AU2021276730A1 - Oligonucleotides - Google Patents

Oligonucleotides Download PDF

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Publication number
AU2021276730A1
AU2021276730A1 AU2021276730A AU2021276730A AU2021276730A1 AU 2021276730 A1 AU2021276730 A1 AU 2021276730A1 AU 2021276730 A AU2021276730 A AU 2021276730A AU 2021276730 A AU2021276730 A AU 2021276730A AU 2021276730 A1 AU2021276730 A1 AU 2021276730A1
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AU
Australia
Prior art keywords
oligonucleotide
bases
modified
region
continuous
Prior art date
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Pending
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AU2021276730A
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English (en)
Inventor
Michael Paul Marie Gantier
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Hudson Institute of Medical Research
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Hudson Institute of Medical Research
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Priority claimed from AU2020901606A external-priority patent/AU2020901606A0/en
Application filed by Hudson Institute of Medical Research filed Critical Hudson Institute of Medical Research
Publication of AU2021276730A1 publication Critical patent/AU2021276730A1/en
Pending legal-status Critical Current

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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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    • 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
    • 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
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
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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
    • C12N15/1138—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 receptors or cell surface proteins
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
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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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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/17—Immunomodulatory nucleic acids
    • C—CHEMISTRY; METALLURGY
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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
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
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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
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • C—CHEMISTRY; METALLURGY
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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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    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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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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    • C12N2330/00—Production
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    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/178—Oligonucleotides characterized by their use miRNA, siRNA or ncRNA

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
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  • Wood Science & Technology (AREA)
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  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Analytical Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2021276730A 2020-05-19 2021-05-19 Oligonucleotides Pending AU2021276730A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
AU2020901606 2020-05-19
AU2020901606A AU2020901606A0 (en) 2020-05-19 Oligonucleotides
PCT/AU2021/050469 WO2021232099A1 (en) 2020-05-19 2021-05-19 Oligonucleotides

Publications (1)

Publication Number Publication Date
AU2021276730A1 true AU2021276730A1 (en) 2023-01-05

Family

ID=78708815

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2021276730A Pending AU2021276730A1 (en) 2020-05-19 2021-05-19 Oligonucleotides

Country Status (10)

Country Link
US (2) US20230348903A1 (https=)
EP (1) EP4153751A4 (https=)
JP (1) JP2023526090A (https=)
KR (1) KR20230013266A (https=)
CN (1) CN115916978A (https=)
AU (1) AU2021276730A1 (https=)
BR (1) BR112022023310A2 (https=)
CA (1) CA3171986A1 (https=)
MX (1) MX2022014495A (https=)
WO (1) WO2021232099A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN115916978A (zh) 2020-05-19 2023-04-04 哈德森医学研究所 寡核苷酸
US20240384268A1 (en) * 2021-04-08 2024-11-21 Hudson Institute of Medical Research Oligonucleotides
CN118460654B (zh) * 2024-07-10 2025-02-14 凯莱英医药集团（天津）股份有限公司 一种Givosiran的制备方法
CN119592567B (zh) * 2024-12-10 2025-12-02 浙江大学 基于肿瘤剪接转换治疗的反义寡核苷酸及其应用

