KR20210111780A - 아밀로이드 전구체 단백질(APP) RNAi 제제 조성물 및 그것의 사용 방법 - Google Patents
아밀로이드 전구체 단백질(APP) RNAi 제제 조성물 및 그것의 사용 방법 Download PDFInfo
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- KR20210111780A KR20210111780A KR1020217022070A KR20217022070A KR20210111780A KR 20210111780 A KR20210111780 A KR 20210111780A KR 1020217022070 A KR1020217022070 A KR 1020217022070A KR 20217022070 A KR20217022070 A KR 20217022070A KR 20210111780 A KR20210111780 A KR 20210111780A
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- 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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- C12N2310/31—Chemical structure of the backbone
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
- C12N2310/3183—Diol linkers, e.g. glycols or propanediols
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- C12N2310/321—2'-O-R Modification
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- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- 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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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- Health & Medical Sciences (AREA)
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- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
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- Psychiatry (AREA)
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- Psychology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862781774P | 2018-12-19 | 2018-12-19 | |
| US62/781,774 | 2018-12-19 | ||
| US201962862472P | 2019-06-17 | 2019-06-17 | |
| US62/862,472 | 2019-06-17 | ||
| US201962928795P | 2019-10-31 | 2019-10-31 | |
| US62/928,795 | 2019-10-31 | ||
| PCT/US2019/067449 WO2020132227A2 (en) | 2018-12-19 | 2019-12-19 | AMYLOID PRECURSOR PROTEIN (APP) RNAi AGENT COMPOSITIONS AND METHODS OF USE THEREOF |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| KR20210111780A true KR20210111780A (ko) | 2021-09-13 |
Family
ID=71101909
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| KR1020217022070A Pending KR20210111780A (ko) | 2018-12-19 | 2019-12-19 | 아밀로이드 전구체 단백질(APP) RNAi 제제 조성물 및 그것의 사용 방법 |
Country Status (20)
| Country | Link |
|---|---|
| US (2) | US11034957B2 (https=) |
| EP (1) | EP3898979A4 (https=) |
| JP (2) | JP2022515193A (https=) |
| KR (1) | KR20210111780A (https=) |
| CN (2) | CN113454222B (https=) |
| AU (1) | AU2019405783B2 (https=) |
| BR (1) | BR112021011895A2 (https=) |
| CA (1) | CA3124090A1 (https=) |
| CL (1) | CL2021001605A1 (https=) |
| CO (1) | CO2021009163A2 (https=) |
| CR (1) | CR20210393A (https=) |
| DO (2) | DOP2021000126A (https=) |
| IL (1) | IL283852A (https=) |
| MX (1) | MX2021007570A (https=) |
| MY (1) | MY206230A (https=) |
| PE (1) | PE20211420A1 (https=) |
| PH (1) | PH12021551482A1 (https=) |
| SG (1) | SG11202105886QA (https=) |
| TW (1) | TW202039844A (https=) |
| WO (1) | WO2020132227A2 (https=) |
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| CN107158445B (zh) | 2012-05-29 | 2021-01-15 | 3M创新有限公司 | 包括聚合物泡沫和中间体的吸收制品 |
| EP3759127A4 (en) | 2018-03-02 | 2022-03-30 | Ionis Pharmaceuticals, Inc. | COMPOUNDS AND METHODS FOR MODULATING AMYLOID BETA PRECURSOR PROTEIN |
| WO2021012082A1 (zh) * | 2019-07-19 | 2021-01-28 | 莫丁丁 | β—淀粉样蛋白环状核糖核酸、多肽及其应用 |
