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1.发明公开METHODS AND COMPOSITIONS USEFUL IN TREATMENT OF DISEASES OR CONDITIONS RELATED TO REPEAT EXPANSION 审中-公开用于疾病的治疗或方法和组合物遭受具有重复加宽CONNECTION
公开(公告)号：EP2536738A1
公开(公告)日：2012-12-26
申请号：EP11740540.7
申请日：2011-02-08
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/315 , C12N2310/322 , C12N2310/3231 , C12N2310/341 , C12N2310/344 , C12N2310/346
摘要： The present invention is drawn to chemically-modified oligomers that are complementary to, and capable of hybridizing within the repeat region of CAG, CUG, or CCUG nucleotide repeat-containing RNAs (NRRs).
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公开(公告)号：EP2751269A1
公开(公告)日：2014-07-09
申请号：EP12827475.0
申请日：2012-08-29
CPC分类号： C12N15/113 , A61K31/7088 , C12N2310/113 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/323 , C12N2310/3341 , C12N2310/341 , C12N2310/343 , C12N2310/344 , C12N2310/351 , C12N2310/3521 , C12N2310/3533 , C12N2310/533 , C12N2320/34
摘要： Described are compounds and methods useful for the treatment and investigation of diseases and disorders associated with expanded repeat-containing RNA molecules.
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3.发明公开
公开(公告)号：EP3027617A2
公开(公告)日：2016-06-08
申请号：EP14832900.6
申请日：2014-07-31
发明人： PRAKASH, Thazha, P. , HU, Jiaxin , LIU, Jing , YU, Dongbo , COREY, David , SWAYZE, Eric, E.
IPC分类号： C07D471/04
CPC分类号： C07H21/02 , C12N15/113 , C12N2310/11 , C12N2310/32 , C12N2320/34
摘要： Described are compounds and methods useful for the treatment and investigation of diseases and disorders associated with expanded repeat-containing RNA molecules. In certain embodiments, compounds and methods useful for the modulation of ATXN-3 pre-mRNA are described. In certain embodiments, compounds and methods useful for the modulation of ATN-1 mRNA are described.
摘要翻译： 描述了用于治疗和研究与包含扩增重复的RNA分子相关的疾病和病症的化合物和方法。 在某些实施方案中，描述了用于调节ATXN-3前体mRNA的化合物和方法。 在某些实施方案中，描述了用于调节ATN-1mRNA的化合物和方法。
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公开(公告)号：EP2751269B1
公开(公告)日：2016-03-23
申请号：EP12827475.0
申请日：2012-08-29
CPC分类号： C12N15/113 , A61K31/7088 , C12N2310/113 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/323 , C12N2310/3341 , C12N2310/341 , C12N2310/343 , C12N2310/344 , C12N2310/351 , C12N2310/3521 , C12N2310/3533 , C12N2310/533 , C12N2320/34
摘要： Described are compounds and methods useful for the treatment and investigation of diseases and disorders associated with expanded repeat-containing RNA molecules.
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5.发明授权DOUBLE STRAND COMPOSITIONS COMPRISING DIFFERENTIALLY MODIFIED STRANDS FOR USE IN GENE MODULATION 有权有使用的基因调节其他的变形STRANDS双组份
公开(公告)号：EP2173358B1
公开(公告)日：2015-10-21
申请号：EP08771759.1
申请日：2008-06-23
发明人： BHAT, Balkrishen , PRAKASH, Thazha, P. , ALLERSON, Charles , KINBERGER, Garth, A. , MARCUSSON, Eric, G. , SWAYZE, Eric, E.