Family Cites Families (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6320041B1 (en) 2001-04-13 2001-11-20 Trilink Biotechnologies, Inc. Pre-activated carbonyl linkers for the modification of oligonucleotides
US8153141B2 (en) 2002-04-04 2012-04-10 Coley Pharmaceutical Gmbh Immunostimulatory G, U-containing oligoribonucleotides
US20050239733A1 (en) 2003-10-31 2005-10-27 Coley Pharmaceutical Gmbh Sequence requirements for inhibitory oligonucleotides
WO2005097993A2 (en) * 2004-02-19 2005-10-20 Coley Pharmaceutical Group, Inc. Immunostimulatory viral rna oligonucleotides
WO2006063152A2 (en) * 2004-12-08 2006-06-15 3M Innovative Properties Company Immunostimulatory combinations and methods
EP1819365B1 (en) 2004-12-09 2014-07-02 Alnylam Pharmaceuticals Inc. Compositions and methods for inducing an immune response in a mammal and methods of avoiding an immune response to oligonucleotide agents such as short interfering RNAs
CN101160401A (zh) * 2005-02-24 2008-04-09 科勒制药集团公司 免疫刺激寡核苷酸
EP1764107A1 (en) * 2005-09-14 2007-03-21 Gunther Hartmann Compositions comprising immunostimulatory RNA oligonucleotides and methods for producing said RNA oligonucleotides
US8076068B2 (en) 2005-09-14 2011-12-13 Gunther Hartmann Method for determining immunostimulatory activity of RNA oligonucleotides
KR20080059595A (ko) * 2005-09-27 2008-06-30 콜리 파마슈티칼 게엠베하 어댑터 올리고뉴클레오티드를 사용하는 ｔｌｒ-매개 면역반응의 조절
PL1957647T3 (pl) * 2005-11-25 2015-07-31 Zoetis Belgium S A Oligorybonukleotydy immunostymulujące
CN101331229B (zh) * 2005-11-25 2012-08-29 科勒制药股份公司 免疫刺激性寡核糖核苷酸
EP2021008B1 (en) * 2006-04-07 2015-12-02 Idera Pharmaceuticals, Inc. Stabilized immune modulatory rna (simra) compounds for tlr7 and tlr8
AU2007268075C1 (en) 2006-06-01 2013-03-07 Trilink Biotechnologies Chemically modified oligonucleotide primers for nucleic acid amplification
AU2007280690C1 (en) * 2006-07-31 2012-08-23 Curevac Gmbh Nucleic acid of formula (I): GIXmGn, or (II): CIXmCn, in particular as an immune-stimulating agent/adjuvant
EP3342415B1 (en) * 2006-08-08 2022-01-26 Rheinische Friedrich-Wilhelms-Universität Bonn Structure and use of 5' phosphate oligonucleotides
WO2008019486A1 (en) 2006-08-16 2008-02-21 Protiva Biotherapeutics, Inc. Nucleic acid modulation of toll-like receptor-mediated immune stimulation
CA2692161C (en) * 2007-07-09 2015-09-29 Idera Pharmaceuticals, Inc. Stabilized immune modulatory rna (simra) compounds
JP5753382B2 (ja) * 2007-11-06 2015-07-22 アディウティーダ ファーマシューティカルズ ゲーエムベーハー 修飾されたオリゴリン酸基を含有する免疫賦活オリゴリボヌクレオチドアナログ
US20100047188A1 (en) * 2008-08-04 2010-02-25 Idera Pharmaceuticals, Inc. Modulation of toll-like receptor 8 expression by antisense oligonucleotides
US9631227B2 (en) 2009-07-06 2017-04-25 Trilink Biotechnologies, Inc. Chemically modified ligase cofactors, donors and acceptors
EP3191591A1 (en) * 2014-09-12 2017-07-19 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
WO2016205323A1 (en) * 2015-06-18 2016-12-22 Alnylam Pharmaceuticals, Inc. Polynucleotde agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
ES2964690T3 (es) 2015-09-21 2024-04-09 Trilink Biotechnologies Llc Método para sintetizar ARN con caperuza 5'
WO2017100587A1 (en) * 2015-12-09 2017-06-15 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting programmed cell death 1 ligand 1 (pd-l1) and methods of use thereof
WO2018184003A1 (en) 2017-03-31 2018-10-04 Dana-Farber Cancer Institute, Inc. Modulating dsrna editing, sensing, and metabolism to increase tumor immunity and improve the efficacy of cancer immunotherapy and/or modulators of intratumoral interferon
US11603532B2 (en) * 2017-06-02 2023-03-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
CN115916978A (zh) 2020-05-19 2023-04-04 哈德森医学研究所 寡核苷酸

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WO2021232099A1 (en) 2021-11-25
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JP2023526090A (ja) 2023-06-20

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