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1. 发明申请

WO2007117438A3 METHODS FOR THE PRODUCTION OF FUNCTIONAL PROTEIN FROM DNA HAVING A NONSENSE MUTATION AND THE TREATMENT OF DISORDERS ASSOCIATED THEREWITH 审中-公开
标题翻译：用于从具有非神经突变的DNA生产功能性蛋白质的方法和与之相关的疾病的治疗
公开(公告)号：WO2007117438A3
公开(公告)日：2008-10-23
申请号：PCT/US2007008268
申请日：2007-03-29
申请人： PTC THERAPEUTICS INC , ALMSTEAD NEIL G , CHEN GUANGMING , HIRAWAT SAMIT , HWANG SEONGWOO , KARP GARY M , MILLER LANGDON , MOON YOUNG-CHOON , REN HONGYU , TAKASUGI JEMES J , WELCH ELLEN M , WILDE RICHARD G , KENNEDY PAUL
发明人： ALMSTEAD NEIL G , CHEN GUANGMING , HIRAWAT SAMIT , HWANG SEONGWOO , KARP GARY M , MILLER LANGDON , MOON YOUNG-CHOON , REN HONGYU , TAKASUGI JEMES J , WELCH ELLEN M , WILDE RICHARD G , KENNEDY PAUL
IPC分类号： A61K31/00 , A61K31/40 , A61K31/41 , A61K31/4162 , A61K31/44 , A61K31/46 , A61P1/00 , A61P5/00 , A61P25/28
CPC分类号： A61K31/4245 , A61K31/00 , A61K31/40 , A61K31/41 , A61K31/4162 , A61K31/44 , A61K31/46
摘要： The present invention relates to functional proteins encoded by nucleic acid sequences comprising a nonsense mutation. The present invention also relates to methods for the production of functional proteins encoded by nucleic acid sequences comprising a nonsense mutation and the use of such proteins for prevention, management and/or treatment of diseases associated with a nonsense mutation(s) in a gene.
摘要翻译：本发明涉及由包含无义突变的核酸序列编码的功能蛋白。本发明还涉及用于产生由包含无义突变的核酸序列编码的功能性蛋白质的方法以及用于预防，管理和/或治疗与基因中的无义突变有关的疾病的这些蛋白质的用途。

2. 发明申请

WO2008039431A2 CRYSTALLINE FORMS OF 3-[5-(2-FHJOROPHENYL)-[1,2,4]OXADIAZOL-3-YL]-BENZOIC ACID 审中-公开
标题翻译： 3- [5-（2-异丙基） - [1,2,4]氧杂双环[3-y]] - 苯甲酸的晶体形式
公开(公告)号：WO2008039431A2
公开(公告)日：2008-04-03
申请号：PCT/US2007020633
申请日：2007-09-24
申请人： PTC THERAPEUTICS INC , ALMSTEAD NEIL G , HWANG PETER SEONGWOO , MOON YOUNG-CHOON
发明人： ALMSTEAD NEIL G , HWANG PETER SEONGWOO , MOON YOUNG-CHOON
IPC分类号： C07D271/06 , A61K31/4245
CPC分类号： A61K31/4245 , C07D271/06
摘要： The present invention relates to crystalline forms of 3-[5-(2-fluorophenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid, pharmaceutical compositions and dosage forms comprising the crystalline forms, methods of making the crystalline forms and methods for their use for the treatment, prevention or management of diseases ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay.
摘要翻译：本发明涉及3- [5-（2-氟苯基） - [1,2,4]恶二唑-3-基] - 苯甲酸的结晶形式，包含结晶形式的药物组合物和剂型，它们用于治疗，预防或控制通过调节过早翻译终止或无义介导的mRNA衰变而改善的疾病的结晶形式和方法。

