JP2022528840A - 安定性が増加した修飾オリゴヌクレオチド - Google Patents
安定性が増加した修飾オリゴヌクレオチド Download PDFInfo
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- JP2022528840A JP2022528840A JP2021557374A JP2021557374A JP2022528840A JP 2022528840 A JP2022528840 A JP 2022528840A JP 2021557374 A JP2021557374 A JP 2021557374A JP 2021557374 A JP2021557374 A JP 2021557374A JP 2022528840 A JP2022528840 A JP 2022528840A
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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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- 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
- C12N15/1138—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 against receptors or cell surface proteins
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12P—FERMENTATION OR ENZYME-USING PROCESSES TO SYNTHESISE A DESIRED CHEMICAL COMPOUND OR COMPOSITION OR TO SEPARATE OPTICAL ISOMERS FROM A RACEMIC MIXTURE
- C12P21/00—Preparation of peptides or proteins
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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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/34—Allele or polymorphism specific uses
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Saccharide Compounds (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025015181A JP2025072451A (ja) | 2019-03-26 | 2025-01-31 | 安定性が増加した修飾オリゴヌクレオチド |
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962824136P | 2019-03-26 | 2019-03-26 | |
| US62/824,136 | 2019-03-26 | ||
| US201962826454P | 2019-03-29 | 2019-03-29 | |
| US62/826,454 | 2019-03-29 | ||
| US201962864792P | 2019-06-21 | 2019-06-21 | |
| US62/864,792 | 2019-06-21 | ||
| PCT/US2020/025017 WO2020198509A2 (en) | 2019-03-26 | 2020-03-26 | Modified oligonucleotides with increased stability |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025015181A Division JP2025072451A (ja) | 2019-03-26 | 2025-01-31 | 安定性が増加した修飾オリゴヌクレオチド |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022528840A true JP2022528840A (ja) | 2022-06-16 |
| JP2022528840A5 JP2022528840A5 (enExample) | 2023-04-03 |
Family
ID=72609530
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021557374A Pending JP2022528840A (ja) | 2019-03-26 | 2020-03-26 | 安定性が増加した修飾オリゴヌクレオチド |
| JP2025015181A Pending JP2025072451A (ja) | 2019-03-26 | 2025-01-31 | 安定性が増加した修飾オリゴヌクレオチド |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025015181A Pending JP2025072451A (ja) | 2019-03-26 | 2025-01-31 | 安定性が増加した修飾オリゴヌクレオチド |
Country Status (4)
| Country | Link |
|---|---|
| US (2) | US11820985B2 (enExample) |
| EP (1) | EP3946369A4 (enExample) |
| JP (2) | JP2022528840A (enExample) |
| WO (1) | WO2020198509A2 (enExample) |
Families Citing this family (24)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20160319278A1 (en) | 2015-04-03 | 2016-11-03 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| WO2017030973A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| US10478503B2 (en) | 2016-01-31 | 2019-11-19 | University Of Massachusetts | Branched oligonucleotides |
| CA3033368A1 (en) | 2016-08-12 | 2018-02-15 | University Of Massachusetts | Conjugated oligonucleotides |
| CN106987604B (zh) * | 2017-03-29 | 2021-05-28 | 北京希诺谷生物科技有限公司 | 一种制备动脉粥样硬化疾病模型犬的方法 |
| 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 |
| EP3781677A4 (en) | 2018-04-16 | 2022-01-19 | University of Massachusetts | COMPOSITIONS AND METHODS FOR IMPROVED GENE EDITTING |
| US11279930B2 (en) | 2018-08-23 | 2022-03-22 | University Of Massachusetts | O-methyl rich fully stabilized oligonucleotides |
| CN113614232A (zh) | 2019-01-18 | 2021-11-05 | 马萨诸塞大学 | 动态药代动力学修饰锚 |
| US11820985B2 (en) | 2019-03-26 | 2023-11-21 | University Of Massachusetts | Modified oligonucleotides with increased stability |
