JP2022513719A - オリゴヌクレオチド組成物及びその方法 - Google Patents
オリゴヌクレオチド組成物及びその方法 Download PDFInfo
- Publication number
- JP2022513719A JP2022513719A JP2021531966A JP2021531966A JP2022513719A JP 2022513719 A JP2022513719 A JP 2022513719A JP 2021531966 A JP2021531966 A JP 2021531966A JP 2021531966 A JP2021531966 A JP 2021531966A JP 2022513719 A JP2022513719 A JP 2022513719A
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- oligonucleotide
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- 0 CC(C)C(C(*)(*)N*)O Chemical compound CC(C)C(C(*)(*)N*)O 0.000 description 42
- WTHODQCXBLEWGP-UHFFFAOYSA-N CN(CCN1C)C1=NP(OC)(ON)=O Chemical compound CN(CCN1C)C1=NP(OC)(ON)=O WTHODQCXBLEWGP-UHFFFAOYSA-N 0.000 description 13
- QGURGJVPHMAYNR-UHFFFAOYSA-N CN(CCN1C)C1=NP(O)(OC)=O Chemical compound CN(CCN1C)C1=NP(O)(OC)=O QGURGJVPHMAYNR-UHFFFAOYSA-N 0.000 description 4
- XIDBUSQQDWWWQX-UHFFFAOYSA-N CN(CCN1C)C1=NP(OC)(OC)=O Chemical compound CN(CCN1C)C1=NP(OC)(OC)=O XIDBUSQQDWWWQX-UHFFFAOYSA-N 0.000 description 2
- LRSKGUZXYPFDQF-UHFFFAOYSA-N CN(CCN1C)C1=NP(OC)(OI)=O Chemical compound CN(CCN1C)C1=NP(OC)(OI)=O LRSKGUZXYPFDQF-UHFFFAOYSA-N 0.000 description 2
- GQWUGGVBSJXIPC-UHFFFAOYSA-N CC(C)(C)OP(N=C1N(C)CCN1C)(ONC)=O Chemical compound CC(C)(C)OP(N=C1N(C)CCN1C)(ONC)=O GQWUGGVBSJXIPC-UHFFFAOYSA-N 0.000 description 1
- YLIVONCKCFURIN-DTWKUNHWSA-N CC(C)(C)S(C[C@H]([C@H]1NCCC1)O)(=O)=O Chemical compound CC(C)(C)S(C[C@H]([C@H]1NCCC1)O)(=O)=O YLIVONCKCFURIN-DTWKUNHWSA-N 0.000 description 1
- LJAMGYRJFWFUGK-DTWKUNHWSA-N CC(C)S(C[C@H]([C@H]1NCCC1)O)(=O)=O Chemical compound CC(C)S(C[C@H]([C@H]1NCCC1)O)(=O)=O LJAMGYRJFWFUGK-DTWKUNHWSA-N 0.000 description 1
- UXUKBISZKZUIQA-UHFFFAOYSA-N CC1=[N+](C)CCN1C Chemical compound CC1=[N+](C)CCN1C UXUKBISZKZUIQA-UHFFFAOYSA-N 0.000 description 1
- OMJADKVVRHOCAC-NKWVEPMBSA-N CS(C[C@H]([C@H]1NCCC1)O)(=O)=O Chemical compound CS(C[C@H]([C@H]1NCCC1)O)(=O)=O OMJADKVVRHOCAC-NKWVEPMBSA-N 0.000 description 1
- JCDWETOKTFWTHA-UHFFFAOYSA-N CS(c1ccccc1)(=O)=O Chemical compound CS(c1ccccc1)(=O)=O JCDWETOKTFWTHA-UHFFFAOYSA-N 0.000 description 1
- JXNHAIBFGMLQOZ-ULUSZKPHSA-N C[C@H](CC1CNCC1)O Chemical compound C[C@H](CC1CNCC1)O JXNHAIBFGMLQOZ-ULUSZKPHSA-N 0.000 description 1
- CSLPJINBPQCCEM-RBUKOAKNSA-N C[Si](C[C@H]1OPN2[C@H]1CCC2)(c1ccccc1)c1ccccc1 Chemical compound C[Si](C[C@H]1OPN2[C@H]1CCC2)(c1ccccc1)c1ccccc1 CSLPJINBPQCCEM-RBUKOAKNSA-N 0.000 description 1
- PCWWMYGSKVMIDU-UONOGXRCSA-N Cc(cc1C)cc(C)c1S(C[C@H]([C@H]1NCCC1)O)(=O)=O Chemical compound Cc(cc1C)cc(C)c1S(C[C@H]([C@H]1NCCC1)O)(=O)=O PCWWMYGSKVMIDU-UONOGXRCSA-N 0.000 description 1
- XBAIQYGAAFCXPL-QWHCGFSZSA-N Cc(cc1C)ccc1S(C[C@H]([C@H]1NCCC1)O)(=O)=O Chemical compound Cc(cc1C)ccc1S(C[C@H]([C@H]1NCCC1)O)(=O)=O XBAIQYGAAFCXPL-QWHCGFSZSA-N 0.000 description 1
- INEMHABDFCKBID-UHFFFAOYSA-N Cc(ccc(C#N)c1)c1Cl Chemical compound Cc(ccc(C#N)c1)c1Cl INEMHABDFCKBID-UHFFFAOYSA-N 0.000 description 1
- CKOARSKDIJCHBN-QWHCGFSZSA-N N#Cc(cc1)ccc1S(C[C@H]([C@H]1NCCC1)O)(=O)=O Chemical compound N#Cc(cc1)ccc1S(C[C@H]([C@H]1NCCC1)O)(=O)=O CKOARSKDIJCHBN-QWHCGFSZSA-N 0.000 description 1
- NWABAORXDMOINC-RYUDHWBXSA-N O[C@@H](CS(c1ccccc1)(=O)=O)[C@H]1NCCC1 Chemical compound O[C@@H](CS(c1ccccc1)(=O)=O)[C@H]1NCCC1 NWABAORXDMOINC-RYUDHWBXSA-N 0.000 description 1
- XGYWUOCODIWYIA-QWHCGFSZSA-N O[C@H](CS(Cc1ccccc1)(=O)=O)[C@H]1NCCC1 Chemical compound O[C@H](CS(Cc1ccccc1)(=O)=O)[C@H]1NCCC1 XGYWUOCODIWYIA-QWHCGFSZSA-N 0.000 description 1
- KPZQEHNSYBBWGQ-GFCCVEGCSA-N O[C@H](CS(c1ccccc1)(=O)=O)C1=NCCC1 Chemical compound O[C@H](CS(c1ccccc1)(=O)=O)C1=NCCC1 KPZQEHNSYBBWGQ-GFCCVEGCSA-N 0.000 description 1
