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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- 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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- 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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- Bioinformatics & Cheminformatics (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 |
| JPWO2020041769A5 JPWO2020041769A5 (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 (18)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP6892433B2 (ja) | 2015-04-03 | 2021-06-23 | ユニバーシティ・オブ・マサチューセッツUniversity Of Massachusetts | 十分に安定化された非対称sirna |
| US10633653B2 (en) | 2015-08-14 | 2020-04-28 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| JP7749201B6 (ja) | 2016-01-31 | 2025-10-21 | ユニバーシティー オブ マサチューセッツ | 分岐オリゴヌクレオチド |
| WO2018031933A2 (en) | 2016-08-12 | 2018-02-15 | University Of Massachusetts | Conjugated oligonucleotides |
| US10844377B2 (en) | 2017-06-23 | 2020-11-24 | University Of Massachusetts | Two-tailed self-delivering siRNA |
| WO2020041769A1 (en) | 2018-08-23 | 2020-02-27 | University Of Massachusetts | O-methyl rich fully stabilized oligonucleotides |
| WO2020150636A1 (en) | 2019-01-18 | 2020-07-23 | University Of Massachusetts | Dynamic pharmacokinetic-modifying anchors |
| MX2022001710A (es) | 2019-08-09 | 2022-05-10 | Univ Massachusetts | Oligonucleótidos modificados químicamente dirigidos a los snp. |
| 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 |
| WO2021173984A2 (en) | 2020-02-28 | 2021-09-02 | University Of Massachusetts | Oligonucleotides for prnp modulation |
| US20210363523A1 (en) * | 2020-03-18 | 2021-11-25 | University Of Massachusetts | Oligonucleotides for mapt modulation |
| AU2021244600A1 (en) * | 2020-03-26 | 2022-11-03 | University Of Massachusetts | Synthesis of modified oligonucleotides with increased stability |
| 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 |
| US20250340883A1 (en) * | 2021-11-18 | 2025-11-06 | Resonance Health Analysis Services Pty Ltd | Method For Treating Cyclophilin B Associated Diseases |
| CN118891366A (zh) * | 2022-03-16 | 2024-11-01 | 第一三共株式会社 | 抑制转铁蛋白受体2的表达的siRNA |
| WO2025063238A1 (ja) * | 2023-09-20 | 2025-03-27 | 第一三共株式会社 | ホスホランバンの発現を抑制するsiRNA |
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| WO2022031591A2 (en) | 2020-08-03 | 2022-02-10 | University Of Massachusetts | Oligonucleotides for htt-1a modulation |
| EP4247391A4 (en) | 2020-11-23 | 2025-11-19 | Univ Massachusetts | OLIGONUCLEOTIDES FOR DGAT2 MODULATION |
| 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 |
| US20230193281A1 (en) | 2021-11-08 | 2023-06-22 | University Of Massachusetts | Oligonucleotides for sod1 modulation |
| US20230313198A1 (en) | 2021-12-17 | 2023-10-05 | University Of Massachusetts | Oligonucleotides for mlh3 modulation |
| WO2023114959A2 (en) | 2021-12-17 | 2023-06-22 | University Of Massachusetts | Oligonucleotides for mlh1 modulation |
| US20230348907A1 (en) | 2022-01-10 | 2023-11-02 | University Of Massachusetts | Oligonucleotides for mecp2 modulation |
| US20230392146A1 (en) | 2022-03-11 | 2023-12-07 | University Of Massachusetts | Oligonucleotides for app modulation |
| US20230416735A1 (en) | 2022-04-14 | 2023-12-28 | University Of Massachusetts | Oligonucleotides for atn1 modulation |
| 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 |
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| 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) |
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| JP2025063212A (ja) | 2025-04-15 |
| US20220251555A1 (en) | 2022-08-11 |
| JP2021533804A (ja) | 2021-12-09 |
| US11279930B2 (en) | 2022-03-22 |
| EP3840759A1 (en) | 2021-06-30 |
| EP3840759A4 (en) | 2022-06-01 |
| US12297430B2 (en) | 2025-05-13 |
| US20200087663A1 (en) | 2020-03-19 |
| WO2020041769A1 (en) | 2020-02-27 |
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