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
-
- 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
-
- 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)
- Chemical & Material Sciences (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Public Health (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Pharmacology & Pharmacy (AREA)
- General Chemical & Material Sciences (AREA)
- Veterinary Medicine (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Saccharide Compounds (AREA)
- Peptides Or Proteins (AREA)
- Enzymes And Modification Thereof (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (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 (enExample) |
| EP (1) | EP2791335B1 (enExample) |
| JP (7) | JP6112569B2 (enExample) |
| CN (2) | CN104126010B (enExample) |
| AU (1) | AU2012353330B2 (enExample) |
| CA (2) | CA3106312C (enExample) |
| ES (1) | ES2707232T3 (enExample) |
| IN (1) | IN2014CN04590A (enExample) |
| WO (1) | WO2013089283A1 (enExample) |
Families Citing this family (54)
| 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 |
| US10428019B2 (en) | 2010-09-24 | 2019-10-01 | Wave Life Sciences Ltd. | Chiral auxiliaries |
| EP2734208B1 (en) | 2011-07-19 | 2017-03-01 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| CN104126010B (zh) * | 2011-12-16 | 2021-04-06 | 国立大学法人东京医科齿科大学 | 嵌合的双链核酸 |
| PL2872485T3 (pl) | 2012-07-13 | 2021-05-31 | Wave Life Sciences Ltd. | Asymetryczna grupa pomocnicza |
| EP2961841B1 (en) * | 2013-03-01 | 2020-01-22 | 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 |
| CN105324119A (zh) * | 2013-06-16 | 2016-02-10 | 国立大学法人东京医科齿科大学 | 具有外显子跳跃效应的双链反义核酸 |
| JP6482475B2 (ja) * | 2014-01-07 | 2019-03-13 | レナセラピューティクス株式会社 | 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 |
| EP3277811B1 (en) | 2015-04-03 | 2020-12-23 | University of Massachusetts | Fully stabilized asymmetric 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 |
| US20180228830A1 (en) * | 2015-10-23 | 2018-08-16 | Rena Therapeutics Inc. | Nucleic acid complex having at least one bulge structure |
| US20180223280A1 (en) * | 2015-10-23 | 2018-08-09 | Rena Therapeutics Inc. | Nucleic acid complex |
| JP6950958B2 (ja) * | 2015-12-01 | 2021-10-13 | 国立大学法人 東京医科歯科大学 | 核酸を含む経腸投与用組成物 |
| WO2017131124A1 (ja) * | 2016-01-26 | 2017-08-03 | 日産化学工業株式会社 | 一本鎖オリゴヌクレオチド |
| MA45328A (fr) | 2016-04-01 | 2019-02-06 | Avidity Biosciences Llc | Compositions acide nucléique-polypeptide et utilisations de celles-ci |
| US11433089B2 (en) | 2016-09-23 | 2022-09-06 | National University Corporation Tokyo Medical And Dental University | Blood-brain barrier permeable heteroduplex nucleic acid |
| US11260134B2 (en) | 2016-09-29 | 2022-03-01 | National University Corporation Tokyo Medical And Dental University | Double-stranded nucleic acid complex having overhang |
| WO2018143475A1 (ja) * | 2017-02-06 | 2018-08-09 | 日産化学工業株式会社 | 一本鎖オリゴヌクレオチド |
| EP3603648B1 (en) | 2017-03-29 | 2025-09-17 | Shionogi & Co., Ltd | Complex of nucleic acid medicine and multibranched lipid |
| US11479773B2 (en) * | 2017-03-31 | 2022-10-25 | Aichi Medical University | Antisense nucleic acid for inhibiting biosynthesis of chondroitin sulfate |
| WO2019004420A1 (ja) | 2017-06-30 | 2019-01-03 | 国立大学法人東京医科歯科大学 | ヘテロ二本鎖型ａｎｔｉｍｉＲ |
| EP3660153A4 (en) * | 2017-07-26 | 2021-04-21 | Nissan Chemical Corporation | SINGLE-STRANDED OLIGONUCLEOTIDE |
