CA3238033A1 - 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
CA3238033A1
CA3238033A1 CA3238033A CA3238033A CA3238033A1 CA 3238033 A1 CA3238033 A1 CA 3238033A1 CA 3238033 A CA3238033 A CA 3238033A CA 3238033 A CA3238033 A CA 3238033A CA 3238033 A1 CA3238033 A1 CA 3238033A1
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Canada
Prior art keywords
chemically modified
linkages
modified oligonucleotide
editing
linkage
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.)
Pending
Application number
CA3238033A
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English (en)
French (fr)
Inventor
Thorsten Stafforst
Ngadhnjim LATIFI
Laura Sophia PFEIFFER
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.)
Eberhard Karls Universitaet Tuebingen
Original Assignee
Eberhard Karls Universitaet Tuebingen
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
Application filed by Eberhard Karls Universitaet Tuebingen filed Critical Eberhard Karls Universitaet Tuebingen
Publication of CA3238033A1 publication Critical patent/CA3238033A1/en
Pending legal-status Critical Current

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    • 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
    • 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
    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele 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)
CA3238033A 2021-11-30 2022-11-29 Antisense oligonucleotides (aso) for efficient and precise rna editing with endogenous adenosine deaminase acting on rna (adar) Pending CA3238033A1 (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
CA3238033A1 true CA3238033A1 (en) 2023-06-08

Family

ID=78820190

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3238033A Pending CA3238033A1 (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
AU2022399905A1 (en) 2024-05-23
WO2023099494A9 (en) 2024-10-17
EP4441216A1 (en) 2024-10-09
KR20240111752A (ko) 2024-07-17
CN118355119A (zh) 2024-07-16

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