AU2016247922B2 - Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof - Google Patents

Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof Download PDF

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AU2016247922B2
AU2016247922B2 AU2016247922A AU2016247922A AU2016247922B2 AU 2016247922 B2 AU2016247922 B2 AU 2016247922B2 AU 2016247922 A AU2016247922 A AU 2016247922A AU 2016247922 A AU2016247922 A AU 2016247922A AU 2016247922 B2 AU2016247922 B2 AU 2016247922B2
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angptl3
nucleotides
dsrna
nucleotide
strand
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AU2016247922A1 (en
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James Butler
Kevin Fitzgerald
Gregory Hinkle
Martin Maier
William Querbes
Stephanie Williams
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Alnylam Pharmaceuticals Inc
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AU2016247922A 2015-04-13 2016-04-13 Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof Active AU2016247922B2 (en)

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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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US (4) US10570393B2 (enExample)
EP (1) EP3283631A1 (enExample)
JP (3) JP6975641B2 (enExample)
KR (2) KR20250005546A (enExample)
CN (2) CN107743522B (enExample)
AU (2) AU2016247922B2 (enExample)
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Families Citing this family (40)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN103890000B (zh) 2011-06-21 2017-09-01 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
KR20250005546A (ko) 2015-04-13 2025-01-09 알닐람 파마슈티칼스 인코포레이티드 안지오포이에틴-유사 3(angptl3) irna 조성물 및 이의 이용 방법
AU2018331383B2 (en) * 2017-09-14 2024-12-05 Arrowhead Pharmaceuticals, Inc. RNAi agents and compositions for inhibiting expression of angiopoietin-like 3 (ANGPTL3), and methods of use
JP7273417B2 (ja) 2017-12-01 2023-05-15 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および複合体ならびに調製方法と使用
KR20250107294A (ko) 2017-12-01 2025-07-11 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 핵산, 이를 포함하는 조성물과 컨쥬게이트, 및 그의 제조 방법과 용도
WO2019105418A1 (zh) 2017-12-01 2019-06-06 苏州瑞博生物技术有限公司 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
WO2019105419A1 (zh) 2017-12-01 2019-06-06 苏州瑞博生物技术有限公司 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
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
DK3732185T3 (da) * 2017-12-29 2025-03-31 Suzhou Ribo Life Science Co Ltd Konjugater og fremstilling og anvendelse heraf
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, PHARMACEUTICAL COMPOSITION AND CONJUGATE WITH NUCLEIC ACID AND THEIR USE
WO2020063198A1 (zh) 2018-09-30 2020-04-02 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
CA3118537A1 (en) * 2018-11-09 2020-05-14 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
JP7507495B2 (ja) 2018-12-28 2024-06-28 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
EP3974530A4 (en) 2019-05-22 2023-07-12 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, PHARMACEUTICAL COMPOSITION, CONJUGATE, METHOD OF MANUFACTURE AND USE
CA3139195A1 (en) 2019-05-22 2020-11-26 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
JP7610849B2 (ja) 2019-05-24 2025-01-09 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
JP2023514000A (ja) * 2019-12-09 2023-04-05 エンピリコ インク． アンジオポエチン様４（ａｎｇｐｔｌ４）関連疾患の処置のためのオリゴヌクレオチド
GB2613225B (en) * 2020-03-04 2024-01-03 Verve Therapeutics Inc Compositions and methods for targeted RNA delivery
CA3174872A1 (en) * 2020-04-07 2021-10-14 Sander Jan Hendrik Van Deventer Gene constructs for silencing angiopoietin-like 3 (angptl3) and uses thereof
CN115516092B (zh) * 2020-09-30 2025-05-16 亿腾医药（苏州）有限公司 血管生成素样3(ANGPTL3)的siRNA及其用途
CN116887842A (zh) * 2020-10-16 2023-10-13 赛诺菲 用于抑制angptl3的新型rna组合物和方法
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
IL305418A (en) * 2021-03-04 2023-10-01 Alnylam Pharmaceuticals Inc Angiopoietin-like 3 (ANGPTL3) IRNA compositions and methods of using them
CA3214660A1 (en) * 2021-03-24 2022-09-29 Atalanta Therapeutics, Inc. Double-stranded sirna having patterned chemical modifications
JP2024530647A (ja) 2021-08-03 2024-08-23 ヴァーヴ・セラピューティクス，インコーポレーテッド 標的ｒｎａ送達のための組成物および方法
WO2023044458A1 (en) * 2021-09-17 2023-03-23 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing angptl4
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
US20230193271A1 (en) 2021-12-22 2023-06-22 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
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
AU2023375521A1 (en) 2022-11-10 2025-05-15 Regeneron Pharmaceuticals, Inc. Treatment of kidney diseases with a combination of angiopoietin like 3 (angptl3) inhibitors and solute carrier family 5 member 2 (slc5a2) inhibitors
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
WO2025232842A1 (zh) * 2024-05-09 2025-11-13 石药集团中奇制药技术（石家庄）有限公司 一种抑制angptl3基因表达的核酸分子

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012177784A2 (en) * 2011-06-21 2012-12-27 Alnylam Pharmaceuticals Angiopoietin-like 3 (angptl3) irna compostions and methods of use thereof

Family Cites Families (51)

