JP7033452B2 - 定義されたマルチコンジュゲートオリゴヌクレオチド - Google Patents
定義されたマルチコンジュゲートオリゴヌクレオチド Download PDFInfo
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- JP7033452B2 JP7033452B2 JP2017565893A JP2017565893A JP7033452B2 JP 7033452 B2 JP7033452 B2 JP 7033452B2 JP 2017565893 A JP2017565893 A JP 2017565893A JP 2017565893 A JP2017565893 A JP 2017565893A JP 7033452 B2 JP7033452 B2 JP 7033452B2
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- stranded oligonucleotide
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- 0 **SC(CC(N1**IN(C(C=C2)=O)C2=O)=O)C1=O Chemical compound **SC(CC(N1**IN(C(C=C2)=O)C2=O)=O)C1=O 0.000 description 3
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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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—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
- A61K47/50—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
- A61K47/51—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
- A61K47/54—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 an organic compound
- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—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
- A61K47/50—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
- A61K47/69—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
- A61K47/6905—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion
- A61K47/6911—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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion the form being a liposome
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/10—Dispersions; Emulsions
- A61K9/127—Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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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- 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
- C12N15/115—Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- 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
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- 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
- 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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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/51—Physical structure in polymeric form, e.g. multimers, concatemers
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
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- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Dispersion Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
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Applications Claiming Priority (15)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562175718P | 2015-06-15 | 2015-06-15 | |
| US201562175714P | 2015-06-15 | 2015-06-15 | |
| US201562175709P | 2015-06-15 | 2015-06-15 | |
| US62/175,718 | 2015-06-15 | ||
| US62/175,714 | 2015-06-15 | ||
| US62/175,709 | 2015-06-15 | ||
| US201562203243P | 2015-08-10 | 2015-08-10 | |
| US62/203,243 | 2015-08-10 | ||
| US201562216318P | 2015-09-09 | 2015-09-09 | |
| US201562216317P | 2015-09-09 | 2015-09-09 | |
| US201562216314P | 2015-09-09 | 2015-09-09 | |
| US62/216,317 | 2015-09-09 | ||
| US62/216,314 | 2015-09-09 | ||
| US62/216,318 | 2015-09-09 | ||
| PCT/US2016/037685 WO2016205410A2 (en) | 2015-06-15 | 2016-06-15 | Defined multi-conjugate oligonucleotides |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018518186A JP2018518186A (ja) | 2018-07-12 |
| JP2018518186A5 JP2018518186A5 (enExample) | 2019-08-08 |
| JP7033452B2 true JP7033452B2 (ja) | 2022-03-10 |
Family
ID=57546088
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017565893A Active JP7033452B2 (ja) | 2015-06-15 | 2016-06-15 | 定義されたマルチコンジュゲートオリゴヌクレオチド |
Country Status (11)
| Country | Link |
|---|---|
| US (3) | US11352629B2 (enExample) |
| EP (2) | EP4541374A2 (enExample) |
| JP (1) | JP7033452B2 (enExample) |
| KR (2) | KR20250016526A (enExample) |
| CN (1) | CN108026527B (enExample) |
| AU (3) | AU2016280709B2 (enExample) |
| CA (1) | CA2988603A1 (enExample) |
| HK (1) | HK1252970A1 (enExample) |
| IL (2) | IL316159A (enExample) |
| MX (2) | MX2017016088A (enExample) |
| WO (1) | WO2016205410A2 (enExample) |
Families Citing this family (32)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR101141544B1 (ko) | 2009-03-13 | 2012-05-03 | 한국과학기술원 | 에스아이알엔에이 다중 접합체 및 이의 제조방법 |
| WO2016161388A1 (en) | 2015-04-03 | 2016-10-06 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| IL316159A (en) | 2015-06-15 | 2024-12-01 | Mpeg La Llc | Defined Oligonucleotide Multimers and Methods for Manufacture Thereof |
| WO2017030973A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| JP7749201B6 (ja) * | 2016-01-31 | 2025-10-21 | ユニバーシティー オブ マサチューセッツ | 分岐オリゴヌクレオチド |
| WO2018013525A1 (en) | 2016-07-11 | 2018-01-18 | Translate Bio Ma, Inc. | Nucleic acid conjugates and uses thereof |
| WO2018013640A1 (en) | 2016-07-15 | 2018-01-18 | University Of Massachusetts | Process of delivering small rnas to sperm |
| CA3033368A1 (en) | 2016-08-12 | 2018-02-15 | University Of Massachusetts | Conjugated oligonucleotides |
