DK2925317T3 - CFTR-korrektor til behandling af genetiske forstyrrelser, der påvirker tværstribet muskel - Google Patents

CFTR-korrektor til behandling af genetiske forstyrrelser, der påvirker tværstribet muskel Download PDF

Info

Publication number
DK2925317T3
DK2925317T3 DK13801517T DK13801517T DK2925317T3 DK 2925317 T3 DK2925317 T3 DK 2925317T3 DK 13801517 T DK13801517 T DK 13801517T DK 13801517 T DK13801517 T DK 13801517T DK 2925317 T3 DK2925317 T3 DK 2925317T3
Authority
DK
Denmark
Prior art keywords
treatment
genetic disorders
striated muscle
disorders affecting
cftr corrector
Prior art date
Application number
DK13801517T
Other languages
English (en)
Inventor
Dorianna Sandona'
Roberta Sacchetto
Elisa Bianchini
Pompeo Volpe
Romeo Betto
Francesco Mascarello
Original Assignee
Univ Degli Studi Padova
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 Univ Degli Studi Padova filed Critical Univ Degli Studi Padova
Application granted granted Critical
Publication of DK2925317T3 publication Critical patent/DK2925317T3/da

Links

Classifications

    • 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
    • A61K31/426—1,3-Thiazoles
    • 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
    • A61K31/40—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil
    • A61K31/403—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with one nitrogen as the only ring hetero atom, e.g. sulpiride, succinimide, tolmetin, buflomedil condensed with carbocyclic rings, e.g. carbazole
    • A61K31/404—Indoles, e.g. pindolol
    • 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
    • A61K31/427—Thiazoles not condensed and containing further heterocyclic 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
    • 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
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/4427—Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems
    • A61K31/443—Non condensed pyridines; Hydrogenated derivatives thereof containing further heterocyclic ring systems containing a five-membered ring with oxygen as a ring hetero atom
    • 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
    • A61K31/435—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
    • A61K31/4709—Non-condensed quinolines and containing further heterocyclic 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
    • 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
    • A61K31/472—Non-condensed isoquinolines, e.g. papaverine
    • 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
    • A61K31/496—Non-condensed piperazines containing further heterocyclic rings, e.g. rifampin, thiothixene or sparfloxacin
    • 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
    • A61K31/506—Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim not condensed and containing further heterocyclic 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system

Landscapes

  • Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Electrotherapy Devices (AREA)
  • Orthopedics, Nursing, And Contraception (AREA)
DK13801517T 2012-12-03 2013-11-29 CFTR-korrektor til behandling af genetiske forstyrrelser, der påvirker tværstribet muskel DK2925317T3 (da)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
IT002065A ITMI20122065A1 (it) 2012-12-03 2012-12-03 Uso dei correttori del cftr nel trattamento delle patologie del muscolo striato
PCT/EP2013/075158 WO2014086687A1 (en) 2012-12-03 2013-11-29 A cftr corrector for the teatment of genetic disorders affecting striated muscle

Publications (1)

Publication Number Publication Date
DK2925317T3 true DK2925317T3 (da) 2019-11-18

Family

ID=47561727

Family Applications (1)

Application Number Title Priority Date Filing Date
DK13801517T DK2925317T3 (da) 2012-12-03 2013-11-29 CFTR-korrektor til behandling af genetiske forstyrrelser, der påvirker tværstribet muskel

Country Status (5)

Country Link
US (2) US9987256B2 (da)
EP (1) EP2925317B1 (da)
DK (1) DK2925317T3 (da)
IT (1) ITMI20122065A1 (da)
WO (1) WO2014086687A1 (da)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8563573B2 (en) 2007-11-02 2013-10-22 Vertex Pharmaceuticals Incorporated Azaindole derivatives as CFTR modulators
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
RU2569678C2 (ru) 2010-04-22 2015-11-27 Вертекс Фармасьютикалз Инкорпорейтед Способ получения циклоалкилкарбоксамидо-индольных соединений
CN105308031B (zh) * 2013-03-04 2017-10-13 健康科学北方研究所 喹啉磺酰基衍生物及其用途
PL3925607T5 (pl) 2014-04-15 2026-06-01 Vertex Pharmaceuticals Incorporated Kompozycje farmaceutyczne do leczenia chorób, w których pośredniczy mukowiscydozowy przezbłonowy regulator przewodnictwa
TW202140422A (zh) 2014-10-06 2021-11-01 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
EA201791500A1 (ru) 2014-12-31 2018-01-31 Оспекс Фармасьютикалз, Инк. Циклопропанкарбоксамидные модуляторы муковисцидозного трансмембранного регулятора проводимости
EP3737375A1 (en) * 2018-01-11 2020-11-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Combination treatment of sarcoglycanopathies

