CN107743522B - 类血管生成素3(ANGPTL3)iRNA组合物及其使用方法 - Google Patents
类血管生成素3(ANGPTL3)iRNA组合物及其使用方法 Download PDFInfo
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- CN107743522B CN107743522B CN201680034444.4A CN201680034444A CN107743522B CN 107743522 B CN107743522 B CN 107743522B CN 201680034444 A CN201680034444 A CN 201680034444A CN 107743522 B CN107743522 B CN 107743522B
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| CN202211369371.8A CN115927335A (zh) | 2015-04-13 | 2016-04-13 | 类血管生成素3(ANGPTL3)iRNA组合物及其使用方法 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562146604P | 2015-04-13 | 2015-04-13 | |
| US62/146,604 | 2015-04-13 | ||
| US201562261361P | 2015-12-01 | 2015-12-01 | |
| US62/261,361 | 2015-12-01 | ||
| PCT/US2016/027271 WO2016168286A1 (en) | 2015-04-13 | 2016-04-13 | Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202211369371.8A Division CN115927335A (zh) | 2015-04-13 | 2016-04-13 | 类血管生成素3(ANGPTL3)iRNA组合物及其使用方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CN107743522A CN107743522A (zh) | 2018-02-27 |
| CN107743522B true CN107743522B (zh) | 2022-11-11 |
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Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201680034444.4A Active CN107743522B (zh) | 2015-04-13 | 2016-04-13 | 类血管生成素3(ANGPTL3)iRNA组合物及其使用方法 |
| CN202211369371.8A Pending CN115927335A (zh) | 2015-04-13 | 2016-04-13 | 类血管生成素3(ANGPTL3)iRNA组合物及其使用方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN202211369371.8A Pending CN115927335A (zh) | 2015-04-13 | 2016-04-13 | 类血管生成素3(ANGPTL3)iRNA组合物及其使用方法 |
Country Status (13)
| Country | Link |
|---|---|
| US (4) | US10570393B2 (enExample) |
| EP (1) | EP3283631A1 (enExample) |
| JP (3) | JP6975641B2 (enExample) |
| KR (2) | KR102749152B1 (enExample) |
| CN (2) | CN107743522B (enExample) |
| AU (2) | AU2016247922B2 (enExample) |
| BR (1) | BR112017021053A2 (enExample) |
| CA (1) | CA2982450A1 (enExample) |
| EA (1) | EA201792263A1 (enExample) |
| IL (2) | IL295159A (enExample) |
| MX (2) | MX392757B (enExample) |
| TW (3) | TWI862799B (enExample) |
| WO (1) | WO2016168286A1 (enExample) |
Families Citing this family (36)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3444348A1 (en) | 2011-06-21 | 2019-02-20 | Alnylam Pharmaceuticals, Inc. | Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof |
| JP6975641B2 (ja) | 2015-04-13 | 2021-12-01 | アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. | アンギオポエチン様3(ANGPTL3)iRNA組成物およびそれらの使用方法 |
| SG11201912188RA (en) * | 2017-09-14 | 2020-01-30 | Arrowhead Pharmaceuticals Inc | Rnai agents and compositions for inhibiting expression of angiopoietin-like 3 (angptl3), and methods of use |
| CN110997917B (zh) | 2017-12-01 | 2024-04-09 | 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 |
| EP3718572B1 (en) | 2017-12-01 | 2024-07-31 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate containing nucleic acid, preparation method and use |
