NO20091826L - Fremgangsmater for dosering av oralt aktive 1,2,4-oksadiazol for nonsense mutasjonsuppresjonsterapi - Google Patents

Fremgangsmater for dosering av oralt aktive 1,2,4-oksadiazol for nonsense mutasjonsuppresjonsterapi

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Publication number
NO20091826L
NO20091826L NO20091826A NO20091826A NO20091826L NO 20091826 L NO20091826 L NO 20091826L NO 20091826 A NO20091826 A NO 20091826A NO 20091826 A NO20091826 A NO 20091826A NO 20091826 L NO20091826 L NO 20091826L
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NO
Norway
Prior art keywords
oxadiazole
nonsense
mutational
repression
therapy
Prior art date
Application number
NO20091826A
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English (en)
Other versions
NO342061B1 (no
Inventor
Samit Hirawat
Langdon Miller
Original Assignee
Ptc Therapeutics Inc
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Publication date
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First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=39102963&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=NO20091826(L) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Ptc Therapeutics Inc filed Critical Ptc Therapeutics Inc
Publication of NO20091826L publication Critical patent/NO20091826L/no
Publication of NO342061B1 publication Critical patent/NO342061B1/no

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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
    • A61K31/41Heterocyclic 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
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/02Inorganic compounds
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/10Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/08Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
    • A61K47/12Carboxylic acids; Salts or anhydrides thereof
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    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/32Macromolecular compounds obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. carbomers, poly(meth)acrylates, or polyvinyl pyrrolidone
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/34Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/36Polysaccharides; Derivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/36Polysaccharides; Derivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
    • A61K47/38Cellulose; Derivatives thereof
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0053Mouth and digestive tract, i.e. intraoral and peroral administration
    • AHUMAN NECESSITIES
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    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0087Galenical forms not covered by A61K9/02 - A61K9/7023
    • A61K9/0095Drinks; Beverages; Syrups; Compositions for reconstitution thereof, e.g. powders or tablets to be dispersed in a glass of water; Veterinary drenches
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    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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    • A61P27/16Otologicals
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    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
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    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/08Drugs for disorders of the metabolism for glucose homeostasis
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    • 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)
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  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
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  • Biochemistry (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
  • Physiology (AREA)
  • Nutrition Science (AREA)
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  • Psychiatry (AREA)

Abstract

Den foreliggende oppfinnelsen er relatert til spesifikke doser av og doseringsregimer for å benytte en 1,2,4-oksadiazolbenzosyreforbindelse i behandling eller forebygging av sykdommer assosiert med nonsense-mutasjoner. Spesielt er oppfinnelsen relatert til spesifikke doser og doseringsregimer for anvendelsen av 3-[5-(2-fluorfenyl)-[1,2,4]oksadiazol-3-yl]-benzosyre i pattedyr som har sykdommer assosiert med nonsense-mutasjoner.
NO20091826A 2006-10-12 2009-05-08 Forbindelse for behandling, forebygging eller håndtering av Duchenne muskulær dystrofi NO342061B1 (no)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US85145006P 2006-10-12 2006-10-12
PCT/US2007/021921 WO2008045566A1 (en) 2006-10-12 2007-10-11 Methods for dosing an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy

Publications (2)

Publication Number Publication Date
NO20091826L true NO20091826L (no) 2009-07-08
NO342061B1 NO342061B1 (no) 2018-03-19

Family

ID=39102963

Family Applications (1)

Application Number Title Priority Date Filing Date
NO20091826A NO342061B1 (no) 2006-10-12 2009-05-08 Forbindelse for behandling, forebygging eller håndtering av Duchenne muskulær dystrofi

Country Status (23)

Country Link
US (7) US9226919B2 (no)
EP (2) EP2073805B1 (no)
JP (4) JP2010506840A (no)
KR (4) KR20160128468A (no)
CN (2) CN103211766B (no)
AU (1) AU2007308067B2 (no)
BR (2) BR122019020961B8 (no)
CA (1) CA2675518C (no)
CL (1) CL2007002923A1 (no)
DK (1) DK2073805T3 (no)
ES (1) ES2538359T3 (no)
HR (1) HRP20150590T1 (no)
IL (2) IL198147A (no)
MX (1) MX2009003909A (no)
NO (1) NO342061B1 (no)
PE (4) PE20160947A1 (no)
PL (1) PL2073805T3 (no)
PT (1) PT2073805E (no)
RS (1) RS54025B1 (no)
RU (1) RU2462247C2 (no)
SI (1) SI2073805T1 (no)
TW (1) TWI405570B (no)
WO (1) WO2008045566A1 (no)

