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121. 发明申请

US20180273954A1 INDUCED EXON INCLUSION IN SPINAL MUSCLE ATROPHY 审中-公开
公开(公告)号：US20180273954A1
公开(公告)日：2018-09-27
申请号：US15921518
申请日：2018-03-14
申请人： Sarepta Therapeutics, Inc.
发明人： Peter Linsley , Brian James Leppert
IPC分类号： C12N15/113 , C12N15/11
摘要： The invention relates to the use of an antisense compound for inducing exon inclusion as a treatment for Spinal Muscle Atrophy (SMA). More particularly it relates to inducing inclusion of exon 7 to restore levels of Survival Motor Neuron (SMN) protein encoded by the Survival Motor Neuron (SMN) gene.

122. 发明申请

US20180223278A1 Functionally-Modified Oligonucleotides And Subunits Thereof 审中-公开
公开(公告)号：US20180223278A1
公开(公告)日：2018-08-09
申请号：US15700718
申请日：2017-09-11
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 , C07F9/6558 , C07F9/6561 , C07F9/6584 , C07H21/00
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.

123. 发明申请

US20180214567A1 Peptide Oligonucleotide Conjugates 审中-公开
公开(公告)号：US20180214567A1
公开(公告)日：2018-08-02
申请号：US15574734
申请日：2016-05-19
申请人： Sarepta Therapeutics, Inc.
发明人： Gunnar J. Hanson , Ming Zhou
IPC分类号： A61K47/64 , C07K7/06 , C07K7/08 , A61K47/18
摘要： Provided herein are oligonucleotides, peptides, and peptide-oligonucleotide-conjugates. Also provided herein are methods of treating a muscle disease, a viral infection, or a bacterial infection in a subject in need thereof, comprising administering to the subject oligonucleotides, peptides, and peptide-oligonucleotide-conjugates described herein.

124. 发明申请

US20180177814A1 EXON SKIPPING OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY 审中-公开
公开(公告)号：US20180177814A1
公开(公告)日：2018-06-28
申请号：US15841261
申请日：2017-12-13
申请人： Sarepta Therapeutics, Inc.
发明人： Marco A. PASSINI , Gunnar J. HANSON
IPC分类号： A61K31/712 , A61K31/7125 , C12N15/113
摘要： Antisense oligomer conjugates complementary to a selected target site in the human dystrophin gene to induce exon 51 skipping are described.

125. 发明申请

US20180161359A1 METHODS AND COMPOUNDS FOR TREATMENT OF LYMPHOCYTE-RELATED DISEASES AND CONDITIONS 审中-公开
公开(公告)号：US20180161359A1
公开(公告)日：2018-06-14
申请号：US15579497
申请日：2016-06-03
申请人： Sarepta Therapeutics, Inc.
发明人： Dan V. Mourich , Gunnar J. Hanson , Frederick Joseph Schnell , Johannes Christian Dworzak
IPC分类号： A61K31/7125 , A61K31/675 , C12N15/113
CPC分类号： A61K31/7125 , A61K31/675 , A61P35/00 , A61P37/06 , C12N15/113 , C12N2310/11 , C12N2310/3233
摘要： Methods for treatment of lymphocyte-related diseases and conditions, such as cancer and automimmune diseases, are provided. The methods comprise administration of an effective amount of an oligomer to a patient in need thereof, wherein the oligomer comprises, inter alia, at least one intersubunit linkage having the following structure: wherein R1, L1, X, Y and Z are as defined herein.

126. 发明申请

US20170198287A1 OLIGONUCLEOTIDE ANALOGUES HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS 审中-公开
公开(公告)号：US20170198287A1
公开(公告)日：2017-07-13
申请号：US15247584
申请日：2016-08-25
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： C12N15/113
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.

