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1. 发明公开

EP3022219A4 SPECIFIC TARGETING OF RNA EXPANDED REPEAT SEQUENCES 审中-公开
标题翻译： SPEZIFISCHES靶向VON RNA扩增重复序列
公开(公告)号：EP3022219A4
公开(公告)日：2017-07-26
申请号：EP14825696
申请日：2014-07-15
申请人： SCRIPPS RESEARCH INST , DISNEY MATTHEW D , GUAN LIRUI , YANG WANG-YONG
发明人： DISNEY MATTHEW D , GUAN LIRUI , YANG WANG-YONG
IPC分类号： C07K5/06 , A61K48/00 , C07D403/14 , C07D495/04 , C07D519/00
CPC分类号： G01N33/5308 , A61K48/00 , C07D403/14 , C07D495/04 , C07D519/00 , G01N2500/04 , G01N2500/10
摘要： The present invention provides small molecule compounds that can form covalent adducts with specific sequences of RNA, such as the hairpin loop r(CUG)exp sequence which is a cause of myotonic dystrophy type 1 (DM1), or the r(CGG)exp sequence which is a cause of fragile X-associated tremor/ataxia syndrome (FXTAS); to methods of making the small molecule compounds; and to methods of using the small molecular compounds in the treatment of DM1 or of FXTAS in patients afflicted therewith. The invention further provides a method for identifying an RNA target of a small molecule drug in vivo, using a small molecule drug conjugated to an RNA-reactive crosslinker group and a reporter group, contacting a cell or nucleic acid extract with the small molecule drug conjugate, then separating RNA targets crosslinked to the small molecule drug conjugate by interaction of the affinity group with a complementary affinity group.
摘要翻译：本发明提供了可以与特定RNA序列形成共价加合物的小分子化合物，例如为肌强直性肌营养不良1型（DM1）的发夹环r（CUG）exp序列或r（CGG）exp序列这是脆性X相关性震颤/共济失调综合征（FXTAS）的原因; 涉及制备小分子化合物的方法; 以及使用该小分子化合物治疗患有DM1或FXTAS的患者的方法。本发明进一步提供了使用与RNA反应性交联剂基团和报道基团缀合的小分子药物，使细胞或核酸提取物与小分子药物缀合物接触，从而体内鉴定小分子药物的RNA靶标的方法然后通过亲和基团与互补亲和基团的相互作用分离与小分子药物缀合物交联的RNA靶标。

2. 发明授权

EP3022219B1 SPECIFIC TARGETING OF RNA EXPANDED REPEAT SEQUENCES 有权
公开(公告)号：EP3022219B1
公开(公告)日：2020-01-15
申请号：EP14825696.9
申请日：2014-07-15
申请人： The Scripps Research Institute , Disney, Matthew D. , Guan, Lirui , Yang, Wang-Yong
发明人： DISNEY, Matthew D. , GUAN, Lirui , YANG, Wang-Yong
IPC分类号： C07K5/06 , C07D403/14 , C07D495/04 , C07D519/00 , A61K48/00

