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 curtoInfo
- 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
Links
Classifications
-
- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07D—HETEROCYCLIC COMPOUNDS
- C07D487/00—Heterocyclic compounds containing nitrogen atoms as the only ring hetero atoms in the condensed system, not provided for by groups C07D451/00 - C07D477/00
- C07D487/02—Heterocyclic 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
- C07D487/04—Ortho-condensed systems
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
- A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P19/00—Drugs for skeletal disorders
-
- 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
- A61P3/00—Drugs for disorders of the metabolism
- A61P3/06—Antihyperlipidemics
-
- 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
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/06—Antianaemics
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.
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) |
Families Citing this family (31)
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US7842470B2 (en) * | 2001-10-09 | 2010-11-30 | Oregon Health & Science University | Method for pharmacoperones correction of GnRHR mutant protein misfolding |
AU2006254796B2 (en) * | 2005-06-08 | 2012-08-16 | Amicus Therapeutics, Inc. | Treatment of CNS disorders associated with mutations in genes encoding lysosomal enzymes |
FR2887549B1 (fr) * | 2005-06-23 | 2009-07-10 | Centre Nat Rech Scient | Nouveaux composes de la famille des iminosucres, leurs utilisations, notamment pour le traitement de maladies lysosomales, ainsi que leur procede de preparation |
MX351004B (es) | 2006-05-16 | 2017-09-26 | Nat Institutes Of Health | Ensayos para diagnosticar y evaluar opciones de tratamiento para enfermedad de fabry. |
WO2007150064A2 (en) | 2006-06-23 | 2007-12-27 | Amicus Therapeutics, Inc. | METHOD FOR THE TREATMENT OF NEUROLOGICAL DISORDERS BY ENHANCING THE ACTIVITY OF β-GLUCOCEREBROSIDASE |
GB0622702D0 (en) * | 2006-11-15 | 2006-12-27 | Univ Dundee | Selective glycosidase inhibitors |
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 |
AU2013202368B2 (en) | 2012-01-25 | 2016-06-16 | Proteostasis Therapeutics, Inc. | Proteasome activity modulating compounds |
AU2013240486B2 (en) * | 2012-03-27 | 2017-10-26 | Amicus Therapeutics, Inc. | Novel compounds for preventing and/or treating lysosomal storage disorders and/or degenerative disorders of the central nervous system |
US20150118221A1 (en) * | 2012-05-08 | 2015-04-30 | The John Hopkins University | Compositions and methods for treating cardiac hypertrophy |
WO2015095963A1 (en) * | 2013-12-23 | 2015-07-02 | Alectos Therapeutics Inc. | Glucocerebrosidase modulators and uses thereof |
FI3215511T3 (fi) | 2014-11-06 | 2024-05-16 | Bial R&D Invest S A | Substituoituja pyratsolo(1,5-a)pyrimidiinejä ja niiden käyttö lääketieteellisten häiriöiden hoitamisessa |
EP3215509B1 (en) | 2014-11-06 | 2020-02-26 | Lysosomal Therapeutics Inc. | Substituted pyrrolo[1,2-a]pyrimidines and their use in the treatment of medical disorders |
US20170333435A1 (en) | 2014-11-06 | 2017-11-23 | Lysosomal Therapeutics Inc. | Substituted imidazo[1,5-a]pyrimidines and their use in the treatment of medical disorders |
MX2018012208A (es) | 2016-04-06 | 2019-03-28 | Lysosomal Therapeutics Inc | Compuestos a base de imidazo [1,5-a] pirimidinil carboxamida y su uso en el tratamiento de trastornos médicos. |
EP3440080A4 (en) | 2016-04-06 | 2020-01-22 | Lysosomal Therapeutics Inc. | PYRAZOLO [1,5-A] PYRIMIDINYL CARBOXAMIDE COMPOUNDS AND THEIR USE IN 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 DISORDERS |
US11168087B2 (en) | 2016-05-05 | 2021-11-09 | Bial—R&D Investments, S.A. | Substituted imidazo[1,2-b]pyridazines, substituted imidazo[1,5-b]pyridazines, related compounds, and their use in the treatment of medical disorders |
WO2017192931A1 (en) | 2016-05-05 | 2017-11-09 | Lysosomal Therapeutics Inc. | SUBSTITUTED IMDAZO[1,2-α]PYRIDINES, SUBSTITUTED IMIDAZO[1,2-α]PYRAZINES, 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 |
EP3501495A1 (en) | 2017-12-21 | 2019-06-26 | InnoMedica Holding AG | Liposomes comprising sphingomyelin |
BR112020026580A2 (pt) | 2018-06-27 | 2021-03-23 | Proteostasis Therapeutics, Inc. (Fem) | Compostos, composição farmacêutica e métodos para tratar um paciente |
EP3814336A1 (en) | 2018-06-27 | 2021-05-05 | Proteostasis Therapeutics, Inc. | Proteasome activity enhancing compounds |
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 |
Family Cites Families (16)
