AU2022399905A1 - Antisense oligonucleotides (aso) for efficient and precise rna editing with endogenous adenosine deaminase acting on rna (adar) - Google Patents

Antisense oligonucleotides (aso) for efficient and precise rna editing with endogenous adenosine deaminase acting on rna (adar) Download PDF

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Publication number
AU2022399905A1
AU2022399905A1 AU2022399905A AU2022399905A AU2022399905A1 AU 2022399905 A1 AU2022399905 A1 AU 2022399905A1 AU 2022399905 A AU2022399905 A AU 2022399905A AU 2022399905 A AU2022399905 A AU 2022399905A AU 2022399905 A1 AU2022399905 A1 AU 2022399905A1
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Prior art keywords
chemically modified
linkages
modified oligonucleotide
editing
nucleotides
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AU2022399905A
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Ngadhnjim LATIFI
Laura Sophia PFEIFFER
Thorsten Stafforst
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Eberhard Karls Universitaet Tuebingen
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Eberhard Karls Universitaet Tuebingen
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/34Allele or polymorphism specific uses

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Cardiology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Epidemiology (AREA)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2022399905A 2021-11-30 2022-11-29 Antisense oligonucleotides (aso) for efficient and precise rna editing with endogenous adenosine deaminase acting on rna (adar) Pending AU2022399905A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP21211372.4 2021-11-30
EP21211372 2021-11-30
PCT/EP2022/083711 WO2023099494A1 (en) 2021-11-30 2022-11-29 Antisense oligonucleotides (aso) for efficient and precise rna editing with endogenous adenosine deaminase acting on rna (adar)

Publications (1)

Publication Number Publication Date
AU2022399905A1 true AU2022399905A1 (en) 2024-05-23

Family

ID=78820190

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2022399905A Pending AU2022399905A1 (en) 2021-11-30 2022-11-29 Antisense oligonucleotides (aso) for efficient and precise rna editing with endogenous adenosine deaminase acting on rna (adar)

Country Status (9)

Country Link
US (1) US20230235329A1 (https=)
EP (1) EP4441216A1 (https=)
JP (1) JP2024544067A (https=)
KR (1) KR20240111752A (https=)
CN (1) CN118355119A (https=)
AU (1) AU2022399905A1 (https=)
CA (1) CA3238033A1 (https=)
IL (1) IL312762A (https=)
WO (1) WO2023099494A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024114908A1 (en) * 2022-11-30 2024-06-06 Eberhard Karls Universität Tübingen Chemically modified antisense oligonucleotides (asos) and compositions comprising the same for rna editing
AU2024325709A1 (en) * 2023-08-15 2026-01-29 Airna Corporation Chemically modified antisense oligonucleotides (asos) and compositions for rna editing
WO2025073902A1 (en) * 2023-10-06 2025-04-10 Eberhard Karls Universität Tübingen Chemical and sequence optimisation of antisense oligonucleotides for adar-mediated rna editing
WO2025172372A1 (en) 2024-02-12 2025-08-21 Airna Corporation Conjugate for targeted delivery of an antisense oligonucleotide
WO2025242018A1 (zh) * 2024-05-20 2025-11-27 时夕(广州)生物科技有限公司 一种经修饰的向导agRNA

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US10941402B2 (en) 2016-09-01 2021-03-09 Proqr Therapeutics Ii B.V. Chemically modified single-stranded RNA-editing oligonucleotides
WO2020001793A1 (en) 2018-06-29 2020-01-02 Eberhard-Karls-Universität Tübingen Artificial nucleic acids for rna editing
CA3158528A1 (en) * 2019-12-23 2021-07-01 Jim SWILDENS Antisense oligonucleotides for nucleotide deamination in the treatment of stargardt disease

Also Published As

Publication number Publication date
IL312762A (en) 2024-07-01
JP2024544067A (ja) 2024-11-27
US20230235329A1 (en) 2023-07-27
WO2023099494A1 (en) 2023-06-08
CA3238033A1 (en) 2023-06-08
WO2023099494A9 (en) 2024-10-17
EP4441216A1 (en) 2024-10-09
KR20240111752A (ko) 2024-07-17
CN118355119A (zh) 2024-07-16

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