DE69824421T2 - Verfahren zur Behandlung von lysosomalen Speicherkrankheiten mit ZNS-Beteiligung - Google Patents

Verfahren zur Behandlung von lysosomalen Speicherkrankheiten mit ZNS-Beteiligung Download PDF

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Publication number
DE69824421T2
DE69824421T2 DE69824421T DE69824421T DE69824421T2 DE 69824421 T2 DE69824421 T2 DE 69824421T2 DE 69824421 T DE69824421 T DE 69824421T DE 69824421 T DE69824421 T DE 69824421T DE 69824421 T2 DE69824421 T2 DE 69824421T2
Authority
DE
Germany
Prior art keywords
disease
storage
mice
dnj
lysosomal storage
Prior art date
Legal status (The legal status 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 status listed.)
Expired - Lifetime
Application number
DE69824421T
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German (de)
English (en)
Other versions
DE69824421D1 (de
Inventor
M. Frances PLATT
R. Gabrielle NEISES
A. Raymond DWEK
Terry D. Butters
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Actelion Pharmaceuticals Ltd
Original Assignee
Monsanto Co
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 Monsanto Co filed Critical Monsanto Co
Publication of DE69824421D1 publication Critical patent/DE69824421D1/de
Application granted granted Critical
Publication of DE69824421T2 publication Critical patent/DE69824421T2/de
Anticipated expiration legal-status Critical
Expired - Lifetime legal-status Critical Current

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7004—Monosaccharides having only carbon, hydrogen and oxygen atoms
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7012—Compounds having a free or esterified carboxyl group attached, directly or through a carbon chain, to a carbon atom of the saccharide radical, e.g. glucuronic acid, neuraminic acid
    • 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
    • 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

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Molecular Biology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Hematology (AREA)
  • Biomedical Technology (AREA)
  • Diabetes (AREA)
  • Neurosurgery (AREA)
  • Obesity (AREA)
  • Neurology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Saccharide Compounds (AREA)
DE69824421T 1997-01-13 1998-01-13 Verfahren zur Behandlung von lysosomalen Speicherkrankheiten mit ZNS-Beteiligung Expired - Lifetime DE69824421T2 (de)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US08/782,321 US5798366A (en) 1993-05-13 1997-01-13 Method for treatment of CNS-involved lysosomal storage diseases
US782321 1997-01-13
PCT/US1998/000031 WO1998030219A1 (en) 1997-01-13 1998-01-13 Method for treatment of cns-involved lysosomal storage diseases

Publications (2)

Publication Number Publication Date
DE69824421D1 DE69824421D1 (de) 2004-07-15
DE69824421T2 true DE69824421T2 (de) 2005-03-03

Family

ID=25125672

Family Applications (1)

Application Number Title Priority Date Filing Date
DE69824421T Expired - Lifetime DE69824421T2 (de) 1997-01-13 1998-01-13 Verfahren zur Behandlung von lysosomalen Speicherkrankheiten mit ZNS-Beteiligung

Country Status (11)

Country Link
US (1) US5798366A (enExample)
EP (2) EP1491196A3 (enExample)
JP (1) JP2001508064A (enExample)
AT (1) ATE268598T1 (enExample)
AU (1) AU5813898A (enExample)
CA (1) CA2278507C (enExample)
DE (1) DE69824421T2 (enExample)
DK (1) DK1007043T3 (enExample)
ES (1) ES2223118T3 (enExample)
PT (1) PT1007043E (enExample)
WO (1) WO1998030219A1 (enExample)