Family Cites Families (46)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2002053161A1 (en) 2000-12-29 2002-07-11 Alteon, Inc. Method for treating fibrotic diseases or other indications
GB0217280D0 (en) 2002-07-25 2002-09-04 Modrec Internat Holdings Ltd Golf trolley
EP1601657A1 (en) 2003-03-12 2005-12-07 Vertex Pharmaceuticals Incorporated Pyrazole modulators of atp-binding cassette transporters
ATE440825T1 (de) 2003-06-06 2009-09-15 Vertex Pharma Pyrimidin-derivate zur verwendung als modulatoren von atp-bindende kassette transportern
US7541466B2 (en) 2003-12-23 2009-06-02 Genzyme Corporation Tetrahydroisoquinoline derivatives for treating protein trafficking diseases
BRPI0507278A (pt) 2004-01-30 2007-06-26 Vertex Pharma moduladores dos transportadores do cassete de ligação ao atp
US7977322B2 (en) 2004-08-20 2011-07-12 Vertex Pharmaceuticals Incorporated Modulators of ATP-binding cassette transporters
WO2005120497A2 (en) 2004-06-04 2005-12-22 The Regents Of The University Of California Compounds having activity in increasing ion transport by mutant-cftr and uses thereof
EP1814578A2 (en) 2004-11-04 2007-08-08 University of Washington Compositions and methods for treatment of protein misfolding and protein aggregation diseases
RU2394036C2 (ru) * 2004-12-20 2010-07-10 Вайет Аналоги рапамицина и их применение при лечении неврологических, пролиферативных и воспалительных заболеваний
JP5385605B2 (ja) 2005-03-11 2014-01-08 バーテックス ファーマシューティカルズ インコーポレイテッド Atp結合カセットトランスポーターのモジュレーター
CA2600869A1 (en) 2005-03-18 2006-09-28 The Regents Of The University Of California Compounds having activity in correcting mutant-cftr processing and uses thereof
RU2008109031A (ru) 2005-08-11 2009-09-20 Вертекс Фармасьютикалз Инкорпорейтед (Us) Модуляторы муковисцидозного трансмембранного регулятора проводимости
PT2404919E (pt) 2005-11-08 2013-10-22 Vertex Pharma Composto heterocíclico útil como um modulador de transportadores de cassete de ligação a atp
AU2006331565A1 (en) 2005-12-27 2007-07-05 Vertex Pharmaceuticals Incorporated Compounds useful in CFTR assays and methods therewith
BRPI0707719A2 (pt) * 2006-02-10 2011-05-10 Summit Corp Plc uso de um composto ou de um sal farmaceuticamente aceitÁvel do mesmo
US7645789B2 (en) 2006-04-07 2010-01-12 Vertex Pharmaceuticals Incorporated Indole derivatives as CFTR modulators
JP5420395B2 (ja) 2006-04-07 2014-02-19 バーテックス ファーマシューティカルズ インコーポレイテッド Atp結合カセット輸送体の調節剤
FR2903905B1 (fr) * 2006-07-18 2009-03-06 Genethon Ass Loi De 1901 Medicaments pour le traitement des sarcoglycanopathies
MX2009004702A (es) 2006-11-03 2009-06-19 Vertex Pharma Derivados de azaindol como moduladores del regulador de conductancia de transmembrana de fibrosis quistica.
US8563573B2 (en) 2007-11-02 2013-10-22 Vertex Pharmaceuticals Incorporated Azaindole derivatives as CFTR modulators
EP2164840A2 (en) 2007-05-09 2010-03-24 Vertex Pharmaceuticals Incorporated Modulators of cftr
JP5283962B2 (ja) 2007-08-06 2013-09-04 国立大学法人 岡山大学 医薬組成物
US20110177999A1 (en) 2007-08-09 2011-07-21 Vertex Pharmaceuticals Incorporated Therapeutic Combinations Useful in Treating CFTR Related Diseases
AU2008303057B2 (en) 2007-09-24 2014-09-04 The University Of Queensland Molecular delivery vesicle
WO2009051910A1 (en) 2007-10-16 2009-04-23 The Regents Of The University Of California Compounds having activity in increasing ion transport by mutant-cftr and uses thereof
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
WO2009062118A2 (en) 2007-11-07 2009-05-14 Foldrx Pharmaceuticals, Inc. Modulation of protein trafficking
WO2009105234A2 (en) 2008-02-19 2009-08-27 Combinatorx, Incorporated Methods and compositions for the treatment of disorders associated with defects of the cystic fibrosis transmembrane conductance regulator gene or protein
CA2716109C (en) 2008-02-28 2016-07-19 Vertex Pharmaceuticals Incorporated Heteroaryl derivatives as cftr modulators
NZ616097A (en) 2008-03-31 2015-04-24 Vertex Pharma Pyridyl derivatives as cftr modulators
AU2009290848A1 (en) 2008-09-11 2010-03-18 Galapagos Nv Method for identifying compounds useful for increasing the functional activity and cell surface expression of CF-associated mutant Cystic Fibrosis transmembrane conductance regulator
US8415292B2 (en) 2008-10-22 2013-04-09 Trustees Of Dartmouth College Compositions and methods for inhibiting the interaction between CFTR and CAL
US20110257223A1 (en) 2008-10-23 2011-10-20 Vertex Pharmaceuticals Incorporated Modulators of Cystic Fibrosis Transmembrane Conductance Regulator
UA104876C2 (uk) 2008-11-06 2014-03-25 Вертекс Фармасьютікалз Інкорпорейтед Модулятори atф-зв'язувальних касетних транспортерів
AU2009326976A1 (en) 2008-12-12 2010-06-17 Universitatsklinikum Munster Compounds having activity in correcting mutant CFTR cellular processing
WO2010068863A2 (en) 2008-12-12 2010-06-17 Cystic Fibrosis Foundation Therapeutics, Inc. Pyrimidine compounds and methods of making and using same
WO2010151747A1 (en) 2009-06-26 2010-12-29 Cystic Fibrosis Foundation Therapeutics, Inc. Pyrimine compounds and methods of making and using same
WO2011008931A2 (en) 2009-07-15 2011-01-20 Cystic Fibrosis Foundation Therapeutics, Inc. Arylpyrimidine compounds and combination therapy comprising same for treating cystic fibrosis & related disorders
US20110086899A1 (en) * 2009-10-14 2011-04-14 Xenon Pharmaceuticals Inc. Pharmaceutical compositions for oral administration
NZ603042A (en) 2010-04-22 2015-02-27 Vertex Pharma Pharmaceutical compositions comprising cftr modulators and administrations thereof
NZ603044A (en) 2010-04-22 2015-08-28 Vertex Pharma Pharmaceutical compositions comprising cftr modulators and administrations thereof
WO2011133953A1 (en) 2010-04-22 2011-10-27 Vertex Pharmaceuticals Incorporated Pharmaceutical compositions and administrations thereof
US20130131146A1 (en) 2010-04-30 2013-05-23 The Johns Hopkins University Compositions and Methods for Treating Pulmonary Conditions
WO2012021974A1 (en) 2010-08-20 2012-02-23 The Hospital For Sick Children Compositions and methods for treatment of cystic fibrosis and diseases associated with aberrant protein cellular processing
CN104478882A (zh) 2010-09-14 2015-04-01 波兰科学院生物化学与生物物理研究所 作为突变cftr蛋白的调节剂的化合物及其在治疗与cftr蛋白失常相关的疾病中的用途

