PE20201283A1 - 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)Info
- Publication number
- PE20201283A1 PE20201283A1 PE2020000340A PE2020000340A PE20201283A1 PE 20201283 A1 PE20201283 A1 PE 20201283A1 PE 2020000340 A PE2020000340 A PE 2020000340A PE 2020000340 A PE2020000340 A PE 2020000340A PE 20201283 A1 PE20201283 A1 PE 20201283A1
- Authority
- PE
- Peru
- Prior art keywords
- apoc3
- irna agents
- iii
- expression
- compositions
- Prior art date
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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
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- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- 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
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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- A61P3/00—Drugs for disorders of the metabolism
- A61P3/04—Anorexiants; Antiobesity agents
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- 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
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- C07H3/02—Monosaccharides
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- 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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- C12N2310/332—Abasic residue
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- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/351—Conjugate
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
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- C12N2310/3521—Methyl
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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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- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Abstract
La presente descripcion se refiere a agentes de iARN, por ejemplo, agentes de iARN de cadena doble, capaces de inhibir la expresion del gen de la Apolipoproteina C-III (tambien 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 administracion a celulas, que incluyen a hepatocitos. Tambien se describen las composiciones farmaceuticas que incluyen uno o mas agentes de iARN de APOC3, opcionalmente con uno o mas productos terapeuticos adicionales. La administracion de los agentes de iARN de APOC3 in vivo proporciona la inhibicion de la expresion del gen APOC3, y puede dar como resultado niveles de trigliceridos y/o niveles de colesterol reducidos en el sujeto. Los agentes de iARN de APOC3 se pueden usar en metodos de tratamiento de enfermedades y trastornos relacionados con APOC3 que incluyen hipertrigliceridemia, enfermedad cardiovascular, y otros trastornos y enfermedades metabolicas relacionadas
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) |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| PE20201283A1 true PE20201283A1 (es) | 2020-11-24 |
Family
ID=65630632
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| PE2020000340A PE20201283A1 (es) | 2017-09-11 | 2018-09-10 | Agentes de iarn y composiciones para inhibir la expresion de la apolipoproteina c-iii (apoc3) |
Country Status (24)
| Country | Link |
|---|---|
| US (4) | US10597657B2 (es) |
| EP (1) | EP3681516A4 (es) |
| JP (2) | JP7438103B2 (es) |
| KR (2) | KR20240161202A (es) |
| CN (2) | CN118879696A (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) | IL273172B1 (es) |
| JO (1) | JOP20200054A1 (es) |
| MX (1) | MX2020002648A (es) |
| MY (1) | MY202025A (es) |
| PE (1) | PE20201283A1 (es) |
| PH (1) | PH12020500332A1 (es) |
| SG (1) | SG11201912178VA (es) |
| TN (1) | TN2020000039A1 (es) |
| TW (2) | TW202415389A (es) |
| UY (1) | UY37871A (es) |
| WO (1) | WO2019051402A1 (es) |
| ZA (1) | ZA202100806B (es) |
Families Citing this family (40)
| 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 |
| KR102403408B1 (ko) * | 2016-09-02 | 2022-05-30 | 애로우헤드 파마슈티컬스 인코포레이티드 | 표적화 리간드 |
| RS63836B1 (sr) | 2017-04-05 | 2023-01-31 | Silence Therapeutics Gmbh | Proizvodi i sastavi |
| CA3074303A1 (en) * | 2017-09-11 | 2019-03-14 | Arrowhead Pharmaceuticals, Inc. | Rnai agents and compositions for inhibiting expression of apolipoprotein c-iii (apoc3) |
| 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 |
| 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 |
| EP3974532A4 (en) | 2019-05-22 | 2024-01-24 | Suzhou Ribo Life Science Co., Ltd. | NUCLEIC ACID, PHARMACEUTICAL COMPOSITION, CONJUGATE, PROCESS OF PREPARATION AND USE |
| EP3974530A4 (en) | 2019-05-22 | 2023-07-12 | Suzhou Ribo Life Science Co., Ltd. | NUCLEIC ACID, PHARMACEUTICAL COMPOSITION, CONJUGATE, METHOD FOR PREPARATION AND USE |
| JP2022543136A (ja) * | 2019-08-05 | 2022-10-07 | 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 |
| KR20230136644A (ko) * | 2021-01-30 | 2023-09-26 | 이-테라퓨틱스 피엘씨 | 무염기 뉴클레오티드를 함유하는 핵산 |
| EP4176061A1 (en) * | 2021-01-30 | 2023-05-10 | E-Therapeutics plc | Nucleic acids containing abasic nucleotides |
| IL309682A (en) * | 2021-06-24 | 2024-02-01 | Sirnaomics Inc | products and preparations |
| CN120989078A (zh) * | 2021-07-16 | 2025-11-21 | 瑞博泰克(山东)生物医药科技有限公司 | 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 |
