MX2020002648A - Agentes de iarn y composiciones para inhibir la expresion de la apolipoproteina c-iii (apoc3). - Google Patents

Agentes de iarn y composiciones para inhibir la expresion de la apolipoproteina c-iii (apoc3).

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Publication number
MX2020002648A
MX2020002648A MX2020002648A MX2020002648A MX2020002648A MX 2020002648 A MX2020002648 A MX 2020002648A MX 2020002648 A MX2020002648 A MX 2020002648A MX 2020002648 A MX2020002648 A MX 2020002648A MX 2020002648 A MX2020002648 A MX 2020002648A
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Prior art keywords
apoc3
rnai agents
iii
expression
apolipoprotein
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MX2020002648A
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English (en)
Inventor
So Wong
Steven Kanner
Tao Pei
Zhen Li
Rui Zhu
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Arrowhead Pharmaceuticals Inc
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Publication date
Application filed by Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of MX2020002648A publication Critical patent/MX2020002648A/es

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Abstract

La presente descripción se refiere a agentes de iARN, por ejemplo, agentes de iARN de cadena doble, capaces de inhibir la expresión del gen de la Apolipoproteína C-III (también denominada APOC3, apoC-III, APOC-III, y APO C-III), y composiciones que incluyen agentes de iARN de APOC3. Los agentes de iARN de APOC3 descritos en la presente pueden estar conjugados con ligandos diana, que incluyen ligandos que incluyen N-acetil-galactosamina, para facilitar la administración a células, que incluyen a hepatocitos. También se describen las composiciones farmacéuticas que incluyen uno o más agentes de iARN de APOC3, opcionalmente con uno o más productos terapéuticos adicionales. La administración de los agentes de iARN de APOC3 in vivo proporciona la inhibición de la expresión del gen APOC3, y puede dar como resultado niveles de triglicéridos y/o niveles de colesterol reducidos en el sujeto. Los agentes de iARN de APOC3 se pueden usar en métodos de tratamiento de enfermedades y trastornos relacionados con APOC3 que incluyen hipertrigliceridemia, enfermedad cardiovascular, y otros trastornos y enfermedades metabólicos relacionados.
MX2020002648A 2017-09-11 2018-09-10 Agentes de iarn y composiciones para inhibir la expresion de la apolipoproteina c-iii (apoc3). MX2020002648A (es)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762556818P 2017-09-11 2017-09-11
US201862643927P 2018-03-16 2018-03-16
US201862720434P 2018-08-21 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)

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MX2020002648A true MX2020002648A (es) 2020-07-22

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MX2020002648A MX2020002648A (es) 2017-09-11 2018-09-10 Agentes de iarn y composiciones para inhibir la expresion de la apolipoproteina c-iii (apoc3).
MX2025015219A MX2025015219A (es) 2017-09-11 2020-03-06 Agentes de iarn y composiciones para inhibir la expresion de la apolipoproteina c-iii (apoc3)

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US (5) US10597657B2 (es)
EP (1) EP3681516A4 (es)
JP (4) JP7438103B2 (es)
KR (2) KR20240161202A (es)
CN (2) CN111107853B (es)
AU (2) AU2018329190B2 (es)
BR (1) BR112020002413A2 (es)
CA (1) CA3074303A1 (es)
CL (2) CL2020000593A1 (es)
CO (1) CO2020002586A2 (es)
CR (1) CR20200108A (es)
EC (1) ECSP20017905A (es)
IL (2) IL273172B2 (es)
JO (1) JOP20200054A1 (es)
MX (2) MX2020002648A (es)
MY (1) MY202025A (es)
NZ (1) NZ763172A (es)
PE (1) PE20201283A1 (es)
PH (1) PH12020500332A1 (es)
SG (1) SG11201912178VA (es)
TN (1) TN2020000039A1 (es)
TW (2) TWI811238B (es)
UY (1) UY37871A (es)
WO (1) WO2019051402A1 (es)
ZA (1) ZA202100806B (es)

