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41. 发明授权

US09066967B2 Oligonucleotide analogues targeting human LMNA 有权
标题翻译：靶向人LMNA的寡核苷酸类似物
公开(公告)号：US09066967B2
公开(公告)日：2015-06-30
申请号：US13708708
申请日：2012-12-07
申请人： Sarepta Therapeutics, Inc.
发明人： Ryszard Kole , Richard Keith Bestwick
IPC分类号： C12N15/113 , A61K31/713 , C12N15/11
CPC分类号： A61K31/713 , A61K47/6455 , C12N15/111 , C12N15/113 , C12N2310/11 , C12N2310/314 , C12N2310/3233 , C12N2310/3513 , C12N2320/33
摘要： Provided are LMNA-targeted antisense oligonucleotides for reducing expression of one or more aberrantly spliced LMNA mRNA isoforms that encode progerin.
摘要翻译：提供了用于减少编码progerin的一个或多个异常剪接的LMNA mRNA同种型的表达的LMNA靶向反义寡核苷酸。

42. 发明申请

US20150080340A1 BORONIC ACID CONJUGATES OF OLIGONUCLEOTIDE ANALOGUES 有权
标题翻译：寡核苷酸类似物的硼酸结合物（BORONIC ACID CONJUGATES OF OLIGONUCLEOTIDE ANALOGUES）
公开(公告)号：US20150080340A1
公开(公告)日：2015-03-19
申请号：US14386720
申请日：2013-03-07
申请人： Sarepta Therapeutics, Inc.
发明人： Gunnar J. Hanson
IPC分类号： C07F9/6596 , C07F9/6558
CPC分类号： C07F9/6596 , C07F9/65583 , C12N15/113 , C12N2310/31 , C12N2310/3233 , C12N2310/331 , C12N2310/351
摘要： Oligonucleotide analogues comprising boronic acid and/or boronic ester moieties are provided. The disclosed compounds are useful for the treatment of diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects.
摘要翻译：提供了包含硼酸和/或硼酸酯部分的寡核苷酸类似物。所公开的化合物可用于治疗其中抑制蛋白质表达或修正异常mRNA剪接产物产生有益治疗效果的疾病。

43. 发明申请

US20150073140A1 OLIGONUCLEOTIDE ANALOGUES HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS 审中-公开
标题翻译：具有修改的互联网链接和/或终端组的寡核苷酸类似物
公开(公告)号：US20150073140A1
公开(公告)日：2015-03-12
申请号：US14298655
申请日：2014-06-06
申请人： Sarepta Therapeutics, Inc.
发明人： Gunnar J. Hanson , Alexander Charles Rudolph , Bao Zhong Cai , Ming Zhou , Dwight D. Weller
IPC分类号： C07F9/6544 , C07F9/26 , C07F9/22
CPC分类号： C12N15/113 , A61K31/496 , A61K31/5377 , C07D265/30 , C07D295/108 , C07F9/222 , C07F9/26 , C07F9/572 , C07F9/59 , C07F9/6544 , C07F9/65583 , C07F9/6561 , C07F9/65846 , C07H21/00 , C12N15/1131 , C12N2310/11 , C12N2310/3233 , C12N2310/3513
摘要： Oligonucleotide analogues comprising modified intersubunit linkages and/or modified 3′ and/or 5′-end groups are provided. The disclosed compounds are useful for the treatment of diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects.
摘要翻译：提供了包含修饰的亚单位间键和/或修饰的3'和/或5'-端基的寡核苷酸类似物。所公开的化合物可用于治疗其中抑制蛋白质表达或修正异常mRNA剪接产物产生有益治疗效果的疾病。

44. 发明申请

US20140330006A1 FUNCTIONALLY-MODIFIED OLIGONUCLEOTIDES AND SUBUNITS THEREOF 有权
标题翻译：功能改性的寡核苷酸及其亚基
公开(公告)号：US20140330006A1
公开(公告)日：2014-11-06
申请号：US14358992
申请日：2012-11-15
申请人： Sarepta Therapeutics, Inc.
发明人： Gunnar J. Hanson , Dwight D. Weller , Bao Zhong Cai , Ming Zhou
IPC分类号： C07F9/6558 , C07F9/6584 , C07F9/6561
CPC分类号： C12N15/113 , C07F9/65583 , C07F9/65586 , C07F9/65616 , C07F9/65846 , C07H21/00 , C12N15/1131 , C12N2310/11 , C12N2310/314 , C12N2310/3233 , C12N2310/346 , C12N2310/351 , C12N2310/3513 , C12N2310/353
摘要： Functionally-modified oligonucleotide analogues comprising modified intersubunit linkages and/or modified 3′ and/or 5′-end groups are provided. The disclosed compounds are useful for the treatment of diseases where inhibition of protein expression or correction of aberrant mRNA splice products produces beneficial therapeutic effects.
摘要翻译：提供包含修饰的亚单位间键和/或修饰的3'和/或5'-端基的功能修饰的寡核苷酸类似物。所公开的化合物可用于治疗其中抑制蛋白质表达或修正异常mRNA剪接产物产生有益治疗效果的疾病。

