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
- Publication number
- 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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- 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
-
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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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
- C12N2310/10—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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- C—CHEMISTRY; METALLURGY
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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
-
- 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
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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 | 2014-11-19 | ||
| EP14193887.8 | 2014-11-19 | ||
| EP14198167.0 | 2014-12-16 | ||
| EP14198167 | 2014-12-16 | ||
| EP15182401.8 | 2015-08-25 | ||
| EP15182401 | 2015-08-25 | ||
| EP15191075 | 2015-10-22 | ||
| EP15191076.7 | 2015-10-22 | ||
| EP15191075.9 | 2015-10-22 | ||
| EP15191074.2 | 2015-10-22 | ||
| EP15191076 | 2015-10-22 | ||
| EP15191074 | 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 (https=) | 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) | US20180016575A1 (https=) |
| EP (2) | EP3221329A1 (https=) |
| JP (2) | JP2017536119A (https=) |
| WO (2) | WO2016079183A1 (https=) |
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 |
|---|---|---|---|---|
| CN102282155B (zh) | 2008-12-02 | 2017-06-09 | 日本波涛生命科学公司 | 磷原子修饰的核酸的合成方法 |
| RU2612521C2 (ru) | 2009-07-06 | 2017-03-09 | Онтории, Инк. | Новые пролекарства нуклеиновых кислот и способы их применения |
| JP5868324B2 (ja) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
| CN103796657B (zh) | 2011-07-19 | 2017-07-11 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
| CA2879066C (en) | 2012-07-13 | 2019-08-13 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant |
| PL2872485T3 (pl) | 2012-07-13 | 2021-05-31 | Wave Life Sciences Ltd. | Asymetryczna grupa pomocnicza |
| KR102213609B1 (ko) | 2012-07-13 | 2021-02-08 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| US11162096B2 (en) | 2013-10-14 | 2021-11-02 | Ionis Pharmaceuticals, Inc | Methods for modulating expression of C9ORF72 antisense transcript |
| US10322173B2 (en) | 2014-01-15 | 2019-06-18 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent |
| JPWO2015108047A1 (ja) | 2014-01-15 | 2017-03-23 | 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 |
| JPWO2015108048A1 (ja) | 2014-01-15 | 2017-03-23 | 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 |
| BR112016016400A2 (pt) | 2014-01-16 | 2017-10-03 | Wave Life Sciences Ltd | Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos |
| EP3234134B1 (en) | 2014-12-17 | 2020-05-27 | ProQR Therapeutics II B.V. | Targeted rna editing |
| US10793855B2 (en) * | 2015-01-06 | 2020-10-06 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of C9ORF72 antisense transcript |
| US10407678B2 (en) | 2015-04-16 | 2019-09-10 | Ionis Pharmaceuticals, Inc. | Compositions for modulating expression of C9ORF72 antisense transcript |
| IL320434A (en) | 2015-07-22 | 2025-06-01 | Wave Life Sciences Ltd | Oligonucleotide preparations and methods |
| US10955407B2 (en) | 2015-10-22 | 2021-03-23 | Roche Innovation Center Copenhagen A/S | In vitro toxicity screening assay |
| WO2017157672A1 (en) * | 2016-03-18 | 2017-09-21 | Roche Innovation Center Copenhagen A/S | Acyl-protected l-lna-guanosine monomers |
| EP3452596A4 (en) | 2016-05-04 | 2020-03-18 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND RELATED METHODS |
| JP7012033B2 (ja) * | 2016-06-17 | 2022-02-10 | エフ.ホフマン-ラ ロシュ アーゲー | インビトロ腎毒性スクリーニングアッセイ |
| CN109328236B (zh) * | 2016-06-17 | 2022-10-25 | 豪夫迈·罗氏有限公司 | 体外肾毒性筛选测定法 |
