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 mutasjonsuppresjonsterapiInfo
- 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
- Authority
- NO
- Norway
- Prior art keywords
- oxadiazole
- nonsense
- mutational
- repression
- therapy
- Prior art date
Links
- BBVIDBNAYOIXOE-UHFFFAOYSA-N 1,2,4-oxadiazole Chemical compound C=1N=CON=1 BBVIDBNAYOIXOE-UHFFFAOYSA-N 0.000 title 1
- 238000000034 method Methods 0.000 title 1
- 230000000869 mutational effect Effects 0.000 title 1
- 238000002560 therapeutic procedure Methods 0.000 title 1
- 108020004485 Nonsense Codon Proteins 0.000 abstract 2
- 230000037434 nonsense mutation Effects 0.000 abstract 2
- -1 1,2,4-oxadiazole benzoic acid compound Chemical class 0.000 abstract 1
- OOUGLTULBSNHNF-UHFFFAOYSA-N 3-[5-(2-fluorophenyl)-1,2,4-oxadiazol-3-yl]benzoic acid Chemical compound OC(=O)C1=CC=CC(C=2N=C(ON=2)C=2C(=CC=CC=2)F)=C1 OOUGLTULBSNHNF-UHFFFAOYSA-N 0.000 abstract 1
- 241000124008 Mammalia Species 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 230000006806 disease prevention Effects 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
Classifications
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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
- A61K31/41—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
-
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
- A61K47/02—Inorganic compounds
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- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
- A61K47/10—Alcohols; Phenols; Salts thereof, e.g. glycerol; Polyethylene glycols [PEG]; Poloxamers; PEG/POE alkyl ethers
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- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/08—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing oxygen, e.g. ethers, acetals, ketones, quinones, aldehydes, peroxides
- A61K47/12—Carboxylic acids; Salts or anhydrides thereof
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- A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
- A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
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- A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
- A61K47/32—Macromolecular compounds obtained by reactions only involving carbon-to-carbon unsaturated bonds, e.g. carbomers, poly(meth)acrylates, or polyvinyl pyrrolidone
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- A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
- A61K47/34—Macromolecular 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
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- A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
- A61K47/36—Polysaccharides; Derivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
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- A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
- A61K47/36—Polysaccharides; Derivatives thereof, e.g. gums, starch, alginate, dextrin, hyaluronic acid, chitosan, inulin, agar or pectin
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- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
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- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Engineering & Computer Science (AREA)
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- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Epidemiology (AREA)
- Inorganic Chemistry (AREA)
- Immunology (AREA)
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- Hematology (AREA)
- Neurosurgery (AREA)
- Biomedical Technology (AREA)
- Heart & Thoracic Surgery (AREA)
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- Biochemistry (AREA)
- Molecular Biology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Physiology (AREA)
- Nutrition Science (AREA)
- Pulmonology (AREA)
- Hospice & Palliative Care (AREA)
- Transplantation (AREA)
- Endocrinology (AREA)
- Vascular Medicine (AREA)
- Urology & Nephrology (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.
