KR20190123351A - 개질된 올리고뉴클레오티드 및 이의 치료 용도 - Google Patents
개질된 올리고뉴클레오티드 및 이의 치료 용도 Download PDFInfo
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- KR20190123351A KR20190123351A KR1020197030864A KR20197030864A KR20190123351A KR 20190123351 A KR20190123351 A KR 20190123351A KR 1020197030864 A KR1020197030864 A KR 1020197030864A KR 20197030864 A KR20197030864 A KR 20197030864A KR 20190123351 A KR20190123351 A KR 20190123351A
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- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Chemical class Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 title abstract description 29
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- A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
- A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
- A61K47/643—Albumins, e.g. HSA, BSA, ovalbumin or a Keyhole Limpet Hemocyanin [KHL]
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- 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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| CN117980003A (zh) * | 2021-09-10 | 2024-05-03 | 嘉德治疗有限责任公司 | 核酸的脂肪酸缀合物 |
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| US8969543B2 (en) * | 2003-04-03 | 2015-03-03 | Bioneer Corporation | SiRNA-hydrophilic polymer conjugates for intracellular delivery of siRNA and method thereof |
| WO2007112414A2 (en) * | 2006-03-27 | 2007-10-04 | Isis Pharmaceuticals, Inc. | Conjugated double strand compositions for use in gene modulation |
| KR101224828B1 (ko) * | 2009-05-14 | 2013-01-22 | (주)바이오니아 | siRNA 접합체 및 그 제조방법 |
| GB0910723D0 (en) * | 2009-06-22 | 2009-08-05 | Sylentis Sau | Novel drugs for inhibition of gene expression |
| CN103649311A (zh) * | 2011-06-03 | 2014-03-19 | 国立大学法人北海道大学 | 寡核苷酸衍生物、包含寡核苷酸衍生物的治疗用医药组合物及诊断用医药组合物、以及miRNA机能抑制用寡核苷酸衍生物 |
| RU2599449C1 (ru) * | 2011-12-15 | 2016-10-10 | Байонир Корпорейшн | Новые конъюгаты олигонуклеотидов и их применение |
| DK3019200T3 (da) * | 2013-07-11 | 2022-06-20 | Alnylam Pharmaceuticals Inc | Oligonukleotid-ligand-konjugater og fremgangsmåde til fremstiling deraf |
| CN105792851B (zh) * | 2013-09-13 | 2023-10-10 | 斯克利普斯研究所 | 修饰的治疗剂及其组合物 |
| AU2014364589B2 (en) * | 2013-12-18 | 2020-02-27 | The California Institute For Biomedical Research | Modified therapeutic agents, stapled peptide lipid conjugates, and compositions thereof |
| JP6773677B2 (ja) * | 2015-03-17 | 2020-10-21 | 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 | 改善されたジスルフィド含有アルキン連結剤 |
| MA41794A (fr) * | 2015-03-18 | 2018-01-23 | The California Institute For Biomedical Res | Agents thérapeutiques modifiés et compositions associées |
| WO2017030973A1 (en) * | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| AU2016326392B2 (en) * | 2015-09-22 | 2021-02-11 | The Regents Of The University Of California | Modified cytotoxins and their therapeutic use |
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| CN110636865A (zh) | 2019-12-31 |
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| SG11201908771YA (en) | 2019-10-30 |
| WO2018175592A1 (en) | 2018-09-27 |
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