Also Published As

Publication number Publication date
EP2925317B1 (en) 2019-09-04
EP2925317A1 (en) 2015-10-07
US20180221347A1 (en) 2018-08-09
US9987256B2 (en) 2018-06-05
ITMI20122065A1 (it) 2014-06-04
WO2014086687A1 (en) 2014-06-12
US20150328217A1 (en) 2015-11-19

Similar Documents

Publication Publication Date Title
HRP20211385T8 (hr) Ciljana modifikacija genoma štakora
IL281178B (en) Methods of treating or preventing cholesterol related disorders
LTC2785706I2 (lt) Pakeistieji 4-fenil-piridinai, skirti su nk-receptoriumi susijusių ligų gydymui
LT3470063T (lt) Farmacinės kompozicijos, skirtos cftr nulemtų ligų gydymui
DK2895156T3 (da) Fremgangsmåde til fremstilling af terapeutiske nanopartikler
DK3520805T3 (da) Sammensætninger til behandling af myelofibrose
LT2916860T (lt) Diabeto gydymui skirta kompozicija, apimanti oksintomodulino analogą
DK3329884T3 (da) Implanterbar indretning til behandling af glaukom
LT2887944T (lt) Alopregnanolonas, skirtas atsparios epileptinės būklės gydymui
LT2914255T (lt) Širdies ir kraujagyslių sistemos sutrikimų gydymas arba prevencija, skiriant kolchiciną
DK2701733T3 (da) Forbindelser til behandling af neuropsykiatriske forstyrrelser
DK2818241T3 (da) Fremgangsmåde til fremstilling af behandlingsapparat udstyret med katalysatorbærende honeycombstruktur
DK2836460T3 (da) Fremgangsmåde til fremstilling af graphen
DK2807170T3 (da) Phragmalin-limonoider til behandlingen af seksuel dysfunktion
DK2814495T3 (da) Fremgangsmåde til behandling af tarmsygdomme, der udviser mindst én inflammatorisk komponent
DK3415158T3 (da) Repeat-variable direster til targeting af nukleotider
DK2809782T3 (da) Forbedret fremgangsmåde til fremstilling af makrokugler
DK2900259T3 (da) Glycosidase-regime til behandling af infektionssygdomme
DK3398594T3 (da) Sammensætninger til forbedring af hjernefunktion
DK3524260T3 (da) Farmaceutiske sammensætninger til behandling af manglende appetit
DK2705847T3 (da) Sammensætning til behandling af psoriasis
DK3901164T3 (da) Glutenrelaterede forstyrrelser
DK3590960T3 (da) Igg-stimuleret remyelinisering af perifere nerver
DK2828396T3 (da) Fremgangsmåde til fremstillingen af g-csf
DK2938350T3 (da) Medicinsk formulering til behandling af hypercholesterolæmi