JP2017536119A - キラルホスホロチオエート連結を含むlnaギャップマーオリゴヌクレオチド - Google Patents
キラルホスホロチオエート連結を含むlnaギャップマーオリゴヌクレオチド Download PDFInfo
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- JP2017536119A JP2017536119A JP2017526918A JP2017526918A JP2017536119A JP 2017536119 A JP2017536119 A JP 2017536119A JP 2017526918 A JP2017526918 A JP 2017526918A JP 2017526918 A JP2017526918 A JP 2017526918A JP 2017536119 A JP2017536119 A JP 2017536119A
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- lna
- phosphorothioate
- nucleoside
- oligonucleotide
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- 0 CC(C(N1)=O)=CN([C@@](C2OC3)O[C@]3(C*)C2OP(N2[C@@]3CCC2)O[C@]3c2ccccc2)C1=O Chemical compound CC(C(N1)=O)=CN([C@@](C2OC3)O[C@]3(C*)C2OP(N2[C@@]3CCC2)O[C@]3c2ccccc2)C1=O 0.000 description 2
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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
- C12N15/117—Nucleic acids having immunomodulatory properties, e.g. containing CpG-motifs
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- 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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/11—Antisense
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/17—Immunomodulatory nucleic acids
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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
- C12N2310/323—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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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
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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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- 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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects
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- C12N2330/00—Production
- C12N2330/30—Production chemically synthesised
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- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Biochemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Immunology (AREA)
- Pharmacology & Pharmacy (AREA)
- General Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (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)
- Enzymes And Modification Thereof (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Saccharide Compounds (AREA)
Applications Claiming Priority (13)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP14193887.8 | 2014-11-19 | ||
| EP14193887 | 2014-11-19 | ||
| EP14198167.0 | 2014-12-16 | ||
| EP14198167 | 2014-12-16 | ||
| EP15182401.8 | 2015-08-25 | ||
| EP15182401 | 2015-08-25 | ||
| EP15191076.7 | 2015-10-22 | ||
| EP15191074 | 2015-10-22 | ||
| EP15191075.9 | 2015-10-22 | ||
| EP15191076 | 2015-10-22 | ||
| EP15191075 | 2015-10-22 | ||
| EP15191074.2 | 2015-10-22 | ||
| PCT/EP2015/076967 WO2016079181A1 (en) | 2014-11-19 | 2015-11-18 | Lna gapmer oligonucleotides comprising chiral phosphorothioate linkages |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2017536119A true JP2017536119A (ja) | 2017-12-07 |
| JP2017536119A5 JP2017536119A5 (enExample) | 2018-11-15 |
Family
ID=54608516
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017526918A Pending JP2017536119A (ja) | 2014-11-19 | 2015-11-18 | キラルホスホロチオエート連結を含むlnaギャップマーオリゴヌクレオチド |
| JP2017526905A Pending JP2017536366A (ja) | 2014-11-19 | 2015-11-18 | Lnaキラルホスホロチオエート |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017526905A Pending JP2017536366A (ja) | 2014-11-19 | 2015-11-18 | Lnaキラルホスホロチオエート |
Country Status (4)
| Country | Link |
|---|---|
| US (2) | US20180112217A1 (enExample) |
| EP (2) | EP3221329A1 (enExample) |
| JP (2) | JP2017536119A (enExample) |
