AU2007260812B2 - Method for the treatment of neurological disorders by enhancing the activity of beta-glucocerebrosidase - Google Patents

Method for the treatment of neurological disorders by enhancing the activity of beta-glucocerebrosidase Download PDF

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Publication number
AU2007260812B2
AU2007260812B2 AU2007260812A AU2007260812A AU2007260812B2 AU 2007260812 B2 AU2007260812 B2 AU 2007260812B2 AU 2007260812 A AU2007260812 A AU 2007260812A AU 2007260812 A AU2007260812 A AU 2007260812A AU 2007260812 B2 AU2007260812 B2 AU 2007260812B2
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Prior art keywords
disease
substituted
activity
glucocerebrosidase
unsubstituted
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AU2007260812A
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AU2007260812A1 (en
Inventor
Brandon Wustman
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Amicus Therapeutics Inc
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Amicus Therapeutics Inc
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    • 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
    • A61K31/185—Acids; Anhydrides, halides or salts thereof, e.g. sulfur acids, imidic, hydrazonic or hydroximic acids
    • A61K31/19—Carboxylic acids, e.g. valproic acid
    • A61K31/194—Carboxylic acids, e.g. valproic acid having two or more carboxyl groups, e.g. succinic, maleic or phthalic acid
    • 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
    • A61K31/4353—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 ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/437—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 ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a five-membered ring having nitrogen as a ring hetero atom, e.g. indolizine, beta-carboline
    • 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
    • A61K31/45—Non condensed piperidines, e.g. piperocaine having oxo groups directly attached to the heterocyclic ring, e.g. cycloheximide
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/02—Muscle relaxants, e.g. for tetanus or cramps
    • 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
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • 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
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A90/00—Technologies having an indirect contribution to adaptation to climate change
    • Y02A90/10—Information and communication technologies [ICT] supporting adaptation to climate change, e.g. for weather forecasting or climate simulation

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Immunology (AREA)
  • Psychology (AREA)
  • Pain & Pain Management (AREA)
  • Hospice & Palliative Care (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Psychiatry (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Hydrogenated Pyridines (AREA)
  • Saccharide Compounds (AREA)
  • Steroid Compounds (AREA)
  • Medicines Containing Plant Substances (AREA)
AU2007260812A 2006-06-23 2007-06-25 Method for the treatment of neurological disorders by enhancing the activity of beta-glucocerebrosidase Ceased AU2007260812B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US81595206P 2006-06-23 2006-06-23
US60/815,952 2006-06-23
PCT/US2007/072016 WO2007150064A2 (en) 2006-06-23 2007-06-25 METHOD FOR THE TREATMENT OF NEUROLOGICAL DISORDERS BY ENHANCING THE ACTIVITY OF β-GLUCOCEREBROSIDASE

Publications (2)

Publication Number Publication Date
AU2007260812A1 AU2007260812A1 (en) 2007-12-27
AU2007260812B2 true AU2007260812B2 (en) 2012-08-16

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ID=38834440

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AU2007260812A Ceased AU2007260812B2 (en) 2006-06-23 2007-06-25 Method for the treatment of neurological disorders by enhancing the activity of beta-glucocerebrosidase

Country Status (15)

Country Link
US (4) US7829579B2 (https=)
EP (1) EP2040548B1 (https=)
JP (1) JP5303458B2 (https=)
KR (1) KR101402554B1 (https=)
AT (1) ATE555788T1 (https=)
AU (1) AU2007260812B2 (https=)
BR (1) BRPI0713442A2 (https=)
CA (1) CA2656643C (https=)
DK (1) DK2040548T3 (https=)
ES (1) ES2387845T3 (https=)
IL (1) IL196151A (https=)
MX (1) MX2009000032A (https=)
PT (1) PT2040548E (https=)
WO (1) WO2007150064A2 (https=)
ZA (1) ZA200900270B (https=)

