公开(公告)号：WO2016183009A2

公开(公告)日：2016-11-17

申请号：PCT/US2016/031471

申请日：2016-05-09

Applicant: DICERNA PHARMACEUTICALS, INC.

Inventor： BROWN, Bob, D. ,  DUDEK, Henryk, T.

IPC: A61K48/00 ,  C07H21/02

CPC classification number: C07H21/02 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/344 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2310/533 ,  C12N2310/321 ,  C12N2310/3521

Abstract: This invention relates to compounds, compositions, and methods useful for reducing AT3 target RNA and protein levels via use of dsRNAs, e.g ., Dicer substrate siRNA (DsiRNA) agents.

Abstract translation: 本发明涉及可用于通过使用dsRNA例如Dicer底物siRNA（DsiRNA）试剂降低AT3靶RNA和蛋白质水平的化合物，组合物和方法。

公开(公告)号：WO2016164896A2

公开(公告)日：2016-10-13

申请号：PCT/US2016/026928

申请日：2016-04-11

Applicant: IONIS PHARMACEUTICALS, INC. ,  THE JOHNS HOPKINS UNIVERSITY

Inventor： RIGO, Frank ,  BENNETT, C., Frank ,  D'YDEWALLE, Constantin, Van Outryve ,  SUMNER, Charlotte, J.

IPC: A61K48/00 ,  C07H21/02

CPC classification number: C07H21/02 ,  C12N15/113 ,  C12N2310/113 ,  C12N2310/315 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2310/321 ,  C12N2310/3525

Abstract: Certain embodiments are directed to methods and compounds for inhibiting SMN-NAT, the natural antisense transcript of SMN. Such methods and compounds are useful for increasing expression of SMN in cells and animals

Abstract translation: 某些实施方案涉及用于抑制SMN的天然反义转录物SMN-NAT的方法和化合物。 这样的方法和化合物可用于增加细胞和动物中SMN的表达

公开(公告)号：WO2016096938A1

公开(公告)日：2016-06-23

申请号：PCT/EP2015/079915

申请日：2015-12-16

Applicant: ROCHE INNOVATION CENTER COPENHAGEN A/S

Inventor： ALBAEK, Nanna ,  GUBLER, Marcel ,  HAGEDORN, Peter ,  HANSEN, Henrik, Frydenlund ,  KOCH, Troels ,  MOISAN, Annie ,  RAVN, Jacob ,  ROSENBOHM, Christoph ,  SEWING, Sabine

IPC: C12N15/11 ,  C12N15/113

CPC classification number: C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2320/53 ,  C12N2330/31 ,  G01N33/5014 ,  G01N33/5088

Abstract: The invention relates to a method of identifying stereodefined phosphrothioate oligonucleotide variants with reduced toxicity by creating and screening libraries of stereodefined chiral phosphorothioate variants for compounds with reduced toxicity, either in vitro or in vivo.

Abstract translation: 本发明涉及通过在体外或体内产生和筛选具有降低的毒性的化合物的立体定向手性硫代磷酸酯变体文库来鉴定具有降低的毒性的立体定向磷酸硫酸酯寡核苷酸变体的方法。

公开(公告)号：WO2016022753A1

公开(公告)日：2016-02-11

申请号：PCT/US2015/043926

申请日：2015-08-06

Applicant: REGULUS THERAPEUTICS INC.

Inventor： BHAT, Balkrishen ,  GIBSON, Neil W. ,  MACKENNA, Diedre ,  WAGNER, Brandee ,  BARTEL, David

IPC: C12N15/113 ,  A61K31/712 ,  C07H21/00

CPC classification number: C12N15/113 ,  A61K31/7088 ,  A61K31/713 ,  A61K45/06 ,  A61K47/549 ,  C12N2310/113 ,  C12N2310/141 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2310/51 ,  C12N2320/30

Abstract: Described herein are compounds comprising modified oligonucleotides that are complementary to miR-103 and/or miR-107 and methods of treating diseases and disorders using the compounds.

