JP6975641B2 - 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 - Google Patents

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 Download PDF

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JP6975641B2
JP6975641B2 JP2017553347A JP2017553347A JP6975641B2 JP 6975641 B2 JP6975641 B2 JP 6975641B2 JP 2017553347 A JP2017553347 A JP 2017553347A JP 2017553347 A JP2017553347 A JP 2017553347A JP 6975641 B2 JP6975641 B2 JP 6975641B2
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angptl3
dsrna
pat
irna
expression
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JP2018512155A5 (enExample
JP2018512155A (ja
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ウィリアム・クアーベス
ケビン・フィッツジェラルド
ジェイムズ・バトラー
ステファニー・ウィリアムズ
グレゴリー・ヒンクル
マルティン・マイアー
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Alnylam Pharmaceuticals Inc
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JP2017553347A 2015-04-13 2016-04-13 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 Active JP6975641B2 (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2021147698A JP2022002518A (ja) 2015-04-13 2021-09-10 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
JP2023169168A JP2024009838A (ja) 2015-04-13 2023-09-29 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

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

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JP2018512155A JP2018512155A (ja) 2018-05-17
JP2018512155A5 JP2018512155A5 (enExample) 2019-05-23
JP6975641B2 true JP6975641B2 (ja) 2021-12-01

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JP2021147698A Pending JP2022002518A (ja) 2015-04-13 2021-09-10 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
JP2023169168A Pending JP2024009838A (ja) 2015-04-13 2023-09-29 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

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JP2023169168A Pending JP2024009838A (ja) 2015-04-13 2023-09-29 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

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US (4) US10570393B2 (enExample)
EP (1) EP3283631A1 (enExample)
JP (3) JP6975641B2 (enExample)
KR (2) KR20250005546A (enExample)
CN (2) CN115927335A (enExample)
AU (2) AU2016247922B2 (enExample)
BR (1) BR112017021053A2 (enExample)
CA (1) CA2982450A1 (enExample)
EA (1) EA201792263A1 (enExample)
IL (2) IL254786B (enExample)
MX (2) MX392757B (enExample)
TW (3) TW202507005A (enExample)
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Families Citing this family (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6110372B2 (ja) 2011-06-21 2017-04-05 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． アンジオポエチン様３（ＡＮＧＰＴＬ３）ｉＲＮＡ組成物及びその使用方法
TW202507005A (zh) 2015-04-13 2025-02-16 美商阿尼拉製藥公司 類血管生成素３（ＡＮＧＰＴＬ３）ｉＲＮＡ組成物及其用途方法
WO2019055633A1 (en) * 2017-09-14 2019-03-21 Arrowhead Pharmaceuticals, Inc. RNAI AGENTS AND COMPOSITIONS FOR INHIBITING ANALOGUE EXPRESSION OF ANGIOPOIETIN 3 (ANGPTL3) AND METHODS OF USE
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
US11414661B2 (en) 2017-12-01 2022-08-16 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
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
JP7365052B2 (ja) 2017-12-01 2023-10-19 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
KR102834361B1 (ko) 2017-12-01 2025-07-17 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 핵산, 이를 포함하는 조성물과 컨쥬게이트, 및 그의 제조 방법과 용도
EP3732185B1 (en) 2017-12-29 2025-02-26 Suzhou Ribo Life Science Co., Ltd. Conjugates and preparation and use thereof
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
EP3842534A4 (en) 2018-08-21 2022-07-06 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, COMPOSITION AND CONJUGATE CONTAINING NUCLEIC ACID AND METHOD OF USE THEREOF
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
AU2019376628B2 (en) * 2018-11-09 2025-08-07 Alnylam Pharmaceuticals, Inc. Modified double stranded oligonucleotides
WO2020099482A2 (en) * 2018-11-13 2020-05-22 Lipigon Pharmaceuticals Ab Angptl 3 oligonucleotides influencing the regulation of the fatty acid metabolism
KR20210110839A (ko) 2018-12-28 2021-09-09 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 핵산, 핵산을 함유하는 조성물 및 접합체, 이의 제조 방법 및 용도
CA3139195A1 (en) 2019-05-22 2020-11-26 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
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
IL296133A (en) * 2020-03-04 2022-11-01 Verve Therapeutics Inc Preparations and methods for administering targeted RNA
JP2023521347A (ja) * 2020-04-07 2023-05-24 ユニキュアー アイピー ビー．ブイ． アンジオポエチン様３（ａｎｇｐｔｌ３）をサイレンシングするための遺伝子構築物及びその使用
WO2022068923A1 (zh) * 2020-09-30 2022-04-07 纳肽得(青岛)生物医药有限公司 血管生成素样3(ANGPTL3)的siRNA及其用途
WO2022079222A1 (en) * 2020-10-16 2022-04-21 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
MX2023010249A (es) 2021-03-04 2023-09-15 Alnylam Pharmaceuticals Inc Composiciones de arni de proteina similar a la angiopoyetina 3 (angptl3) y metodos para usarlas.
US20240182892A1 (en) * 2021-03-24 2024-06-06 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
IL310929A (en) * 2021-09-23 2024-04-01 Shanghai Argo Biopharmaceutical Co Ltd Preparations and methods for inhibiting expression of angiopoietin-like protein 3
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
IL313191A (en) 2021-12-22 2024-07-01 Regeneron Pharma Treatment of kidney diseases using angiopoietin-like inhibitors 3
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
WO2024027518A1 (zh) * 2022-08-04 2024-02-08 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
CN119585431A (zh) * 2022-08-08 2025-03-07 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
CN120265775A (zh) 2022-11-10 2025-07-04 瑞泽恩制药公司 用血管生成素样3(angptl3)抑制剂和溶质载体家族5成员2(slc5a2)抑制剂的组合治疗肾脏疾病
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
WO2025073276A1 (zh) * 2023-10-07 2025-04-10 齐鲁制药有限公司 一种iRNA组合物及其使用方法
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