3. 发明申请

WO2004009533A1 ACETYLAMINO BENZOIC ACID COMPOUNDS AND THEIR USE FOR NONSENSE SUPPRESSION AND THE TREATMENT OF DISEASE 审中-公开
标题翻译：乙酰氨基苯甲酸化合物及其用于非感染抑制和疾病治疗的用途
公开(公告)号：WO2004009533A1
公开(公告)日：2004-01-29
申请号：PCT/US2003/023183
申请日：2003-07-23
申请人： PTC THERAPEUTICS, INC. , TULARIK INC. , WILDE, Richard, G. , WELCH. Ellen, M. , TAKASUGI, James, Jan , ALMSTEAD, Neil, G. , RUBENSTEIN, Steven, Marc , BECKMANN, Holger
发明人： WILDE, Richard, G. , WELCH. Ellen, M. , TAKASUGI, James, Jan , ALMSTEAD, Neil, G. , RUBENSTEIN, Steven, Marc , BECKMANN, Holger
IPC分类号： C07C235/24
CPC分类号： C07D207/273 , A61K31/16 , A61K31/33 , C07C235/24 , C07C323/62 , C07C327/42 , C07C2601/08 , C07C2602/08 , C07C2602/10 , C07D209/48 , C07D213/64 , C07D239/38 , C07D257/04 , C07D271/06 , C07D317/64 , C07D405/12
摘要： Novel acetylamino benzoic acid compounds, methods of using and pharmaceutical compositions comprising an acetylamino benzoic acid derivative are disclosed. The methods include methods of treating or preventing a disease ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay, or ameliorating one or more symptoms associated therewith.
摘要翻译：公开了新型乙酰氨基苯甲酸化合物，使用方法和包含乙酰氨基苯甲酸衍生物的药物组合物。所述方法包括治疗或预防通过调节过早翻译终止或无义介导的mRNA衰变而改善的疾病或改善与其相关的一种或多种症状的方法。

4. 发明申请

WO2008039431B1 CRYSTALLINE FORMS OF 3-[5-(2-FHJOROPHENYL)-[1,2,4]OXADIAZOL-3-YL]-BENZOIC ACID 审中-公开
标题翻译： 3- [5-（2-氟苯基） - [1,2,4]恶二唑-3-基] - 苯甲酸的结晶形式
公开(公告)号：WO2008039431B1
公开(公告)日：2008-07-10
申请号：PCT/US2007020633
申请日：2007-09-24
申请人： PTC THERAPEUTICS INC , ALMSTEAD NEIL G , HWANG PETER SEONGWOO , MOON YOUNG-CHOON
发明人： ALMSTEAD NEIL G , HWANG PETER SEONGWOO , MOON YOUNG-CHOON
IPC分类号： C07D271/06 , A61K31/4245
CPC分类号： A61K31/4245 , C07D271/06
摘要： The present invention relates to crystalline forms of 3- [5- (2 -fluorophenyl) - [l,2,4]oxadiazol-3-yl] -benzoic acid (formula (I)), pharmaceutical compositions and dosage forms comprising the crystalline forms, methods of making the crystalline forms and methods for their use for the treatment, prevention or management of diseases ameliorated by modulation of premature translation termination or nonsense -mediated mRNA decay.
摘要翻译：本发明涉及3- [5-（2-氟苯基） - [1,2,4]恶二唑-3-基] - 苯甲酸（式（I））的结晶形式，包含结晶的药物组合物和剂型形式，制造晶体形式的方法以及它们用于治疗，预防或控制通过调节过早翻译终止或无义介导的mRNA衰变而改善的疾病的方法。

5. 发明申请

WO2004009610A3 USE OF NUCLEOSIDE COMPOUNDS FOR NONSENSE SUPPRESSION AND THE TREATMENT OF GENETIC DISEASES 审中-公开
标题翻译：核苷类化合物用于非感染抑制和治疗遗传性疾病的用途
公开(公告)号：WO2004009610A3
公开(公告)日：2005-10-06
申请号：PCT/US0323185
申请日：2003-07-23
申请人： PTC THERAPEUTICS INC , TULARIK INC , WILDE RICHARD G , ALMSTEAD NEIL G , WELCH ELLEN M , BECKMANN HOLGER
发明人： WILDE RICHARD G , ALMSTEAD NEIL G , WELCH ELLEN M , BECKMANN HOLGER
IPC分类号： C07H19/00 , C07H17/02 , A61K31/7064 , A61P7/00 , A61P25/00 , A61P37/00
CPC分类号： A61K31/506 , A61K31/7072 , C07H19/00
摘要： The invention encompasses nucleoside compounds, compositions comprising the compounds and methods for treating or preventing diseases associated with nonsense mutations of mRNA by administering these compounds or compositions.
摘要翻译：本发明包括核苷化合物，包含该化合物的组合物和通过施用这些化合物或组合物来治疗或预防与无义突变mRNA相关的疾病的方法。