Families Citing this family (44)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2017535552A (ja) 2014-11-17 2017-11-30 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. アポリポタンパク質C3(APOC3)iRNA組成物およびその使用方法
SG11201901841TA (en) * 2016-09-02 2019-03-28 Arrowhead Pharmaceuticals Inc Targeting ligands
DK3607069T3 (da) 2017-04-05 2022-11-21 Silence Therapeutics Gmbh Produkter og sammensætninger
KR20240161202A (ko) * 2017-09-11 2024-11-12 애로우헤드 파마슈티컬스 인코포레이티드 아포지단백질 C-III (APOC3)의 발현을 억제하기 위한 RNAi 작용제 및 조성물
EP3719126B1 (en) 2017-12-01 2025-01-01 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
EP3719127A4 (en) 2017-12-01 2021-10-20 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, COMPOSITION AND CONJUGATE WITH IT, MANUFACTURING METHOD AND USE
WO2020135673A1 (zh) * 2018-12-28 2020-07-02 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
AU2020280439B2 (en) 2019-05-22 2025-08-14 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
US12590304B2 (en) 2019-05-22 2026-03-31 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
KR20220011689A (ko) 2019-05-22 2022-01-28 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 핵산, 약제학적 조성물, 접합체, 제조 방법, 및 용도
CN113795582B (zh) 2019-05-24 2024-05-24 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
MX2022001460A (es) * 2019-08-05 2022-02-22 Arrowhead Pharmaceuticals Inc Metodos para el tratamiento de enfermedades y trastornos relacionados con apoc3.
JP7735288B2 (ja) 2020-02-18 2025-09-08 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 アポリポタンパク質C3(APOC3)iRNA組成物およびその使用法
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
EP4176061A1 (en) * 2021-01-30 2023-05-10 E-Therapeutics plc Nucleic acids containing abasic nucleotides
AU2022212580A1 (en) * 2021-01-30 2023-07-27 E-Therapeutics Plc Nucleic acids containing abasic nucleotides
AU2022299514A1 (en) * 2021-06-24 2024-01-18 Sirnaomics, Inc. Products and compositions
EP4372085A4 (en) * 2021-07-16 2026-01-21 Suzhou Ribo Life Science Co Ltd Nucleic acid, composition and conjugate, container, preparation process and use
WO2023059948A1 (en) * 2021-10-08 2023-04-13 E-Therapeutics Plc Nucleic acids containing abasic nucleosides
IL312811A (en) 2021-11-16 2024-07-01 Shanghai Argo Biopharmaceutical Co Ltd Composition and method for inhibiting angiotensinogen (agt) protein expression
JP2024543195A (ja) 2021-12-01 2024-11-19 ディセルナ ファーマシューティカルズ インコーポレイテッド Apoc3発現を調節するための組成物及び方法
TW202340469A (zh) * 2022-01-20 2023-10-16 大陸商上海拓界生物醫藥科技有限公司 一種dsrna、其應用及製備方法
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
CN117756866A (zh) 2022-01-30 2024-03-26 大睿生物医药科技(上海)有限公司 含有n-乙酰半乳糖胺的靶向配体
CN119487191A (zh) 2022-06-14 2025-02-18 大睿生物医药科技(上海)有限公司 调控PCSK9基因活性的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
EP4555088A1 (en) * 2022-07-11 2025-05-21 Sanegene Bio USA Inc. Optimized 2'- modified ribose derivatives and methods of use
AU2023313030A1 (en) * 2022-07-27 2025-01-09 E-Therapeutics Plc Nucleic acid compounds
CN120615125A (zh) * 2022-07-27 2025-09-09 e-生物有限公司 核酸化合物
CN120322552A (zh) * 2022-07-27 2025-07-15 e-生物有限公司 核酸化合物
CN116814621B (zh) * 2022-08-05 2025-09-09 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
WO2024165571A2 (en) * 2023-02-06 2024-08-15 E-Therapeutics Plc Inhibitors of expression and/or function
WO2024165568A2 (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
WO2025007961A1 (zh) 2023-07-06 2025-01-09 大睿生物 调控AGT表达的dsRNA分子
TW202526017A (zh) 2023-09-14 2025-07-01 美商Ionis製藥公司 用於減少apociii表現的化合物及方法
WO2025087442A1 (zh) * 2023-10-27 2025-05-01 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
CA2361201A1 (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
DE60040274D1 (de) 1999-03-10 2008-10-30 Phogen Ltd Verabreichung von nukleinsäuren und proteinen an zellen
EP1272630A2 (en) 2000-03-16 2003-01-08 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
CA2429814C (en) 2000-12-01 2014-02-18 Thomas Tuschl Rna interference mediating small rna molecules
