AU2018345683B2 - Modified Cpf1 guide RNA - Google Patents

Modified Cpf1 guide RNA Download PDF

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AU2018345683B2
AU2018345683B2 AU2018345683A AU2018345683A AU2018345683B2 AU 2018345683 B2 AU2018345683 B2 AU 2018345683B2 AU 2018345683 A AU2018345683 A AU 2018345683A AU 2018345683 A AU2018345683 A AU 2018345683A AU 2018345683 B2 AU2018345683 B2 AU 2018345683B2
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Australia
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sequence
nucleic acid
crrna
cpfl
extension
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AU2018345683A
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AU2018345683A1 (en
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Kunwoo Lee
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Breeze Bio Inc
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GenEdit Inc
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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
    • 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
    • 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
    • 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
    • C12N15/88—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3519—Fusion with another nucleic acid
    • 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

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  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
AU2018345683A 2017-10-02 2018-10-02 Modified Cpf1 guide RNA Active AU2018345683B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2025202594A AU2025202594A1 (en) 2017-10-02 2025-04-11 Modified Cpf1 guide RNA

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201762567123P 2017-10-02 2017-10-02
US62/567,123 2017-10-02
US201862617138P 2018-01-12 2018-01-12
US62/617,138 2018-01-12
US201862697327P 2018-07-12 2018-07-12
US62/697,327 2018-07-12
PCT/US2018/054027 WO2019070762A1 (en) 2017-10-02 2018-10-02 RNA GUIDE CPF1 MODIFIED

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2025202594A Division AU2025202594A1 (en) 2017-10-02 2025-04-11 Modified Cpf1 guide RNA

Publications (2)

Publication Number Publication Date
AU2018345683A1 AU2018345683A1 (en) 2020-04-23
AU2018345683B2 true AU2018345683B2 (en) 2025-05-08

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AU2018345683A Active AU2018345683B2 (en) 2017-10-02 2018-10-02 Modified Cpf1 guide RNA
AU2025202594A Pending AU2025202594A1 (en) 2017-10-02 2025-04-11 Modified Cpf1 guide RNA

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AU2025202594A Pending AU2025202594A1 (en) 2017-10-02 2025-04-11 Modified Cpf1 guide RNA

Country Status (12)

Country Link
US (1) US12606823B2 (https=)
EP (1) EP3692154A1 (https=)
JP (2) JP2020537540A (https=)
KR (2) KR102907044B1 (https=)
CN (2) CN120290559A (https=)
AU (2) AU2018345683B2 (https=)
BR (1) BR112020006601A2 (https=)
CA (1) CA3077189A1 (https=)
IL (2) IL273595B2 (https=)
MX (1) MX2020003339A (https=)
SG (1) SG11202003083PA (https=)
WO (1) WO2019070762A1 (https=)

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GB201616590D0 (en) 2016-09-29 2016-11-16 Oxford Nanopore Technologies Limited Method
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
CN120290559A (zh) 2017-10-02 2025-07-11 基因编辑有限公司 修饰的cpf1引导rna
US11268092B2 (en) 2018-01-12 2022-03-08 GenEdit, Inc. Structure-engineered guide RNA
WO2019178426A1 (en) * 2018-03-14 2019-09-19 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
GB201905651D0 (en) * 2019-04-24 2019-06-05 Lightbio Ltd Nucleic acid constructs and methods for their manufacture
GB201913997D0 (en) * 2019-09-27 2019-11-13 Oxford Nanopore Tech Ltd Method
EP4076479A4 (en) * 2019-12-18 2024-05-22 Editas Medicine, Inc. GENETICALLY MODIFIED CELLS FOR THERAPY
CN113234701B (zh) * 2020-10-20 2022-08-16 珠海舒桐医疗科技有限公司 一种Cpf1蛋白及基因编辑系统
CN116848240A (zh) * 2020-12-07 2023-10-03 因思科瑞普特公司 核酸引导的切口酶编辑中的gRNA稳定
IL316048A (en) * 2022-04-13 2024-11-01 Caribou Biosciences Inc Therapeutic applications of V-type CRISPR systems
US20240167029A1 (en) * 2022-06-27 2024-05-23 University Of Massachusetts Modified guide rnas for crispr genome editing
IL324634A (en) 2023-05-15 2026-01-01 Nchroma Bio Inc Compositions and methods for epigenetic regulation of hbv gene expression
WO2025212120A1 (en) * 2024-04-05 2025-10-09 Arbor Biotechnologies, Inc. Chemical modifications of guide rnas for crispr nucleases

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WO2017070598A1 (en) * 2015-10-23 2017-04-27 Caribou Biosciences, Inc. Engineered crispr class 2 cross-type nucleic-acid targeting nucleic acids
WO2018204493A1 (en) * 2017-05-04 2018-11-08 The Trustees Of The University Of Pennsylvania Compositions and methods for gene editing in t cells using crispr/cpf1

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Also Published As

Publication number Publication date
EP3692154A1 (en) 2020-08-12
AU2018345683A1 (en) 2020-04-23
US12606823B2 (en) 2026-04-21
US20200299689A1 (en) 2020-09-24
IL273595B2 (en) 2026-02-01
CN111770994B (zh) 2025-03-18
IL273595B1 (en) 2025-10-01
MX2020003339A (es) 2020-11-06
CA3077189A1 (en) 2019-04-11
CN120290559A (zh) 2025-07-11
SG11202003083PA (en) 2020-05-28
JP2020537540A (ja) 2020-12-24
WO2019070762A1 (en) 2019-04-11
IL273595A (en) 2020-05-31
BR112020006601A2 (pt) 2020-12-08
KR102907044B1 (ko) 2026-01-02
KR20260011199A (ko) 2026-01-22
AU2025202594A1 (en) 2025-05-22
IL323269A (en) 2025-11-01
KR20200106485A (ko) 2020-09-14
CN111770994A (zh) 2020-10-13
AU2025202594A9 (en) 2025-09-04
JP2024116119A (ja) 2024-08-27

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