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GB0622702D0 (en) * 2006-11-15 2006-12-27 Univ Dundee Selective glycosidase inhibitors
EP2154969B1 (en) 2007-05-16 2015-11-18 The Brigham and Women's Hospital, Inc. Treatment of synucleinopathies
AU2009262670B2 (en) * 2008-06-26 2013-06-20 Zevra Denmark A/S Use of Hsp70 as a regulator of enzymatic activity
WO2010056413A2 (en) * 2008-11-14 2010-05-20 Parkinson's Institute COMPOSITIONS AND METHODS FOR THE TREATMENT OF ALTERED α-SYNUCLEIN FUNCTION
GB0906159D0 (en) * 2009-04-09 2009-05-20 Summit Corp Plc Drug combination for the treatment of proteostatic diseases
CA2758187C (en) * 2009-04-09 2017-03-07 Robert Boyd Methods for preventing and/or treating degenerative disorders of the central nervous system
US8940766B2 (en) * 2009-04-09 2015-01-27 Amicus Therapeutics, Inc. Methods for preventing and/or treating lysosomal storage disorders
US9206457B2 (en) * 2009-05-26 2015-12-08 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
US20110070643A1 (en) * 2009-05-26 2011-03-24 Do Hung V Utilization of Pharmacological Chaperones to Improve Manufacturing and Purification of Biologics
AU2010281403B2 (en) * 2009-07-28 2016-03-10 Takeda Pharmaceutical Company Limited Compositions and methods for treating Gaucher disease
WO2011049737A1 (en) * 2009-10-19 2011-04-28 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating lysosomal storage disorders
SI2490532T1 (sl) * 2009-10-19 2017-03-31 Amicus Therapeutics, Inc. Novi sestavki za preventivo in/ali zdravljenje degenerativnih motenj centralnega živčnega sistema
WO2011049787A1 (en) * 2009-10-19 2011-04-28 Amicus Therapeutics, Inc. Method for treating alzheimer's disease using pharmacological chaperones to increase the activity of gangliosidases
CA2817773A1 (en) 2010-11-30 2012-06-07 Orphazyme Aps Methods for increasing intracellular activity of hsp70
DK2707101T3 (da) 2011-05-12 2019-05-13 Proteostasis Therapeutics Inc Proteostaseregulatorer
EP2533051A1 (en) 2011-06-06 2012-12-12 Centogene GmbH Method for the diagnosis of Gaucher's disease
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JP2016503405A (ja) * 2012-11-05 2016-02-04 ジェンザイム・コーポレーション タンパク質症を処置するための組成物および方法
WO2014089449A1 (en) 2012-12-07 2014-06-12 Rush University Nedical Center Composition and method for treating neuronal ceroid lipofuscinosis
WO2015095963A1 (en) 2013-12-23 2015-07-02 Alectos Therapeutics Inc. Glucocerebrosidase modulators and uses thereof
US9675627B2 (en) 2014-04-14 2017-06-13 Amicus Therapeutics, Inc. Dosing regimens for treating and/or preventing cerebral amyloidoses
AU2015317447B2 (en) 2014-09-15 2021-02-25 Zevra Denmark A/S Arimoclomol formulation
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EP3292206B8 (en) 2015-05-07 2022-02-09 Takeda Pharmaceutical Company Limited Glucocerebrosidase gene therapy for parkinson's disease
CA3020305A1 (en) 2016-04-06 2017-10-12 Lysosomal Therapeutics Inc. Imidazo [1,5-a]pyrimidinyl carboxamide compounds and their use in the treatment of medical disorders
US11192892B2 (en) 2016-04-06 2021-12-07 Bial—R&D Investments, S.A. Substituted pyrazolo[1,5-a]pyrimidines for the treatment of medical disorders
EP3440081A4 (en) 2016-04-06 2019-09-18 Lysosomal Therapeutics Inc. PYRROLO [1,2-A] PYRIMIDINYL-CARBOXAMIDE COMPOUNDS AND THEIR USE IN THE TREATMENT OF MEDICAL DISORDER
US10898476B2 (en) 2016-04-13 2021-01-26 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
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CN112585130A (zh) 2018-06-27 2021-03-30 蛋白质平衡治疗股份有限公司 蛋白酶体活性增强化合物
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Patent Citations (2)

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US6916829B2 (en) * 1998-06-01 2005-07-12 Mount Sinai School Of Medicine Of New York University Method for enhancing mutant enzyme activity in gaucher disease

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Publication number Publication date
US7829579B2 (en) 2010-11-09
DK2040548T3 (da) 2012-08-13
US9119845B2 (en) 2015-09-01
IL196151A (en) 2015-08-31
ES2387845T3 (es) 2012-10-02
US20150025109A1 (en) 2015-01-22
EP2040548A4 (en) 2009-12-23
EP2040548B1 (en) 2012-05-02
US20080009516A1 (en) 2008-01-10
KR101402554B1 (ko) 2014-06-19
US10064851B2 (en) 2018-09-04
IL196151A0 (en) 2009-11-18
CA2656643A1 (en) 2007-12-27
EP2040548A2 (en) 2009-04-01
JP5303458B2 (ja) 2013-10-02
ATE555788T1 (de) 2012-05-15
BRPI0713442A2 (pt) 2012-03-06
US20170027919A1 (en) 2017-02-02
US20110052613A1 (en) 2011-03-03
CA2656643C (en) 2015-08-25
WO2007150064A3 (en) 2008-02-21
AU2007260812A1 (en) 2007-12-27
ZA200900270B (en) 2011-02-23
US9402837B2 (en) 2016-08-02
WO2007150064A2 (en) 2007-12-27
JP2009541489A (ja) 2009-11-26
MX2009000032A (es) 2009-01-23
PT2040548E (pt) 2012-08-02
KR20090021393A (ko) 2009-03-03

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