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1. 发明公开

EP2797592A1 COMPOUNDS FOR TREATING SPINAL MUSCULAR ATROPHY 有权
标题翻译：用于治疗脊髓肌萎缩症的化合物
公开(公告)号：EP2797592A1
公开(公告)日：2014-11-05
申请号：EP12862080.4
申请日：2012-12-28
申请人： PTC Therapeutics, Inc. , F.Hoffmann-La Roche AG
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
CPC分类号： C07D487/04 , C07D231/12 , C07D311/16 , C07D311/18 , C07D405/04 , C07D405/12 , C07D405/14 , C07D413/04 , C07D413/14 , C07D417/04 , C07D417/14 , C07D471/04 , C07D491/04 , C07D495/04 , C07D513/04 , C07D519/00
摘要： Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy. In a specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN2 into mRNA that is transcribed from the SMN2 gene. In another specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 into mRNA that is transcribed from the SMN1 gene. In yet another embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 and SMN2 into mRNA that is transcribed from the SMN1 and SMN2 genes, respectively.
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。在具体的实施方案中，本文提供了可用于调节SMN2的外显子7包含到从SMN2基因转录的mRNA中的形式的化合物。在另一个具体的实施方案中，本文提供了可用于调节SMN1的外显子7包含到从SMN1基因转录的mRNA中的形式的化合物。在又一个实施方案中，本文提供了可用于调节SMN1和SMN2的外显子7包含在分别从SMN1和SMN2基因转录的mRNA中的形式的化合物。

2. 发明申请

WO2013101974A8 COMPOUNDS FOR TREATING SPINAL MUSCULAR ATROPHY 审中-公开
公开(公告)号：WO2013101974A8
公开(公告)日：2013-07-04
申请号：PCT/US2012/071899
申请日：2012-12-28
申请人： PTC THERAPEUTICS, INC. , F. HOFFMANN-LA ROCHE AG
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
摘要： Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy. In a specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN2 into mRNA that is transcribed from the SMN2 gene. In another specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 into mRNA that is transcribed from the SMN1 gene. In yet another embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 and SMN2 into mRNA that is transcribed from the SMN1 and SMN2 genes, respectively.

3. 发明申请

WO2013101974A1 COMPOUNDS FOR TREATING SPINAL MUSCULAR ATROPHY 审中-公开
标题翻译：用于治疗脊柱肌瘤的化合物
公开(公告)号：WO2013101974A1
公开(公告)日：2013-07-04
申请号：PCT/US2012/071899
申请日：2012-12-28
申请人： PTC THERAPEUTICS, INC. , F. HOFFMANN-LA ROCHE AG
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
CPC分类号： C07D487/04 , C07D231/12 , C07D311/16 , C07D311/18 , C07D405/04 , C07D405/12 , C07D405/14 , C07D413/04 , C07D413/14 , C07D417/04 , C07D417/14 , C07D471/04 , C07D491/04 , C07D495/04 , C07D513/04 , C07D519/00
摘要： Provided herein are compounds, compositions thereof and uses therewith for treating spinal muscular atrophy. In a specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN2 into mRNA that is transcribed from the SMN2 gene. In another specific embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 into mRNA that is transcribed from the SMN1 gene. In yet another embodiment, provided herein are compounds of a form that may be used to modulate the inclusion of exon 7 of SMN1 and SMN2 into mRNA that is transcribed from the SMN1 and SMN2 genes, respectively.
摘要翻译：本文提供了化合物，其组合物，并用于治疗脊髓性肌肉萎缩。在一个具体实施方案中，本文提供了可用于调节SMN2外显子7包含在从SMN2基因转录的mRNA中的形式的化合物。在另一个具体实施方案中，本文提供了可用于调节SMN1外显子7包含在从SMN1基因转录的mRNA中的形式的化合物。在另一个实施方案中，本文提供的形式的化合物可用于调节SMN1和SMN2的外显子7包含在分别从SMN1和SMN2基因转录的mRNA中。

