HK1111897A1 - Compositions of an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy - Google Patents
Compositions of an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapyInfo
- Publication number
- HK1111897A1 HK1111897A1 HK08106626.1A HK08106626A HK1111897A1 HK 1111897 A1 HK1111897 A1 HK 1111897A1 HK 08106626 A HK08106626 A HK 08106626A HK 1111897 A1 HK1111897 A1 HK 1111897A1
- Authority
- HK
- Hong Kong
- Prior art keywords
- oxadiazole
- compositions
- nonsense mutation
- orally active
- suppression therapy
- Prior art date
Links
Classifications
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- 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
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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- A61K9/0053—Mouth and digestive tract, i.e. intraoral and peroral administration
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- A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
- A61K9/141—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers
- A61K9/145—Intimate drug-carrier mixtures characterised by the carrier, e.g. ordered mixtures, adsorbates, solid solutions, eutectica, co-dried, co-solubilised, co-kneaded, co-milled, co-ground products, co-precipitates, co-evaporates, co-extrudates, co-melts; Drug nanoparticles with adsorbed surface modifiers with organic compounds
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Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- General Health & Medical Sciences (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Organic Chemistry (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Neurology (AREA)
- Epidemiology (AREA)
- Physical Education & Sports Medicine (AREA)
- Endocrinology (AREA)
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- Orthopedic Medicine & Surgery (AREA)
- Obesity (AREA)
- Rheumatology (AREA)
- Urology & Nephrology (AREA)
- Pulmonology (AREA)
- Heart & Thoracic Surgery (AREA)
- Cardiology (AREA)
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- Physiology (AREA)
- Nutrition Science (AREA)
- Psychology (AREA)
- Gastroenterology & Hepatology (AREA)
- Child & Adolescent Psychology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
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Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US66978905P | 2005-04-08 | 2005-04-08 | |
US78739506P | 2006-03-30 | 2006-03-30 | |
PCT/US2006/012887 WO2006110483A1 (en) | 2005-04-08 | 2006-04-06 | Compositions of an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy |
Publications (1)
Publication Number | Publication Date |
---|---|
HK1111897A1 true HK1111897A1 (en) | 2008-08-22 |
Family
ID=36763092
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
HK08106626.1A HK1111897A1 (en) | 2005-04-08 | 2008-06-17 | Compositions of an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy |
Country Status (17)
Country | Link |
---|---|
US (7) | US8716321B2 (da) |
EP (2) | EP1874306B1 (da) |
JP (3) | JP5800451B2 (da) |
CN (1) | CN103720688A (da) |
AU (1) | AU2006235115C1 (da) |
BR (1) | BRPI0609089A2 (da) |
CA (1) | CA2603402C (da) |
DK (2) | DK1874306T3 (da) |
ES (2) | ES2390804T3 (da) |
HK (1) | HK1111897A1 (da) |
IL (2) | IL186433A (da) |
MX (1) | MX2007012206A (da) |
NO (2) | NO339909B1 (da) |
NZ (1) | NZ562197A (da) |
PL (2) | PL1874306T3 (da) |
PT (2) | PT2402002T (da) |
WO (1) | WO2006110483A1 (da) |
Families Citing this family (49)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
PL2910551T3 (pl) | 2003-04-11 | 2017-09-29 | Ptc Therapeutics, Inc. | Związki kwasu 1,2,4-oksadiazolobenzoesowego i ich zastosowanie do supresji mutacji nonsensownych i leczenia choroby |
NZ562197A (en) | 2005-04-08 | 2010-09-30 | Ptc Therapeutics Inc | Compositions of an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy |
WO2007117438A2 (en) | 2006-03-30 | 2007-10-18 | Ptc Therapeutics, Inc. | Methods for the production of functional protein from dna having a nonsense mutation and the treatment of disorders associated therewith |
US10022352B2 (en) | 2006-04-07 | 2018-07-17 | Vertex Pharmaceuticals Incorporated | Modulators of ATP-binding cassette transporters |
US7776905B2 (en) | 2006-04-07 | 2010-08-17 | Vertex Pharmaceuticals Incorporated | Modulators of ATP-binding cassette transporters |
