公开(公告)号：US11920134B2

公开(公告)日：2024-03-05

申请号：US16846535

申请日：2020-04-13

申请人： Arrowhead Pharmaceuticals, Inc.

发明人： John Frederick Boylan ,  Birgit Bramlage ,  Markus Hossbach ,  John Reidhaar-Olson

IPC分类号： C12N15/113 ,  A01K67/00 ,  A61K48/00 ,  C12N9/02

CPC分类号： C12N15/1137 ,  A01K67/00 ,  C12N9/0093 ,  C12N15/1135 ,  C12Y117/04001 ,  A61K48/00 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/33 ,  C12N2310/335 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2310/50 ,  C12N2310/53 ,  C12N2310/321 ,  C12N2310/3521

摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a RRM2 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA or nucleic acid molecules or vectors encoding the same together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of a RRM2 gene using said pharmaceutical composition; and methods for inhibiting the expression of RRM2 in a cell.

公开(公告)号：US20230235333A1

公开(公告)日：2023-07-27

申请号：US18010945

申请日：2021-06-17

申请人： RIBOMIC INC

发明人： Kazuteru Aoki ,  Marie Mitsui

IPC分类号： C12N15/115 ,  C12N15/10 ,  G01N33/50

CPC分类号： C12N15/115 ,  C12N15/1048 ,  G01N33/5041 ,  C12N2310/16 ,  C12N2310/322 ,  C12N2310/53 ,  C12N2310/3515 ,  C12N2310/313

摘要： The present invention provides an aptamer that binds to IL-21, an aptamer that binds to IL-21 and inhibits the binding of IL-21 and a receptor thereof, and an aptamer that binds to IL-21 and contains a nucleotide sequence represented by the formula (1): CGRYKACY wherein R is A or G, Y is C or U, and K is G or U.

公开(公告)号：US20190233821A1

公开(公告)日：2019-08-01

申请号：US16381288

申请日：2019-04-11

申请人： SIRNA THERAPEUTICS, INC.

发明人： Leonid Beigelman ,  James McSwiggen ,  Chandra Vargeese

IPC分类号： C12N15/113 ,  C12N15/82 ,  C12N15/11 ,  C07H21/02

CPC分类号： C12N15/113 ,  C07H21/02 ,  C12N15/111 ,  C12N15/1131 ,  C12N15/1137 ,  C12N15/1138 ,  C12N15/8218 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/322 ,  C12N2310/3231 ,  C12N2310/344 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2310/53 ,  C12N2320/32 ,  C12N2320/51 ,  C12Y304/21021

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：US20180320181A1

公开(公告)日：2018-11-08

申请号：US16021165

申请日：2018-06-28

申请人： National University Corporation Tokyo Medical and Dental University ,  Osaka University

发明人： Takanori Yokota ,  Kazutaka Nishina ,  Satoshi Obika ,  Hidehiro Mizusawa

IPC分类号： C12N15/113 ,  C12N15/11

CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N2310/11 ,  C12N2310/15 ,  C12N2310/3181 ,  C12N2310/3231 ,  C12N2310/341 ,  C12N2310/3513 ,  C12N2310/3515 ,  C12N2310/53 ,  C12N2320/52

摘要： A method of reducing the level of a transcription product in a cell comprising contacting with the cell a composition comprising a double-stranded nucleic acid complex comprising a first nucleic acid strand annealed to a second nucleic acid strand, wherein: (i) the first nucleic acid strand hybridizes to the transcription product and comprises (a) a region consisting of at least 4 consecutive nucleotides that are recognized by RNase H when the strand is hybridized to the transcription product, (b) one or more nucleotide analogs located on 5′ terminal side of the region, (c) one or more nucleotide analogs located on 3′ terminal side of the region and (d) a total number of nucleotides and nucleotide analogs ranging from 8 to 35 nucleotides and (ii) the second nucleic acid strand comprises (a) nucleotides and optionally nucleotide analogs and (b) at least 4 consecutive RNA nucleotides.