3. 发明公开

EP3022219A2 SPECIFIC TARGETING OF RNA EXPANDED REPEAT SEQUENCES 审中-公开
标题翻译：特异性靶向VON RNA扩增重复序列
公开(公告)号：EP3022219A2
公开(公告)日：2016-05-25
申请号：EP14825696.9
申请日：2014-07-15
申请人： The Scripps Research Institute , Disney, Matthew D. , Guan, Lirui , Yang, Wang-yong
发明人： DISNEY, Matthew D. , GUAN, Lirui , YANG, Wang-Yong
IPC分类号： C07K5/06
CPC分类号： G01N33/5308 , A61K48/00 , C07D403/14 , C07D495/04 , C07D519/00 , G01N2500/04 , G01N2500/10
摘要： The present invention provides small molecule compounds that can form covalent adducts with specific sequences of RNA, such as the hairpin loop r(CUG)exp sequence which is a cause of myotonic dystrophy type 1 (DM1), or the r(CGG)exp sequence which is a cause of fragile X-associated tremor/ataxia syndrome (FXTAS); to methods of making the small molecule compounds; and to methods of using the small molecular compounds in the treatment of DM1 or of FXTAS in patients afflicted therewith. The invention further provides a method for identifying an RNA target of a small molecule drug in vivo, using a small molecule drug conjugated to an RNA-reactive crosslinker group and a reporter group, contacting a cell or nucleic acid extract with the small molecule drug conjugate, then separating RNA targets crosslinked to the small molecule drug conjugate by interaction of the affinity group with a complementary affinity group.
摘要翻译：本发明提供可以形成具有特定RNA序列的共价加合物的小分子化合物，例如作为强直性营养不良型1（DM1）或r（CGG）exp序列的发夹环r（CUG）exp序列这是造成脆弱X型震颤/共济失调综合征（FXTAS）的原因; 制备小分子化合物的方法; 以及使用小分子化合物治疗患有DM1或FXTAS的患者的方法。本发明还提供了使用与RNA反应性交联剂基团和报道基团缀合的小分子药物，使细胞或核酸提取物与小分子药物缀合物接触的方法，用于在体内鉴定小分子药物的RNA靶标的方法，然后通过亲和基团与互补亲和基团的相互作用分离与小分子药物偶联物交联的RNA靶标。

4. 发明专利

AU2014290206B2 Specific targeting of RNA expanded repeat sequences 未知
公开(公告)号：AU2014290206B2
公开(公告)日：2018-03-29
申请号：AU2014290206
申请日：2014-07-15
申请人： DISNEY MATTHEW D , GUAN LIRUI , SCRIPPS RESEARCH INST , YANG WANG YONG
发明人： DISNEY MATTHEW D , GUAN LIRUI , YANG WANG-YONG
IPC分类号： C07K5/06 , A61K38/10
摘要： The present invention provides small molecule compounds that can form covalent adducts with specific sequences of RNA, such as the hairpin loop r(CUG)

5. 发明专利

CA2955136A1 SPECIFIC TARGETING OF RNA EXPANDED REPEAT SEQUENCES 未知
公开(公告)号：CA2955136A1
公开(公告)日：2015-01-22
申请号：CA2955136
申请日：2014-07-15
申请人： SCRIPPS RESEARCH INST , DISNEY MATTHEW D , GUAN LIRUI , YANG WANG-YONG
发明人： DISNEY MATTHEW D , GUAN LIRUI , YANG WANG-YONG
IPC分类号： A61K38/10
摘要： The present invention provides small molecule compounds that can form covalent adducts with specific sequences of RNA, such as the hairpin loop r(CUG)exp sequence which is a cause of myotonic dystrophy type 1 (DM1), or the r(CGG)exp sequence which is a cause of fragile X-associated tremor/ataxia syndrome (FXTAS); to methods of making the small molecule compounds; and to methods of using the small molecular compounds in the treatment of DM1 or of FXTAS in patients afflicted therewith. The invention further provides a method for identifying an RNA target of a small molecule drug in vivo, using a small molecule drug conjugated to an RNA-reactive crosslinker group and a reporter group, contacting a cell or nucleic acid extract with the small molecule drug conjugate, then separating RNA targets crosslinked to the small molecule drug conjugate by interaction of the affinity group with a complementary affinity group.