127. 发明授权

US09534220B2 Antisense antibacterial method and compound 有权
标题翻译：反义抗菌法及化合物
公开(公告)号：US09534220B2
公开(公告)日：2017-01-03
申请号：US13680790
申请日：2012-11-19
申请人： Sarepta Therapeutics, Inc.
发明人： Bruce L. Geller , Jesse D. Deere , Patrick L. Iversen
IPC分类号： C12N15/113 , C07F9/6558 , C07F9/6561
CPC分类号： C12N15/1137 , C07F9/65583 , C07F9/65613 , C12N15/113 , C12N2310/11 , C12N2310/3145 , C12N2310/3233
摘要： A method and antisense compound for inhibiting the growth of pathogenic bacterial cells are disclosed. The compound contains no more than 12 nucleotide bases and has a targeting nucleic acid sequence of no fewer than 10 bases in length that is complementary to a target sequence containing or within 10 bases, in a downstream direction, of the translational start codon of a bacterial mRNA that encodes a bacterial protein essential for bacterial replication. The compound binds to a target mRNA with a Tm of between 50° to 60° C. The relatively short antisense compounds are substantially more active than conventional antisense compounds having a targeting base sequence of 15 or more bases.
摘要翻译：公开了一种用于抑制病原菌细胞生长的方法和反义化合物。该化合物含有不超过12个核苷酸碱基，并且具有长度不小于10个碱基的靶向核酸序列，该靶向核酸序列与在细菌的翻译起始密码子的下游方向上含有或在10个碱基内的靶序列互补编码细菌复制必需的细菌蛋白质的mRNA。该化合物以Tm在50℃至60℃之间的靶mRNA结合。相对较短的反义化合物比具有15个或更多碱基的靶向碱基序列的常规反义化合物显着更具活性。

128. 发明授权

US09487786B2 Immunosuppression compound and treatment method 有权
公开(公告)号：US09487786B2
公开(公告)日：2016-11-08
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： C12N15/11 , A61K48/00 , C07H21/02 , C07H21/04 , 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.

129. 发明授权

US09347063B2 Oligonucleotide analog and method for treating flavivirus infections 有权
标题翻译：寡核苷酸类似物和治疗黄病毒感染的方法
公开(公告)号：US09347063B2
公开(公告)日：2016-05-24
申请号：US14103603
申请日：2013-12-11
申请人： Sarepta Therapeutics, Inc.
发明人： Patrick L. Iversen , David A. Stein
IPC分类号： C07H21/04 , C12N15/113
CPC分类号： C12N15/1131 , C12N2310/11 , C12N2310/314 , C12N2310/3145 , C12N2310/3233
摘要： A method of inhibiting replication of a flavivirus in animal cells, and an oligonucleotide compound for use in the method are disclosed. The oligonucleotide analog (i) has a nuclease-resistant backbone, (ii) is capable of uptake by the cells, (iii) contains between 8-40 nucleotide bases, and (iv) has a sequence of at least 8 bases complementary to a region of the virus' positive strand RNA genome that includes at least a portion of SEQ ID NOS:1-4. Exposure of cells infected with a flavivirus to the analog is effective to form within the cells, a heteroduplex structure composed of the virus ssRNA and the oligonucleotide, characterized by a Tm of dissociation of at least 45° C., and having disrupted base pairing between the virus's 5′ and 3′ cyclization sequences.
摘要翻译：公开了抑制黄病毒在动物细胞中的复制的方法和用于该方法的寡核苷酸化合物。寡核苷酸类似物（i）具有核酸酶抗性主链，（ii）能够被细胞吸收，（iii）含有8-40个核苷酸碱基，和（iv）具有至少8个碱基互补的序列包含SEQ ID NO：1-4的至少一部分的病毒“正链RNA”基因组的区域。用黄病毒感染的细胞暴露于类似物是有效的在细胞内形成，由病毒ssRNA和寡核苷酸组成的异源双链结构，其特征在于解离的Tm至少为45℃，并且具有中间的碱基配对病毒的5'和3'环化序列。

130. 发明申请

US20160102307A1 OLIGONUCLEOTIDE ANALOGUES TARGETING HUMAN LMNA 审中-公开
标题翻译：命名人体LMNA的寡核苷酸模拟物
公开(公告)号：US20160102307A1
公开(公告)日：2016-04-14
申请号：US14709355
申请日：2015-05-11
申请人： Sarepta Therapeutics, Inc.
发明人： Ryszard Kole , Richard Keith Bestwick
IPC分类号： C12N15/113 , A61K31/713 , A61K47/48
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。

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