KR20230013266A - 올리고뉴클레오티드 - Google Patents

올리고뉴클레오티드 Download PDF

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KR20230013266A
KR20230013266A KR1020227044467A KR20227044467A KR20230013266A KR 20230013266 A KR20230013266 A KR 20230013266A KR 1020227044467 A KR1020227044467 A KR 1020227044467A KR 20227044467 A KR20227044467 A KR 20227044467A KR 20230013266 A KR20230013266 A KR 20230013266A
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oligonucleotide
bases
modified
region
consecutive
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Korean (ko)
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마이클 폴 마리에 간티에
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허드슨 인스티튜트 오브 메디컬 리서치
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KR1020227044467A 2020-05-19 2021-05-19 올리고뉴클레오티드 Pending KR20230013266A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
AU2020901606 2020-05-19
AU2020901606A AU2020901606A0 (en) 2020-05-19 Oligonucleotides
PCT/AU2021/050469 WO2021232099A1 (en) 2020-05-19 2021-05-19 Oligonucleotides

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KR20230013266A true KR20230013266A (ko) 2023-01-26

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KR1020227044467A Pending KR20230013266A (ko) 2020-05-19 2021-05-19 올리고뉴클레오티드

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US (2) US20230348903A1 (https=)
EP (1) EP4153751A4 (https=)
JP (1) JP2023526090A (https=)
KR (1) KR20230013266A (https=)
CN (1) CN115916978A (https=)
AU (1) AU2021276730A1 (https=)
BR (1) BR112022023310A2 (https=)
CA (1) CA3171986A1 (https=)
MX (1) MX2022014495A (https=)
WO (1) WO2021232099A1 (https=)

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Publication number Priority date Publication date Assignee Title
CN115916978A (zh) 2020-05-19 2023-04-04 哈德森医学研究所 寡核苷酸
US20240384268A1 (en) * 2021-04-08 2024-11-21 Hudson Institute of Medical Research Oligonucleotides
CN118460654B (zh) * 2024-07-10 2025-02-14 凯莱英医药集团(天津)股份有限公司 一种Givosiran的制备方法
CN119592567B (zh) * 2024-12-10 2025-12-02 浙江大学 基于肿瘤剪接转换治疗的反义寡核苷酸及其应用

Family Cites Families (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6320041B1 (en) 2001-04-13 2001-11-20 Trilink Biotechnologies, Inc. Pre-activated carbonyl linkers for the modification of oligonucleotides
US8153141B2 (en) 2002-04-04 2012-04-10 Coley Pharmaceutical Gmbh Immunostimulatory G, U-containing oligoribonucleotides
US20050239733A1 (en) 2003-10-31 2005-10-27 Coley Pharmaceutical Gmbh Sequence requirements for inhibitory oligonucleotides
WO2005097993A2 (en) * 2004-02-19 2005-10-20 Coley Pharmaceutical Group, Inc. Immunostimulatory viral rna oligonucleotides
WO2006063152A2 (en) * 2004-12-08 2006-06-15 3M Innovative Properties Company Immunostimulatory combinations and methods
EP1819365B1 (en) 2004-12-09 2014-07-02 Alnylam Pharmaceuticals Inc. Compositions and methods for inducing an immune response in a mammal and methods of avoiding an immune response to oligonucleotide agents such as short interfering RNAs
CN101160401A (zh) * 2005-02-24 2008-04-09 科勒制药集团公司 免疫刺激寡核苷酸
EP1764107A1 (en) * 2005-09-14 2007-03-21 Gunther Hartmann Compositions comprising immunostimulatory RNA oligonucleotides and methods for producing said RNA oligonucleotides
US8076068B2 (en) 2005-09-14 2011-12-13 Gunther Hartmann Method for determining immunostimulatory activity of RNA oligonucleotides
KR20080059595A (ko) * 2005-09-27 2008-06-30 콜리 파마슈티칼 게엠베하 어댑터 올리고뉴클레오티드를 사용하는 tlr-매개 면역반응의 조절
PL1957647T3 (pl) * 2005-11-25 2015-07-31 Zoetis Belgium S A Oligorybonukleotydy immunostymulujące
CN101331229B (zh) * 2005-11-25 2012-08-29 科勒制药股份公司 免疫刺激性寡核糖核苷酸
EP2021008B1 (en) * 2006-04-07 2015-12-02 Idera Pharmaceuticals, Inc. Stabilized immune modulatory rna (simra) compounds for tlr7 and tlr8
AU2007268075C1 (en) 2006-06-01 2013-03-07 Trilink Biotechnologies Chemically modified oligonucleotide primers for nucleic acid amplification
AU2007280690C1 (en) * 2006-07-31 2012-08-23 Curevac Gmbh Nucleic acid of formula (I): GIXmGn, or (II): CIXmCn, in particular as an immune-stimulating agent/adjuvant
EP3342415B1 (en) * 2006-08-08 2022-01-26 Rheinische Friedrich-Wilhelms-Universität Bonn Structure and use of 5' phosphate oligonucleotides
WO2008019486A1 (en) 2006-08-16 2008-02-21 Protiva Biotherapeutics, Inc. Nucleic acid modulation of toll-like receptor-mediated immune stimulation
CA2692161C (en) * 2007-07-09 2015-09-29 Idera Pharmaceuticals, Inc. Stabilized immune modulatory rna (simra) compounds
JP5753382B2 (ja) * 2007-11-06 2015-07-22 アディウティーダ ファーマシューティカルズ ゲーエムベーハー 修飾されたオリゴリン酸基を含有する免疫賦活オリゴリボヌクレオチドアナログ
US20100047188A1 (en) * 2008-08-04 2010-02-25 Idera Pharmaceuticals, Inc. Modulation of toll-like receptor 8 expression by antisense oligonucleotides
US9631227B2 (en) 2009-07-06 2017-04-25 Trilink Biotechnologies, Inc. Chemically modified ligase cofactors, donors and acceptors
EP3191591A1 (en) * 2014-09-12 2017-07-19 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
WO2016205323A1 (en) * 2015-06-18 2016-12-22 Alnylam Pharmaceuticals, Inc. Polynucleotde agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
ES2964690T3 (es) 2015-09-21 2024-04-09 Trilink Biotechnologies Llc Método para sintetizar ARN con caperuza 5'
WO2017100587A1 (en) * 2015-12-09 2017-06-15 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting programmed cell death 1 ligand 1 (pd-l1) and methods of use thereof
WO2018184003A1 (en) 2017-03-31 2018-10-04 Dana-Farber Cancer Institute, Inc. Modulating dsrna editing, sensing, and metabolism to increase tumor immunity and improve the efficacy of cancer immunotherapy and/or modulators of intratumoral interferon
US11603532B2 (en) * 2017-06-02 2023-03-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
CN115916978A (zh) 2020-05-19 2023-04-04 哈德森医学研究所 寡核苷酸

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