CN102625661B - 用于预防和/或治疗溶酶体贮积失调的方法 - Google Patents

用于预防和/或治疗溶酶体贮积失调的方法 Download PDF

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Publication number
CN102625661B
CN102625661B CN201080023544.XA CN201080023544A CN102625661B CN 102625661 B CN102625661 B CN 102625661B CN 201080023544 A CN201080023544 A CN 201080023544A CN 102625661 B CN102625661 B CN 102625661B
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China
Prior art keywords
piperidines
glycol
chloromethyl
compound
purposes
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Expired - Fee Related
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CN201080023544.XA
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English (en)
Chinese (zh)
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CN102625661A (zh
Inventor
R·博伊德
G·李
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Amicus Therapeutics Inc
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Amicus Therapeutics Inc
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/34—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase
    • 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
    • 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
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D211/00—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
    • C07D211/04—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D211/06—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members
    • C07D211/36—Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom having no double bonds between ring members or between ring members and non-ring members with hetero atoms or with carbon atoms having three bonds to hetero atoms with at the most one bond to halogen, e.g. ester or nitrile radicals, directly attached to ring carbon atoms
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00—Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90—Enzymes; Proenzymes
    • G01N2333/914—Hydrolases (3)
    • G01N2333/924—Hydrolases (3) acting on glycosyl compounds (3.2)

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  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Genetics & Genomics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Analytical Chemistry (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Hospice & Palliative Care (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Psychiatry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Hydrogenated Pyridines (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CN201080023544.XA 2009-04-09 2010-04-09 用于预防和/或治疗溶酶体贮积失调的方法 Expired - Fee Related CN102625661B (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US16810909P 2009-04-09 2009-04-09
US61/168,109 2009-04-09
PCT/US2010/030472 WO2010118283A1 (en) 2009-04-09 2010-04-09 Methods for preventing and/or treating lysosomal storage disorders

Publications (2)

Publication Number Publication Date
CN102625661A CN102625661A (zh) 2012-08-01
CN102625661B true CN102625661B (zh) 2015-05-27

Family

ID=42934559

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201080023544.XA Expired - Fee Related CN102625661B (zh) 2009-04-09 2010-04-09 用于预防和/或治疗溶酶体贮积失调的方法

Country Status (11)

Country Link
US (1) US8940766B2 (https=)
EP (1) EP2416656B1 (https=)
JP (1) JP5645918B2 (https=)
CN (1) CN102625661B (https=)
AU (1) AU2010233188B2 (https=)
BR (1) BRPI1010517A2 (https=)
CA (1) CA2758271C (https=)
ES (1) ES2564093T3 (https=)
IL (1) IL215583A (https=)
NZ (1) NZ595629A (https=)
WO (1) WO2010118283A1 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2932982B1 (en) 2005-05-17 2018-10-03 Amicus Therapeutics, Inc. A method for the treatment of pompe disease using 1-deoxynojirimycin and derivatives
CA2758187C (en) * 2009-04-09 2017-03-07 Robert Boyd Methods for preventing and/or treating degenerative disorders of the central nervous system
SI2490532T1 (sl) 2009-10-19 2017-03-31 Amicus Therapeutics, Inc. Novi sestavki za preventivo in/ali zdravljenje degenerativnih motenj centralnega živčnega sistema
WO2011049737A1 (en) 2009-10-19 2011-04-28 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating lysosomal storage disorders
KR20200032244A (ko) * 2012-03-07 2020-03-25 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
ES2771849T3 (es) * 2014-02-12 2020-07-07 Amicus Therapeutics Inc Derivados de azúcares que comprenden restos que contienen azufre, métodos para prepararlos y métodos para emplearlos en el tratamiento de MPS IIIC
WO2016054231A1 (en) 2014-09-30 2016-04-07 Amicus Therapeutics, Inc. Highly potent acid alpha-glucosidase with enhanced carbohydrates
KR102510941B1 (ko) 2015-12-30 2023-03-20 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
IL325155A (en) 2016-03-30 2026-02-01 Amicus Therapeutics Inc Formulations containing recombinant acid alpha-glucosidase
US10227577B2 (en) 2016-03-30 2019-03-12 Amicus Therapeutics, Inc. Method for selection of high M6P recombinant proteins
EP3624831B1 (en) 2017-05-15 2023-03-29 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase
US20220184185A1 (en) 2018-07-25 2022-06-16 Modernatx, Inc. Mrna based enzyme replacement therapy combined with a pharmacological chaperone for the treatment of lysosomal storage disorders

