CL2020000593A1 - Agentes rnai y composiciones para inhibir la expresión de apoliproteína c-iii (apoc3). - Google Patents

Agentes rnai y composiciones para inhibir la expresión de apoliproteína c-iii (apoc3).

Info

Publication number
CL2020000593A1
CL2020000593A1 CL2020000593A CL2020000593A CL2020000593A1 CL 2020000593 A1 CL2020000593 A1 CL 2020000593A1 CL 2020000593 A CL2020000593 A CL 2020000593A CL 2020000593 A CL2020000593 A CL 2020000593A CL 2020000593 A1 CL2020000593 A1 CL 2020000593A1
Authority
CL
Chile
Prior art keywords
apoc3
irna agents
iii
expression
compositions
Prior art date
Application number
CL2020000593A
Other languages
English (en)
Spanish (es)
Inventor
Rui Zhu
Tao Pei
Zhen Li
Steven Kanner
So Wong
Original Assignee
Arrowhead Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of CL2020000593A1 publication Critical patent/CL2020000593A1/es

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/18—Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/04—Anorexiants; Antiobesity agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H3/00—Compounds containing only hydrogen atoms and saccharide radicals having only carbon, hydrogen, and oxygen atoms
    • C07H3/02—Monosaccharides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/11—Antisense
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/332—Abasic residue
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen

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  • Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
  • Animal Behavior & Ethology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • General Engineering & Computer Science (AREA)
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  • Plant Pathology (AREA)
  • Microbiology (AREA)
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  • Dermatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
CL2020000593A 2017-09-11 2020-03-09 Agentes rnai y composiciones para inhibir la expresión de apoliproteína c-iii (apoc3). CL2020000593A1 (es)

Applications Claiming Priority (3)

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

Publications (1)

Publication Number Publication Date
CL2020000593A1 true CL2020000593A1 (es) 2020-09-25

Family

ID=65630632

Family Applications (2)

Application Number Title Priority Date Filing Date
CL2020000593A CL2020000593A1 (es) 2017-09-11 2020-03-09 Agentes rnai y composiciones para inhibir la expresión de apoliproteína c-iii (apoc3).
CL2024003470A CL2024003470A1 (es) 2017-09-11 2024-11-13 Agente de iarn para inhibir la expresión de un gen de apoc3; composición; y uso.

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CL2024003470A CL2024003470A1 (es) 2017-09-11 2024-11-13 Agente de iarn para inhibir la expresión de un gen de apoc3; composición; y uso.

Country Status (25)

Country Link
US (5) US10597657B2 (https=)
EP (1) EP3681516A4 (https=)
JP (4) JP7438103B2 (https=)
KR (2) KR20240161202A (https=)
CN (2) CN118879696A (https=)
AU (2) AU2018329190B2 (https=)
BR (1) BR112020002413A2 (https=)
CA (1) CA3074303A1 (https=)
CL (2) CL2020000593A1 (https=)
CO (1) CO2020002586A2 (https=)
CR (1) CR20200108A (https=)
EC (1) ECSP20017905A (https=)
IL (2) IL273172B2 (https=)
JO (1) JOP20200054A1 (https=)
MX (2) MX2020002648A (https=)
MY (1) MY202025A (https=)
NZ (1) NZ763172A (https=)
PE (1) PE20201283A1 (https=)
PH (1) PH12020500332A1 (https=)
SG (1) SG11201912178VA (https=)
TN (1) TN2020000039A1 (https=)
TW (2) TWI811238B (https=)
UY (1) UY37871A (https=)
WO (1) WO2019051402A1 (https=)
ZA (1) ZA202100806B (https=)