| SG11201906728TA (en) * | 2017-02-06 | 2019-08-27 | Mpeg La Llc | Multimeric oligonucleotides having decreased kidney clearance |
| CN110799647A (zh) | 2017-06-23 | 2020-02-14 | 马萨诸塞大学 | 双尾自递送sirna及相关的方法 |
| AU2018330495A1 (en) | 2017-09-08 | 2020-03-26 | Mina Therapeutics Limited | Stabilized hnf4a sarna compositions and methods of use |
| WO2019140102A1 (en) | 2018-01-10 | 2019-07-18 | Translate Bio Ma, Inc. | Compositions and methods for facilitating delivery of synthetic nucleic acids to cells |
| CN111886231B (zh) | 2018-04-05 | 2023-08-01 | 株式会社Lg化学 | 基于咔唑的化合物和包含其的有机发光器件 |
| EP3833763A4 (en) | 2018-08-10 | 2023-07-19 | University of Massachusetts | MODIFIED OLIGONUCLEOTIDES FOR TARGETING SNPS |
| US11279930B2 (en) | 2018-08-23 | 2022-03-22 | University Of Massachusetts | O-methyl rich fully stabilized oligonucleotides |
| AU2019346148A1 (en) * | 2018-09-28 | 2021-05-27 | Sirnaomics, Inc. | Multi-targeting nucleic acid constructs composed of multiple oligonucleotides that modulate gene expression through complimentary interactions with targets |
| JP2022523467A (ja) | 2019-01-18 | 2022-04-25 | ユニバーシティ・オブ・マサチューセッツ | 動的な薬物動態を修飾するアンカー |
| JP2022523226A (ja) * | 2019-03-04 | 2022-04-21 | エムペグ エルエイ リミテッド ライアビリティ カンパニー | 増強された生理活性を有する多量体オリゴヌクレオチド |
| WO2020210207A2 (en) * | 2019-04-11 | 2020-10-15 | Northeastern University | Oligonucleotide-polymer miktoarm conjugates and methods of use |
| CA3144467A1 (en) * | 2019-07-30 | 2021-02-04 | Jonathan Miles Brown | Subcutaneous delivery of multimeric oligonucleotides with enhanced bioactivity |
| CN114502730A (zh) | 2019-08-09 | 2022-05-13 | 马萨诸塞大学 | 经化学修饰的靶向snp的寡核苷酸 |
| US12365894B2 (en) | 2019-09-16 | 2025-07-22 | University Of Massachusetts | Branched lipid conjugates of siRNA for specific tissue delivery |
| CN111041025B (zh) | 2019-12-17 | 2021-06-18 | 深圳市瑞吉生物科技有限公司 | 基于结合N-乙酰半乳糖胺多肽的mRNA靶向分子及其制备方法 |
| CN112111524B (zh) * | 2020-01-10 | 2024-02-27 | 深圳瑞吉生物科技有限公司 | mRNA-GalNAc靶向分子的制备方法及其体内递送系统和应用 |
| WO2021222635A2 (en) * | 2020-04-30 | 2021-11-04 | Mpeg La, L.L.C. | Multimeric oligonucleotides with divided strands |
| CA3178559A1 (en) * | 2020-05-19 | 2021-11-25 | Jonathan Miles Brown | Orthogonally linked multimeric oligonucleotides |
| CN111744019B (zh) | 2020-07-01 | 2023-08-04 | 深圳瑞吉生物科技有限公司 | 基于甘露糖的mRNA靶向递送系统及其应用 |
| JP2024506042A (ja) * | 2021-02-08 | 2024-02-08 | ラクティゲン セラピューティクス | 多価オリゴヌクレオチド薬剤およびその使用方法 |
| EP4359539A4 (en) | 2021-06-23 | 2025-05-14 | University Of Massachusetts | ANTI-FLT1 OLIGONUCLEOTIDE COMPOUNDS OPTIMIZED FOR THE TREATMENT OF PREECLAMPSIA AND OTHER ANGIOGENIC DISORDERS |
| CN115010794A (zh) * | 2022-06-30 | 2022-09-06 | 武汉瑞佶生物科技有限公司 | 蛋白质介导的mRNA靶向分子及其制备方法和应用 |
| WO2024061157A1 (en) * | 2022-09-19 | 2024-03-28 | Kylonova (Xiamen) Biopharma Co., Ltd. | Carbohydrate-oligonucleotide conjugates, pharmaceutical compositions, and therapeutic applications |
| WO2024228030A2 (en) | 2023-05-04 | 2024-11-07 | Argonaute RNA Limited | Dual silencing |
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| WO2008109105A2 (en) | 2007-03-06 | 2008-09-12 | Flagship Ventures | Methods and compositions for improved therapeutic effects with sirna |
| JP2011518784A (ja) | 2008-04-11 | 2011-06-30 | アルニラム ファーマスーティカルズ インコーポレイテッド | 標的リガンドをエンドソーム分解性成分と組み合わせることによる核酸の部位特異的送達 |
| US20130064786A1 (en) | 2011-09-14 | 2013-03-14 | Korea Advanced Institute Of Science And Technology | siRNA HYDROGEL AND METHOD FOR MANUFACTURING THE SAME |
| US20140309281A1 (en) | 2009-03-13 | 2014-10-16 | Kaist Ip Co. Ltd. | Multi-conjugate of sirna and preparing method thereof |
| JP2014527819A (ja) | 2011-09-14 | 2014-10-23 | ラナ セラピューティクス インコーポレイテッド | 多量体オリゴヌクレオチド化合物 |
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| US5539082A (en) | 1993-04-26 | 1996-07-23 | Nielsen; Peter E. | Peptide nucleic acids |
| GB9207381D0 (en) | 1992-04-03 | 1992-05-13 | Ici Plc | Synthesis of oligonucleotides |
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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 |
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| EP3307889A4 (en) | 2019-06-26 |
| US20240191240A1 (en) | 2024-06-13 |
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| CN108026527A (zh) | 2018-05-11 |
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| WO2016205410A3 (en) | 2017-02-09 |
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| IL256169B1 (en) | 2025-04-01 |
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| MX2024001378A (es) | 2024-02-27 |
| AU2024278244A1 (en) | 2025-01-02 |
| WO2016205410A2 (en) | 2016-12-22 |
| AU2016280709A1 (en) | 2018-01-04 |
| AU2022283737A1 (en) | 2023-02-02 |
| CA2988603A1 (en) | 2016-12-22 |
| IL256169A (en) | 2018-03-29 |
| EP3307889A2 (en) | 2018-04-18 |
| US11352629B2 (en) | 2022-06-07 |
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| EP4541374A2 (en) | 2025-04-23 |
| AU2022283737B2 (en) | 2024-09-19 |
| KR102761411B1 (ko) | 2025-02-03 |
| US11767531B2 (en) | 2023-09-26 |
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