| JP2019213978A (ja) * | 2019-09-30 | 2019-12-19 | 株式会社三洋物産 | 遊技機 |
| AU2021315992A1 (en) * | 2020-07-28 | 2023-02-09 | Ionis Pharmaceuticals, Inc. | Compounds and methods for reducing app expression |
| US20240294906A1 (en) * | 2020-07-29 | 2024-09-05 | Alnylam Pharmaceuticals, Inc. | Atxn2 irna compositions and methods of use thereof for treating or preventing atxn2-associated neurodegenerative diseases |
| EP4284390A4 (en) * | 2021-01-29 | 2025-10-29 | Alnylam Pharmaceuticals Inc | ARNI COMPOSITIONS AND METHODS FOR SILENCER OF AMYLOID PRECURSOR PROTEIN (APP) |
| WO2022187617A1 (en) * | 2021-03-05 | 2022-09-09 | Alnylam Pharmaceuticals, Inc. | Dosing of sirna compounds to the cisterna magna |
| US20240287512A1 (en) * | 2021-05-11 | 2024-08-29 | Dicerna Pharmaceuticals, Inc. | Lipid conjugation for targeting neurons of the central nervous system |
| EP4399311A4 (en) * | 2021-09-10 | 2025-11-26 | Alnylam Pharmaceuticals Inc | APP ARNI COMPOSITIONS AND THEIR METHODS OF USE FOR TREATMENT OR PREVENTION OF DISEASES CHARACTERIZED BY ENLARGED ENDOSOMES |
| KR20240067943A (ko) | 2021-09-24 | 2024-05-17 | 알닐람 파마슈티칼스 인코포레이티드 | 미소관 연관 단백질 타우(MAPT) iRNA 제제 조성물 및 이의 사용 방법 |
| JP2024542125A (ja) * | 2021-10-28 | 2024-11-13 | ザ・ホンコン・ユニバーシティー・オブ・サイエンス・アンド・テクノロジー | アミロイド病変に対する治療アプローチとしてのapp発現のcrispr媒介性操作 |
| TW202335674A (zh) * | 2021-11-05 | 2023-09-16 | 美商黛瑟納製藥公司 | 靶定中樞神經系統的星狀細胞之脂質結合物 |
| WO2023081500A2 (en) * | 2021-11-08 | 2023-05-11 | Dicerna Pharmaceuticals, Inc. | RNAi OLIGONUCLEOTIDE CONJUGATES |
| WO2023173061A2 (en) * | 2022-03-11 | 2023-09-14 | University Of Massachusetts | Oligonucleotides for app modulation |
| WO2023207615A1 (zh) * | 2022-04-26 | 2023-11-02 | 南京明德新药研发有限公司 | 一类含由天然核苷酸组成突出端的双链RNAi化合物 |
| WO2023224979A1 (en) * | 2022-05-16 | 2023-11-23 | University Of Massachusetts | Optimized sirna scaffolds |
| WO2023230465A1 (en) * | 2022-05-23 | 2023-11-30 | Switch Therapeutics Inc. | Antisense oligonucleotides |
| 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 |
| AU2024326438A1 (en) * | 2023-08-22 | 2026-04-02 | Eli Lilly And Company | AMYLOID PRECURSOR PROTEIN (APP) RNAi AGENTS |
| WO2025045195A1 (zh) * | 2023-08-31 | 2025-03-06 | 上海拓界生物医药科技有限公司 | 靶向APP的RNAi剂及其医药用途 |
| AU2024354107A1 (en) * | 2023-09-28 | 2026-04-16 | Bebetter Med Inc. | Sirna inhibiting expression of amyloid precursor protein (app) gene, drug, and use |
| WO2025077711A1 (en) * | 2023-10-10 | 2025-04-17 | Shanghai Argo Biopharmaceutical Co., Ltd. | Compositions and methods for inhibiting expression of amyloid precursor protein (app) |
| WO2025228429A1 (zh) * | 2024-04-30 | 2025-11-06 | 北京安龙生物医药有限公司 | 靶向淀粉样前体蛋白基因的寡核苷酸及其用途 |
| CN118979036B (zh) * | 2024-08-01 | 2025-08-19 | 北京尧景基因技术有限公司 | 抑制APP基因表达的siRNA及其缀合物和应用 |
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| US20080032942A1 (en) * | 2000-08-30 | 2008-02-07 | Mcswiggen James | RNA interference mediated treatment of Alzheimer's disease using short interfering nucleic acid (siNA) |
| US20050209179A1 (en) * | 2000-08-30 | 2005-09-22 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of Alzheimer's disease using short interfering nucleic acid (siNA) |
| WO2004007718A2 (en) | 2002-07-10 | 2004-01-22 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | Rna-interference by single-stranded rna molecules |
