公开(公告)号：EP4306116A3

公开(公告)日：2024-04-17

申请号：EP23184286.5

申请日：2019-10-12

申请人： Peking University ,  EdiGene Therapeutics (Beijing) Inc.

发明人： WEI, Wensheng ,  QU, Liang ,  YI, Zongyi ,  ZHU, Shiyou ,  WANG, Chunhui ,  CAO, Zhongzheng ,  ZHOU, Zhuo ,  YUAN, Pengfei

IPC分类号： C12N15/113 ,  C12N15/11 ,  C12N15/10 ,  A61K31/712

CPC分类号： C12N15/113 ,  A61K31/712 ,  C12N15/102 ,  C12N15/111 ,  C12N2310/2020170501 ,  A61K31/7105 ,  A61K31/7125

摘要： The present application provides methods for editing RNA by introducing a deaminase-recruiting RNA in a host cell for deamination of an adenosine in a target RNA. The present application further provides deaminase-recruiting RNAs used in the RNA editing methods and compositions comprising the same.

公开(公告)号：EP3449926A1

公开(公告)日：2019-03-06

申请号：EP18185820.0

申请日：2010-06-17

申请人： Biogen MA Inc. ,  COLD SPRING HARBOR LABORATORY

发明人： BENNETT, C. Frank ,  HUNG, Gene ,  RIGO, Frank ,  KRAINER, Adrian R. ,  HUA, Yimin ,  PASSINI, Marco A. ,  SHIHABUDDIN, Lamya ,  CHENG, Seng H. ,  KLINGER, Katherine W.

IPC分类号： A61K31/7088 ,  C12N15/113

CPC分类号： A61K31/7088 ,  A61K31/712 ,  C12N15/113 ,  C12N2310/11 ,  C12N2320/33

摘要： Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders, including spinal muscular atrophy.

公开(公告)号：EP3384028A1

公开(公告)日：2018-10-10

申请号：EP16869430.5

申请日：2016-11-30

申请人： The University of British Columbia

发明人： WANG, Yuzhuo ,  CHOI, Stephen Yiu Chuen

IPC分类号： C12N15/113 ,  A61K31/7088 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  A61P35/00 ,  C07H21/04

CPC分类号： A61K31/7088 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  C07H21/04 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2320/31

摘要： Provided herein are compositions, method and uses for modulating MCT4 activity or for the treatment of cancer. The compositions comprise antisense oligonucleotides (ASO) for administration to a cancer cell, wherein the cancer cell may be characterized by elevated expression of MCT4. The cancer may be selected from one or more of: prostate cancer; renal cell carcinoma; breast cancer; cervical cancer; liver cancer; bladder cancer; and small cell lung cancer pr. The prostate cancer may be castration-resistant prostate cancer (CRPC).

公开(公告)号：EP2841579B1

公开(公告)日：2018-10-10

申请号：EP13720699.1

申请日：2013-04-24

申请人： Regulus Therapeutics Inc.

发明人： BHAT, Balkrishen ,  MARCUSSON, Eric

IPC分类号： C12N15/113 ,  A61K31/712 ,  A61P35/00 ,  A61P13/12 ,  A61P43/00 ,  A61K45/06

CPC分类号： C12N15/113 ,  A61K31/712 ,  A61K31/7125 ,  A61K45/06 ,  C12N15/1135 ,  C12N2310/11 ,  C12N2310/113 ,  C12N2310/141 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/323 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/343 ,  C12N2310/346 ,  C12N2320/30 ,  C12N2320/31 ,  C12N2320/51 ,  C12N2310/3525

摘要： Described herein are compositions and methods for the inhibition of miR-21 activity. The compositions have certain nucleoside modification patterns that yield potent inhibitors of miR-21 activity. The compositions may be used to inhibit miR-21, and also to treat diseases associated with abnormal expression of miR-21, such as fibrosis and cancer.

