NO20090583L - Terapi for alfa-galaktosidase A-mangel - Google Patents

Terapi for alfa-galaktosidase A-mangel

Info

Publication number
NO20090583L
NO20090583L NO20090583A NO20090583A NO20090583L NO 20090583 L NO20090583 L NO 20090583L NO 20090583 A NO20090583 A NO 20090583A NO 20090583 A NO20090583 A NO 20090583A NO 20090583 L NO20090583 L NO 20090583L
Authority
NO
Norway
Prior art keywords
galactosidase
therapy
deficiency
alpha
sample
Prior art date
Application number
NO20090583A
Other languages
English (en)
Other versions
NO330687B1 (no
Inventor
Richard F Selden
Marianne Borowski
Francis P Gillespie
Carol M Kinoshita
Douglas A Treco
Melanie D Williams
Original Assignee
Shire Human Genetic Therapies
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Publication of NO20090583L publication Critical patent/NO20090583L/no
Application filed by Shire Human Genetic Therapies filed Critical Shire Human Genetic Therapies
Publication of NO330687B1 publication Critical patent/NO330687B1/no

Links

Classifications

    • 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

Landscapes

  • 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)
NO20090583A 1996-09-13 2009-02-06 Fremgangsmate for rensing av human alfa-galaktosidase A NO330687B1 (no)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US71261496A 1996-09-13 1996-09-13
PCT/US1997/016603 WO1998011206A2 (en) 1996-09-13 1997-09-12 THERAPY FOR α-GALACTOSIDASE A DEFICIENCY

Publications (2)

Publication Number Publication Date
NO20090583L true NO20090583L (no) 1999-05-10
NO330687B1 NO330687B1 (no) 2011-06-06

Family

ID=24862863

Family Applications (4)

Application Number Title Priority Date Filing Date
NO19991225A NO328443B1 (no) 1996-09-13 1999-03-12 DNA-molekyl, ekspresjonskonstruksjon, dyrket human celle, kloncellestamme, kloncellelinje, protein, fremgangsmate for fremstilling av glykosylert human alfa-galaktosidase A og human alfa-galaktosidase A samt anvendelse av en human celle som er genetisk modifisert.
NO20090583A NO330687B1 (no) 1996-09-13 2009-02-06 Fremgangsmate for rensing av human alfa-galaktosidase A
NO20110496A NO340408B1 (no) 1996-09-13 2011-03-31 Fremgangsmåte for å produsere humant alfa-galaktosidase A, og farmasøytisk preparat
NO20141168A NO20141168L (no) 1996-09-13 2014-09-29 Fremgangsmåte for rensing av human alfa-galaktosidase A fra en prøve

Family Applications Before (1)

Application Number Title Priority Date Filing Date
NO19991225A NO328443B1 (no) 1996-09-13 1999-03-12 DNA-molekyl, ekspresjonskonstruksjon, dyrket human celle, kloncellestamme, kloncellelinje, protein, fremgangsmate for fremstilling av glykosylert human alfa-galaktosidase A og human alfa-galaktosidase A samt anvendelse av en human celle som er genetisk modifisert.

Family Applications After (2)

Application Number Title Priority Date Filing Date
NO20110496A NO340408B1 (no) 1996-09-13 2011-03-31 Fremgangsmåte for å produsere humant alfa-galaktosidase A, og farmasøytisk preparat
NO20141168A NO20141168L (no) 1996-09-13 2014-09-29 Fremgangsmåte for rensing av human alfa-galaktosidase A fra en prøve

Country Status (20)

