JP2022545118A - O-メチルリッチ完全安定化オリゴヌクレオチド - Google Patents
O-メチルリッチ完全安定化オリゴヌクレオチド Download PDFInfo
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- JP2022545118A JP2022545118A JP2022512459A JP2022512459A JP2022545118A JP 2022545118 A JP2022545118 A JP 2022545118A JP 2022512459 A JP2022512459 A JP 2022512459A JP 2022512459 A JP2022512459 A JP 2022512459A JP 2022545118 A JP2022545118 A JP 2022545118A
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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
- C12N15/111—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
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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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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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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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
- C12N2310/32—Chemical structure of the sugar
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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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C—CHEMISTRY; METALLURGY
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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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- C—CHEMISTRY; METALLURGY
- 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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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- C—CHEMISTRY; METALLURGY
- 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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/52—Physical structure branched
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- 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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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025064358A JP2025123220A (ja) | 2019-08-23 | 2025-04-09 | O-メチルリッチ完全安定化オリゴヌクレオチド |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962891185P | 2019-08-23 | 2019-08-23 | |
| US62/891,185 | 2019-08-23 | ||
| US202062982534P | 2020-02-27 | 2020-02-27 | |
| US62/982,534 | 2020-02-27 | ||
| PCT/US2020/047492 WO2021041247A1 (en) | 2019-08-23 | 2020-08-21 | O-methyl rich fully stabilized oligonucleotides |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025064358A Division JP2025123220A (ja) | 2019-08-23 | 2025-04-09 | O-メチルリッチ完全安定化オリゴヌクレオチド |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022545118A true JP2022545118A (ja) | 2022-10-25 |
| JP2022545118A5 JP2022545118A5 (https=) | 2023-06-30 |
Family
ID=74684325
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022512459A Pending JP2022545118A (ja) | 2019-08-23 | 2020-08-21 | O-メチルリッチ完全安定化オリゴヌクレオチド |
| JP2025064358A Pending JP2025123220A (ja) | 2019-08-23 | 2025-04-09 | O-メチルリッチ完全安定化オリゴヌクレオチド |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025064358A Pending JP2025123220A (ja) | 2019-08-23 | 2025-04-09 | O-メチルリッチ完全安定化オリゴヌクレオチド |
Country Status (4)
| Country | Link |
|---|---|
| US (1) | US20210115442A1 (https=) |
| EP (1) | EP4017504A4 (https=) |
| JP (2) | JP2022545118A (https=) |
| WO (1) | WO2021041247A1 (https=) |
Families Citing this family (15)
| 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 |
| 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 |
| 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 |
| 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 |
| US20210363523A1 (en) * | 2020-03-18 | 2021-11-25 | University Of Massachusetts | Oligonucleotides for mapt modulation |
| WO2021242883A1 (en) | 2020-05-26 | 2021-12-02 | University Of Massachusetts | Synthetic oligonucleotides having regions of block and cluster modifications |
| WO2022204430A1 (en) * | 2021-03-24 | 2022-09-29 | Atalanta Therapeutics, Inc. | Double-stranded sirna having patterned chemical modifications |
