BRPI0416217A - composto derivado de hidroxipiperidina, métodos para a sìntese de um composto derivado de hidroxipiperidina para inibir glicocerebrosidase, para aumentar a atividade de glicocerebrosidase numa célula de mamìfero e para estabilizar glicocerebrosidade, composição farmacêutica, métodos para inibir -glicosidade, e para tratar doença de gaucher e derivado hidroxipiperidina com ligante flexìvel curto - Google Patents

composto derivado de hidroxipiperidina, métodos para a sìntese de um composto derivado de hidroxipiperidina para inibir glicocerebrosidase, para aumentar a atividade de glicocerebrosidase numa célula de mamìfero e para estabilizar glicocerebrosidade, composição farmacêutica, métodos para inibir -glicosidade, e para tratar doença de gaucher e derivado hidroxipiperidina com ligante flexìvel curto

Info

Publication number
BRPI0416217A
BRPI0416217A BRPI0416217-0A BRPI0416217A BRPI0416217A BR PI0416217 A BRPI0416217 A BR PI0416217A BR PI0416217 A BRPI0416217 A BR PI0416217A BR PI0416217 A BRPI0416217 A BR PI0416217A
Authority
BR
Brazil
Prior art keywords
hydroxypiperidine
methods
inhibit
derivative
glucocerebrosidase
Prior art date
Application number
BRPI0416217-0A
Other languages
English (en)
Inventor
Jian-Qiang Fan
Xiaoxiang Zhu
Kamlesh Sheth
Original Assignee
Amicus Therapeutics Inc
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 Amicus Therapeutics Inc filed Critical Amicus Therapeutics Inc
Publication of BRPI0416217A publication Critical patent/BRPI0416217A/pt
Publication of BRPI0416217A8 publication Critical patent/BRPI0416217A8/pt

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07DHETEROCYCLIC COMPOUNDS
    • C07D487/00Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
    • C07D487/02Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00 in which the condensed system contains two hetero rings
    • C07D487/04Ortho-condensed systems
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06Antianaemics

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biomedical Technology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Hydrogenated Pyridines (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Low-Molecular Organic Synthesis Reactions Using Catalysts (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Nitrogen Condensed Heterocyclic Rings (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)

Abstract

"COMPOSTO DERIVADO DE HIDROXIPIPERIDINA, MéTODOS PARA A SìNTESE DE UM COMPOSTO DERIVADO DE HIDROXIPIPERIDINA PARA INIBIR GLICOCEREBROSIDASE, PARA AUMENTAR A ATIVIDADE DE GLICOCEREBROSIDASE NUMA CéLULA DE MAMìFERO E PARA ESTABILIZAR GLICOCEREBROSIDADE, COMPOSIçãO FARMACêUTICA, MéTODOS PARA INIBIR <225>GLICOSIDASE, E PARA TRATAR DOENçA DE GAUCHER E DERIVADO DE HIDROXIPIPERIDINA COM LIGANTE FLEXìVEL CURTO". A presente invenção refere-se a derivados de hidroxipiperidina (HP)novos com (i) uma carga positiva na posição correspondente à posição anomérica de um anel de piranose; (ii) um ligante flexível curto procedente da posição correspondente do oxigênio no anel numa piranose; e (iii) uma porção lipofílica conectada ao ligante e seus sais farmaceuticamente aceitáveis. O ligante pode estar ausente se a porção lipofílica corresponder a uma cadeia hidrocarbónica com uma extensão linear de 6 ou mais carbonos. A presente invenção provê ainda um método para tratar indivíduos com doença de Gaucher administrando-se o derivado de HP na forma de 'chaperonas' (proteínas ligantes) para a glicocerebrosidade mutante associada com a doença.
BRPI0416217A 2003-11-12 2004-11-12 composto derivado de hidroxipiperidina, métodos para a síntese de um composto derivado de hidroxipiperidina para inibir glicocerebrosidase, para aumentar a atividade de glicocerebrosidase numa célula de mamífero e para estabilizar glicocerebrosidade, composição farmacêutica, métodos para inibir beta-glicosidade, e para tratar doença de gaucher e derivado hidroxipiperidina com ligante flexível curto BRPI0416217A8 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US51949603P 2003-11-12 2003-11-12
PCT/US2004/037704 WO2005046612A2 (en) 2003-11-12 2004-11-12 Hydroxy piperidine derivatives to treat gaucher disease

Publications (2)

Publication Number Publication Date
BRPI0416217A true BRPI0416217A (pt) 2007-01-02
BRPI0416217A8 BRPI0416217A8 (pt) 2018-05-08

Family

ID=34590422

Family Applications (2)

