公开(公告)号：EP2999785A1

公开(公告)日：2016-03-30

申请号：EP14730051.1

申请日：2014-05-22

申请人： Alnylam Pharmaceuticals, Inc.

发明人： SEHGAL, Alfica ,  CHARISSE, Klaus ,  BETTENCOURT, Brian ,  MAIER, Martin ,  RAJEEV, Kallanthottathil G. ,  HINKLE, Gregory ,  MANOHARAN, Muthiah

IPC分类号： C12N15/113 ,  A61K31/712 ,  A61K31/713 ,  A61P1/16 ,  A61P11/00 ,  A61P35/00

CPC分类号： C12N15/113 ,  A61K31/712 ,  A61K31/713 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/32 ,  C12N2310/321 ,  C12N2310/331 ,  C12N2310/332 ,  C12N2310/343 ,  C12N2310/344 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2320/32 ,  C12N2320/34 ,  C12N2320/51 ,  C12N2330/30 ,  C12N2310/322 ,  C12N2310/3533 ,  C12N2310/3521

摘要： The invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the Serpina1 gene, and methods of using such RNAi agents to inhibit expression of Serpina1 and methods of treating subjects having a Serpina1 associated disease, such as a liver disorder.

摘要翻译： 本发明涉及靶向Serpina1基因的RNAi试剂，例如双链RNAi试剂，以及使用这种RNAi试剂抑制Serpina1表达的方法以及治疗患有Serpina1相关疾病例如肝脏疾病的受试者的方法。

公开(公告)号：EP2793906A4

公开(公告)日：2016-01-13

申请号：EP12860538

申请日：2012-12-21

申请人： MODERNA THERAPEUTICS INC

发明人： BANCEL STEPHANE ,  DE FOUGEROLLES ANTONIN

IPC分类号： A61K31/7088

CPC分类号： A01N1/0226 ,  A61K31/7088 ,  A61K31/7115 ,  A61K31/712 ,  A61K38/1866 ,  A61K48/00

公开(公告)号：EP2792367A4

公开(公告)日：2015-09-30

申请号：EP12857103

申请日：2012-12-12

申请人： KYOWA HAKKO KIRIN CO LTD

发明人： KUBOYAMA TAKESHI ,  ERA TOMOHIRO ,  NAOI TOMOYUKI

IPC分类号： A61K47/18 ,  A61K9/00 ,  A61K9/127 ,  A61K9/51 ,  A61K31/7088 ,  A61K38/00 ,  C07C211/21 ,  C12N15/88

CPC分类号： A61K9/5123 ,  A61K9/0019 ,  A61K31/7088 ,  A61K31/712 ,  A61K31/713 ,  A61K47/18 ,  C07C211/21 ,  C12N15/111 ,  C12N15/113 ,  C12N15/88 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2320/32

摘要： The present invention provides a lipid nano-particles, which allow nucleic acids to be easily introduced into cells, comprising a cationic lipid represented by formula (I) (wherein: R 1 and R 2 are, the same or different, alkenyl, etc, and X 3 is absent or is alkyl, etc, X 1 and X 2 are hydrogen atoms, or are combined together to form a single bond or alkylene, and Y 1 is absent or anion, L 1 is a single bond, etc, R 3 is alkyl, etc), and the like.

摘要翻译： 本发明提供一种脂质纳米颗粒，其允许将核酸容易地引入细胞，其包含由式（I）表示的阳离子脂质（其中：R 1和R 2相同或不同，烯基等） 并且X 3不存在或者是烷基等，X 1和X 2是氢原子，或者组合在一起形成单键或亚烷基，Y 1不存在或阴离子，L 1是单键等，R 3是烷基等）等。

公开(公告)号：EP2850187A1

公开(公告)日：2015-03-25

申请号：EP13790838.0

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 ,  C12N15/63 ,  A61K31/7088 ,  A61P35/00

CPC分类号： C12N15/1137 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  C07H21/00 ,  C07H21/02 ,  C07H21/04 ,  C12N15/1135 ,  C12N2310/11 ,  C12N2310/31 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3231 ,  C12N2310/346 ,  C12N2310/351 ,  C12Y301/03016 ,  C12Y301/03048 ,  C12Y301/03067 ,  C12N2310/3521

摘要： Aspects of the invention provide single stranded oligonucleotides for activating or enhancing expression of PTEN. Further aspects provide compositions and kits comprising single stranded oligonucleotides for activating or enhancing expression of PTEN. Methods for modulating expression of PTEN 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 PTEN.

公开(公告)号：EP2849760A4

公开(公告)日：2015-03-25

申请号：EP13791584

申请日：2013-05-17

申请人： REPLICOR INC

发明人： BAZINET MICHEL ,  VAILLANT ANDREW

IPC分类号： A61K31/7088 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  A61K31/713 ,  A61P3/06 ,  A61P21/00 ,  A61P25/28 ,  A61P31/12 ,  A61P31/14 ,  A61P31/16

CPC分类号： A61K31/7088 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  A61K31/713 ,  A61K31/7135

摘要： It is described pharmaceutical compositions and methods for the treatment of viral infections, hypercholesterolemia, hypertriglyceridemia, Alzheimer's disease, prion disease and Duchene's muscular dystrophy with oligonucleotide chelate complexes.