公开(公告)号：EP3365444A1

公开(公告)日：2018-08-29

申请号：EP16785159.1

申请日：2016-10-21

申请人： spRNA GmbH

发明人： PASCOLO, Steve

IPC分类号： C12N15/11

CPC分类号： A61K47/55 ,  A61K9/08 ,  A61K31/7115 ,  A61K31/712 ,  A61K47/02 ,  A61K47/12 ,  A61K47/549 ,  C12N15/117 ,  C12N2310/17 ,  C12N2310/351 ,  C12N2320/31 ,  C12N2310/335 ,  C12N2310/3533

摘要： A composition including an immunostimulating RNA molecule in a dication containing solution wherein the RNA includes a chemical modification which is toxic to cancer or tumor cells. Pharmaceutical compositions incorporate the immunostimulating RNA with tumor cytotoxicity. Methods for treating cancer and tumors use the solution of the immunostimulating RNA with tumor cytotoxicity.

公开(公告)号：EP2951305B1

公开(公告)日：2018-08-15

申请号：EP14704307.9

申请日：2014-01-30

申请人： F. Hoffmann-La Roche AG

发明人： ALBÆK, Nanna ,  HANSEN, Henrik Frydenlund ,  KAMMLER, Susanne ,  RAVN, Jacob ,  ØRUM, Henrik ,  TURNER, Mark ,  KRAMPERT, Monika ,  HADWIGER, Philipp ,  OTTOSEN, Søren ,  LINDOW, Morten

IPC分类号： C12N15/113 ,  A61K31/713 ,  A61K47/55

CPC分类号： C12N15/113 ,  A61K31/712 ,  A61K31/713 ,  A61K47/55 ,  A61K47/64 ,  C12N15/1131 ,  C12N15/1137 ,  C12N2310/11 ,  C12N2310/113 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/341 ,  C12N2310/351 ,  C12N2310/3513 ,  C12N2320/30 ,  C12N2330/30

摘要： The invention provides LNA therapeutics oligonucleotide carbohydrate conjugates with considerably enhanced potency, extended therapeutic index and reduced toxicity.

公开(公告)号：EP3357499A1

公开(公告)日：2018-08-08

申请号：EP17001941.8

申请日：2013-12-18

申请人： Sirna Therapeutics, Inc.

发明人： Zewge, Daniel ,  Copeland, Gregory, T. ,  LI, Zhen ,  Armstrong, Joseph, D., III

IPC分类号： A61K31/712 ,  C07H21/02

CPC分类号： C12N15/113 ,  A61K31/712 ,  C07H21/02 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/332 ,  C12N2330/30

摘要： This invention relates to the post-synthetic chemical modifications of RNA at the 2' - position on the ribose rings via orthogonal chemistry involving amidation reactions plus metal catalyzed alkyne-azide cycloaddition (click) reactions. RNA interference (RNAi) is an evolutionarily conserved cellular mechanism of post-transcriptional gene silencing found in fungi, plants and animals that uses small RNA molecules to inhibit gene expression in a sequence-specific manner. The RNAi machinery can be harnessed to destruct any mRNA of a known sequence.

公开(公告)号：EP3351632A1

公开(公告)日：2018-07-25

申请号：EP16845971.7

申请日：2016-09-16

申请人： The University Of Tokyo ,  Genedesign, Inc.

发明人： IBA, Hideo ,  HARAGUCHI, Takeshi ,  NANKAI, Hirokazu ,  SATO, Hideaki

IPC分类号： C12N15/113 ,  A61K31/712 ,  A61K31/713 ,  A61K48/00 ,  A61P31/12 ,  A61P35/00

CPC分类号： C12N15/113 ,  A61K31/712 ,  A61K31/713 ,  A61K48/00 ,  C12N2310/141 ,  C12N2310/3231

摘要： The present invention pertains to the improvement of a miRNA inhibitor (a synthesized Tough Decoy (S-TuD)). The present invention provides a miRNA inhibitory complex including RNA or an analog thereof, wherein the RNA inhibitory complex includes at least one double-stranded structure and a miRNA binding sequence, each of two strands of the miRNA binding sequence being bound to two strands of at least one end of the double-stranded structure, and the miRNA inhibitory complex further includes at least one crosslinked nucleic acid.

