KR20220009971A - 최종당화산물 수용체 알엔에이의 조절인자 및 조절 - Google Patents

최종당화산물 수용체 알엔에이의 조절인자 및 조절 Download PDF

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Publication number
KR20220009971A
KR20220009971A KR1020217039458A KR20217039458A KR20220009971A KR 20220009971 A KR20220009971 A KR 20220009971A KR 1020217039458 A KR1020217039458 A KR 1020217039458A KR 20217039458 A KR20217039458 A KR 20217039458A KR 20220009971 A KR20220009971 A KR 20220009971A
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rage
aon
exon
mrna
expression
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KR1020217039458A
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Korean (ko)
Inventor
스티븐 윌턴
멀린 크리스토퍼 토마스
카를로스 로사도
라엘린 제인 피커링
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모나쉬 유니버시티
머독 유니버시티
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Priority claimed from AU2019901641A external-priority patent/AU2019901641A0/en
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Publication of KR20220009971A publication Critical patent/KR20220009971A/ko
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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
    • C12N15/113—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
    • 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
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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
    • 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
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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
    • C12N2310/323—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
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    • 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
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    • 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
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C—CHEMISTRY; METALLURGY
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    • 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)
KR1020217039458A 2019-05-14 2020-05-07 최종당화산물 수용체 알엔에이의 조절인자 및 조절 Pending KR20220009971A (ko)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
AU2019901641A AU2019901641A0 (en) 2019-05-14 Modulators and Modulation of the Receptor for Advanced Glycation End-Products RNA
AU2019901641 2019-05-14
AU2019902095 2019-06-17
AU2019902095A AU2019902095A0 (en) 2019-06-17 Modulators and Modulation of the Receptor for Advanced Glycation End-Products RNA
AU2019902772 2019-08-02
AU2019902772A AU2019902772A0 (en) 2019-08-02 Modulators and Modulation of the Receptor for Advanced Glycation End-Products RNA
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

Publications (1)

Publication Number Publication Date
KR20220009971A true KR20220009971A (ko) 2022-01-25

Family

ID=73290056

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020217039458A Pending KR20220009971A (ko) 2019-05-14 2020-05-07 최종당화산물 수용체 알엔에이의 조절인자 및 조절

Country Status (12)

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

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
AU2023447004A1 (en) * 2023-05-05 2025-10-16 RAGE Biotech Pty Ltd Methods of monitoring splicing
KR20260027924A (ko) * 2023-05-19 2026-03-03 레이지 바이오텍 피티와이 리미티드 연기 노출의 영향 최소화 방법
KR20260027923A (ko) * 2023-05-19 2026-03-03 레이지 바이오텍 피티와이 리미티드 폐 섬유증 치료 방법
AU2024352066A1 (en) * 2023-09-27 2026-04-09 Changchun Genescience Pharmaceutical Co., Ltd. New sirna, composition comprising same, and use
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

Family Cites Families (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US5149797A (en) 1990-02-15 1992-09-22 The Worcester Foundation For Experimental Biology Method of site-specific alteration of rna and production of encoded polypeptides
US20020123476A1 (en) 1991-03-19 2002-09-05 Emanuele R. Martin Therapeutic delivery compositions and methods of use thereof
US6806084B1 (en) 1992-06-04 2004-10-19 The Regents Of The University Of California Methods for compositions for in vivo gene delivery
ATE330644T1 (de) 1995-12-18 2006-07-15 Angiotech Biomaterials Corp Vernetzten polymerisatmassen und verfahren für ihre verwendung
DE69834038D1 (de) 1997-07-01 2006-05-18 Isis Pharmaceutical Inc Zusammensetzungen und verfahren zur verabreichung von oligonukleotiden über die speiseröhre
JP2002537343A (ja) 1999-02-23 2002-11-05 アイシス・ファーマシューティカルス・インコーポレーテッド 多重粒子製剤
US6287860B1 (en) 2000-01-20 2001-09-11 Isis Pharmaceuticals, Inc. Antisense inhibition of MEKK2 expression
JP3837494B2 (ja) * 2001-03-19 2006-10-25 国立大学法人金沢大学 可溶型ｒａｇｅタンパク質
US7026121B1 (en) * 2001-06-08 2006-04-11 Expression Diagnostics, Inc. Methods and compositions for diagnosing and monitoring transplant rejection
US6905827B2 (en) * 2001-06-08 2005-06-14 Expression Diagnostics, Inc. Methods and compositions for diagnosing or monitoring auto immune and chronic inflammatory diseases
US6965025B2 (en) 2001-12-10 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of connective tissue growth factor expression
JP2008539698A (ja) * 2005-04-29 2008-11-20 ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル 転写後レベルでの核酸発現調節のための方法および組成物
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
JP2010233542A (ja) * 2009-03-31 2010-10-21 Kanazawa Univ Ｒａｇｅ遺伝子の２種類のスプライシングバリアントを区別して増幅可能なプライマーセット及びプローブ
US20100303776A1 (en) * 2009-04-16 2010-12-02 The University Of North Carolina At Chapel Hill Methods and compositions for regulated expression of multiple nucleic acids
US9315298B2 (en) 2011-08-25 2016-04-19 Db Equipment As Accessory bag having reinforced sidewalls and variable length
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
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

Also Published As

Publication number Publication date
WO2020227758A8 (en) 2021-11-18
JP2022533358A (ja) 2022-07-22
US20240141360A1 (en) 2024-05-02
US20210388363A1 (en) 2021-12-16
US11441148B2 (en) 2022-09-13
AU2025223886A1 (en) 2025-09-18
WO2020227758A1 (en) 2020-11-19
US20230015481A1 (en) 2023-01-19
EP3969590A1 (en) 2022-03-23
CA3140209A1 (en) 2020-11-19
AU2020276337B2 (en) 2025-05-29
SG11202112144RA (en) 2021-11-29
US12565656B2 (en) 2026-03-03
JP2025106369A (ja) 2025-07-15
AU2020276337A1 (en) 2021-12-02
MX2021013858A (es) 2022-03-22
US11674143B2 (en) 2023-06-13
IL288040A (en) 2022-01-01
CN114127288A (zh) 2022-03-01
EP3969590A4 (en) 2024-05-15
BR112021022714A2 (pt) 2022-01-18

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