6. 发明申请

WO2004009610A2 USE OF NUCLEOSIDE COMPOUNDS FOR NONSENSE SUPPRESSION AND THE TREATMENT OF GENETIC DISEASES 审中-公开
标题翻译：核苷类化合物用于非感染抑制和治疗遗传性疾病的用途
公开(公告)号：WO2004009610A2
公开(公告)日：2004-01-29
申请号：PCT/US2003/023185
申请日：2003-07-23
申请人： PTC THERAPEUTICS, INC. , TULARIK INC. , WILDE, Richard, G. , ALMSTEAD, Neil, G. , WELCH, Ellen, M. , BECKMANN, Holger
发明人： WILDE, Richard, G. , ALMSTEAD, Neil, G. , WELCH, Ellen, M. , BECKMANN, Holger
IPC分类号： C07H19/00
CPC分类号： A61K31/506 , A61K31/7072 , C07H19/00
摘要： The invention encompasses nucleoside compounds, compositions comprising the compounds and methods for treating or preventing diseases associated with nonsense mutations of mRNA by administering these compounds or compositions.
摘要翻译：本发明包括核苷化合物，包含该化合物的组合物和通过施用这些化合物或组合物来治疗或预防与无义突变mRNA相关的疾病的方法。

7. 发明申请

WO02083837B1 METHODS FOR IDENTIFYING SMALL MOLECULES THAT BIND SPECIFIC RNA STRUCTURAL MOTIFS 审中-公开
标题翻译：识别特异性RNA结构运动的小分子的方法
公开(公告)号：WO02083837B1
公开(公告)日：2003-01-30
申请号：PCT/US0211758
申请日：2002-04-11
申请人： PTC THERAPEUTICS INC , ALMSTEAD NEIL G
发明人： ALMSTEAD NEIL G
IPC分类号： C12N15/10 , C12Q1/68 , C12M1/38 , C12M1/40
CPC分类号： C12N15/115 , C12N15/1048
摘要： The present invention relates to a method for screening and identifying test compounds that bind to a preselected target ribonucleic acid ("RNA"). Direct, non-competitive binding assays are advantageously used to screen bead-based libraries of compounds for those that selectively bind to a preselected target RNA. Binding of target RNA molecules to a particular test compound is detected using any physical method that measures the altered physical property of the target RNA bound to a test compound. The structure of the test compound attached to the labeled RNA is also determined. The methods used will depend, in part, on the nature of the library screened. The methods of the present invention provide a simple, sensitive assay for high-throughput screening of libraries of compounds to identify pharmaceutical leads.
摘要翻译：本发明涉及筛选和鉴定结合预选靶核糖核酸（“RNA”）的测试化合物的方法。直接的非竞争性结合测定法有利地用于筛选选择性结合预选靶RNA的化合物的基于珠的文库。使用测量与测试化合物结合的靶RNA的改变的物理性质的任何物理方法检测靶RNA分子与特定测试化合物的结合。还测定了附着于标记的RNA的测试化合物的结构。所使用的方法部分取决于所筛选图书馆的性质。本发明的方法提供了用于识别药物引线的化合物文库的高通量筛选的简单，灵敏的测定。

