JP2017536366A - Lnaキラルホスホロチオエート - Google Patents
Lnaキラルホスホロチオエート Download PDFInfo
- Publication number
- JP2017536366A JP2017536366A JP2017526905A JP2017526905A JP2017536366A JP 2017536366 A JP2017536366 A JP 2017536366A JP 2017526905 A JP2017526905 A JP 2017526905A JP 2017526905 A JP2017526905 A JP 2017526905A JP 2017536366 A JP2017536366 A JP 2017536366A
- Authority
- JP
- Japan
- Prior art keywords
- lna
- phosphorothioate
- oligonucleotide
- nucleotide
- nucleoside
- 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.)
- Pending
Links
- 0 CC12C=*(C3)C3=C=CC1CC*2 Chemical compound CC12C=*(C3)C3=C=CC1CC*2 0.000 description 13
- YPMHBZQROFTOSU-UHFFFAOYSA-N OC(C1NCCC1)c1ccccc1 Chemical compound OC(C1NCCC1)c1ccccc1 YPMHBZQROFTOSU-UHFFFAOYSA-N 0.000 description 1
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- 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
- C12N15/117—Nucleic acids having immunomodulatory properties, e.g. containing CpG-motifs
-
- 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
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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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- 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
- C12N15/113—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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- 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
- 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
- C12N2310/17—Immunomodulatory 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
- C12N2310/30—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
- C12N2310/323—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
- C12N2310/33—Chemical structure of the base
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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
- C12N2310/341—Gapmers, i.e. of the type ===---===
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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
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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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
- 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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- C—CHEMISTRY; METALLURGY
- 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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- 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
- C12N2330/00—Production
- C12N2330/30—Production chemically synthesised
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- Health & Medical Sciences (AREA)
- 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)
- Saccharide Compounds (AREA)
- Enzymes And Modification Thereof (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (13)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP14193887 | 2014-11-19 | ||
| EP14193887.8 | 2014-11-19 | ||
| EP14198167 | 2014-12-16 | ||
| EP14198167.0 | 2014-12-16 | ||
| EP15182401 | 2015-08-25 | ||
| EP15182401.8 | 2015-08-25 | ||
| EP15191076.7 | 2015-10-22 | ||
| EP15191074 | 2015-10-22 | ||
| EP15191075 | 2015-10-22 | ||
| EP15191076 | 2015-10-22 | ||
| EP15191074.2 | 2015-10-22 | ||
| EP15191075.9 | 2015-10-22 | ||
| PCT/EP2015/076971 WO2016079183A1 (en) | 2014-11-19 | 2015-11-18 | Lna chiral phosphorothioates |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2017536366A true JP2017536366A (ja) | 2017-12-07 |
| JP2017536366A5 JP2017536366A5 (enExample) | 2018-11-15 |
Family
ID=54608516
Family Applications (2)
| 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 |
| 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 |
Family Applications After (1)
| 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 |
Country Status (4)
| Country | Link |
|---|---|
| US (2) | US20180016575A1 (enExample) |
| EP (2) | EP3221329A1 (enExample) |
| JP (2) | JP2017536366A (enExample) |
| WO (2) | WO2016079183A1 (enExample) |
Cited By (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2021508689A (ja) * | 2017-12-22 | 2021-03-11 | 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 | ホスホロジチオアートヌクレオシド間結合を含むギャップマーオリゴヌクレオチド |
Families Citing this family (44)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG171914A1 (en) | 2008-12-02 | 2011-07-28 | Chiralgen Ltd | Method for the synthesis of phosphorus atom modified nucleic acids |
| US9744183B2 (en) | 2009-07-06 | 2017-08-29 | Wave Life Sciences Ltd. | Nucleic acid prodrugs and methods of use thereof |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| RU2014105311A (ru) | 2011-07-19 | 2015-08-27 | Уэйв Лайф Сайенсес Пте. Лтд. | Способы синтеза функционализованных нуклеиновых кислот |
| PT2872485T (pt) | 2012-07-13 | 2021-03-05 | Wave Life Sciences Ltd | Grupo auxiliar assimétrico |
| KR102712879B1 (ko) | 2012-07-13 | 2024-10-04 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| BR112015000723A2 (pt) | 2012-07-13 | 2017-06-27 | Shin Nippon Biomedical Laboratories Ltd | adjuvante de ácido nucléico quiral |
| US11162096B2 (en) | 2013-10-14 | 2021-11-02 | Ionis Pharmaceuticals, Inc | Methods for modulating expression of C9ORF72 antisense transcript |
| 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 |
| JPWO2015108046A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤 |
| PT3094728T (pt) | 2014-01-16 | 2022-05-19 | Wave Life Sciences Ltd | Desenho quiral |