4. 发明申请

WO2005003316A2 RNA PROCESSING PROTEIN COMPLEXES AND USES THEREOF 审中-公开
标题翻译： RNA加工蛋白复合物及其用途
公开(公告)号：WO2005003316A2
公开(公告)日：2005-01-13
申请号：PCT/US2004/021334
申请日：2004-07-02
申请人： PTC THERAPEUTICS, INC. , TROTTA, Christopher, R. , PAUSHKIN, Sergey
发明人： TROTTA, Christopher, R. , PAUSHKIN, Sergey
IPC分类号： C12N
CPC分类号： C12Q1/6897 , C07K14/47 , C12N9/22
摘要： The invention provides human protein complexes with endonuclease activity. In particular, the invention provides human protein complexes with tRNA splicing endonuclease activity and/or 3' end pre-mRNA endonuclease activity. The invention also provides a splice variant of human Sen2, namely human Sen2deltaEx8, and human protein complexes comprising human Sen2deltaEx8. The human Sen2deltaEx8 complexes have pre-tRNA cleavage activity and/or 3' end pre-mRNA endonuclease activity. The invention also provides human protein complexes with pre-ribosomal RNA cleavage activity. The invention also provides antibodies that immunospecifically bind to a complex described herein or a component thereof, and methods of diagnosing, preventing, treating, managing or ameliorating a disorder utilizing such antibodies. The present invention also provides methods utilizing the complexes described herein, inter alia , in screening, diagnosis, and therapy. The invention further provides methods of preparing and purifying the complexes. The present invention further provides methods of identifying a compound that modulates the expression of a component of a complex described herein, the formation of a complex described herein or the activity of a complex described herein, and methods of preventing, treating, managing or ameliorating a disorder, such as a proliferative disorder, or a symptom thereof utilizing a compound identified in accordance with the methods.
摘要翻译：本发明提供具有内切核酸酶活性的人蛋白复合物。特别地，本发明提供具有tRNA剪接核酸内切酶活性和/或3'末端pre-mRNA内切酶活性的人蛋白复合物。本发明还提供人Sen2的剪接变体，即人Sen2deltaEx8，以及包含人Sen2deltaEx8的人蛋白复合物。人Sen2deltaEx8复合物具有pre-tRNA切割活性和/或3'末端pre-mRNA内切核酸酶活性。本发明还提供具有前核糖体RNA切割活性的人蛋白复合物。本发明还提供免疫特异性结合本文所述的复合物或其组分的抗体，以及利用此类抗体诊断，预防，治疗，控制或改善病症的方法。本发明还提供了利用本文所述的复合物，特别是在筛选，诊断和治疗中的方法。本发明还提供了制备和纯化复合物的方法。本发明还提供了鉴定调节本文所述复合物组分的表达，本文所述的复合物的形成或本文所述复合物的活性的化合物的方法，以及预防，治疗，治疗或改善障碍，例如增殖性疾病，或其症状，利用根据该方法鉴定的化合物。

5. 发明公开

EP1649002A2 RNA PROCESSING PROTEIN COMPLEXES AND USES THEREOF 审中-公开
标题翻译： RNA加工蛋白复合物及其用途
公开(公告)号：EP1649002A2
公开(公告)日：2006-04-26
申请号：EP04777457.5
申请日：2004-07-02
申请人： PTC Therapeutics, Inc.
发明人： TROTTA, Christopher, R. , PAUSHKIN, Sergey
IPC分类号： C12N1/00
CPC分类号： C12Q1/6897 , C07K14/47 , C12N9/22
摘要： The invention provides human protein complexes with endonuclease activity. In particular, the invention provides human protein complexes with tRNA splicing endonuclease activity and/or 3' end pre-mRNA endonuclease activity. The invention also provides a splice variant of human Sen2, namely human Sen2deltaEx8, and human protein complexes comprising human Sen2deltaEx8. The human Sen2deltaEx8 complexes have pre-tRNA cleavage activity and/or 3' end pre-mRNA endonuclease activity. The invention also provides human protein complexes with pre-ribosomal RNA cleavage activity. The invention also provides antibodies that immunospecifically bind to a complex described herein or a component thereof, and methods of diagnosing, preventing, treating, managing or ameliorating a disorder utilizing such antibodies. The present invention also provides methods utilizing the complexes described herein, inter alia, in screening, diagnosis, and therapy. The invention further provides methods of preparing and purifying the complexes. The present invention further provides methods of identifying a compound that modulates the expression of a component of a complex described herein, the formation of a complex described herein or the activity of a complex described herein, and methods of preventing, treating, managing or ameliorating a disorder, such as a proliferative disorder, or a symptom thereof utilizing a compound identified in accordance with the methods.

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