| WO (2) | WO2016079183A1 (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2022521510A (ja) * | 2019-02-20 | 2022-04-08 | ロシュ イノベーション センター コペンハーゲン エーエス | ホスホノアセテートギャップマー型オリゴヌクレオチド |
Families Citing this family (45)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9394333B2 (en) | 2008-12-02 | 2016-07-19 | Wave Life Sciences Japan | Method for the synthesis of phosphorus atom modified nucleic acids |
| MX342945B (es) | 2009-07-06 | 2016-10-18 | Ontorii Inc * | Profármacos de ácido nucleico novedosos y métodos de uso de los mismos. |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| KR20240148947A (ko) | 2012-07-13 | 2024-10-11 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| SG11201500243WA (en) | 2012-07-13 | 2015-04-29 | Shin Nippon Biomedical Lab Ltd | Chiral nucleic acid adjuvant |
| SG11201500239VA (en) | 2012-07-13 | 2015-03-30 | Wave Life Sciences Japan | Asymmetric auxiliary group |
| US11162096B2 (en) | 2013-10-14 | 2021-11-02 | Ionis Pharmaceuticals, Inc | Methods for modulating expression of C9ORF72 antisense transcript |
| EP3095460A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent |
| WO2015108047A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
| WO2015108048A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
| ES2917473T3 (es) | 2014-01-16 | 2022-07-08 | Wave Life Sciences Ltd | Diseño quiral |
| CA2968336C (en) | 2014-12-17 | 2021-11-23 | Proqr Therapeutics Ii B.V. | Construct for site directed editing of an adenosine nucleotide in target rna |
| US10793855B2 (en) * | 2015-01-06 | 2020-10-06 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of C9ORF72 antisense transcript |
| WO2016167780A1 (en) | 2015-04-16 | 2016-10-20 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of c9orf72 antisense transcript |
| CN108025089A (zh) | 2015-07-22 | 2018-05-11 | 波涛生命科学有限公司 | 寡核苷酸组合物及其方法 |
| WO2017067970A1 (en) | 2015-10-22 | 2017-04-27 | Roche Innovation Center Copenhagen A/S | In vitro toxicity screening assay |
| US11267843B2 (en) * | 2016-03-18 | 2022-03-08 | Roche Innovation Center Copenhagen A/S | Stereodefining L-monomers |
| CN109477108A (zh) | 2016-05-04 | 2019-03-15 | 波涛生命科学有限公司 | 寡核苷酸组合物和其方法 |
| US11479818B2 (en) * | 2016-06-17 | 2022-10-25 | Hoffmann-La Roche Inc. | In vitro nephrotoxicity screening assay |
| EP3472346B1 (en) | 2016-06-17 | 2023-01-18 | F. Hoffmann-La Roche AG | Papd5 and papd7 inhibitors for treating a hepatitis b infection |
| WO2017216340A1 (en) * | 2016-06-17 | 2017-12-21 | F. Hoffmann-La Roche Ag | In vitro nephrotoxicity screening assay |
| 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 |
| EP3507366B1 (en) | 2016-09-01 | 2020-10-07 | ProQR Therapeutics II B.V. | Chemically modified single-stranded rna-editing oligonucleotides |
| CN114621302A (zh) * | 2016-11-14 | 2022-06-14 | 学校法人东京理科大学 | 聚合性化合物、化合物、及硼烷磷酸酯低聚物的制造方法 |
| JP7296882B2 (ja) | 2016-11-23 | 2023-06-23 | ウェイブ ライフ サイエンシズ リミテッド | ホスホラミダイト及びオリゴヌクレオチド合成のための組成物及び方法 |
| WO2018130583A1 (en) * | 2017-01-13 | 2018-07-19 | Roche Innovation Center Copenhagen A/S | Antisense oligonucleotides for modulating nfkb1 expression |
| WO2018130585A1 (en) * | 2017-01-13 | 2018-07-19 | Roche Innovation Center Copenhagen A/S | Antisense oligonucleotides for modulating relb expression |
| EP3571300A1 (en) | 2017-01-19 | 2019-11-27 | ProQR Therapeutics II B.V. | Oligonucleotide complexes for use in rna editing |
| EP3694995A1 (en) | 2017-10-13 | 2020-08-19 | Roche Innovation Center Copenhagen A/S | Methods for identifying improved stereodefined phosphorothioate oligonucleotide variants of antisense oligonucleotides utilising sub-libraries of partially stereodefined oligonucleotides |
| TW202424191A (zh) * | 2017-10-16 | 2024-06-16 | 瑞士商赫孚孟拉羅股份公司 | 用於降低PAPD5及PAPD7 mRNA以治療B型肝炎感染之核酸分子 |
| HRP20250804T1 (hr) | 2017-12-01 | 2025-09-12 | The Texas A&M University System | Antisense liječenje angelmanovog sindroma |