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HUE047569T2 (hu) 2003-04-11 2020-04-28 Ptc Therapeutics Inc 1,2,4-oxadiazol-benzoesav vegyület és nonszensz szupresszióra történõ alkalmazása
RU2462246C2 (ru) * 2006-03-30 2012-09-27 ПиТиСи ТЕРАПЬЮТИКС, ИНК. Способы получения функционального белка из днк, имеющей нонсенс-мутацию, и лечения нарушений, ассоциированных с ней
RS54025B1 (sr) * 2006-10-12 2015-10-30 Ptc Therapeutics Inc. Postupci doziranja oralno aktivnog 1,2,4-oksadiazola za supresionu terapiju nonsens mutacije
MY162940A (en) 2009-08-19 2017-07-31 Eisai R&D Man Co Ltd Quinoline derivative-containing pharmaceutical composition
JP6038128B2 (ja) 2011-06-03 2016-12-07 エーザイ・アール・アンド・ディー・マネジメント株式会社 レンバチニブ化合物に対する甲状腺癌対象及び腎臓癌対象の反応性を予測及び評価するためのバイオマーカー
KR102497273B1 (ko) * 2014-03-06 2023-02-07 피티씨 테라퓨틱스, 인크. 1,2,4-옥사디아졸 벤조산의 약학 조성물 및 염
PT3524595T (pt) 2014-08-28 2022-09-19 Eisai R&D Man Co Ltd Derivado de quinolina altamente puro e método para produção do mesmo
LT3263106T (lt) * 2015-02-25 2024-01-10 Eisai R&D Management Co., Ltd. Chinolino darinių kartumo sumažinimo būdas
MX373231B (es) 2015-06-16 2020-05-08 Eisai R&D Man Co Ltd Agente anticancerigeno.
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JP2018531975A (ja) 2015-10-30 2018-11-01 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 てんかんの治療方法
US12303505B2 (en) 2017-02-08 2025-05-20 Eisai R&D Management Co., Ltd. Tumor-treating pharmaceutical composition
RU2019134940A (ru) 2017-05-16 2021-06-16 Эйсай Ар Энд Ди Менеджмент Ко., Лтд. Лечение гепатоцеллюлярной карциномы
EA202090980A1 (ru) 2017-10-19 2020-10-07 СИПЛА ЮЭсЭй, ИНК Синтез антибактериальных аминогликозидных аналогов
EP3713934B1 (en) 2017-11-23 2022-04-13 Universita' degli Studi di Palermo Oxadiazole derivatives for the treatment of genetic diseases due to nonsense mutations
EP3952867A4 (en) * 2019-04-10 2023-01-11 PTC Therapeutics, Inc. METHODS OF TREATMENT OF NONSENSE MUTATION MEDIATED DUCHENNE MUSCULAR DYSTROPHY IN PEDIATRIC PATIENTS
KR102378381B1 (ko) * 2021-08-23 2022-03-24 라이보텍(주) 넌센스-매개 mRNA 분해를 억제하기 위한 화합물
US20240156805A1 (en) * 2021-02-16 2024-05-16 Ribotech Co., Ltd. Compound for inhibiting nonsense-mediated mrna decay
EP4337267A1 (en) * 2021-05-11 2024-03-20 RegenxBio Inc. Treatment of duchenne muscular dystrophy and combinations thereof
KR102665711B1 (ko) * 2021-12-21 2024-05-14 재단법인 아산사회복지재단 Ras 활성도를 이용한 신경섬유종증 치료제에 대한 감수성 예측용 바이오마커 조성물 및 Ras 활성 억제제를 포함하는 신경섬유종증 예방 또는 치료용 약학적 조성물
CN119638642A (zh) * 2023-12-12 2025-03-18 暨南大学 双环类化合物及其药物组合物和应用

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BR122019020961B1 (pt) 2021-03-09
PT2073805E (pt) 2015-07-13
US20180140583A1 (en) 2018-05-24
MX2009003909A (es) 2009-05-25
KR101758341B1 (ko) 2017-07-14
PL2073805T3 (pl) 2015-08-31
RU2462247C2 (ru) 2012-09-27
TWI405570B (zh) 2013-08-21
JP6333227B2 (ja) 2018-05-30
DK2073805T3 (da) 2015-06-15
US20080114039A1 (en) 2008-05-15
PE20160947A1 (es) 2016-09-26
JP6129713B2 (ja) 2017-05-17
CA2675518A1 (en) 2008-04-17
IL198147A0 (en) 2009-12-24
CA2675518C (en) 2017-11-14
KR20150064244A (ko) 2015-06-10
PE20212245A1 (es) 2021-11-24
JP2014058533A (ja) 2014-04-03
IL236519A (en) 2017-03-30
BR122019020961B8 (pt) 2021-07-27
US20170304275A1 (en) 2017-10-26
BRPI0719276A2 (pt) 2014-04-29
US9737513B2 (en) 2017-08-22
EP2073805A1 (en) 2009-07-01
KR20090074231A (ko) 2009-07-06
CN101553226A (zh) 2009-10-07
PE20080982A1 (es) 2008-07-19
KR20160128468A (ko) 2016-11-07
AU2007308067A1 (en) 2008-04-17
HRP20150590T1 (hr) 2015-07-03
US20170049752A1 (en) 2017-02-23
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US20210038572A1 (en) 2021-02-11
IL236519A0 (en) 2015-02-26
RU2009117590A (ru) 2010-11-20
SI2073805T1 (sl) 2015-10-30
CN103211766A (zh) 2013-07-24
US20160101087A1 (en) 2016-04-14
US9877952B2 (en) 2018-01-30
PE20120670A1 (es) 2012-06-01
AU2007308067B2 (en) 2013-05-30
CN101553226B (zh) 2015-05-13
US10172836B2 (en) 2019-01-08
IL198147A (en) 2016-02-29
CL2007002923A1 (es) 2008-06-06
WO2008045566A1 (en) 2008-04-17
EP2073805B1 (en) 2015-03-04
ES2538359T3 (es) 2015-06-19
US9226919B2 (en) 2016-01-05
WO2008045566B1 (en) 2008-06-19
TW200820968A (en) 2008-05-16
CN103211766B (zh) 2015-03-11
HK1170690A1 (en) 2013-03-08
EP2460520A1 (en) 2012-06-06
JP2010506840A (ja) 2010-03-04
US10849885B2 (en) 2020-12-01
RS54025B1 (sr) 2015-10-30
HK1136214A1 (en) 2010-06-25
KR20170100072A (ko) 2017-09-01
NO342061B1 (no) 2018-03-19
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