| EP4455285A3 (en) | 2017-12-01 | 2025-02-26 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof |
| CN118291457A (zh) | 2017-12-01 | 2024-07-05 | 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 |
| AU2018374219C1 (en) | 2017-12-01 | 2023-05-11 | Suzhou Ribo Life Science Co., Ltd. | Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method therefor and use thereof |
| KR102617947B1 (ko) | 2017-12-29 | 2023-12-27 | 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 | 접합체와 제조 및 그 용도 |
| TWI851574B (zh) * | 2018-05-14 | 2024-08-11 | 美商阿尼拉製藥公司 | 血管收縮素原(AGT)iRNA組成物及其使用方法 |
| WO2020038377A1 (zh) | 2018-08-21 | 2020-02-27 | 苏州瑞博生物技术有限公司 | 一种核酸、含有该核酸的药物组合物和缀合物及其用途 |
| CN111655297A (zh) | 2018-09-30 | 2020-09-11 | 苏州瑞博生物技术有限公司 | 一种siRNA缀合物及其制备方法和用途 |
| US20210388356A1 (en) * | 2018-11-09 | 2021-12-16 | Alnylam Pharmaceuticals, Inc. | Modified double stranded oligonucleotide |
| WO2020099482A2 (en) * | 2018-11-13 | 2020-05-22 | Lipigon Pharmaceuticals Ab | Angptl 3 oligonucleotides influencing the regulation of the fatty acid metabolism |
| EP3903830A4 (en) | 2018-12-28 | 2023-04-26 | Suzhou Ribo Life Science Co., Ltd. | NUCLEIC ACID, COMPOSITION AND CONJUGATE WITH NUCLEIC ACID, PROCESS FOR THEIR PREPARATION AND ITS USE |
| CN113227376B (zh) | 2019-05-22 | 2024-04-09 | 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 |
| CN119607006A (zh) * | 2019-12-09 | 2025-03-14 | 艾姆皮瑞克公司 | 用于治疗血管生成素样4(angptl4)相关疾病的寡核苷酸 |
| GB2627637B (en) | 2020-03-04 | 2024-11-20 | Verve Therapeutics Inc | A method for reducing the risk of coronary disease |
| WO2021204872A1 (en) * | 2020-04-07 | 2021-10-14 | Uniqure Ip B.V. | Gene constructs for silencing angiopoietin-like 3 (angptl3) and uses thereof |
| CN120384079A (zh) * | 2020-09-30 | 2025-07-29 | 亿腾医药(苏州)有限公司 | 血管生成素样3的siRNA及其用途 |
| EP4229200A1 (en) * | 2020-10-16 | 2023-08-23 | Sanofi | Novel rna compositions and methods for inhibiting angptl3 |
| CN114685585B (zh) * | 2020-12-31 | 2024-06-04 | 苏州瑞博生物技术股份有限公司 | 核苷酸序列、双链寡核苷酸、药物组合物与缀合物及制备方法和用途 |
| AU2022231003A1 (en) | 2021-03-04 | 2023-09-14 | Alnylam Pharmaceuticals, Inc. | Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof |
| WO2022204430A1 (en) * | 2021-03-24 | 2022-09-29 | Atalanta Therapeutics, Inc. | Double-stranded sirna having patterned chemical modifications |
| JP2024535852A (ja) * | 2021-09-17 | 2024-10-02 | アルナイラム ファーマシューティカルズ, インコーポレイテッド | ANGPTL4をサイレンシングするためのiRNA組成物および方法 |
| TWI877520B (zh) * | 2021-09-23 | 2025-03-21 | 大陸商上海舶望製藥有限公司 | 用於抑制血管生成素樣3(angptl3)蛋白表達的組合物和方法 |
| US20230159930A1 (en) * | 2021-11-19 | 2023-05-25 | Sanegene Bio Usa Inc. | Double stranded rna targeting angiopoietin-like 3 (angptl-3) and methods of use thereof |
| CA3241896A1 (en) | 2021-12-22 | 2023-06-29 | Regeneron Pharmaceuticals, Inc. | Treatment of kidney diseases with angiopoietin like 3 (angptl3) inhibitors |
| WO2023134705A1 (zh) * | 2022-01-11 | 2023-07-20 | 上海金中锘美生物医药科技有限公司 | 抑制angptl3表达的rna干扰剂及其用途 |
| CN116987696A (zh) * | 2022-08-04 | 2023-11-03 | 北京福元医药股份有限公司 | 用于抑制angptl3基因表达的双链核糖核酸及其修饰物、缀合物和用途 |