Family Cites Families (52)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5783701A (en) 1992-09-08 1998-07-21 Vertex Pharmaceuticals, Incorporated Sulfonamide inhibitors of aspartyl protease
DE69424406T2 (de) 1993-02-19 2000-10-26 Nippon Shinyaku Co., Ltd. Arzneistoffzusammensetzung, die ein nukleinsäurecopolymer enthält
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
WO2000022114A1 (en) 1998-10-09 2000-04-20 Ingene, Inc. PRODUCTION OF ssDNA $i(IN VIVO)
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
WO1999014226A2 (en) 1997-09-12 1999-03-25 Exiqon A/S Bi- and tri-cyclic nucleoside, nucleotide and oligonucleotide analogues
ID25551A (id) 1997-12-24 2000-10-12 Vertex Pharma Bahan baku obat penghambat protease aspartil
US6436989B1 (en) 1997-12-24 2002-08-20 Vertex Pharmaceuticals, Incorporated Prodrugs of aspartyl protease inhibitors
KR20010099682A (ko) 1998-10-09 2001-11-09 추후보충 단일가닥 ｄｎａ의 효소 합성
US6319946B1 (en) 1999-02-12 2001-11-20 Vertex Pharmaceuticals Incorporated 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
CZ302719B6 (cs) 2000-12-01 2011-09-21 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Izolovaná molekula dvouretezcové RNA, zpusob její výroby a její použití
AU2002323151A1 (en) 2001-08-13 2003-03-03 University Of Pittsburgh Application of lipid vehicles and use for drug delivery
EP1560931B1 (en) 2002-11-14 2011-07-27 Dharmacon, Inc. Functional and hyperfunctional sirna
ATE541928T1 (de) 2005-03-31 2012-02-15 Calando Pharmaceuticals Inc Inhibitoren der untereinheit 2 der ribonukleotidreduktase und ihre verwendungen
MX2009006082A (es) 2006-12-08 2009-08-18 Lexicon Pharmaceuticals Inc Anticuerpos monoclonales contra la proteina 3 similar a angiopoyetina (angptl3).
EP4074344A1 (en) 2007-12-04 2022-10-19 Arbutus Biopharma Corporation Targeting lipids
JP2011511004A (ja) 2008-01-31 2011-04-07 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 ＰＣＳＫ９遺伝子を標的とするｄｓＲＮＡを送達するための最適化された方法
EP3584320A1 (en) 2008-09-25 2019-12-25 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of serum amyloid a gene
PT2937418T (pt) 2008-10-20 2018-01-23 Alnylam Pharmaceuticals Inc Composições e métodos de inibição da expressão de transtirretina
NZ593618A (en) 2008-12-10 2013-02-22 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
HRP20211619T1 (hr) 2009-06-10 2022-02-04 Arbutus Biopharma Corporation Poboljšana formulacija lipida
NZ597504A (en) 2009-06-15 2013-10-25 Alnylam Pharmaceuticals Inc Lipid formulated dsrna targeting the pcsk9 gene
US9512164B2 (en) 2009-07-07 2016-12-06 Alnylam Pharmaceuticals, Inc. Oligonucleotide end caps
