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CA2500498A1 - Inhibiteurs de proteine regulant la permeabilite transmembranaire de la fibrose kystique (cftr) et uitlisations - Google Patents

Inhibiteurs de proteine regulant la permeabilite transmembranaire de la fibrose kystique (cftr) et uitlisations Download PDF

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Publication number
CA2500498A1
CA2500498A1 CA002500498A CA2500498A CA2500498A1 CA 2500498 A1 CA2500498 A1 CA 2500498A1 CA 002500498 A CA002500498 A CA 002500498A CA 2500498 A CA2500498 A CA 2500498A CA 2500498 A1 CA2500498 A1 CA 2500498A1
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group
compound
hydrogen
formula
independently chosen
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CA002500498A
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English (en)
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CA2500498C (fr
Inventor
Alan Verkman
Tonghui Ma
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University of California San Diego UCSD
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Individual
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Priority claimed from US10/262,573 external-priority patent/US20040063695A1/en
Application filed by Individual filed Critical Individual
Publication of CA2500498A1 publication Critical patent/CA2500498A1/fr
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Publication of CA2500498C publication Critical patent/CA2500498C/fr
Anticipated expiration legal-status Critical
Expired - Fee Related legal-status Critical Current

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    • 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
    • A61K31/54Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one sulfur as the ring hetero atoms, e.g. sulthiame
    • A61K31/549Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one sulfur as the ring hetero atoms, e.g. sulthiame having two or more nitrogen atoms in the same ring, e.g. hydrochlorothiazide
    • 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
    • A61K31/54Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with at least one nitrogen and one sulfur as the ring hetero atoms, e.g. sulthiame
    • 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
    • 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
    • A61P1/04Drugs for disorders of the alimentary tract or the digestive system for ulcers, gastritis or reflux esophagitis, e.g. antacids, inhibitors of acid secretion, mucosal protectants
    • 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
    • A61P1/12Antidiarrhoeals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00Drugs for disorders of the respiratory system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/04Antibacterial agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • A61P31/18Antivirals for RNA viruses for HIV
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P33/00Antiparasitic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P39/00General protective or antinoxious agents
    • A61P39/02Antidotes
    • 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
    • A61P5/00Drugs for disorders of the endocrine system

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Communicable Diseases (AREA)
  • Virology (AREA)
  • Oncology (AREA)
  • Epidemiology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Molecular Biology (AREA)
  • AIDS & HIV (AREA)
  • Diabetes (AREA)
  • Endocrinology (AREA)
  • Pulmonology (AREA)
  • Toxicology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Thiazole And Isothizaole Compounds (AREA)
  • Plural Heterocyclic Compounds (AREA)

Abstract

L'invention concerne des compositions, des préparations pharmaceutiques et des procédés permettant d'inhiber la protéine CFTR, utiles pour l'étude et le traitement des maladies dont la médiation est assurée par la protéine CFTR. Les compositions et les préparations pharmaceutiques peuvent comprendre un ou plusieurs composés thiazolidinone, et par ailleurs un ou plusieurs vecteurs, excipients et/ou adjuvants pharmaceutiquement acceptables. Les procédés consistent, selon certaines variantes, à administrer au patient soufrant d'une maladie ou d'une affection dont la médiation est assurée par la protéine CFTR une quantité efficace de composé thiazolidinone. Sous d'autres variantes, on décrit des procédés inhibant la protéine CFTR qui consistent à mettre en contact des cellules du patient avec une quantité efficace de composé thiazolidinone. L'invention concerne également un modèle animal non humain de maladie dont la médiation est assurée par la protéine CFTR, résultant de l'administration d'un composé thiazolidinone à un animal non humain, en quantité suffisante pour inhiber la protéine CFTR.
CA2500498A 2002-09-30 2003-09-30 Inhibiteurs de proteine regulant la permeabilite transmembranaire de la fibrose kystique (cftr) et uitlisations Expired - Fee Related CA2500498C (fr)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US10/262,573 2002-09-30
US10/262,573 US20040063695A1 (en) 2002-09-30 2002-09-30 Cystic fibrosis transmembrane conductance regulator protein inhibitors and uses thereof
US48025303P 2003-06-20 2003-06-20
US60/480,253 2003-06-20
PCT/US2003/031005 WO2004028480A2 (fr) 2002-09-30 2003-09-30 Inhibiteurs de proteine regulant la permeabilite transmembranaire de la fibrose kystique (cftr) et uitlisations

Publications (2)

Publication Number Publication Date
CA2500498A1 true CA2500498A1 (fr) 2004-04-08
CA2500498C CA2500498C (fr) 2012-08-21

Family

ID=32044981

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2500498A Expired - Fee Related CA2500498C (fr) 2002-09-30 2003-09-30 Inhibiteurs de proteine regulant la permeabilite transmembranaire de la fibrose kystique (cftr) et uitlisations

Country Status (13)

