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1. 发明申请

WO2014059353A2 OLIGOMERIC COMPOUNDS COMPRISING BICYCLIC NUCLEOSIDES AND USES THEREOF 审中-公开
标题翻译：包含双相核苷酸的低分子化合物及其用途
公开(公告)号：WO2014059353A2
公开(公告)日：2014-04-17
申请号：PCT/US2013/064660
申请日：2013-10-11
申请人： ISIS PHARMACEUTICALS, INC.
发明人： SETH, Punit, P. , FREIER, Susan, M. , MONIA, Brett, P. , SWAYZE, Eric, E.
IPC分类号： A61K48/00 , C07H21/02
CPC分类号： C12N15/113 , C12N15/111 , C12N2310/11 , C12N2310/315 , C12N2310/32 , C12N2310/321 , C12N2310/322 , C12N2310/323 , C12N2310/3231 , C12N2310/34 , C12N2310/341 , C12N2310/346 , C12N2310/3521 , C12N2310/3525 , C12N2320/30 , C12N2320/51
摘要： 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.
摘要翻译：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活性或表达的调节。

2. 发明申请

WO2016077689A1 MODULATORY POLYNUCLEOTIDES 审中-公开
标题翻译：调节多核苷酸
公开(公告)号：WO2016077689A1
公开(公告)日：2016-05-19
申请号：PCT/US2015/060564
申请日：2015-11-13
申请人： VOYAGER THERAPEUTICS, INC.
发明人： HOU, Jinzhao , NONNENMACHER, Mathieu E. , ZHOU, Pengcheng
IPC分类号： C07H21/02 , C12N15/113 , C12N15/85
CPC分类号： C12N15/1137 , C12N7/00 , C12N15/111 , C12N15/86 , C12N2310/14 , C12N2310/141 , C12N2310/34 , C12N2310/531 , C12N2330/50 , C12N2750/14121 , C12N2750/14143 , C12Y115/01001
摘要： The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of modulatory polynucleotides.
摘要翻译：本发明涉及用于制备，制备和治疗性使用调节性多核苷酸的组合物和方法。

3. 发明申请

WO2013177468A2 METHODS AND COMPOSITIONS FOR MODULATING APOLIPOPROTEIN(A) EXPRESSION 审中-公开
标题翻译：调节APOLIPOPROTEIN（A）表达的方法和组合物
公开(公告)号：WO2013177468A2
公开(公告)日：2013-11-28
申请号：PCT/US2013042532
申请日：2013-05-23
申请人： ISIS PHARMACEUTICALS INC
发明人： CROOKE ROSANNE M , GRAHAM MARK J , FREIER SUSAN M
IPC分类号： A61K48/00
CPC分类号： C12N15/113 , C12N2310/11 , C12N2310/315 , C12N2310/32 , C12N2310/321 , C12N2310/3231 , C12N2310/3341 , C12N2310/34 , C12N2310/341 , C12N2310/346 , C12Y304/21007 , C12N2310/3525
摘要： Disclosed herein are antisense compounds and methods for decreasing apo(a) to treat, prevent, ameliorate diseases, disorders or conditions related to apo(a) or Lp(a). Certain diseases, disorders or conditions related to apo(a) or Lp(a) include inflammatory, cardiovascular and/or metabolic diseases, disorders or conditions. The antisense compounds disclosed herein can be used to treat such diseases, disorders or conditions in an individual in need thereof.
摘要翻译：本文公开了用于减少apo（a）以治疗，预防，改善与apo（a）或Lp（a）相关的疾病，病症或病症的反义化合物和方法。与apo（a）或Lp（a）相关的某些疾病，病症或病症包括炎性，心血管和/或代谢疾病，病症或病症。本文公开的反义化合物可用于治疗有需要的个体中的这些疾病，病症或病症。

4. 发明申请

WO2013103632A1 DOUBLE-STRANDED RNA COMPOUNDS TO CASP2 AND USES THEREOF 审中-公开
标题翻译：双链RNA化合物CASP2及其用途
公开(公告)号：WO2013103632A1
公开(公告)日：2013-07-11
申请号：PCT/US2013/020012
申请日：2013-01-03
申请人： QUARK PHARMACEUTICALS, INC. , ERLICH, Shai , THOMPSON, James D. , OZDEN, Rabia
发明人： ERLICH, Shai , THOMPSON, James D. , OZDEN, Rabia
IPC分类号： C12N15/113 , A61K31/713 , A61P27/02
CPC分类号： C12N15/1137 , A61M5/002 , C12N15/1135 , C12N2310/11 , C12N2310/14 , C12N2310/317 , C12N2310/321 , C12N2310/34 , C12N2310/344 , C12N2310/3521 , C12N2320/32 , C12N2320/35 , C12Y304/22055
摘要： The present disclosure relates to methods of treating a patient suffering from or at risk of developing an ocular disease, disorder or injury, and includes treatment regimens using a double-stranded RNA compound that down-regulates CASP2 expression, or a pharmaceutically acceptable salt thereof.
摘要翻译：本公开涉及治疗患有或有风险发生眼部疾病，障碍或损伤的患者的方法，并且包括使用下调CASP2表达的双链RNA化合物或其药学上可接受的盐的治疗方案。