8. 发明申请

WO2008130370A1 HYDROXYLATED 1,2,4-OXADIAZOLE BENZOIC ACID COMPOUNDS, COMPOSITIONS THEREOF AND THE USE FOR NONSENSE SUPPRESSION 审中-公开
标题翻译：羟基苯氧基-1,2,4-氧杂苯并噻唑酸化合物及其组合物及其用于非抑制作用
公开(公告)号：WO2008130370A1
公开(公告)日：2008-10-30
申请号：PCT/US2007/020631
申请日：2007-09-24
申请人： PTC THERAPEUTICS, INC. , ALMSTEAD, Neil, G. , HWANG, Peter, Seongwoo , MOON, Young-choon , WELCH, Ellen, M.
发明人： ALMSTEAD, Neil, G. , HWANG, Peter, Seongwoo , MOON, Young-choon , WELCH, Ellen, M.
IPC分类号： A61K31/4245 , C07D271/06 , C07D413/04 , A61P43/00 , A61P35/00
CPC分类号： A61K31/4245 , C07D271/06
摘要： Novel hydroxylated 1,2,4-oxadiazole benzoic acid compounds, methods of using and pharmaceutical compositions comprising a hydroxylated 1,2,4-oxadiazole benzoic acid derivative are disclosed. The methods include methods of treating or preventing a disease ameliorated by modulation of premature translation termination or nonsense-mediated mRNA decay, or ameliorating one or more symptoms associated therewith.
摘要翻译：公开了新型羟基化的1,2,4-恶二唑苯甲酸化合物，使用方法和包含羟基化的1,2,4-恶二唑苯甲酸衍生物的药物组合物。所述方法包括治疗或预防通过调节过早翻译终止或无义介导的mRNA衰变而改善的疾病或改善与其相关的一种或多种症状的方法。

9. 发明申请

WO2008039431A3 CRYSTALLINE FORMS OF 3-[5-(2-FHJOROPHENYL)-[1,2,4]OXADIAZOL-3-YL]-BENZOIC ACID 审中-公开
公开(公告)号：WO2008039431A3
公开(公告)日：2008-04-03
申请号：PCT/US2007/020633
申请日：2007-09-24
申请人： PTC THERAPEUTICS, INC. , ALMSTEAD, Neil, G. , HWANG, Peter, Seongwoo , MOON, Young-Choon
发明人： ALMSTEAD, Neil, G. , HWANG, Peter, Seongwoo , MOON, Young-Choon
IPC分类号： C07D271/06 , A61K31/4245
摘要： The present invention relates to crystalline forms of 3- [5- (2 -fluorophenyl) - [l,2,4]oxadiazol-3-yl] -benzoic acid (formula (I)), pharmaceutical compositions and dosage forms comprising the crystalline forms, methods of making the crystalline forms and methods for their use for the treatment, prevention or management of diseases ameliorated by modulation of premature translation termination or nonsense -mediated mRNA decay.

10. 发明申请

WO2008030570A1 PROCESSES FOR THE PREPARATION OF 1,2,4-OXADIAZOLE BENZOIC ACIDS 审中-公开
标题翻译：制备1,2,4-氧杂萘并苯甲酸的方法
公开(公告)号：WO2008030570A1
公开(公告)日：2008-03-13
申请号：PCT/US2007/019561
申请日：2007-09-06
申请人： PTC THERAPEUTICS, INC. , ALMSTEAD, Neil, G. , HWANG, Peter, Seongwoo , PINES, Seemon , MOON, Young-choon , TAKASUGI, James, J.
发明人： ALMSTEAD, Neil, G. , HWANG, Peter, Seongwoo , PINES, Seemon , MOON, Young-choon , TAKASUGI, James, J.
IPC分类号： C07D271/06
CPC分类号： C07D271/06
摘要： Provided herein are processes for the preparation of compounds useful for the treatment, prevention or management of diseases associated with a nonsense mutation. More specifically, provided herein are processes for the synthesis of 1,2,4-oxadiazoles. In particular, provided herein are processes useful for the preparation of 3-[5-(2-fluorophenyl)- [1,2,4]oxadiazol-3-yl]-benzoic acid.
摘要翻译：本文提供了制备用于治疗，预防或控制与无义突变有关的疾病的化合物的方法。更具体地，本文提供了合成1,2,4-恶二唑的方法。特别地，本文提供了可用于制备3- [5-（2-氟苯基） - [1,2,4]恶二唑-3-基] - 苯甲酸的方法。

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