公开(公告)号：US20170166898A1

公开(公告)日：2017-06-15

申请号：US15376633

申请日：2016-12-12

申请人： Nitto Denko Corporation

发明人： Kenjirou Minomi ,  Jens Harborth ,  Cima Cina ,  Wenbin Ying ,  Jane Zheng ,  Narendra Vaish

IPC分类号： C12N15/113

CPC分类号： C12N15/1137 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/343 ,  C12Y205/01018 ,  C12N2310/3521 ,  C12N2310/3533

摘要： This invention provides compounds, compositions and methods for modulating the expression of target genes using RNA interference. RNAi structures and molecules of this invention can be used for modulating or silencing the expression of genes, with high levels of RNAi activity and reduced off target actions. Advantageous structures include siRNAs targeted to any gene having one or more 2′-deoxy nucleotides located in the seed region. The RNA interference molecules can be used in methods for preventing or treating diseases.

公开(公告)号：US20170159053A1

公开(公告)日：2017-06-08

申请号：US15371300

申请日：2016-12-07

申请人： Alnylam Pharmaceuticals, Inc.

发明人： Akin Akinc ,  Benny Sorensen ,  Pushkal Garg ,  Gabriel Robbie

IPC分类号： C12N15/113

CPC分类号： C12N15/113 ,  A61K31/713 ,  A61K47/549 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2320/35 ,  C12N2310/3521 ,  C12N2310/322 ,  C12N2310/3533 ,  C12N2310/335

摘要： The invention relates to iRNA, e.g., double stranded ribonucleic acid (dsRNA), compositions targeting the Serpinc1 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of Serpinc1 and to treat subjects having a Serpinc1-associated disease, e.g., a bleeding disorder, such as a hemophilia.

公开(公告)号：US20170130226A1

公开(公告)日：2017-05-11

申请号：US15320929

申请日：2015-06-23

申请人： ALTERMUNE LIMITED

发明人： Bradley HALL ,  Paul HATALA

IPC分类号： C12N15/115

CPC分类号： C12N15/115 ,  C12N2310/16 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/344 ,  C12N2310/351 ,  C12N2310/3513 ,  C12N2320/51 ,  C12N2310/3533 ,  C12N2310/3521

摘要： The invention relates to novel aptamers, in particular aptamers which are capable of binding to EGFR. The invention also relates to cancer cell binding complexes comprising said aptamers and the use of said cancer cell binding complexes in the treatment of cancer.

公开(公告)号：US09631192B2

公开(公告)日：2017-04-25

申请号：US14358942

申请日：2012-11-19

申请人： The United States of America, as represented by the Secretary, Department of Health & Human Services

发明人： Bruce A. Shapiro ,  Kirill A. Afonin ,  Mathias D. Viard ,  Eckart H. Bindewald ,  Luc Jaeger ,  Arti N. Santhanam

IPC分类号： C07H21/02 ,  C07H21/04 ,  A61K48/00 ,  C12N15/113 ,  C12N15/11

CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N15/1132 ,  C12N15/1135 ,  C12N2310/14 ,  C12N2310/16 ,  C12N2310/322 ,  C12N2310/3519 ,  C12N2320/51 ,  C12N2310/3533

摘要： Auto-recognizing therapeutic R/DNA chimeric nanoparticles (R/DNA NP) are described that are pairs of DNA/RNA hybrids where the DNA molecules have complementary toehold sequences that promote the re-association of the R/DNA NPs when mixed resulting in the formation of RNA/RNA duplexes that act as siRNAs.

公开(公告)号：US09574192B2

公开(公告)日：2017-02-21

申请号：US14284745

申请日：2014-05-22

申请人： Alnylam Pharmaceuticals, Inc.

发明人： Alfica Sehgal ,  Klaus Charisse ,  Brian Bettencourt ,  Martin Maier ,  Kallanthottathil G. Rajeev ,  Gregory Hinkle ,  Muthiah Manoharan

IPC分类号： C07H21/02 ,  C07H21/04 ,  A61K48/00 ,  C12N15/113 ,  A61K31/712 ,  A61K31/713

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相关疾病如肝脏疾病的受试者的方法。

公开(公告)号：US20170044535A1

公开(公告)日：2017-02-16

申请号：US15299549

申请日：2016-10-21

申请人： INTEGRATED DNA TECHNOLOGIES, INC.

发明人： Michael Allen Collingwood ,  Ashley Mae Jacobi ,  Garrett Richard Rettig ,  Mollie Sue Schubert ,  Mark Aaron Behlke

IPC分类号： C12N15/113

CPC分类号： C12N15/111 ,  C12N2310/10 ,  C12N2310/20 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/346 ,  C12N2310/531 ,  C12N2320/51 ,  C12N2320/53 ,  C12N2310/3521 ,  C12N2310/3533

摘要： This invention pertains to modified compositions for use in CRISPR systems, and their methods of use. In particular, length-modified and chemically-modified forms of crRNA and tracrRNA are described for use as a reconstituted guide RNA for interaction with Cas9 of CRISPR systems. The resultant length-modified and chemically-modified forms of crRNA and tracrRNA are economical to produce and can be tailored to have unique properties relevant to their biochemical and biological activity in the context of the CRISPR Cas9 endonuclease system.

公开(公告)号：US20160369279A1

公开(公告)日：2016-12-22

申请号：US15251155

申请日：2016-08-30

申请人： Sirna Therapeutics, Inc.

