ES2707232T3 - Acido nucleico bicatenario quimérico - Google Patents
Acido nucleico bicatenario quimérico Download PDFInfo
- Publication number
- ES2707232T3 ES2707232T3 ES12815856T ES12815856T ES2707232T3 ES 2707232 T3 ES2707232 T3 ES 2707232T3 ES 12815856 T ES12815856 T ES 12815856T ES 12815856 T ES12815856 T ES 12815856T ES 2707232 T3 ES2707232 T3 ES 2707232T3
- Authority
- ES
- Spain
- Prior art keywords
- nucleic acid
- nucleotides
- strand
- dna
- double
- 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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Links
- 0 *[C@](C1OC2)O[C@]2(CO)C1N=O Chemical compound *[C@](C1OC2)O[C@]2(CO)C1N=O 0.000 description 1
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
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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
-
- 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
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
-
- 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
- C12N2310/15—Nucleic acids forming more than 2 strands, e.g. TFOs
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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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
- C12N2310/3181—Peptide nucleic acid, PNA
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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
- C12N2310/32—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
- 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
- C12N2310/341—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
- 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
- C12N2310/3513—Protein; Peptide
-
- 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
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
-
- 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
-
- 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
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/52—Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Zoology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- General Chemical & Material Sciences (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Enzymes And Modification Thereof (AREA)
- Saccharide Compounds (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2011275488 | 2011-12-16 | ||
| PCT/JP2012/083180 WO2013089283A1 (en) | 2011-12-16 | 2012-12-17 | Chimeric double-stranded nucleic acid |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ES2707232T3 true ES2707232T3 (es) | 2019-04-03 |
Family
ID=47559626
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| ES12815856T Active ES2707232T3 (es) | 2011-12-16 | 2012-12-17 | Acido nucleico bicatenario quimérico |
Country Status (9)
| Country | Link |
|---|---|
| US (6) | US9816089B2 (instruction) |
| EP (1) | EP2791335B1 (instruction) |
| JP (7) | JP6112569B2 (instruction) |
| CN (2) | CN112386605A (instruction) |
| AU (1) | AU2012353330B2 (instruction) |
| CA (2) | CA2859581C (instruction) |
| ES (1) | ES2707232T3 (instruction) |
| IN (1) | IN2014CN04590A (instruction) |
| WO (1) | WO2013089283A1 (instruction) |