Country Link
EP (1) EP1549321A4 (fr)
JP (1) JP4977319B2 (fr)
KR (1) KR20050061501A (fr)
CN (1) CN100356922C (fr)
AP (1) AP2005003292A0 (fr)
AU (1) AU2003277162C1 (fr)
BR (1) BR0314943A (fr)
CA (1) CA2500498C (fr)
EA (1) EA009847B1 (fr)
MX (1) MXPA05003366A (fr)
NZ (1) NZ538809A (fr)
PL (1) PL376147A1 (fr)
WO (1) WO2004028480A2 (fr)

Cited By (4)

* Cited by examiner, † Cited by third party
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US8207205B2 (en) 2008-04-21 2012-06-26 Institute For Oneworld Health Compounds, compositions and methods comprising oxadiazole derivatives
US8236838B2 (en) 2008-04-21 2012-08-07 Institute For Oneworld Health Compounds, compositions and methods comprising isoxazole derivatives
US8283351B2 (en) 2007-04-02 2012-10-09 Institute For Oneworld Health Cyclic and acyclic hydrazine derivatives compositions including them and uses thereof
US8343976B2 (en) 2009-04-20 2013-01-01 Institute For Oneworld Health Compounds, compositions and methods comprising pyrazole derivatives

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US10022352B2 (en) 2006-04-07 2018-07-17 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
PL2674428T3 (pl) 2006-04-07 2017-01-31 Vertex Pharmaceuticals Incorporated Modulatory transporterów z kasetą wiążącą ATP
USRE50453E1 (en) 2006-04-07 2025-06-10 Vertex Pharmaceuticals Incorporated Indole derivatives as CFTR modulators
US7645789B2 (en) 2006-04-07 2010-01-12 Vertex Pharmaceuticals Incorporated Indole derivatives as CFTR modulators
US8563573B2 (en) 2007-11-02 2013-10-22 Vertex Pharmaceuticals Incorporated Azaindole derivatives as CFTR modulators
AU2008302598B2 (en) * 2007-08-24 2014-07-17 Vertex Pharmaceuticals Incorporated Isothiazolopyridinones useful for the treatment of (inter alia) Cystic Fibrosis
SI2578571T1 (sl) 2007-11-16 2016-01-29 Vertex Pharmaceuticals Incorporated Izokinolinski modulatorji prenašalcev z atp-vezavno kaseto
HRP20170241T2 (hr) 2007-12-07 2023-03-17 Vertex Pharmaceuticals Incorporated Čvrsti oblici 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioksol-5-il)ciklopropankarboksamido)-3-metilpiridin-2-il)benzojeve kiseline
AU2009228307A1 (en) * 2008-03-25 2009-10-01 The Regents Of The University Of California Water soluble small molecule inhibitors of the cystic fibrosis transmembrane conductance regulator
DK2615085T3 (en) 2008-03-31 2015-10-05 Vertex Pharma Pyridyl derivatives as CFTR modulators
US20110237528A1 (en) * 2008-09-19 2011-09-29 Institute For Oneworld Health Compositions and methods comprising imidazole and triazole derivatives
US20120219543A1 (en) * 2009-10-20 2012-08-30 Raphael Scharfmann Methods and pharmaceutical compositions for the treatment of disorders of glucose homeostasis
NZ700556A (en) 2010-03-25 2016-04-29 Vertex Pharma Solid forms of (r)-1(2,2-difluorobenzo[d][1,3]dioxol-5-yl)-n-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1h-indol-5-yl) cyclopropanecarboxamide
US8802868B2 (en) 2010-03-25 2014-08-12 Vertex Pharmaceuticals Incorporated Solid forms of (R)-1(2,2-difluorobenzo[D][1,3]dioxo1-5-yl)-N-(1-(2,3-dihydroxypropyl-6-fluoro-2-(1-hydroxy-2-methylpropan2-yl)-1H-Indol-5-yl)-Cyclopropanecarboxamide
DK3150198T3 (da) 2010-04-07 2021-11-01 Vertex Pharma Farmaceutiske sammensætninger af 3-(6-(1-(2,2-difluorbenzo[d][1,3]dioxol-5-yl)-cyclopropancarboxamido)-3-methylpyriodin-2-yl)benzoesyre og indgivelse deraf
RS54783B1 (sr) 2010-04-07 2016-10-31 Vertex Pharma Čvrste forme 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioksol-5-il)ciklopropankarboksiamido)-3-metilpiridin-2-il)benzoeve kiseline
CA2797118C (fr) 2010-04-22 2021-03-30 Vertex Pharmaceuticals Incorporated Procede de production de composes de cycloalkylcarboxamido-indole
US8563593B2 (en) 2010-06-08 2013-10-22 Vertex Pharmaceuticals Incorporated Formulations of (R)-1-(2,2-difluorobenzo[D] [1,3] dioxol-5-yl)-N-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl)cyclopropanecarboxamide
BR112013004443A8 (pt) 2010-08-23 2018-01-02 Vertex Pharma composições farmacêuticas de (r)-1-(2,2-difluorbenzo[d] [1,3]dioxol-5-il)-n-(1-(2,3-di-hidróxipropil)-6-flúor-2-(1-hidróxi-2-metilpropan-2-il)-1h-indol-5-il)ciclopropano carboxamida e administração das mesmas
CN102133402B (zh) * 2011-03-24 2013-06-12 首都医科大学附属北京同仁医院 囊性纤维化跨膜转导调节因子抑制剂在制备治疗糖尿病药物中的应用
WO2012153775A1 (fr) 2011-05-10 2012-11-15 国立大学法人神戸大学 Dérivé de thioxothiazolidine ayant un effet inhibiteur sur la fonction ras
MX357328B (es) 2011-11-08 2018-07-05 Vertex Pharma Moduladores de trasportadores de casete enlazante de atp.
SG10201606135TA (en) 2012-01-25 2016-09-29 Vertex Pharma Formulations of 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl) cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid
EP2872122A1 (fr) 2012-07-16 2015-05-20 Vertex Pharmaceuticals Incorporated Compositions pharmaceutiques de (r)-1-(2,2-diflurorbenzo[d][1,3]dioxol-5-yl)-n-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1h-indol-5-yl) cyclopropanecarboxamide et son administration
SMT202500078T1 (it) 2012-11-02 2025-03-12 Vertex Pharma Composizioni farmaceutiche per il trattamento di malattie mediate da cftr
FR2999191B1 (fr) 2012-12-12 2016-02-05 Lesaffre & Cie Souches probiotiques pour le traitement et/ou la prevention de la diarrhee
RS62140B1 (sr) 2014-04-15 2021-08-31 Vertex Pharma Farmaceutske kompozicije za lečenje bolesti posredovanih transmembranskim regulatorom provodljivosti cistične fibroze
IL313498A (en) 2014-10-06 2024-08-01 Vertex Pharma Modulators of the cystic fibrosis transmembrane conductance regulator
CN104398509B (zh) * 2014-11-13 2017-01-11 四川大学华西第二医院 CFTR抑制剂CFTRinh-172在制备防治白血病细胞介导的疾病的药物中的应用
CN104788423B (zh) * 2015-03-13 2016-10-26 成都理工大学 一种新的囊性纤维化跨膜传导调节因子抑制剂
AU2016377782B2 (en) * 2015-12-24 2021-07-29 The Regents Of The University Of California CFTR regulators and methods of use thereof
MX2021013639A (es) 2016-12-09 2022-09-30 Vertex Pharma Forma cristalina del compuesto 1, un modulador del regulador de conductancia transmembrana de fibrosis quística, procesos para su preparación, composiciones farmacéuticas del compuesto 1, y su uso en el tratamiento de fibrosis quística.
AU2018304168B2 (en) 2017-07-17 2023-05-04 Vertex Pharmaceuticals Incorporated Methods of treatment for cystic fibrosis
US11465985B2 (en) 2017-12-08 2022-10-11 Vertex Pharmaceuticals Incorporated Processes for making modulators of cystic fibrosis transmembrane conductance regulator
CN114007623A (zh) * 2019-06-12 2022-02-01 加利福尼亚大学董事会 治疗胆汁酸性腹泻的方法

