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81. 发明申请

US20170292125A1 MULTIPLE EXON SKIPPING COMPOSITIONS FOR DMD 审中-公开
公开(公告)号：US20170292125A1
公开(公告)日：2017-10-12
申请号：US15349778
申请日：2016-11-11
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.

82. 发明申请

US20160376587A1 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：US20160376587A1
公开(公告)日：2016-12-29
申请号：US15007017
申请日：2016-01-26
申请人： Sarepta Therapeutics, Inc.
发明人： Gunnar J. Hanson , Dwight D. Weller , Bao Zhong Cai , Ming Zhou
IPC分类号： C12N15/113
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。

83. 发明授权

US09499583B2 Antibacterial antisense oligonucleotide and method 有权
公开(公告)号：US09499583B2
公开(公告)日：2016-11-22
申请号：US14977451
申请日：2015-12-21
申请人： Sarepta Therapeutics, Inc.
发明人： Dwight D. Weller , Bruce L. Geller , Patrick L. Iversen , Lucas D. Tilley , Jed N. Hassinger
IPC分类号： C07K7/02
CPC分类号： C07K7/02 , A61K48/00 , C07D295/205 , C07D413/04 , C07D473/34 , C07F9/65583 , C08F112/08 , C12N15/111 , C12N15/113 , C12N2310/11 , C12N2310/3233 , C12N2310/3513 , C12N2320/50
摘要： A method for enhancing, by at least 10 fold, the antibacterial activity of an antisense oligonucleotide composed of morpholino subunits linked by phosphorus-containing intersubunit linkages. The method includes one or both of: conjugating an arginine-rich carrier to a 3′ or 5′ end of the oligonucleotide and modifying the oligonucleotide to contain 20%-50% intersubunit linkages that are positively charged at physiological pH. Also disclosed is an antisense oligonucleotide having enhanced antibacterial activity by virtue of one or both modifications.

84. 发明授权

US09469664B2 Oligonucleotide analogues having modified intersubunit linkages and/or terminal groups 有权
标题翻译：具有修饰的亚单位间键和/或末端基团的寡核苷酸类似物
公开(公告)号：US09469664B2
公开(公告)日：2016-10-18
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 , C07F9/22 , C07F9/26 , C07D265/30 , C07D295/108 , C07F9/59 , C07F9/6561 , C07F9/6584 , C07F9/572 , C07F9/6558 , C07H21/00 , A61K31/496 , A61K31/5377
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 analogs 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。

85. 发明申请

US20150368653A1 ANTISENSE MODULATION OF NUCLEAR HORMONE RECEPTORS 审中-公开
标题翻译：核激素受体的抗原调节
公开(公告)号：US20150368653A1
公开(公告)日：2015-12-24
申请号：US14716141
申请日：2015-05-19
申请人： Sarepta Therapeutics, Inc.
发明人： Patrick L. Iversen
IPC分类号： C12N15/113
CPC分类号： C12N15/1138 , C12N2310/11 , C12N2310/314 , C12N2310/3233 , C12N2310/3513
摘要： Provided are antisense oligonucleotides and other agents that target and modulate nuclear hormone receptors (NHRs) such as the glucocorticoid receptor (GR), compositions that comprise the same, and methods of use thereof.
摘要翻译：提供靶向和调节核激素受体（NHR）的反义寡核苷酸和其它试剂，例如糖皮质激素受体（GR），包含其的组合物及其使用方法。

86. 发明申请

US20150361428A1 EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY 审中-公开
标题翻译：用于治疗肌肉晶状体的脱皮组合物
公开(公告)号：US20150361428A1
公开(公告)日：2015-12-17
申请号：US14743856
申请日：2015-06-18
申请人： Sarepta Therapeutics, Inc.
发明人： Richard K. Bestwick , Diane Elizabeth Frank
IPC分类号： C12N15/113
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/31 , C12N2310/314 , C12N2310/3181 , C12N2310/32 , C12N2310/321 , C12N2310/3233 , C12N2310/3341 , C12N2310/346 , C12N2310/351 , C12N2310/3513 , C12N2320/33
摘要： Antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon 53 skipping are described.
摘要翻译：描述了能够结合人肌营养不良蛋白基因中选定的靶位点以引发外显子53跳跃的反义分子。

