AU2020276337B2 - Modulators and modulation of the receptor for advanced glycation end-products RNA - Google Patents

Modulators and modulation of the receptor for advanced glycation end-products RNA Download PDF

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Publication number
AU2020276337B2
AU2020276337B2 AU2020276337A AU2020276337A AU2020276337B2 AU 2020276337 B2 AU2020276337 B2 AU 2020276337B2 AU 2020276337 A AU2020276337 A AU 2020276337A AU 2020276337 A AU2020276337 A AU 2020276337A AU 2020276337 B2 AU2020276337 B2 AU 2020276337B2
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aon
rage
exon
antisense oligonucleotide
mrna
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AU2020276337A1 (en
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Raelene Jane Pickering
Carlos Rosado
Merlin Christopher Thomas
Stephen Wilton
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Murdoch University
Monash University
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Murdoch University
Monash University
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • 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
    • 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
    • C12N2310/30—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
    • C12N2310/3233—Morpholino-type ring
    • 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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • 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
    • 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
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
AU2020276337A 2019-05-14 2020-05-07 Modulators and modulation of the receptor for advanced glycation end-products RNA Active AU2020276337B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2025223886A AU2025223886A1 (en) 2019-05-14 2025-08-29 Modulators and modulation of the receptor for advanced glycation end-products RNA

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
AU2019901641 2019-05-14
AU2019901641A AU2019901641A0 (en) 2019-05-14 Modulators and Modulation of the Receptor for Advanced Glycation End-Products RNA
AU2019902095A AU2019902095A0 (en) 2019-06-17 Modulators and Modulation of the Receptor for Advanced Glycation End-Products RNA
AU2019902095 2019-06-17
AU2019902772A AU2019902772A0 (en) 2019-08-02 Modulators and Modulation of the Receptor for Advanced Glycation End-Products RNA
AU2019902772 2019-08-02
AU2019903900 2019-10-16
AU2019903900A AU2019903900A0 (en) 2019-10-16 Modulators and Modulation of the Receptor for Advanced Glycation End-Products RNA
PCT/AU2020/050449 WO2020227758A1 (en) 2019-05-14 2020-05-07 Modulators and modulation of the receptor for advanced glycation end-products rna

Related Child Applications (1)

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AU2025223886A Division AU2025223886A1 (en) 2019-05-14 2025-08-29 Modulators and modulation of the receptor for advanced glycation end-products RNA

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AU2020276337A1 AU2020276337A1 (en) 2021-12-02
AU2020276337B2 true AU2020276337B2 (en) 2025-05-29

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AU2020276337A Active AU2020276337B2 (en) 2019-05-14 2020-05-07 Modulators and modulation of the receptor for advanced glycation end-products RNA
AU2025223886A Pending AU2025223886A1 (en) 2019-05-14 2025-08-29 Modulators and modulation of the receptor for advanced glycation end-products RNA

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US (3) US11441148B2 (enExample)
EP (1) EP3969590A4 (enExample)
JP (2) JP2022533358A (enExample)
KR (1) KR20220009971A (enExample)
CN (1) CN114127288A (enExample)
AU (2) AU2020276337B2 (enExample)
BR (1) BR112021022714A2 (enExample)
CA (1) CA3140209A1 (enExample)
IL (1) IL288040A (enExample)
MX (1) MX2021013858A (enExample)
SG (1) SG11202112144RA (enExample)
WO (1) WO2020227758A1 (enExample)

Families Citing this family (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022533358A (ja) 2019-05-14 2022-07-22 モナシュ ユニバーシティ 終末糖化産物受容体ｒｎａのモジュレーター及び調節
WO2024229505A1 (en) * 2023-05-05 2024-11-14 RAGE Biotech Pty Ltd Administration of oligonucleotides
KR20260028678A (ko) * 2023-05-05 2026-03-04 레이지 바이오텍 피티와이 리미티드 스플라이싱 모니터링 방법
WO2024239040A1 (en) * 2023-05-19 2024-11-28 RAGE Biotech Pty Ltd Methods of minimising the effects of smoke exposure
WO2024239039A1 (en) * 2023-05-19 2024-11-28 RAGE Biotech Pty Ltd Methods of treating pulmonary fibrosis
CN119709736A (zh) * 2023-09-27 2025-03-28 长春金赛药业有限责任公司 一种新的siRNA、包含其的组合物及用途
WO2025076595A1 (en) * 2023-10-12 2025-04-17 RAGE Biotech Pty Ltd Rnai agents targeting rage
WO2025222258A1 (en) * 2024-04-24 2025-10-30 RAGE Biotech Pty Ltd Methods of treating or preventing obstructive pulmonary disease
WO2025236048A1 (en) * 2024-05-17 2025-11-20 RAGE Biotech Pty Ltd Methods of treating the effects of smoke

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WO2019222693A1 (en) * 2018-05-17 2019-11-21 Lifesplice Pharma Llc Splice modulating oligonucleotides targeting receptor for advanced glycation end products and methods of use

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Also Published As

Publication number Publication date
US20230015481A1 (en) 2023-01-19
US20210388363A1 (en) 2021-12-16
MX2021013858A (es) 2022-03-22
BR112021022714A2 (pt) 2022-01-18
US20240141360A1 (en) 2024-05-02
US11674143B2 (en) 2023-06-13
WO2020227758A1 (en) 2020-11-19
EP3969590A4 (en) 2024-05-15
JP2022533358A (ja) 2022-07-22
IL288040A (en) 2022-01-01
US11441148B2 (en) 2022-09-13
EP3969590A1 (en) 2022-03-23
CA3140209A1 (en) 2020-11-19
US12565656B2 (en) 2026-03-03
JP2025106369A (ja) 2025-07-15
KR20220009971A (ko) 2022-01-25
CN114127288A (zh) 2022-03-01
AU2020276337A1 (en) 2021-12-02
SG11202112144RA (en) 2021-11-29
AU2025223886A1 (en) 2025-09-18
WO2020227758A8 (en) 2021-11-18

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