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) | KR20090074231A (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) |
Families Citing this family (20)
| 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 |
| CN101505739A (zh) * | 2006-03-30 | 2009-08-12 | Ptc医疗公司 | 由具有无义突变的dna生产功能蛋白的方法以及相关疾病的治疗 |
| EP2073805B1 (en) * | 2006-10-12 | 2015-03-04 | PTC Therapeutics, Inc. | Methods for dosing an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy |
| EP2714937B1 (en) | 2011-06-03 | 2018-11-14 | Eisai R&D Management Co., Ltd. | Biomarkers for predicting and assessing responsiveness of thyroid and kidney cancer subjects to lenvatinib compounds |
| TWI636978B (zh) | 2014-03-06 | 2018-10-01 | 美商Ptc治療公司 | 1,2,4-二唑苯甲酸之鹽及醫藥組合物 |
| CN113683564A (zh) | 2014-08-28 | 2021-11-23 | 卫材R&D管理有限公司 | 高纯度喹啉衍生物及其生产方法 |
| LT3263106T (lt) * | 2015-02-25 | 2024-01-10 | Eisai R&D Management Co., Ltd. | Chinolino darinių kartumo sumažinimo būdas |
| CN107801379B (zh) | 2015-06-16 | 2021-05-25 | 卫材R&D管理有限公司 | 抗癌剂 |
| RU2718048C2 (ru) | 2015-08-20 | 2020-03-30 | Эйсай Ар Энд Ди Менеджмент Ко., Лтд. | Противоопухолевое терапевтическое средство |
| KR20180081516A (ko) | 2015-10-30 | 2018-07-16 | 피티씨 테라퓨틱스, 인크. | 간질 치료 방법 |
| WO2018147275A1 (ja) | 2017-02-08 | 2018-08-16 | エーザイ・アール・アンド・ディー・マネジメント株式会社 | 腫瘍治療用医薬組成物 |
| RU2019134940A (ru) | 2017-05-16 | 2021-06-16 | Эйсай Ар Энд Ди Менеджмент Ко., Лтд. | Лечение гепатоцеллюлярной карциномы |
| TWI801438B (zh) | 2017-10-19 | 2023-05-11 | 中國大陸商軒竹(北京)醫藥科技有限公司 | 抗菌胺基糖苷類似物之合成 |
| US11203578B2 (en) | 2017-11-23 | 2021-12-21 | Universita'degli Studi Dipalermo | Oxadiazole derivatives for the treatment of genetic diseases due to nonsense mutations |
| KR20210151131A (ko) * | 2019-04-10 | 2021-12-13 | 피티씨 테라퓨틱스, 인크. | 소아 환자에서 넌센스 돌연변이 매개된 뒤센 근이영양증의 치료 방법 |
| WO2022177267A1 (ko) * | 2021-02-16 | 2022-08-25 | 라이보텍(주) | 넌센스-매개 mrna 분해를 억제하기 위한 화합물 |
| KR102378381B1 (ko) * | 2021-08-23 | 2022-03-24 | 라이보텍(주) | 넌센스-매개 mRNA 분해를 억제하기 위한 화합물 |
| WO2022241030A1 (en) * | 2021-05-11 | 2022-11-17 | 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 |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6660753B2 (en) * | 1999-08-19 | 2003-12-09 | Nps Pharmaceuticals, Inc. | Heteropolycyclic compounds and their use as metabotropic glutamate receptor antagonists |
| AU2002313633B2 (en) * | 2001-06-08 | 2007-03-01 | Cytovia, Inc. | Substituted 3-aryl-5-aryl-[1,2,4]-oxadiazoles and analogs |
| MY151199A (en) * | 2001-11-02 | 2014-04-30 | Rigel Pharmaceuticals Inc | Substituted diphenyl heterocycles useful for treating hcv infection |
| US20040132726A1 (en) * | 2002-08-09 | 2004-07-08 | Astrazeneca Ab And Nps Pharmaceuticals, Inc. | New compounds |
| GB0303503D0 (en) * | 2003-02-14 | 2003-03-19 | Novartis Ag | Organic compounds |
| 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 |
| US20050075375A1 (en) * | 2003-05-14 | 2005-04-07 | Anadys Pharmaceuticals, Inc. | Heterocyclic compounds for treating hepatitis C virus |
| KR20060123493A (ko) * | 2003-12-23 | 2006-12-01 | 알자 코포레이션 | 제어된 전달을 위한 약물 조성물의 용해도를 증가시키는방법 및 제형 |
| 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 |
| US20070010553A1 (en) * | 2005-07-01 | 2007-01-11 | Astrazeneca Ab | New use |
| DK2059513T3 (da) | 2006-09-08 | 2013-05-06 | Ptc Therapeutics Inc | Fremgangsmåde til fremstillingen af 1,2,4-oxadiazolbenzoesyrer |
| AU2007300542B2 (en) | 2006-09-25 | 2012-05-17 | Ptc Therapeutics, Inc. | Crystalline forms of 3-[5-(2-fluorophenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid |
| EP2073805B1 (en) | 2006-10-12 | 2015-03-04 | PTC Therapeutics, Inc. | Methods for dosing an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy |
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2007
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