PL2600865T3 - Związek użyteczny do leczenia chorób, w których pośredniczy mutacja nonsensowna oraz kompozycje farmaceutyczne zawierające wymieniony związek - Google Patents

Związek użyteczny do leczenia chorób, w których pośredniczy mutacja nonsensowna oraz kompozycje farmaceutyczne zawierające wymieniony związek

Info

Publication number
PL2600865T3
PL2600865T3 PL11736401T PL11736401T PL2600865T3 PL 2600865 T3 PL2600865 T3 PL 2600865T3 PL 11736401 T PL11736401 T PL 11736401T PL 11736401 T PL11736401 T PL 11736401T PL 2600865 T3 PL2600865 T3 PL 2600865T3
Authority
PL
Poland
Prior art keywords
compound
nonsense
mutation
treatment
pharmaceutical composition
Prior art date
Application number
PL11736401T
Other languages
English (en)
Inventor
Fabrice Lejeune
Benoit Deprez
Terence Beghyn
Sara Sofia Gonzalez-Hilarion
Original Assignee
Institut National De La Sante Et De La Rech Medicale
Pasteur Institut
Univ De Lille
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 Institut National De La Sante Et De La Rech Medicale, Pasteur Institut, Univ De Lille filed Critical Institut National De La Sante Et De La Rech Medicale
Publication of PL2600865T3 publication Critical patent/PL2600865T3/pl

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    • 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
    • A61K31/4353—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 ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/436—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 ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having oxygen as a ring hetero atom, e.g. rapamycin
    • 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
    • A61K31/12—Ketones
    • A61K31/122—Ketones having the oxygen directly attached to a ring, e.g. quinones, vitamin K1, anthralin
    • 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
    • A61K31/335—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/35—Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin having six-membered rings with one oxygen as the only 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
    • A61K31/41—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
    • A61K31/4245—Oxadiazoles
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/02—Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
    • A—HUMAN NECESSITIES
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    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
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    • A—HUMAN NECESSITIES
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    • A61P5/00—Drugs for disorders of the endocrine system
    • A—HUMAN NECESSITIES
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    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/14—Drugs for disorders of the endocrine system of the thyroid hormones, e.g. T3, T4
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    • A61P5/38—Drugs for disorders of the endocrine system of the suprarenal hormones
    • A—HUMAN NECESSITIES
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    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis

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  • Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Biomedical Technology (AREA)
  • Hematology (AREA)
  • Neurosurgery (AREA)
  • Endocrinology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Obesity (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Urology & Nephrology (AREA)
  • Psychology (AREA)
  • Vascular Medicine (AREA)
  • Ophthalmology & Optometry (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Pulmonology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pain & Pain Management (AREA)
  • Emergency Medicine (AREA)
  • Immunology (AREA)
  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
PL11736401T 2010-08-05 2011-07-29 Związek użyteczny do leczenia chorób, w których pośredniczy mutacja nonsensowna oraz kompozycje farmaceutyczne zawierające wymieniony związek PL2600865T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
FR1056472 2010-08-05
EP11736401.8A EP2600865B1 (en) 2010-08-05 2011-07-29 Compound useful for the treatment of nonsense-mutation-mediated diseases and pharmaceutical composition comprising said compound
PCT/EP2011/063126 WO2012016930A1 (en) 2010-08-05 2011-07-29 Compound useful for the treatment of nonsense-mutation-mediated diseases and pharmaceutical composition comprising said compound

Publications (1)

Publication Number Publication Date
PL2600865T3 true PL2600865T3 (pl) 2019-05-31

Family

ID=43447136

Family Applications (1)

Application Number Title Priority Date Filing Date
PL11736401T PL2600865T3 (pl) 2010-08-05 2011-07-29 Związek użyteczny do leczenia chorób, w których pośredniczy mutacja nonsensowna oraz kompozycje farmaceutyczne zawierające wymieniony związek

Country Status (10)

Country Link
US (2) US9913829B2 (pl)
EP (1) EP2600865B1 (pl)
JP (1) JP5877200B2 (pl)
CN (1) CN103189060A (pl)
CA (1) CA2806825C (pl)
DK (1) DK2600865T3 (pl)
ES (1) ES2710854T3 (pl)
IL (1) IL224544B (pl)
PL (1) PL2600865T3 (pl)
WO (1) WO2012016930A1 (pl)