Images
Classifications
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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
- 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
-
- 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
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
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- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Physics & Mathematics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024209791A JP2025041652A (ja) | 2018-12-06 | 2024-12-02 | オリゴヌクレオチド組成物及びその方法 |
Applications Claiming Priority (11)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862776432P | 2018-12-06 | 2018-12-06 | |
| US62/776,432 | 2018-12-06 | ||
| PCT/US2019/027109 WO2019200185A1 (en) | 2018-04-12 | 2019-04-11 | Oligonucleotide compositions and methods of use thereof |
| USPCT/US2019/027109 | 2019-04-11 | ||
| USPCT/US2019/031672 | 2019-05-10 | ||
| PCT/US2019/031672 WO2019217784A1 (en) | 2018-05-11 | 2019-05-10 | Oligonucleotide compositions and methods of use thereof |
| US201962916194P | 2019-10-16 | 2019-10-16 | |
| US201962916192P | 2019-10-16 | 2019-10-16 | |
| US62/916,192 | 2019-10-16 | ||
| US62/916,194 | 2019-10-16 | ||
| PCT/US2019/065058 WO2020118246A1 (en) | 2018-12-06 | 2019-12-06 | Oligonucleotide compositions and methods thereof |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024209791A Division JP2025041652A (ja) | 2018-12-06 | 2024-12-02 | オリゴヌクレオチド組成物及びその方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022513719A true JP2022513719A (ja) | 2022-02-09 |
| JPWO2020118246A5 JPWO2020118246A5 (enExample) | 2023-03-09 |
Family
ID=70973999
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021531966A Ceased JP2022513719A (ja) | 2018-12-06 | 2019-12-06 | オリゴヌクレオチド組成物及びその方法 |
| JP2024209791A Pending JP2025041652A (ja) | 2018-12-06 | 2024-12-02 | オリゴヌクレオチド組成物及びその方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024209791A Pending JP2025041652A (ja) | 2018-12-06 | 2024-12-02 | オリゴヌクレオチド組成物及びその方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (1) | US20220186217A1 (enExample) |
| EP (1) | EP3891284A4 (enExample) |
| JP (2) | JP2022513719A (enExample) |
| CN (1) | CN113383078A (enExample) |
| AU (1) | AU2019392928A1 (enExample) |
| CA (1) | CA3122271A1 (enExample) |
| SG (1) | SG11202105626TA (enExample) |
| WO (1) | WO2020118246A1 (enExample) |
Families Citing this family (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2872485B1 (en) | 2012-07-13 | 2020-12-16 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| EP2872147B1 (en) | 2012-07-13 | 2022-12-21 | Wave Life Sciences Ltd. | Method for making chiral oligonucleotides |
| CN106068325B (zh) | 2014-01-16 | 2021-07-09 | 波涛生命科学有限公司 | 手性设计 |
| RU2708237C2 (ru) | 2014-08-22 | 2019-12-05 | Общество с ограниченной ответственностью "НооГен" | Модифицированные олигонуклеотиды и способ их получения |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| MA43822A (fr) | 2016-03-13 | 2018-11-28 | Wave Life Sciences Ltd | Compositions et procédés de synthèse de phosphoramidite et d'oligonucléotides |
| JP2019520339A (ja) | 2016-06-03 | 2019-07-18 | 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、その組成物および方法 |
| CN109477103A (zh) | 2016-06-22 | 2019-03-15 | ProQR治疗上市公司Ⅱ | 单链rna-编辑寡核苷酸 |
| NZ751483A (en) | 2016-09-01 | 2022-07-01 | Proqr Therapeutics Ii Bv | Chemically modified single-stranded rna-editing oligonucleotides |
| US11873316B2 (en) | 2016-11-23 | 2024-01-16 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
