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公开(公告)号：EP2812004A2
公开(公告)日：2014-12-17
申请号：EP13747100.9
申请日：2013-02-08
发明人： QI, Hongyan , CHOI, Soongyu , DAKKA, Amal , KARP, Gary, Mitchell , NARASIMHAN, Jana , NARYSHKIN, Nikolai , TURPOFF, Anthony, A. , WEETALL, Marla, L. , WELCH, Ellen , WOLL, Matthew, G. , YANG, Tianle , ZHANG, Nanjing , ZHANG, Xiaoyan , ZHAO, Xin , GREEN, Luke , PINARD, Emmanuel , RATNI, Hasane
IPC分类号： A61K31/53 , A01N43/90 , A61K31/519 , C07D239/70
CPC分类号： C07D471/04 , A61K31/519 , C07D239/70 , C07D487/04 , C07D519/00
摘要： Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.
摘要翻译： 本文提供了化合物，其组合物以及用于治疗脊髓性肌萎缩的用途。
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公开(公告)号：EP2812004B1
公开(公告)日：2018-06-27
申请号：EP13747100.9
申请日：2013-02-08
发明人： QI, Hongyan , CHOI, Soongyu , DAKKA, Amal , KARP, Gary, Mitchell , NARASIMHAN, Jana , NARYSHKIN, Nikolai , TURPOFF, Anthony, A. , WEETALL, Marla, L. , WELCH, Ellen , WOLL, Matthew, G. , YANG, Tianle , ZHANG, Nanjing , ZHANG, Xiaoyan , ZHAO, Xin , GREEN, Luke , PINARD, Emmanuel , RATNI, Hasane
IPC分类号： A61K31/53 , A61K31/519 , C07D471/04 , C07D487/04 , C07D519/00 , A61K31/4375 , A61P25/00
CPC分类号： C07D471/04 , A61K31/519 , C07D239/70 , C07D487/04 , C07D519/00
摘要： Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.
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公开(公告)号：EP3013345B1
公开(公告)日：2017-11-22
申请号：EP14816651.5
申请日：2014-06-23
发明人： DAKKA, Amal , GREEN, Luke , KARP, Gary , NARASIMHAN, Jana , NARYSHKIN, Nikolai , PINARD, Emmanuel , QI, Hongyan , RATNI, Hasane , RISHER, Nicole , WEETALL, Marla , WOLL, Matthew
IPC分类号： C07D263/56 , C07D413/12 , C07D471/04 , C07D487/04 , C07D487/10 , A61K31/497 , A61K31/4965 , A61P25/28
CPC分类号： C07D519/00 , C07D263/56 , C07D413/12 , C07D471/04 , C07D487/04 , C07D487/10
摘要： The present invention provides compounds of formula (I) wherein A, B, X, Y, R1 and R2 are as described herein, as well as pharmaceutically acceptable salts thereof. Further the present invention is concerned with the manufacture of the compounds of formula (I), pharmaceutical compositions comprising them and their use as medicaments.
