JP2020522510A - オリゴヌクレオチド組成物及びその使用方法 - Google Patents
オリゴヌクレオチド組成物及びその使用方法 Download PDFInfo
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- JP2020522510A JP2020522510A JP2019566651A JP2019566651A JP2020522510A JP 2020522510 A JP2020522510 A JP 2020522510A JP 2019566651 A JP2019566651 A JP 2019566651A JP 2019566651 A JP2019566651 A JP 2019566651A JP 2020522510 A JP2020522510 A JP 2020522510A
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- Prior art keywords
- oligonucleotide
- composition
- fmfmm
- independently
- oligonucleotides
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
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- 0 CCC(C)=C(C)N(C)*(CCCN(C)*)*(C)C Chemical compound CCC(C)=C(C)N(C)*(CCCN(C)*)*(C)C 0.000 description 70
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- NMILGIZTAZXMTM-UHFFFAOYSA-N CCCN1CCOCC1 Chemical compound CCCN1CCOCC1 NMILGIZTAZXMTM-UHFFFAOYSA-N 0.000 description 2
- OJAWOLWHEQUTDE-UHFFFAOYSA-N Cc1c[n](C)nn1 Chemical compound Cc1c[n](C)nn1 OJAWOLWHEQUTDE-UHFFFAOYSA-N 0.000 description 2
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- KQXRSYAEUNHBEO-UHFFFAOYSA-N CCOC(C(C)(C)CCN1CCOCC1)=O Chemical compound CCOC(C(C)(C)CCN1CCOCC1)=O KQXRSYAEUNHBEO-UHFFFAOYSA-N 0.000 description 1
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- QFDYDSLVRDKIBH-UHFFFAOYSA-N CNC(CCO)=N Chemical compound CNC(CCO)=N QFDYDSLVRDKIBH-UHFFFAOYSA-N 0.000 description 1
- VUYROTORXLFCQN-KFCGZGSFSA-N CNC(COCCC(NCCCN)=O)(COCCC(NCCCN)=O)COCCC(NCCCNC(CCCCO[C@H](C(C1O)O)O[C@H](CO)[C@H]1O)=O)=O Chemical compound CNC(COCCC(NCCCN)=O)(COCCC(NCCCN)=O)COCCC(NCCCNC(CCCCO[C@H](C(C1O)O)O[C@H](CO)[C@H]1O)=O)=O VUYROTORXLFCQN-KFCGZGSFSA-N 0.000 description 1
- DHACZVGAFGZKPQ-UHFFFAOYSA-N COC(C1N(CCSC)CCC1)=O Chemical compound COC(C1N(CCSC)CCC1)=O DHACZVGAFGZKPQ-UHFFFAOYSA-N 0.000 description 1
- FYIRKZMLZLJCLR-UHFFFAOYSA-N COC(C=C1)=CCC1C(NCCCNC(CCO)=O)=O Chemical compound COC(C=C1)=CCC1C(NCCCNC(CCO)=O)=O FYIRKZMLZLJCLR-UHFFFAOYSA-N 0.000 description 1
- MEHOOWGQMMFWHJ-XJGBXTMCSA-N C[C@H]([C@H](C1)OC(C)(C)C)O[C@H]1NC(NC(/C(/C)=C\C)=O)=O Chemical compound C[C@H]([C@H](C1)OC(C)(C)C)O[C@H]1NC(NC(/C(/C)=C\C)=O)=O MEHOOWGQMMFWHJ-XJGBXTMCSA-N 0.000 description 1
- LMYRWZFENFIFIT-UHFFFAOYSA-N Cc(cc1)ccc1S(N)(=O)=O Chemical compound Cc(cc1)ccc1S(N)(=O)=O LMYRWZFENFIFIT-UHFFFAOYSA-N 0.000 description 1
- OPTASPLRGRRNAP-UHFFFAOYSA-N NC(C=CN1)=NC1=O Chemical compound NC(C=CN1)=NC1=O OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 description 1
- RAFOGKOTRLOFKP-UHFFFAOYSA-N NC(CCCCCCCCCCC(O)=O)=O Chemical compound NC(CCCCCCCCCCC(O)=O)=O RAFOGKOTRLOFKP-UHFFFAOYSA-N 0.000 description 1
- YZVWMFBVTTUSAW-UHFFFAOYSA-N SCCN1CCOCC1 Chemical compound SCCN1CCOCC1 YZVWMFBVTTUSAW-UHFFFAOYSA-N 0.000 description 1
- OYKOJVLEGGJKSD-UHFFFAOYSA-N SCN1CCOCC1 Chemical compound SCN1CCOCC1 OYKOJVLEGGJKSD-UHFFFAOYSA-N 0.000 description 1
Images
Classifications
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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
-
- 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
- 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
-
- 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
-
- 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
- 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