Families Citing this family (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6465488B1 (en) 1997-12-11 2002-10-15 Chancellor, Masters & Scholars Of The University Of Oxford Inhibition of glycolipid biosynthesis
US6274597B1 (en) * 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
US6610703B1 (en) 1998-12-10 2003-08-26 G.D. Searle & Co. Method for treatment of glycolipid storage diseases
GB0100889D0 (en) 2001-01-12 2001-02-21 Oxford Glycosciences Uk Ltd Compounds
GB9909064D0 (en) * 1999-04-20 1999-06-16 Oxford Glycosciences Uk Ltd Therapies
WO2000066105A2 (en) * 1999-04-30 2000-11-09 The Trustees Of Boston College Treatment of epilepsy with imino sugars
ATE234626T1 (de) 1999-07-26 2003-04-15 Searle & Co Verwendung von langkettigen n-alkyl derivaten von deoxynojirimycin mit glucocerebrosidase enzyme zur herstellung eines medikaments zur behandlung von mit glykolipiden akkumulation zusammenhängenden krankheiten
US6770468B1 (en) 1999-09-14 2004-08-03 Genzyme Glycobiology Research Institute, Inc. Phosphodiester-α-GlcNAcase of the lysosomal targeting pathway
US6534300B1 (en) 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
ATE384736T1 (de) * 2001-04-30 2008-02-15 Zystor Therapeutics Inc Subzelluläres targeting von therapeutischen proteinen
US20040005309A1 (en) * 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US7629309B2 (en) * 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US20030072761A1 (en) * 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
US20030124652A1 (en) * 2001-12-21 2003-07-03 Novazyme Pharmaceuticals, Inc. Methods of producing high mannose glycoproteins in complex carbohydrate deficient cells
US6905856B2 (en) 2001-12-21 2005-06-14 Genzyme Glycobiology Research Institute, Inc. Soluble GlcNAc phosphotransferase
US6800472B2 (en) 2001-12-21 2004-10-05 Genzyme Glycobiology Research Institute, Inc. Expression of lysosomal hydrolase in cells expressing pro-N-acetylglucosamine-1-phosphodiester α-N-acetyl glucosimanidase
EP1534676B1 (en) * 2002-07-17 2012-09-12 Actelion Pharmaceuticals Ltd. Piperidinetriol derivatives as inhibitors of glycosylceramide synthase
JP4585851B2 (ja) * 2002-07-17 2010-11-24 アクテリオン ファーマシューティカルズ リミテッド グルコシルセラミドシンターゼの阻害剤としてのピペリジントリオール誘導体
WO2004055157A2 (en) * 2002-08-13 2004-07-01 Whitley Chester B Methods of using vectors to treat metabolic disorders
WO2004069190A2 (en) 2003-01-31 2004-08-19 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiency disorders
GB0313677D0 (en) * 2003-06-13 2003-07-16 Oxford Glycosciences Uk Ltd Novel compound
GB0313678D0 (en) * 2003-06-13 2003-07-16 Oxford Glycosciences Uk Ltd Novel compounds
ES2344302T3 (es) * 2004-02-10 2010-08-24 Zystor Therapeutics , Inc. Alfa glucosidasa acida y fragmentos de la misma.
US20050208090A1 (en) * 2004-03-18 2005-09-22 Medtronic, Inc. Methods and systems for treatment of neurological diseases of the central nervous system
DE102004029310A1 (de) * 2004-06-17 2005-12-29 Clariant Gmbh Hochkonzentrierte, wässrige Formulierungen von Oligo-und Polyestern
EP1773401B1 (en) * 2004-06-21 2013-01-02 Medtronic, Inc. Medical systems and methods for delivering compositions to cells
WO2006037069A1 (en) * 2004-09-28 2006-04-06 Allergan, Inc. Compositions comprising nb-dnj, ne-dnj or d-glucaro-delta-lactam and their uses for the treatment of pain and other neurological conditions
US8500720B2 (en) * 2005-05-09 2013-08-06 Medtronic, Inc Method and apparatus for treatment of cardiac disorders
ES2572148T3 (es) 2005-05-17 2016-05-30 Amicus Therapeutics Inc Un método para el tratamiento de la enfermedad de Pompe usando 1-desoxinojirimicina y derivados
JP2010509344A (ja) * 2006-11-13 2010-03-25 ザイストール セラピューティクス， インコーポレイテッド ポンペ病を治療するための方法
US20080140056A1 (en) * 2006-12-06 2008-06-12 Medtronic, Inc. Method for delivering large molecules to the brain
US20110223147A1 (en) 2008-05-07 2011-09-15 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
US20100119502A1 (en) * 2008-11-11 2010-05-13 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
EP3679942A1 (en) 2009-06-17 2020-07-15 BioMarin Pharmaceutical Inc. Formulations for lysosomal enzymes
CA2731346A1 (en) * 2010-02-09 2011-08-09 The Hospital For Sick Children Compounds for the treatment of lysosomal storage diseases
EP3318277A1 (en) * 2016-11-04 2018-05-09 Institut du Cerveau et de la Moelle Epiniere-ICM Inhibitors of glucosylceramide synthase for the treatment of motor neuron diseases

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB1555654A (en) * 1977-06-25 1979-11-14 Exxon Research Engineering Co Agricultural burner apparatus
US4065562A (en) * 1975-12-29 1977-12-27 Nippon Shinyaku Co., Ltd. Method and composition for reducing blood glucose levels
NO154918C (no) * 1977-08-27 1987-01-14 Bayer Ag Analogifremgangsmaate til fremstilling av terapeutisk aktive derivater av 3,4,5-trihydroksypiperidin.
GB2020278B (en) * 1978-05-03 1983-02-23 Nippon Shinyaku Co Ltd Moranoline dervitives
US4849430A (en) * 1988-03-09 1989-07-18 Monsanto Company Method of inhibiting virus
US5011829A (en) * 1989-06-02 1991-04-30 G. D. Searle & Co. Pharmaceutical composition and method of inhibiting virus
US5030638A (en) * 1990-02-26 1991-07-09 G. D. Searle & Co. Method of antiviral enhancement
US6291657B1 (en) * 1993-05-13 2001-09-18 Monsanto Company Deoxygalactonojirimycin derivatives

Also Published As

Publication number Publication date
WO1998030219A1 (en) 1998-07-16
AU5813898A (en) 1998-08-03
CA2278507A1 (en) 1998-07-16
CA2278507C (en) 2007-08-28
EP1007043B9 (en) 2004-12-22
JP2001508064A (ja) 2001-06-19
EP1007043B1 (en) 2004-06-09
DE69824421D1 (de) 2004-07-15
ES2223118T3 (es) 2005-02-16
EP1007043A1 (en) 2000-06-14
DK1007043T3 (da) 2004-10-11
ATE268598T1 (de) 2004-06-15
EP1491196A3 (en) 2005-03-16
US5798366A (en) 1998-08-25
PT1007043E (pt) 2004-10-29
EP1491196A2 (en) 2004-12-29

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Owner name: ACTELION PHARMACEUTICALS LTD., ALLSCHWIL, CH