EP1399189A1 (en) 2001-06-11 2004-03-24 Universite De Montreal Compositions and methods for enhancing nucleic acid transfer into cells
JP5170934B2 (ja) 2001-08-16 2013-03-27 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 改良dnaリポフェクションならびに/または徐放性プロドラッグおよび薬物療法のための試薬の合成と使用
EP1519714B1 (en) 2002-06-28 2010-10-20 Protiva Biotherapeutics Inc. Method and apparatus for producing liposomes
US20050008617A1 (en) 2002-09-28 2005-01-13 Massachusetts Institute Of Technology Compositions and methods for delivery of short interfering RNA and short hairpin RNA
AU2003295600A1 (en) 2002-11-14 2004-06-15 Dharmacon, Inc. Functional and hyperfunctional sirna
EP1585756B1 (en) 2002-11-26 2010-04-21 University of Massachusetts Delivery of sirnas
US20040208921A1 (en) 2003-01-14 2004-10-21 Ho Rodney J. Y. Lipid-drug formulations and methods for targeted delivery of lipid-drug complexes to lymphoid tissues
US7598227B2 (en) * 2003-04-16 2009-10-06 Isis Pharmaceuticals Inc. Modulation of apolipoprotein C-III expression
CA2532228C (en) 2003-07-16 2017-02-14 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering rna
EP1660657A1 (en) 2003-08-28 2006-05-31 Novartis AG Interfering rna duplex having blunt-ends and 3'-modifications
WO2005116204A1 (ja) 2004-05-11 2005-12-08 Rnai Co., Ltd. Rna干渉を生じさせるポリヌクレオチド、および、これを用いた遺伝子発現抑制方法
CN101346393B (zh) 2005-11-02 2015-07-22 普洛体维生物治疗公司 修饰的siRNA分子及其应用
WO2007107162A2 (en) 2006-03-23 2007-09-27 Santaris Pharma A/S Small internally segmented interfering rna
GB0608838D0 (en) 2006-05-04 2006-06-14 Novartis Ag Organic compounds
CN101500548A (zh) 2006-08-18 2009-08-05 弗·哈夫曼-拉罗切有限公司 用于体内递送多核苷酸的多缀合物
KR101629017B1 (ko) 2007-05-22 2016-06-10 아크투루스 쎄라퓨틱스, 인크. 히드록시메틸 치환된 rna 올리고뉴클레오티드 및 rna 복합체
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
ES2535419T3 (es) 2007-12-27 2015-05-11 Protiva Biotherapeutics Inc. Silenciamiento de expresión de quinasa tipo polo usando ARN interferente
US8188060B2 (en) 2008-02-11 2012-05-29 Dharmacon, Inc. Duplex oligonucleotides with enhanced functionality in gene regulation
US9023820B2 (en) * 2009-01-26 2015-05-05 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein C-III expression
CA3009891C (en) 2009-12-23 2020-09-15 Novartis Ag Lipids, lipid compositions, and methods of using them
SI2539451T1 (sl) 2010-02-24 2016-04-29 Arrowhead Research Corporation Sestavki za ciljano dostavo sirna
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
MX390699B (es) 2011-06-21 2025-03-21 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibicion de genes para apolipoproteina c-iii (apoc3).
WO2013032829A1 (en) 2011-08-26 2013-03-07 Arrowhead Research Corporation Poly(vinyl ester) polymers for in vivo nucleic acid delivery
CA2842041A1 (en) 2012-04-18 2013-10-24 Arrowhead Research Corporation Poly(acrylate) polymers for in vivo nucleic acid delivery
US20150299696A1 (en) * 2012-05-02 2015-10-22 Sirna Therapeutics, Inc. SHORT INTERFERING NUCLEIC ACID (siNA) COMPOSITIONS
EP3564374A1 (en) * 2013-06-21 2019-11-06 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of target nucleic acids
WO2015051366A2 (en) 2013-10-04 2015-04-09 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
JP2017535552A (ja) * 2014-11-17 2017-11-30 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. アポリポタンパク質C3(APOC3)iRNA組成物およびその使用方法
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
CA3011946A1 (en) 2016-03-07 2017-09-14 Arrowhead Pharmaceuticals, Inc. Targeting ligands for therapeutic compounds
SG11201901841TA (en) 2016-09-02 2019-03-28 Arrowhead Pharmaceuticals Inc Targeting ligands
JP2020522510A (ja) * 2017-06-02 2020-07-30 ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. オリゴヌクレオチド組成物及びその使用方法
KR20240161202A (ko) * 2017-09-11 2024-11-12 애로우헤드 파마슈티컬스 인코포레이티드 아포지단백질 C-III (APOC3)의 발현을 억제하기 위한 RNAi 작용제 및 조성물

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