CA2500498C - 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
CA2500498C
CA2500498C CA2500498A CA2500498A CA2500498C CA 2500498 C CA2500498 C CA 2500498C CA 2500498 A CA2500498 A CA 2500498A CA 2500498 A CA2500498 A CA 2500498A CA 2500498 C CA2500498 C CA 2500498C
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group
cftr
hydroxyl
nitro
compound
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Expired - Fee Related
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CA2500498A
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CA2500498A1 (fr
Inventor
Alan Verkman
Tonghui Ma
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University of California San Diego UCSD
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University of California San Diego UCSD
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Priority claimed from US10/262,573 external-priority patent/US20040063695A1/en
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Publication of CA2500498A1 publication Critical patent/CA2500498A1/fr
Application granted granted Critical
Publication of CA2500498C publication Critical patent/CA2500498C/fr
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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)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Communicable Diseases (AREA)
  • Epidemiology (AREA)
  • Oncology (AREA)
  • Virology (AREA)
  • Tropical Medicine & Parasitology (AREA)
  • Diabetes (AREA)
  • Endocrinology (AREA)
  • AIDS & HIV (AREA)
  • Molecular Biology (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 US20040063695A1 (en) 2002-09-30 2002-09-30 Cystic fibrosis transmembrane conductance regulator protein inhibitors and uses thereof
US10/262,573 2002-09-30
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 CA2500498A1 (fr) 2004-04-08
CA2500498C true CA2500498C (fr) 2012-08-21

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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 (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8796321B2 (en) 2008-04-21 2014-08-05 Path Drug Solutions Compounds, compositions and methods comprising oxadiazole derivatives