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- 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
-
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
-
- 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
- C12N2310/30—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
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/01—Carboxylic ester hydrolases (3.1.1)
- C12Y301/01003—Triacylglycerol lipase (3.1.1.3)
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- Engineering & Computer Science (AREA)
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- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
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- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (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)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023122841A JP2023145620A (ja) | 2017-06-02 | 2023-07-27 | オリゴヌクレオチド組成物及びその使用方法 |
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762514769P | 2017-06-02 | 2017-06-02 | |
| US201762514771P | 2017-06-02 | 2017-06-02 | |
| US62/514,771 | 2017-06-02 | ||
| US62/514,769 | 2017-06-02 | ||
| US201862656949P | 2018-04-12 | 2018-04-12 | |
| US62/656,949 | 2018-04-12 | ||
| US201862670686P | 2018-05-11 | 2018-05-11 | |
| US201862670709P | 2018-05-11 | 2018-05-11 | |
| US62/670,686 | 2018-05-11 | ||
| US62/670,709 | 2018-05-11 | ||
| PCT/US2018/035687 WO2018223056A1 (en) | 2017-06-02 | 2018-06-01 | Oligonucleotide compositions and methods of use thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023122841A Division JP2023145620A (ja) | 2017-06-02 | 2023-07-27 | オリゴヌクレオチド組成物及びその使用方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020522510A true JP2020522510A (ja) | 2020-07-30 |
| JP2020522510A5 JP2020522510A5 (enExample) | 2021-07-26 |
Family
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Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019566651A Pending JP2020522510A (ja) | 2017-06-02 | 2018-06-01 | オリゴヌクレオチド組成物及びその使用方法 |
| JP2023122841A Pending JP2023145620A (ja) | 2017-06-02 | 2023-07-27 | オリゴヌクレオチド組成物及びその使用方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023122841A Pending JP2023145620A (ja) | 2017-06-02 | 2023-07-27 | オリゴヌクレオチド組成物及びその使用方法 |
Country Status (5)
| Country | Link |
|---|---|
| US (2) | US11603532B2 (enExample) |
| EP (1) | EP3630199A4 (enExample) |
| JP (2) | JP2020522510A (enExample) |
| CN (2) | CN111050806A (enExample) |
| WO (1) | WO2018223056A1 (enExample) |
Families Citing this family (71)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2879023C (en) | 2012-07-13 | 2017-03-28 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| CN104661664B (zh) | 2012-07-13 | 2020-07-03 | 波涛生命科学有限公司 | 手性控制 |
| MX2016009290A (es) | 2014-01-16 | 2017-02-28 | Wave Life Sciences Ltd | Diseño quiral. |
| RU2708237C2 (ru) | 2014-08-22 | 2019-12-05 | Общество с ограниченной ответственностью "НооГен" | Модифицированные олигонуклеотиды и способ их получения |
| WO2016161388A1 (en) | 2015-04-03 | 2016-10-06 | University Of Massachusetts | Fully stabilized asymmetric sirna |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| WO2017030973A1 (en) | 2015-08-14 | 2017-02-23 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| SG10201912902TA (en) | 2015-10-09 | 2020-02-27 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods thereof |
