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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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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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING 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
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/34Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving hydrolase
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic 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
    • A61K31/44Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445Non condensed piperidines, e.g. piperocaine
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D211/00Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings
    • C07D211/04Heterocyclic compounds containing hydrogenated pyridine rings, not condensed with other rings with only hydrogen or carbon atoms directly attached to the ring nitrogen atom
    • C07D211/06Heterocyclic 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
    • C07D211/36Heterocyclic 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
    • GPHYSICS
    • G01MEASURING; TESTING
    • G01NINVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2333/00Assays involving biological materials from specific organisms or of a specific nature
    • G01N2333/90Enzymes; Proenzymes
    • G01N2333/914Hydrolases (3)
    • G01N2333/924Hydrolases (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)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Analytical Chemistry (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Molecular Biology (AREA)
  • Physics & Mathematics (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (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

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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 (enExample)
EP (1) EP2416656B1 (enExample)
JP (1) JP5645918B2 (enExample)
CN (1) CN102625661B (enExample)
AU (1) AU2010233188B2 (enExample)
BR (1) BRPI1010517A2 (enExample)
CA (1) CA2758271C (enExample)
ES (1) ES2564093T3 (enExample)
IL (1) IL215583A (enExample)
NZ (1) NZ595629A (enExample)
WO (1) WO2010118283A1 (enExample)

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
US8304429B2 (en) * 2009-04-09 2012-11-06 Amicus Therapeutics, Inc. Methods for preventing and/or treating degenerative disorders of the central nervous system
EP2490533B1 (en) 2009-10-19 2015-09-16 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating lysosomal storage disorders
RU2015147509A (ru) 2009-10-19 2019-01-14 Амикус Терапьютикс, Инк. Новые композиции для предупреждения и/или лечения дегенеративных расстройств центральной нервной системы
KR20140135222A (ko) 2012-03-07 2014-11-25 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병의 치료를 위한 고농도 알파-글루코시다제 조성물
MX2016010365A (es) * 2014-02-12 2016-11-30 Amicus Therapeutics Inc Derivados de azucares que comprenden restos que contienen azufre, metodos para prepararlos y metodos para emplearlos en el tratamiento de mps iiic.
SI4273241T1 (sl) 2014-09-30 2025-03-31 Amicus Therapeutics, Inc. Zelo močna kislinska alfa-glukozidaza z okrepljenimi ogljikovimi hidrati
KR102510941B1 (ko) 2015-12-30 2023-03-20 아미쿠스 세라퓨틱스, 인코포레이티드 폼페병 치료용의 강화된 산 알파-글루코시다제
NZ786723A (en) 2016-03-30 2025-07-25 Amicus Therapeutics Inc Method for selection of high m6p recombinant proteins
CA3019128A1 (en) 2016-03-30 2017-10-05 Amicus Therapeutics, Inc. Formulations comprising recombinant acid alpha-glucosidase
DK3624831T5 (da) 2017-05-15 2024-09-02 Amicus Therapeutics Inc Rekombinant human sur alfa-glucosidase
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

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
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ES2179831T3 (es) 1992-09-29 2003-02-01 Inhale Therapeutic Syst Liberacion en los pulmones de fragmentos activos de hormona paratiroidiana.
ATE243196T1 (de) * 1994-03-09 2003-07-15 Novo Nordisk As Piperidine und pyrrolidine
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
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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
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WO2008134628A2 (en) * 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
WO2010048532A1 (en) 2008-10-24 2010-04-29 Amicus Therapeutics, Inc. Multiple compartment dosing model
US8304429B2 (en) * 2009-04-09 2012-11-06 Amicus Therapeutics, Inc. 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
AU2010233188B2 (en) 2015-07-16
US8940766B2 (en) 2015-01-27
EP2416656A4 (en) 2012-09-05
WO2010118283A1 (en) 2010-10-14
BRPI1010517A2 (pt) 2017-01-31
EP2416656B1 (en) 2015-12-23
NZ595629A (en) 2013-04-26
IL215583A0 (en) 2011-12-29
JP2012523431A (ja) 2012-10-04
AU2010233188A1 (en) 2011-11-03
EP2416656A1 (en) 2012-02-15
JP5645918B2 (ja) 2014-12-24
CA2758271A1 (en) 2010-10-14
ES2564093T3 (es) 2016-03-17
US20100260740A1 (en) 2010-10-14
IL215583A (en) 2016-09-29
CA2758271C (en) 2018-06-05
CN102625661A (zh) 2012-08-01

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