专利快速检索-快速检索全球专利，免费商用专利数据库-IPRDB

111. 发明申请

WO2004067728A3 METHODS AND SYSTEMS FOR THE IDENTIFICATION OF RNA REGULATORY SEQUENCES AND COMPOUNDS THAT MODULATE THEIR FUNCTION 审中-公开
公开(公告)号：WO2004067728A3
公开(公告)日：2004-08-12
申请号：PCT/US2004/000423
申请日：2004-01-09
申请人： PTC THERAPEUTICS
发明人： PELLEGRINI, Matthew, C. , TROTTA, Christopher, R. , HUQ, Shah, I.
IPC分类号： C12Q1/68
摘要： The present invention is directed to a non-cell based method for identifying RNA regulatory sequences involved in translational control. The method includes: combining a translational extract; an RNA test sequence; and a reporter mRNA under suitable conditions for translation of the reporter mRNA. The method also includes measuring the effect of the test sequence on the translation of the reporter mRNA, wherein a test sequence that modifies the translation of the reporter mRNA includes an RNA regulatory element. The invention also provides methods and systems for identifying test compounds that modulate the regulatory activity of an RNA regulatory sequence. In the method, an RNA regulatory sequence, which regulates translation of a reporter mRNA, is combined with a translation extract; the reporter mRNA; and at least one test compound. The method further includes measuring the effect of the test compound on the ability of the RNA regulatory sequence to regulate the translation of the reporter mRNA.

112. 发明申请

WO2004065561A2 METHODS FOR IDENTIFYING COMPOUNDS THAT MODULATE UNTRANSLATED REGION-DEPENDENT GENE EXPRESSION AND METHODS OF USING SAME 审中-公开
标题翻译：用于识别不依赖地区的相关基因表达的化合物的鉴定方法及其使用方法
公开(公告)号：WO2004065561A2
公开(公告)日：2004-08-05
申请号：PCT/US2004/001643
申请日：2004-01-21
申请人： PTC THERAPEUTICS, INC. , CAO, Liangxian , TRIFILLIS, Panayiota
发明人： CAO, Liangxian , TRIFILLIS, Panayiota
IPC分类号： C12N
CPC分类号： C12Q1/6897 , C07K14/52 , C12Q1/6886 , C12Q2600/136 , C12Q2600/158 , G01N33/5011 , G01N33/5023 , G01N2333/515 , G01N2500/00
摘要： The present invention relates to methods for identifying compounds that modulate untranslated region-dependent expression of a target gene. The invention particularly relates to using untranslated regions of a target gene or fragments thereof linked to a reporter gene to identify compounds that modulate untranslated region-dependent expression of a target gene. 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：本发明涉及用于鉴定调节靶基因的非翻译区域依赖性表达的化合物的方法。本发明特别涉及使用与报告基因连接的靶基因的非翻译区或其片段来鉴定调节靶基因的非翻译区域依赖性表达的化合物。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。

113. 发明申请

WO2004010106A2 METHODS FOR IDENTIFYING SMALL MOLEDULES THAT MODULATE PREMATURE TRANSLATION TERMINATION AND NONSENSE MEDIATED mRNA DECAY 审中-公开
标题翻译：鉴定调节早期翻译终止和非感染介导的mRNA衰变的小模型的方法
公开(公告)号：WO2004010106A2
公开(公告)日：2004-01-29
申请号：PCT/US2003/023075
申请日：2003-07-24
申请人： PTC THERAPEUTICS, INC. , WELCH, Ellen, M. , ALMSTEAD, Neil, Gregory , RANDO, Robert, F. , PELLEGRINI, Mathew, C.
发明人： WELCH, Ellen, M. , ALMSTEAD, Neil, Gregory , RANDO, Robert, F. , PELLEGRINI, Mathew, C.
IPC分类号： G01N
CPC分类号： B82Y30/00 , G01N33/5308 , G01N2500/04
摘要： The present invention relates to a method for screening and identifying compounds that modulate premature translation termination and/or nonsense-mediated messenger ribonucleic acid ("mRNA") by interacting with a preselected target ribonucleic acid ("RNA"). In particular, the present invention relates to identifying compounds that bind to regions of the 28S ribosomal RNA ("rRNA") and analogs thereof. Direct, noncompetitive binding assays are advantageously used to screen libraries of compounds for those that selectively bind to a preselected target RNA. Binding of target RNA molecules to a particular compound is detected using any physical method that measures the altered physical property of the target RNA bound to a compound. The structure of the 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：本发明涉及用于筛选和鉴定通过与预先选择的靶核糖核酸（“mRNA”）相互作用来调节过早翻译终止和/或无义介导的信使核糖核酸（“mRNA”）的化合物 ; RNA＆QUOT）。具体而言，本发明涉及鉴定与28S核糖体RNA（“rRNA”）及其类似物的区域结合的化合物。直接非竞争性结合测定法有利地用于筛选选择性结合预先选择的靶RNA的化合物文库。使用任何物理方法检测靶RNA分子与特定化合物的结合，所述物理方法测量与化合物结合的靶RNA的改变的物理性质。与标记的RNA连接的化合物的结构也被确定。所用的方法部分取决于筛选的图书馆的性质。本发明的方法提供了用于高通量筛选化合物文库以鉴定药物导致的简单，灵敏的测定。