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ID27787A (id) * 1998-08-21 2001-04-26 Viro Pharma Inc Senyawa, komposisi dan metode untuk pengobatan atau pencegahan infeksi yang disebabkan oleh virus dan penyakit-penyakit yang berkaitan
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Cited By (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8283351B2 (en) 2007-04-02 2012-10-09 Institute For Oneworld Health Cyclic and acyclic hydrazine derivatives compositions including them and uses thereof
US8207205B2 (en) 2008-04-21 2012-06-26 Institute For Oneworld Health Compounds, compositions and methods comprising oxadiazole derivatives
US8236838B2 (en) 2008-04-21 2012-08-07 Institute For Oneworld Health Compounds, compositions and methods comprising isoxazole derivatives
US8343976B2 (en) 2009-04-20 2013-01-01 Institute For Oneworld Health Compounds, compositions and methods comprising pyrazole derivatives

Also Published As

Publication number Publication date
AU2003277162B2 (en) 2009-07-16
CN100356922C (zh) 2007-12-26
PL376147A1 (en) 2005-12-27
KR20050061501A (ko) 2005-06-22
EA009847B1 (ru) 2008-04-28
EP1549321A4 (fr) 2007-05-23
AU2003277162A1 (en) 2004-04-19
WO2004028480A3 (fr) 2004-07-01
JP4977319B2 (ja) 2012-07-18
BR0314943A (pt) 2005-08-02
AU2003277162C1 (en) 2009-12-24
CN1684686A (zh) 2005-10-19
EA200500583A1 (ru) 2005-12-29
MXPA05003366A (es) 2005-10-05
JP2006503853A (ja) 2006-02-02
EP1549321A2 (fr) 2005-07-06
AP2005003292A0 (en) 2005-06-30
WO2004028480A2 (fr) 2004-04-08
CA2500498C (fr) 2012-08-21
NZ538809A (en) 2008-06-30

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