5. 发明申请

WO2016168286A1 ANGIOPOIETIN-LIKE 3 (ANGPTL3) IRNA COMPOSITIONS AND METHODS OF USE THEREOF 审中-公开
标题翻译： ANGIOPOIETIN-LIKE 3（ANGPTL3）IRNA组合物及其使用方法
公开(公告)号：WO2016168286A1
公开(公告)日：2016-10-20
申请号：PCT/US2016/027271
申请日：2016-04-13
申请人： ALNYLAM PHARMACEUTICALS, INC.
发明人： QUERBES, William , FITZGERALD, Kevin , BUTLER, James , WILLIAMS, Stephanie , HINKLE, Gregory , MAIER, Martin
IPC分类号： C12N15/113 , A61K31/713 , C07H21/02
CPC分类号： C12N15/1136 , A61K31/713 , A61K47/549 , C12N2310/14 , C12N2310/315 , C12N2310/34 , C12N2310/344 , C12N2310/346 , C12N2310/351 , C12N2310/3515 , C12N2320/32 , C12N2320/35 , C12N2320/51 , C12N2310/321 , C12N2310/3521 , C12N2310/322 , C12N2310/3533
摘要： The invention relates to double-stranded ribonucleic acid (dsRNA) compositions targeting the ANGPTL3 gene, as well as methods of inhibiting epression of ANGPTL3 and methods of treating subjects having a disorder of lipid metabolism, such as hyperlipidemia or hypertriglyceridemia, using such dsRNA compositions.
摘要翻译：本发明涉及靶向ANGPTL3基因的双链核糖核酸（dsRNA）组合物，以及抑制ANGPTL3进展的方法以及使用这种dsRNA组合物治疗具有脂质代谢紊乱（例如高脂血症或高甘油三酯血症）的受试者的方法。

6. 发明申请

WO2016130943A1 HYBRID OLIGONUCLEOTIDES AND USES THEREOF 审中-公开
标题翻译：混合寡核苷酸及其用途
公开(公告)号：WO2016130943A1
公开(公告)日：2016-08-18
申请号：PCT/US2016/017790
申请日：2016-02-12
申请人： RANA THERAPEUTICS, INC.
发明人： OZSOLAK, Fatih
IPC分类号： C12N15/113 , C12N15/63 , A61K31/7105
CPC分类号： A61K31/7105 , C12N15/111 , C12N15/113 , C12N2310/141 , C12N2310/152 , C12N2310/34 , A61K2300/00
摘要： Provided herein are hybrid oligonucleotides comprising a region that promotes cleavage of a nucleic acid and a region that protects a nucleic acid from exonuclease activity. Such hybrid oligonucleotides are useful for modulating the expression of genes. Related compositions and methods are also provided. In some embodiments, methods are provided for treating a disease, such as by administering a hybrid oligonucleotide.
摘要翻译：本文提供了包含促进核酸切割的区域和保护核酸免受核酸外切酶活性的区域的杂交寡核苷酸。这样的杂交寡核苷酸可用于调节基因的表达。还提供了相关的组合物和方法。在一些实施方案中，提供了用于治疗疾病的方法，例如通过施用杂交寡核苷酸。

7. 发明申请

WO2013173635A1 COMPOSITIONS AND METHODS FOR MODULATING GENE EXPRESSION 审中-公开
标题翻译：调节基因表达的组合物和方法
公开(公告)号：WO2013173635A1
公开(公告)日：2013-11-21
申请号：PCT/US2013/041434
申请日：2013-05-16
申请人： RANA THERAPEUTICS, INC. , THE GENERAL HOSPITAL CORPORATION D/B/A MASSACHUSETTS GENERAL HOSPITAL
发明人： KRIEG, Arthur, M. , SUBRAMANIAN, Romesh , MCSWIGGEN, James , LEE, Jeannie, T.
IPC分类号： C12N15/11 , C07H21/00 , A61K31/7088
CPC分类号： A61K31/7088 , C07H21/00 , C07H21/02 , C07H21/04 , C12N15/113 , C12N2310/11 , C12N2310/34 , C12N2310/343
摘要： Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of a target gene. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of a target gene. Methods for modulating expression of a target gene using the single stranded oligonucleotides are also provided. Further aspects of the invention provide methods for selecting a candidate oligonucleotide for activating or enhancing expression of a target gene.
摘要翻译：本发明的方面提供了用于激活或增强靶基因表达的单链寡核苷酸。其它方面提供了包含用于激活或增强靶基因表达的单链寡核苷酸的组合物和试剂盒。还提供了使用单链寡核苷酸调节靶基因表达的方法。本发明的其他方面提供了用于选择用于激活或增强靶基因表达的候选寡核苷酸的方法。

