公开(公告)号：WO2019145386A1

公开(公告)日：2019-08-01

申请号：PCT/EP2019/051684

申请日：2019-01-24

申请人： ROCHE INNOVATION CENTER COPENHAGEN A/S

发明人： HAGEDORN, Peter ,  PEDERSEN, Lykke

IPC分类号： C12N15/113

CPC分类号： C12N15/1137 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/3521 ,  C12N2310/3525 ,  C12N2310/3533

摘要： The present invention relates to antisense oligonucleotides that are capable of reducing expression of CSNK1D in a target cell. The oligonucleotides hybridize to CSNK1D pre-mRNA. The present invention further relates to conjugates of the oligonucleotide and pharmaceutical compositions and methods for treatment or alleviation of cancers, such as breast cancer,liver cancer, lung cancer, cervical cancer, melanoma, glioblastoma, medulloblastoma, pancreatic cancer, prostate cancer, renal cancer, bladder cancer, endometrial cancer, stomach cancer, colon cancer, testis cancer, urothelial cancer, or a cancer that metastasizes to the brain, lymph nodes, lung, or bone.

公开(公告)号：WO2018165564A1

公开(公告)日：2018-09-13

申请号：PCT/US2018/021768

申请日：2018-03-09

申请人： IONIS PHARMACEUTICALS, INC.

发明人： OESTERGAARD, Michael ,  SETH, Punit, P.

IPC分类号： C07F9/6561 ,  C12N15/113

CPC分类号： C07F9/6533 ,  C07F9/2408 ,  C07F9/65583 ,  C07F9/6561 ,  C07H21/00 ,  C12N15/1136 ,  C12N2310/11 ,  C12N2310/3145 ,  C12N2310/3231 ,  C12N2310/3233 ,  C12N2310/3341 ,  C12N2310/341

摘要： The present invention provides morpholino modified oligomeric compounds having at least one monomer subunit having Formula III, compounds having Formula I useful for making certain of the morpholino modified oligomeric compounds and methods of using the oligomeric compounds. In certain embodiments, the oligomeric compounds provided herein provide for an improved toxicity profile. 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 of activity or expression of the target nucleic acid in a cell.

公开(公告)号：WO2018118587A1

公开(公告)日：2018-06-28

申请号：PCT/US2017/066115

申请日：2017-12-13

申请人： AGENOVIR CORPORATION

发明人： SLOAN, Derek, D. ,  LEDOUX, Sarah ,  HOUSLEY, Michael, Patrick

IPC分类号： A61K38/46 ,  A61K45/06 ,  C12N9/16 ,  C12N15/11 ,  C12N15/113 ,  C12N15/52

CPC分类号： C12N9/22 ,  A61K9/51 ,  A61K9/513 ,  A61K9/5161 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  A61K38/465 ,  A61K45/06 ,  A61K47/549 ,  C12N15/11 ,  C12N15/113 ,  C12N15/1133 ,  C12N2310/20 ,  C12N2310/3341 ,  C12N2310/321 ,  C12N2310/3521

摘要： Provided are antiviral compositions that use a CRISPR-associated (Cas) endonuclease and a guide RNA that work together to eliminate viral nucleic acid from host cells, e.g., human cells. The guide RNA includes a target sequence complementary to a target sequence within a genome of a virus and at least one modified nucleotide. The Cas endonuclease may be provided as mRNa, and either or both of the mRNA and the guide RNA include a modified or non-naturally occuring nucleotide. Also provided are methods of making and using such compositions.

公开(公告)号：WO2016105516A8

公开(公告)日：2017-07-06

申请号：PCT/US2015000269

申请日：2015-12-23

申请人： CELGENE CORP ,  CELGENE ALPINE INVEST COMPANY II LLC

发明人： SMITH SCOTT ANDREW ,  LI XIAOBIN ,  ROSSITER GUILLERMO ,  MARTIN PHILIPPE L ,  DEWACKER SETH R ,  USISKIN KEITH ,  CLINE GARY ALLAN

IPC分类号： A61K48/00

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/3341 ,  C12N2310/346 ,  C12N2320/35 ,  C12N2310/321 ,  C12N2310/3521

摘要： Described herein are methods of treating inflammatory bowel disease (I BD) in a patient having IBD using SMAD7 antisense oligonucleotides. In one aspect, provided herein is a method for treating or managing inflammatory bowel disease (IBD) in a patient having IBD, wherein the method comprises (a) administering to the patient a SMAD7 antisense-oligonucleotide (SMAD7 AON) during a first treatment period at a first dose; and (b) administering to the patient the SMAD7 antisense-oligonucleotide during a second treatment period at a second dose.