公开(公告)号：US20180291381A1

公开(公告)日：2018-10-11

申请号：US16013776

申请日：2018-06-20

申请人： Silence Therapeutics GMBH

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

IPC分类号： C12N15/113 ,  A61K31/713 ,  C12Q1/68

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：US20180223286A1

公开(公告)日：2018-08-09

申请号：US15678024

申请日：2017-08-15

申请人： Silence Therapeutics GMBH

发明人： Klaus GIESE ,  Jörg KAUFMANN ,  Anke KLIPPEL-GIESE

IPC分类号： C12N15/113 ,  C12N5/10 ,  C12N15/11 ,  C12Q1/68 ,  A61K31/713 ,  A61K38/00

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：US20180195074A1

公开(公告)日：2018-07-12

申请号：US15906054

申请日：2018-02-27

申请人： Arrowhead Pharmaceuticals, Inc.

发明人： John Frederick Boylan ,  Birgit Bramlage ,  Markus Hossbach ,  John Reidhaar-Olson

IPC分类号： C12N15/113

CPC分类号： C12N15/1137 ,  A01K67/00 ,  A61K48/00 ,  C12N9/0093 ,  C12N15/1135 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/33 ,  C12N2310/335 ,  C12N2310/346 ,  C12N2310/351 ,  C12N2310/50 ,  C12N2310/53 ,  C12Y117/04001 ,  C12N2310/3521

摘要： The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a RRM2 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA or nucleic acid molecules or vectors encoding the same together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of a RRM2 gene using said pharmaceutical composition; and methods for inhibiting the expression of RRM2 in a cell.

公开(公告)号：US20180179526A1

公开(公告)日：2018-06-28

申请号：US15870380

申请日：2018-01-12

申请人： Alnylam Pharmaceuticals, Inc.

发明人： Roland Kreutzer ,  Stefan Limmer

IPC分类号： C12N15/113 ,  C12N15/11

CPC分类号： C12N15/113 ,  A61K31/713 ,  A61K38/00 ,  C12N15/111 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/53 ,  C12N2330/30 ,  Y02A50/411 ,  Y02A50/467

摘要： The invention relates to an isolated RNA that mediates RNA interference of an mRNA to which it corresponds and a method of mediating RNA interference of mRNA of a gene in a cell or organism using the isolated RNA.

公开(公告)号：US20180142241A1

公开(公告)日：2018-05-24

申请号：US15849887

申请日：2017-12-21

申请人： CITY OF HOPE ,  INTEGRATED DNA TECHNOLOGIES, INC.

发明人： John J. Rossi ,  Mark Behlke ,  Dongho Kim

IPC分类号： C12N15/113 ,  C12N15/11

CPC分类号： C12N15/113 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/33 ,  C12N2310/53 ,  C12N2310/533 ,  C12N2320/35 ,  C12N2320/51

摘要： The invention is directed to compositions and methods for selectively reducing the expression of a gene product from a desired target gene in a cell, as well as for treating diseases caused by the expression of the gene. More particularly, the invention is directed to compositions that contain double stranded RNA (“dsRNA”), and methods for preparing them, that are capable of reducing the expression of target genes in eukaryotic cells. The dsRNA has a first oligonucleotide sequence that is between 25 and about 30 nucleotides in length and a second oligonucleotide sequence that anneals to the first sequence under biological conditions. In addition, a region of one of the sequences of the dsRNA having a sequence length of at least 19 nucleotides is sufficiently complementary to a nucleotide sequence of the RNA produced from the target gene to trigger the destruction of the target RNA by the RNAi machinery.

公开(公告)号：US09976139B2

公开(公告)日：2018-05-22

申请号：US14664659

申请日：2015-03-20

申请人： MONSANTO TECHNOLOGY LLC

发明人： Shihshieh Huang ,  Thomas M. Malvar ,  Michael H. Luethy

IPC分类号： C12N15/113 ,  C12N15/82 ,  C07H21/02 ,  C12N9/06 ,  C12N15/11 ,  A61K48/00

CPC分类号： C12N15/113 ,  A61K48/00 ,  C07H21/02 ,  C12N9/0028 ,  C12N15/111 ,  C12N15/8218 ,  C12N15/8251 ,  C12N15/8254 ,  C12N2310/11 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/53 ,  C12N2330/30

摘要： Anti-sense-oriented RNA gene suppression agents in the form of a loop of anti-sense-oriented RNA is produced in cells of transgenic organisms, e.g. plants, by transcription from a recombinant DNA construct which comprises in 5′ to 3′ order a promoter element operably linked to an anti-sense-oriented DNA element and a complementary DNA element.