公开(公告)号：EP2842577A1

公开(公告)日：2015-03-04

申请号：EP14189625.8

申请日：2011-03-11

申请人： Daiichi Sankyo Company, Limited

发明人： Kawashima, Kayoko ,  Koshimizu, Uichi

IPC分类号： A61K48/00 ,  A61K31/7105 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  A61K35/76 ,  A61P9/00 ,  A61P9/04 ,  A61P9/10

CPC分类号： C12N5/0657 ,  A61K31/7105 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  A61K35/34 ,  C12N15/113 ,  C12N2310/141 ,  C12N2320/11

摘要： The present invention addresses the problems of providing a method for proliferating cardiomyocytes using a miRNA that promotes the proliferation of cardiomyocytes, a vector for use in the treatment of heart disease, a pharmaceutical composition for treating heart disease, and so forth.
The present invention provides a method for proliferating cardiomyocytes using a miRNA having the cardiomyocyte proliferation promoting action, a vector for use in the treatment of heart disease that comprises said miRNA, a pharmaceutical composition for treating heart disease that comprises said vector, and so forth. A particularly preferred miRNA is one that is selected from the group consisting of mature miRNAs, i.e., miR-148a, miR-148b, miR-152, and miR-373, and precursors of said miRNAs, as well as variants and analogs thereof.

摘要翻译： 本发明解决了提供使用促进心肌细胞增殖的miRNA增殖心肌细胞的方法，用于治疗心脏病的载体，用于治疗心脏病的药物组合物等的问题。 本发明提供使用具有心肌细胞增殖作用的miRNA增殖心肌细胞的方法，用于治疗心脏病的载体，其包含所述miRNA，用于治疗包括所述载体的心脏病的药物组合物等。 特别优选的miRNA是选自成熟miRNA，即miR-148a，miR-148b，miR-152和miR-373以及所述miRNA的前体以及其变体和类似物的miRNA。

公开(公告)号：EP2841579A1

公开(公告)日：2015-03-04

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

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.

摘要翻译： 本文描述了抑制miR-21活性的组合物和方法。 组合物具有产生miR-21活性的有效抑制剂的某些核苷修饰模式。 该组合物可用于抑制miR-21，并且还可以治疗与miR-21异常表达相关的疾病，例如纤维化和癌症。

公开(公告)号：EP2809400A1

公开(公告)日：2014-12-10

申请号：EP13743020.3

申请日：2013-02-04

申请人： Antisense Therapeutics Ltd

发明人： TACHAS, George

IPC分类号： A61P5/08 ,  A61K31/7088 ,  A61K38/27

CPC分类号： A61K38/27 ,  A61K31/7088 ,  A61K31/7115 ,  A61K31/712 ,  A61K45/06 ,  A61K2300/00

摘要： The present disclosure relates to a method for treatment or prevention of diseases have an increased level of insulin-like growth factor I (IGF-I). The method comprises administration of a growth hormone (GH) variant having antagonistic activity in combination with an oligonucleotide targeted to growth hormone receptor (GHR) to a subject in need.

摘要翻译： 本发明涉及一种用于治疗或预防疾病有胰岛素样生长因子I（IGF-I）增加的水平。 该方法包括具有在寡核苷酸靶向生长激素受体（GHR），以在需要的受试者中组合拮抗活性的生长激素（GH）的变体的施用。

公开(公告)号：EP2793906A2

公开(公告)日：2014-10-29

申请号：EP12860538.3

申请日：2012-12-21

申请人： Moderna Therapeutics, Inc.

发明人： BANCEL, Stephane ,  DE FOUGEROLLES, Antonin

IPC分类号： A61K31/7088

CPC分类号： A01N1/0226 ,  A61K31/7088 ,  A61K31/7115 ,  A61K31/712 ,  A61K38/1866 ,  A61K48/00

摘要： The invention relates to compositions and methods for the manufacture and optimization of modified mRNA molecules for their use in improving organ viability and/or longevity.

公开(公告)号：EP2771463A1

公开(公告)日：2014-09-03

申请号：EP12843053.5

申请日：2012-10-25

申请人： Isis Pharmaceuticals, Inc.

发明人： FREIER, Susan M. ,  BHANOT, Sanjay

IPC分类号： C12N15/11 ,  C07H21/04 ,  C12N5/00

CPC分类号： C12N15/1138 ,  A61K31/7088 ,  A61K31/7115 ,  A61K31/712 ,  A61K31/7125 ,  A61K45/06 ,  C07H21/04 ,  C12N2310/11 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/3525 ,  C12N2310/3521

摘要： Provided herein are methods, compounds, and compositions for reducing expression of GCCR 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.