Families Citing this family (44)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
HK1244843A1 (zh) 2014-11-17 2018-08-17 Alnylam Pharmaceuticals, Inc. 载脂蛋白c3(apoc3)irna组合物及其使用方法
MX2018009853A (es) * 2016-09-02 2018-11-09 Arrowhead Pharmaceuticals Inc Ligandos de direccion.
JP7155239B2 (ja) 2017-04-05 2022-10-18 サイレンス・セラピューティクス・ゲーエムベーハー 産物及び組成物
US10597657B2 (en) * 2017-09-11 2020-03-24 Arrowhead Pharmaceuticals, Inc. RNAi agents and compositions for inhibiting expression of apolipoprotein C-III (APOC3)
CN110945130B (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
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
US12496347B2 (en) * 2018-12-28 2025-12-16 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
WO2020233680A1 (zh) 2019-05-22 2020-11-26 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
JP7610268B2 (ja) 2019-05-22 2025-01-08 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
AU2020280438B2 (en) 2019-05-22 2025-03-06 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
EP3978609A4 (en) 2019-05-24 2024-02-07 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
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
KR20220143106A (ko) * 2020-02-18 2022-10-24 알닐람 파마슈티칼스 인코포레이티드 아포지질단백질 C3 (APOC3) iRNA 조성물 및 이의 사용 방법
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
WO2022162155A1 (en) * 2021-01-30 2022-08-04 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
CN117580953B (zh) * 2021-07-16 2025-07-18 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
CA3232053A1 (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
WO2023102469A2 (en) 2021-12-01 2023-06-08 Dicerna Pharmaceuticals, Inc. Compositions and methods for modulating apoc3 expression
US20260076990A1 (en) * 2022-01-20 2026-03-19 Tuojie Biotech (Shanghai) Co., Ltd. Dsrna, use thereof and preparation method therefor
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
EP4471140A1 (en) 2022-01-30 2024-12-04 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
JP2025520796A (ja) 2022-06-27 2025-07-03 大睿生物 アポリポタンパク質Ｃ３発現を阻害するｓｉＲＮＡ
IL317824A (en) * 2022-07-11 2025-02-01 Sanegene Bio Usa Inc Ribose derivatives modified at the '2' position and methods of use
AU2023313321A1 (en) * 2022-07-27 2025-01-09 E-Therapeutics Plc Nucleic acid compounds
IL318229A (en) * 2022-07-27 2025-03-01 E Therapeutics Plc Nucleic acid compounds
JP2025524135A (ja) * 2022-07-27 2025-07-25 イー－セラピューティクス・ピーエルシー 核酸化合物
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
WO2024032680A1 (zh) * 2022-08-11 2024-02-15 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
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
EP2314700A1 (en) 1999-01-28 2011-04-27 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
US8137695B2 (en) 2006-08-18 2012-03-20 Arrowhead Madison Inc. Polyconjugates for in vivo delivery of polynucleotides
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
US20040259247A1 (en) 2000-12-01 2004-12-23 Thomas Tuschl Rna interference mediating small rna molecules
WO2002100435A1 (en) 2001-06-11 2002-12-19 Centre Hospitalier Universitaire De Montreal Compositions and methods for enhancing nucleic acid transfer into cells
CA2456977C (en) 2001-08-16 2011-06-07 The Trustees Of The University Of Pennsylvania Synthesis and use of reagents for improved dna lipofection and/or slow release prodrug and drug therapies
US7901708B2 (en) 2002-06-28 2011-03-08 Protiva Biotherapeutics, Inc. Liposomal apparatus and manufacturing methods
MXPA05003287A (es) 2002-09-28 2005-07-05 Massachusetts Inst Technology Influenza terapeutica.
EP2305812A3 (en) 2002-11-14 2012-06-06 Dharmacon, Inc. Fuctional and hyperfunctional sirna
ES2343318T3 (es) 2002-11-26 2010-07-28 University Of Massachusetts Administracion de arnsis.
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
ES2559828T3 (es) 2003-07-16 2016-02-16 Protiva Biotherapeutics Inc. ARN de interferencia encapsulado en lípidos
CN1845993B (zh) 2003-08-28 2010-06-23 诺瓦提斯公司 具有平端和3＇修饰的干扰rna双链体
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
US8101741B2 (en) 2005-11-02 2012-01-24 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
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
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
WO2009082817A1 (en) 2007-12-27 2009-07-09 Protiva Biotherapeutics, Inc. Silencing of polo-like kinase expression using interfering rna
US8188060B2 (en) 2008-02-11 2012-05-29 Dharmacon, Inc. Duplex oligonucleotides with enhanced functionality in gene regulation
CA2750561C (en) * 2009-01-26 2017-10-10 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein c-iii expression
WO2011076807A2 (en) 2009-12-23 2011-06-30 Novartis Ag Lipids, lipid compositions, and methods of using them
DK2539451T3 (da) 2010-02-24 2016-04-04 Arrowhead Res Corp Sammensætninger til målrettet tilførsel af siRNA
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
CN103649103A (zh) 2011-06-21 2014-03-19 阿尔尼拉姆医药品有限公司 用于抑制载脂蛋白c-iii(apoc3)基因表达的组合物与方法
WO2013032829A1 (en) 2011-08-26 2013-03-07 Arrowhead Research Corporation Poly(vinyl ester) polymers for in vivo nucleic acid delivery
JP2015515530A (ja) 2012-04-18 2015-05-28 アローヘッド リサーチ コーポレイション インビボ核酸送達のためのポリ（アクリラート）ポリマー
WO2013165816A2 (en) * 2012-05-02 2013-11-07 Merck Sharp & Dohme Corp. 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
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
HK1244843A1 (zh) * 2014-11-17 2018-08-17 Alnylam Pharmaceuticals, Inc. 载脂蛋白c3(apoc3)irna组合物及其使用方法
US20160272970A1 (en) * 2015-03-17 2016-09-22 Arrowhead Madison Inc. RNA Interference Agents
JOP20160211B1 (ar) * 2015-10-01 2021-08-17 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
KR102515329B1 (ko) 2016-03-07 2023-03-29 애로우헤드 파마슈티컬스 인코포레이티드 치료용 화합물을 위한 표적화 리간드
MX2018009853A (es) 2016-09-02 2018-11-09 Arrowhead Pharmaceuticals Inc Ligandos de direccion.
US11603532B2 (en) * 2017-06-02 2023-03-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
US10597657B2 (en) * 2017-09-11 2020-03-24 Arrowhead Pharmaceuticals, Inc. RNAi agents and compositions for inhibiting expression of apolipoprotein C-III (APOC3)

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