AU2013324716B2 - Oligomers with improved off-target profile - Google Patents

Oligomers with improved off-target profile Download PDF

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Publication number
AU2013324716B2
AU2013324716B2 AU2013324716A AU2013324716A AU2013324716B2 AU 2013324716 B2 AU2013324716 B2 AU 2013324716B2 AU 2013324716 A AU2013324716 A AU 2013324716A AU 2013324716 A AU2013324716 A AU 2013324716A AU 2013324716 B2 AU2013324716 B2 AU 2013324716B2
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hsa
mir
monomers
oligomer
affinity region
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AU2013324716A1 (en
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Thorleif Moeller
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Guandong Mijinjia Biotechnology Co Ltd
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Guandong Mijinjia Biotechnology Co Ltd
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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
    • C12N15/1131—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 against viruses
    • 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
    • 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
    • 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
    • C12N2310/11—Antisense
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • 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
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/321—2'-O-R Modification
    • 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
    • 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 ===---===
    • 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
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2013324716A 2012-09-26 2013-09-26 Oligomers with improved off-target profile Active AU2013324716B2 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201261705668P 2012-09-26 2012-09-26
DKPA201200587 2012-09-26
DKPA201200587 2012-09-26
US61/705,668 2012-09-26
PCT/DK2013/050308 WO2014048441A1 (en) 2012-09-26 2013-09-26 Oligomers with improved off-target profile

Publications (2)

Publication Number Publication Date
AU2013324716A1 AU2013324716A1 (en) 2015-04-09
AU2013324716B2 true AU2013324716B2 (en) 2019-06-13

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Family Applications (1)

Application Number Title Priority Date Filing Date
AU2013324716A Active AU2013324716B2 (en) 2012-09-26 2013-09-26 Oligomers with improved off-target profile

Country Status (7)

Country Link
US (1) US9725721B2 (enExample)
EP (1) EP2900822B1 (enExample)
JP (1) JP6352269B2 (enExample)
CN (1) CN104955950B (enExample)
AU (1) AU2013324716B2 (enExample)
CA (1) CA2886116C (enExample)
WO (1) WO2014048441A1 (enExample)