US7645789B2 (en) | 2006-04-07 | 2010-01-12 | Vertex Pharmaceuticals Incorporated | Indole derivatives as CFTR modulators |
RU2495030C2 (ru) * | 2006-09-08 | 2013-10-10 | ПиТиСи ТЕРАПЬЮТИКС, ИНК. | Способы получения 1,2,4-оксадиазолбензойных кислот |
BRPI0717107A2 (pt) | 2006-09-25 | 2013-10-15 | Ptc Therapeutics Inc | FORMA DE CRISTAL, FORMA DE DOSAGEM FARMACÊUTICA, E, MÉTODO PARA MODULAR TERMINAÇÃO DE TRANSLAÇÃO PREMATURA OU DETERIORAÇÃO DE mRNA |
US8101641B2 (en) | 2006-09-25 | 2012-01-24 | Ptc Therapeutics, Inc. | Hydroxylated 1,2,4-oxadiazole benzoic acid compounds and compositions thereof |
AU2016262645B2 (en) * | 2006-10-12 | 2018-05-17 | Ptc Therapeutics, Inc. | Methods for dosing an orally active 1,2,4-oxadiazole for nonsense mutation suppression therapy |
US9226919B2 (en) * | 2006-10-12 | 2016-01-05 | Ptc Therapeutics, Inc. | Methods for dosing an orally active 1,2,4-oxadiazole |
US8563573B2 (en) | 2007-11-02 | 2013-10-22 | Vertex Pharmaceuticals Incorporated | Azaindole derivatives as CFTR modulators |
GB0715087D0 (en) | 2007-08-03 | 2007-09-12 | Summit Corp Plc | Drug combinations for the treatment of duchenne muscular dystrophy |
HUE033195T2 (en) | 2007-08-03 | 2017-11-28 | Summit (Oxford) Ltd | Combinations of drugs for the treatment of Duchenne muscular dystrophy |
SI2578571T1 (sl) | 2007-11-16 | 2016-01-29 | Vertex Pharmaceuticals Incorporated | Izokinolinski modulatorji prenašalcev z atp-vezavno kaseto |
ES2790830T3 (es) | 2007-12-07 | 2020-10-29 | Vertex Pharma | Formas solidas de ácido 3-(6-(1-(2,2-difluorobenzo[d][1,3]dioxol-5-il)ciclopropanocarboxamido)-3-metilpiridin-2-il) benzoico |
CN101981011B (zh) | 2008-03-31 | 2014-05-07 | 沃泰克斯药物股份有限公司 | 作为cftr调节剂的吡啶基衍生物 |
RU2573830C2 (ru) | 2010-03-25 | 2016-01-27 | Вертекс Фармасьютикалз Инкорпорейтед | ТВЕРДЫЕ ФОРМЫ (R)-1-(2,2-ДИФТОРБЕНЗО[d][1,3]ДИОКСОЛ-5-ИЛ)-N-(2,3-ДИГИДРОКСИПРОПИЛ)-6-ФТОР-2-(1-ГИДРОКСИ-2-МЕТИЛПРОПАН-2-ИЛ)-1H-ИНДОЛ-5-ИЛ)ЦИКЛОПРОПАНКАРБОКСАМИДА |
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 |
NZ708598A (en) | 2010-04-07 | 2017-05-26 | Vertex Pharma | Pharmaceutical compositions of 3-(6-(1-(2,2-difluorobenzo[d](1,3)dioxol-5-yl) cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid and administration thereof |
TWI549950B (zh) | 2010-04-07 | 2016-09-21 | 維泰克斯製藥公司 | 3-(6-(1-(2,2-二氟苯并[d][1,3]二氧雜環戊烯-5-基)環丙烷甲醯胺基)-3-甲基吡啶-2-基)苯甲酸之固體形式 |
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US8563593B2 (en) | 2010-06-08 | 2013-10-22 | Vertex Pharmaceuticals Incorporated | Formulations of (R)-1-(2,2-difluorobenzo[D] [1,3] dioxol-5-yl)-N-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl)cyclopropanecarboxamide |
CN103189060A (zh) | 2010-08-05 | 2013-07-03 | 里尔法律与医疗卫生第二大学 | 用于对无义突变介导的疾病进行治疗的化合物和含有所述化合物的药物组合物 |
NZ606805A (en) | 2010-08-23 | 2015-03-27 | Vertex Pharma | Pharmaceutical composition of (r)-1-(2,2-difluorobenzo[d][1,3]dioxol-5-yl)-n-(1-(2,3-dihydroxy propyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1h-indol-5-yl) cyclopropanecarboxamide and administration therof |
IN2014KN00885A (da) | 2011-11-08 | 2015-10-02 | Vertex Pharma | |
CN104168890A (zh) | 2012-01-25 | 2014-11-26 | 沃泰克斯药物股份有限公司 | 3-(6-(1-(2,2-二氟苯并[d][1,3]二氧杂环戊烯-5-基)环丙烷甲酰胺基)-3-甲基吡啶-2-基)苯甲酸的制剂 |
AU2013290444B2 (en) | 2012-07-16 | 2018-04-26 | Vertex Pharmaceuticals Incorporated | Pharmaceutical compositions of (R)-1-(2,2-diflurorbenzo[d][1,3]dioxol-5-yl)-N-(1-(2,3-dihydroxypropyl)-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl) cyclopropanecarboxamide and administration thereof |
SI2914248T2 (sl) | 2012-11-02 | 2023-12-29 | Vertex Pharmaceuticals Incorporated, | Farmacevtski sestavki za zdravljenje bolezni, pri katerih posreduje CFTR |
WO2014153643A1 (en) * | 2013-03-26 | 2014-10-02 | The University Of British Columbia | Compositions and methods for use thereof in the treatment of aniridia |
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 |
LT3925607T (lt) | 2014-04-15 | 2023-11-27 | Vertex Pharmaceuticals Incorporated | Farmacinė kompozicija, skirta gydyti ligas, kurias nulemia cistinės fibrozės transmembraninio laidumo reguliatorius |
US20170196842A1 (en) * | 2014-06-06 | 2017-07-13 | Ptc Therapeutics, Inc. | Methods of treatment using a 1,2,4-oxadiazole benzoic acid |
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