WO2006101740A2 - Compounds having activity in correcting mutant-cftr processing and uses thereof - Google Patents

Compounds having activity in correcting mutant-cftr processing and uses thereof Download PDF

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Publication number
WO2006101740A2
WO2006101740A2 PCT/US2006/008267 US2006008267W WO2006101740A2 WO 2006101740 A2 WO2006101740 A2 WO 2006101740A2 US 2006008267 W US2006008267 W US 2006008267W WO 2006101740 A2 WO2006101740 A2 WO 2006101740A2
Authority
WO
WIPO (PCT)
Prior art keywords
group
cftr
pharmaceutical composition
pharmaceutically acceptable
chosen
Prior art date
Application number
PCT/US2006/008267
Other languages
English (en)
French (fr)
Other versions
WO2006101740A3 (en
Inventor
Alan Verkman
Nicoletta Pedemonte
Luis J.V. Galietta
Original Assignee
The Regents Of The University Of California
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 The Regents Of The University Of California filed Critical The Regents Of The University Of California
Priority to JP2008501913A priority Critical patent/JP2008538107A/ja
Priority to MX2007011512A priority patent/MX2007011512A/es
Priority to BRPI0608453-2A priority patent/BRPI0608453A2/pt
Priority to US11/908,591 priority patent/US8143295B2/en
Priority to CA002600869A priority patent/CA2600869A1/en
Priority to AU2006227833A priority patent/AU2006227833A1/en
Priority to EP06737438A priority patent/EP1863522A4/en
Publication of WO2006101740A2 publication Critical patent/WO2006101740A2/en
Priority to IL185999A priority patent/IL185999A0/en
Publication of WO2006101740A3 publication Critical patent/WO2006101740A3/en

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D277/00—Heterocyclic compounds containing 1,3-thiazole or hydrogenated 1,3-thiazole rings
    • C07D277/60—Heterocyclic compounds containing 1,3-thiazole or hydrogenated 1,3-thiazole rings condensed with carbocyclic rings or ring systems
    • C07D277/62—Benzothiazoles
    • C07D277/68—Benzothiazoles 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 in position 2
    • C07D277/82—Nitrogen atoms
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/41—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/425—Thiazoles
    • A61K31/428—Thiazoles condensed with carbocyclic rings
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic 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
    • A61K31/47—Quinolines; Isoquinolines
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/517—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with carbocyclic ring systems, e.g. quinazoline, perimidine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P11/00—Drugs for disorders of the respiratory system
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D417/00—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00
    • C07D417/02—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00 containing two hetero rings
    • C07D417/04—Heterocyclic compounds containing two or more hetero rings, at least one ring having nitrogen and sulfur atoms as the only ring hetero atoms, not provided for by group C07D415/00 containing two hetero rings directly linked by a ring-member-to-ring-member bond

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Pulmonology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Quinoline Compounds (AREA)
  • Thiazole And Isothizaole Compounds (AREA)
  • Plural Heterocyclic Compounds (AREA)
PCT/US2006/008267 2005-03-18 2006-03-08 Compounds having activity in correcting mutant-cftr processing and uses thereof WO2006101740A2 (en)

Priority Applications (8)

Application Number Priority Date Filing Date Title
JP2008501913A JP2008538107A (ja) 2005-03-18 2006-03-08 変異型ｃｆｔｒプロセシングの修正において活性を有する化合物及びその用途
MX2007011512A MX2007011512A (es) 2005-03-18 2006-03-08 Compuestos que tienen actividad para corregir procesamiento de cftr-mutante y usos de los mismos.
BRPI0608453-2A BRPI0608453A2 (pt) 2005-03-18 2006-03-08 compostos tendo atividade na correção de processamento de cftr mutante e usos destes
US11/908,591 US8143295B2 (en) 2005-03-18 2006-03-08 Compounds having activity in correcting mutant-CFTR processing and uses thereof
CA002600869A CA2600869A1 (en) 2005-03-18 2006-03-08 Compounds having activity in correcting mutant-cftr processing and uses thereof
AU2006227833A AU2006227833A1 (en) 2005-03-18 2006-03-08 Compounds having activity in correcting mutant-CFTR processing and uses thereof
EP06737438A EP1863522A4 (en) 2005-03-18 2006-03-08 CORRECTIVE TO THE PROCESSING OF MUTANT CFTR IMPACT COMPOUNDS AND ITS APPLICATIONS
IL185999A IL185999A0 (en) 2005-03-18 2007-09-17 Compounds having activity in correcting mutant-cftr processing and uses thereof