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US3105833A (en) * | 1962-04-03 | 1963-10-01 | Grace W R & Co | 4, 5-dihydroxy-piperidazines and their production |
DE2848117A1 (de) * | 1978-11-06 | 1980-05-14 | Bayer Ag | Derivate des 2-hydroxymethyl-3,4,5- trihydroxy-piperidins, ihre herstellung und verwendung zur beeinflussung des kohlenhydrat- und fettstoffwechsels |
DE3737523A1 (de) * | 1987-11-05 | 1989-05-18 | Bayer Ag | Verwendung von substituierten hydroxypiperidinen als antivirale mittel |
US5179023A (en) * | 1989-03-24 | 1993-01-12 | Research Corporation Technologies, Inc. | Recombinant α-galactosidase a therapy for Fabry disease |
US5401650A (en) * | 1990-10-24 | 1995-03-28 | The Mount Sinai School Of Medicine Of The City University Of New York | Cloning and expression of biologically active α-galactosidase A |
US6291657B1 (en) * | 1993-05-13 | 2001-09-18 | Monsanto Company | Deoxygalactonojirimycin derivatives |
JP3561546B2 (ja) * | 1995-01-06 | 2004-09-02 | 四国化成工業株式会社 | 新規5,6,7,8−テトラヒドロ−イミダゾ〔1,2−a〕ピリジン化合物 |
US5962313A (en) * | 1996-01-18 | 1999-10-05 | Avigen, Inc. | Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme |
WO1997031901A1 (en) * | 1996-02-29 | 1997-09-04 | Mikael Bols | Hydroxyhexahydropyridazines |
US6177447B1 (en) * | 1996-07-15 | 2001-01-23 | Universiteit Van Amsterdam | Deoxynojirimycin derivatives and their uses as glucosylceramidase inhibitors |
US6083725A (en) * | 1996-09-13 | 2000-07-04 | Transkaryotic Therapies, Inc. | Tranfected human cells expressing human α-galactosidase A protein |
CA2305768A1 (en) * | 1997-10-29 | 2000-02-24 | Genzyme Corporation | Compositions and methods for treating lysosomal storage disease |
US6274597B1 (en) * | 1998-06-01 | 2001-08-14 | Mount Sinai School Of Medicine Of New York University | Method of enhancing lysosomal α-Galactosidase A |
WO2001007078A1 (en) * | 1999-07-26 | 2001-02-01 | G.D. Searle & Co. | Use of long-chain n-alkyl derivates of deoxynojirimycin and a glucocerebrosidase enzyme for the manufacture of medicament for the treatment of glycolipid storage diseases |
US7138262B1 (en) * | 2000-08-18 | 2006-11-21 | Shire Human Genetic Therapies, Inc. | High mannose proteins and methods of making high mannose proteins |
AU2003284886A1 (en) | 2002-10-21 | 2004-05-13 | William E. Balch | CHEMICAL CHAPERONES AND THEIR EFFECT UPON THE CELLULAR ACTIVITY OF Beta-GLUCOSIDASE |
-
2004
- 2004-11-12 ES ES04810772T patent/ES2402182T3/es active Active
- 2004-11-12 CN CNA2004800402413A patent/CN1901907A/zh active Pending
- 2004-11-12 JP JP2006539870A patent/JP4767172B2/ja not_active Expired - Fee Related
- 2004-11-12 EP EP04810772A patent/EP1682504B1/en active Active
- 2004-11-12 EP EP04810771A patent/EP1682134A4/en not_active Withdrawn
- 2004-11-12 CN CN2004800402432A patent/CN1906162B/zh not_active Expired - Fee Related
- 2004-11-12 BR BRPI0416216-1A patent/BRPI0416216A/pt not_active IP Right Cessation
- 2004-11-12 CA CA2545435A patent/CA2545435C/en not_active Expired - Fee Related
- 2004-11-12 US US10/988,428 patent/US7741340B2/en active Active
- 2004-11-12 WO PCT/US2004/037703 patent/WO2005046611A2/en active Application Filing
- 2004-11-12 US US10/988,427 patent/US20050137223A1/en not_active Abandoned
- 2004-11-12 BR BRPI0416217A patent/BRPI0416217A8/pt not_active Application Discontinuation
- 2004-11-12 WO PCT/US2004/037704 patent/WO2005046612A2/en active Application Filing
- 2004-11-12 AU AU2004289327A patent/AU2004289327A1/en not_active Abandoned
- 2004-11-12 CA CA002545433A patent/CA2545433A1/en not_active Abandoned
- 2004-11-12 AU AU2004289328A patent/AU2004289328B2/en not_active Ceased
- 2004-11-12 JP JP2006539869A patent/JP2007510753A/ja active Pending
-
2006
- 2006-05-04 IL IL175444A patent/IL175444A/en active IP Right Grant
- 2006-05-07 IL IL175472A patent/IL175472A0/en unknown
-
2010
- 2010-04-08 US US12/756,382 patent/US20100189708A1/en not_active Abandoned
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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B06F | Objections, documents and/or translations needed after an examination request according [chapter 6.6 patent gazette] | ||
B07D | Technical examination (opinion) related to article 229 of industrial property law [chapter 7.4 patent gazette] | ||
B07E | Notification of approval relating to section 229 industrial property law [chapter 7.5 patent gazette] |
Free format text: NOTIFICACAO DE ANUENCIA RELACIONADA COM O ART 229 DA LPI |
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B06T | Formal requirements before examination [chapter 6.20 patent gazette] | ||
B11E | Dismissal acc. art. 34 of ipl - requirements for examination incomplete | ||
B11T | Dismissal of application maintained [chapter 11.20 patent gazette] |