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

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EP1027069B1 (en) * 1997-10-29 2006-07-26 Genzyme Corporation Gene therapy for gaucher disease
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US6274597B1 (en) 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
AU2012241170B2 (en) * 1999-03-11 2016-07-28 Shire Human Genetic Therapies, Inc. Treatment of alpha-Galactosidase A deficiency
AU2016250357B2 (en) * 1999-03-11 2018-11-01 Shire Human Genetic Therapies, Inc. Treatment of alpha-Galactosidase A deficiency
AU2004242550B2 (en) * 1999-03-11 2008-04-03 Shire Human Genetic Therapies, Inc. Treatment of alpha-Galactosidase A deficiency
EP2275559A3 (en) * 1999-09-28 2011-03-23 Shire Human Genetic Therapies, Inc. Optimized messenger RNA
WO2002064799A2 (en) 1999-09-28 2002-08-22 Transkaryotic Therapies, Inc. Optimized messenger rna
WO2001042442A2 (en) 1999-12-10 2001-06-14 Cytos Biotechnology Ag Activation of endogenous genes by genomic introduction of a replicon
DE60138535D1 (de) 2000-02-04 2009-06-10 Children S Hospital Res Founda Verwendung von lysosomal acid lipase zur behandlung von atherosklerose und ähnlichen krankheiten
US20020095135A1 (en) 2000-06-19 2002-07-18 David Meeker Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US7138262B1 (en) * 2000-08-18 2006-11-21 Shire Human Genetic Therapies, Inc. High mannose proteins and methods of making high mannose proteins
IL140110A0 (en) 2000-12-05 2002-02-10 Applied Research Systems Improvement of homogeneity and secretion of recombinant proteins in mammalian systems
DK1503788T3 (da) 2002-04-25 2011-10-17 Shire Human Genetic Therapies Behandling af alpha-galactosidase A-deficiens
PT1587923E (pt) 2003-01-22 2011-12-07 Univ Duke Construções melhoradas para a expressão de polipéptidos lisossomais
JP4368925B2 (ja) 2005-11-18 2009-11-18 財団法人 東京都医学研究機構 基質特異性を変換した新規高機能酵素
AR059089A1 (es) * 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
ES2391657T3 (es) 2006-02-07 2012-11-28 Shire Human Genetic Therapies, Inc. Composiciones estabilizadas de proteínas que tienen un resto tiol libre
CN101404927A (zh) 2006-02-09 2009-04-08 建新公司 慢速脑室内递送
US8569032B2 (en) 2007-05-18 2013-10-29 Tokyo Metropolitan Institute Of Medical Science Proteins having acquired A-galactosidase activity
EP3608330B1 (en) 2008-12-16 2022-11-09 Genzyme Corporation Synthetic intermediates for preparing oligosaccharide-protein conjugates
AU2010281403B2 (en) 2009-07-28 2016-03-10 Takeda Pharmaceutical Company Limited Compositions and methods for treating Gaucher disease
KR101742346B1 (ko) 2010-04-23 2017-06-15 시나게바 바이오파르마, 코포레이션 리소솜 축적병 효소
WO2012012461A2 (en) 2010-07-19 2012-01-26 Shire Human Genetic Therapies, Inc. Mannose receptor c type 1 (mrc1) codon optimized cell line and uses thereof
BR122019021757B1 (pt) 2010-09-09 2020-12-08 Alexion Pharmaceuticals, Inc composição farmacêutica para tratamento de um paciente humano sofrendo de uma deficiência de lipase ácida lisossomal (lal)
EP2675472A4 (en) 2011-02-15 2014-09-17 Synageva Biopharma Corp METHOD FOR THE TREATMENT OF LACK OF LYSOSOMAL SAURER LIPASE
KR20140091721A (ko) * 2011-11-02 2014-07-22 제넨테크, 인크. 오버로드 및 용리 크로마토그래피
WO2013130963A1 (en) 2012-03-02 2013-09-06 Shire Human Genetic Therapies, Inc. Compositions and methods for treating type iii gaucher disease
BR112014032919B1 (pt) * 2012-06-29 2023-03-28 Universitat De Barcelona Conjugado e seu método de produção, lipossomas, uso e método de produção dos mesmos, bem como composição farmacêutica
WO2014016873A1 (en) 2012-07-26 2014-01-30 Jcr Pharmaceuticals Co., Ltd. Method for production of recombinant human alpha-galactosidase a
MX379269B (es) 2012-10-24 2025-03-11 Genzyme Corp Elucion de biomoleculas de resinas multimodales utilizando mes y mops como modificadores de fase movil.
TWI793159B (zh) 2013-10-23 2023-02-21 美商健臻公司 重組醣蛋白及其用途
CA2970638A1 (en) 2014-12-22 2016-06-30 Codexis, Inc. Human alpha-galactosidase variants
WO2016116966A1 (en) 2015-01-22 2016-07-28 Jcr Pharmaceuticals Co., Ltd. Method for purification of recombinant human alpha-galactosidase a from material containing contaminant host cell proteins
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
WO2019004446A1 (ja) 2017-06-30 2019-01-03 富士フイルム株式会社 ライソゾーム病処置剤
ES2716305B2 (es) 2017-12-11 2019-11-27 Fund Biomedica Galicia Sur Uso de chaperonas farmacologicas para el tratamiento de enfermedades de deposito lisosomal
MY207075A (en) 2018-12-20 2025-01-28 Codexis Inc Human alpha-galactosidase variants
CN111308095A (zh) * 2020-03-04 2020-06-19 北京师范大学 用于诊断前列腺癌的尿液蛋白标记物
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Also Published As

Publication number Publication date
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
DK2374876T3 (en) 2016-05-30
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 (en) 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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