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1. 发明申请

US20110053975A1 CHEMICAL MOLECULES THAT INHIBIT THE SLICING MECHANISM FOR TREATING DISEASES RESULTING FROM SPLICING ANOMALIES 有权
标题翻译：化学分子，禁止用于治疗由分离异常引起的疾病的机制
公开(公告)号：US20110053975A1
公开(公告)日：2011-03-03
申请号：US12811931
申请日：2009-01-12
申请人： Jamal Tazi , David Grierson , Florence Mahuteau-Betzer , Pierre Roux
发明人： Jamal Tazi , David Grierson , Florence Mahuteau-Betzer , Pierre Roux
IPC分类号： A61K31/4709 , C07D213/72 , C07D211/72 , C07D401/12 , C07D401/14 , C07D249/04 , C07D217/22 , A61K31/444 , A61K31/4439 , A61K31/4164 , A61K31/4192 , A61P31/12
CPC分类号： A61K31/166 , A61K31/417 , A61K31/4192 , A61K31/4406 , A61K31/4409 , A61K31/4439 , A61K31/4725 , C07C237/30 , C07D213/38 , C07D213/56 , C07D213/74 , C07D213/75 , C07D213/82 , C07D233/61 , C07D233/64 , C07D249/06 , C07D401/12 , C07D401/14
摘要： The present invention relates to a compound of one of the formulas I to XXI; a pharmaceutical composition comprising at least one such compound; and the use of at least one such compound in preparing a drug to treat, in a subject, a genetic disease resulting from at least one splicing anomaly.
摘要翻译：本发明涉及式I至XXI之一的化合物; 包含至少一种此类化合物的药物组合物; 以及至少一种这样的化合物在制备药物中的用途，以在受试者中治疗由至少一个拼接异常引起的遗传疾病。

2. 发明授权

US08604063B2 Chemical molecules that inhibit the slicing mechanism for treating diseases resulting from splicing anomalies 有权
标题翻译：抑制切片机制用于治疗由拼接异常引起的疾病的化学分子
公开(公告)号：US08604063B2
公开(公告)日：2013-12-10
申请号：US12811931
申请日：2009-01-12
申请人： Jamal Tazi , David Grierson , Florence Mahuteau-Betzer , Pierre Roux
发明人： Jamal Tazi , David Grierson , Florence Mahuteau-Betzer , Pierre Roux
IPC分类号： A61K31/44 , A61K31/235 , C07D213/72
CPC分类号： A61K31/166 , A61K31/417 , A61K31/4192 , A61K31/4406 , A61K31/4409 , A61K31/4439 , A61K31/4725 , C07C237/30 , C07D213/38 , C07D213/56 , C07D213/74 , C07D213/75 , C07D213/82 , C07D233/61 , C07D233/64 , C07D249/06 , C07D401/12 , C07D401/14
摘要： The present invention relates to a compound of one of the formulas I to XXI; a pharmaceutical composition comprising at least one such compound; and the use of at least one such compound in preparing a drug to treat, in a subject, a genetic disease resulting from at least one splicing anomaly.
摘要翻译：本发明涉及式I至XXI之一的化合物; 包含至少一种这样的化合物的药物组合物; 以及至少一种这样的化合物在制备药物中的用途，以在受试者中治疗由至少一个拼接异常引起的遗传疾病。

3. 发明授权

US08895726B2 Compounds derived from indole and pharmaceutical compositions containing them 有权
标题翻译：衍生自吲哚的化合物和含有它们的药物组合物
公开(公告)号：US08895726B2
公开(公告)日：2014-11-25
申请号：US12527071
申请日：2008-02-19
申请人： Fabrice Lejeune , Jamal Tazi , David Grierson , Christian Rivalle , Florence Mahuteau-Betzer
发明人： Fabrice Lejeune , Jamal Tazi , David Grierson , Christian Rivalle , Florence Mahuteau-Betzer
IPC分类号： C07D345/00 , C07D517/00 , C07D471/04 , C07D471/14 , C07D401/12
CPC分类号： C07D471/14 , C07D401/12 , C07D471/04
摘要： The present invention relates to a compound derived from indole corresponding to the following formula II: wherein X represents N, CR8 or N+R8, wherein R8 represents a hydrogen atom, a hydroxyl or alkyl or methoxy group optionally substituted with a phenyl group; R2, R3 and R4 independently represent a hydrogen atom or a halogen atom or an optionally substituted alkyl, amine, alkene, ester, sulfonamide, ether or benzyl group; R5 represents a hydrogen atom or an optionally substituted, saturated or unsaturated alkyl group, amine, benzyl group; R6 represents an optionally substituted C1-C3 alkyl group; R7 represents a hydrogen atom or an optionally substituted C1-C3 alkyl group and R7 is absent when the ring A is in the b position, and A represents a ring; R9 and R10 represent together a carbon bond or independently represent an R11 OR11, SR11 group; wherein R11 represents a hydrogen atom, an optionally substituted, saturated or unsaturated, C1-C3 alkyl group, which may contain one or more sulfur, oxygen or nitrogen atoms; pharmaceutically acceptable salts of said compounds, their isomers and/or a mixture thereof; pharmaceutical composition comprising such a compound; the use of such a compound for preparing a drug intended to treat a genetic disease resulting from at least one mutation causing the occurrence of an early termination codon.
摘要翻译：本发明涉及对应于下式II的衍生自吲哚的化合物：其中X表示N，CR8或N + R8，其中R8表示氢原子，羟基或任选被苯基取代的烷基或甲氧基; R2，R3和R4独立地表示氢原子或卤素原子或任选取代的烷基，胺，烯烃，酯，磺酰胺，醚或苄基; R5表示氢原子或任选取代的饱和或不饱和的烷基，胺，苄基; R 6表示任选取代的C 1 -C 3烷基; R7表示氢原子或任意取代的C1-C3烷基，当环A在b位时，R7不存在，A表示环; R 9和R 10一起表示碳键或独立地表示R11 OR11，SR11基团; 其中R11表示氢原子，任选取代的，饱和或不饱和的C 1 -C 3烷基，其可以含有一个或多个硫，氧或氮原子; 所述化合物的药学上可接受的盐，它们的异构体和/或它们的混合物; 包含这种化合物的药物组合物; 使用这种化合物来制备旨在治疗由引起早期终止密码子的发生的至少一个突变引起的遗传疾病的药物。