87. 发明申请

US20150184165A1 IMMUNOSUPPRESSION COMPOUND AND TREATMENT METHOD 审中-公开
公开(公告)号：US20150184165A1
公开(公告)日：2015-07-02
申请号：US14581485
申请日：2014-12-23
申请人： SAREPTA THERAPEUTICS, INC.
发明人： Dan V. Mourich , Patrick L. Iversen , Dwight D. Weller
IPC分类号： C12N15/113
CPC分类号： C12N15/1138 , C12N15/113 , C12N2310/11 , C12N2310/314 , C12N2310/3233 , C12N2310/3513
摘要： A method and compound for suppressing an immune response in a mammalian subject, for the treatment or prevention of an autoimmune condition or transplantation rejection are disclosed. The compound is an antisense oligonucleotide analog compound having a targeting sequence complementary to a preprocessed CTLA-4 mRNA region identified by SEQ ID NO: 22 in SEQ ID NO: 1, spanning the splice junction between intron 1 and exon 2 of the preprocessed mRNA of the subject. The compound is effective, when administered to a subject, to form within host cells, a heteroduplex structure (i) composed of the preprocessed CTLA-4 mRNA and the oligonucleotide compound, (ii) characterized by a Tm of dissociation of at least 45° C., and (iii) resulting in an increased ratio of processed mRNA encoding ligand-independent CTLA-4 to processed mRNA encoding full-length CTLA-4.

88. 发明申请

US20150152415A1 MULTIPLE EXON SKIPPING COMPOSITIONS FOR DMD 审中-公开
公开(公告)号：US20150152415A1
公开(公告)日：2015-06-04
申请号：US14523610
申请日：2014-10-24
申请人： Sarepta Therapeutics, Inc.
发明人： Peter SAZANI , Ryszard KOLE
IPC分类号： C12N15/113
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.

89. 发明申请

US20150141321A1 COMPOUND AND METHOD FOR TREATING MYOTONIC DYSTROPHY 审中-公开
标题翻译：用于处理微电子显微镜的化合物和方法
公开(公告)号：US20150141321A1
公开(公告)日：2015-05-21
申请号：US14464561
申请日：2014-08-20
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。

90. 发明授权

US08933216B2 Immunosuppression compound and treatment method 有权
标题翻译：免疫抑制化合物和治疗方法
公开(公告)号：US08933216B2
公开(公告)日：2015-01-13
申请号：US13940098
申请日：2013-07-11
申请人： Sarepta Therapeutics, Inc.
发明人： Dan V. Mourich , Patrick L. Iversen , Dwight D. Weller
IPC分类号： C07H21/02 , C07H21/04
CPC分类号： C12N15/1138 , C12N15/113 , C12N2310/11 , C12N2310/314 , C12N2310/3233 , C12N2310/3513
摘要： A method and compound for suppressing an immune response in a mammalian subject, for the treatment or prevention of an autoimmune condition or transplantation rejection are disclosed. The compound is an antisense oligonucleotide analog compound having a targeting sequence complementary to a preprocessed CTLA-4 mRNA region identified by SEQ ID NO: 22 in SEQ ID NO: 1, spanning the splice junction between intron 1 and exon 2 of the preprocessed mRNA of the subject. The compound is effective, when administered to a subject, to form within host cells, a heteroduplex structure (i) composed of the preprocessed CTLA-4 mRNA and the oligonucleotide compound, (ii) characterized by a Tm of dissociation of at least 45° C., and (iii) resulting in an increased ratio of processed mRNA encoding ligand-independent CTLA-4 to processed mRNA encoding full-length CTLA-4.
摘要翻译：公开了用于抑制哺乳动物受试者的免疫应答的方法和化合物，用于治疗或预防自身免疫病症或移植排斥反应。该化合物是具有与SEQ ID NO：1中SEQ ID NO：22鉴定的预处理的CTLA-4mRNA区域互补的靶序列的反义寡核苷酸类似物化合物，其跨越了预处理mRNA的内含子1和外显子2之间的剪接连接主题。该化合物在给受试者施用时在宿主细胞内形成，由预处理的CTLA-4mRNA和寡核苷酸化合物组成的异源双链体结构（i），（ii）特征在于解离的Tm至少为45° （iii）导致编码与配体无关的CTLA-4的加工的mRNA与编码全长CTLA-4的加工的mRNA的比例增加。

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