| EP4257691A3 (en) | 2017-12-14 | 2024-01-10 | Ionis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| US11674141B2 (en) | 2018-02-28 | 2023-06-13 | National University Corporation Tokyo Medical And Dental University | Ischemic-lesion-site-specific gene therapy |
| 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 |
| WO2019182294A1 (ko) * | 2018-03-19 | 2019-09-26 | 주식회사 케이티 | 비디오 신호 처리 방법 및 장치 |
| MX2020009765A (es) | 2018-03-20 | 2021-01-08 | Tokyo Inst Tech | Oligonucleotido antisentido reducido en toxicidad. |
| 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 |
| AU2020207935A1 (en) * | 2019-01-18 | 2021-08-26 | University Of Massachusetts | Dynamic pharmacokinetic-modifying anchors |
| CN113677374A (zh) | 2019-04-08 | 2021-11-19 | 国立大学法人东京医科齿科大学 | 肌疾病治疗用药物组合物 |
| EP4005602A4 (en) | 2019-07-30 | 2024-06-12 | Shionogi & Co., Ltd | NUCLEIC ACID AGENT TARGETED AGAINST MURF1 |
| EP4043565A4 (en) * | 2019-10-11 | 2024-01-24 | National University Corporation Tokyo Medical and Dental University | MODIFIED HETERONUCLEIC ACID |
| EP4090744A4 (en) * | 2020-01-17 | 2024-04-10 | Anastasia Khvorova | UNIVERSAL DYNAMIC PHARMACOKINETIC MODIFYING ANCHORS |
| KR20220133925A (ko) | 2020-01-31 | 2022-10-05 | 가부시키가이샤산와카가쿠켄큐쇼 | Atn1의 안티센스 올리고뉴클레오티드 |
| WO2021177418A1 (ja) | 2020-03-04 | 2021-09-10 | 日産化学株式会社 | Ｃａｌｍ２のアンチセンスオリゴヌクレオチド |
| JPWO2021187392A1 (enExample) * | 2020-03-16 | 2021-09-23 | ||
| WO2021242883A1 (en) | 2020-05-26 | 2021-12-02 | University Of Massachusetts | Synthetic oligonucleotides having regions of block and cluster modifications |
| CN115968374A (zh) | 2020-06-15 | 2023-04-14 | Liid制药股份有限公司 | 交联型核苷及核苷酸 |
| CA3202708A1 (en) | 2020-11-23 | 2022-05-27 | Alpha Anomeric Sas | Nucleic acid duplexes |
| US20240141414A1 (en) * | 2021-02-26 | 2024-05-02 | Northwestern University | Cell-free biosensors with dna strand displacement circuits |
| CA3221584A1 (en) | 2021-05-25 | 2022-12-01 | National University Corporation Tokyo Medical And Dental University | Heteronucleic acid containing scpbna or amna |
| BR112023024384A2 (pt) | 2021-05-31 | 2024-02-15 | Rena Therapeutics Inc | Complexo de ácido nucleico ligado ao ligante |
| WO2023022229A1 (ja) | 2021-08-19 | 2023-02-23 | 国立大学法人東京医科歯科大学 | モルホリノ核酸を含む修飾ヘテロ核酸 |
| US20240390508A1 (en) | 2021-08-21 | 2024-11-28 | Takeda Pharmaceutical Company Limited | Human transferrin receptor binding peptide-drug conjugate |
| JPWO2023042876A1 (enExample) | 2021-09-15 | 2023-03-23 | ||
| EP4415827A4 (en) * | 2021-10-14 | 2025-12-03 | Univ Arizona State | PROGRAMMABLE DISTRIBUTION PLATFORMS FOR RNA STRUCTURES |
| EP4428238A1 (en) | 2021-11-02 | 2024-09-11 | Rena Therapeutics Inc. | Ligand-bound nucleic acid complex |
| 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 (enExample) | 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 |
| CA2780741C (en) * | 2009-10-12 | 2023-04-04 | Smith Holdings, Llc | Methods and compositions for modulating gene expression using oligonucleotide based drugs administered in vivo or in vitro |
| CN104126010B (zh) * | 2011-12-16 | 2021-04-06 | 国立大学法人东京医科齿科大学 | 嵌合的双链核酸 |
| CA2913499A1 (en) * | 2013-05-30 | 2014-12-04 | National University Corporation Tokyo Medical And Dental University | Double-stranded agents for delivering therapeutic oligonucleotides |
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