Families Citing this family (52)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| KR101885383B1 (ko) | 2009-07-06 | 2018-08-03 | 웨이브 라이프 사이언시스 리미티드 | 신규한 핵산 프로드러그 및 그의 사용 방법 |
| WO2012039448A1 (ja) | 2010-09-24 | 2012-03-29 | 株式会社キラルジェン | 不斉補助基 |
| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| EP2791335B1 (en) * | 2011-12-16 | 2018-11-14 | National University Corporation Tokyo Medical and Dental University | Chimeric double-stranded nucleic acid |
| AU2013288048A1 (en) | 2012-07-13 | 2015-01-22 | Wave Life Sciences Ltd. | Asymmetric auxiliary group |
| AU2014222150A1 (en) * | 2013-03-01 | 2015-09-10 | National University Corporation Tokyo Medical And Dental University | Chimeric single-stranded antisense polynucleotides and double-stranded antisense agent |
| JP6300212B2 (ja) * | 2013-03-21 | 2018-03-28 | 国立大学法人 東京医科歯科大学 | 二重鎖核酸結合剤、当該結合剤−二重鎖核酸複合体、当該複合体を含有する医薬品組成物、及び、当該複合体の製造方法 |
| JP6604544B2 (ja) * | 2013-06-16 | 2019-11-13 | 国立大学法人 東京医科歯科大学 | エクソンスキッピング効果を有する二本鎖アンチセンス核酸 |
| WO2015105083A1 (ja) * | 2014-01-07 | 2015-07-16 | 塩野義製薬株式会社 | アンチセンスオリゴヌクレオチド及び糖誘導体を含む二本鎖オリゴヌクレオチド |
| US9714288B2 (en) | 2014-09-30 | 2017-07-25 | The Regents Of The University Of California | Antisense compounds and uses thereof |
| JP6892433B2 (ja) | 2015-04-03 | 2021-06-23 | ユニバーシティ・オブ・マサチューセッツUniversity Of Massachusetts | 十分に安定化された非対称sirna |
| US10633653B2 (en) | 2015-08-14 | 2020-04-28 | University Of Massachusetts | Bioactive conjugates for oligonucleotide delivery |
| EP3353306A4 (en) | 2015-09-25 | 2019-06-05 | Ionis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| WO2017068791A1 (ja) * | 2015-10-23 | 2017-04-27 | レナセラピューティクス株式会社 | 少なくとも1つのバルジ構造を有する核酸複合体 |
| US20180223280A1 (en) * | 2015-10-23 | 2018-08-09 | Rena Therapeutics Inc. | Nucleic acid complex |
| WO2017094745A1 (ja) * | 2015-12-01 | 2017-06-08 | 国立大学法人大阪大学 | 核酸を含む経腸投与用組成物 |
| BR112018015164A2 (pt) * | 2016-01-26 | 2018-12-26 | Nissan Chemical Corp | oligonucleotídeo de fita simples |
| MA45328A (fr) * | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Compositions acide nucléique-polypeptide et utilisations de celles-ci |
| EP3517610A4 (en) * | 2016-09-23 | 2020-05-27 | National University Corporation Tokyo Medical and Dental University | Blood-brain barrier permeable heteroduplex nucleic acid |
| WO2018062510A1 (ja) | 2016-09-29 | 2018-04-05 | 国立大学法人東京医科歯科大学 | オーバーハングを有する二本鎖核酸複合体 |
| TWI830693B (zh) | 2017-02-06 | 2024-02-01 | 日商日產化學工業股份有限公司 | 單股寡核苷酸 |
| WO2018181428A1 (ja) | 2017-03-29 | 2018-10-04 | 塩野義製薬株式会社 | 核酸医薬及び多分岐脂質の複合体 |
| JP7141621B2 (ja) * | 2017-03-31 | 2022-09-26 | 学校法人 愛知医科大学 | コンドロイチン硫酸生合成を阻害するアンチセンス核酸 |
| EP3647423A4 (en) * | 2017-06-30 | 2021-03-24 | National University Corporation Tokyo Medical and Dental University | STRAIGHT DOUBLE-STRANDED ANTI-MIRROR |
| US11555188B2 (en) | 2017-07-26 | 2023-01-17 | Nissan Chemical Corporation | Single-stranded oligonucleotide |
| JP2021506239A (ja) | 2017-12-14 | 2021-02-22 | アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. | 複合アンチセンス化合物及びその使用 |
| WO2019167995A1 (ja) | 2018-02-28 | 2019-09-06 | 国立大学法人東京医科歯科大学 | 虚血病変部位特異的な遺伝子治療法 |
| KR20190110043A (ko) * | 2018-03-19 | 2019-09-27 | 주식회사 케이티 | 비디오 신호 처리 방법 및 장치 |
| JP7333079B2 (ja) | 2018-03-19 | 2023-08-24 | 国立大学法人 東京医科歯科大学 | 毒性が軽減した核酸 |