Abstract translation: 本文描述了包含与miR-103和/或miR-107互补的修饰的寡核苷酸的化合物和使用该化合物治疗疾病和病症的方法。

公开(公告)号：WO2014059356A3

公开(公告)日：2015-07-16

申请号：PCT/US2013064666

申请日：2013-10-11

Applicant: ISIS PHARMACEUTICALS INC

Inventor： OESTERGAARD MICHAEL ,  SETH PUNIT P ,  SWAYZE ERIC E

IPC: A61K31/7125

CPC classification number: C07H21/02 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/3125 ,  C12N2310/313 ,  C12N2310/315 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/335 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2320/34 ,  C12N2310/321 ,  C12N2310/3525 ,  C12N2310/322 ,  C12N2310/3533

Abstract: The present invention provides oligomeric compounds. 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, activity, or expression of the target nucleic acid in a cell. In certain embodiments, hybridization results in selective modulation of the amount, activity, or expression of a target Huntingtin gene or Huntingtin transcript in a cell.

Abstract translation: 本发明提供低聚化合物。 某些这样的寡聚化合物可用于与互补核酸杂交，包括但不限于细胞中的核酸。 在某些实施方案中，杂交导致细胞中靶核酸的量，活性或表达的调节。 在某些实施方案中，杂交导致选择性调节细胞中靶标Huntingtin基因或Huntingtin转录物的量，活性或表达。

公开(公告)号：WO2015051318A1

公开(公告)日：2015-04-09

申请号：PCT/US2014/059160

申请日：2014-10-03

Applicant: ALNYLAM PHARMACEUTICALS, INC. ,  ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI

Inventor： BETTENCOURT, Brian ,  FITZGERALD, Kevin ,  QUERBES, William ,  DESNICK, Robert, J. ,  YASUDA, Makiko

IPC: C12N15/113 ,  A61K31/7088 ,  A61K31/713 ,  A61P3/00

CPC classification number: C12N15/1137 ,  A61K9/0019 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/351 ,  C12Q1/6876 ,  C12Y203/01037 ,  C12N2310/3521 ,  C12N2310/322 ,  C12N2310/3533

Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ALAS1 gene, and methods of using such dsRNA compositions to alter (e.g., inhibit) expression of ALAS1.

Abstract translation: 本发明涉及靶向ALAS1基因的双链核糖核酸（dsRNA）组合物，以及使用这种dsRNA组合物改变（例如抑制）ALAS1表达的方法。

公开(公告)号：WO2012128785A1

公开(公告)日：2012-09-27

申请号：PCT/US2011/051042

申请日：2011-09-09

Applicant: CITY OF HOPE ,  YU, Hua ,  KORTYLEWSKI, Marcin ,  JOVE, Richard ,  SWIDERSKI, Piotr, Marek ,  ROSSI, John, J.

Inventor： YU, Hua ,  KORTYLEWSKI, Marcin ,  JOVE, Richard ,  SWIDERSKI, Piotr, Marek ,  ROSSI, John, J.

IPC: C12N15/11 ,  C07H21/04

CPC classification number: A61K31/713 ,  C12N15/111 ,  C12N15/113 ,  C12N15/117 ,  C12N2310/14 ,  C12N2310/17 ,  C12N2310/315 ,  C12N2310/3183 ,  C12N2310/345 ,  C12N2310/3519 ,  C12N2320/31

Abstract: The disclosure relates to methods and compositions for improving immune function and treating diseases, including cancer, infectious diseases and autoimmune diseases. More particularly, the disclosure relates to multifunctional molecules that are capable of being delivered to cells of interest for blocking STAT3 for the treatment of diseases and the improvement of immune function.

Abstract translation: 本公开涉及用于改善免疫功能并治疗疾病的方法和组合物，包括癌症，感染性疾病和自身免疫性疾病。 更具体地，本公开涉及能够被递送到感兴趣的细胞以阻断STAT3以治疗疾病和改善免疫功能的多功能分子。

公开(公告)号：WO2009055076A2

公开(公告)日：2009-04-30

申请号：PCT/US2008/012220

申请日：2008-10-27

Applicant: DYNAVAX TECHNOLOGIES CORPORATION ,  BARRAT, Franck ,  COFFMAN, Robert, L. ,  MATRAY, Tracy ,  GUIDUCCI, Cristiana

Inventor： BARRAT, Franck ,  COFFMAN, Robert, L. ,  MATRAY, Tracy ,  GUIDUCCI, Cristiana

IPC: C12N15/11

CPC classification number: C12N15/117 ,  A61K31/711 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  A61K31/713 ,  A61K45/06 ,  C12N2310/17 ,  C12N2310/314 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/331 ,  C12N2310/3341 ,  C12N2310/336 ,  C12N2310/345 ,  C12N2310/3521

Abstract: The application relates to the use of immunoregulatory polynucleotides and/or immunoregulatory compounds in combination with other therapeutic agents. The application further relates to immunoregulatory polynucleotides and/or immunoregulatory compounds comprising a modified immunoregulatory sequence. It also relates to the administration of the immunoregulatory polynucleotides and/or immunoregulatory compounds comprising a modified immunoregulatory sequence to regulate an immune response.

