JP2020533334A5 - - Google Patents

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Publication number
JP2020533334A5
JP2020533334A5 JP2020514257A JP2020514257A JP2020533334A5 JP 2020533334 A5 JP2020533334 A5 JP 2020533334A5 JP 2020514257 A JP2020514257 A JP 2020514257A JP 2020514257 A JP2020514257 A JP 2020514257A JP 2020533334 A5 JP2020533334 A5 JP 2020533334A5
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Japan
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rnai agent
agent according
nucleotides
composition
sense strand
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JP2020514257A
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English (en)
Japanese (ja)
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JP7438103B2 (ja
JP2020533334A (ja
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Priority claimed from PCT/US2018/050248 external-priority patent/WO2019051402A1/en
Publication of JP2020533334A publication Critical patent/JP2020533334A/ja
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Priority to JP2023051640A priority Critical patent/JP2023073358A/ja
Application granted granted Critical
Publication of JP7438103B2 publication Critical patent/JP7438103B2/ja
Priority to JP2025285010A priority patent/JP2026042850A/ja
Priority to JP2025285009A priority patent/JP2026042849A/ja
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JP2020514257A 2017-09-11 2018-09-10 アポリポタンパク質Ｃ－ＩＩＩ（ＡＰＯＣ３）の発現を阻害するためのＲＮＡｉ剤および組成物 Active JP7438103B2 (ja)

Priority Applications (3)

Application Number Priority Date Filing Date Title
JP2023051640A JP2023073358A (ja) 2017-09-11 2023-03-28 アポリポタンパク質Ｃ－ＩＩＩ（ＡＰＯＣ３）の発現を阻害するためのＲＮＡｉ剤および組成物
JP2025285010A JP2026042850A (ja) 2017-09-11 2025-12-26 アポリポタンパク質Ｃ－ＩＩＩ（ＡＰＯＣ３）の発現を阻害するためのＲＮＡｉ剤および組成物
JP2025285009A JP2026042849A (ja) 2017-09-11 2025-12-26 アポリポタンパク質Ｃ－ＩＩＩ（ＡＰＯＣ３）の発現を阻害するためのＲＮＡｉ剤および組成物

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762556818P 2017-09-11 2017-09-11
US62/556,818 2017-09-11
US201862643927P 2018-03-16 2018-03-16
US62/643,927 2018-03-16
US201862720434P 2018-08-21 2018-08-21
US62/720,434 2018-08-21
PCT/US2018/050248 WO2019051402A1 (en) 2017-09-11 2018-09-10 RNA INTERFERENCE AGENTS AND COMPOSITIONS FOR INHIBITING THE EXPRESSION OF APOLIPOPROTEIN C-III (APOC3)

Related Child Applications (1)

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JP2023051640A Division JP2023073358A (ja) 2017-09-11 2023-03-28 アポリポタンパク質Ｃ－ＩＩＩ（ＡＰＯＣ３）の発現を阻害するためのＲＮＡｉ剤および組成物

Publications (3)

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JP2020533334A JP2020533334A (ja) 2020-11-19
JP2020533334A5 true JP2020533334A5 (enExample) 2021-10-07
JP7438103B2 JP7438103B2 (ja) 2024-02-26

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JP2020514257A Active JP7438103B2 (ja) 2017-09-11 2018-09-10 アポリポタンパク質Ｃ－ＩＩＩ（ＡＰＯＣ３）の発現を阻害するためのＲＮＡｉ剤および組成物
JP2023051640A Withdrawn JP2023073358A (ja) 2017-09-11 2023-03-28 アポリポタンパク質Ｃ－ＩＩＩ（ＡＰＯＣ３）の発現を阻害するためのＲＮＡｉ剤および組成物
JP2025285009A Pending JP2026042849A (ja) 2017-09-11 2025-12-26 アポリポタンパク質Ｃ－ＩＩＩ（ＡＰＯＣ３）の発現を阻害するためのＲＮＡｉ剤および組成物
JP2025285010A Pending JP2026042850A (ja) 2017-09-11 2025-12-26 アポリポタンパク質Ｃ－ＩＩＩ（ＡＰＯＣ３）の発現を阻害するためのＲＮＡｉ剤および組成物

