CA2853609A1 - Inhibition of viral gene expression - Google Patents

Inhibition of viral gene expression Download PDF

Info

Publication number
CA2853609A1
CA2853609A1 CA2853609A CA2853609A CA2853609A1 CA 2853609 A1 CA2853609 A1 CA 2853609A1 CA 2853609 A CA2853609 A CA 2853609A CA 2853609 A CA2853609 A CA 2853609A CA 2853609 A1 CA2853609 A1 CA 2853609A1
Authority
CA
Canada
Prior art keywords
nucleic acid
acid molecule
seq
modified
sirna
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.)
Abandoned
Application number
CA2853609A
Other languages
English (en)
French (fr)
Inventor
Patrick Arbuthnot
Justin HEAN
Abdullah Ely
Musa MARIMANI
Jolanta BRZEZINSKA
Jennifer D'ONOFRIO
Maximilian C. R. BUFF
Joachim W. Engels
Stefan Bernhardt
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Goethe Universitaet Frankfurt am Main
University of the Witwatersrand, Johannesburg
Original Assignee
Goethe Universitaet Frankfurt am Main
University of the Witwatersrand, Johannesburg
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Goethe Universitaet Frankfurt am Main, University of the Witwatersrand, Johannesburg filed Critical Goethe Universitaet Frankfurt am Main
Publication of CA2853609A1 publication Critical patent/CA2853609A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA 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
    • C12N15/113—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
    • C12N2310/30—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
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3527—Other alkyl chain
    • 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
    • C12N2320/30—Special therapeutic applications

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Oncology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Communicable Diseases (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CA2853609A 2011-10-28 2012-10-26 Inhibition of viral gene expression Abandoned CA2853609A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
ZA2011/07890 2011-10-28
ZA201107890 2011-10-28
PCT/IB2012/055915 WO2013061295A1 (en) 2011-10-28 2012-10-26 Inhibition of viral gene expression

Publications (1)

Publication Number Publication Date
CA2853609A1 true CA2853609A1 (en) 2013-05-02

Family

ID=47326248

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2853609A Abandoned CA2853609A1 (en) 2011-10-28 2012-10-26 Inhibition of viral gene expression

Country Status (9)

Country Link
US (1) US20140350080A1 (https=)
EP (1) EP2771466A1 (https=)
CN (1) CN104011209A (https=)
AP (1) AP2014007650A0 (https=)
BR (1) BR112014010134A2 (https=)
CA (1) CA2853609A1 (https=)
IN (1) IN2014CN03921A (https=)
RU (1) RU2014121304A (https=)
WO (1) WO2013061295A1 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2870246B1 (en) 2012-07-03 2019-09-11 BioMarin Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
CN107849567B (zh) * 2015-06-26 2024-07-23 苏州瑞博生物技术股份有限公司 一种siRNA、含有该siRNA的药物组合物和缀合物及它们的应用
US20170137821A1 (en) 2015-07-17 2017-05-18 Arcturus Therapeutics, Inc. Molecules and agents for treating hepatitis b virus
CA2996722A1 (en) 2015-07-17 2017-01-26 Arcturus Therapeutics, Inc. Compositions and agents against hepatitis b virus and uses thereof
CN110678447B (zh) * 2017-06-16 2023-12-12 卫材R&D管理有限公司 经修饰的核酸单体化合物及寡核酸类似物
CA3083970A1 (en) * 2017-12-01 2019-06-06 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof
US20210395751A1 (en) * 2018-10-31 2021-12-23 The University Of Sydney Compositions and methods for treating viral infections
WO2020135673A1 (zh) 2018-12-28 2020-07-02 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
AU2020280439B2 (en) 2019-05-22 2025-08-14 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
KR20220011689A (ko) 2019-05-22 2022-01-28 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 핵산, 약제학적 조성물, 접합체, 제조 방법, 및 용도
US12590304B2 (en) 2019-05-22 2026-03-31 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
CN113795582B (zh) 2019-05-24 2024-05-24 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN1814614A (zh) * 2005-02-06 2006-08-09 中国人民解放军军事医学科学院毒物药物研究所 核酸、肽核酸衍生物及它们的用途
KR20100069679A (ko) * 2007-09-17 2010-06-24 재단법인 목암생명공학연구소 ＨＢＶ 또는 ＨＣＶ의 유전자 발현을 하향 조절하는 ｓｉＲＮＡ의 혈청 안정성 향상 및 면역 반응 저감 방법

Also Published As

Publication number Publication date
US20140350080A1 (en) 2014-11-27
BR112014010134A2 (pt) 2019-09-24
AP2014007650A0 (en) 2014-05-31
EP2771466A1 (en) 2014-09-03
RU2014121304A (ru) 2015-12-10
IN2014CN03921A (https=) 2015-09-04
WO2013061295A1 (en) 2013-05-02
CN104011209A (zh) 2014-08-27

Similar Documents

Publication Publication Date Title
CA2853609A1 (en) Inhibition of viral gene expression
AU2020244546B2 (en) Chiral control
JP7684373B2 (ja) オフターゲット効果が低下した修飾ｒｎａ剤
EP1789553B1 (en) Oligonucleotides comprising a non-phosphate backbone linkage
EP2970968B1 (en) Bridged bicyclic nucleosides
AU2007209481B2 (en) LNA modified phosphorothiolated oligonucleotides
JP7667775B2 (ja) ヌクレオチド類似体を含有するオリゴヌクレオチド
WO2019170731A1 (en) Nucleotide precursors, nucleotide analogs and oligomeric compounds containing the same
JP2017140030A (ja) 治療剤のためのｕｎａオリゴマーおよびアミダイト
WO2010039543A2 (en) Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating rna interference
JP2020505032A (ja) エンドソーム切断可能なリンカー
Brzezinska et al. Synthesis of 2′-O-guanidinopropyl-modified nucleoside phosphoramidites and their incorporation into siRNAs targeting hepatitis B virus
US20090069263A1 (en) 4'-thioarabinonucleotide-containing oligonucleotides, compounds and methods for their preparation and uses thereof
WO2021132589A1 (ja) ポリヌクレオチド及び医薬組成物
JP2026503499A (ja) 修飾されたヌクレオシド単量体及び二本鎖リボ核酸
WO2025119303A1 (en) Abasic modification compound, gene modulating agents and the use thereof
Malinowska Cross-linking methods to identify the mRNA targets of microRNAs
WO2025036984A1 (en) Chemically modified antisense oligonucleotides (asos) and compositions for rna editing
WO2025223315A1 (zh) 一种用于肝脏特异性递送的多靶标双链rna偶联物及药物组合物
CA3236838A1 (en) Stabilized rna agents
WO2025188589A1 (en) NOVEL NUCLEOTIDES AND OLIGONUCLEOTIDES AND RNAi AGENTS COMPRISING THE SAME
Malek-Adamian Synthesis of Novel 2′, 4′-Modified Nucleoside Analogues and Their Properties in Oligonucleotides and Nanoparticle Development for Targeted Delivery to Glioblastoma Multiformes Drr-Expressing Cells
KR20260053296A (ko) Rna 편집을 위한 화학적으로 변형된 안티센스 올리고뉴클레오티드(asos) 및 조성물
Bogojeski Synthesis and characterization of novel self-delivering amino acid-RNA conjugates for the development of potent siRNA prodrugs

Legal Events

Date Code Title Description
FZDE Discontinued

Effective date: 20161026