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US66350105P 2005-03-18 2005-03-18
US60/663,501 2005-03-18

Publications (2)

Publication Number Publication Date
WO2006101740A2 true WO2006101740A2 (en) 2006-09-28
WO2006101740A3 WO2006101740A3 (en) 2009-06-04

Family

ID=37024308

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2006/008267 WO2006101740A2 (en) 2005-03-18 2006-03-08 Compounds having activity in correcting mutant-cftr processing and uses thereof

Country Status (12)

Country Link
US (1) US8143295B2 (US20080318984A1-20081225-C00018.png)
EP (1) EP1863522A4 (US20080318984A1-20081225-C00018.png)
JP (1) JP2008538107A (US20080318984A1-20081225-C00018.png)
KR (1) KR20070114820A (US20080318984A1-20081225-C00018.png)
CN (1) CN101605543A (US20080318984A1-20081225-C00018.png)
AU (1) AU2006227833A1 (US20080318984A1-20081225-C00018.png)
BR (1) BRPI0608453A2 (US20080318984A1-20081225-C00018.png)
CA (1) CA2600869A1 (US20080318984A1-20081225-C00018.png)
IL (1) IL185999A0 (US20080318984A1-20081225-C00018.png)
MX (1) MX2007011512A (US20080318984A1-20081225-C00018.png)
RU (1) RU2007138584A (US20080318984A1-20081225-C00018.png)
WO (1) WO2006101740A2 (US20080318984A1-20081225-C00018.png)

Cited By (53)