45. 发明授权

US08865883B2 Multiple exon skipping compositions for DMD 有权
标题翻译：用于DMD的多个外显子跳过组合物
公开(公告)号：US08865883B2
公开(公告)日：2014-10-21
申请号：US13830253
申请日：2013-03-14
申请人： Sarepta Therapeutics, Inc.
发明人： Peter Sazani , Ryszard Kole
IPC分类号： C12N15/113 , C12N15/11
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/321 , C12N2310/3233 , C12N2310/331 , C12N2310/3341 , C12N2310/3513 , C12N2320/30 , C12N2320/33
摘要： Provided are antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping, and methods of use thereof to treat muscular dystrophy.
摘要翻译：提供能够结合人肌营养不良蛋白基因中的选定靶位点以诱导外显子跳跃的反义分子及其用于治疗肌营养不良症的方法。

46. 发明授权

US08835402B2 Compound and method for treating myotonic dystrophy 有权
标题翻译：用于治疗强直性营养不良的化合物和方法
公开(公告)号：US08835402B2
公开(公告)日：2014-09-16
申请号：US14038314
申请日：2013-09-26
申请人： Sarepta Therapeutics, Inc.
发明人： Ryszard Kole , Gunnar J. Hanson
IPC分类号： C12N15/113
CPC分类号： C12N15/1137 , C07K2319/33 , C12N15/113 , C12N15/87 , C12N2310/113 , C12N2310/3233 , C12N2810/40
摘要： Provided are 9-base morpholino antisense compounds targeted to polyCUG repeats in the 3′UTR region of dystrophia myotonica protein kinase (DMPK) mRNA, and related methods for treating myotonic dystrophy DM1.
摘要翻译：提供的是靶向dCKG mRNA的3'UTR区域中的polyCUG重复序列的9位吗啉反义化合物，以及用于治疗强直性营养不良DM1的相关方法。

47. 发明申请

US20140044750A1 ANTISENSE ANTIVIRAL COMPOUNDS AND METHODS FOR TREATING A FILOVIRUS INFECTION 有权
标题翻译：抗真菌化合物和治疗FILOVIRUS感染的方法
公开(公告)号：US20140044750A1
公开(公告)日：2014-02-13
申请号：US13957261
申请日：2013-08-01
申请人： Sarepta Therapeutics, Inc.
发明人： Patrick L. Iversen , Dwight D. Weller
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , A61K31/675 , A61K31/713 , C12N15/1131 , C12N2310/11 , C12N2310/3233
摘要： The present invention provides antisense antiviral compounds, compositions, and methods of their use and production, mainly for inhibiting the replication of viruses of the Filoviridae family, including Ebola and Marburg viruses. The compounds, compositions, and methods also relate to the treatment of viral infections in mammals including primates by Ebola and Marburg viruses. The antisense antiviral compounds include phosphorodiamidate morpholino oligonucleotides (PMOplus) having a nuclease resistant backbone, about 15-40 nucleotide bases, at least two but typically no more than half piperazine-containing intersubunit linkages, and a targeting sequence that is targeted against the AUG start site region of Ebola virus VP35, Ebola virus VP24, Marburg virus VP24, or Marburg virus NP, including combinations and mixtures thereof.
摘要翻译：本发明提供反义抗病毒化合物，组合物及其使用和生产方法，主要用于抑制丝状病毒科病毒的复制，包括埃博拉病毒和马尔堡病毒。化合物，组合物和方法还涉及治疗包括埃博拉和马尔堡病毒在内的哺乳动物中的病毒感染。反义抗病毒化合物包括具有核酸酶抗性主链的磷酸二亚胺吗啉代寡核苷酸（PMOplus），约15-40个核苷酸碱基，至少两个但通常不超过一半的含哌嗪的亚单位间连接，以及针对AUG起始靶向序列埃博拉病毒VP35的位点区域，埃博拉病毒VP24，马尔堡病毒VP24或马尔堡病毒NP，包括其组合和混合物。

48. 发明公开

US20240042049A1 EXON SKIPPING OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY 审中-公开
公开(公告)号：US20240042049A1
公开(公告)日：2024-02-08
申请号：US18050785
申请日：2022-10-28
申请人： Sarepta Therapeutics, Inc.
发明人： Richard K. BESTWICK , Frederick Joseph SCHNELL
IPC分类号： A61K47/68 , A61K9/00 , C07K14/47 , C12N15/113 , C12N15/62
CPC分类号： A61K47/6807 , A61K9/0019 , C07K14/4707 , C12N2310/3233 , C12N15/62 , C12N2310/11 , C12N15/113
摘要： Antisense oligomers complementary to a selected target site in the human dystrophin gene to induce exon 52 skipping are described.

49. 发明授权

US11642364B2 Exon skipping oligomer conjugates for muscular dystrophy 有权
公开(公告)号：US11642364B2
公开(公告)日：2023-05-09
申请号：US17736148
申请日：2022-05-04
申请人： Sarepta Therapeutics, Inc.
发明人： Marco A. Passini , Gunnar J. Hanson
IPC分类号： A61K31/712 , A61K31/7125 , A61P21/00 , C12N15/113 , A61K47/64
CPC分类号： A61K31/712 , A61K31/7125 , A61K47/645 , A61P21/00 , C12N15/113 , C12N2310/111
摘要： Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 51 skipping are described.

50. 发明申请

US20230110479A1 EXON SKIPPING OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY 有权
公开(公告)号：US20230110479A1
公开(公告)日：2023-04-13
申请号：US17730508
申请日：2022-04-27
申请人： Sarepta Therapeutics, Inc.
发明人： Gunnar J. HANSON , Marco A. PASSINI , Frederick Joseph SCHNELL
IPC分类号： A61K47/68 , A61K9/00 , C07K14/47 , C12N15/113 , C12N15/62
摘要： Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 52 skipping are described.

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