ATE286120T1 - Therapie für alpha galactosidase a insuffizienz - Google Patents

Therapie für alpha galactosidase a insuffizienz

Info

Publication number
ATE286120T1
ATE286120T1 AT97942567T AT97942567T ATE286120T1 AT E286120 T1 ATE286120 T1 AT E286120T1 AT 97942567 T AT97942567 T AT 97942567T AT 97942567 T AT97942567 T AT 97942567T AT E286120 T1 ATE286120 T1 AT E286120T1
Authority
AT
Austria
Prior art keywords
insufficiency
therapy
alpha galactosidase
galactosidase
sample
Prior art date
Application number
AT97942567T
Other languages
English (en)
Inventor
Richard F Selden
Marianne Borowski
Frances P Gillespie
Carol M Kinoshita
Douglas A Treco
Melanie D Williams
Original Assignee
Transkaryotic Therapies Inc
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
Application filed by Transkaryotic Therapies Inc filed Critical Transkaryotic Therapies Inc
Application granted granted Critical
Publication of ATE286120T1 publication Critical patent/ATE286120T1/de

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
    • 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
    • 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
    • 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)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Cardiology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • General Engineering & Computer Science (AREA)
  • Endocrinology (AREA)
  • Diabetes (AREA)
  • Pain & Pain Management (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Urology & Nephrology (AREA)
  • Hospice & Palliative Care (AREA)
  • Biophysics (AREA)
AT97942567T 1996-09-13 1997-09-12 Therapie für alpha galactosidase a insuffizienz ATE286120T1 (de)

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 (1)

Publication Number Publication Date
ATE286120T1 true ATE286120T1 (de) 2005-01-15

Family

ID=24862863

Family Applications (1)

Application Number Title Priority Date Filing Date
AT97942567T ATE286120T1 (de) 1996-09-13 1997-09-12 Therapie für alpha galactosidase a insuffizienz

Country Status (20)

Country Link
EP (4) EP1538202B1 (de)
JP (7) JP4001925B2 (de)
KR (2) KR20050084473A (de)
CN (1) CN1268741C (de)
AT (1) ATE286120T1 (de)
AU (1) AU4424497A (de)
CA (1) CA2265464C (de)
DE (1) DE69732129T2 (de)
DK (2) DK1538202T3 (de)
ES (3) ES2234032T3 (de)
HU (2) HU230275B1 (de)
IL (2) IL128960A (de)
MX (1) MX340738B (de)
NO (4) NO328443B1 (de)
NZ (2) NZ506214A (de)
PL (1) PL190041B1 (de)
PT (1) PT1538202E (de)
RU (1) RU2179034C2 (de)
TW (1) TW585919B (de)
WO (1) WO1998011206A2 (de)

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US6083725A (en) 1996-09-13 2000-07-04 Transkaryotic Therapies, Inc. Tranfected human cells expressing human α-galactosidase A protein
IL135578A0 (en) * 1997-10-29 2001-05-20 Genzyme Corp Compositions and methods for treating lysosomal storage disease
EP1658857A1 (de) * 1997-10-29 2006-05-24 Genzyme Corporation Zusammensetzungen und Methoden zum Behandlung von lysosomalen Lagerungskrankheit
US6274597B1 (en) 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
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
AU2012241170B2 (en) * 1999-03-11 2016-07-28 Shire Human Genetic Therapies, Inc. Treatment of alpha-Galactosidase A deficiency
WO2002064799A2 (en) 1999-09-28 2002-08-22 Transkaryotic Therapies, Inc. Optimized messenger rna
EP2275559A3 (de) * 1999-09-28 2011-03-23 Shire Human Genetic Therapies, Inc. Optimierte Boten-RNA (mRNA)
WO2001042442A2 (en) 1999-12-10 2001-06-14 Cytos Biotechnology Ag Activation of endogenous genes by genomic introduction of a replicon
US6849257B2 (en) 2000-02-04 2005-02-01 Children's Hospital Research Foundation Lipid hydrolysis therapy for atherosclerosis and related diseases
AU2001269923A1 (en) 2000-06-19 2002-01-02 Genzyme Corporation 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
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BRPI0618583A2 (pt) 2005-11-18 2011-09-06 Tokyo Metropolitan Org Med Res enzima altamente funcional tendo especifidade ao substrato modificada
AR059089A1 (es) * 2006-01-20 2008-03-12 Genzyme Corp Administracion intraventricular de una enzima para enfermedades de almacenamiento lisosomal
HRP20120994T1 (hr) 2006-02-07 2012-12-31 Shire Human Genetic Therapies, Inc. Stabilizirana smjesa glukocerebrozidaze
HUE040490T2 (hu) 2006-02-09 2019-03-28 Genzyme Corp Lassú intraventrikuláris szállítás
WO2008143354A1 (ja) * 2007-05-18 2008-11-27 Tokyo Metropolitan Organization For Medical Research 酵素補充療法用医薬組成物
CN102369023A (zh) 2008-12-16 2012-03-07 建新公司 寡糖-蛋白缀合物
NZ713967A (en) 2009-07-28 2017-01-27 Shire Human Genetic Therapies Compositions and methods for treating gaucher disease
FI3205351T3 (fi) 2010-04-23 2023-07-06 Alexion Pharma Inc Lysosomaalisen kertymätaudin entsyymi
EP2595651B1 (de) 2010-07-19 2017-03-29 Shire Human Genetic Therapies, Inc. Kodonoptimierte zelllinie für den mannoserezeptor-c vom typ 1 (mrc1) und anwendungen davon
KR20150038636A (ko) 2010-09-09 2015-04-08 시나게바 바이오파르마, 코포레이션 환자에서 리소좀 산 리파제 결핍증의 치료를 위한 리소좀 산 리파제의 용도
WO2012112681A1 (en) 2011-02-15 2012-08-23 Shire Human Genetic Therapies, Inc. Methods for treating lysosomal acid lipase deficiency
HUE035685T2 (en) * 2011-11-02 2018-05-28 Hoffmann La Roche Overload and elute chromatography
CN104519905A (zh) 2012-03-02 2015-04-15 夏尔人类遗传性治疗公司 用于治疗iii型戈谢病的组合物和方法
US9744247B2 (en) * 2012-06-29 2017-08-29 Consejo Superior De Investigaciones Científicas (Csic) Functionalized liposomes useful for the delivery of bioactive compounds
WO2014016873A1 (en) * 2012-07-26 2014-01-30 Jcr Pharmaceuticals Co., Ltd. Method for production of recombinant human alpha-galactosidase a
JP6226435B2 (ja) * 2012-07-26 2017-11-08 Ｊｃｒファーマ株式会社 組換えヒトα−ガラクトシダーゼＡの製造方法
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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
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Also Published As