Application Number Title Priority Date Filing Date
BRPI0416216-1A BRPI0416216A (pt) 2003-11-12 2004-11-12 composto de glicoimidazol e polihidroxiciclohexenila e método para sintetizar composto de glicoimidazol e polihidroxiciclohexenila, métodos para inibir glicocerebrosidase, para aumentar a atividade de glicocerebrosidase e para estabilizar glicocerebrosidade, composição farmacêutica, métodos para inibir uma -glicosidade, e para tratar doença de gaucher, derivado de glicoimidazol e derivados de polihidroxiciclohexilamina
BRPI0416217A BRPI0416217A8 (pt) 2003-11-12 2004-11-12 composto derivado de hidroxipiperidina, métodos para a síntese de um composto derivado de hidroxipiperidina para inibir glicocerebrosidase, para aumentar a atividade de glicocerebrosidase numa célula de mamífero e para estabilizar glicocerebrosidade, composição farmacêutica, métodos para inibir beta-glicosidade, e para tratar doença de gaucher e derivado hidroxipiperidina com ligante flexível curto

Family Applications Before (1)

Application Number Title Priority Date Filing Date
BRPI0416216-1A BRPI0416216A (pt) 2003-11-12 2004-11-12 composto de glicoimidazol e polihidroxiciclohexenila e método para sintetizar composto de glicoimidazol e polihidroxiciclohexenila, métodos para inibir glicocerebrosidase, para aumentar a atividade de glicocerebrosidase e para estabilizar glicocerebrosidade, composição farmacêutica, métodos para inibir uma -glicosidade, e para tratar doença de gaucher, derivado de glicoimidazol e derivados de polihidroxiciclohexilamina

Country Status (10)

Country Link
US (3) US7741340B2 (pt)
EP (2) EP1682504B1 (pt)
JP (2) JP4767172B2 (pt)
CN (2) CN1901907A (pt)
AU (2) AU2004289327A1 (pt)
BR (2) BRPI0416216A (pt)
CA (2) CA2545435C (pt)
ES (1) ES2402182T3 (pt)
IL (2) IL175444A (pt)
WO (2) WO2005046611A2 (pt)

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CA2683713A1 (en) * 2007-04-13 2008-10-23 Amicus Therapeutics, Inc. Treatment of gaucher disease with specific pharmacological chaperones and monitoring treatment using surrogate markers
US9999618B2 (en) * 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
WO2008144773A1 (en) 2007-05-22 2008-11-27 Amicus Therapeutics, Inc. New method for preparing isofagomine and its derivatives
WO2010118283A1 (en) * 2009-04-09 2010-10-14 Amicus Therapeutics, Inc. Methods for preventing and/or treating lysosomal storage disorders
MX354506B (es) 2010-12-08 2018-01-16 Us Health Pirazolopirimidinas sustituidas como activadores de glucocerebrosidasa.
US9017979B2 (en) 2011-04-11 2015-04-28 Roche Molecular Systems, Inc. DNA polymerases with improved activity
SI2707101T1 (sl) 2011-05-12 2019-06-28 Proteostasis Therapeutics, Inc. Regulatorji proteostaze
WO2013079580A1 (en) 2011-11-29 2013-06-06 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for the treatment of darier disease
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Also Published As

Publication number Publication date
BRPI0416216A (pt) 2006-12-26
AU2004289328B2 (en) 2011-05-19
US20100189708A1 (en) 2010-07-29
CA2545435A1 (en) 2005-05-26
CN1906162B (zh) 2012-05-23
EP1682504A2 (en) 2006-07-26
WO2005046611A2 (en) 2005-05-26
CA2545435C (en) 2014-08-19
IL175472A0 (en) 2006-09-05
BRPI0416217A8 (pt) 2018-05-08
WO2005046612A2 (en) 2005-05-26
EP1682134A4 (en) 2008-10-15
CN1906162A (zh) 2007-01-31
CA2545433A1 (en) 2005-05-26
WO2005046612A3 (en) 2005-07-14
US20050130972A1 (en) 2005-06-16
IL175444A (en) 2013-05-30
US7741340B2 (en) 2010-06-22
EP1682134A2 (en) 2006-07-26
EP1682504A4 (en) 2008-10-22
AU2004289328A1 (en) 2005-05-26
JP2007510753A (ja) 2007-04-26
IL175444A0 (en) 2006-09-05
ES2402182T3 (es) 2013-04-29
WO2005046611A3 (en) 2005-09-09
EP1682504B1 (en) 2012-12-26
AU2004289327A1 (en) 2005-05-26
US20050137223A1 (en) 2005-06-23
CN1901907A (zh) 2007-01-24
JP2007512253A (ja) 2007-05-17
JP4767172B2 (ja) 2011-09-07

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