CA2656643C - 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
CA2656643C
CA2656643C CA2656643A CA2656643A CA2656643C CA 2656643 C CA2656643 C CA 2656643C CA 2656643 A CA2656643 A CA 2656643A CA 2656643 A CA2656643 A CA 2656643A CA 2656643 C CA2656643 C CA 2656643C
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CA
Canada
Prior art keywords
isofagomine
substituted
activity
unsubstituted
gcase
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 - Fee Related
Application number
CA2656643A
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English (en)
French (fr)
Other versions
CA2656643A1 (en
Inventor
Brandon Wustman
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Individual
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Amicus Therapeutics Inc
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Application filed by Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of CA2656643A1 publication Critical patent/CA2656643A1/en
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Publication of CA2656643C publication Critical patent/CA2656643C/en
Expired - Fee Related legal-status Critical Current
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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)
  • Animal Behavior & Ethology (AREA)
  • Chemical & Material Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Epidemiology (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Immunology (AREA)
  • Psychology (AREA)
  • Pain & Pain Management (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Rheumatology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Hydrogenated Pyridines (AREA)
  • Saccharide Compounds (AREA)
  • Plural Heterocyclic Compounds (AREA)
  • Steroid Compounds (AREA)
  • Medicines Containing Plant Substances (AREA)
CA2656643A 2006-06-23 2007-06-25 Method for the treatment of neurological disorders by enhancing the activity of .beta.-glucocerebrosidase Expired - Fee Related CA2656643C (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
CA2656643A1 CA2656643A1 (en) 2007-12-27
CA2656643C true CA2656643C (en) 2015-08-25

Family

ID=38834440

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2656643A Expired - Fee Related CA2656643C (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 (enExample)
EP (1) EP2040548B1 (enExample)
JP (1) JP5303458B2 (enExample)
KR (1) KR101402554B1 (enExample)
AT (1) ATE555788T1 (enExample)
AU (1) AU2007260812B2 (enExample)
BR (1) BRPI0713442A2 (enExample)
CA (1) CA2656643C (enExample)
DK (1) DK2040548T3 (enExample)
ES (1) ES2387845T3 (enExample)
IL (1) IL196151A (enExample)
MX (1) MX2009000032A (enExample)
PT (1) PT2040548E (enExample)
WO (1) WO2007150064A2 (enExample)
ZA (1) ZA200900270B (enExample)