Publication number Publication date
PL332188A1 (en) 1999-08-30
WO1998011206A2 (en) 1998-03-19
NO20090583L (no) 1999-05-10
NO991225D0 (no) 1999-03-12
EP2374876A2 (de) 2011-10-12
HU1000445D0 (en) 2010-11-29
ES2581828T3 (es) 2016-09-07
HK1162580A1 (en) 2012-08-31
JP2007289201A (ja) 2007-11-08
WO1998011206A3 (en) 1998-08-13
EP1538202A3 (de) 2007-05-30
KR100547402B1 (ko) 2006-02-01
EP2327775A2 (de) 2011-06-01
CA2265464A1 (en) 1998-03-19
JP2001504324A (ja) 2001-04-03
JP2007000145A (ja) 2007-01-11
IL184637A (en) 2015-07-30
EP2374876A3 (de) 2012-10-24
EP0935651A2 (de) 1999-08-18
AU4424497A (en) 1998-04-02
KR20050084473A (ko) 2005-08-26
CN1268741C (zh) 2006-08-09
HU230275B1 (hu) 2015-11-30
EP1538202B1 (de) 2014-01-22
HU227189B1 (en) 2010-10-28
PL190041B1 (pl) 2005-10-31
HK1074058A1 (en) 2005-10-28
ES2458292T3 (es) 2014-04-30
KR20000036078A (ko) 2000-06-26
CN1230220A (zh) 1999-09-29
EP0935651B1 (de) 2004-12-29
NZ334721A (en) 2001-01-26
CA2265464C (en) 2007-06-26
IL128960A (en) 2007-10-31
HK1022173A1 (zh) 2000-07-28
HU9904666A (en) 2000-05-28
RU2179034C2 (ru) 2002-02-10
MX340738B (es) 2016-07-22
ES2234032T3 (es) 2005-06-16
JP2009060918A (ja) 2009-03-26
NO340408B1 (no) 2017-04-18
JP4313405B2 (ja) 2009-08-12
JP2009102321A (ja) 2009-05-14
NO328443B1 (no) 2010-02-22
NO991225L (no) 1999-05-10
JP5590788B2 (ja) 2014-09-17
DE69732129T2 (de) 2005-12-08
NO20141168L (no) 1999-05-10
DK2374876T3 (en) 2016-05-30
NZ506214A (en) 2002-11-26
EP2327775A3 (de) 2012-10-24
JP2012019793A (ja) 2012-02-02
JP4001925B2 (ja) 2007-10-31
TW585919B (en) 2004-05-01
EP1538202A2 (de) 2005-06-08
JP4313381B2 (ja) 2009-08-12
IL184637A0 (en) 2009-09-22
HUP9904666A3 (en) 2001-09-28
IL128960A0 (en) 2000-02-17
DE69732129D1 (de) 2005-02-03
NO20110496L (no) 1999-05-10
PT1538202E (pt) 2014-04-22
EP2374876B1 (de) 2016-04-13
JP2015044830A (ja) 2015-03-12
DK1538202T3 (en) 2014-03-24
NO330687B1 (no) 2011-06-06

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