| EP4413130A1 (en) * | 2021-10-08 | 2024-08-14 | 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 |
| JP2025504845A (ja) * | 2022-01-20 | 2025-02-19 | 上海拓界生物医薬科技有限公司 | dsRNA、その使用及び調製方法 |
| 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 |
| 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 |
| 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 |
| AU2023307152A1 (en) * | 2022-07-11 | 2025-01-09 | Sanegene Bio Usa Inc. | Optimized 2'- modified ribose derivatives and methods of use |
| WO2024023256A1 (en) * | 2022-07-27 | 2024-02-01 | 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 |
| KR20250043473A (ko) * | 2022-07-27 | 2025-03-28 | 이-테라퓨틱스 피엘씨 | 핵산 화합물 |
| 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 |
| CN121219413A (zh) * | 2023-02-06 | 2025-12-26 | e-生物有限公司 | 表达和/或功能的抑制剂 |
| 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 |
| WO2025059466A1 (en) | 2023-09-14 | 2025-03-20 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing apociii expression |
| CN120344664A (zh) * | 2023-10-27 | 2025-07-18 | 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 |
| WO2025133348A1 (en) * | 2023-12-22 | 2025-06-26 | 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 |
| 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 |
Family Cites Families (49)
| 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 |
| WO2000044914A1 (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 |
| WO2000053722A2 (en) | 1999-03-10 | 2000-09-14 | Phogen Limited | Delivery of nucleic acids and proteins to cells |
| US8137695B2 (en) | 2006-08-18 | 2012-03-20 | Arrowhead Madison Inc. | Polyconjugates for in vivo delivery of polynucleotides |
| JP2003526367A (ja) | 2000-03-16 | 2003-09-09 | ジェネティカ インコーポレイテッド | Rna干渉の方法とrna干渉組成物 |
| US6680068B2 (en) | 2000-07-06 | 2004-01-20 | The General Hospital Corporation | Drug delivery formulations and targeting |
| IL155991A0 (en) | 2000-12-01 | 2003-12-23 | Max Planck Gesellschaft | 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 |
| AU2002324723B2 (en) | 2001-08-16 | 2007-10-25 | The Trustees Of The University Of Pennsylvania | Synthesis and use of reagents for improved DNA lipofection and/or slow release pro-drug and drug therapies |
| EP2338478B1 (en) | 2002-06-28 | 2014-07-23 | Protiva Biotherapeutics Inc. | Method for producing liposomes |
| AU2003279010A1 (en) | 2002-09-28 | 2004-04-19 | Massachusetts Institute Of Technology | Compositions and methods for delivery of short interfering rna and short hairpin rna |
| EP2305813A3 (en) | 2002-11-14 | 2012-03-28 | Dharmacon, Inc. | Fuctional and hyperfunctional sirna |
| EP1585756B1 (en) | 2002-11-26 | 2010-04-21 | University of Massachusetts | Delivery of sirnas |
| WO2004064731A2 (en) | 2003-01-14 | 2004-08-05 | University Of Washington | Lipid-drug formulations and methods for targeted delivery of lipid-drug complexes to lymlplhoid tissues |
| US7598227B2 (en) * | 2003-04-16 | 2009-10-06 | Isis Pharmaceuticals Inc. | Modulation of apolipoprotein C-III expression |
| CN101291653B (zh) | 2003-07-16 | 2012-06-27 | 普洛体维生物治疗公司 | 脂质包封的干扰rna |
| KR20060087531A (ko) | 2003-08-28 | 2006-08-02 | 노파르티스 아게 | 블런트-말단 및 3'-변형체를 갖는 간섭 rna 이중나선 |
| CA2566286A1 (en) | 2004-05-11 | 2005-12-08 | Rnai Co., Ltd. | Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same |
| 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 |
| CA2644347C (en) | 2006-03-23 | 2017-05-30 | Santaris Pharma A/S | Small internally segmented interfering rna |
| GB0608838D0 (en) | 2006-05-04 | 2006-06-14 | Novartis Ag | Organic compounds |
| CA2687850C (en) | 2007-05-22 | 2017-11-21 | Mdrna, Inc. | Oligomers for therapeutics |
| 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 |
| EP2238251B1 (en) | 2007-12-27 | 2015-02-11 | 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 |
| KR101762466B1 (ko) | 2009-12-23 | 2017-07-27 | 노파르티스 아게 | 지질, 지질 조성물 및 이의 사용 방법 |
| CN102869774B (zh) | 2010-02-24 | 2018-02-09 | 箭头研究公司 | 用于靶向递送siRNA的组合物 |
| US8501930B2 (en) | 2010-12-17 | 2013-08-06 | Arrowhead Madison Inc. | Peptide-based in vivo siRNA delivery system |
| KR102540778B1 (ko) | 2011-06-21 | 2023-06-07 | 알닐람 파마슈티칼스 인코포레이티드 | 아포리포단백질 c-iii(apoc3) 유전자의 발현 억제를 위한 조성물 및 방법 |
| BR112014004585A2 (pt) | 2011-08-26 | 2017-06-13 | Arrowhead Res Corp | polímeros de poli(éster vinílico) para liberação de ácido nucleico in vivo |
| AU2012377385A1 (en) | 2012-04-18 | 2014-01-23 | 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 |
| EP3169784B1 (en) * | 2014-07-16 | 2020-06-10 | 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 |
| US20160272970A1 (en) * | 2015-03-17 | 2016-09-22 | Arrowhead Madison Inc. | RNA Interference Agents |
| JOP20210043A1 (ar) * | 2015-10-01 | 2017-06-16 | 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 |
| KR102403408B1 (ko) * | 2016-09-02 | 2022-05-30 | 애로우헤드 파마슈티컬스 인코포레이티드 | 표적화 리간드 |
| WO2018223056A1 (en) * | 2017-06-02 | 2018-12-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| CA3074303A1 (en) * | 2017-09-11 | 2019-03-14 | Arrowhead Pharmaceuticals, Inc. | Rnai agents and compositions for inhibiting expression of apolipoprotein c-iii (apoc3) |
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