| WO2024032608A1 (zh) * | 2022-08-08 | 2024-02-15 | 大睿生物医药科技(上海)有限公司 | 调控ANGPTL3基因活性的siRNA分子 |
| KR20250099703A (ko) | 2022-11-10 | 2025-07-02 | 리제너론 파마슈티칼스 인코포레이티드 | 안지오포이에틴 유사 3(angptl3) 억제제 및 용질 운반체 패밀리 5 구성원 2(slc5a2) 억제제의 조합을 이용한 신장 질환의 치료 |
| WO2024227059A2 (en) * | 2023-04-27 | 2024-10-31 | Aligos Therapeutics, Inc. | Short interfering nucleic acid (sina) molecules targeting angptl 3 and angptl 8 and methods of using the same |
| TW202530409A (zh) * | 2023-10-07 | 2025-08-01 | 大陸商齊魯製藥有限公司 | iRNA組合物及其使用方法 |
| WO2025169133A1 (en) | 2024-02-09 | 2025-08-14 | Astrazeneca Ab | Double-stranded rnai agents and compositions for reducing expression of angiopoietin-like 3 (angptl3) and methods of use thereof |
| WO2025201277A1 (en) * | 2024-03-25 | 2025-10-02 | Shanghai Argo Biopharmaceutical Co., Ltd. | Method for treating dyslipidemia with angiopoietin-like 3 (angptl3) -targeted rnai agents |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2011085271A3 (en) * | 2010-01-08 | 2011-09-09 | Isis Pharmaceuticals, Inc. | Modulation of angiopoietin-like 3 expression |
| CN103890000A (zh) * | 2011-06-21 | 2014-06-25 | 阿尔尼拉姆医药品有限公司 | 血管生成素样3(ANGPTL3)iRNA组合物及其使用方法 |
| CN104105790A (zh) * | 2011-11-18 | 2014-10-15 | 阿尔尼拉姆医药品有限公司 | 修饰的RNAi试剂 |
Family Cites Families (49)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5783701A (en) | 1992-09-08 | 1998-07-21 | Vertex Pharmaceuticals, Incorporated | Sulfonamide inhibitors of aspartyl protease |
| ES2142934T3 (es) | 1993-02-19 | 2000-05-01 | Nippon Shinyaku Co Ltd | Derivado del glicerol, dispositivo y composicion farmaceutica. |
| US6191105B1 (en) | 1993-05-10 | 2001-02-20 | Protein Delivery, Inc. | Hydrophilic and lipophilic balanced microemulsion formulations of free-form and/or conjugation-stabilized therapeutic agents such as insulin |
| US6054299A (en) | 1994-04-29 | 2000-04-25 | Conrad; Charles A. | Stem-loop cloning vector and method |
| US5858401A (en) | 1996-01-22 | 1999-01-12 | Sidmak Laboratories, Inc. | Pharmaceutical composition for cyclosporines |
| US5994316A (en) | 1996-02-21 | 1999-11-30 | The Immune Response Corporation | Method of preparing polynucleotide-carrier complexes for delivery to cells |
| EP2253639A1 (en) | 1997-09-12 | 2010-11-24 | Exiqon A/S | Bi- and tri-cyclic nucleoside, nucleotide and oligonucleoide analogues |
| AU2092599A (en) | 1997-12-24 | 1999-07-19 | Vertex Pharmaceuticals Incorporated | Prodrugs of aspartyl protease inhibitors |
| US6436989B1 (en) | 1997-12-24 | 2002-08-20 | Vertex Pharmaceuticals, Incorporated | Prodrugs of aspartyl protease inhibitors |
| BR9914773A (pt) | 1998-10-09 | 2002-02-05 | Ingene Inc | Conjunto de elementos genéricos, método para a produção de dna de cordão único, transcrição de mrna, construção de ácido nucléico, transcrição de ssdna, vetor, sistema vetor, célula hospedeira, conjunto para a produção de uma sequência de ácido nucléico de cordão único, método para a produção in vivo ou in vitro de uma sequência de ácido nucléico de cordão único, transcrição de cdna de cordão único, ácido nucléico inibidor, molécula heteroduplex, e composição farmacêutica |