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公开(公告)号：EP3406252B1
公开(公告)日：2020-05-13
申请号：EP18179491.8
申请日：2013-02-08
发明人： QI, Hongyan , CHOI, Soongyu , DAKKA, Amal , KARP, Gary, Mitchell , NARASIMHAN, Jana , NARYSHKIN, Nikolai A. , TURPOFF, Anthony, A. , WEETALL, Marla, L. , WELCH, Ellen , WOLL, Matthew, G. , YANG, Tianle , ZHANG, Nanjing , ZHANG, Xiaoyan , ZHAO, Xin , GREEN, Luke , PINARD, Emmanuel , RATNI, Hasane
IPC分类号： A61K31/53 , A61K31/519 , C07D471/04 , C07D487/04 , C07D519/00 , A61K31/4375 , A61P25/00
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公开(公告)号：EP2797592B1
公开(公告)日：2019-08-28
申请号：EP12862080.4
申请日：2012-12-28
发明人： WOLL, Matthew G. , CHEN, Guangming , CHOI, Soongyu , DAKKA, Amal , HUANG, Song , KARP, Gary Mitchell , LEE, Chang-Sun , LI, Chunshi , NARASIMHAN, Jana , NARYSHKIN, Nikolai , PAUSHKIN, Sergey , QI, Hongyan , TURPOFF, Anthony A. , WEETALL, Marla L. , WELCH, Ellen , YANG, Tianle , ZHANG, Nanjing , ZHANG, Xiaoyan , ZHAO, Xin , PINARD, Emmanuel , RATNI, Hasane
IPC分类号： A61K31/353 , C07D413/14 , C07D405/14 , C07D311/16 , C07D311/18 , C07D231/12 , C07D405/04 , C07D405/12 , C07D413/04 , C07D417/04 , C07D471/04 , C07D487/04 , C07D491/04 , C07D495/04 , C07D513/04 , C07D519/00 , A61P25/00 , C07D417/14
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公开(公告)号：EP3406252A1
公开(公告)日：2018-11-28
申请号：EP18179491.8
申请日：2013-02-08
发明人： QI, Hongyan , CHOI, Soongyu , DAKKA, Amal , KARP, Gary, Mitchell , NARASIMHAN, Jana , NARYSHKIN, Nikolai A. , TURPOFF, Anthony, A. , WEETALL, Marla, L. , WELCH, Ellen , WOLL, Matthew, G. , YANG, Tianle , ZHANG, Nanjing , ZHANG, Xiaoyan , ZHAO, Xin , GREEN, Luke , PINARD, Emmanuel , RATNI, Hasane
IPC分类号： A61K31/53 , A61K31/519 , C07D471/04 , C07D487/04 , C07D519/00 , A61K31/4375 , A61P25/00
摘要： Provided herein are compounds of formula (IIa1), compositions thereof and uses therewith for treating spinal muscular atrophy.
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公开(公告)号：EP4249472A3
公开(公告)日：2023-12-13
申请号：EP23173467.4
申请日：2016-05-27
IPC分类号： A01N43/90 , A61K31/519 , C07D239/70
摘要： In one aspect, described herein is a recognition element for splicing (REMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains a REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises a REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene construct comprising a REMS, and uses of those artificial gene constructs to modulate functional protein production. In another aspect, provided herein are methods for altering endogenous genes to comprise a REMS, and the use of a compound described herein to modulate the functional protein produced from such altered endogenous genes.
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公开(公告)号：EP3924050A1
公开(公告)日：2021-12-22
申请号：EP20714316.5
申请日：2020-02-10
发明人： WANG, Jiashi , ARNOLD, Michael, A. , DAKKA, Amal , KARP, Gary, Mitchell , NARASIMHAN, Jana , NARYSHKIN, Nikolai, A. , ZHANG, Nanjing
IPC分类号： A61P25/00 , C07D487/04 , A61K31/519
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公开(公告)号：EP3638318A1
公开(公告)日：2020-04-22
申请号：EP18817883.4
申请日：2018-06-13
发明人： BHATTACHARYYA, Anuradha , DAKKA, Amal , EFFENBERGER, Kerstin , GABBETA, Vijayalakshmi , JANI, Minakshi, B. , LI, Wencheng , NARYSHKIN, Nikolai , TROTTA, Christopher , WIEDINGER, Kari
IPC分类号： A61K48/00 , A61K31/711 , C12N15/11 , A61K31/501
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公开(公告)号：EP2809322A1
公开(公告)日：2014-12-10
申请号：EP13740919.9
申请日：2013-01-25
发明人： CHEN, Guangming , DAKKA, Amal , KARP, Gary Mitchell , LI, Chunshi , NARASIMHAN, Jana , NARYSHKIN, Nikolai , WEETALL, Marla L. , WELCH, Ellen , ZHAO, Xin , WEETAL, Marla L.
摘要： Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy.
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