| CN111868244A (zh) | 2018-03-20 | 2020-10-30 | 国立大学法人东京工业大学 | 降低了毒性的反义寡核苷酸 |
| WO2019182109A1 (ja) * | 2018-03-22 | 2019-09-26 | 国立大学法人東京医科歯科大学 | ヘテロ核酸のbbb通過脂質リガンド |
| 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 |
| WO2020209285A1 (ja) * | 2019-04-08 | 2020-10-15 | 国立大学法人東京医科歯科大学 | 筋疾患治療用医薬組成物 |
| EP4005602A4 (en) | 2019-07-30 | 2024-06-12 | Shionogi & Co., Ltd | NUCLEIC ACID AGENT TARGETED AGAINST MURF1 |
| WO2021070959A1 (ja) * | 2019-10-11 | 2021-04-15 | 国立大学法人東京医科歯科大学 | 修飾ヘテロ核酸 |
| WO2021146548A1 (en) * | 2020-01-17 | 2021-07-22 | Anastasia Khvorova | Universal dynamic pharmacokinetic-modifying anchors |
| EP4098747A4 (en) | 2020-01-31 | 2025-03-26 | Sanwa Kagaku Kenkyusho Co., Ltd. | ATN1 ANTISENSE OLIGONUCLEOTIDE |
| US20230159925A1 (en) | 2020-03-04 | 2023-05-25 | Nissan Chemical Corporation | Antisense oligonucleotide of calm2 |
| WO2021187392A1 (ja) * | 2020-03-16 | 2021-09-23 | 国立大学法人東京医科歯科大学 | モルホリノ核酸を含むヘテロ核酸 |
| US20230322840A1 (en) | 2020-06-15 | 2023-10-12 | Liid Pharmaceuticals, Inc. | Bridged nucleoside and nucleotide |
| CA3202708A1 (en) | 2020-11-23 | 2022-05-27 | Alpha Anomeric Sas | Nucleic acid duplexes |
| WO2022183102A1 (en) * | 2021-02-26 | 2022-09-01 | Northwestern University | Cell-free biosensors with dna strand displacement circuits |
| EP4349986A1 (en) | 2021-05-25 | 2024-04-10 | National University Corporation Tokyo Medical and Dental University | Heteronucleic acid containing scpbna or amna |
| EP4353266A1 (en) | 2021-05-31 | 2024-04-17 | Rena Therapeutics Inc. | Ligand-bound nucleic acid complex |
| EP4389891A1 (en) | 2021-08-19 | 2024-06-26 | National University Corporation Tokyo Medical and Dental University | Modified heteronucleic acid containing morpholino nucleic acid |
| WO2023026994A1 (ja) | 2021-08-21 | 2023-03-02 | 武田薬品工業株式会社 | ヒトトランスフェリンレセプター結合ペプチド-薬物コンジュゲート |
| WO2023042876A1 (ja) * | 2021-09-15 | 2023-03-23 | 国立大学法人東京医科歯科大学 | 2'-修飾ヌクレオシドを含むヘテロ核酸 |
| JPWO2023080159A1 (instruction) | 2021-11-02 | 2023-05-11 | ||
| CN118660965A (zh) * | 2021-12-07 | 2024-09-17 | Liid制药股份有限公司 | 反义寡核苷酸复合体 |
| JPWO2024071099A1 (instruction) | 2022-09-29 | 2024-04-04 | ||
| GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
| WO2024228398A1 (ja) * | 2023-05-02 | 2024-11-07 | 国立大学法人東京医科歯科大学 | グアニン四重鎖構造を誘導するための核酸剤 |
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| US8541569B2 (en) * | 2008-09-06 | 2013-09-24 | Chemgenes Corporation | Phosphoramidites for synthetic RNA in the reverse direction, efficient RNA synthesis and convenient introduction of 3'-end ligands, chromophores and modifications of synthetic RNA |
| AU2010306940A1 (en) * | 2009-10-12 | 2012-06-07 | Smith, Larry | Methods and compositions for modulating gene expression using oligonucleotide based drugs administered in vivo or in vitro |
| EP2791335B1 (en) * | 2011-12-16 | 2018-11-14 | National University Corporation Tokyo Medical and Dental University | Chimeric double-stranded nucleic acid |
| ES2700277T3 (es) * | 2013-05-30 | 2019-02-14 | Univ Nat Corp Tokyo Medical & Dental | Agentes de doble cadena para el suministro de oligonucleótidos terapéuticos |
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