| AU6430599A (en) | 1998-10-09 | 2000-05-01 | Cytogenix, Inc. | Enzymatic synthesis of ssdna |
| TWI260322B (en) | 1999-02-12 | 2006-08-21 | Vertex Pharma | Inhibitors of aspartyl protease |
| IT1318539B1 (it) | 2000-05-26 | 2003-08-27 | Italfarmaco Spa | Composizioni farmaceutiche a rilascio prolungato per lasomministrazione parenterale di sostanze idrofile biologicamente |
| CZ308053B6 (cs) | 2000-12-01 | 2019-11-27 | Max Planck Gesellschaft | Izolovaná molekula dvouřetězcové RNA, způsob její výroby a její použití |
| WO2003015698A2 (en) | 2001-08-13 | 2003-02-27 | University Of Pittsburgh | Application of lipid vehicles and use for drug delivery |
| US8090542B2 (en) | 2002-11-14 | 2012-01-03 | Dharmacon Inc. | Functional and hyperfunctional siRNA |
| JP2008537551A (ja) | 2005-03-31 | 2008-09-18 | カランド ファーマシューティカルズ, インコーポレイテッド | リボヌクレオチドレダクターゼサブユニット2の阻害剤およびその使用 |
| KR101503937B1 (ko) | 2006-12-08 | 2015-03-18 | 렉시컨 파마슈티컬스 인코퍼레이티드 | Angptl3 에 대한 단일클론 항체 |
| WO2009073809A2 (en) | 2007-12-04 | 2009-06-11 | Alnylam Pharmaceuticals, Inc. | Carbohydrate conjugates as delivery agents for oligonucleotides |
| AU2009241591A1 (en) | 2008-01-31 | 2009-11-05 | Alnylam Pharmaceuticals, Inc. | Optimized methods for delivery of DSRNA targeting the PCSK9 gene |
| WO2010036962A1 (en) | 2008-09-25 | 2010-04-01 | Alnylam Pharmaceuticals, Inc. | Lipid formulated compositions and methods for inhibiting expression of serum amyloid a gene |
| PL2937418T3 (pl) | 2008-10-20 | 2018-04-30 | Alnylam Pharmaceuticals, Inc. | Kompozycje i sposoby hamowania ekspresji transtyretyny |
| EP2373382B1 (en) | 2008-12-10 | 2017-02-15 | Alnylam Pharmaceuticals, Inc. | Gnaq targeted dsrna compositions and methods for inhibiting expression |
| EP2380108A4 (en) * | 2009-01-20 | 2013-01-23 | Videojet Technologies Inc | DATE PRINTING PROCESS |
| LT2440183T (lt) | 2009-06-10 | 2018-08-10 | Arbutus Biopharma Corporation | Patobulinta lipido kompozicija |
| MX2011013421A (es) | 2009-06-15 | 2012-03-16 | Alnylam Pharmaceuticals Inc | Arnds formulado con lipido de direccionamiento del gen pcsk9. |
| WO2011005861A1 (en) | 2009-07-07 | 2011-01-13 | Alnylam Pharmaceuticals, Inc. | Oligonucleotide end caps |
| JP5992911B2 (ja) * | 2010-09-21 | 2016-09-14 | ポピュレーション ジェネティクス テクノロジーズ リミテッド | 分子計数による対立遺伝子呼び出しの信頼度の増加 |
| EP3453762B1 (en) * | 2012-05-02 | 2021-04-21 | Sirna Therapeutics, Inc. | Short interfering nucleic acid (sina) compositions |
| EP2880162B1 (en) | 2012-08-03 | 2017-07-05 | Alnylam Pharmaceuticals, Inc. | Modified rnai agents |
| DK2879718T3 (da) * | 2012-08-06 | 2023-06-19 | Alnylam Pharmaceuticals Inc | Proces til fremstilling af kulhydratkonjugerede RNA-stoffer |
| EP2994167B1 (en) * | 2013-05-06 | 2020-05-06 | Alnylam Pharmaceuticals, Inc. | Dosages and methods for delivering lipid formulated nucleic acid molecules |