Family Applications After (3)

Application Number Title Priority Date Filing Date
JP2023051640A Withdrawn JP2023073358A (ja) 2017-09-11 2023-03-28 アポリポタンパク質Ｃ－ＩＩＩ（ＡＰＯＣ３）の発現を阻害するためのＲＮＡｉ剤および組成物
JP2025285009A Pending JP2026042849A (ja) 2017-09-11 2025-12-26 アポリポタンパク質Ｃ－ＩＩＩ（ＡＰＯＣ３）の発現を阻害するためのＲＮＡｉ剤および組成物
JP2025285010A Pending JP2026042850A (ja) 2017-09-11 2025-12-26 アポリポタンパク質Ｃ－ＩＩＩ（ＡＰＯＣ３）の発現を阻害するためのＲＮＡｉ剤および組成物

Country Status (26)

Country Link
US (5) US10597657B2 (enExample)
EP (1) EP3681516A4 (enExample)
JP (4) JP7438103B2 (enExample)
KR (2) KR102723833B1 (enExample)
CN (2) CN111107853B (enExample)
AU (2) AU2018329190B2 (enExample)
BR (1) BR112020002413A2 (enExample)
CA (1) CA3074303A1 (enExample)
CL (2) CL2020000593A1 (enExample)
CO (1) CO2020002586A2 (enExample)
CR (1) CR20200108A (enExample)
EC (1) ECSP20017905A (enExample)
IL (2) IL273172B2 (enExample)
JO (1) JOP20200054A1 (enExample)
MX (2) MX2020002648A (enExample)
MY (1) MY202025A (enExample)
NZ (1) NZ763172A (enExample)
PE (1) PE20201283A1 (enExample)
PH (1) PH12020500332A1 (enExample)
SG (1) SG11201912178VA (enExample)
TN (1) TN2020000039A1 (enExample)
TW (2) TWI811238B (enExample)
UA (1) UA130735C2 (enExample)
UY (1) UY37871A (enExample)
WO (1) WO2019051402A1 (enExample)
ZA (1) ZA202100806B (enExample)

