JP2022515193A - アミロイド前駆体タンパク質(APP)RNAi薬剤組成物およびその使用方法 - Google Patents
アミロイド前駆体タンパク質(APP)RNAi薬剤組成物およびその使用方法 Download PDFInfo
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- JP2022515193A JP2022515193A JP2021536024A JP2021536024A JP2022515193A JP 2022515193 A JP2022515193 A JP 2022515193A JP 2021536024 A JP2021536024 A JP 2021536024A JP 2021536024 A JP2021536024 A JP 2021536024A JP 2022515193 A JP2022515193 A JP 2022515193A
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- 0 COC[C@](C1)N(*)CC1OC Chemical compound COC[C@](C1)N(*)CC1OC 0.000 description 10
- HXJJFAFABXEKLD-SSDOTTSWSA-N B[C@@H](C1)OC(COC)=C1OC Chemical compound B[C@@H](C1)OC(COC)=C1OC HXJJFAFABXEKLD-SSDOTTSWSA-N 0.000 description 1
- XOJVXYVKUFVBQE-UHFFFAOYSA-N CC[n](cc1)c(cc2)c1cc2[N+]([O-])=O Chemical compound CC[n](cc1)c(cc2)c1cc2[N+]([O-])=O XOJVXYVKUFVBQE-UHFFFAOYSA-N 0.000 description 1
- ORZQZDFOABYORG-UHFFFAOYSA-N COCC(OCC1)=C1OC Chemical compound COCC(OCC1)=C1OC ORZQZDFOABYORG-UHFFFAOYSA-N 0.000 description 1
- AUIBUIVVOOFMLT-UHFFFAOYSA-N COCC(OCC1OC)=C1OC Chemical compound COCC(OCC1OC)=C1OC AUIBUIVVOOFMLT-UHFFFAOYSA-N 0.000 description 1
- AVCGGVFMLBZIGD-JGVFFNPUSA-N COC[C@H]1OCCC[C@@H]1OC Chemical compound COC[C@H]1OCCC[C@@H]1OC AVCGGVFMLBZIGD-JGVFFNPUSA-N 0.000 description 1
- LSKCTDOPSVEIPU-UHFFFAOYSA-N C[n]1c(nc(N)nc2)c2nc1 Chemical compound C[n]1c(nc(N)nc2)c2nc1 LSKCTDOPSVEIPU-UHFFFAOYSA-N 0.000 description 1
- CQYLGSKQLBWLHF-UHFFFAOYSA-N Cc(c(F)c1)cc(C)c1F Chemical compound Cc(c(F)c1)cc(C)c1F CQYLGSKQLBWLHF-UHFFFAOYSA-N 0.000 description 1
- DRJSEGTWOQNMSY-UHFFFAOYSA-N Cc1cccc2c1nc[n]2C Chemical compound Cc1cccc2c1nc[n]2C DRJSEGTWOQNMSY-UHFFFAOYSA-N 0.000 description 1
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024090554A JP2024113043A (ja) | 2018-12-19 | 2024-06-04 | アミロイド前駆体タンパク質(APP)RNAi薬剤組成物およびその使用方法 |
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 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024090554A Division JP2024113043A (ja) | 2018-12-19 | 2024-06-04 | アミロイド前駆体タンパク質(APP)RNAi薬剤組成物およびその使用方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2022515193A true JP2022515193A (ja) | 2022-02-17 |
| JP2022515193A5 JP2022515193A5 (https=) | 2022-12-26 |
| JPWO2020132227A5 JPWO2020132227A5 (https=) | 2022-12-26 |
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Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021536024A Pending JP2022515193A (ja) | 2018-12-19 | 2019-12-19 | アミロイド前駆体タンパク質(APP)RNAi薬剤組成物およびその使用方法 |
| JP2024090554A Pending JP2024113043A (ja) | 2018-12-19 | 2024-06-04 | アミロイド前駆体タンパク質(APP)RNAi薬剤組成物およびその使用方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024090554A Pending JP2024113043A (ja) | 2018-12-19 | 2024-06-04 | アミロイド前駆体タンパク質(APP)RNAi薬剤組成物およびその使用方法 |
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=) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2024518564A (ja) * | 2021-05-11 | 2024-05-01 | 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 | 中枢神経系のニューロンを標的とするための脂質コンジュゲーション |
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| 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 |
Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2005003350A2 (en) * | 2003-06-27 | 2005-01-13 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED TREATMENT OF ALZHEIMER’S DISEASE USING SHORT INTERFERING NUCLEIC ACID (siNA) |
Family Cites Families (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| 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 |
-
2019
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Patent Citations (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2005003350A2 (en) * | 2003-06-27 | 2005-01-13 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED TREATMENT OF ALZHEIMER’S DISEASE USING SHORT INTERFERING NUCLEIC ACID (siNA) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2024518564A (ja) * | 2021-05-11 | 2024-05-01 | 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 | 中枢神経系のニューロンを標的とするための脂質コンジュゲーション |
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