公开(公告)号：EP3222724B1

公开(公告)日：2018-10-31

申请号：EP17160954.8

申请日：2003-08-05

申请人： Silence Therapeutics GmbH

发明人： Giese, Klaus ,  Kaufmann, Jörg ,  Klippel-Giese, Anke

IPC分类号： C12N15/113 ,  A61K31/713 ,  C07H21/00 ,  C12Q1/68 ,  A01K67/027 ,  C12N5/10

CPC分类号： C12N15/1137 ,  A61K31/713 ,  A61K38/00 ,  C12N5/10 ,  C12N15/111 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/3183 ,  C12N2310/32 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/343 ,  C12N2310/346 ,  C12N2310/3521 ,  C12N2310/3525 ,  C12N2310/53 ,  C12N2310/531 ,  C12N2320/51 ,  C12Q1/68 ,  C12Y207/11001

摘要： The present invention is related to a ribonucleic acid comprising a double stranded structure whereby the double- stranded structure comprises a first strand and a second strand, whereby the first strand comprises a first stretch of contiguous nucleotides and whereby said first stretch is at least partially complementary to a target nucleic acid, and the second strand comprises a second stretch of contiguous nucleotides whereby said second stretch is at least partially identical to a target nucleic acid, and whereby the double stranded structure is blunt ended.

公开(公告)号：EP1730280B1

公开(公告)日：2018-10-24

申请号：EP05732497.2

申请日：2005-03-23

申请人： Curis, Inc. ,  Genentech, Inc.

发明人： BUMCROT, David, A.

IPC分类号： C12N15/11

CPC分类号： C12N15/113 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/53

摘要： The instant application relates to methods and reagents for modulating the Hedgehog signaling pathway using RNA interference technology (RNAi). The application provides potential targets of the Hedgehog RNAi, methods to identify additional Hedgehog signaling pathway components, methods to inhibit Hedgehog signaling targets using siRNA, and their uses in the treatment of a number of disease conditions.

公开(公告)号：EP1309726B2

公开(公告)日：2018-10-03

申请号：EP01922870.9

申请日：2001-03-30

申请人： WHITEHEAD INSTITUTE FOR BIOMEDICAL RESEARCH ,  Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. ,  MASSACHUSETTS INSTITUTE OF TECHNOLOGY ,  University of Massachusetts

发明人： TUSCHL, Thomas ,  SHARP, Phillip, A. ,  ZAMORE, Phillip, D. ,  BARTEL, David, P.

IPC分类号： C12N15/11

CPC分类号： C12N15/113 ,  A01K67/0336 ,  A01K2207/05 ,  A01K2217/075 ,  A01K2227/703 ,  A01K2267/03 ,  A61K38/00 ,  C07H21/02 ,  C12N15/1079 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/321 ,  C12N2310/53 ,  C12N2330/30 ,  C12Q1/66 ,  C12N2310/3521

摘要： The present invention relates to a Drosophila in vitro system which was used to demonstrate that dsRNA is processed to RNA segments 21-23 nucleotides (nt) in length. Furthermore, when these 21-23 nt fragments are purified and added back to Drosophila extracts, they mediate RNA interference in the absence of long dsRNA. Thus, these 21-23 nt fragments are the sequence-specific mediators of RNA degradation. A molecular signal, which may be their specific length, must be present in these 21-23 nt fragments to recruit cellular factors involved in RNAi. This present invention encompasses these 21-23 nt fragments and their use for specifically inactivating gene function. The use of these fragments (or chemically synthesized oligonucleotides of the same or similar nature) enables the targeting of specific mRNAs for degradation in mammalian cells, where the use of long dsRNAs to elicit RNAi is usually not practical, presumably because of the deleterious effects of the interferon response. This specific targeting of a particular gene function is useful in functional genomic and therapeutic applications.

公开(公告)号：EP3137119A4

公开(公告)日：2018-05-09

申请号：EP15785528

申请日：2015-04-28

申请人： RXI PHARMACEUTICALS CORP

发明人： BYRNE MICHAEL ,  BULOCK KAREN G ,  CARDIA JAMES

IPC分类号： A61K48/00

CPC分类号： C12N15/1135 ,  C12N15/113 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/3515 ,  C12N2310/53 ,  C12N2320/32 ,  C12N2320/35

摘要： Aspects of the invention relate to methods for treating cancer by administering to a subject in need thereof a therapeutically effective amount of a nucleic acid molecule that is directed against a gene encoding mouse double minute 1 homolog (MDM1), mouse double minute 2 homolog (MDM2), mouse double minute 3 homolog (MDM3), mouse double minute 4 homolog (MDM4) or V-myc myelocytomatosis viral related oncogene (MYCN) for treating cancer. Further aspects of the invention relate to nucleic acid molecules and compositions comprising nucleic acid molecules.

公开(公告)号：EP2385123B1

公开(公告)日：2018-04-25

申请号：EP11175570.8

申请日：2002-09-27

申请人： Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V.