8. 发明申请

WO2016190795A1 BLOCKING OLIGONUCLEOTIDES 审中-公开
标题翻译：阻塞OLIGONUCLEOTIDES
公开(公告)号：WO2016190795A1
公开(公告)日：2016-12-01
申请号：PCT/SE2016/050303
申请日：2016-04-11
申请人： KRJUTSKOV, Kaarel , SALUMETS, Andres , KERE, Juha
发明人： KRJUTSKOV, Kaarel , SALUMETS, Andres , KERE, Juha
IPC分类号： C12N15/113 , C12Q1/68
CPC分类号： C12Q1/6848 , C12N15/1096 , C12N15/11 , C12N2310/34 , C12Q1/6844 , C12Q2521/107 , C12Q2537/163
摘要： A blocking oligonucleotide (10, 10A, 10A', 10A", 10B, 10C, 10D, 10E) comprises a 3'-end complementary sequence (13) complementary to a 3'-end sequence (23) of a globin mRNA molecule (20; 20A, 20B) and a poly-A complementary sequence (12) of at least one nucleotide complementary to at least a portion of a poly-A sequence (22) of the globin mRNA molecule (20, 20A, 20B). The blocking oligonucleotide (10, 10A, 10A', 10A", 10B, 10C, 10D, 10E) is capable of inhibiting binding of a reverse transcription anchored poly-T primer (30) to the globin mRNA molecule (20, 20A, 20B) and thereby significantly reducing synthesis of globin cDNA from globin mRNA molecules (20, 20A, 20B) present in a sample. This high reduction of globin cDNA by the blocking oligonucleotide (10, 10A, 10A', 10A", 10B, 10C, 10D, 10E) is achieved without any significant degradation of mRNA molecules present in the sample.
摘要翻译：封闭寡核苷酸（10,10A，10A'，10A“，10B，10C，10D，10E）包含与球蛋白mRNA分子的3'末端序列（23）互补的3'末端互补序列（13） 20,20A，20B）和与所述珠蛋白mRNA分子（20,20A，20B）的poly-A序列（22）的至少一部分互补的至少一个核苷酸的多聚A互补序列（12）。阻断寡核苷酸（10,10A，10A'，10A“，10B，10C，10D，10E）能够抑制逆转录锚定多T引物（30）与球蛋白mRNA分子（20,20A，20B）的结合，从而显着减少样品中存在的球蛋白mRNA分子（20,20A，20B）的珠蛋白cDNA的合成。实现了通过阻断寡核苷酸（10,10A，10A'，10A“，10B，10C，10D，10E）高效降低珠蛋白cDNA，而没有样品中存在的mRNA分子的任何显着降解。

9. 发明申请

WO2015168605A1 METHODS FOR TREATMENT OF DISORDERS IN THE FRONT OF THE EYE UTILIZING NUCLEIC ACID MOLECULES 审中-公开
标题翻译：用于治疗使用核酸分子的眼睛前面的疾病的方法
公开(公告)号：WO2015168605A1
公开(公告)日：2015-11-05
申请号：PCT/US2015/028860
申请日：2015-05-01
申请人： RXI PHARMACEUTICALS CORPORATION
发明人： BYRNE, Michael , PAVCO, Pamela, A. , BULOCK, Karen, G.
IPC分类号： A61K48/00 , C07H21/02
CPC分类号： C12N15/1137 , A61K31/7088 , C12N15/1136 , C12N2310/11 , C12N2310/14 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/34 , C12N2310/346 , C12N2310/351 , C12N2310/3515 , C12N2310/3521 , C12N2310/3533 , C12N2320/32 , C12N2320/51 , C12N2320/52
摘要： Aspects of the invention relate to methods for treating an ocular disorder associated with the front of the eye, comprising administering to the eye of a subject in need thereof a therapeutic RNA molecule, in an effective amount to treat an ocular disorder associated with the front of the eye.
摘要翻译：本发明的方面涉及用于治疗与眼睛前部相关的眼部疾病的方法，包括向有需要的受试者的眼睛施用有效量的治疗性RNA分子，以治疗与前方相关的眼部疾病眼。

10. 发明申请

WO2012142458A1 ANTISENSE MODULATION OF PTP1B EXPRESSION 审中-公开
标题翻译： PTP1B表达的抗原调节
公开(公告)号：WO2012142458A1
公开(公告)日：2012-10-18
申请号：PCT/US2012/033588
申请日：2012-04-13
申请人： ISIS PHARMACEUTICALS, INC. , BHANOT, Sanjay , FREIER, Susan, M.
发明人： BHANOT, Sanjay , FREIER, Susan, M.
IPC分类号： C07H21/04 , C12N15/113
CPC分类号： C12N15/1137 , A61K31/7088 , C12N2310/11 , C12N2310/315 , C12N2310/321 , C12N2310/322 , C12N2310/3231 , C12N2310/3341 , C12N2310/34 , C12N2310/341 , C12N2310/346 , C12N2320/30 , C12Y301/03048 , C12N2310/3525
摘要： Provided herein are methods, compounds, and compositions for reducing expression of PTPIB mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent, delay, or ameliorate metabolic disease, for example, diabetes, or a symptom thereof.
摘要翻译：本文提供了用于减少动物中PTPIB mRNA和蛋白质表达的方法，化合物和组合物。这些方法，化合物和组合物可用于治疗，预防，延迟或改善代谢疾病，例如糖尿病或其症状。

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