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040204379A1 (en) * 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20050130972A1 (en) * 2003-11-12 2005-06-16 Amicus Therapeutics, Inc. Hydroxy piperidine derivatives to treat gaucher disease
US20060079533A1 (en) * 2001-03-23 2006-04-13 Nieman James A Methods of treating alzheimer's disease
US20080009516A1 (en) * 2006-06-23 2008-01-10 Wustman Brandon A Method for the treatment of neurological disorders by enhancing the activity of beta-glucocerebrosidase

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US4407957A (en) 1981-03-13 1983-10-04 Damon Corporation Reversible microencapsulation of a core material
CA2109085C (en) 1991-04-25 2003-03-11 Keith E. Dionne Implantable biocompatible immunoisolatory vehicle for delivery of selected therapeutic products
US5383851A (en) 1992-07-24 1995-01-24 Bioject Inc. Needleless hypodermic injection device
US5569189A (en) 1992-09-28 1996-10-29 Equidyne Systems, Inc. hypodermic jet injector
ES2179831T3 (es) 1992-09-29 2003-02-01 Inhale Therapeutic Syst Liberacion en los pulmones de fragmentos activos de hormona paratiroidiana.
WO1995024391A1 (en) * 1994-03-09 1995-09-14 Novo Nordisk A/S Piperidines and pyrrolidines
FR2718357B1 (fr) 1994-04-06 1997-10-03 Defarges Alain Moreau Perfectionnements apportés à un dispositif d'injection par jet sans aiguille.
US5844102A (en) 1994-07-07 1998-12-01 University Of Maryland Baltimore County Glycohydrolase inhibitors, their preparation and use thereof
US5599302A (en) 1995-01-09 1997-02-04 Medi-Ject Corporation Medical injection system and method, gas spring thereof and launching device using gas spring
US5780014A (en) 1995-04-14 1998-07-14 Inhale Therapeutic Systems Method and apparatus for pulmonary administration of dry powder alpha 1-antitrypsin
US5654007A (en) 1995-06-07 1997-08-05 Inhale Therapeutic Systems Methods and system for processing dispersible fine powders
JP2006507359A (ja) 2002-10-28 2006-03-02 ノボ・ノルデイスク・エー／エス 心循環器疾患の治療のためのグリコーゲンホスホリラーゼ阻害剤の使用
US7446098B2 (en) 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
EP2150254A4 (en) 2007-04-26 2010-11-10 Amicus Therapeutics Inc DOSAGES FOR THE TREATMENT OF LYSOSOMAL STORAGE DISEASES WITH PHARMACOLOGICAL CHAPTERONES
US8321148B2 (en) 2008-10-24 2012-11-27 Amicus Therapeutics, Inc. Multiple compartment dosing model
CA2758187C (en) * 2009-04-09 2017-03-07 Robert Boyd Methods for preventing and/or treating degenerative disorders of the central nervous system

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040204379A1 (en) * 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US20060079533A1 (en) * 2001-03-23 2006-04-13 Nieman James A Methods of treating alzheimer's disease
US20050130972A1 (en) * 2003-11-12 2005-06-16 Amicus Therapeutics, Inc. Hydroxy piperidine derivatives to treat gaucher disease
US20080009516A1 (en) * 2006-06-23 2008-01-10 Wustman Brandon A Method for the treatment of neurological disorders by enhancing the activity of beta-glucocerebrosidase

Also Published As

Publication number Publication date
IL215583A (en) 2016-09-29
JP5645918B2 (ja) 2014-12-24
EP2416656A4 (en) 2012-09-05
CA2758271A1 (en) 2010-10-14
AU2010233188A1 (en) 2011-11-03
US20100260740A1 (en) 2010-10-14
EP2416656A1 (en) 2012-02-15
BRPI1010517A2 (pt) 2017-01-31
WO2010118283A1 (en) 2010-10-14
CN102625661A (zh) 2012-08-01
CA2758271C (en) 2018-06-05
US8940766B2 (en) 2015-01-27
NZ595629A (en) 2013-04-26
JP2012523431A (ja) 2012-10-04
EP2416656B1 (en) 2015-12-23
AU2010233188B2 (en) 2015-07-16
ES2564093T3 (es) 2016-03-17
IL215583A0 (en) 2011-12-29

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