| 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 |
| WO2023018705A1 (en) * | 2021-08-10 | 2023-02-16 | University Of Massachusetts | Compositions and methods for sirna treatment of muscular dystrophy |
| JP2025532984A (ja) * | 2022-09-30 | 2025-10-03 | ユニバーシティー オブ マサチューセッツ | オリゴヌクレオチドの眼送達 |
Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2012521766A (ja) * | 2009-03-27 | 2012-09-20 | メルク・シャープ・エンド・ドーム・コーポレイション | 低分子干渉核酸(siNA)を用いた高親和性IgE受容体α鎖(FCεR1α)遺伝子発現のRNA干渉媒介性阻害 |
| JP2016506240A (ja) * | 2012-12-05 | 2016-03-03 | アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. | PCSK9iRNA組成物及びその使用方法 |
| JP2018537952A (ja) * | 2015-10-09 | 2018-12-27 | 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およびその方法 |
| JP2019503394A (ja) * | 2016-01-31 | 2019-02-07 | ユニバーシティ・オブ・マサチューセッツUniversity Of Massachusetts | 分岐オリゴヌクレオチド |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| NL2000439C2 (nl) * | 2006-01-20 | 2009-03-16 | Quark Biotech | Therapeutische toepassingen van inhibitoren van RTP801. |
| AU2007299705B2 (en) * | 2006-09-22 | 2012-09-06 | Dharmacon, Inc. | Duplex oligonucleotide complexes and methods for gene silencing by RNA interference |
| CA2715289C (en) * | 2008-02-11 | 2019-12-24 | Rxi Pharmaceuticals Corporation | Modified rnai polynucleotides and uses thereof |
| WO2012058210A1 (en) * | 2010-10-29 | 2012-05-03 | Merck Sharp & Dohme Corp. | RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (siNA) |
| US20160319278A1 (en) * | 2015-04-03 | 2016-11-03 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| DK3607069T3 (da) * | 2017-04-05 | 2022-11-21 | Silence Therapeutics Gmbh | Produkter og sammensætninger |
| WO2019075419A1 (en) * | 2017-10-13 | 2019-04-18 | Dicerna Pharmaceuticals, Inc. | METHODS AND COMPOSITIONS FOR INHIBITING LDHA EXPRESSION |
| WO2020033899A1 (en) * | 2018-08-10 | 2020-02-13 | University Of Massachusetts | Modified oligonucleotides targeting snps |
| US11279930B2 (en) * | 2018-08-23 | 2022-03-22 | University Of Massachusetts | O-methyl rich fully stabilized oligonucleotides |
| CN113811311A (zh) * | 2019-03-15 | 2021-12-17 | 马萨诸塞大学 | 用于组织特异性apoe调节的寡核苷酸 |
| JP2022528487A (ja) * | 2019-03-29 | 2022-06-13 | ユニバーシティ・オブ・マサチューセッツ | C9orf72のオリゴヌクレオチドベースの調節 |
| KR20220047989A (ko) * | 2019-08-09 | 2022-04-19 | 유니버시티 오브 매사추세츠 | Snp를 표적화하는 화학적으로 변형된 올리고뉴클레오타이드 |
| WO2021195510A1 (en) * | 2020-03-27 | 2021-09-30 | University Of Massachusetts | Dual-acting sirna based modulation of c9orf72 |
| EP4158028A4 (en) * | 2020-05-28 | 2024-10-02 | University Of Massachusetts | OLIGONUCLEOTIDES FOR THE MODULATION OF SARS-COV-2 |
-
2020
- 2020-08-21 WO PCT/US2020/047492 patent/WO2021041247A1/en not_active Ceased
- 2020-08-21 JP JP2022512459A patent/JP2022545118A/ja active Pending
- 2020-08-21 EP EP20856904.6A patent/EP4017504A4/en active Pending
- 2020-08-21 US US16/999,759 patent/US20210115442A1/en active Pending
-
2025
- 2025-04-09 JP JP2025064358A patent/JP2025123220A/ja active Pending
Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2012521766A (ja) * | 2009-03-27 | 2012-09-20 | メルク・シャープ・エンド・ドーム・コーポレイション | 低分子干渉核酸(siNA)を用いた高親和性IgE受容体α鎖(FCεR1α)遺伝子発現のRNA干渉媒介性阻害 |
| JP2016506240A (ja) * | 2012-12-05 | 2016-03-03 | アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. | PCSK9iRNA組成物及びその使用方法 |
| JP2018537952A (ja) * | 2015-10-09 | 2018-12-27 | 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およびその方法 |
| JP2019503394A (ja) * | 2016-01-31 | 2019-02-07 | ユニバーシティ・オブ・マサチューセッツUniversity Of Massachusetts | 分岐オリゴヌクレオチド |
Non-Patent Citations (1)
| Title |
|---|
| 日薬理誌, vol. 148, JPN6025040233, 2016, pages 100 - 104, ISSN: 0005695065 * |
Also Published As
| Publication number | Publication date |
|---|---|
| WO2021041247A1 (en) | 2021-03-04 |
| JP2025123220A (ja) | 2025-08-22 |
| EP4017504A4 (en) | 2024-01-24 |
| US20210115442A1 (en) | 2021-04-22 |
| EP4017504A1 (en) | 2022-06-29 |
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