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US7645789B2 (en) 2006-04-07 2010-01-12 Vertex Pharmaceuticals Incorporated Indole derivatives as CFTR modulators
NZ571803A (en) 2006-04-07 2011-12-22 Vertex Pharma Amide indole derivatives as modulators of ATP-binding cassette transporters
US10022352B2 (en) 2006-04-07 2018-07-17 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
USRE50453E1 (en) 2006-04-07 2025-06-10 Vertex Pharmaceuticals Incorporated Indole derivatives as CFTR modulators
US8563573B2 (en) 2007-11-02 2013-10-22 Vertex Pharmaceuticals Incorporated Azaindole derivatives as CFTR modulators
EP2142498A2 (fr) 2007-04-02 2010-01-13 Institute for Oneworld Health Composés inhibiteurs de cftr et leurs utilisations
HUE028426T2 (en) * 2007-08-24 2016-12-28 Vertex Pharma Isothiazolopyridinones for (inter alia) treating cystic fibrosis
SI2217572T1 (sl) 2007-11-16 2014-05-30 Vertex Pharmaceuticals Incorporated Izokinolinski modulatorji prenaĺ alcev z atp-vezavno kaseto
SI2225230T1 (sl) 2007-12-07 2017-03-31 Vertex Pharmaceuticals Incorporated Trdne oblike 3-(6-(1-2,2-difluorobenzo(d)(1,3)dioxol-5-il)ciklopropan- karboksamido)-3-metilpiridin-2-il) benzojske kisline
EP2279029A2 (fr) * 2008-03-25 2011-02-02 The Regents of the University of California Inhibiteurs à petite molécule soluble dans l eau du régulateur de conductance transmembranaire de fibrose kystique
EP2271621B1 (fr) 2008-03-31 2013-11-20 Vertex Pharmaceuticals Incorporated Dérivés de pyridyle en tant que modulateur du cftr
US8236838B2 (en) 2008-04-21 2012-08-07 Institute For Oneworld Health Compounds, compositions and methods comprising isoxazole derivatives
JP2012503005A (ja) * 2008-09-19 2012-02-02 インスティテュート フォア ワンワールド ヘルス イミダゾール誘導体およびトリアゾール誘導体を含む、化合物、組成物ならびに方法。
US8343976B2 (en) 2009-04-20 2013-01-01 Institute For Oneworld Health Compounds, compositions and methods comprising pyrazole derivatives
EP2490696A1 (fr) * 2009-10-20 2012-08-29 INSERM (Institut National de la Santé et de la Recherche Médicale) Méthodes et compositions pharmaceutiques pour le traitement des troubles de l'homéostasie du glucose
US20110251253A1 (en) 2010-03-25 2011-10-13 Vertex Pharmaceuticals Incorporated 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
HRP20211752T1 (hr) 2010-04-07 2022-02-18 Vertex Pharmaceuticals Incorporated Farmaceutski pripravci 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioksol-5-il)ciklopropankarboksamido)-3-metilpiridin-2-il)benzojeve kiseline i njihova primjena
HRP20160682T1 (hr) 2010-04-07 2016-07-29 Vertex Pharmaceuticals Incorporated Čvrsti oblici 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioksol-5-il)ciklopropankarboksiamido)-3-metilpiridin-2-il)benzojeve kiseline
ES2608474T3 (es) 2010-04-22 2017-04-11 Vertex Pharmaceuticals Incorporated Proceso de producción de compuestos indol cycloalkylcarboxamido
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
CA2808501A1 (fr) 2010-08-23 2012-03-01 Vertex Pharmaceuticals Incorporated Composition pharmaceutique a base de (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-yle)cyclopropanecarboxamide et son administration
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
US9056862B2 (en) 2011-05-10 2015-06-16 National University Corporation Kobe University Thioxothiazolidine derivative having Ras function inhibitory effect
IN2014KN00885A (fr) 2011-11-08 2015-10-02 Vertex Pharma
WO2013112804A1 (fr) 2012-01-25 2013-08-01 Vertex Pharmaceuticals Incorporated Formulations d'acide 3-(6-(1-(2,2-difluorobenzo [d] [1,3]dioxol-5-yl) cyclopropanecarboxamido)-3-méthylpyridin-2-yl) benzoïque
WO2014014841A1 (fr) 2012-07-16 2014-01-23 Vertex Pharmaceuticals Incorporated Compositions pharmaceutiques de (r)-1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl)-n-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-méthylpropan-2-yl)-1h-indol-5-yl)cyclopropane- carboxamide et leur administration
LT3470063T (lt) 2012-11-02 2025-02-10 Vertex Pharmaceuticals Incorporated Farmacinės kompozicijos, skirtos cftr nulemtų ligų gydymui
FR2999191B1 (fr) 2012-12-12 2016-02-05 Lesaffre & Cie Souches probiotiques pour le traitement et/ou la prevention de la diarrhee
ES2885181T3 (es) 2014-04-15 2021-12-13 Vertex Pharma Composiciones farmacéuticas para el tratamiento de enfermedades mediadas por el regulador de la conductancia transmembrana de fibrosis quística
TWI735416B (zh) 2014-10-06 2021-08-11 美商維泰克斯製藥公司 囊腫纖維化症跨膜傳導調節蛋白之調節劑
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
JP6894902B2 (ja) 2015-12-24 2021-06-30 ザ・リージエンツ・オブ・ザ・ユニバーシテイー・オブ・カリフオルニア Cftr制御因子及びこの使用方法
CN110267948B (zh) 2016-12-09 2023-12-08 弗特克斯药品有限公司 囊性纤维化跨膜传导调控剂的调节剂、药物组合物、治疗方法和制备所述调节剂的方法
MA49631A (fr) 2017-07-17 2020-05-27 Vertex Pharma Méthodes de traitement de la fibrose kystique
KR102716244B1 (ko) 2017-12-08 2024-10-14 버텍스 파마슈티칼스 인코포레이티드 낭포성 섬유증 막횡단 전도 조절자의 조정제의 제조 방법
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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* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8796321B2 (en) 2008-04-21 2014-08-05 Path Drug Solutions Compounds, compositions and methods comprising oxadiazole derivatives

Also Published As

Publication number Publication date
KR20050061501A (ko) 2005-06-22
EP1549321A2 (fr) 2005-07-06
JP2006503853A (ja) 2006-02-02
EA009847B1 (ru) 2008-04-28
WO2004028480A2 (fr) 2004-04-08
AU2003277162C1 (en) 2009-12-24
NZ538809A (en) 2008-06-30
WO2004028480A3 (fr) 2004-07-01
CN1684686A (zh) 2005-10-19
AP2005003292A0 (en) 2005-06-30
AU2003277162A1 (en) 2004-04-19
CA2500498A1 (fr) 2004-04-08
CN100356922C (zh) 2007-12-26
EP1549321A4 (fr) 2007-05-23
JP4977319B2 (ja) 2012-07-18
BR0314943A (pt) 2005-08-02
AU2003277162B2 (en) 2009-07-16
MXPA05003366A (es) 2005-10-05
PL376147A1 (en) 2005-12-27
EA200500583A1 (ru) 2005-12-29

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