4. 发明申请

US20110003843A1 NOVEL COMPOUNDS DERIVED FROM INDOLE AND PHARMACEUTICAL COMPOSITIONS CONTAINING THEM 有权
标题翻译：从含有它们的药物和药物组合物衍生的新化合物
公开(公告)号：US20110003843A1
公开(公告)日：2011-01-06
申请号：US12527071
申请日：2008-02-19
申请人： Fabrice Lejeune , Jamal Tazi , David Grierson , Christian Rivalle , Florence Mahuteau-Betzer
发明人： Fabrice Lejeune , Jamal Tazi , David Grierson , Christian Rivalle , Florence Mahuteau-Betzer
IPC分类号： A61K31/4745 , C07D471/14 , C07D471/04 , C07D217/24 , A61K31/47 , A61P9/00 , A61P21/00 , A61P27/02 , A61P11/00 , A61P17/00 , A61P13/00 , A61P1/00 , A61P15/08
CPC分类号： C07D471/14 , C07D401/12 , C07D471/04
摘要： The present invention relates to a compound derived from indole corresponding to the following formula II: wherein X represents N, CR8 or N+R8, wherein R8 represents a hydrogen atom, a hydroxyl or alkyl or methoxy group optionally substituted with a phenyl group; R2, R3 and R4 independently represent a hydrogen atom or a halogen atom or an optionally substituted alkyl, amine, alkene, ester, sulfonamide, ether or benzyl group; R5 represents a hydrogen atom or an optionally substituted, saturated or unsaturated alkyl group, amine, benzyl group; R6 represents an optionally substituted C1-C3 alkyl group; R7 represents a hydrogen atom or an optionally substituted C1-C3 alkyl group and R7 is absent when the ring A is in the b position, and A represents a ring; R9 and R10 represent together a carbon bond or independently represent an R11 OR11, SR11 group; wherein R11 represents a hydrogen atom, an optionally substituted, saturated or unsaturated, C1-C3 alkyl group, which may contain one or more sulfur, oxygen or nitrogen atoms; pharmaceutically acceptable salts of said compounds, their isomers and/or a mixture thereof; pharmaceutical composition comprising such a compound; the use of such a compound for preparing a drug intended to treat a genetic disease resulting from at least one mutation causing the occurrence of an early termination codon.
摘要翻译：本发明涉及对应于下式II的衍生自吲哚的化合物：其中X表示N，CR8或N + R8，其中R8表示氢原子，羟基或任选被苯基取代的烷基或甲氧基; R2，R3和R4独立地表示氢原子或卤素原子或任选取代的烷基，胺，烯烃，酯，磺酰胺，醚或苄基; R5表示氢原子或任选取代的饱和或不饱和的烷基，胺，苄基; R 6表示任选取代的C 1 -C 3烷基; R7表示氢原子或任意取代的C1-C3烷基，当环A在b位时，R7不存在，A表示环; R 9和R 10一起表示碳键或独立地表示R11 OR11，SR11基团; 其中R11表示氢原子，任选取代的，饱和或不饱和的C 1 -C 3烷基，其可以含有一个或多个硫，氧或氮原子; 所述化合物的药学上可接受的盐，它们的异构体和/或它们的混合物; 包含这种化合物的药物组合物; 使用这种化合物来制备旨在治疗由引起早期终止密码子的发生的至少一个突变引起的遗传疾病的药物。

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