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1. 发明专利

AU2014290206A1 Specific targeting of RNA expanded repeat sequences 未知
公开(公告)号：AU2014290206A1
公开(公告)日：2017-05-11
申请号：AU2014290206
申请日：2014-07-15
申请人： DISNEY MATTHEW , SCRIPPS RESEARCH INST , GUAN LIRUI , 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)

2. 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)

3. 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.

4. 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

5. 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。

6. 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。

7. 发明申请

WO2003002518A1 NOVEL 2,4-DIFLUOROBENZAMIDE DERIVATIVES AS ANTIVIRAL AGENTS 审中-公开
标题翻译：新的2,4-二氟苯甲酰胺衍生物作为抗病毒剂
公开(公告)号：WO2003002518A1
公开(公告)日：2003-01-09
申请号：PCT/KR2002/001239
申请日：2002-06-28
申请人： DONG HWA PHARM. IND. CO., LTD. , CHUNG, Yong-Ho , LEE, Chi-Woo , YANG, Wang-Yong , LEE, Hak-Dong , PARK, Whui-Jung , CHANG, Yoon-Young , LEE, Jin-Soo
发明人： CHUNG, Yong-Ho , LEE, Chi-Woo , YANG, Wang-Yong , LEE, Hak-Dong , PARK, Whui-Jung , CHANG, Yoon-Young , LEE, Jin-Soo
IPC分类号： C07C233/11
CPC分类号： C07D249/08 , C07D231/12 , C07D231/56 , C07D233/56 , C07D401/12 , C07D403/12
摘要： 2,4-difluorobenzamide derivatives and their pharmaceutically acceptable salts, represented by following formula (1), are described in which the derivatives have excellent inhibitory effects on proliferation of hepatitis B virus (HBV) and hepatitis C virus (HCV) so that they can be easily used as an effective ingredient against viruses. In addition, the process for preparing the derivatives is also described.
摘要翻译：描述了以下式（1）表示的2,4-二氟苯甲酰胺衍生物及其药学上可接受的盐，其中衍生物对乙型肝炎病毒（HBV）和丙型肝炎病毒（HCV）的增殖具有优异的抑制作用，容易被用作抗病毒的有效成分。此外，还描述了制备衍生物的方法。

8. 发明申请

WO2002079187A1 METHOXY-1,3,5-TRIAZINE DERIVATIVES AS ANTIVIRAL AGENTS 审中-公开
标题翻译：甲氧基-1,3,5-三唑衍生物作为抗病毒剂
公开(公告)号：WO2002079187A1
公开(公告)日：2002-10-10
申请号：PCT/KR2002/000565
申请日：2002-03-30
申请人： DONG WHA PHARM. IND. CO., LTD , CHUNG, Yong-Ho , LEE, Chi-Woo , YANG, Wang-Yong , LEE, Hak-Dong , PARK, Whui-Jung , CHANG, Yoon-Young , LEE, Jin-Soo
发明人： CHUNG, Yong-Ho , LEE, Chi-Woo , YANG, Wang-Yong , LEE, Hak-Dong , PARK, Whui-Jung , CHANG, Yoon-Young , LEE, Jin-Soo
IPC分类号： C07D401/02
CPC分类号： C07D403/14 , C07D401/14 , C07D403/12 , C07D413/14
摘要： Methoxy-1,3,5-triazine derivatives and their pharmaceutically acceptable salts are described in which the derivatives have excellent inhibitory effects on proliferation of hepatitis B virus(HBV) and hepatitis C virus(HCV) so that they can be easily used as an effective ingredient against viruses. In addition, the process for preparing the derivatives is also described.
摘要翻译：描述了甲氧基-1,3,5-三嗪衍生物及其药学上可接受的盐，其中所述衍生物对乙型肝炎病毒（HBV）和丙型肝炎病毒（HCV）的增殖具有优异的抑制作用，使得它们可以容易地用作针对病毒的有效成分。此外，还描述了制备衍生物的方法。

9. 发明申请

WO9958526A8 NOVEL 2,5-PYRIDINEDICARBOXYLIC ACID DERIVATIVES 审中-公开
标题翻译：新的2,5-吡啶二羧酸衍生物
公开(公告)号：WO9958526A8
公开(公告)日：2001-03-08
申请号：PCT/KR9900213
申请日：1999-05-01
申请人： DONG WHA PHARM IND CO LTD , YOON SUNG JOON , LEE SANG WOOK , SIM HYEONG SU , PARK YONG KYUN , YANG WANG YONG , KIM JONG WOO , HAN JAE JIN , YOON JE IN , PARK SANG JIN , PARK HEE JEOUNG , SIN DONG HYUK , CHANG HWAN BONG
发明人： YOON SUNG JOON , LEE SANG WOOK , SIM HYEONG SU , PARK YONG KYUN , YANG WANG YONG , KIM JONG WOO , HAN JAE JIN , YOON JE IN , PARK SANG JIN , PARK HEE JEOUNG , SIN DONG HYUK , CHANG HWAN BONG
IPC分类号： C07D213/82 , C07D401/14 , C07D405/12 , C07D405/14 , C07D409/14 , C07D413/14 , C07D417/14 , A61K31/50
CPC分类号： C07D213/82 , C07D401/14 , C07D405/12 , C07D405/14 , C07D409/14 , C07D413/14 , C07D417/14
摘要： The present invention relates to novel 2,5-pyridinedicarboxylic acid derivatives and more particularly, to novel 2,5-pyridinedicarboxylic acid derivatives of formula (1) and its pharmaceutically acceptable salts as anti retroviral agents, having inhibitory activities against the proliferation of retrovirus such as hepatitis B virus and human immunodeficiency virus, including its preparing method, wherein, R1, R2, R3 and n are the same as defined in the detailed description of the invention, respectively.
摘要翻译：本发明涉及新的2,5-吡啶二羧酸衍生物，更具体地说，涉及新的式（1）的2,5-吡啶二羧酸衍生物及其药学上可接受的盐作为抗逆转录病毒剂，具有抗逆转录病毒增殖的抑制活性作为乙型肝炎病毒和人类免疫缺陷病毒，包括其制备方法，其中R1，R2，R3和n分别与本发明的详细描述中所定义相同。

10. 发明申请

WO2015009678A3 SPECIFIC TARGETING OF RNA EXPANDED REPEAT SEQUENCES 审中-公开
标题翻译： RNA扩增重复序列的特异性靶向
公开(公告)号：WO2015009678A3
公开(公告)日：2015-03-19
申请号：PCT/US2014046626
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
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。

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