JP2024539093A - 2’-デオキシ-2’-f-2’-c-メチルヌクレオチドを有するオリゴヌクレオチド - Google Patents
2’-デオキシ-2’-f-2’-c-メチルヌクレオチドを有するオリゴヌクレオチド Download PDFInfo
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- JP2024539093A JP2024539093A JP2024523413A JP2024523413A JP2024539093A JP 2024539093 A JP2024539093 A JP 2024539093A JP 2024523413 A JP2024523413 A JP 2024523413A JP 2024523413 A JP2024523413 A JP 2024523413A JP 2024539093 A JP2024539093 A JP 2024539093A
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- alkyl
- dsrna
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- nucleoside
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- C—CHEMISTRY; METALLURGY
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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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- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
- C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
- C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
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- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
- C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
- C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
- C07H19/06—Pyrimidine radicals
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
- C07H19/02—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof sharing nitrogen
- C07H19/04—Heterocyclic radicals containing only nitrogen atoms as ring hetero atom
- C07H19/16—Purine radicals
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- C—CHEMISTRY; METALLURGY
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- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H23/00—Compounds containing boron, silicon or a metal, e.g. chelates or vitamin B12
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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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- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/312—Phosphonates
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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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-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
- C12N2310/32—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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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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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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- Chemical & Material Sciences (AREA)
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Biomedical Technology (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Engineering & Computer Science (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Saccharide Compounds (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202163257289P | 2021-10-19 | 2021-10-19 | |
| US63/257,289 | 2021-10-19 | ||
| PCT/US2022/047102 WO2023069495A1 (en) | 2021-10-19 | 2022-10-19 | Oligonucleotides with 2'-deoxy-2'-f-2'-c-methyl nucleotides |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2024539093A true JP2024539093A (ja) | 2024-10-28 |
| JP2024539093A5 JP2024539093A5 (https=) | 2025-10-23 |
Family
ID=86059614
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024523413A Pending JP2024539093A (ja) | 2021-10-19 | 2022-10-19 | 2’-デオキシ-2’-f-2’-c-メチルヌクレオチドを有するオリゴヌクレオチド |
Country Status (4)
| Country | Link |
|---|---|
| US (1) | US20250223588A1 (https=) |
| EP (1) | EP4419536A4 (https=) |
| JP (1) | JP2024539093A (https=) |
| WO (1) | WO2023069495A1 (https=) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2024259190A2 (en) * | 2023-06-15 | 2024-12-19 | Alnylam Pharmaceuticals, Inc. | 5'-modified monomers, oligonucleotides and double-stranded rnas |
| CN118063533B (zh) * | 2023-09-18 | 2024-08-27 | 广州必贝特医药股份有限公司 | 修饰的核苷酸化合物、其寡聚核苷酸及其应用 |
| WO2025260011A1 (en) * | 2024-06-14 | 2025-12-18 | Alnylam Pharmaceuticals, Inc. | 5'-modified monomers, oligonucleotides and double-stranded rnas |
| WO2025259992A1 (en) * | 2024-06-14 | 2025-12-18 | Alnylam Pharmaceuticals, Inc. | 5'-modified monomers, oligonucleotides and double-stranded rnas |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2008100447A2 (en) * | 2007-02-09 | 2008-08-21 | Gilead Sciences, Inc. | Nucleoside analogs for antiviral treatment |
| US20110130440A1 (en) * | 2008-03-26 | 2011-06-02 | Alnylam Pharmaceuticals, Inc. | Non-natural ribonucleotides, and methods of use thereof |
| US20120059045A1 (en) * | 2008-10-24 | 2012-03-08 | Isis Pharmaceuticals, Inc. | Methods of using oligomeric compounds comprising 2'-substituted nucleosides |
| AU2010221419B2 (en) * | 2009-03-02 | 2015-10-01 | Alnylam Pharmaceuticals, Inc. | Nucleic acid chemical modifications |
| WO2011005860A2 (en) * | 2009-07-07 | 2011-01-13 | Alnylam Pharmaceuticals, Inc. | 5' phosphate mimics |
| HUE051986T2 (hu) * | 2014-06-24 | 2021-04-28 | Janssen Biopharma Inc | Helyettesített nukleozidok, nukleotidek és analógjaik virális fertõzés kezelésére való alkalmazásra |
| IL316808A (en) * | 2014-08-20 | 2025-01-01 | Alnylam Pharmaceuticals Inc | Modified double-stranded RNA materials and their uses |
| WO2020051398A1 (en) * | 2018-09-06 | 2020-03-12 | Aptamir Therapeutics, Inc. | Metabolic benefits of short mir-22 mirna antagomir therapies |
| US11820985B2 (en) * | 2019-03-26 | 2023-11-21 | University Of Massachusetts | Modified oligonucleotides with increased stability |
| AU2020358016A1 (en) * | 2019-10-02 | 2022-04-21 | Dicerna Pharmaceuticals, Inc. | Chemical modifications of small interfering RNA with minimal fluorine content |
-
2022
- 2022-10-19 WO PCT/US2022/047102 patent/WO2023069495A1/en not_active Ceased
- 2022-10-19 EP EP22884399.1A patent/EP4419536A4/en active Pending
- 2022-10-19 JP JP2024523413A patent/JP2024539093A/ja active Pending
- 2022-10-19 US US18/702,640 patent/US20250223588A1/en active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| EP4419536A4 (en) | 2026-04-15 |
| WO2023069495A1 (en) | 2023-04-27 |
| US20250223588A1 (en) | 2025-07-10 |
| EP4419536A1 (en) | 2024-08-28 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20240716 |
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| A521 | Request for written amendment filed |
Free format text: JAPANESE INTERMEDIATE CODE: A523 Effective date: 20251015 |
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| A621 | Written request for application examination |
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