IL312762A - ANTISENSE OLIGONUCLEOTIDES (ASO) for efficient and accurate RNA editing with an endogenous adaminase that acts on RNA (ADAR) - Google Patents

ANTISENSE OLIGONUCLEOTIDES (ASO) for efficient and accurate RNA editing with an endogenous adaminase that acts on RNA (ADAR)

Info

Publication number
IL312762A
IL312762A IL312762A IL31276224A IL312762A IL 312762 A IL312762 A IL 312762A IL 312762 A IL312762 A IL 312762A IL 31276224 A IL31276224 A IL 31276224A IL 312762 A IL312762 A IL 312762A
Authority
IL
Israel
Prior art keywords
rna
adar
aso
efficient
antisense oligonucleotides
Prior art date
Application number
IL312762A
Other languages
English (en)
Hebrew (he)
Inventor
Ngadhnjim Latifi
Laura Sophia Pfeiffer
Thorsten Stafforst
Original Assignee
Eberhard Karls Universit?T T?Bingen
Ngadhnjim Latifi
Laura Sophia Pfeiffer
Thorsten Stafforst
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 Universit?T T?Bingen, Ngadhnjim Latifi, Laura Sophia Pfeiffer, Thorsten Stafforst filed Critical Eberhard Karls Universit?T T?Bingen
Publication of IL312762A publication Critical patent/IL312762A/en

Links

Classifications

    • 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

Landscapes

  • 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)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Cardiology (AREA)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
IL312762A 2021-11-30 2022-11-29 ANTISENSE OLIGONUCLEOTIDES (ASO) for efficient and accurate RNA editing with an endogenous adaminase that acts on RNA (ADAR) IL312762A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
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
IL312762A true IL312762A (en) 2024-07-01

Family

ID=78820190

Family Applications (1)

Application Number Title Priority Date Filing Date
IL312762A IL312762A (en) 2021-11-30 2022-11-29 ANTISENSE OLIGONUCLEOTIDES (ASO) for efficient and accurate RNA editing with an endogenous adaminase that acts on RNA (ADAR)

Country Status (9)

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

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
CN121794378A (zh) * 2023-08-15 2026-04-03 艾尔纳公司 用于rna编辑的化学修饰的反义寡核苷酸（aso）及组合物
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
CN110352244B (zh) * 2016-09-01 2023-03-21 ProQR治疗上市公司Ⅱ 化学修饰的编辑rna的单链寡核苷酸
JP7347830B2 (ja) 2018-06-29 2023-09-20 エバーハルト カール ウニヴェルジテート テュービンゲン Ｒｎａ編集のための人工核酸
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
WO2023099494A1 (en) 2023-06-08
CA3238033A1 (en) 2023-06-08
JP2024544067A (ja) 2024-11-27
WO2023099494A9 (en) 2024-10-17
EP4441216A1 (en) 2024-10-09
KR20240111752A (ko) 2024-07-17
AU2022399905A1 (en) 2024-05-23
US20230235329A1 (en) 2023-07-27
CN118355119A (zh) 2024-07-16

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