CN108026527B - 确定的多偶联寡核苷酸 - Google Patents
确定的多偶联寡核苷酸 Download PDFInfo
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- CN108026527B CN108026527B CN201680048179.5A CN201680048179A CN108026527B CN 108026527 B CN108026527 B CN 108026527B CN 201680048179 A CN201680048179 A CN 201680048179A CN 108026527 B CN108026527 B CN 108026527B
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- isolated compound
- cleavable
- oligonucleotides
- stranded
- sirna
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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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- 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
- A61K47/51—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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- C12N15/00—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
- C12N15/113—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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—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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- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biochemistry (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Dispersion Chemistry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (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)
- Medicinal Preparation (AREA)
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 | |
| US62/175,709 | 2015-06-15 | ||
| US62/175,718 | 2015-06-15 | ||
| US62/175,714 | 2015-06-15 | ||
| US201562203243P | 2015-08-10 | 2015-08-10 | |
| US62/203,243 | 2015-08-10 | ||
| US201562216318P | 2015-09-09 | 2015-09-09 | |
| US201562216314P | 2015-09-09 | 2015-09-09 | |
| US201562216317P | 2015-09-09 | 2015-09-09 | |
| US62/216,318 | 2015-09-09 | ||
| US62/216,314 | 2015-09-09 | ||
| US62/216,317 | 2015-09-09 | ||
| PCT/US2016/037685 WO2016205410A2 (en) | 2015-06-15 | 2016-06-15 | Defined multi-conjugate oligonucleotides |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CN108026527A CN108026527A (zh) | 2018-05-11 |
| CN108026527B true CN108026527B (zh) | 2022-05-10 |
Family
ID=57546088
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CN201680048179.5A Active CN108026527B (zh) | 2015-06-15 | 2016-06-15 | 确定的多偶联寡核苷酸 |
Country Status (11)
| Country | Link |
|---|---|
| US (3) | US11352629B2 (https=) |
| EP (2) | EP4541374A3 (https=) |
| JP (1) | JP7033452B2 (https=) |
| KR (2) | KR20250016526A (https=) |
| CN (1) | CN108026527B (https=) |
| AU (3) | AU2016280709B2 (https=) |
| CA (1) | CA2988603A1 (https=) |
| HK (1) | HK1252970A1 (https=) |
| IL (2) | IL316159A (https=) |
| MX (2) | MX2017016088A (https=) |
| WO (1) | WO2016205410A2 (https=) |
Families Citing this family (33)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR101141544B1 (ko) | 2009-03-13 | 2012-05-03 | 한국과학기술원 | 에스아이알엔에이 다중 접합체 및 이의 제조방법 |
| EP3277811B1 (en) | 2015-04-03 | 2020-12-23 | 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 |
| US10633653B2 (en) | 2015-08-14 | 2020-04-28 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| WO2017132669A1 (en) * | 2016-01-31 | 2017-08-03 | University Of Massachusetts | Branched oligonucleotides |
| 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 |
| EP3576752A4 (en) * | 2017-02-06 | 2020-12-16 | Mpeg La, LLC | MULTIMERIC OLIGONUCLEOTIDES WITH REDUCED RENAL CLEARANCE |
| JP7406793B2 (ja) | 2017-06-23 | 2023-12-28 | ユニバーシティー オブ マサチューセッツ | 2テイル自己デリバリー型siRNAおよび関連方法 |
| CA3075205A1 (en) | 2017-09-08 | 2019-03-14 | 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 |
| WO2020033899A1 (en) | 2018-08-10 | 2020-02-13 | University Of Massachusetts | Modified oligonucleotides targeting snps |
| JP7627042B2 (ja) | 2018-08-23 | 2025-02-05 | ユニバーシティー オブ マサチューセッツ | O-メチルリッチ完全安定化オリゴヌクレオチド |
| CN113164507A (zh) * | 2018-09-28 | 2021-07-23 | 圣诺制药公司 | 由多个寡核苷酸构成的通过与靶标的互补相互作用调控基因表达的多靶向核酸构建体 |
| CN113614232A (zh) | 2019-01-18 | 2021-11-05 | 马萨诸塞大学 | 动态药代动力学修饰锚 |
| CA3132505A1 (en) * | 2019-03-04 | 2020-09-10 | Mpeg La, L.L.C. | Multimeric oligonucleotides with enhanced bioactivity |
| US12478684B2 (en) * | 2019-04-11 | 2025-11-25 | Northeastern University | Oligonucleotide-polymer miktoarm conjugates and methods of use |
| AU2020319911A1 (en) * | 2019-07-30 | 2022-02-24 | Mpeg La, L.L.C. | Subcutaneous delivery of multimeric oligonucleotides with enhanced bioactivity |
| MX2022001710A (es) | 2019-08-09 | 2022-05-10 | Univ Massachusetts | Oligonucleótidos modificados químicamente dirigidos a los snp. |
| 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 |
| AU2021263932B2 (en) * | 2020-04-30 | 2025-07-03 | 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 |
| WO2021242883A1 (en) | 2020-05-26 | 2021-12-02 | University Of Massachusetts | Synthetic oligonucleotides having regions of block and cluster modifications |
| 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 |
| US20240318174A1 (en) * | 2021-02-08 | 2024-09-26 | Ractigen Therapeutics | Multi-Valent Oligonucleotide Agent and Methods of Use Thereof |
| AU2022299169A1 (en) | 2021-06-23 | 2024-02-08 | Beth Israel Deaconess Medical Center, Inc. | Optimized anti-flt1 oligonucleotide compounds for 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 |
| JP2025531341A (ja) * | 2022-09-19 | 2025-09-19 | カイロンノヴァ (シャーメン) バイオファーマ カンパニー,リミテッド | 炭水化物-オリゴヌクレオチド複合体、薬物組成物および治療への応用 |
| WO2024228030A2 (en) | 2023-05-04 | 2024-11-07 | Argonaute RNA Limited | Dual silencing |
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| WO2016205410A2 (en) | 2016-12-22 |
| AU2022283737B2 (en) | 2024-09-19 |
| HK1252970A1 (zh) | 2019-06-06 |
| JP7033452B2 (ja) | 2022-03-10 |
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| MX2024001378A (es) | 2024-02-27 |
| US20190085331A1 (en) | 2019-03-21 |
| IL316159A (en) | 2024-12-01 |
| EP3307889A4 (en) | 2019-06-26 |
| KR20250016526A (ko) | 2025-02-03 |
| EP3307889A2 (en) | 2018-04-18 |
| IL256169B2 (en) | 2025-08-01 |
| KR102761411B1 (ko) | 2025-02-03 |
| US20220290148A1 (en) | 2022-09-15 |
| CN108026527A (zh) | 2018-05-11 |
| IL256169A (en) | 2018-03-29 |
| US11767531B2 (en) | 2023-09-26 |
| AU2016280709A1 (en) | 2018-01-04 |
| KR20180039621A (ko) | 2018-04-18 |
| EP4541374A3 (en) | 2026-03-18 |
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| CA2988603A1 (en) | 2016-12-22 |
| AU2016280709B2 (en) | 2022-09-15 |
| AU2024278244A1 (en) | 2025-01-02 |
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