| US20170037409A1 (en) | 2013-12-24 | 2017-02-09 | Ionis Pharmaceuticals, Inc. | Modulation of angiopoietin-like 3 expression |
| AU2015252895B2 (en) * | 2014-05-01 | 2021-07-15 | Ionis Pharmaceuticals, Inc. | Compositions and methods for modulating angiopoietin-like 3 expression |
| IL316808A (en) * | 2014-08-20 | 2025-01-01 | Alnylam Pharmaceuticals Inc | Modified double-stranded RNA materials and their uses |
| US20180245077A1 (en) | 2015-03-20 | 2018-08-30 | Protiva Biotherapeutics, Inc. | Compositions and methods for treating hypertriglyceridemia |
| JP6975641B2 (ja) | 2015-04-13 | 2021-12-01 | アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. | アンギオポエチン様3(ANGPTL3)iRNA組成物およびそれらの使用方法 |
| SG11201912188RA (en) | 2017-09-14 | 2020-01-30 | Arrowhead Pharmaceuticals Inc | Rnai agents and compositions for inhibiting expression of angiopoietin-like 3 (angptl3), and methods of use |
| CN118291457A (zh) | 2017-12-01 | 2024-07-05 | 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 |
| UY38003A (es) | 2017-12-12 | 2019-06-28 | Amgen Inc | Construcciones de arni para inhibir la expresión de pnpla3 y métodos de uso de las mismas |
| CN113166761B (zh) | 2018-12-10 | 2025-09-09 | 美国安进公司 | 用于抑制pnpla3表达的rnai构建体 |
| CN114728017B (zh) | 2019-10-14 | 2025-10-28 | 阿斯利康(瑞典)有限公司 | Pnpla3表达的调节剂 |
| CA3172117A1 (en) | 2020-03-18 | 2021-09-23 | Bob D. Brown | Compositions and methods for inhibiting angptl3 expression |
| AU2021346205A1 (en) | 2020-09-17 | 2023-05-18 | Astrazeneca Ab | Pharmaceutical compositions comprising an antisense oligonucleotide for oral administration |
| AU2022231003A1 (en) | 2021-03-04 | 2023-09-14 | Alnylam Pharmaceuticals, Inc. | Angiopoietin-like 3 (angptl3) irna compositions and methods of use thereof |
| US20250333730A1 (en) | 2021-06-21 | 2025-10-30 | Shanghai Junshi Biosciences Co., Ltd. | Sirna for inhibiting angptl3 gene expression and use thereof |
| JP2025527670A (ja) | 2022-08-22 | 2025-08-22 | 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 | Angptl3関連疾患及び障害の治療方法 |
| WO2024164008A1 (en) | 2023-02-03 | 2024-08-08 | Sirnaomics, Inc. | Products and compositions |
| WO2025021831A1 (en) | 2023-07-24 | 2025-01-30 | Astrazeneca Ab | Multivalent cargo-carrying complexes and uses thereof |
-
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- 2023-06-20 US US18/337,495 patent/US12492401B2/en active Active
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Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2011085271A3 (en) * | 2010-01-08 | 2011-09-09 | Isis Pharmaceuticals, Inc. | Modulation of angiopoietin-like 3 expression |
| CN102753186A (zh) * | 2010-01-08 | 2012-10-24 | Isis制药公司 | 血管生成素样3表达的调节 |
| CN103890000A (zh) * | 2011-06-21 | 2014-06-25 | 阿尔尼拉姆医药品有限公司 | 血管生成素样3(ANGPTL3)iRNA组合物及其使用方法 |
| CN104105790A (zh) * | 2011-11-18 | 2014-10-15 | 阿尔尼拉姆医药品有限公司 | 修饰的RNAi试剂 |
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