MX2021014769A - Composiciones que comprenden oligonucleotidos modificados reversiblemente, y sus usos. - Google Patents
Composiciones que comprenden oligonucleotidos modificados reversiblemente, y sus usos.Info
- Publication number
- MX2021014769A MX2021014769A MX2021014769A MX2021014769A MX2021014769A MX 2021014769 A MX2021014769 A MX 2021014769A MX 2021014769 A MX2021014769 A MX 2021014769A MX 2021014769 A MX2021014769 A MX 2021014769A MX 2021014769 A MX2021014769 A MX 2021014769A
- Authority
- MX
- Mexico
- Prior art keywords
- glutathione
- sensitive
- moiety
- oligonucleotide
- compositions
- Prior art date
Links
- 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 3
- 239000000203 mixture Substances 0.000 title 1
- RWSXRVCMGQZWBV-WDSKDSINSA-N glutathione Chemical compound OC(=O)[C@@H](N)CCC(=O)N[C@@H](CS)C(=O)NCC(O)=O RWSXRVCMGQZWBV-WDSKDSINSA-N 0.000 abstract 12
- 108010024636 Glutathione Proteins 0.000 abstract 6
- 229960003180 glutathione Drugs 0.000 abstract 6
- 108091034117 Oligonucleotide Proteins 0.000 abstract 4
- 239000002777 nucleoside Substances 0.000 abstract 3
- 239000002773 nucleotide Substances 0.000 abstract 3
- 125000003729 nucleotide group Chemical group 0.000 abstract 3
- 229910052799 carbon Inorganic materials 0.000 abstract 2
- 238000000034 method Methods 0.000 abstract 2
- 239000000178 monomer Substances 0.000 abstract 2
- 150000003833 nucleoside derivatives Chemical class 0.000 abstract 2
- OKTJSMMVPCPJKN-UHFFFAOYSA-N Carbon Chemical group [C] OKTJSMMVPCPJKN-UHFFFAOYSA-N 0.000 abstract 1
- 239000003443 antiviral agent Substances 0.000 abstract 1
- 238000001727 in vivo Methods 0.000 abstract 1
- 239000003112 inhibitor Substances 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- -1 nucleoside phosphoramidites Chemical class 0.000 abstract 1
- 238000002515 oligonucleotide synthesis Methods 0.000 abstract 1
- 150000008300 phosphoramidites Chemical class 0.000 abstract 1
Classifications
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- C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
- C07H19/06—Pyrimidine radicals
- C07H19/10—Pyrimidine radicals with the saccharide radical esterified by phosphoric or polyphosphoric acids
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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
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Abstract
En el presente documento se describen oligonucleótidos sensibles a glutatión y métodos para usarlos. Cualquier oligonucleótido de interés puede modificarse con una fracción sensible a glutatión, incluyendo los oligonucleótidos utilizados para liberación in vivo, tales como moléculas inhibidoras de ácido nucleico. Típicamente, la fracción sensible a glutatión se usa para modificar reversiblemente el carbono 2' de una fracción azúcar en uno o más nucleótidos en el oligonucleótido, aunque otras posiciones de carbono también pueden modificarse con la fracción sensible a glutatión. También se describen monómeros de nucleótidos y nucleósidos sensibles a glutatión, que incluyen fosforamiditos de nucleósido sensibles a glutatión que pueden usarse, por ejemplo, en métodos de síntesis de oligonucleótidos estándar. Además, los monómeros de nucleótidos y nucleósidos sensibles a glutatión sin un fosforamidito se pueden usar terapéuticamente, por ejemplo, como agentes antivirales.
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Families Citing this family (28)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2017321892A1 (en) * | 2016-09-02 | 2019-02-28 | Dicerna Pharmaceuticals, Inc. | 4'-phosphate analogs and oligonucleotides comprising the same |
| WO2019136157A2 (en) | 2018-01-05 | 2019-07-11 | Dicerna Pharmaceuticals, Inc. | Reducing beta-catenin and ido expression to potentiate immunotherapy |
| CA3086409A1 (en) | 2018-01-16 | 2019-07-25 | Dicerna Pharmaceuticals, Inc. | Compositions and methods for inhibiting aldh2 expression |
| SG11202110896WA (en) | 2019-04-04 | 2021-10-28 | Dicerna Pharmaceuticals Inc | Compositions and methods for inhibiting gene expression in the central nervous system |
| JP2023517008A (ja) * | 2020-03-03 | 2023-04-21 | ネクスモス カンパニー リミテッド | グルタチオンに特異的に結合するdnaアプタマー及びその用途 |
| CA3187220A1 (en) | 2020-08-04 | 2022-02-10 | Dicerna Pharmaceuticals, Inc. | Systemic delivery of oligonucleotides |