6. 发明申请

WO2015031819A1 MODULARLY ASSEMBLED SMALL MOLECULES FOR THE TREATMENT OF MYOTONIC DYSTROPHY TYPE 1 审中-公开
标题翻译：用于处理MYOTONIC DYSTROPYY TYPE 1的模块化组装小分子
公开(公告)号：WO2015031819A1
公开(公告)日：2015-03-05
申请号：PCT/US2014/053520
申请日：2014-08-29
申请人： THE SCRIPPS RESEARCH INSTITUTE , DISNEY, Matthew D. , RZUCZEK, Suzanne
发明人： DISNEY, Matthew D. , RZUCZEK, Suzanne
IPC分类号： A61K38/00 , A61K31/497 , C07D239/00
CPC分类号： A61K47/65 , A61K31/496 , A61K38/00 , A61K47/542 , A61K47/55 , C07D233/22
摘要： To study RNA function using small molecules, we designed bioactive, modularly assembled small molecules that target the noncoding expanded RNA repeat that causes myotonic dystrophy type 1 (Dml), r(CUG)exp. Different modular assembly scaffolds were investigated including polyamines, alpha-peptides, beta-peptides, and peptide tertiary amides (PT As). Based on activity as assessed by improvement of DM1 -associated defects, stability against proteases, cellular permeability, and toxicity, we discovered that constrained backbones, namely PT As, are optimal.
摘要翻译：为了研究使用小分子的RNA功能，我们设计了生物活性，模块化组装的小分子，其靶向引起强直性营养不良1型（Dml），r（CUG）exp的非编码扩增RNA重复。研究了不同的模块化组装支架，包括多胺，α-肽，β-肽和肽叔酰胺（PTAs）。基于通过改善DM1相关缺陷，对蛋白酶的稳定性，细胞通透性和毒性评估的活性，我们发现受限制的骨架即PT As是最佳的。

7. 发明申请

WO2008103489A3 RNA TARGETING COMPOUNDS AND METHODS FOR MAKING AND USING SAME 审中-公开
标题翻译： RNA靶向化合物及其制备和使用方法
公开(公告)号：WO2008103489A3
公开(公告)日：2008-12-18
申请号：PCT/US2008002438
申请日：2008-02-25
申请人： UNIV NEW YORK STATE RES FOUND , DISNEY MATTHEW D
发明人： DISNEY MATTHEW D
IPC分类号： A61K38/16
CPC分类号： C07K4/00 , A61K31/7008 , C07D249/04 , C07K9/00 , C07K14/001 , Y10T436/143333
摘要： Disclosed are RNA targeting compounds having the formula : wherein j is an integer from 1 to 100; each i is the same or different and is zero or an integer from 1 to 100; each Z1 represents the same or different linking moiety; each R1 is the same or different and represents an alkyl group or an aryl group; each Q1 represents the same or different RNA binding ligand; Q2 is an alkyl group; Q3 is a halogen, an alkyl group, an aryl group, or an amine. Also disclosed are RNA targeting compounds that include a polymer backbone and two or more pendant RNA binding ligands that are bound to the polymer backbone. Methods for using the subject RNA targeting compounds to treat myotonic dystrophy and other diseases are also disclosed, as are compounds that can be used to prepare the subject RNA targeting compounds.
摘要翻译：公开了具有下式的RNA靶向化合物：其中j是1至100的整数; 每个i相同或不同，为零或1至100的整数; 每个Z 1代表相同或不同的连接部分; 每个R 1相同或不同，表示烷基或芳基; 每个Q1代表相同或不同的RNA结合配体; Q2是烷基; Q3是卤素，烷基，芳基或胺。还公开了包含聚合物主链和结合至聚合物主链的两个或更多个侧挂RNA结合配体的RNA靶向化合物。还公开了使用目标RNA靶向化合物治疗肌强直性营养不良和其他疾病的方法，以及可用于制备目标RNA靶向化合物的化合物。

8. 发明申请

WO2019246120A3 MICROARRAY AND METHOD OF IDENTIFYING INTERACTIONS BETWEEN COMPOUNDS AND RNA 审中-公开
公开(公告)号：WO2019246120A3
公开(公告)日：2019-12-26
申请号：PCT/US2019/037762
申请日：2019-06-18
申请人： DISNEY, Matthew D. , CHILDS-DISNEY, Jessica L. , VELAGAPUDI, Sai Pradeep , TRAN, Tuan
发明人： DISNEY, Matthew D. , CHILDS-DISNEY, Jessica L. , VELAGAPUDI, Sai Pradeep , TRAN, Tuan
IPC分类号： B01J19/00 , C12Q1/6804 , C12Q1/6837 , C12Q1/6883 , G01N33/50
摘要： The invention provides a microarray having a substrate coated with a gel and a plurality of compounds that are non-covalently adhered to the gel at discrete locations. The invention also provides a method of identifying binding interactions between a compound and an RNA by using a microarray. The invention also provides a method of treating a subject suffering from an RNA mediated disease by administering compounds identified has having a binding interaction with the RNA which mediate the disease. For example, the invention provides a method of preventing or reducing cancer metathesis in a subject by administering to the subject a topoisomerase inhibitor which binds with miR-21.