CN111700901A (zh) 2010-01-08 2020-09-25 Ionis制药公司 血管生成素样3表达的调节
ES2690753T3 (es) * 2010-09-21 2018-11-22 Agilent Technologies, Inc. Aumento de la confianza en las identificaciones de alelos con el recuento molecular
JP6110372B2 (ja) 2011-06-21 2017-04-05 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． アンジオポエチン様３（ＡＮＧＰＴＬ３）ｉＲＮＡ組成物及びその使用方法
DK3366775T4 (da) 2011-11-18 2025-10-27 Alnylam Pharmaceuticals Inc Modificerede rnai-midler
EP3358013B1 (en) * 2012-05-02 2020-06-24 Sirna Therapeutics, Inc. Short interfering nucleic acid (sina) compositions
WO2014022739A2 (en) 2012-08-03 2014-02-06 Alnylam Pharmaceuticals, Inc. Modified rnai agents
CA2879693A1 (en) 2012-08-06 2014-02-13 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugated rna agents and process for their preparation
EP2994167B1 (en) * 2013-05-06 2020-05-06 Alnylam Pharmaceuticals, Inc. Dosages and methods for delivering lipid formulated nucleic acid molecules
KR102298476B1 (ko) 2013-12-24 2021-09-03 아이오니스 파마수티컬즈, 인코포레이티드 안지오포이에틴-유사 3 발현의 조절
EP3137605B1 (en) * 2014-05-01 2020-10-28 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
CA2979998A1 (en) * 2015-03-20 2016-09-29 Protiva Biotherapeutics, Inc. Compositions and methods for treating hypertriglyceridemia
TW202507005A (zh) 2015-04-13 2025-02-16 美商阿尼拉製藥公司 類血管生成素３（ＡＮＧＰＴＬ３）ｉＲＮＡ組成物及其用途方法
WO2019055633A1 (en) 2017-09-14 2019-03-21 Arrowhead Pharmaceuticals, Inc. RNAI AGENTS AND COMPOSITIONS FOR INHIBITING ANALOGUE EXPRESSION OF ANGIOPOIETIN 3 (ANGPTL3) AND METHODS OF USE
US11414661B2 (en) 2017-12-01 2022-08-16 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
AR113490A1 (es) 2017-12-12 2020-05-06 Amgen Inc CONSTRUCCIONES DE ARNi PARA INHIBIR LA EXPRESIÓN DE PNPLA3 Y MÉTODOS DE USO DE LAS MISMAS
TW202039843A (zh) 2018-12-10 2020-11-01 美商安進公司 用於抑制ｐｎｐｌａ３表現之ｒｎａｉ構建體及其使用方法
EP4045062A1 (en) 2019-10-14 2022-08-24 Astrazeneca AB Modulators of pnpla3 expression
BR112022018667A2 (pt) 2020-03-18 2022-11-29 Dicerna Pharmaceuticals Inc Composições e métodos para inibir a expressão de ang-ptl3
JP2023542136A (ja) 2020-09-17 2023-10-05 アストラゼネカ・アクチエボラーグ 経口投与用のアンチセンスオリゴヌクレオチドを含む医薬組成物
MX2023010249A (es) 2021-03-04 2023-09-15 Alnylam Pharmaceuticals Inc Composiciones de arni de proteina similar a la angiopoyetina 3 (angptl3) y metodos para usarlas.
KR20240023635A (ko) 2021-06-21 2024-02-22 상하이 준스 바이오사이언스 컴퍼니 리미티드 ANGPTL3 유전자 발현을 억제하는 siRNA 및 이의 용도
CN120112639A (zh) 2022-08-22 2025-06-06 箭头药业股份有限公司 Angptl3相关疾病和病症的治疗方法
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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