Families Citing this family (45)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2968114A1 (en) 2014-11-17 2016-05-26 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
JP6989521B2 (ja) * 2016-09-02 2022-01-05 アローヘッド ファーマシューティカルズ インコーポレイテッド 標的化リガンド
RS63836B1 (sr) 2017-04-05 2023-01-31 Silence Therapeutics Gmbh Proizvodi i sastavi
TN2020000039A1 (en) * 2017-09-11 2021-10-04 Arrowhead Pharmaceuticals Inc Rnai agents and compositions for inhibiting expression of apolipoprotein c-iii (apoc3)
US11660347B2 (en) 2017-12-01 2023-05-30 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing same, preparation method, and use thereof
CN110997919B (zh) 2017-12-01 2024-04-02 苏州瑞博生物技术股份有限公司 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途
JP7261494B2 (ja) 2017-12-01 2023-04-20 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用
CN112423794A (zh) * 2018-12-28 2021-02-26 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
JP7663951B2 (ja) 2019-05-22 2025-04-17 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、薬物組成物及び複合体ならびに調製方法と使用
AU2020280439B2 (en) 2019-05-22 2025-08-14 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
CN113811613B (zh) 2019-05-22 2024-04-05 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
WO2020238766A1 (zh) 2019-05-24 2020-12-03 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
JP7829478B2 (ja) * 2019-08-05 2026-03-13 アローヘッド ファーマシューティカルズ インコーポレイテッド Ａｐｏｃ３関連疾患および障害の処置のための方法
WO2021167841A1 (en) 2020-02-18 2021-08-26 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
CN114480382A (zh) * 2020-11-12 2022-05-13 北京大学 用于抑制SURF4基因表达的RNAi试剂和组合物
JP2024504503A (ja) * 2021-01-30 2024-01-31 イー セラピューティクス パブリック リミテッド カンパニー 脱塩基ヌクレオチドを含有する核酸
WO2022162155A1 (en) * 2021-01-30 2022-08-04 E-Therapeutics Plc Nucleic acids containing abasic nucleotides
AU2022299514A1 (en) * 2021-06-24 2024-01-18 Sirnaomics, Inc. Products and compositions
WO2023284559A1 (zh) * 2021-07-16 2023-01-19 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
WO2023059948A1 (en) * 2021-10-08 2023-04-13 E-Therapeutics Plc Nucleic acids containing abasic nucleosides
WO2023088227A1 (zh) 2021-11-16 2023-05-25 上海舶望制药有限公司 抑制血管紧张素原(agt)蛋白表达的组合物和方法
EP4441218A2 (en) 2021-12-01 2024-10-09 Dicerna Pharmaceuticals, Inc. Compositions and methods for modulating apoc3 expression
EP4467645A4 (en) * 2022-01-20 2026-01-21 Tuojie Biotech Shanghai Co Ltd DOUBLE-STRANDED RNA, ITS USES AND PREPARATION PROCESS
TW202345865A (zh) * 2022-01-24 2023-12-01 大陸商上海舶望製藥有限公司 抑制LPA(Apo(a))蛋白表達的組合物和方法
US20250161461A1 (en) 2022-01-30 2025-05-22 Rona Bioscience, Limited Targeting ligand containing n-acetylgalactosamine
JP2025520520A (ja) 2022-06-14 2025-07-03 大睿生物 ＰＣＳＫ９遺伝子活性を調節するｓｉＲＮＡ分子
CN119421952A (zh) * 2022-06-27 2025-02-11 大睿生物医药科技(上海)有限公司 抑制载脂蛋白C3表达的siRNA
WO2024015796A1 (en) * 2022-07-11 2024-01-18 Sanegene Bio Usa Inc. Optimized 2'- modified ribose derivatives and methods of use
KR20250043473A (ko) * 2022-07-27 2025-03-28 이-테라퓨틱스 피엘씨 핵산 화합물
EP4562148A1 (en) * 2022-07-27 2025-06-04 E-Therapeutics plc Nucleic acid compounds
EP4562147A1 (en) * 2022-07-27 2025-06-04 E-Therapeutics plc Nucleic acid compounds
CN121271867A (zh) * 2022-08-05 2026-01-06 厦门甘宝利生物医药有限公司 一种抑制apoc3基因表达的rna抑制剂及其应用
CN119744305A (zh) * 2022-08-11 2025-04-01 益杰立科(上海)生物科技有限公司 一种表观编辑靶点的方法及用途
WO2024165568A2 (en) * 2023-02-06 2024-08-15 E-Therapeutics Plc Inhibitors of expression and/or function
WO2024165571A2 (en) * 2023-02-06 2024-08-15 E-Therapeutics Plc Inhibitors of expression and/or function
WO2024188173A1 (zh) * 2023-03-10 2024-09-19 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及其用途
CN121399258A (zh) 2023-07-06 2026-01-23 大睿生物医药科技(上海)有限公司 调控AGT表达的dsRNA分子
AU2024342933A1 (en) 2023-09-14 2026-03-26 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing apociii expression
CN120344664A (zh) * 2023-10-27 2025-07-18 北京安龙生物医药有限公司 靶向脂蛋白a的寡核苷酸及其用途
WO2025125576A2 (en) * 2023-12-15 2025-06-19 E-Therapeutics Plc Inhibitors of expression and/or function
EP4686757A1 (en) * 2024-07-31 2026-02-04 e-therapeutics PLC Inhibitors of expression and/or function
WO2025133348A1 (en) * 2023-12-22 2025-06-26 E-Therapeutics Plc Inhibitors of expression and/or function
WO2025184301A1 (en) * 2024-02-29 2025-09-04 Arrowhead Pharmaceuticals, Inc. Rnai agents for dual inhibition of expression of apolipoprotein c-iii (apoc3) and proprotein convertase subtilisin kexin 9 (pcsk9), compositions thereof, and methods of use
WO2025237176A1 (zh) * 2024-05-11 2025-11-20 深圳信立泰药业股份有限公司 用于抑制GPR75基因表达的RNAi剂及其制备方法和用途
KR20260032888A (ko) * 2024-08-30 2026-03-10 올릭스 주식회사 APOC3 유전자를 표적으로 하는 RNAi 제제 및 이의 용도