114. 发明申请

WO2004001010A2 METHODS FOR IDENTIFYING SMALL MOLECULES THAT MODULATE PREMATURE TRANSLATION TERMINATION AND NONSENSE MEDIATED mRNA DECAY 审中-公开
标题翻译：用于鉴定调节初步翻译终止和非延迟介导的mRNA衰减的小分子的方法
公开(公告)号：WO2004001010A2
公开(公告)日：2003-12-31
申请号：PCT/US2003/019760
申请日：2003-06-23
申请人： PTC THERAPEUTICS, INC. , WELCH, Ellen , ZHUO, Jin
发明人： WELCH, Ellen , ZHUO, Jin
IPC分类号： C12N
CPC分类号： C12Q1/6897
摘要： The present invention relates to methods for identifying compounds that modulate premature translation termination and/or nonsense-mediated mRNA decay by screening and identifying compounds that modulate the post-transcriptional expression of any gene with a premature translation stop codon. The invention particularly relates to using any gene encoding a premature stop codon to identify compounds that modulate premature translation termination and/or nonsense-mediated mRNA decay. A compound that modulates premature translation termination and/or nonsense-mediated mRNA decay of a target gene is identified using standard methods known in the art to measure changes in translation or mRNA stability of the gene product or mRNA of the gene with the premature stop codon. 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：本发明涉及通过筛选和鉴定调节具有过早翻译终止密码子的任何基因的转录后表达的化合物来鉴定调节过早翻译终止和/或无义介导的mRNA衰变的化合物的方法。本发明特别涉及使用编码过早终止密码子的任何基因来鉴定调节过早翻译终止和/或无义介导的mRNA衰变的化合物。使用本领域已知的标准方法鉴定调节前体转录终止和/或无义介导的mRNA基因的衰老的化合物，以测量基因产物或基因mRNA的mRNA稳定性的变化，所述基因产物或mRNA的早期终止密码子。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。

115. 发明申请

WO0144516A3 METHODS OF ASSAYING FOR COMPOUNDS THAT INHIBIT PREMATURE TRANSLATION TERMINATION AND NONSENSE-MEDIATED RNA DECAY 审中-公开
标题翻译：测定禁止翻译终止和非延迟RNA降解的化合物的方法
公开(公告)号：WO0144516A3
公开(公告)日：2002-06-06
申请号：PCT/US0034232
申请日：2000-12-13
申请人： TULARIK INC , PTC THERAPEUTICS INC
发明人： BECKMANN HOLGER , LEARNED MARC , PELTZ STUART , CZAPLINSKI KEVIN
IPC分类号： G01N33/50 , C12N15/09 , C12Q1/02 , C12Q1/66 , C12Q1/68 , G01N33/15 , G01N33/53 , G01N33/58
CPC分类号： G01N33/5008 , C12Q1/6897 , G01N2333/70503 , G01N2333/90 , G01N2500/00
摘要： The present application provides methods of assaying for compounds that inhibit premature translation termination and nonsense mediated RNA decay in cells.
摘要翻译：本申请提供了测定抑制细胞中过早翻译终止和无义介导的RNA衰变的化合物的方法。

116. 发明申请

WO2023039369A1 METHODS FOR TREATING NEUROGENERATIVE DISEASES 审中-公开
公开(公告)号：WO2023039369A1
公开(公告)日：2023-03-16
申请号：PCT/US2022/075967
申请日：2022-09-06
申请人： PTC THERAPEUTICS, INC.
发明人： BARRAZA, Scott J. , TRIMMER, Jeffrey K. , YU, Yong , ZHANG, Xiaoyan
IPC分类号： A61P25/28 , A61K31/4365 , A61P25/16
摘要： The present description relates to methods of treating neurodegenerative diseases characterized by the accumulation of aberrant forms of the microtubule associated protein Tau (MAPT) using substituted thieno[3,2‑b]pyridine compounds, forms, and pharmaceutical compositions thereof.