| EP3712269A1 (en) | 2014-12-17 | 2020-09-23 | ProQR Therapeutics II B.V. | Targeted rna editing |
| WO2016112132A1 (en) * | 2015-01-06 | 2016-07-14 | 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 |
| MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| EP3394258B1 (en) * | 2015-10-22 | 2021-09-22 | 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 |
| MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
| WO2017216325A1 (en) * | 2016-06-17 | 2017-12-21 | F. Hoffmann-La Roche Ag | In vitro nephrotoxicity screening assay |
| JP7049271B2 (ja) * | 2016-06-17 | 2022-04-06 | 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 |
| MA45496A (fr) | 2016-06-17 | 2019-04-24 | Hoffmann La Roche | Molécules d'acide nucléique pour la réduction de l'arnm de padd5 ou pad7 pour le traitement d'une infection par l'hépatite b |
| 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 |
| KR102501980B1 (ko) | 2016-09-01 | 2023-02-20 | 프로큐알 테라퓨틱스 Ⅱ 비.브이. | 화학적으로 변형된 단일 가닥 rna-편집 올리고뉴클레오타이드 |
| JP7115669B2 (ja) * | 2016-11-14 | 2022-08-09 | 学校法人東京理科大学 | 重合性化合物、化合物、及び、ボラノホスフェートオリゴマーの製造方法 |
| US11873316B2 (en) | 2016-11-23 | 2024-01-16 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
| WO2018130583A1 (en) * | 2017-01-13 | 2018-07-19 | Roche Innovation Center Copenhagen A/S | Antisense oligonucleotides for modulating nfkb1 expression |
| EP3568481A1 (en) * | 2017-01-13 | 2019-11-20 | 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 |
| CN118291458A (zh) | 2017-12-01 | 2024-07-05 | 德克萨斯A&M大学体系 | 天使综合征反义治疗 |
| BR112020012427A8 (pt) * | 2017-12-22 | 2024-04-02 | Roche Innovation Ct Copenhagen As | Oligonucleotídeos, sal farmaceuticamente aceitável, conjugado, composição farmacêutica e processo para a fabricação de um oligonucleotídeo |
| JP2021508327A (ja) * | 2017-12-22 | 2021-03-04 | 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 | 新規のチオホスホラミダイト |
| CA3088112A1 (en) | 2018-01-12 | 2019-07-18 | Bristol-Myers Squibb Company | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
| BR112020014011A2 (pt) * | 2018-01-12 | 2020-12-08 | Bristol-Myers Squibb Company | Oligonucleotídeos antissenso dirigidos à alfa-sinucleína e usos dos mesmos |
| CN112236438B (zh) | 2018-04-13 | 2024-10-15 | 百时美施贵宝公司 | 基于磷(v)的试剂,其制备方法,以及其在制备立体限定的有机磷(v)化合物中的用途 |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| RU2768285C1 (ru) | 2018-07-03 | 2022-03-23 | Ф. Хоффманн-Ля Рош Аг | Олигонуклеотиды для модуляции экспрессии тау-белка |
| WO2020009151A1 (ja) * | 2018-07-04 | 2020-01-09 | 国立大学法人名古屋大学 | タウのスプライシングを制御するアンチセンスオリゴヌクレオチド及びその用途 |
| 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 |
| MX2021014206A (es) | 2019-05-31 | 2022-01-06 | Aligos Therapeutics Inc | Oligonucleotidos gapmer modificados y metodos de uso. |
| AU2021209076A1 (en) | 2020-01-16 | 2022-07-07 | Bristol-Myers Squibb Company | Reagents and their use for modular enantiodivergent synthesis of C–P bonds |
| 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 |
|---|---|---|---|---|
| JP2002520420A (ja) * | 1998-07-14 | 2002-07-09 | アイシス・ファーマシューティカルス・インコーポレーテッド | 部位特異的キラルホスホロチオエート・ヌクレオシド間結合を有するオリゴヌクレオチド |
| WO2014118267A1 (en) * | 2013-01-30 | 2014-08-07 | Santaris Pharma A/S | Lna oligonucleotide carbohydrate conjugates |
Family Cites Families (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2007310989B2 (en) * | 2006-10-18 | 2014-05-29 | Isis Pharmaceuticals, Inc. | Antisense compounds |
| KR102712879B1 (ko) * | 2012-07-13 | 2024-10-04 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
-
2015
- 2015-11-18 JP JP2017526905A patent/JP2017536366A/ja active Pending
- 2015-11-18 EP EP15797651.5A patent/EP3221329A1/en not_active Withdrawn
- 2015-11-18 JP JP2017526918A patent/JP2017536119A/ja active Pending
- 2015-11-18 WO PCT/EP2015/076971 patent/WO2016079183A1/en not_active Ceased
- 2015-11-18 EP EP15797652.3A patent/EP3220921A1/en not_active Withdrawn
- 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
Patent Citations (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2002520420A (ja) * | 1998-07-14 | 2002-07-09 | アイシス・ファーマシューティカルス・インコーポレーテッド | 部位特異的キラルホスホロチオエート・ヌクレオシド間結合を有するオリゴヌクレオチド |
| WO2014118267A1 (en) * | 2013-01-30 | 2014-08-07 | Santaris Pharma A/S | Lna oligonucleotide carbohydrate conjugates |
Non-Patent Citations (4)
| Title |
|---|
| BIOORGANIC AND MEDICINAL CHEMISTRY LETTERS, vol. 11, JPN6019029882, 2001, pages 1001 - 1003, ISSN: 0004224932 * |
| J. MED. DENT. SCI., vol. 60, JPN6019029880, 2013, pages 9 - 16, ISSN: 0004224929 * |
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| JP2021508689A (ja) * | 2017-12-22 | 2021-03-11 | 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 | ホスホロジチオアートヌクレオシド間結合を含むギャップマーオリゴヌクレオチド |
| JP2024041845A (ja) * | 2017-12-22 | 2024-03-27 | 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 | ホスホロジチオアートヌクレオシド間結合を含むギャップマーオリゴヌクレオチド |
| JP7476101B2 (ja) | 2017-12-22 | 2024-04-30 | 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 | ホスホロジチオアートヌクレオシド間結合を含むギャップマーオリゴヌクレオチド |
Also Published As
| Publication number | Publication date |
|---|---|
| US20180112217A1 (en) | 2018-04-26 |
| EP3221329A1 (en) | 2017-09-27 |
| WO2016079181A1 (en) | 2016-05-26 |
| US20180016575A1 (en) | 2018-01-18 |
| WO2016079183A1 (en) | 2016-05-26 |
| JP2017536119A (ja) | 2017-12-07 |
| EP3220921A1 (en) | 2017-09-27 |
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