| US11603532B2 (en) | 2017-06-02 | 2023-03-14 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| TW201904587A (zh) | 2017-06-02 | 2019-02-01 | 新加坡商波濤生命科學有限公司 | 寡核苷酸組合物及其使用方法 |
| CN111051281A (zh) | 2017-06-21 | 2020-04-21 | 波涛生命科学有限公司 | 用于合成的化合物、组合物和方法 |
| JP2020534253A (ja) | 2017-08-08 | 2020-11-26 | ウェーブ ライフ サイエンシーズ リミテッド | オリゴヌクレオチド組成物及びその方法 |
| KR20200052369A (ko) | 2017-09-18 | 2020-05-14 | 웨이브 라이프 사이언시스 리미티드 | 올리고뉴클레오티드 제조 기술 |
| US11596646B2 (en) | 2017-10-12 | 2023-03-07 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| AU2019265904A1 (en) | 2018-05-11 | 2020-11-12 | 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 |
| SG11202100934PA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11168141B2 (en) | 2018-08-02 | 2021-11-09 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| US20230089442A1 (en) * | 2019-03-20 | 2023-03-23 | Pachamuthu Kandasamy | Technologies useful for oligonucleotide preparation |
| AU2020395113A1 (en) | 2019-12-02 | 2022-06-09 | Shape Therapeutics Inc. | Therapeutic editing |
| US11969475B2 (en) | 2021-07-09 | 2024-04-30 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US11771776B2 (en) | 2021-07-09 | 2023-10-03 | Dyne Therapeutics, Inc. | Muscle targeting complexes and uses thereof for treating dystrophinopathies |
| WO2023154528A1 (en) * | 2022-02-11 | 2023-08-17 | Wave Life Sciences Ltd. | Stereoselective technologies for chiral compounds |
| EP4479535A1 (en) | 2022-02-14 | 2024-12-25 | ProQR Therapeutics II B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| EP4555085A1 (en) | 2022-07-15 | 2025-05-21 | ProQR Therapeutics II B.V. | Chemically modified oligonucleotides for adar-mediated rna editing |
| JP2025524566A (ja) | 2022-07-15 | 2025-07-30 | プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ | Ａｄａｒ媒介ｒｎａ編集のためのオリゴヌクレオチドおよびその使用 |
| GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
| KR20250113455A (ko) | 2022-11-24 | 2025-07-25 | 프로큐알 테라퓨틱스 Ⅱ 비.브이. | 유전성 hfe-혈색소침착증의 치료를 위한 안티센스 올리고뉴클레오티드 |
| GB202218090D0 (en) | 2022-12-01 | 2023-01-18 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency |
| KR20250115388A (ko) | 2022-12-09 | 2025-07-30 | 프로큐알 테라퓨틱스 Ⅱ 비.브이. | 심혈관 질환 치료용 안티센스 올리고뉴클레오타이드 |
| GB202300865D0 (en) | 2023-01-20 | 2023-03-08 | Proqr Therapeutics Ii Bv | Delivery of oligonucleotides |
| EP4669753A1 (en) | 2023-02-20 | 2025-12-31 | ProQR Therapeutics II B.V. | Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease |
| GB202304363D0 (en) | 2023-03-24 | 2023-05-10 | Proqr Therapeutics Ii Bv | Chemically modified antisense oligonucleotides for use in RNA editing |
| WO2024206175A1 (en) | 2023-03-24 | 2024-10-03 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of neurological disorders |
| WO2024200472A1 (en) | 2023-03-27 | 2024-10-03 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of liver disease |
| TW202516003A (zh) | 2023-06-16 | 2025-04-16 | 荷蘭商Ｐｒｏｑｒ治療上市公司Ｉｉ | 用於治療神經退化性疾病之反義寡核苷酸 |
| WO2025051946A1 (en) | 2023-09-07 | 2025-03-13 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of metabolic disorders |
| WO2025072881A2 (en) * | 2023-09-28 | 2025-04-03 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| WO2025072879A1 (en) * | 2023-09-28 | 2025-04-03 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| WO2025104239A1 (en) | 2023-11-16 | 2025-05-22 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of classic galactosemia |
| WO2025132708A1 (en) | 2023-12-20 | 2025-06-26 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of huntington's disease |
| GB202404661D0 (en) | 2024-04-02 | 2024-05-15 | Proqr Therapeutics Ii Bv | Antisense oligoncleotides for the treatment of liver disease |