117. 发明申请

WO2023288325A2 RECOMBINANT REELIN GENE THERAPY 审中-公开
公开(公告)号：WO2023288325A2
公开(公告)日：2023-01-19
申请号：PCT/US2022/073809
申请日：2022-07-15
申请人： PTC THERAPEUTICS INC.
发明人： WEEBER, Edwin J. , GRACI, Jason , WONG, Helen , PICK, Joseph Edward
IPC分类号： C12N15/86 , A61K48/00
摘要： The disclosure describes novel Reelin repeat fusion proteins for use as a therapeutic in the treatment of a disease or disorder of the nervous system. Intracerebroventricular administration of AAV vectors expressing a Reelin repeat fusion protein is shown to rescue impairment of spine density in the hippocampus of reeler and attenuate hyperactivity of FMR-/- knockout mice in an open field assay.

118. 发明申请

WO2023283466A1 PHARMACEUTICAL COMPOSITIONS COMPRISING 2,3,5-TRIMETHYL-6-NONYLCYCLOHEXA-2,5-DIENE-1,4-DIONE 审中-公开
公开(公告)号：WO2023283466A1
公开(公告)日：2023-01-12
申请号：PCT/US2022/036589
申请日：2022-07-08
申请人： PTC THERAPEUTICS, INC. , PINNAMANENI, Swathi , DALI, Mandar V. , PATEL, Dhaval , UDDIN, Akm Nasir
发明人： PINNAMANENI, Swathi , DALI, Mandar V. , PATEL, Dhaval , UDDIN, Akm Nasir
IPC分类号： A61K9/107 , A61K31/122 , A61K31/047 , A61K36/63 , A61K36/899 , A61K47/14 , A61K47/26 , A61K47/44 , A61K9/1075 , A61P1/00
摘要： Disclosed herein is pharmaceutical compositions of Compound 1, and/or the hydroquinone form thereof, and methods useful for treating or suppressing a disease or disorder such as an α-synucleinpathy, a tauopathy, an autistic spectrum disorder, a pervasive developmental disorder, a liver disease, and liver damage in a subject using such pharmaceutical compositions.

119. 发明申请

WO2022104058A1 TABLET FOR USE IN TREATING HUNTINGTON'S DISEASE AND METHOD OF MAKING THE SAME 审中-公开
公开(公告)号：WO2022104058A1
公开(公告)日：2022-05-19
申请号：PCT/US2021/059139
申请日：2021-11-12
申请人： PTC THERAPEUTICS INC.
发明人： PINNAMANENI, Swathi , UDDIN, Akm, Nasir , DALI, Mandar, Vasant
IPC分类号： A61K9/20
摘要： The present description relates to a tablet formulation of 2-[3-(2,2,6,6- tetramethylpiperidin-4-yl)-3H-[l,2,3]triazolo[4,5-c]pyridazin-6-yl]-5-(2H-l,2,3-triazol-2- yl)phenol, a compound for use in treating Huntington's disease, and a method of making the same.

120. 发明申请

WO2021150983A1 METHODS FOR TREATING PARKINSON'S DISEASE WITH SEPIAPTERIN 审中-公开
公开(公告)号：WO2021150983A1
公开(公告)日：2021-07-29
申请号：PCT/US2021/014745
申请日：2021-01-22
申请人： PTC THERAPEUTICS MP, INC.
发明人： SMITH, Neil
IPC分类号： A61K31/519 , A61P25/16 , A61K31/198
摘要： The present invention features methods for treating Parkinson's disease using sepiapterin, or a pharmaceutically acceptable salt thereof.

你已经成功收藏专利！

检索式保存成功!

IPRDB

热门服务

关于我们

友情链接

联系方式