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

Info

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
Authority
AU
Australia
Prior art keywords
aon
rage
exon
antisense oligonucleotide
mrna
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.)
Active
Application number
AU2020276337A
Other languages
English (en)
Other versions
AU2020276337A1 (en
Inventor
Raelene Jane Pickering
Carlos Rosado
Merlin Christopher Thomas
Stephen Wilton
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.)
Murdoch University
Monash University
Original Assignee
Murdoch University
Monash University
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
Priority claimed from AU2019901641A external-priority patent/AU2019901641A0/en
Application filed by Murdoch University, Monash University filed Critical Murdoch University
Publication of AU2020276337A1 publication Critical patent/AU2020276337A1/en
Application granted granted Critical
Publication of AU2020276337B2 publication Critical patent/AU2020276337B2/en
Priority to AU2025223886A priority Critical patent/AU2025223886A1/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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

Landscapes

  • 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
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

Related Child Applications (1)

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

Publications (2)

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

Family

ID=73290056

Family Applications (2)

Application Number Title Priority Date Filing Date
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

Family Applications After (1)

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

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

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Family Cites Families (18)

* 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
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

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
KALEA, A. et al., 'Alternative splicing of RAGE: roles in biology and disease', Frontiers in Bioscience. June 2011, vol 16, pages 2756-2770. *

Also Published As

Publication number Publication date
WO2020227758A8 (en) 2021-11-18
JP2022533358A (ja) 2022-07-22
KR20220009971A (ko) 2022-01-25
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
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

Similar Documents

Publication Publication Date Title
AU2020276337B2 (en) Modulators and modulation of the receptor for advanced glycation end-products RNA
US20230407309A1 (en) Antisense oligomers for treatment of disease
CA3193706A1 (en) Treatment of optic atrophy
US20250066782A1 (en) Novel Retinitis Pigmentosa Treatment
US11459563B2 (en) Treatment for NEAT1 associated disease
RU2820247C2 (ru) Модуляторы и модуляция рнк рецептора конечных продуктов глубокого гликирования
US20220275369A1 (en) Antisense therapy for ptp1b related conditions
JP6885596B2 (ja) 多発性硬化症の治療
US20250368988A1 (en) Compositions and Methods for Treating FUS Associated Diseases
HK40069896A (en) Modulators and modulation of the receptor for advanced glycation end-products rna
WO2026025143A1 (en) Antisense treatment for metabolic diseases
AU2024364517A1 (en) Treatments for psoriasis
WO2025081230A1 (en) Treatments for psoriasis
WO2025000015A1 (en) Antisense oligomer treatment for bone loss

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)