发明人： Steven Bartz ,  Duncan Brown ,  Michael Robinson

IPC分类号： C12N15/113 ,  A61K47/34 ,  A61K47/28 ,  A61K47/24 ,  A61K31/713 ,  A61K47/18

CPC分类号： C12N15/1131 ,  A61K9/5123 ,  A61K31/713 ,  A61K47/18 ,  A61K47/24 ,  A61K47/28 ,  A61K47/34 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2320/32 ,  C12N2310/321 ,  C12N2310/3521 ,  C12N2310/322 ,  C12N2310/3533 ,  C12N2310/317 ,  C12N2310/332

摘要： The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of HBV gene expression and/or activity, and/or modulate a HBV gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against HBV gene expression.

公开(公告)号：US20160354404A1

公开(公告)日：2016-12-08

申请号：US15072016

申请日：2016-03-16

申请人： Alnylam Pharmaceuticals, Inc.

发明人： Gregory Hinkle ,  Maria Frank-Kamenetsky ,  Kevin Fitzgerald

IPC分类号： A61K31/713 ,  C12N15/113

CPC分类号： A61K31/713 ,  C12N15/1137 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/314 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/3233 ,  C12N2310/332 ,  C12N2310/3515 ,  C12Y304/21061 ,  C12N2310/3521 ,  C12N2310/3527 ,  C12N2310/3533 ,  C12N2310/3531

摘要： The invention relates to sirens targeting a PCs gene, and methods of using sirens to inhibit expression of PCs and to treat PCs related disorders, e.g., hyperlipidemia.

摘要翻译： 本发明涉及靶向PCs基因的警报器，以及使用警报器抑制PC表达和治疗PC相关疾病例如高脂血症的方法。

公开(公告)号：US20160340679A1

公开(公告)日：2016-11-24

申请号：US15224805

申请日：2016-08-01

申请人： Alnylam Pharmaceuticals, Inc.

发明人： Kevin Fitzgerald ,  Brian Bettencourt ,  Gregory Hinkle ,  Jennifer Willoughby

IPC分类号： C12N15/113 ,  A61K31/713

CPC分类号： C12N15/1137 ,  A61K31/713 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/335 ,  C12N2310/3515 ,  C12N2310/3521 ,  C12N2310/3533

摘要： The present invention relates to RNAi agents, e.g., double-stranded RNAi agents, targeting the ketohexokinase (KHK) gene, and methods of using such RNAi agents to inhibit expression of KHK and methods of treating subjects having a KHK-associated disorder, e.g., liver disease (e.g., fatty liver, steatohepatitis), dyslipidemia (e.g., hyperlipidemia, high LDL cholesterol, low HDL cholesterol, hypertriglyceridemia, postprandial hypertriglyceridemia), disorders of glycemic control (e.g., insulin resistance, diabetes), cardiovascular disease (e.g., hypertension, endothelial cell dysfunction), kidney disease (e.g., acute kidney disorder, tubular dysfunction, proinflammatory changes to the proximal tubules), metabolic syndrome, adipocyte dysfunction, visceral adipose deposition, obesity, hyperuricemia, gout, eating disorders, and excessive sugar craving.

摘要翻译： 本发明涉及靶向酮基己糖激酶（KHK）基因的RNAi试剂，例如双链RNAi试剂，以及使用这种RNAi试剂抑制KHK表达的方法以及治疗具有KHK相关病症的受试者的方法， 肝脏疾病（例如脂肪肝，脂肪性肝炎），血脂异常（例如高脂血症，高LDL胆固醇，低HDL胆固醇，高甘油三酯血症，餐后高甘油三酯血症），血糖控制障碍（例如胰岛素抵抗，糖尿病），心血管疾病（例如高血压 ，内皮细胞功能障碍），肾脏疾病（例如急性肾脏疾病，管状功能障碍，近端小管的炎性改变），代谢综合征，脂肪细胞功能障碍，内脏脂肪沉积，肥胖症，高尿酸血症，痛风，进食障碍和过度糖渴求。

公开(公告)号：US20160296550A1

公开(公告)日：2016-10-13

申请号：US15144429

申请日：2016-05-02

申请人： Ophthotech Corporation

发明人： Samir Patel ,  Richard Everett ,  Douglas Brooks ,  Shane Xinxin Tian

IPC分类号： A61K31/7088 ,  A61K9/00 ,  C12N15/115 ,  A61K9/14 ,  C07K16/22 ,  A61K39/395 ,  A61K45/06

CPC分类号： A61K31/7088 ,  A61K9/0048 ,  A61K9/143 ,  A61K31/713 ,  A61K39/3955 ,  A61K45/06 ,  C07K16/22 ,  C07K2317/24 ,  C07K2317/55 ,  C07K2317/76 ,  C12N15/115 ,  C12N2310/16 ,  C12N2310/314 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/351 ,  C12N2320/30 ,  C12N2320/31 ,  A61K2300/00 ,  C12N2310/3533 ,  C12N2310/3521

摘要： The present invention relates to methods for treating and preventing ophthalmological disease and disorders, comprising administering Antagonist A or another pharmaceutically acceptable salt thereof, optionally in combination with another treatment, to a subject in need thereof. The present invention also relates to methods for treating and preventing ophthalmological disease and disorders, comprising administering an anti-C5 agent (e.g., ARC1905), optionally in combination with another treatment, to a subject in need thereof.