摘要翻译： 本文描述了使用SMAD7反义寡核苷酸治疗患有IBD的患者的炎症性肠病（IBD）的方法。 在一个方面，本文提供了用于治疗或控制患有IBD的患者中的炎症性肠病（IBD）的方法，其中所述方法包括（a）在第一治疗期间向患者施用SMAD7反义寡核苷酸（SMAD7 AON） 以第一剂量; 和（b）在第二次治疗期间以第二剂量向患者施用SMAD7反义寡核苷酸。

公开(公告)号：WO2017100587A1

公开(公告)日：2017-06-15

申请号：PCT/US2016/065846

申请日：2016-12-09

申请人： ALNYLAM PHARMACEUTICALS, INC.

发明人： HINKLE, Gregory

IPC分类号： C12N15/113 ,  A61K31/712 ,  A61P31/06

CPC分类号： A61K31/712 ,  A61K31/7125 ,  A61K45/06 ,  A61K47/549 ,  C12N15/1138 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/321 ,  C12N2310/3521

摘要： The invention relates to polynucleotide agents targeting programmed cell death 1 ligand 1 (PD-L1) gene, and methods of using such polynucleotide agents to inhibit expression of PD-L1 and to treat subjects having a PD-L1-associated disorder.

摘要翻译： 本发明涉及靶向程序性细胞死亡1配体1（PD-L1）基因的多核苷酸试剂，以及使用此类多核苷酸试剂抑制PD-L1表达并治疗患有PD- L1相关的疾病。

公开(公告)号：WO2017004261A1

公开(公告)日：2017-01-05

申请号：PCT/US2016/040191

申请日：2016-06-29

申请人： IONIS PHARMACEUTICALS, INC.

发明人： RAHDAR, Meghdad ,  PRAKASH, Thazha, P. ,  SWAYZE, Eric, E. ,  BENNETT, Frank, C.

IPC分类号： C12N9/22 ,  C12N15/63 ,  A61K31/7088 ,  C07H19/00

CPC分类号： C12N15/11 ,  A61K38/00 ,  A61K48/00 ,  C07H21/00 ,  C12N7/00 ,  C12N9/22 ,  C12N9/96 ,  C12N15/102 ,  C12N15/111 ,  C12N15/907 ,  C12N2310/20 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/344 ,  C12N2310/351 ,  C12N2740/15043 ,  C12N2750/14343 ,  C12N2800/80

摘要： The present disclosure provides compounds comprising modified oligonucleotides for use in CRISPR. In certain embodiments, such modified oligonucleotides provide improved properties of crRNA. In cetain embodiments, such modified oligonucleotides provide improved properties of scrRNA.

摘要翻译： 本公开提供了包含用于CRISPR的修饰的寡核苷酸的化合物。 在某些实施方案中，这样的修饰的寡核苷酸提供了改进的crRNA的性质。 在切割实施方案中，这种修饰的寡核苷酸提供了scrRNA的改进的性质。

公开(公告)号：WO2016141145A1

公开(公告)日：2016-09-09

申请号：PCT/US2016/020610

申请日：2016-03-03

申请人： IONIS PHARMACEUTICALS, INC. ,  BAYLOR COLLEGE OF MEDICINE

发明人： ZOGHBI, Huda, Y. ,  SZTAINBERG, Ezequiel ,  FREIER, Susan, M.

IPC分类号： C12N15/11 ,  A61K31/712 ,  A61K31/7088

CPC分类号： C12N15/113 ,  A61K31/7125 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/322 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2320/32 ,  C12N2310/321 ,  C12N2310/3525

摘要： Disclosed herein are methods for decreasing MECP2 mRNA and protein expression. Such methods are useful to treat, prevent, or ameliorate MECP2 associated disorders and syndromes. Such MECP2 associated disorders include MECP2 duplication syndrome.