| EP4219767A1 (en) | 2016-06-17 | 2023-08-02 | F. Hoffmann-La Roche AG | Papd5 and papd7 inhibitors for treating a hepatitis b infection |
| WO2017220751A1 (en) | 2016-06-22 | 2017-12-28 | Proqr Therapeutics Ii B.V. | Single-stranded rna-editing oligonucleotides |
| CN110352244B (zh) | 2016-09-01 | 2023-03-21 | ProQR治疗上市公司Ⅱ | 化学修饰的编辑rna的单链寡核苷酸 |
| CN109937206B (zh) * | 2016-11-14 | 2022-04-01 | 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 |
| US11873316B2 (en) | 2016-11-23 | 2024-01-16 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
| EP3568479A1 (en) * | 2017-01-13 | 2019-11-20 | Roche Innovation Center Copenhagen A/S | Antisense oligonucleotides for modulating nfkb1 expression |
| US20190345496A1 (en) * | 2017-01-13 | 2019-11-14 | 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 |
| CN111511914B (zh) * | 2017-10-16 | 2023-11-17 | 豪夫迈·罗氏有限公司 | 减少PAPD5和PAPD7 mRNA的核酸分子用于治疗乙型肝炎感染 |
| KR102723989B1 (ko) | 2017-12-01 | 2024-11-01 | 더 텍사스 에이 & 엠 유니버시티 시스템 | 엔젤만 증후군 안티센스 치료 |
| CA3084170A1 (en) * | 2017-12-22 | 2019-06-27 | Roche Innovation Center Copenhagen A/S | Gapmer oligonucleotides comprising a phosphorodithioate internucleoside linkage |
| US11597926B2 (en) | 2017-12-22 | 2023-03-07 | Roche Innovation Center Copenhagen A/S | Thiophosphoramidites |
| WO2019122279A1 (en) * | 2017-12-22 | 2019-06-27 | Roche Innovation Center Copenhagen A/S | Oligonucleotides comprising a phosphorodithioate internucleoside linkage |
| SG11202006528XA (en) * | 2018-01-12 | 2020-08-28 | Bristol Myers Squibb Co | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
| CN120310791A (zh) | 2018-01-12 | 2025-07-15 | 百时美施贵宝公司 | 靶向α-突触核蛋白的反义寡核苷酸及其用途 |
| SG11202008827XA (en) | 2018-04-13 | 2020-10-29 | Bristol Myers Squibb Co | 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 |
| PE20210346A1 (es) | 2018-07-03 | 2021-02-25 | Hoffmann La Roche | Oligonucleotidos para modular la expresion de tau |
| EP3819378A4 (en) * | 2018-07-04 | 2022-09-14 | Aichi Medical University | OLIGONUCLEOTIDES FOR CONTROL OF TAU SPLICING AND THEIR USES |
| SG11202112741XA (en) | 2019-05-31 | 2021-12-30 | Aligos Therapeutics Inc | Modified gapmer oligonucleotides and methods of use |
| MX2022008123A (es) | 2020-01-16 | 2022-07-21 | Bristol Myers Squibb Co | Reactivos y su uso para modular la sintesis enantiodivergente de enlaces c-p. |
| WO2022200633A1 (en) * | 2021-03-26 | 2022-09-29 | Neumirna Therapeutics Aps | Microrna-27b inhibitors |
| EP4590683A1 (en) | 2022-09-19 | 2025-07-30 | Bristol-Myers Squibb Company | Novel phosphorous (v)-based reagents, processes for the preparation thereof, and their use in making stereo-defined organophoshorous (v) compounds |
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 |
| AU2014211406B2 (en) * | 2013-01-30 | 2019-07-18 | Roche Innovation Center Copenhagen A/S | LNA oligonucleotide carbohydrate conjugates |
-
2015
- 2015-11-18 WO PCT/EP2015/076971 patent/WO2016079183A1/en not_active Ceased
- 2015-11-18 US US15/527,765 patent/US20180016575A1/en not_active Abandoned
- 2015-11-18 US US15/527,832 patent/US20180112217A1/en not_active Abandoned
- 2015-11-18 WO PCT/EP2015/076967 patent/WO2016079181A1/en not_active Ceased
- 2015-11-18 JP JP2017526918A patent/JP2017536119A/ja active Pending
- 2015-11-18 EP EP15797651.5A patent/EP3221329A1/en not_active Withdrawn
- 2015-11-18 EP EP15797652.3A patent/EP3220921A1/en not_active Withdrawn
- 2015-11-18 JP JP2017526905A patent/JP2017536366A/ja active Pending
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 |
|---|---|
| WO2016079181A1 (en) | 2016-05-26 |
| EP3220921A1 (en) | 2017-09-27 |
| EP3221329A1 (en) | 2017-09-27 |
| US20180016575A1 (en) | 2018-01-18 |
| WO2016079183A1 (en) | 2016-05-26 |
| JP2017536366A (ja) | 2017-12-07 |
| US20180112217A1 (en) | 2018-04-26 |
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