JP6694382B2 - 標的核酸を調節するための組成物および方法 - Google Patents
標的核酸を調節するための組成物および方法 Download PDFInfo
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- JP6694382B2 JP6694382B2 JP2016521893A JP2016521893A JP6694382B2 JP 6694382 B2 JP6694382 B2 JP 6694382B2 JP 2016521893 A JP2016521893 A JP 2016521893A JP 2016521893 A JP2016521893 A JP 2016521893A JP 6694382 B2 JP6694382 B2 JP 6694382B2
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- 0 CCNCC(C1OC)OC(*)C1N Chemical compound CCNCC(C1OC)OC(*)C1N 0.000 description 19
- DFBDVSZENGIVAQ-OIAFMQTASA-N C[C@@H](C(CCC1C(C(C2)C(C)(C3)C(C4)(C5)C3C4OC)C5[O](C)C)C1(C)C2OC)C(C)(C)CC(C)(C)OC Chemical compound C[C@@H](C(CCC1C(C(C2)C(C)(C3)C(C4)(C5)C3C4OC)C5[O](C)C)C1(C)C2OC)C(C)(C)CC(C)(C)OC DFBDVSZENGIVAQ-OIAFMQTASA-N 0.000 description 1
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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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- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
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- 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
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- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/32—Chemical structure of the sugar
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/34—Spatial arrangement of the modifications
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- C12N2310/00—Structure or type of the nucleic acid
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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
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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- C12N2320/30—Special therapeutic applications
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- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance
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- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biomedical Technology (AREA)
- Biochemistry (AREA)
- Organic Chemistry (AREA)
- Public Health (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Obesity (AREA)
- Hematology (AREA)
- Diabetes (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361838190P | 2013-06-21 | 2013-06-21 | |
| US61/838,190 | 2013-06-21 | ||
| US201361871683P | 2013-08-29 | 2013-08-29 | |
| US61/871,683 | 2013-08-29 | ||
| PCT/US2014/043731 WO2014205451A2 (en) | 2013-06-21 | 2014-06-23 | Compositions and methods for modulation of target nucleic acids |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020029006A Division JP2020099335A (ja) | 2013-06-21 | 2020-02-25 | 標的核酸を調節するための組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016523087A JP2016523087A (ja) | 2016-08-08 |
| JP2016523087A5 JP2016523087A5 (enExample) | 2017-08-03 |
| JP6694382B2 true JP6694382B2 (ja) | 2020-05-13 |
Family
ID=52105553
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016521893A Active JP6694382B2 (ja) | 2013-06-21 | 2014-06-23 | 標的核酸を調節するための組成物および方法 |
| JP2020029006A Withdrawn JP2020099335A (ja) | 2013-06-21 | 2020-02-25 | 標的核酸を調節するための組成物および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020029006A Withdrawn JP2020099335A (ja) | 2013-06-21 | 2020-02-25 | 標的核酸を調節するための組成物および方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (3) | US20160122761A1 (enExample) |
| EP (3) | EP3011028B1 (enExample) |
| JP (2) | JP6694382B2 (enExample) |
| AU (2) | AU2014284152B2 (enExample) |
| CA (1) | CA2916252A1 (enExample) |
| WO (1) | WO2014205451A2 (enExample) |
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| RU2015151857A (ru) | 2008-12-02 | 2019-01-15 | Уэйв Лайф Сайенсес Джапан, Инк. | Способ синтеза модифицированных по атому фосфора нуклеиновых кислот |
| MX342945B (es) | 2009-07-06 | 2016-10-18 | Ontorii Inc * | Profármacos de ácido nucleico novedosos y métodos de uso de los mismos. |
| US10428019B2 (en) | 2010-09-24 | 2019-10-01 | Wave Life Sciences Ltd. | Chiral auxiliaries |
| EP3248982A1 (en) | 2011-07-19 | 2017-11-29 | Wave Life Sciences Ltd. | Thiosulfonate reagents for the synthesis of functionalized nucleic acids |
| CA2879023C (en) | 2012-07-13 | 2017-03-28 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| KR102482890B1 (ko) | 2013-05-01 | 2022-12-30 | 아이오니스 파마수티컬즈, 인코포레이티드 | 아포지질단백질 (a) 발현을 조절하는 조성물 및 방법 |
| DK3019200T3 (da) * | 2013-07-11 | 2022-06-20 | Alnylam Pharmaceuticals Inc | Oligonukleotid-ligand-konjugater og fremgangsmåde til fremstiling deraf |
| MX2016009290A (es) | 2014-01-16 | 2017-02-28 | Wave Life Sciences Ltd | Diseño quiral. |
| CN110903337A (zh) | 2014-05-01 | 2020-03-24 | Ionis制药公司 | 用于调节生长激素受体表达的组合物和方法 |
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| WO2015179693A1 (en) | 2014-05-22 | 2015-11-26 | Isis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| EP3151839A4 (en) * | 2014-06-06 | 2018-02-28 | Ionis Pharmaceuticals, Inc. | Compositions and methods for enhanced intestinal absorption of conjugated oligomeric compounds |
| KR20170068469A (ko) | 2014-10-10 | 2017-06-19 | 에프. 호프만-라 로슈 아게 | GalNAc 포스포라미다이트, 그의 핵산 컨쥬게이트 및 그의 용도 |
| US10358497B2 (en) | 2015-09-29 | 2019-07-23 | Amgen Inc. | Methods of treating cardiovascular disease with an ASGR inhibitor |
| CA2999341A1 (en) | 2015-11-06 | 2017-05-11 | Ionis Pharmaceuticals, Inc. | Modulating apolipoprotein (a) expression |
| DK4119569T3 (da) | 2015-11-06 | 2024-08-12 | Ionis Pharmaceuticals Inc | Konjugerede antisense-forbindelser til anvendelse i behandling |
| MA45478A (fr) | 2016-04-11 | 2019-02-20 | Arbutus Biopharma Corp | Compositions de conjugués d'acides nucléiques ciblés |
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| CN108239644B (zh) * | 2016-12-23 | 2021-05-28 | 苏州瑞博生物技术股份有限公司 | 一种小干扰核酸和药物组合物及其用途 |
| KR20250145702A (ko) | 2017-04-11 | 2025-10-13 | 아뷰터스 바이오파마 코포레이션 | 표적화 조성물 |
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| KR102834361B1 (ko) | 2017-12-01 | 2025-07-17 | 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 | 핵산, 이를 포함하는 조성물과 컨쥬게이트, 및 그의 제조 방법과 용도 |
| US11414661B2 (en) | 2017-12-01 | 2022-08-16 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof |
| US11414665B2 (en) | 2017-12-01 | 2022-08-16 | Suzhou Ribo Life Science Co., Ltd. | Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof |
| JP7365052B2 (ja) * | 2017-12-01 | 2023-10-19 | スーチョウ リボ ライフ サイエンス カンパニー、リミテッド | 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用 |
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| JP2021533800A (ja) | 2018-08-21 | 2021-12-09 | スーチョウ リボ ライフ サイエンス カンパニー、リミテッドSuzhou Ribo Life Science Co., Ltd. | 核酸、当該核酸を含む薬物組成物及び複合体ならびにその使用 |
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| EP3011028A2 (en) | 2016-04-27 |
| JP2016523087A (ja) | 2016-08-08 |
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