| KR20220047989A (ko) | 2019-08-09 | 2022-04-19 | 유니버시티 오브 매사추세츠 | Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드 |
| US12365894B2 (en) | 2019-09-16 | 2025-07-22 | University Of Massachusetts | Branched lipid conjugates of siRNA for specific tissue delivery |
| US12339155B2 (en) | 2020-03-25 | 2025-06-24 | Kuwana Metals, Ltd. | Sensor |
| JP7837560B2 (ja) | 2020-03-26 | 2026-03-31 | ユニバーシティー オブ マサチューセッツ | 安定性の向上を有する修飾オリゴヌクレオチドの合成 |
| WO2021242883A1 (en) | 2020-05-26 | 2021-12-02 | University Of Massachusetts | Synthetic oligonucleotides having regions of block and cluster modifications |
| EP4158028A4 (en) | 2020-05-28 | 2024-10-02 | University Of Massachusetts | OLIGONUCLEOTIDES FOR THE MODULATION OF SARS-COV-2 |
| EP4314296A2 (en) | 2021-03-29 | 2024-02-07 | Alnylam Pharmaceuticals, Inc. | Huntingtin (htt) irna agent compositions and methods of use thereof |
| CN117677699A (zh) | 2021-06-23 | 2024-03-08 | 马萨诸塞大学 | 用于治疗先兆子痫和其他血管生成病症的优化抗flt1寡核苷酸化合物 |
| WO2023069495A1 (en) * | 2021-10-19 | 2023-04-27 | Alnylam Pharmaceuticals, Inc. | Oligonucleotides with 2'-deoxy-2'-f-2'-c-methyl nucleotides |
| WO2023114989A2 (en) * | 2021-12-17 | 2023-06-22 | University Of Massachusetts | Oligonucleotides for mlh3 modulation |
| US20240052344A1 (en) * | 2022-05-04 | 2024-02-15 | University Of Massachusetts | Oligonucleotides for pms1 modulation |
| US20250388617A1 (en) * | 2022-06-27 | 2025-12-25 | Rona Bioscience, Limited | Double-stranded rna comprising nucleotide analog capable of reducing off-target toxicity |
| WO2025264952A2 (en) * | 2024-06-21 | 2025-12-26 | Eli Lilly And Company | Novel rna therapeutics and uses thereof |
| WO2026006109A2 (en) * | 2024-06-24 | 2026-01-02 | Eli Lilly And Company | Novel rna therapeutics and uses thereof |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
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| WO2010033247A2 (en) * | 2008-09-22 | 2010-03-25 | Rxi Pharmaceuticals Corporation | Reduced size self-delivering rnai compounds |
| JP2018516091A (ja) * | 2015-04-03 | 2018-06-21 | ユニバーシティ・オブ・マサチューセッツUniversity Of Massachusetts | ハンチンチンmRNAをターゲティングするオリゴヌクレオチド化合物 |
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| US5610289A (en) | 1990-07-27 | 1997-03-11 | Isis Pharmaceuticals, Inc. | Backbone modified oligonucleotide analogues |
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| JPH0641183A (ja) | 1992-07-23 | 1994-02-15 | Mitsubishi Kasei Corp | オリゴヌクレオチド単分子膜 |
| US6291438B1 (en) | 1993-02-24 | 2001-09-18 | Jui H. Wang | Antiviral anticancer poly-substituted phenyl derivatized oligoribonucleotides and methods for their use |
| WO1994022890A1 (en) | 1993-03-31 | 1994-10-13 | Sterling Winthop Inc. | Novel 5'-substituted nucleosides and oligomers produced therefrom |
| WO1996003500A1 (en) | 1994-07-26 | 1996-02-08 | Ltt Institute Co., Ltd. | Sustance with antiviral activity |
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| US20100240730A1 (en) * | 2002-02-20 | 2010-09-23 | Merck Sharp And Dohme Corp. | RNA Interference Mediated Inhibition of Gene Expression Using Chemically Modified Short Interfering Nucleic Acid (siNA) |
| AU2003285071A1 (en) | 2002-10-30 | 2004-06-07 | Nikolaos Avlonitis | Antiprotozoal ring-substituted phospholipids |
| DE10326302A1 (de) | 2003-06-11 | 2004-12-30 | Gnothis Holding Sa | Bi-Fluorophor-markierte Sonden zum Nachweis von Nukleinsäuren |
| US20050176045A1 (en) | 2004-02-06 | 2005-08-11 | Dharmacon, Inc. | SNP discriminatory siRNA |
| EP1789553B1 (en) | 2004-06-30 | 2014-03-26 | Alnylam Pharmaceuticals Inc. | Oligonucleotides comprising a non-phosphate backbone linkage |
