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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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    • 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
    • A61K31/435Heterocyclic 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
    • A61K31/4353Heterocyclic 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
    • A61K31/436Heterocyclic 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/12Ketones
    • A61K31/122Ketones having the oxygen directly attached to a ring, e.g. quinones, vitamin K1, anthralin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/335Heterocyclic compounds having oxygen as the only ring hetero atom, e.g. fungichromin
    • A61K31/35Heterocyclic 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
    • 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
    • A61K31/41Heterocyclic 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
    • A61K31/4245Oxadiazoles
    • 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
    • A61K31/435Heterocyclic 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
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    • AHUMAN NECESSITIES
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    • A61P13/02Drugs for disorders of the urinary system of urine or of the urinary tract, e.g. urine acidifiers
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    • A61P3/10Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
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    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/12Drugs for disorders of the metabolism for electrolyte homeostasis
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    • A61P35/00Antineoplastic agents
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    • AHUMAN NECESSITIES
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    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
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    • A61P5/00Drugs for disorders of the endocrine system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P5/14Drugs for disorders of the endocrine system of the thyroid hormones, e.g. T3, T4
    • AHUMAN NECESSITIES
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    • A61P7/02Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • AHUMAN NECESSITIES
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    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • AHUMAN NECESSITIES
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    • A61P7/06Antianaemics
    • AHUMAN NECESSITIES
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    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs 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)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Epidemiology (AREA)
  • Neurology (AREA)
  • Hematology (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Endocrinology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Obesity (AREA)
  • Cardiology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Urology & Nephrology (AREA)
  • Immunology (AREA)
  • Pulmonology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Ophthalmology & Optometry (AREA)
  • Vascular Medicine (AREA)
  • Pain & Pain Management (AREA)
  • Psychology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Emergency Medicine (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)

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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
BR112015015075A2 (pt) 2012-12-24 2019-01-15 Univ Ramot agentes para tratar doenças genéticas resultantes de mutações sem sentido e métodos para identificar as mesmas.
WO2014179734A1 (en) 2013-05-02 2014-11-06 The Regents Of The University Of Michigan Deuterated amlexanox
WO2015119624A1 (en) * 2014-02-07 2015-08-13 The Regents Of The University Of Michigan COMBINATIONS OF IKKε/TBK1 INHIBITORS WITH BETA ADRENERGIC AGONISTS OR SYMPATHETIC NERVOUS SYSTEM ACTIVATORS
CU24438B1 (es) 2014-06-03 2019-09-04 Novartis Ag Compuestos derivados de 1,6 naftiridina-diona activos como supresores de mutación sin sentido
US9884862B2 (en) 2014-06-03 2018-02-06 Novartis Ag Pyridopyrimidinedione derivatives
US9896448B2 (en) 2014-06-03 2018-02-20 Novartis Ag Pyrimido[4,5-b]quinoline-4,5(3H, 10H)-dione derivatives
AR100771A1 (es) * 2014-06-06 2016-11-02 Ptc Therapeutics Inc Métodos de tratamiento que utilizan el ácido 1,2,4-oxadiazolbenzoico
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
KR20180081516A (ko) * 2015-10-30 2018-07-16 피티씨 테라퓨틱스, 인크. 간질 치료 방법
CA3048193A1 (en) 2015-12-23 2017-06-29 Moonshot Pharma Llc Methods for inducing an immune response by promoting premature termination codon read-through
US10214536B2 (en) 2016-01-29 2019-02-26 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
JP2020525434A (ja) * 2017-06-22 2020-08-27 ムーンショット ファーマ エルエルシー アンレキサノクス及び免疫調節剤を含む組成物で癌を治療する方法
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
EP3178816A1 (en) * 2003-04-11 2017-06-14 PTC Therapeutics, Inc. 1,2,4-oxadiazole benzoic acid compound and its use for nonsense suppression and the treatment of disease
JP5800451B2 (ja) * 2005-04-08 2015-10-28 ピーティーシー セラピューティクス,インコーポレーテッド ナンセンス突然変異抑制治療用の、経口的に活性な1,2,4−オキサジアゾール組成物
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
EP2175845A2 (en) * 2007-07-11 2010-04-21 Cardoz AB New combination for use in the treatment of inflammatory disorders
US20100009934A1 (en) 2008-06-09 2010-01-14 Combinatorx, Incorporated Beta adrenergic receptor agonists for the treatment of b-cell proliferative disorders
US20120065165A1 (en) * 2008-10-31 2012-03-15 Arginetix, Inc Compositions and methods of treating endothelial disorders
JP2012528844A (ja) * 2009-06-01 2012-11-15 バイオコピア リミテッド 好中球によって引き起こされる疾患の治療におけるアンレキサノクスの使用

Also Published As

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

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