-26
JP5590788B2 (ja) 2014-09-17
JP2001504324A (ja) 2001-04-03
EP1538202B1 (en) 2014-01-22
EP2327775A2 (en) 2011-06-01
WO1998011206A2 (en) 1998-03-19
DE69732129T2 (de) 2005-12-08
HU230275B1 (hu) 2015-11-30
NO20090583L (no) 1999-05-10
CA2265464C (en) 2007-06-26
CN1230220A (zh) 1999-09-29
EP2374876A2 (en) 2011-10-12
JP2012019793A (ja) 2012-02-02
EP1538202A3 (en) 2007-05-30
MX340738B (es) 2016-07-22
PL190041B1 (pl) 2005-10-31
AU4424497A (en) 1998-04-02
NZ506214A (en) 2002-11-26
NO20141168L (no) 1999-05-10
NO340408B1 (no) 2017-04-18
HU227189B1 (en) 2010-10-28
KR20000036078A (ko) 2000-06-26
JP2007289201A (ja) 2007-11-08
HK1074058A1 (en) 2005-10-28
EP2374876B1 (en) 2016-04-13
JP2009102321A (ja) 2009-05-14
JP2015044830A (ja) 2015-03-12
NO328443B1 (no) 2010-02-22
NZ334721A (en) 2001-01-26
EP2374876A3 (en) 2012-10-24
PL332188A1 (en) 1999-08-30
EP0935651A2 (en) 1999-08-18
ES2458292T3 (es) 2014-04-30
NO991225L (no) 1999-05-10
RU2179034C2 (ru) 2002-02-10
JP2007000145A (ja) 2007-01-11
ES2234032T3 (es) 2005-06-16
NO20110496L (no) 1999-05-10
JP4001925B2 (ja) 2007-10-31
NO330687B1 (no) 2011-06-06
HUP9904666A3 (en) 2001-09-28
IL128960A0 (en) 2000-02-17
DK1538202T3 (da) 2014-03-24
EP0935651B1 (en) 2004-12-29
EP1538202A2 (en) 2005-06-08
KR100547402B1 (ko) 2006-02-01
HK1022173A1 (zh) 2000-07-28
DE69732129D1 (de) 2005-02-03

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