| US20200339982A1 (en) * | 2017-12-22 | 2020-10-29 | Roche Innovation Center Copenhagen A/S | Oligonucleotides comprising a phosphorodithioate internucleoside linkage |
| KR20200104302A (ko) * | 2017-12-22 | 2020-09-03 | 로슈 이노베이션 센터 코펜하겐 에이/에스 | 신규 티오포스포르아미다이트 |
| KR20200104347A (ko) * | 2017-12-22 | 2020-09-03 | 로슈 이노베이션 센터 코펜하겐 에이/에스 | 포스포로디티오에이트 인터뉴클레오시드 연결을 포함하는 갭머 올리고뉴클레오티드 |
| MX2020007433A (es) | 2018-01-12 | 2020-09-14 | Bristol Myers Squibb Co | Oligonucleotidos antisentido que actuan sobre alfa-sinucleina, y usos de estos. |
| EA202091693A1 (ru) | 2018-01-12 | 2021-04-14 | Бристол-Маерс Сквибб Компани | Антисмысловые олигонуклеотиды, целенаправленно воздействующие на альфа-синуклеин, и их применения |
| EP3774824A1 (en) | 2018-04-13 | 2021-02-17 | Bristol-Myers Squibb Company | Novel phosphorous (v)-based reagents, processes for the preparation thereof, and their use in making stereo-defined organophoshorous (v) compounds |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| PE20220013A1 (es) | 2018-07-03 | 2022-01-11 | Hoffmann La Roche | Oligonucleotidos para modular la expresion de tau |
| CN112469827B (zh) * | 2018-07-04 | 2024-05-24 | 学校法人爱知医科大学 | 控制Tau的剪接的反义寡核苷酸及其用途 |
| US11466274B2 (en) | 2019-05-31 | 2022-10-11 | Aligos Therapeutics, Inc. | Modified gapmer oligonucleotides and methods of use |
| WO2021146391A1 (en) | 2020-01-16 | 2021-07-22 | Bristol-Myers Squibb Company | Reagents and their use for modular enantiodivergent synthesis of c–p bonds |
| AU2022242781A1 (en) * | 2021-03-26 | 2023-10-26 | Neumirna Therapeutics Aps | Microrna-27b inhibitors |
| CN120187734A (zh) | 2022-09-19 | 2025-06-20 | 百时美施贵宝公司 | 新的基于磷(v)的试剂、其制备方法以及其在制备立体定义的有机磷(v)化合物中的用途 |
Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2010507579A (ja) * | 2006-10-18 | 2010-03-11 | アイシス ファーマシューティカルズ, インコーポレーテッド | アンチセンス化合物 |
| WO2014012081A2 (en) * | 2012-07-13 | 2014-01-16 | Ontorii, Inc. | Chiral control |
Family Cites Families (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US6242589B1 (en) * | 1998-07-14 | 2001-06-05 | Isis Pharmaceuticals, Inc. | Phosphorothioate oligonucleotides having modified internucleoside linkages |
| DK2951305T3 (en) * | 2013-01-30 | 2018-10-29 | Hoffmann La Roche | LNA oligonucleotide KULHYDRATKONJUGATER |
-
2015
- 2015-11-18 JP JP2017526918A patent/JP2017536119A/ja active Pending
- 2015-11-18 US US15/527,832 patent/US20180112217A1/en not_active Abandoned
- 2015-11-18 EP EP15797651.5A patent/EP3221329A1/en not_active Withdrawn
- 2015-11-18 US US15/527,765 patent/US20180016575A1/en not_active Abandoned
- 2015-11-18 JP JP2017526905A patent/JP2017536366A/ja active Pending
- 2015-11-18 EP EP15797652.3A patent/EP3220921A1/en not_active Withdrawn
- 2015-11-18 WO PCT/EP2015/076971 patent/WO2016079183A1/en not_active Ceased
- 2015-11-18 WO PCT/EP2015/076967 patent/WO2016079181A1/en not_active Ceased
Patent Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2010507579A (ja) * | 2006-10-18 | 2010-03-11 | アイシス ファーマシューティカルズ, インコーポレーテッド | アンチセンス化合物 |
| WO2014012081A2 (en) * | 2012-07-13 | 2014-01-16 | Ontorii, Inc. | Chiral control |
Non-Patent Citations (1)
| Title |
|---|
| NUCLEIC ACIDS RES., vol. 42, no. 22, JPN6019032985, 14 November 2014 (2014-11-14), pages 13456 - 13468, ISSN: 0004237397 * |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2022521510A (ja) * | 2019-02-20 | 2022-04-08 | ロシュ イノベーション センター コペンハーゲン エーエス | ホスホノアセテートギャップマー型オリゴヌクレオチド |
Also Published As
| Publication number | Publication date |
|---|---|
| US20180016575A1 (en) | 2018-01-18 |
| WO2016079181A1 (en) | 2016-05-26 |
| US20180112217A1 (en) | 2018-04-26 |
| EP3220921A1 (en) | 2017-09-27 |
| JP2017536366A (ja) | 2017-12-07 |
| EP3221329A1 (en) | 2017-09-27 |
| WO2016079183A1 (en) | 2016-05-26 |
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