公开(公告)号：EP3350328A1

公开(公告)日：2018-07-25

申请号：EP16775903.4

申请日：2016-09-12

申请人： Alnylam Pharmaceuticals, Inc.

发明人： HINKLE, Gregory

IPC分类号： C12N15/113

CPC分类号： C12N15/1137 ,  A61K31/7008 ,  A61K31/712 ,  A61K31/7125 ,  C12N15/113 ,  C12N2310/111 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/351

摘要： The invention relates to polynucleotide agents targeting a Patatin-Like Phospholipase Domain Containing 3 (PNPLA3) gene, and methods of using such polynucleotide agents to inhibit expression of a PNPLA3 gene and methods of treating subjects having Nonalcoholic Fatty Liver Disease (NAFLD) and/or a PNPLA3-associated disorder.

公开(公告)号：EP3279325A1

公开(公告)日：2018-02-07

申请号：EP16772732.0

申请日：2016-03-28

申请人： Nissan Chemical Industries, Ltd. ,  The University of Tokyo

发明人： YOSHIMOTO, Keitaro ,  KIMURA, Keiko ,  FURUSHO, Hitoshi

IPC分类号： C12N15/115 ,  A61K31/7088 ,  A61K31/711 ,  A61K31/712 ,  A61K31/7125 ,  A61K48/00 ,  A61P3/10 ,  A61P9/00 ,  A61P19/02 ,  A61P29/00 ,  A61P35/00 ,  A61P43/00 ,  C12N15/09 ,  C12Q1/02 ,  C12Q1/68 ,  G01N33/50

CPC分类号： C12N15/115 ,  A61K31/7088 ,  A61K31/711 ,  A61K31/712 ,  A61K31/7125 ,  A61K48/00 ,  C12N15/09 ,  C12N15/1048 ,  C12N2310/16 ,  C12N2310/3517 ,  C12Q1/02 ,  C12Q1/68 ,  G01N33/50 ,  G01N33/5308 ,  G01N33/588 ,  G01N33/74 ,  G01N2333/71 ,  G01N2800/7014

摘要： [Problem] To provide a novel nucleic acid aptamer for a vascular endothelial growth factor receptor, said nucleic acid aptamer being useful for the diagnosis and treatment of various diseases associated with VEGFs that can regulate angiogenesis and receptors for the VEGFs, e.g., tumor angiogenesis, diabetic retina and chronic rheumatoid arthritis. [Solution] A nucleic acid aptamer characterized by comprising a nucleotide sequence represented by any one of SEQ ID NOs: 1 to 5, and also characterized by being capable of bonding to a human VEGF receptor specifically. In a preferred embodiment of the nucleic acid aptamer, a primer recognition sequence, a fluorescent label, or a biotin molecule, an avidin molecule, a streptavidin molecule or other specific binding tag peptide may be linked to the 5'- or 3'-terminal of the nucleic acid aptamer for the purpose of making it possible to detect the nucleic acid aptamer easily.

摘要翻译： 为了提供用于血管内皮生长因子受体的新型核酸适体，所述核酸适体可用于诊断和治疗与VEGF相关的各种疾病，所述疾病可调节VEGF的血管生成和受体，例如肿瘤血管生成， 糖尿病视网膜和慢性风湿性关节炎。 [解决方案]核酸适体，其特征在于，包含序列号1〜5中任一项所示的碱基序列，其特征还在于能够与人VEGF受体特异性结合。 在核酸适体的优选实施方案中，可将引物识别序列，荧光标记或生物素分子，抗生物素蛋白分子，链霉抗生物素蛋白分子或其他特异性结合标签肽连接至5'-或3'-末端 为了能够容易地检测核酸适体，可以使用核酸适体。