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PH12023550488A1 (en) 2012-04-25 2024-06-24 Sanofi Sa Microrna compounds and methods for modulating mir-21 activity
JP6430945B2 (ja) 2012-10-02 2018-11-28 センテナリ インスティテュート オブ キャンサー メディシン アンド セル バイオロジーＣｅｎｔｅｎａｒｙ Ｉｎｓｔｉｔｕｔｅ Ｏｆ Ｃａｎｃｅｒ Ｍｅｄｉｃｉｎｅ Ａｎｄ Ｃｅｌｌ Ｂｉｏｌｏｇｙ Ｒｎａ活性及び血管透過性の調節
UA116639C2 (uk) 2012-10-09 2018-04-25 Рег'Юлес Терап'Ютікс Інк. Способи лікування синдрому альпорта
TW201446791A (zh) 2013-05-01 2014-12-16 Regulus Therapeutics Inc 用於調節mir-122之微小rna化合物及方法
WO2015061536A1 (en) 2013-10-25 2015-04-30 Regulus Therapeutics Inc. Microrna compounds and methods for modulating mir-21 activity
KR102633955B1 (ko) 2014-06-13 2024-02-06 도레이 카부시키가이샤 대장암의 검출 키트 또는 디바이스 및 검출 방법
JP6804975B2 (ja) * 2014-06-13 2020-12-23 東レ株式会社 乳がんの検出キット又はデバイス及び検出方法
CN106460068B (zh) * 2014-06-16 2024-08-23 东丽株式会社 胃癌的检测试剂盒或装置以及检测方法
CN119842899A (zh) 2014-06-18 2025-04-18 东丽株式会社 肝癌的检测试剂盒或装置以及检测方法
JP6385732B2 (ja) * 2014-06-27 2018-09-05 国立大学法人北海道大学 免疫応答制御剤
RU2017105342A (ru) 2014-08-07 2018-09-13 Регулус Терапьютикс Инк. НАЦЕЛИВАНИЕ НА микроРНК ПРИ РАССТРОЙСТВАХ ОБМЕНА ВЕЩЕСТВ
BR112017003608B1 (pt) * 2014-09-16 2023-11-07 Toray Industries, Inc MÉTODO PARA A ANÁLISE COMPARATIVA DO NÍVEL DE EXPRESSÃO DE miRNA(S) ALVO(S) E CHIP PARA ANÁLISE DA EXPRESSÃO DE miRNA
WO2016115609A1 (en) * 2015-01-20 2016-07-28 Katholieke Universiteit Leuven Colon cancer
WO2016137235A2 (ko) 2015-02-25 2016-09-01 (주)바이오니아 마이크로 rna를 유효 성분으로 포함하는 암 치료용 약학 조성물
CN104826133B (zh) * 2015-05-01 2020-11-24 北京博奥医学检验所有限公司 一种与肺腺癌有关的miRNA及其药物组合物
CN107058470B (zh) * 2016-11-03 2020-09-01 中国人民解放军陆军军医大学 一种通过联合miR-136和miR-4791预测急性高山病发病风险的诊断试剂盒
CN107058472B (zh) * 2016-11-03 2020-10-27 中国人民解放军陆军军医大学 一种通过四种血浆microRNA联合诊断急性高山病的诊断试剂盒
CN107058471B (zh) * 2016-11-03 2020-09-01 中国人民解放军陆军军医大学 一种通过四种microRNA生物标志物联合预测急性高山病发病风险的试剂盒
CN107058475B (zh) * 2016-11-03 2020-10-27 中国人民解放军陆军军医大学 一种联合miR-676、miR-181b和miR-193b诊断急性高山病的试剂盒
CN107058474B (zh) * 2016-11-03 2020-09-01 中国人民解放军陆军军医大学 一种通过血浆microRNA-3591-3p诊断急性高山病的诊断试剂盒
CN107058473B (zh) * 2016-11-03 2020-10-27 中国人民解放军陆军军医大学 一种联合miR-676、miR-181b和miR-3591诊断急性高山病的试剂盒
CN108251422A (zh) * 2016-12-29 2018-07-06 昆山彭济凯丰生物科技有限公司 通过miR-6069进行抗癌的方法和药物及其应用
CN108295086B (zh) * 2017-01-13 2021-11-02 昆山彭济凯丰生物科技有限公司 通过miR-4418进行抗癌的方法和药物及其应用
CN108295260A (zh) * 2017-01-13 2018-07-20 彭长庚 通过miR-1915-3p进行抗癌的方法和药物及其应用
CN108310381A (zh) * 2017-01-16 2018-07-24 昆山彭济凯丰生物科技有限公司 通过miR-6511b-3p进行抗癌的方法和药物及其应用
CN107502609A (zh) * 2017-09-08 2017-12-22 中国科学院深圳先进技术研究院 一种靶向cJun信号通路的miRNA及其制备方法和应用
KR102070969B1 (ko) * 2018-04-10 2020-01-29 한국한의학연구원 통증 판별용 마커
CN112400020B (zh) 2018-05-08 2024-11-19 莱古路斯治疗法股份有限公司 作为具有hcv抗病毒活性与降低的高胆红素血症副作用的mir-122抑制剂的galnac缀合的经修饰的寡核苷酸
CN109468375B (zh) * 2018-12-28 2021-05-14 固安博健生物技术有限公司 分子标志物在骨质疏松症中的应用
CN113993588B (zh) * 2019-06-05 2024-03-01 癌症干细胞科技公司 癌治疗剂
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Publication number Publication date
CA2886116A1 (en) 2014-04-03
CN104955950B (zh) 2019-04-26
CA2886116C (en) 2022-06-07
AU2013324716A1 (en) 2015-04-09
EP2900822A4 (en) 2016-05-25
EP2900822B1 (en) 2021-11-24
JP2016513950A (ja) 2016-05-19
JP6352269B2 (ja) 2018-07-04
WO2014048441A1 (en) 2014-04-03
US9725721B2 (en) 2017-08-08
US20160145620A1 (en) 2016-05-26
CN104955950A (zh) 2015-09-30
EP2900822A1 (en) 2015-08-05

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