* 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
EP0685234B1 (en) 1993-02-19 2000-05-10 Nippon Shinyaku Company, Limited Drug composition containing nucleic acid copolymer
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
DE69829760T3 (de) 1997-09-12 2016-04-14 Exiqon A/S Bi- und tri-zyklische - nukleosid, nukleotid und oligonukleotid-analoga
US6436989B1 (en) 1997-12-24 2002-08-20 Vertex Pharmaceuticals, Incorporated Prodrugs of aspartyl protease inhibitors
AP2000001856A0 (en) 1997-12-24 2000-09-30 Vertex Pharma Prodrugs of aspartyl protease inhibitors.
AU6298899A (en) 1998-10-09 2000-05-01 Ingene, Inc. Production of ssdna (in vivo)
AU6430599A (en) 1998-10-09 2000-05-01 Cytogenix, Inc. Enzymatic synthesis of ssdna
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
KR100909681B1 (ko) 2000-12-01 2009-07-29 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. Ｒｎａ 간섭을 매개하는 작은 ｒｎａ 분자
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
CN101855241B (zh) 2006-12-08 2013-11-06 莱克康制药公司 针对angptl3的单克隆抗体
CA2910760C (en) 2007-12-04 2019-07-09 Muthiah Manoharan Targeting lipids
WO2009134487A2 (en) 2008-01-31 2009-11-05 Alnylam Pharmaceuticals, Inc. Optimized methods for delivery of dsrna targeting the pcsk9 gene
EP2334793B1 (en) 2008-09-25 2016-04-06 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of serum amyloid a gene
SG10201809460SA (en) 2008-10-20 2018-11-29 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expression of transthyretin
CA2746514C (en) 2008-12-10 2018-11-27 Alnylam Pharmaceuticals, Inc. Gnaq targeted dsrna compositions and methods for inhibiting expression
US20120017784A1 (en) * 2009-01-20 2012-01-26 James Butcher Method of providing a printed date
CA2764609C (en) 2009-06-10 2018-10-02 Alnylam Pharmaceuticals, Inc. Improved cationic lipid of formula i
KR20120050429A (ko) 2009-06-15 2012-05-18 알닐람 파마슈티칼스 인코포레이티드 Ｐｃｓｋ9 유전자를 표적으로 하는 지질 제형된 ｄｓｒｎａ
US9512164B2 (en) 2009-07-07 2016-12-06 Alnylam Pharmaceuticals, Inc. Oligonucleotide end caps
EP2521556B1 (en) * 2010-01-08 2018-05-30 Ionis Pharmaceuticals, Inc. Modulation of angiopoietin-like 3 expression
ES2523140T3 (es) * 2010-09-21 2014-11-21 Population Genetics Technologies Ltd. Aumento de la confianza en las identificaciones de alelos con el recuento molecular
HK1200191A1 (en) * 2011-11-18 2015-07-31 Alnylam Pharmaceuticals, Inc. Modified rnai agents
EP2844261B1 (en) * 2012-05-02 2018-10-17 Sirna Therapeutics, Inc. SHORT INTERFERING NUCLEIC ACID (siNA) COMPOSITIONS
CA2879683C (en) 2012-08-03 2023-02-14 Alnylam Pharmaceuticals, Inc. Modified rnai agents
JP2015525797A (ja) 2012-08-06 2015-09-07 アルニラム・ファーマシューティカルズ・インコーポレーテッド 糖質コンジュゲートｒｎａ剤およびその調製方法
US10246708B2 (en) * 2013-05-06 2019-04-02 Alnylam Pharmaceuticals, Inc. Dosages and methods for delivering lipid formulated nucleic acid molecules
CN105814204B (zh) 2013-12-24 2020-04-28 Ionis制药公司 促血管生成素样3表达的调节
ES2844593T3 (es) * 2014-05-01 2021-07-22 Ionis Pharmaceuticals Inc Composiciones y procedimientos para modular la expresión de la angiopoyetina de tipo 3
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
KR20250005546A (ko) 2015-04-13 2025-01-09 알닐람 파마슈티칼스 인코포레이티드 안지오포이에틴-유사 3(angptl3) irna 조성물 및 이의 이용 방법
AU2018331383B2 (en) 2017-09-14 2024-12-05 Arrowhead Pharmaceuticals, Inc. RNAi agents and compositions for inhibiting expression of angiopoietin-like 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
EP3894555A2 (en) 2018-12-10 2021-10-20 Amgen Inc. Rnai constructs for inhibiting pnpla3 expression
JP7757277B2 (ja) 2019-10-14 2025-10-21 アストラゼネカ・アクチエボラーグ Ｐｎｐｌａ３発現のモジュレーター
IL296549A (en) 2020-03-18 2022-11-01 Dicerna Pharmaceuticals Inc Compositions and methods for inhibiting angptl3 expression
IL301221A (en) 2020-09-17 2023-05-01 Astrazeneca Ab Pharmaceutical compositions comprising an antisense oligonucleotide for oral administration
IL305418A (en) 2021-03-04 2023-10-01 Alnylam Pharmaceuticals Inc Angiopoietin-like 3 (ANGPTL3) IRNA compositions and methods of using them
KR20240023635A (ko) 2021-06-21 2024-02-22 상하이 준스 바이오사이언스 컴퍼니 리미티드 ANGPTL3 유전자 발현을 억제하는 siRNA 및 이의 용도
CA3264922A1 (en) 2022-08-22 2024-02-29 Arrowhead Pharmaceuticals, Inc. METHODS FOR THE TREATMENT OF DISEASES AND DISORDERS RELATED TO ANGPTL3
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

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2012177784A2 (en) * 2011-06-21 2012-12-27 Alnylam Pharmaceuticals Angiopoietin-like 3 (angptl3) irna compostions and methods of use thereof

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