JP7627042B2 - 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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- JP7627042B2 JP7627042B2 JP2021509991A JP2021509991A JP7627042B2 JP 7627042 B2 JP7627042 B2 JP 7627042B2 JP 2021509991 A JP2021509991 A JP 2021509991A JP 2021509991 A JP2021509991 A JP 2021509991A JP 7627042 B2 JP7627042 B2 JP 7627042B2
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- C—CHEMISTRY; METALLURGY
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- C12Y—ENZYMES
- C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
- C12Y207/10—Protein-tyrosine kinases (2.7.10)
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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
- C12N15/113—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
- C12N15/1137—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 enzymes
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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
- C12N2310/312—Phosphonates
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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
- 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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- 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
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/345—Spatial arrangement of the modifications having at least two different backbone modifications
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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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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- 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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/52—Physical structure branched
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- Biomedical Technology (AREA)
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- Chemical & Material Sciences (AREA)
- General Engineering & Computer Science (AREA)
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- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
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- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2025006720A JP2025063212A (ja) | 2018-08-23 | 2025-01-17 | O-メチルリッチ完全安定化オリゴヌクレオチド |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862721993P | 2018-08-23 | 2018-08-23 | |
| US62/721,993 | 2018-08-23 | ||
| PCT/US2019/048027 WO2020041769A1 (en) | 2018-08-23 | 2019-08-23 | O-methyl rich fully stabilized oligonucleotides |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2025006720A Division JP2025063212A (ja) | 2018-08-23 | 2025-01-17 | O-メチルリッチ完全安定化オリゴヌクレオチド |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021533804A JP2021533804A (ja) | 2021-12-09 |
| JP2021533804A5 JP2021533804A5 (enExample) | 2022-08-31 |
| JP7627042B2 true JP7627042B2 (ja) | 2025-02-05 |
Family
ID=69591135
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021509991A Active JP7627042B2 (ja) | 2018-08-23 | 2019-08-23 | O-メチルリッチ完全安定化オリゴヌクレオチド |
| JP2025006720A Pending JP2025063212A (ja) | 2018-08-23 | 2025-01-17 | 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 |
|---|---|---|---|
| JP2025006720A Pending JP2025063212A (ja) | 2018-08-23 | 2025-01-17 | 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 (2) | US11279930B2 (enExample) |
| EP (1) | EP3840759A4 (enExample) |
| JP (2) | JP7627042B2 (enExample) |
| WO (1) | WO2020041769A1 (enExample) |
Families Citing this family (21)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3929293A3 (en) | 2015-04-03 | 2022-03-16 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| WO2017030973A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| WO2017132669A1 (en) | 2016-01-31 | 2017-08-03 | University Of Massachusetts | Branched oligonucleotides |
| WO2018031933A2 (en) | 2016-08-12 | 2018-02-15 | University Of Massachusetts | Conjugated oligonucleotides |
| WO2018237245A1 (en) | 2017-06-23 | 2018-12-27 | University Of Massachusetts | TWO TAIL SELF-ADMINISTRATION SIRNA AND ASSOCIATED METHODS |
| BR112021002440A2 (pt) | 2018-08-10 | 2021-05-04 | University Of Massachusetts | oligonucleotídeos modificados visando snps |
| WO2020041769A1 (en) | 2018-08-23 | 2020-02-27 | 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 |
| US20210115442A1 (en) * | 2019-08-23 | 2021-04-22 | University Of Massachusetts | O-methyl rich fully stabilized oligonucleotides |
| US12365894B2 (en) | 2019-09-16 | 2025-07-22 | University Of Massachusetts | Branched lipid conjugates of siRNA for specific tissue delivery |
| EP4110913A4 (en) * | 2020-02-28 | 2024-07-24 | University of Massachusetts | OLIGONUCLEOTIDES FOR PRNP MODULATION |
| JP2023518439A (ja) * | 2020-03-18 | 2023-05-01 | ユニバーシティー オブ マサチューセッツ | Mapt調節のためのオリゴヌクレオチド |
| AU2021244600A1 (en) * | 2020-03-26 | 2022-11-03 | University Of Massachusetts | Synthesis of modified oligonucleotides with increased stability |
| CA3174079A1 (en) | 2020-05-26 | 2021-12-02 | Anastasia Khvorova | Synthetic oligonucleotides having regions of block and cluster modifications |
| IL309334A (en) * | 2021-06-23 | 2024-02-01 | Univ Massachusetts | Anti-FLT1 optimized oligonucleotide compounds for the treatment of preeclampsia and other angiogenic disorders |
| WO2023014654A2 (en) * | 2021-08-02 | 2023-02-09 | University Of Massachusetts | Oligonucleotides for htt-1a modulation |
| WO2023081908A2 (en) | 2021-11-08 | 2023-05-11 | University Of Massachusetts | Oligonucleotides for sod1 modulation |
| WO2023087062A1 (en) * | 2021-11-18 | 2023-05-25 | Resonance Health Analysis Services Pty Ltd | Method for treating cyclophilin b associated diseases |
| IL315659A (en) * | 2022-03-16 | 2024-11-01 | Daiichi Sankyo Co Ltd | siRNA FOR SUPPRESSING EXPRESSION OF TRANSFERRIN RECEPTOR-2 |
| TW202528539A (zh) * | 2023-09-20 | 2025-07-16 | 日商第一三共股份有限公司 | 抑制受磷蛋白之表現的siRNA |
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- 2019-08-23 JP JP2021509991A patent/JP7627042B2/ja active Active
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Patent Citations (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20140288148A1 (en) | 2002-02-20 | 2014-09-25 | Sirna Therapeutics Inc | RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA) |
| US20110046206A1 (en) | 2007-06-22 | 2011-02-24 | Isis Pharmaceuticals, Inc. | Double strand compositions comprising differentially modified strands for use in gene modulation |
| JP2017534290A (ja) | 2014-11-10 | 2017-11-24 | 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. | D型肝炎ウイルス(HDV)iRNA組成物及びその使用方法 |
| JP2017538679A (ja) | 2014-11-10 | 2017-12-28 | 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. | B型肝炎ウイルス(HBV)iRNA組成物及びその使用方法 |
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|---|---|
| US20200087663A1 (en) | 2020-03-19 |
| EP3840759A4 (en) | 2022-06-01 |
| US11279930B2 (en) | 2022-03-22 |
| WO2020041769A1 (en) | 2020-02-27 |
| EP3840759A1 (en) | 2021-06-30 |
| US20220251555A1 (en) | 2022-08-11 |
| JP2025063212A (ja) | 2025-04-15 |
| JP2021533804A (ja) | 2021-12-09 |
| US12297430B2 (en) | 2025-05-13 |
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