| 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 |
| CN114685589A (zh) | 2016-03-13 | 2022-07-01 | 波涛生命科学有限公司 | 用于亚磷酰胺和寡核苷酸合成的组合物和方法 |
| MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| US11013757B2 (en) | 2016-06-03 | 2021-05-25 | Wave Life Sciences Ltd. | Oligonucleotides, compositions and methods thereof |
| WO2017220751A1 (en) | 2016-06-22 | 2017-12-28 | Proqr Therapeutics Ii B.V. | Single-stranded rna-editing oligonucleotides |
| ES2837076T3 (es) | 2016-09-01 | 2021-06-29 | Proqr Therapeutics Ii Bv | Oligonucleótidos para la edición de ARN de cadena sencilla modificados químicamente |
| WO2018098264A1 (en) | 2016-11-23 | 2018-05-31 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
| JOP20190215A1 (ar) | 2017-03-24 | 2019-09-19 | Ionis Pharmaceuticals Inc | مُعدّلات التعبير الوراثي عن pcsk9 |
| WO2018223081A1 (en) | 2017-06-02 | 2018-12-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| WO2018223056A1 (en) | 2017-06-02 | 2018-12-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| JP7402696B2 (ja) | 2017-06-21 | 2023-12-21 | ウェイブ ライフ サイエンシズ リミテッド | 合成のための化合物、組成物、及び方法 |
| CN110799647A (zh) | 2017-06-23 | 2020-02-14 | 马萨诸塞大学 | 双尾自递送sirna及相关的方法 |
| EP3664816A4 (en) | 2017-08-08 | 2021-05-19 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED PROCESSES |
| SG11201912178VA (en) * | 2017-09-11 | 2020-01-30 | Arrowhead Pharmaceuticals Inc | Rnai agents and compositions for inhibiting expression of apolipoprotein c-iii (apoc3) |
| AU2018333065B2 (en) | 2017-09-18 | 2024-04-18 | Wave Life Sciences Ltd. | Technologies for oligonucleotide preparation |
| WO2019075357A1 (en) | 2017-10-12 | 2019-04-18 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS |
| UY38003A (es) * | 2017-12-12 | 2019-06-28 | Amgen Inc | Construcciones de arni para inhibir la expresión de pnpla3 y métodos de uso de las mismas |
| SG11202010910QA (en) | 2018-05-07 | 2020-12-30 | Alnylam Pharmaceuticals Inc | Extrahepatic delivery |
| CA3098624A1 (en) | 2018-05-11 | 2019-11-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| EP3833763A4 (en) | 2018-08-10 | 2023-07-19 | University of Massachusetts | MODIFIED OLIGONUCLEOTIDES FOR TARGETING SNPS |
| US11279930B2 (en) | 2018-08-23 | 2022-03-22 | University Of Massachusetts | O-methyl rich fully stabilized oligonucleotides |
| CN113166761B (zh) * | 2018-12-10 | 2025-09-09 | 美国安进公司 | 用于抑制pnpla3表达的rnai构建体 |
| GB201821269D0 (en) * | 2018-12-28 | 2019-02-13 | Nippon Shinyaku Co Ltd | Myostatin signal inhibitor |
| JP2022523467A (ja) | 2019-01-18 | 2022-04-25 | ユニバーシティ・オブ・マサチューセッツ | 動的な薬物動態を修飾するアンカー |
| US11820985B2 (en) | 2019-03-26 | 2023-11-21 | University Of Massachusetts | Modified oligonucleotides with increased stability |
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| WO2017062862A2 (en) * | 2015-10-09 | 2017-04-13 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
Also Published As
| Publication number | Publication date |
|---|---|
| CN120884604A (zh) | 2025-11-04 |
| US20240132894A1 (en) | 2024-04-25 |
| CN111050806A (zh) | 2020-04-21 |
| EP3630199A1 (en) | 2020-04-08 |
| US20200157545A1 (en) | 2020-05-21 |
| JP2023145620A (ja) | 2023-10-11 |
| WO2018223056A1 (en) | 2018-12-06 |
| EP3630199A4 (en) | 2021-11-10 |
| US11603532B2 (en) | 2023-03-14 |
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