| WO2025213142A2 (en) * | 2024-04-05 | 2025-10-09 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| WO2025224230A1 (en) | 2024-04-25 | 2025-10-30 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of fatty liver disease |
Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2003025139A2 (en) * | 2001-09-17 | 2003-03-27 | Mayo Foundation For Medical Education And Research | Cobalamin mediated delivery of nucleic acids, analogs and derivatives thereof |
| JP2017527277A (ja) * | 2014-08-22 | 2017-09-21 | ノーゲン エルエルシー | 修飾オリゴヌクレオチドおよびそれらの合成のための方法 |
| WO2018067973A1 (en) * | 2015-10-09 | 2018-04-12 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6617442B1 (en) * | 1999-09-30 | 2003-09-09 | Isis Pharmaceuticals, Inc. | Human Rnase H1 and oligonucleotide compositions thereof |
| HUE028662T2 (en) * | 2007-10-26 | 2016-12-28 | Academisch Ziekenhuis Leiden | Preparations and methods for controlling muscle disorders |
| PT2344637E (pt) * | 2008-10-27 | 2015-03-23 | Academisch Ziekenhuis Leiden | Métodos e meios para o salto eficiente do exão 45 no pré-mrna de distrofia muscular de duchenne |
| WO2011109427A2 (en) * | 2010-03-01 | 2011-09-09 | Alnylam Pharmaceuticals, Inc. | Improving the biological activity of sirna through modulation of its thermodynamic profile |
| EP2872147B1 (en) * | 2012-07-13 | 2022-12-21 | Wave Life Sciences Ltd. | Method for making chiral oligonucleotides |
| CN106068325B (zh) * | 2014-01-16 | 2021-07-09 | 波涛生命科学有限公司 | 手性设计 |
| EP3514234A1 (en) * | 2014-03-12 | 2019-07-24 | Nippon Shinyaku Co., Ltd. | Antisense nucleic acid |
| BR112018007066A2 (pt) * | 2015-10-09 | 2018-10-23 | Sarepta Therapeutics Inc | composições e métodos para tratamento da distrofia muscular de duchene e distúrbios relacionados |
| US20180028554A1 (en) * | 2016-07-05 | 2018-02-01 | Biomarin Technologies B.V. | Oligomers Having Bicyclic Scaffold Moeities |
-
2019
- 2019-12-06 SG SG11202105626TA patent/SG11202105626TA/en unknown
- 2019-12-06 JP JP2021531966A patent/JP2022513719A/ja not_active Ceased
- 2019-12-06 WO PCT/US2019/065058 patent/WO2020118246A1/en not_active Ceased
- 2019-12-06 CA CA3122271A patent/CA3122271A1/en active Pending
- 2019-12-06 US US17/311,285 patent/US20220186217A1/en not_active Abandoned
- 2019-12-06 CN CN201980090833.2A patent/CN113383078A/zh active Pending
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- 2019-12-06 AU AU2019392928A patent/AU2019392928A1/en active Pending
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Patent Citations (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2003025139A2 (en) * | 2001-09-17 | 2003-03-27 | Mayo Foundation For Medical Education And Research | Cobalamin mediated delivery of nucleic acids, analogs and derivatives thereof |
| JP2017527277A (ja) * | 2014-08-22 | 2017-09-21 | ノーゲン エルエルシー | 修飾オリゴヌクレオチドおよびそれらの合成のための方法 |
| WO2018067973A1 (en) * | 2015-10-09 | 2018-04-12 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
Non-Patent Citations (1)
| Title |
|---|
| NAT. CHEM. BIOL., vol. Vol.8, pp.555-561, JPN6021048020, 2012, ISSN: 0005178026 * |
Also Published As
| Publication number | Publication date |
|---|---|
| CA3122271A1 (en) | 2020-06-11 |
| CN113383078A (zh) | 2021-09-10 |
| AU2019392928A1 (en) | 2021-06-17 |
| EP3891284A4 (en) | 2023-04-12 |
| SG11202105626TA (en) | 2021-06-29 |
| WO2020118246A1 (en) | 2020-06-11 |
| US20220186217A1 (en) | 2022-06-16 |
| EP3891284A1 (en) | 2021-10-13 |
| JP2025041652A (ja) | 2025-03-26 |
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