Abstract translation: 本申请涉及免疫调节多核苷酸和/或免疫调节化合物与其它治疗剂组合的用途。 本申请还涉及包含修饰的免疫调节序列的免疫调节多核苷酸和/或免疫调节化合物。 它还涉及免疫调节多核苷酸和/或免疫调节化合物的施用，其包含修饰的免疫调节序列以调节免疫应答。

公开(公告)号：WO2007027775A3

公开(公告)日：2007-08-02

申请号：PCT/US2006033866

申请日：2006-08-29

Applicant: ISIS PHARMACEUTICALS INC ,  ESAU CHRISTINE ,  BHANOT SANJAY

Inventor： ESAU CHRISTINE ,  BHANOT SANJAY

IPC: A61K31/711 ,  C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/11 ,  C12N2310/113 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/345 ,  C12N2310/346 ,  C12N2310/3525

Abstract: Methods are provided for the treatment of cardiovascular or metabolic diseases characterized by elevated serum total cholesterol, elevated serum LDL-cholesterol, or elevated serum triglycerides, through the administration of an oligomeric compound which modulates the levels or activity of miR-122a. Further provided are methods for reducing hepatic steatosis or liver tissue triglyceride accumulation through the administration of an oligomeric compound which modulates the levels or activity of miR-122a. Such methods employ oligomeric compounds which hybridize with or sterically interfere with nucleic acid molecules comprising or encoding miR-122a. Such oligomeric compounds may include one or more modifications thereon, which may improve the activity, stability, or nuclease resistance of the oligomeric compound. These modified oligomeric compounds are used as single compounds or in compositions, including pharmaceutical compositions, to modulate or mimic the targeted nucleic acid comprising or encoding miR-122a.

Abstract translation: 提供了通过施用调节miR-122a的水平或活性的寡聚化合物来治疗以血清总胆固醇升高，血清LDL-胆固醇升高或血清甘油三酯升高为特征的心血管或代谢疾病的方法。 还提供了通过施用调节miR-122a的水平或活性的寡聚化合物来减少肝脂肪变性或肝组织甘油三酯积聚的方法。 这样的方法使用与包含miR-122a或编码miR-122a的核酸分子杂交或空间干扰的寡聚化合物。 这样的低聚化合物可以包括其上的一个或多个修饰，其可以改善低聚化合物的活性，稳定性或核酸酶抗性。 这些改性的低聚化合物用作单一化合物或组合物，包括药物组合物，以调节或模拟包含或编码miR-122a的靶向核酸。

公开(公告)号：WO2006135434A3

公开(公告)日：2007-05-10

申请号：PCT/US2005037731

申请日：2005-10-20

Applicant: COLEY PHARM GROUP INC ,  COLEY PHARM GMBH ,  KRIEG ARTHUR M ,  SAMULOWITZ ULRIKE ,  VOLLMER JORG ,  UHLMANN EUGEN

Inventor： KRIEG ARTHUR M ,  SAMULOWITZ ULRIKE ,  VOLLMER JORG ,  UHLMANN EUGEN

IPC: A61K9/00 ,  A61K31/7125 ,  C12N15/117

CPC classification number: C12N15/117 ,  A61K9/0043 ,  A61K9/0073 ,  A61K39/39 ,  A61K2039/55561 ,  C07H21/02 ,  C12N2310/17 ,  C12N2310/315 ,  C12N2310/345

Abstract: The invention relates to specific C-Class semi-soft CpG immunostimulatory oligonucleotides that are useful for stimulating an immune response. In particular the oligonucleotides are useful for treating allergy, such as allergic rhinitis and asthma, cancer and infectious disease, such as hepatitis B and hepatitis C.

Abstract translation: 本发明涉及可用于刺激免疫应答的特异性C-class半软CpG免疫刺激性寡核苷酸。 特别地，寡核苷酸可用于治疗过敏，例如过敏性鼻炎和哮喘，癌症和感染性疾病，例如乙型肝炎和丙型肝炎。