AU2024260090A1 - Non-natural polynucleotides for modification of target nucleotide sequence - Google Patents

Non-natural polynucleotides for modification of target nucleotide sequence

Info

Publication number
AU2024260090A1
AU2024260090A1 AU2024260090A AU2024260090A AU2024260090A1 AU 2024260090 A1 AU2024260090 A1 AU 2024260090A1 AU 2024260090 A AU2024260090 A AU 2024260090A AU 2024260090 A AU2024260090 A AU 2024260090A AU 2024260090 A1 AU2024260090 A1 AU 2024260090A1
Authority
AU
Australia
Prior art keywords
nucleotide
naturally occurring
nucleotides
nucleic acid
occurring polynucleotide
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
AU2024260090A
Other languages
English (en)
Inventor
Keiko Hayami
Naoki Inoue
Takato NOUMI
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.)
Eurus Therapeutics Inc
Original Assignee
Eurus Therapeutics Inc
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 Eurus Therapeutics Inc filed Critical Eurus Therapeutics Inc
Publication of AU2024260090A1 publication Critical patent/AU2024260090A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • 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
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/3525—MOE, methoxyethoxy
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Saccharide Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
AU2024260090A 2023-04-26 2024-04-26 Non-natural polynucleotides for modification of target nucleotide sequence Pending AU2024260090A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2023-071927 2023-04-26
JP2023071927 2023-04-26
PCT/JP2024/016395 WO2024225425A1 (ja) 2023-04-26 2024-04-26 標的ヌクレオチド配列の改変のための非天然型ポリヌクレオチド

Publications (1)

Publication Number Publication Date
AU2024260090A1 true AU2024260090A1 (en) 2025-12-04

Family

ID=93256810

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2024260090A Pending AU2024260090A1 (en) 2023-04-26 2024-04-26 Non-natural polynucleotides for modification of target nucleotide sequence

Country Status (7)

Country Link
EP (1) EP4703472A1 (https=)
JP (2) JP7660960B2 (https=)
KR (1) KR20250168544A (https=)
CN (1) CN121443735A (https=)
AU (1) AU2024260090A1 (https=)
IL (1) IL324176A (https=)
WO (1) WO2024225425A1 (https=)

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2006020560A (ja) * 2004-07-07 2006-01-26 National Institute Of Agrobiological Sciences 核酸を配列特異的に標識する方法、およびそれを利用した新規核酸検出法
WO2007073149A1 (en) * 2005-12-22 2007-06-28 Keygene N.V. Alternative nucleotides for improved targeted nucleotide exchange
CN102016035A (zh) * 2006-11-02 2011-04-13 犹他大学研究基金会 可用于等位基因特异pcr的寡聚核苷酸
EP2167660A1 (en) * 2007-06-22 2010-03-31 Keygene N.V. Targeted nucleotide exchange with improved modified oligonucleotides
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
EP2970979B1 (en) * 2013-03-14 2018-11-21 Somalogic, Inc. Aptamers that bind to il-6 and their use in treating or diagnosing il-6 mediated conditions
EP3115457B1 (en) 2014-03-05 2019-10-02 National University Corporation Kobe University Genomic sequence modification method for specifically converting nucleic acid bases of targeted dna sequence, and molecular complex for use in same
EP4545649A3 (en) * 2016-11-11 2025-06-04 Bio-Rad Laboratories, Inc. Methods for processing nucleic acid samples
DK3636753T5 (da) 2017-06-08 2024-08-26 Univ Osaka Fremgangsmåde til fremstilling af DNA-redigeret eukaryotisk celle
EP4347818A2 (en) * 2021-06-01 2024-04-10 Arbor Biotechnologies, Inc. Gene editing systems comprising a crispr nuclease and uses thereof

Also Published As

Publication number Publication date
IL324176A (en) 2025-12-01
EP4703472A1 (en) 2026-03-04
WO2024225425A1 (ja) 2024-10-31
KR20250168544A (ko) 2025-12-02
JPWO2024225425A1 (https=) 2024-10-31
JP7660960B2 (ja) 2025-04-14
JP2025092562A (ja) 2025-06-19
CN121443735A (zh) 2026-01-30

Similar Documents

Publication Publication Date Title
US11560555B2 (en) Engineered proteins
US12577546B2 (en) Programmable DNA base editing by Nme2Cas9-deaminase fusion proteins
US20220033858A1 (en) Crispr oligoncleotides and gene editing
US20240102007A1 (en) Gene editing systems comprising a crispr nuclease and uses thereof
AU740702B2 (en) Cell-free chimeraplasty and eukaryotic use of heteroduplex mutational vectors
WO2018213351A1 (en) Thermostable rna-guided endonucleases and methods of use thereof
WO2017181107A2 (en) Modified cpf1 mrna, modified guide rna, and uses thereof
US12018297B2 (en) Nuclease-mediated nucleic acid modification
US20240218339A1 (en) Class ii, type v crispr systems
EP4230737A1 (en) Novel enhanced base editing or revising fusion protein and use thereof
US20230365992A1 (en) Novel enhanced base editing or revising fusion protein and use thereof
AU2024260090A1 (en) Non-natural polynucleotides for modification of target nucleotide sequence
WO2020111229A1 (ja) 溶液を用いた薬物送達系
CN117693585A (zh) Ii类v型crispr系统
US20240425850A1 (en) Noncanonical crRNA for Highly Efficient Genome Editing
WO2026094932A1 (ja) 標的ヌクレオチド配列の改変のための二本鎖ヌクレオチド複合体
WO2026050560A1 (en) Cas9 fusion proteins with internal nls: compositions and methods
WO2024081383A2 (en) Compositions and methods for targeting, editing, or modifying genes