| US8309356B2 (en) * | 2009-04-01 | 2012-11-13 | Yale University | Pseudocomplementary oligonucleotides for targeted gene therapy |
| WO2010118263A1 (en) | 2009-04-08 | 2010-10-14 | University Of Massachusetts | Single-nucleotide polymorphism (snp) targeting therapies for the treatment of huntington's disease |
| US8957040B2 (en) | 2010-02-08 | 2015-02-17 | Isis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
| WO2011125943A1 (ja) | 2010-04-01 | 2011-10-13 | 日本新薬株式会社 | 修飾オリゴヌクレオチド |
| RU2013132681A (ru) | 2010-12-16 | 2015-01-27 | Вертекс Фармасьютикалз Инкорпорейтед | Ингибиторы репликации вирусов гриппа |
| CA2822059A1 (en) | 2010-12-16 | 2012-06-21 | Vertex Pharmaceuticals Incorporated | Inhibitors of influenza viruses replication |
| CN103562205A (zh) | 2010-12-16 | 2014-02-05 | 沃泰克斯药物股份有限公司 | 流感病毒复制的抑制剂 |
| GB201105137D0 (en) | 2011-03-28 | 2011-05-11 | Isis Innovation | Therapeutic molecules for use in the suppression of Parkinson's disease |
| KR102104125B1 (ko) | 2011-04-21 | 2020-05-29 | 재단법인 한국파스퇴르연구소 | 소염 화합물 |
| EP3007705A4 (en) * | 2013-06-12 | 2017-02-15 | Oncoimmunin, Inc. | Systemic in vivo delivery of oligonucleotides |
| WO2015113004A2 (en) | 2014-01-27 | 2015-07-30 | The Board Of Trustee Of The Leland Stanford Junior University | Oligonucleotides and methods for treatment of cardiomyopathy using rna interference |
| US20170051286A1 (en) | 2014-05-01 | 2017-02-23 | Larry J. Smith | METHODS AND MODIFICATIONS THAT PRODUCE ssRNAi COMPOUNDS WITH ENHANCED ACTIVITY, POTENCY AND DURATION OF EFFECT |
| US10478503B2 (en) * | 2016-01-31 | 2019-11-19 | University Of Massachusetts | Branched oligonucleotides |
| CN110352244B (zh) * | 2016-09-01 | 2023-03-21 | ProQR治疗上市公司Ⅱ | 化学修饰的编辑rna的单链寡核苷酸 |
| JP2020522510A (ja) | 2017-06-02 | 2020-07-30 | ウェイブ ライフ サイエンシズ リミテッドWave Life Sciences Ltd. | オリゴヌクレオチド組成物及びその使用方法 |
| WO2020033899A1 (en) * | 2018-08-10 | 2020-02-13 | University Of Massachusetts | Modified oligonucleotides targeting snps |
| US11820985B2 (en) | 2019-03-26 | 2023-11-21 | University Of Massachusetts | Modified oligonucleotides with increased stability |
| ES3014076T3 (en) | 2020-03-26 | 2025-04-16 | Nutrivert Inc | Lipophilic enantiomers of desacetylglucosamine muramyl dipeptide with anti-inflammatory and growth promoting activity |
| JP7837560B2 (ja) | 2020-03-26 | 2026-03-31 | ユニバーシティー オブ マサチューセッツ | 安定性の向上を有する修飾オリゴヌクレオチドの合成 |
-
2020
- 2020-03-26 US US16/831,470 patent/US11820985B2/en active Active
- 2020-03-26 WO PCT/US2020/025017 patent/WO2020198509A2/en not_active Ceased
- 2020-03-26 JP JP2021557374A patent/JP2022528840A/ja active Pending
- 2020-03-26 EP EP20777915.8A patent/EP3946369A4/en active Pending
-
2023
- 2023-09-01 US US18/459,992 patent/US20240141362A1/en active Pending
-
2025
- 2025-01-31 JP JP2025015181A patent/JP2025072451A/ja active Pending
Patent Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2010033247A2 (en) * | 2008-09-22 | 2010-03-25 | Rxi Pharmaceuticals Corporation | Reduced size self-delivering rnai compounds |
| JP2018516091A (ja) * | 2015-04-03 | 2018-06-21 | ユニバーシティ・オブ・マサチューセッツUniversity Of Massachusetts | ハンチンチンmRNAをターゲティングするオリゴヌクレオチド化合物 |
Non-Patent Citations (1)
| Title |
|---|
| MOLECULAR THERAPY-NUCLEIC ACIDS, vol. 4, no. 12, JPN5018004887, 1 December 2015 (2015-12-01), pages 266 - 1, ISSN: 0005285953 * |
Also Published As
| Publication number | Publication date |
|---|---|
| WO2020198509A3 (en) | 2020-11-05 |
| WO2020198509A2 (en) | 2020-10-01 |
| JP2025072451A (ja) | 2025-05-09 |
| US20240141362A1 (en) | 2024-05-02 |
| EP3946369A2 (en) | 2022-02-09 |
| EP3946369A4 (en) | 2023-10-18 |
| US20200385740A1 (en) | 2020-12-10 |
| US11820985B2 (en) | 2023-11-21 |
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