| WO2022077024A1 (en) | 2020-10-08 | 2022-04-14 | Dicerna Pharmaceuticals, Inc. | Selective delivery of oligonucleotides to glial cells |
| WO2022147223A2 (en) * | 2020-12-31 | 2022-07-07 | Alnylam Pharmaceuticals, Inc. | 2'-modified nucleoside based oligonucleotide prodrugs |
| TWI857290B (zh) | 2021-04-14 | 2024-10-01 | 美商戴瑟納製藥股份有限公司 | 用於調節pnpla3表現之組合物及方法 |
| CA3227661A1 (en) | 2021-08-05 | 2023-02-09 | Weimin Wang | 1'-alkyl modified ribose derivatives and methods of use |
| US20230241224A1 (en) | 2021-09-22 | 2023-08-03 | Sanegene Bio Usa Inc. | 2'-alkyl or 3'- alkyl modified ribose derivatives and methods of use |
| US20230105319A1 (en) * | 2021-09-24 | 2023-04-06 | Crispr Therapeutics Ag | PRODRUG INCORPORATED sgRNA SYNTHESIS |
| WO2023059695A1 (en) | 2021-10-05 | 2023-04-13 | Sanegene Bio Usa Inc. | Polyhydroxylated cyclopentane derivatives and methods of use |
| 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 |
| CA3252536A1 (en) | 2022-02-22 | 2023-08-31 | Sanegene Bio Usa Inc. | CARBOCYCLIC RIBONUCLEOTIDE DERIVATIVES MODIFIED AT THE 5' POSITION AND METHODS OF USE |
| KR20250005108A (ko) | 2022-04-15 | 2025-01-09 | 다이서나 파마수이티컬, 인크. | Scap 활성을 조절하기 위한 조성물 및 방법 |
| WO2023220561A1 (en) | 2022-05-09 | 2023-11-16 | Sanegene Bio Usa Inc. | Double stranded rna targeting 17-beta hydroxysteroiddehydrogenase 13 (hsd17b13) and methods of use thereof |
| AU2023307152A1 (en) | 2022-07-11 | 2025-01-09 | Sanegene Bio Usa Inc. | Optimized 2'- modified ribose derivatives and methods of use |
| US20240052348A1 (en) | 2022-08-05 | 2024-02-15 | Sanegene Bio Usa Inc. | Double stranded rna targeting angiotensinogen (agt) and methods of use thereof |
| AU2023358547A1 (en) | 2022-10-14 | 2025-04-24 | Sanegene Bio Usa Inc. | Small interfering rna targeting c3 and uses thereof |
| WO2024112800A1 (en) * | 2022-11-22 | 2024-05-30 | Ionis Pharmaceuticals, Inc. | Methods for deprotecting and purifying oligonucleotide compounds |
| CN120569477A (zh) | 2022-12-19 | 2025-08-29 | 美国圣因生物股份有限公司 | 靶向cfb的小干扰rna及其用途 |
| WO2024175111A2 (en) | 2023-02-24 | 2024-08-29 | Suzhou Sanegene Bio Inc. | Small interfering rna targeting hbv and uses thereof |
| WO2024186673A2 (en) | 2023-03-03 | 2024-09-12 | Sanegene Bio Usa Inc. | Small interfering rna targeting apoc3 and uses thereof |
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| WO2025024523A1 (en) | 2023-07-24 | 2025-01-30 | Sanegene Bio Usa Inc. | Lipid-based enhancement agent for rna delivery and therapy |
| WO2025228441A2 (en) | 2024-04-30 | 2025-11-06 | Sanegene Bio Usa Inc. | Small interfering rna targeting inhbe and uses thereof |
| WO2026017140A2 (en) | 2024-07-18 | 2026-01-22 | Sanegene Bio Usa Inc. | Small interfering rna targeting fxi and uses thereof |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6114513A (en) * | 1990-01-11 | 2000-09-05 | Isis Pharmaceuticals, Inc. | Thiol-derivatized oligonucleotides |
| EP0537299A1 (en) | 1990-03-29 | 1993-04-21 | Gilead Sciences, Inc. | Oligonucleotide-transport agent disulfide conjugates |
| US7282590B2 (en) | 2004-02-12 | 2007-10-16 | The Research Foundation Of State University Of New York | Drug conjugates |
| US20110008395A1 (en) | 2007-07-06 | 2011-01-13 | Vladimir Torchilin | Mixed micelles including amphipathic conjugates of rna agents, and uses thereof |
| EP3067359A1 (en) | 2008-09-23 | 2016-09-14 | Scott G. Petersen | Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating rna interference |
| KR101885383B1 (ko) | 2009-07-06 | 2018-08-03 | 웨이브 라이프 사이언시스 리미티드 | 신규한 핵산 프로드러그 및 그의 사용 방법 |
| US8927513B2 (en) | 2009-07-07 | 2015-01-06 | Alnylam Pharmaceuticals, Inc. | 5′ phosphate mimics |
| WO2013188607A1 (en) * | 2012-06-14 | 2013-12-19 | The Trustees Of The University Of Pennsylvania | One step n-terminal tagging of proteins |
| WO2014088923A1 (en) | 2012-12-06 | 2014-06-12 | Merck Sharp & Dohme Corp. | Nucleoside kinase bypass compositions and methods |
| CN104903307A (zh) | 2012-12-06 | 2015-09-09 | 默沙东公司 | 二硫化物屏蔽的前体药物组合物和方法 |
| EP2845607A1 (en) | 2013-09-09 | 2015-03-11 | University of Vienna | Antisense oligonucleotides with improved pharmacokinetic properties |
| CN106061981A (zh) | 2013-11-06 | 2016-10-26 | 索尔斯蒂斯生物有限公司 | 具有二硫化物基团的多核苷酸构建体 |
| WO2015188197A2 (en) * | 2014-06-06 | 2015-12-10 | Solstice Biologics, Ltd. | Polynucleotide constructs having bioreversible and non-bioreversible groups |
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