Family Cites Families (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
US5030453A (en) 1983-03-24 1991-07-09 The Liposome Company, Inc. Stable plurilamellar vesicles
NL9201440A (nl) 1992-08-11 1994-03-01 Univ Leiden Triantennaire clusterglycosiden, hun bereiding en toepassing.
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US6977244B2 (en) 1996-10-04 2005-12-20 Board Of Regents, The University Of Texas Systems Inhibition of Bcl-2 protein expression by liposomal antisense oligodeoxynucleotides
US5891467A (en) 1997-01-31 1999-04-06 Depotech Corporation Method for utilizing neutral lipids to modify in vivo release from multivesicular liposomes
WO2000044914A1 (en) 1999-01-28 2000-08-03 Medical College Of Georgia Research Institute, Inc. Composition and method for in vivo and in vitro attenuation of gene expression using double stranded rna
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
JP2002537828A (ja) 1999-03-10 2002-11-12 フォゲン リミティド 細胞への物質のデリバリー
WO2001068836A2 (en) 2000-03-16 2001-09-20 Genetica, Inc. Methods and compositions for rna interference
US6680068B2 (en) 2000-07-06 2004-01-20 The General Hospital Corporation Drug delivery formulations and targeting
KR100872437B1 (ko) 2000-12-01 2008-12-05 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. Ｒｎａ 간섭을 매개하는 작은 ｒｎａ 분자
WO2002100435A1 (en) 2001-06-11 2002-12-19 Centre Hospitalier Universitaire De Montreal Compositions and methods for enhancing nucleic acid transfer into cells
DE60236520D1 (de) 2001-08-16 2010-07-08 Univ Pennsylvania Synthese und verwendung von reagenzien für die verbesserte dna-lipofektion und/oder prodrug- und arzneimitteltherapien mit langsamer freisetzung
EP1519714B1 (en) 2002-06-28 2010-10-20 Protiva Biotherapeutics Inc. Method and apparatus for producing liposomes
JP2006512906A (ja) 2002-09-28 2006-04-20 マサチューセッツ インスティチュート オブ テクノロジー インフルエンザ治療剤
JP2006507841A (ja) 2002-11-14 2006-03-09 ダーマコン， インコーポレイテッド 機能的ｓｉＲＮＡおよび超機能的ｓｉＲＮＡ
ATE465255T1 (de) 2002-11-26 2010-05-15 Univ Massachusetts Verabreichung von sirnas
WO2004064731A2 (en) 2003-01-14 2004-08-05 University Of Washington Lipid-drug formulations and methods for targeted delivery of lipid-drug complexes to lymlplhoid tissues
US7598227B2 (en) * 2003-04-16 2009-10-06 Isis Pharmaceuticals Inc. Modulation of apolipoprotein C-III expression
SG190613A1 (en) 2003-07-16 2013-06-28 Protiva Biotherapeutics Inc Lipid encapsulated interfering rna
CA2536333C (en) 2003-08-28 2013-01-08 Jan Weiler Interfering rna duplex having blunt-ends and 3'-modifications
EP1752536A4 (en) 2004-05-11 2008-04-16 Alphagen Co Ltd POLYNUCLEOTIDE CAUSING RNA INTERFERENCE AND METHOD OF REGULATING GENE EXPRESSION WITH THE USE OF THE SAME
WO2007051303A1 (en) 2005-11-02 2007-05-10 Protiva Biotherapeutics, Inc. MODIFIED siRNA MOLECULES AND USES THEREOF