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WO2007075946A1 (en) * 2005-12-27 2007-07-05 Vertex Pharmaceuticals Incorporated Compounds useful in cftr assays and methods therewith
WO2009023509A2 (en) * 2007-08-09 2009-02-19 Vertex Pharmaceuticals Incorporated Therapeutic combinations useful in treating cftr related diseases
WO2009046784A1 (en) 2007-10-09 2009-04-16 Merck Patent Gmbh Pyridine derivatives useful as glucokinase activators
WO2009051909A1 (en) * 2007-10-16 2009-04-23 The Regents Of The University Of California Compounds having activity in correcting mutant-cftr cellular processing and uses thereof
WO2009150137A2 (en) 2008-06-10 2009-12-17 Novartis Ag Organic compounds
WO2011050325A1 (en) 2009-10-22 2011-04-28 Vertex Pharmaceuticals Incorporated Compositions for treatment of cystic fibrosis and other chronic diseases
EP2332933A1 (en) 2007-05-07 2011-06-15 Novartis AG Epithelial sodium channel (ENaC) inhibitors
WO2011119984A1 (en) 2010-03-25 2011-09-29 Vertex Pharmaceuticals Incorporated Solid forms of (r)-1(2,2-difluorobenzo[d][1,3]dioxol-5-yl)-n-(1-(2,3-dihyderoxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1h-indol-5-yl) cyclopropanecarboxamide
WO2011127290A2 (en) 2010-04-07 2011-10-13 Vertex Pharmaceuticals Incorporated Solid forms of 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl) cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid
WO2011127241A2 (en) 2010-04-07 2011-10-13 Vertex Pharmaceuticals Incorporated Pharmaceutical compositions of 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl) cyclopropanecarboxamido)-3-methylpyriodin-2-yl)benzoic acid and administration thereof
WO2011133751A2 (en) 2010-04-22 2011-10-27 Vertex Pharmaceuticals Incorporated Process of producing cycloalkylcarboxamido-indole compounds
WO2012027247A2 (en) 2010-08-23 2012-03-01 Vertex Pharmaceuticals Incorporated Pharmaceutical composition of (r)-1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl)-n-(1-(2,3-dihydroxy propyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1h-indol-5-yl) cyclopropanecarboxamide and administration therof
WO2012036573A2 (en) 2010-09-14 2012-03-22 Instytut Biochemii I Biofizyki Pan Compounds as modulators of a mutant cftr protein and their use for treating diseases associated with cftr protein malfunction
WO2012035158A1 (en) 2010-09-17 2012-03-22 Novartis Ag Pyrazine derivatives as enac blockers
EP2444120A1 (en) 2007-12-10 2012-04-25 Novartis AG Spirocyclic amiloride analogues as ENac blockers
US8283351B2 (en) 2007-04-02 2012-10-09 Institute For Oneworld Health Cyclic and acyclic hydrazine derivatives compositions including them and uses thereof
WO2012170061A1 (en) 2011-06-08 2012-12-13 Vertex Pharmaceuticals Incorporated Formulations of (r)-1-(2,2-diflurobenzo[d][1,3] dioxol-5-yl)-n-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methyl propan-2-yl)-1h-indol-5-yl)cyclopropanecarboxamide
US8343976B2 (en) 2009-04-20 2013-01-01 Institute For Oneworld Health Compounds, compositions and methods comprising pyrazole derivatives
WO2013003360A1 (en) * 2011-06-29 2013-01-03 Janssen Pharmaceutica Nv Methods of treatment using allosteric processing inhibitors for matrix metalloproteinases
EP2578571A1 (en) 2007-11-16 2013-04-10 Vertex Pharmaceuticals Incorporated Isoquinoline modulators of ATP-binding cassette transporters
WO2013070961A1 (en) 2011-11-08 2013-05-16 Vertex Pharmaceuticals Incorporated Modulators of atp-binding cassette transporters
EP2615085A1 (en) 2008-03-31 2013-07-17 Vertex Pharmaceuticals Incorporated Pyridyl derivatives as CFTR modulators
WO2013112804A1 (en) 2012-01-25 2013-08-01 Vertex Pharmaceuticals Incorporated Formulations of 3-(6-(1-(2.2-difluorobenzo[d][1,3]dioxol-5-yl) cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid
WO2013140319A1 (en) 2012-03-19 2013-09-26 Novartis Ag Crystalline form of a succinate salt
WO2014071122A1 (en) 2012-11-02 2014-05-08 Vertex Pharmaceuticals Incorporated Pharmaceutical compositions for the treatment of cftr mediated diseases
WO2014086687A1 (en) 2012-12-03 2014-06-12 Universita' Degli Studi Di Padova A cftr corrector for the teatment of genetic disorders affecting striated muscle
US8753857B2 (en) 2011-06-29 2014-06-17 Janssen Pharmaceutica N.V. Crystal structure of the pro form of a matrix metalloproteinase and an allosteric processing inhibitor
US8798939B2 (en) 2011-06-29 2014-08-05 Janssen Pharmaceutica N.V. Methods for designing, selecting and/or optimizing allosteric processing inhibitors for matrix metalloproteinases
US8952049B2 (en) 2006-04-07 2015-02-10 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
WO2015160787A1 (en) 2014-04-15 2015-10-22 Vertex Pharmaceuticals Incorporated Pharmaceutical compositions for the treatment of cystic fibrosis transmembrane conductance regulator mediated diseases
WO2016057572A1 (en) 2014-10-06 2016-04-14 Mark Thomas Miller Modulators of cystic fibrosis transmembrane conductance regulator
WO2016087665A2 (en) 2014-12-05 2016-06-09 Centre National De La Recherche Scientifique (Cnrs) Compounds for treating cystic fibrosis
WO2016123253A1 (en) * 2015-01-27 2016-08-04 Rutgers, The State University Of New Jersey Hydrazne derivatives for the treatment of cancer
WO2016129005A1 (en) * 2015-02-09 2016-08-18 Luigina Romani Thymosin alpha 1 for use in treatment of cystic fibrosis
ITUB20153714A1 (it) * 2015-09-18 2017-03-18 Luigina Romani Timosina alfa 1 per l?uso nel trattamento della fibrosi cistica.
WO2017060880A1 (en) * 2015-10-09 2017-04-13 AbbVie S.à.r.l. Potentiator-corrector combinations useful in the treatment of cystic fibrosis
EP3170818A1 (en) 2007-12-07 2017-05-24 Vertex Pharmaceuticals Incorporated Solid forms of 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl) cyclopropanecarboxamido)-3-methylpyridin-2-yl) benzoic acid
US9670163B2 (en) 2005-12-28 2017-06-06 Vertex Pharmaceuticals Incorporated Solid forms of N-[2,4-bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinoline-3-carboxamide
US9701639B2 (en) 2014-10-07 2017-07-11 Vertex Pharmaceuticals Incorporated Co-crystals of modulators of cystic fibrosis transmembrane conductance regulator
US9732080B2 (en) 2006-11-03 2017-08-15 Vertex Pharmaceuticals Incorporated Azaindole derivatives as CFTR modulators
US9751839B2 (en) 2009-03-20 2017-09-05 Vertex Pharmaceuticals Incorporated Process for making modulators of cystic fibrosis transmembrane conductance regulator
US9758510B2 (en) 2006-04-07 2017-09-12 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
US10022352B2 (en) 2006-04-07 2018-07-17 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
US10058546B2 (en) 2012-07-16 2018-08-28 Vertex Pharmaceuticals Incorporated Pharmaceutical compositions of (R)-1-(2,2-difluorobenzo[D][1,3]dioxo1-5-y1)-N-(1-(2,3-dihydroxypropy1)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-y1)-1H-indol-5-y1) cyclopropanecarbox-amide and administration thereof
US10081621B2 (en) 2010-03-25 2018-09-25 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
US10221133B2 (en) 2015-01-27 2019-03-05 Rutgers, The State University Of New Jersey (Thio, oxo, and seleno) semicarbazone complexes with zinc and their use for treating cancer
US10272046B2 (en) 2012-02-27 2019-04-30 Vertex Pharmaceuticals Incorporated Pharmaceutical composition and administrations thereof
WO2019138012A1 (en) 2018-01-11 2019-07-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Combination treatment of sarcoglycanopathies
WO2020049189A1 (en) 2018-09-09 2020-03-12 Qanatpharma Gmbh Use of cftr modulators for treating cerebrovascular conditions
US10604480B2 (en) 2015-01-27 2020-03-31 Rutgers, The State University Of New Jersey (Thio, oxo, and seleno) semicarbazone derivatives and their use for treating cancer
US10646481B2 (en) 2008-08-13 2020-05-12 Vertex Pharmaceuticals Incorporated Pharmaceutical composition and administrations thereof
US10662192B2 (en) 2004-06-24 2020-05-26 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
US10729671B2 (en) 2015-01-27 2020-08-04 Rutgers, The State University Of New Jersey Zinc complexes of hydrazones and (thio)semicarbazones and their use for the treatment of cancer