IPC分类号： C12N15/113 , C12N15/11
CPC分类号： C12N15/111 , C12N15/113 , C12N15/1137 , C12N2310/14 , C12N2310/315 , C12N2310/317 , C12N2310/321 , C12N2310/322 , C12N2310/323 , C12N2310/341 , C12N2310/346 , C12N2310/3515 , C12N2310/3525 , C12N2310/3533 , C12N2320/51 , C12Y301/03048 , C12N2310/3521
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公开(公告)号：EP2906258A2
公开(公告)日：2015-08-19
申请号：EP13847957.1
申请日：2013-10-15
IPC分类号： A61K48/00
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/313 , C12N2310/315 , C12N2310/322 , C12N2310/3233 , C12N2310/3341 , C12N2310/341 , C12N2310/346 , C12N2310/3525 , C12N2310/3533
摘要： Disclosed herein are compositions and methods for reducing expression of C90RF72 mRNA and protein in an animal with C90RF72 specific inhibitors. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C90RF72 specific inhibitors include antisense compounds. Examples of neurodegenerative diseases that can be treated, prevented, and ameliorated with the administration C90RF72 specific inhibitors include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), corticalbasal degeneration syndrome (CBD), atypical Parkinsonian syndrome, and olivopontocerellar degeneration (OPCD).
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公开(公告)号：EP2906255A2
公开(公告)日：2015-08-19
申请号：EP13844964.0
申请日：2013-10-11
CPC分类号： C12N15/113 , C07H21/00 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/323 , C12N2310/341 , C12N2310/346 , C12N2310/3525 , C12N2310/3533
摘要： The present disclosure provides oligomeric compounds. Certain such oligomeric compounds are useful for hybridizing to a complementary nucleic acid, including but not limited, to nucleic acids in a cell. In certain embodiments, hybridization results in modulation of the amount activity or expression of the target nucleic acid in a cell.
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公开(公告)号：EP2023939B1
公开(公告)日：2012-06-27
申请号：EP07811874.2
申请日：2007-05-07
发明人： BHANOT, Sanjay , GEARY, Richard, S. , MCKAY, Robert , MONIA, Brett, P. , SETH, Punit, P. , SIWKOWSKI, Andrew, M. , SWAYZE, Eric, E. , WANCEWICZ, Edward
IPC分类号： C12N15/113 , A61K31/712 , A61P3/00 , A61P9/00
CPC分类号： C12N15/113 , C12N15/1137 , C12N2310/11 , C12N2310/31 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/341 , C12N2310/3515 , C12Y203/0102 , C12N2310/3521
摘要： The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense compound are useful for the reduction of target nucleic acids and/or proteins in cells, tissues, and animals with increased potency and improved therapeutic index. Thus, provided herein are short antisense compounds comprising high-affinity nucleotide modifications useful for reducing a target RNA in vivo. Such short antisense compounds are effective at lower doses than previously described antisense compounds, allowing for a reduction in toxicity and cost of treatment. In addition, the described short antisense compounds have greater potential for oral dosing.
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9.发明授权
公开(公告)号：EP2176280B1
公开(公告)日：2011-12-21
申请号：EP08781239.2
申请日：2008-07-01
发明人： SWAYZE, Eric, E. , SETH, Punit, P.
摘要： The present disclosure describes 6-disubstituted bicyclic nucleosides, oligomeric compounds prepared therefrom and methods of using the oligomeric compounds. More particularly, the 6-disubstituted bicyclic nucleosides each comprise a 2'-O-C(Ri)(R2)-4' or 2'-O- C=(R3)(R.4)-4' bridge wherein each R is, independently a substituent group and Ri and R2 include H. The 6-disubstituted bicyclic nucleosides are useful for enhancing properties of oligomeric compounds including nuclease resistance. In certain embodiments, the oligomeric compounds provided herein hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA.
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10.发明公开
公开(公告)号：EP2176280A2
公开(公告)日：2010-04-21
申请号：EP08781239.2
申请日：2008-07-01
发明人： SWAYZE, Eric, E. , SETH, Punit, P.
摘要： The present disclosure describes 6-disubstituted bicyclic nucleosides, oligomeric compounds prepared therefrom and methods of using the oligomeric compounds. More particularly, the 6-disubstituted bicyclic nucleosides each comprise a 2'-O-C(Ri)(R2)-4' or 2'-O- C=(R3)(R.4)-4' bridge wherein each R is, independently a substituent group and Ri and R2 include H. The 6-disubstituted bicyclic nucleosides are useful for enhancing properties of oligomeric compounds including nuclease resistance. In certain embodiments, the oligomeric compounds provided herein hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA.
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