AU2007229161B2 (en) 2006-03-23 2012-07-12 Roche Innovation Center Copenhagen A/S Small internally segmented interfering RNA
GB0608838D0 (en) 2006-05-04 2006-06-14 Novartis Ag Organic compounds
BRPI0715375A2 (pt) 2006-08-18 2013-06-18 Hoffmann La Roche policonjugados para distribuiÇço in vivo de polinucleotÍdeos
BRPI0811170B8 (pt) 2007-05-22 2021-05-25 Arcturus Therapeutics Inc oligonucleotídeos de rna e complexos de rna substituídos por hidroximetila
US7615374B2 (en) 2007-09-25 2009-11-10 Wisconsin Alumni Research Foundation Generation of clonal mesenchymal progenitors and mesenchymal stem cell lines under serum-free conditions
JP5697988B2 (ja) 2007-12-27 2015-04-08 プロチバ バイオセラピューティクス インコーポレイティッド 干渉ｒｎａを使用したポロ様キナーゼ発現のサイレンシング方法
US8188060B2 (en) 2008-02-11 2012-05-29 Dharmacon, Inc. Duplex oligonucleotides with enhanced functionality in gene regulation
WO2010083615A1 (en) * 2009-01-26 2010-07-29 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein c-iii expression
AU2010334911A1 (en) 2009-12-23 2012-07-12 Novartis Ag Lipids, lipid compositions, and methods of using them
EP3023495B1 (en) 2010-02-24 2019-05-08 Arrowhead Pharmaceuticals, Inc. Compositions for targeted delivery of sirna
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
KR20230084331A (ko) 2011-06-21 2023-06-12 알닐람 파마슈티칼스 인코포레이티드 아포리포단백질 c-iii(apoc3) 유전자의 발현 억제를 위한 조성물 및 방법
KR20140051357A (ko) 2011-08-26 2014-04-30 애로우헤드 리서치 코오포레이션 Ｉｎ Ｖｉｖｏ 핵산 전달용 폴리(비닐 에스테르) 고분자
AU2012377385A1 (en) 2012-04-18 2014-01-23 Arrowhead Research Corporation Poly(acrylate) polymers for in vivo nucleic acid delivery
EP3358013B1 (en) * 2012-05-02 2020-06-24 Sirna Therapeutics, Inc. Short interfering nucleic acid (sina) compositions
JP6694382B2 (ja) * 2013-06-21 2020-05-13 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 標的核酸を調節するための組成物および方法
EP3052627B1 (en) 2013-10-04 2018-08-22 Novartis AG Novel formats for organic compounds for use in rna interference
EP3736334A1 (en) * 2014-07-16 2020-11-11 Arrowhead Pharmaceuticals, Inc. Rnai compositions to treat apoc3-related diseases
CA2968114A1 (en) * 2014-11-17 2016-05-26 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
WO2016149020A1 (en) * 2015-03-17 2016-09-22 Arrowhead Research Corporation Rna interference agents
JOP20160211B1 (ar) * 2015-10-01 2021-08-17 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
US10246709B2 (en) 2016-03-07 2019-04-02 Arrowhead Pharmaceuticals, Inc. Targeting ligands for therapeutic compounds
JP6989521B2 (ja) * 2016-09-02 2022-01-05 アローヘッド ファーマシューティカルズ インコーポレイテッド 標的化リガンド
CN111050806A (zh) * 2017-06-02 2020-04-21 波涛生命科学有限公司 寡核苷酸组合物及其使用方法
TN2020000039A1 (en) * 2017-09-11 2021-10-04 Arrowhead Pharmaceuticals Inc Rnai agents and compositions for inhibiting expression of apolipoprotein c-iii (apoc3)

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