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Cited By (105)

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US10662192B2 (en) 2004-06-24 2020-05-26 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
WO2007075946A1 (en) * 2005-12-27 2007-07-05 Vertex Pharmaceuticals Incorporated Compounds useful in cftr assays and methods therewith
US11291662B2 (en) 2005-12-28 2022-04-05 Vertex Pharmaceuticals Incorporated Solid forms of n-[2,4-bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinoline-3-carboxamide
US9670163B2 (en) 2005-12-28 2017-06-06 Vertex Pharmaceuticals Incorporated Solid forms of N-[2,4-bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinoline-3-carboxamide
US9931334B2 (en) 2005-12-28 2018-04-03 Vertex Pharmaceuticals Incorporated Solid forms of N[2,4-bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinoline-3-carboxamide
US10537565B2 (en) 2005-12-28 2020-01-21 Vertex Pharmaceuticals Incorporated Solid forms of N-[2,4-bis(1,1-dimethylethyl)-5-hydroxyphenyl]-1,4-dihydro-4-oxoquinoline-3-carboxamide
US9974781B2 (en) 2006-04-07 2018-05-22 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
US8952049B2 (en) 2006-04-07 2015-02-10 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
US10239867B2 (en) 2006-04-07 2019-03-26 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
US11639347B2 (en) 2006-04-07 2023-05-02 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
US10022352B2 (en) 2006-04-07 2018-07-17 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
US9758510B2 (en) 2006-04-07 2017-09-12 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
US10987348B2 (en) 2006-04-07 2021-04-27 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
US10975061B2 (en) 2006-04-07 2021-04-13 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
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