发明人： Tuschl, Thomas ,  Lagos-Quintana, Mariana ,  Lendeckel, Winfried ,  Dammann, Jutta ,  Rauhut, Reinhard

IPC分类号： A61K38/00 ,  C12Q1/68 ,  C12N15/113

CPC分类号： C12N15/113 ,  A61K38/00 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/53 ,  C12N2330/10 ,  C12Q1/6876 ,  C12Q2600/136 ,  C12Q2600/178

摘要： In Caenorhabditis elegans, lin-4 and let-7 encode 22- and 21-nucleotide RNAs, respectively, that function as key regulators of developmental timing. Because the appearance of these short RNAs is regulated during development, they are also referred to as "small temporal RNAs" (stRNAs). We show that many more 21- and 22-nt expressed RNAs, termed microRNAs (miRNAs), exist in invertebrates and vertebrates, and that some of these novel RNAs, similar to let-7 stRNA, are also highly conserved. This suggests that sequence-specific post-transcriptional regulatory mechanisms mediated by small RNAs are more general than previously appreciated.

公开(公告)号：EP2213736B1

公开(公告)日：2018-03-07

申请号：EP10002422.3

申请日：2002-01-09

申请人： Alnylam Europe AG

发明人： Kreutzer, Roland ,  Limmer, Stefan ,  Vornlocher, Hans-Peter ,  Hadwiger, Philipp ,  Geick, Anke ,  Ocker, Matthias ,  Herold, Christoph ,  Schuppan, Detlef

IPC分类号： C12N15/11 ,  A61K31/713 ,  C12N15/88 ,  A61P35/00

CPC分类号： C12N15/113 ,  C12N15/1131 ,  C12N2310/152 ,  C12N2310/315 ,  C12N2310/3183 ,  C12N2310/33 ,  C12N2310/351 ,  C12N2310/3511 ,  C12N2310/53

公开(公告)号：EP2933333B1

公开(公告)日：2018-02-21

申请号：EP15169933.7

申请日：2011-07-08

申请人： Bonac Corporation

发明人： Ohgi, Tadaaki ,  Hayashi, Hirotake ,  Hamasaki, Tomohiro ,  Shirohzu, Hisao ,  Itoh, Akihiro ,  Suzuki, Hiroshi

IPC分类号： C12N15/113 ,  C12N15/11

CPC分类号： C12N15/111 ,  C12N2310/14 ,  C12N2310/318 ,  C12N2310/53 ,  C12N2310/531 ,  C12N2310/533

摘要： Provided is a novel nucleic acid molecule that can inhibit the expression of a gene and can be produces easily and efficiently. The nucleic acid molecule is a single-stranded nucleic acid molecule including an expression inhibitory sequence that inhibits expression of a target gene. The single-stranded nucleic acid molecule includes, in sequence from the 5' side to the 3' side: a 5' side region (Xc); an inner region (Z); and a 3' side region (Yc). The inner region (Z) is composed of an inner 5' side region (X) and an inner 3' side region (Y) that are linked to each other. The 5' side region (Xc) is complementary to the inner 5' side region (X). The 3' side region (Yc) is complementary to the inner 3' side region (Y). At least one of the inner region (Z), the 5' side region (Xc), and the 3' side region (Yc) includes the expression inhibitory sequence. According to this single-stranded nucleic acid molecule, it is possible to inhibit the expression of the target gene.

公开(公告)号：EP2902406B1

公开(公告)日：2018-01-31

申请号：EP14195627.6

申请日：2003-02-20

申请人： Sirna Therapeutics, Inc.

发明人： MCSWIGGEN, James ,  BEIGELMAN, Leonid ,  CHOWRIRA, Bharat ,  PAVCO, Pamela ,  FOSNAUGH, Kathy ,  JAMISON, Sharon ,  USMAN, Nassim ,  THOMPSON, James

IPC分类号： C07H21/02 ,  C07H21/04 ,  C12Q1/68 ,  C12N15/85 ,  C12N15/86 ,  C12P19/34 ,  A61K48/00

CPC分类号： C12N15/8218 ,  A61K38/00 ,  A61K47/54 ,  C07H21/02 ,  C12N15/111 ,  C12N15/1131 ,  C12N15/1138 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/318 ,  C12N2310/321 ,  C12N2310/322 ,  C12N2310/332 ,  C12N2310/344 ,  C12N2310/346 ,  C12N2310/53 ,  C12N2320/51 ,  C12N2330/30 ,  Y02A50/393 ,  C12N2310/3521

摘要： The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified 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 capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.

公开(公告)号：EP2314687B1

公开(公告)日：2017-12-27

申请号：EP10179492.3

申请日：2004-01-15

申请人： Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V.

发明人： Tuschl, Thomas ,  Achsel, Tilmann ,  Lührmann, Reinhard ,  Dammann, Jutta

IPC分类号： C12N15/11

CPC分类号： C12N15/111 ,  A01K2217/05 ,  A01K2217/075 ,  A61K38/00 ,  A61K48/0058 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/53 ,  C12N2330/30

摘要： The invention relates to vectors for the inducible expression of RNA molecules in eukaryotic, particularly mammalian cells and transgenic animals.

公开(公告)号：EP2930245B1

公开(公告)日：2017-11-22

申请号：EP15163041.5

申请日：2008-10-13

申请人： Sigma-Aldrich Co. LLC

发明人： Zhang, Min ,  Ross, James S.

IPC分类号： C12N15/113 ,  C12N9/24

CPC分类号： C12N15/1137 ,  C12N9/2402 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/53 ,  C12N2510/02 ,  C12Y302/01018

摘要： The present invention provides compositions and methods for the production of glycoproteins with enhanced sialylation. In particular, the invention provides cell lines comprising disrupted sialidase expression and methods of using the cell lines to produce glycoproteins with enhanced sialylation.