9. 发明申请

WO2015021415A1 TRANSCRIPTOME-WIDE DESIGN OF SELECTIVE, BIOACTIVE SMALL MOLECULES TARGETING RNA 审中-公开
标题翻译：选择性，生物活性小分子靶向RNA的转录式设计
公开(公告)号：WO2015021415A1
公开(公告)日：2015-02-12
申请号：PCT/US2014/050399
申请日：2014-08-08
申请人： THE SCRIPPS RESEARCH INSTITUTE , DISNEY, Matthew D. , VELAGAPUDI, Sai
发明人： DISNEY, Matthew D. , VELAGAPUDI, Sai
IPC分类号： C07D235/18 , C07D403/14 , C07H11/00 , C40B40/08 , G06E1/00 , G06N3/00
CPC分类号： G06F19/16 , C07D235/18 , C07D235/20 , C07D403/14 , C07H15/04 , C07H15/234 , G01N33/5308 , G01N2500/04 , G06F19/22
摘要： Methods and computer systems are described herein for identifying small molecules that bind to selected RNA structural features (e.g., to RNA secondary structures). Also described are compounds and compositions that modulate RNA function and/or activity.
摘要翻译：本文描述了用于鉴定结合所选RNA结构特征（例如，到RNA二级结构）的小分子的方法和计算机系统。还描述了调节RNA功能和/或活性的化合物和组合物。

10. 发明申请

WO2008103489A2 RNA TARGETING COMPOUNDS AND METHODS FOR MAKING AND USING SAME 审中-公开
标题翻译： RNA靶向化合物及其制备和使用方法
公开(公告)号：WO2008103489A2
公开(公告)日：2008-08-28
申请号：PCT/US2008/002438
申请日：2008-02-25
申请人： THE RESEARCH FOUNDATION OF STATE UNIVERSITY OF NEWYORK , DISNEY, Matthew, D.
发明人： DISNEY, Matthew, D.
IPC分类号： G01N33/00
CPC分类号： C07K4/00 , A61K31/7008 , C07D249/04 , C07K9/00 , C07K14/001 , Y10T436/143333
摘要： Disclosed are RNA targeting compounds having the formula : wherein j is an integer from 1 to 100; each i is the same or different and is zero or an integer from 1 to 100; each Z 1 represents the same or different linking moiety; each R 1 is the same or different and represents an alkyl group or an aryl group; each Q 1 represents the same or different RNA binding ligand; Q 2 is an alkyl group; Q 3 is a halogen, an alkyl group, an aryl group, or an amine. Also disclosed are RNA targeting compounds that include a polymer backbone and two or more pendant RNA binding ligands that are bound to the polymer backbone. Methods for using the subject RNA targeting compounds to treat myotonic dystrophy and other diseases are also disclosed, as are compounds that can be used to prepare the subject RNA targeting compounds.
摘要翻译：公开了具有下式的RNA靶向化合物：其中j是1至100的整数; 每个i相同或不同，为零或1至100的整数; 每个Z 1代表相同或不同的连接部分; 每个R 1相同或不同，表示烷基或芳基; 每个Q 1表示相同或不同的RNA结合配体; Q 2是烷基; Q 3是卤素，烷基，芳基或胺。还公开了包含聚合物主链和结合至聚合物主链的两个或更多个侧挂RNA结合配体的RNA靶向化合物。还公开了使用目标RNA靶向化合物治疗肌强直性营养不良和其他疾病的方法，以及可用于制备目标RNA靶向化合物的化合物。

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