Families Citing this family (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2659763T3 (es) 2011-02-14 2018-03-19 The Regents Of The University Of Michigan Composiciones y procedimientos para el tratamiento de obesidad y trastornos relacionados
PE20151062A1 (es) 2012-12-13 2015-08-05 Novartis Ag PIRIMIDO-[4,5-b]-QUINOLINA-4,5(3H,10H)-DIONAS COMO SUPRESORAS DE MUTACION SIN SENTIDO
WO2014102778A2 (en) 2012-12-24 2014-07-03 Ramot At Tel-Aviv University Ltd. Agents for treating genetic diseases resulting from nonsense mutations, and methods for identifying the same
US9365581B2 (en) 2013-05-02 2016-06-14 The Regents Of The University Of Michigan Deuterated amlexanox
CA2938126A1 (en) * 2014-02-07 2015-08-13 The Regents Of The University Of Michigan Combinations of ikke/tbk1 inhibitors with beta adrenergic agonists or sympathetic nervous system activators
WO2015186061A1 (en) 2014-06-03 2015-12-10 Novartis Ag Pyridopyrimidinedione derivatives
EP3152208B1 (en) * 2014-06-03 2019-05-08 Novartis AG Pyrimido[4,5-b]quinoline-4,5(3h,10h)-dione derivatives as suppressors of non-sens mutations
CN106459043B (zh) * 2014-06-03 2018-12-28 诺华股份有限公司 萘啶二酮衍生物
WO2015188037A1 (en) * 2014-06-06 2015-12-10 Ptc Therapeutics, Inc. Methods of treatment using a 1,2,4-oxadiazole benzoic acid
WO2015198329A1 (en) 2014-06-25 2015-12-30 Bio Blast Pharma Ltd. Injectable formulations for intrathecal administration of antibiotic agents
WO2016073545A1 (en) 2014-11-06 2016-05-12 Concert Pharmaceuticals, Inc. Phenyloxadiazole benzoic acids
CN104450727A (zh) * 2014-11-13 2015-03-25 深圳华大基因科技有限公司 X-连锁低磷酸盐血症性佝偻病的致病基因及其编码蛋白质和应用
HK1251965A1 (zh) * 2015-10-30 2019-05-10 Ptc医疗公司 用於治疗癫痫的方法
WO2017112954A1 (en) 2015-12-23 2017-06-29 Moonshot Pharma Llc Methods for inducing an immune response by promoting premature termination codon read-through
EP3407974A4 (en) * 2016-01-29 2019-08-28 The Regents Of The University Of Michigan Amlexanox Analogs
WO2018050706A1 (en) * 2016-09-13 2018-03-22 Marco Cipolli Method of treatment of shwachman-diamond syndrome
FR3057776A1 (fr) 2016-10-21 2018-04-27 Museum National D'histoire Naturelle Lepista flaccida destine a etre utilise dans le traitement d'une maladie due a une mutation non-sens
FR3057774B1 (fr) 2016-10-21 2025-11-07 Museum Nat Dhistoire Naturelle Derives de la purine pour leur utilisation dans le traitement ou la prevention de maladies dues a une mutation non-sens
EP3641752A4 (en) * 2017-06-22 2021-03-17 Moonshot Pharma LLC METHOD OF TREATMENT OF CANCER WITH COMPOSITIONS WITH AMLEXANOX AND IMMUNE MODULATORS
CN109200053A (zh) * 2018-11-17 2019-01-15 王海玲 一种抗癫痫外用药物及其用途

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2887054T3 (es) 2003-04-11 2021-12-21 Ptc Therapeutics Inc Compuesto de ácido 1,2,4-oxadiazol benzoico y su uso para la supresión sin sentido y el tratamiento de enfermedades
CA2603402C (en) * 2005-04-08 2017-10-31 Ptc Therapeutics, Inc. Compositions of an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy
US7772255B2 (en) 2005-12-13 2010-08-10 Supratek Pharma, Inc. Method of treating tumors with azaxanthones
WO2008021210A2 (en) 2006-08-11 2008-02-21 Combinatorx, Incorporated Methods and compositions for the treatment of neurodegenerative disorders
FR2912745A1 (fr) 2007-02-19 2008-08-22 Centre Nat Rech Scient Nouveaux composes derives d'indole et compositions pharmaceutiques les contenant
CA2693628A1 (en) * 2007-07-11 2009-01-15 Cardoz Ab New combination for use in the treatment of imflammatory disorders
WO2009151569A2 (en) 2008-06-09 2009-12-17 Combinatorx, Incorporated Beta adrenergic receptor agonists for the treatment of b-cell proliferative disorders
WO2010062366A1 (en) * 2008-10-31 2010-06-03 Arginetix, Inc. Compositions and methods of treating endothelial disorders
US20120125325A1 (en) * 2009-06-01 2012-05-24 Robin Mark Bannister The use of amlexanox in the therapy of neutrophil-driven diseases

Also Published As

Publication number Publication date
CA2806825A1 (en) 2012-02-09
ES2710854T3 (es) 2019-04-29
US10265302B2 (en) 2019-04-23
JP5877200B2 (ja) 2016-03-02
IL224544B (en) 2018-07-31
CN103189060A (zh) 2013-07-03
JP2013535479A (ja) 2013-09-12
WO2012016930A1 (en) 2012-02-09
EP2600865B1 (en) 2018-11-14
US9913829B2 (en) 2018-03-13
US20180147191A1 (en) 2018-05-31
EP2600865A1 (en) 2013-06-12
DK2600865T3 (en) 2019-02-25
CA2806825C (en) 2018-08-21
US20130217717A1 (en) 2013-08-22

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