摘要翻译： 本文公开了减少MECP2 mRNA和蛋白质表达的方法。 这些方法可用于治疗，预防或改善MECP2相关疾病和综合征。 这样的MECP2相关疾病包括MECP2复制综合征。

公开(公告)号：WO2016118612A2

公开(公告)日：2016-07-28

申请号：PCT/US2016/014108

申请日：2016-01-20

申请人： MIRAGEN THERAPEUTICS, INC.

发明人： DALBY, Christina Marie ,  GALLANT-BEHM, Corrie Lynn ,  JACKSON, Aimee ,  HUTNICK, Kathryn ,  SETO, Anita

CPC分类号： C12N15/113 ,  C12N2310/113 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/3341

摘要： The present invention provides oligonucleotide inhibitors of miR-92 and methods of using said inhibitors for inhibiting the function and/or activity of miR-92 in a subject in need thereof. The present invention also provides methods for evaluating or monitoring the efficacy of a therapeutic for promoting wound healing and selecting a subject for treatment with a therapeutic that modulates miR-92 function and/or activity.

摘要翻译： 本发明提供miR-92的寡核苷酸抑制剂和使用所述抑制剂抑制有需要的受试者中miR-92的功能和/或活性的方法。 本发明还提供了用于评估或监测用于促进伤口愈合的治疗剂的功效的方法，并且选择受试者用用于调节miR-92功能和/或活性的治疗剂进行治疗。

公开(公告)号：WO2016103042A1

公开(公告)日：2016-06-30

申请号：PCT/IB2015/002574

申请日：2015-12-23

申请人： GUANGZHOU RIBOBIO CO., LTD. ,  GUANGZHOU INSTITUTES OF BIOMEDICINE AND HEALTH, CHINESE ACADEMY OF SCIENCES

发明人： YANG, Xiuqun ,  WANG, Qiuyun ,  TORTORELLA, Micky, Daniel ,  ZHANG, Bill, Biliang ,  WANG, Zhe

IPC分类号： C12N15/113 ,  C12N15/11 ,  A61K48/00 ,  A61P29/00 ,  A61P19/02

CPC分类号： C12N15/1137 ,  A61K31/713 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3341 ,  C12Y304/24014 ,  C12Y304/24082 ,  C12N2310/3521 ,  C12N2310/3533 ,  A61K2300/00

摘要： Provided are siRNAs or chemically-modified siRNAs targeted against ADAMTS-5 or ADAM17 for inhibiting the expression of ADAMTS-5 or ADAM17. Use of the siRNAs for treating an ADAMTS-5 or ADAM17 associated disease, such as arthritis and other inflammation-related diseases, by injecting the siRNAs or preparations comprising the siRNAs into an articular cavity of the patient, are also provided.

摘要翻译： 提供靶向针对ADAMTS-5或ADAM17的siRNA或化学修饰的siRNA以抑制ADAMTS-5或ADAM17的表达。 还提供了通过将siRNA或包含siRNA的制剂注射到患者的关节腔中来治疗ADAMTS-5或ADAM17相关疾病（例如关节炎和其它炎症相关疾病）的用途。

公开(公告)号：WO2016069694A3

公开(公告)日：2016-06-23

申请号：PCT/US2015057717

申请日：2015-10-28

申请人： ALNYLAM PHARMACEUTICALS INC

发明人： HINKLE GREGORY

IPC分类号： C12N15/113 ,  A61K31/7088 ,  C07H21/02

CPC分类号： C12N15/113 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2320/35 ,  C12N2310/321 ,  C12N2310/3525 ,  C12N2310/3521

摘要： The invention relates to polynucleotide agents targeting the Serpinc 1 (AT3) gene, and methods of using such polynucleotide agents to inhibit expression of Serpinc 1 and to treat subjects having a bleeding disorder, e.g., a hemophilia.

摘要翻译： 本发明涉及靶向Serpinc1（AT3）基因的多核苷酸试剂，以及使用这种多核苷酸试剂抑制Serpinc1表达并治疗出血性疾病例如血友病的受试者的方法。