| US7851615B2 (en) * | 2003-04-17 | 2010-12-14 | Alnylam Pharmaceuticals, Inc. | Lipophilic conjugated iRNA agents |
| CA2528963A1 (en) * | 2003-06-27 | 2005-01-13 | Sirna Therapeutics, Inc. | Rna interference mediated treatment of alzheimer's disease using short interfering nucleic acid (sina) |
| GB0324854D0 (en) * | 2003-10-24 | 2003-11-26 | Expresson Biosystems Ltd | App/ena antisense |
| EP2239328A3 (en) * | 2005-08-18 | 2011-01-05 | Alnylam Pharmaceuticals Inc. | Methods and compositions for treating neurological disease |
| JP5252622B2 (ja) * | 2007-01-17 | 2013-07-31 | 独立行政法人産業技術総合研究所 | 高いヌクレアーゼ耐性と優れたrna干渉効果を発現可能な二本鎖rna |
| JP5683261B2 (ja) * | 2008-03-11 | 2015-03-11 | 学校法人 埼玉医科大学 | 癌の予防乃至治療に好適な二本鎖核酸分子、癌細胞増殖抑制剤、並びに医薬 |
| EP2510098B1 (en) * | 2009-12-09 | 2015-02-11 | Quark Pharmaceuticals, Inc. | Methods and compositions for treating diseases, disorders or injury of the cns |
| CN103221549A (zh) * | 2010-08-31 | 2013-07-24 | 默沙东公司 | 用于递送寡核苷酸的新型简单化学实体和方法 |
| US9127274B2 (en) * | 2012-04-26 | 2015-09-08 | Alnylam Pharmaceuticals, Inc. | Serpinc1 iRNA compositions and methods of use thereof |
| CN109810977A (zh) * | 2012-05-26 | 2019-05-28 | 株式会社博纳克 | 具有递送功能的基因表达调控用单链核酸分子 |
| US10125369B2 (en) * | 2012-12-05 | 2018-11-13 | Alnylam Pharmaceuticals, Inc. | PCSK9 iRNA compositions and methods of use thereof |
| US10004814B2 (en) * | 2013-11-11 | 2018-06-26 | Sirna Therapeutics, Inc. | Systemic delivery of myostatin short interfering nucleic acids (siNA) conjugated to a lipophilic moiety |
| IL316808A (en) * | 2014-08-20 | 2025-01-01 | Alnylam Pharmaceuticals Inc | Modified double-stranded RNA materials and their uses |
| TWI864340B (zh) * | 2014-10-10 | 2024-12-01 | 美商艾爾妮蘭製藥公司 | 用於抑制hao1(羥酸氧化酶1(乙醇酸鹽氧化酶))基因表現的組合物及方法 |
| TWI710633B (zh) * | 2014-11-10 | 2020-11-21 | 美商阿尼拉製藥公司 | B型肝炎病毒(HBV)iRNA組成物及其用途方法 |
| CA3000046A1 (en) * | 2015-10-16 | 2017-04-20 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for use in treating alzheimer's disease |
| WO2019217459A1 (en) * | 2018-05-07 | 2019-11-14 | Alnylam Pharmaceuticals, Inc. | Extrahepatic delivery |
| UY38562A (es) * | 2019-01-29 | 2020-08-31 | Ionis Pharmaceuticals Inc | Compuestos y métodos para reducir la expresión de app |
| US20220307024A1 (en) * | 2019-06-17 | 2022-09-29 | Alnylam Pharmaceuticals, Inc. | Delivery of oligonucleotides to the striatum |
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2019
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| TW202039844A (zh) | 2020-11-01 |
| BR112021011895A2 (pt) | 2021-09-08 |
| CN113454222B (zh) | 2025-01-14 |
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| SG11202105886QA (en) | 2021-07-29 |
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| PH12021551482A1 (en) | 2022-04-11 |
| US20220002724A1 (en) | 2022-01-06 |
| EP3898979A4 (en) | 2022-10-12 |
| AU2019405783A1 (en) | 2021-07-01 |
| MX2021007570A (es) | 2021-08-24 |
| JP2024113043A (ja) | 2024-08-21 |
| CL2021001605A1 (es) | 2021-12-24 |
| JP2022515193A (ja) | 2022-02-17 |
| MY206230A (en) | 2024-12-05 |
| WO2020132227A3 (en) | 2020-07-30 |
| US11034957B2 (en) | 2021-06-15 |
| US20200339991A1 (en) | 2020-10-29 |
| CO2021009163A2 (es) | 2021-10-29 |
| AU2019405783B2 (en) | 2026-03-26 |
| PE20211420A1 (es) | 2021-08-03 |
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