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DK2040548T3 (da) 2006-06-23 2012-08-13 Amicus Therapeutics Inc Fremgangsmåde til behandling af neurologiske forstyrrelser ved forøgelse af aktiviteten af beta-glucocerebrosidase
GB0622702D0 (en) * 2006-11-15 2006-12-27 Univ Dundee Selective glycosidase inhibitors
CN104383556B (zh) 2007-05-16 2018-06-05 布里格姆妇女医院 突触核蛋白病的治疗
JP5616336B2 (ja) * 2008-06-26 2014-10-29 オルファザイム エーピーエスＯｒｐｈａｚｙｍｅ Ａｐｓ 酵素活性の調節因子としてのＨｓｐ７０の使用
AU2009314447A1 (en) * 2008-11-14 2010-05-20 Neuraltus Pharmaceuticals, Inc. Compositions and methods for the treatment of altered alpha-synuclein function
JP5645918B2 (ja) * 2009-04-09 2014-12-24 アミカス セラピューティックス インコーポレイテッド リソソーム蓄積障害の予防および／または治療方法
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
US9206457B2 (en) * 2009-05-26 2015-12-08 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
EP2435459B1 (en) * 2009-05-26 2016-05-04 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of acid beta-glucosidase
AU2010281403B2 (en) * 2009-07-28 2016-03-10 Takeda Pharmaceutical Company Limited Compositions and methods for treating Gaucher disease
RU2015147509A (ru) * 2009-10-19 2019-01-14 Амикус Терапьютикс, Инк. Новые композиции для предупреждения и/или лечения дегенеративных расстройств центральной нервной системы
WO2011049737A1 (en) * 2009-10-19 2011-04-28 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating lysosomal storage disorders
DK2490712T3 (en) * 2009-10-19 2015-10-05 Amicus Therapeutics Inc PROCESS FOR THE TREATMENT OF ALZHEIMER'S DISEASE BY USING Pharmacological chaperones TO INCREASE ACTIVITY OF GANGLIOSIDASER
US9662375B2 (en) 2010-11-30 2017-05-30 Orphazyme Aps Methods for increasing intracellular activity of Hsp70
TR201906779T4 (tr) 2011-05-12 2019-05-21 Proteostasis Therapeutics Inc Proteostaz regülatörleri.
EP2533051A1 (en) 2011-06-06 2012-12-12 Centogene GmbH Method for the diagnosis of Gaucher's disease
AU2012272815B2 (en) * 2011-06-22 2017-09-07 The General Hospital Corporation Treatment of proteinopathies
WO2013112706A1 (en) 2012-01-25 2013-08-01 Proteostasis Therapeutics, Inc. Proteasome activity modulating compounds
HK1214521A1 (zh) * 2012-11-05 2016-07-29 Genzyme Corporation 用於治療蛋白質病的組合物和方法
WO2014089449A1 (en) * 2012-12-07 2014-06-12 Rush University Nedical Center Composition and method for treating neuronal ceroid lipofuscinosis
JP2017502961A (ja) 2013-12-23 2017-01-26 アレクトス セラピューティックス インコーポレイテッド グルコセレブロシダーゼモジュレーターおよびその使用
US9675627B2 (en) 2014-04-14 2017-06-13 Amicus Therapeutics, Inc. Dosing regimens for treating and/or preventing cerebral amyloidoses
US10709700B2 (en) 2014-09-15 2020-07-14 Orphazyme A/S Arimoclomol formulation
EP3215511B1 (en) 2014-11-06 2024-04-17 Bial-R&D Investments, S.A. Substituted pyrazolo(1,5-a)pyrimidines and their use in the treatment of medical disorders
CA2985235A1 (en) 2015-05-07 2016-11-10 Shire Human Genetic Therapies, Inc. Glucocerebrosidase gene therapy for parkinson's disease
KR20180133461A (ko) 2016-04-06 2018-12-14 리소소말 테라퓨틱스 인크. 피라졸로[1,5-a]피리미디닐 카르복스아미드 화합물 및 의학적 장애의 치료에서의 그의 용도
WO2017176961A1 (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
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WO2017178029A1 (en) 2016-04-13 2017-10-19 Orphazyme Aps Heat shock proteins and cholesterol homeostasis
ES2831764T3 (es) 2016-04-29 2021-06-09 Orphazyme As Arimoclomol para el tratamiento de trastornos asociados a glucocerebrosidasa
WO2017192930A1 (en) 2016-05-05 2017-11-09 Lysosomal Therapeutics Inc. SUBSTITUTED IMIDAZO[1,2-b]PYRIDAZINES, SUBSTITUTED IMIDAZO[1,5-b]PYRIDAZINES, RELATED COMPOUNDS, AND THEIR USE IN THE TREATMENT OF MEDICAL DISORDERS
EP3264092A1 (en) * 2016-07-01 2018-01-03 Centogene AG Use of lyso-gb1 as druggable target
EA202091032A1 (ru) * 2017-10-26 2020-07-17 Шайр Хьюман Дженетик Терапиз, Инк. Составы, содержащие глюкоцереброзидазу и изофагомин
CN108428452B (zh) 2018-03-14 2019-12-13 百度在线网络技术（北京）有限公司 终端支架和远场语音交互系统
WO2020006296A1 (en) 2018-06-27 2020-01-02 Proteostasis Therapeutics, Inc. Proteasome activity enhancing compounds
WO2020006269A1 (en) 2018-06-27 2020-01-02 Proteostasis Therapeutics, Inc. Proteasome activity enhancing compounds
WO2020023390A1 (en) 2018-07-25 2020-01-30 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders
KR20210113606A (ko) * 2018-12-05 2021-09-16 워싱턴 유니버시티 신경 질환을 검출, 예방, 회복 및 치료하는 방법
CN113906012A (zh) * 2019-04-25 2022-01-07 武田药品工业有限公司 异法戈明盐、使用方法和调配物
CN111450306B (zh) * 2020-03-06 2021-11-19 大连理工大学 外用纳米羟基磷灰石/聚多巴胺湿黏附型止血粉及其制备方法
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GB2355074A (en) * 1996-10-15 2001-04-11 Imperial College Method for predicting susceptibility to bovine spongiform encephalopathy
US6274597B1 (en) * 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
JP5589165B2 (ja) 2003-01-31 2014-09-17 マウント シナイ スクール オブ メディシン オブ ニューヨーク ユニバーシティー タンパク質欠損性障害の治療のための併用療法
US20050137223A1 (en) 2003-11-12 2005-06-23 Amicus Therapeutics, Inc. Glucoimidazole and polyhydroxycyclohexenyl amine derivatives to treat gaucher disease
ES2485369T3 (es) * 2005-06-08 2014-08-13 Amicus Therapeutics, Inc. Tratamiento de trastornos del SNC asociados con mutaciones en genes que codifican enzimas lisosómicas
WO2007140212A2 (en) * 2006-05-24 2007-12-06 Amicus Therapeutics, Inc. Tartrate salt of isofagomine and methods of use
DK2040548T3 (da) 2006-06-23 2012-08-13 Amicus Therapeutics Inc Fremgangsmåde til behandling af neurologiske forstyrrelser ved forøgelse af aktiviteten af beta-glucocerebrosidase
KR101789000B1 (ko) * 2007-05-22 2017-10-20 아미쿠스 세라퓨틱스, 인코포레이티드 이소파고민과 이의 유도체를 제조하는 새로운 방법

Also Published As

Publication number Publication date
IL196151A0 (en) 2009-11-18
WO2007150064A2 (en) 2007-12-27
IL196151A (en) 2015-08-31
BRPI0713442A2 (pt) 2012-03-06
US9119845B2 (en) 2015-09-01
JP5303458B2 (ja) 2013-10-02
DK2040548T3 (da) 2012-08-13
ES2387845T3 (es) 2012-10-02
US20080009516A1 (en) 2008-01-10
US10064851B2 (en) 2018-09-04
AU2007260812B2 (en) 2012-08-16
AU2007260812A1 (en) 2007-12-27
KR20090021393A (ko) 2009-03-03
CA2656643A1 (en) 2007-12-27
ZA200900270B (en) 2011-02-23
EP2040548A4 (en) 2009-12-23
JP2009541489A (ja) 2009-11-26
ATE555788T1 (de) 2012-05-15
EP2040548A2 (en) 2009-04-01
US20170027919A1 (en) 2017-02-02
EP2040548B1 (en) 2012-05-02
US9402837B2 (en) 2016-08-02
US7829579B2 (en) 2010-11-09
US20150025109A1 (en) 2015-01-22
WO2007150064A3 (en) 2008-02-21
MX2009000032A (es) 2009-01-23
PT2040548E (pt) 2012-08-02
US20110052613A1 (en) 2011-03-03
KR101402554B1 (ko) 2014-06-19

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