AU2020206997A1 - A non-toxic Cas9 enzyme and application thereof - Google Patents

A non-toxic Cas9 enzyme and application thereof Download PDF

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Publication number
AU2020206997A1
AU2020206997A1 AU2020206997A AU2020206997A AU2020206997A1 AU 2020206997 A1 AU2020206997 A1 AU 2020206997A1 AU 2020206997 A AU2020206997 A AU 2020206997A AU 2020206997 A AU2020206997 A AU 2020206997A AU 2020206997 A1 AU2020206997 A1 AU 2020206997A1
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AU
Australia
Prior art keywords
cells
cell
cas9
polynucleotide
polypeptide
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.)
Abandoned
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AU2020206997A
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English (en)
Inventor
Christopher HACKLEY
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.)
Crisp HR Therapeutics Inc
Original Assignee
Crisp HR Therapeutics Inc
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Filing date
Publication date
Application filed by Crisp HR Therapeutics Inc filed Critical Crisp HR Therapeutics Inc
Publication of AU2020206997A1 publication Critical patent/AU2020206997A1/en
Abandoned 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
AU2020206997A 2019-01-07 2020-01-06 A non-toxic Cas9 enzyme and application thereof Abandoned AU2020206997A1 (en)

Applications Claiming Priority (9)

Application Number Priority Date Filing Date Title
US201962789347P 2019-01-07 2019-01-07
US62/789,347 2019-01-07
US201962823477P 2019-03-25 2019-03-25
US62/823,477 2019-03-25
US201962824164P 2019-03-26 2019-03-26
US62/824,164 2019-03-26
US201962855612P 2019-05-31 2019-05-31
US62/855,612 2019-05-31
PCT/US2020/012438 WO2020146290A1 (en) 2019-01-07 2020-01-06 A non-toxic cas9 enzyme and application thereof

Publications (1)

Publication Number Publication Date
AU2020206997A1 true AU2020206997A1 (en) 2021-08-26

Family

ID=71520905

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020206997A Abandoned AU2020206997A1 (en) 2019-01-07 2020-01-06 A non-toxic Cas9 enzyme and application thereof

Country Status (8)

Country Link
US (3) US20210403922A1 (https=)
EP (1) EP3908659A4 (https=)
JP (1) JP2022516647A (https=)
CN (1) CN113811611A (https=)
AU (1) AU2020206997A1 (https=)
CA (1) CA3125009A1 (https=)
GB (2) GB2596660B (https=)
WO (1) WO2020146290A1 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3204496A1 (en) 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
EP4243608A1 (en) * 2020-11-11 2023-09-20 Leibniz-Institut für Pflanzenbiochemie (IPB) Fusion protein for editing endogenous dna of a eukaryotic cell
AU2021409732A1 (en) * 2020-12-23 2023-07-20 Intellia Therapeutics, Inc. Compositions and methods for reducing hla-a in a cell
EP4317549A4 (en) * 2021-03-31 2025-03-26 Ddbio. Co, Ltd., (Shang Hai) Vector and method for screening functional antigen-binding protein
CN113481184A (zh) * 2021-08-06 2021-10-08 北京大学 融合蛋白以及其使用方法
EP4396340A4 (en) * 2021-09-01 2025-09-24 Univ Leland Stanford Junior RNA-GUIDED GENOME RECOMBINATION AT THE KILOBASE SCALE
WO2023168235A2 (en) * 2022-03-01 2023-09-07 Crisp-Hr Therapeutics, Inc. Systems and methods of gene editing
CN116286741A (zh) * 2022-03-03 2023-06-23 清华大学 5’→3’核酸外切酶在基因编辑系统中的用途和基因编辑系统及其编辑方法
EP4286401A1 (en) * 2022-06-03 2023-12-06 Technische Universität Dresden Use of an artificial protein or of a nucleic acid encoding the artificial protein for providing a functional ribonucleoprotein complex
JP2025012122A (ja) * 2023-07-12 2025-01-24 国立研究開発法人農業・食品産業技術総合研究機構 遺伝子組換えチョウ目昆虫
WO2025137069A1 (en) * 2023-12-18 2025-06-26 University Of Maryland, Baltimore Compositions and methods for enhanced genome editing using cas9 fusion proteins

Family Cites Families (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4357457B1 (en) * 2012-10-23 2024-10-16 Toolgen Incorporated Composition for cleaving a target dna comprising a guide rna specific for the target dna and cas protein-encoding nucleic acid or cas protein, and use thereof
US9388430B2 (en) * 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US20150165054A1 (en) * 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
WO2016007604A1 (en) * 2014-07-09 2016-01-14 Gen9, Inc. Compositions and methods for site-directed dna nicking and cleaving
EP3204496A1 (en) * 2014-10-10 2017-08-16 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
CA2963820A1 (en) * 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
US10155938B2 (en) * 2015-04-14 2018-12-18 City Of Hope Coexpression of CAS9 and TREX2 for targeted mutagenesis
GB201510296D0 (en) * 2015-06-12 2015-07-29 Univ Wageningen Thermostable CAS9 nucleases
US20170175140A1 (en) * 2015-12-16 2017-06-22 Regents Of The University Of Minnesota Methods for using a 5'-exonuclease to increase homologous recombination in eukaryotic cells
US11274288B2 (en) * 2016-02-16 2022-03-15 Emendobio Inc. Compositions and methods for promoting homology directed repair mediated gene editing
US9982267B2 (en) * 2016-10-12 2018-05-29 Feldan Bio Inc. Rationally-designed synthetic peptide shuttle agents for delivering polypeptide cargos from an extracellular space to the cytosol and/or nucleus of a target eukaryotic cell, uses thereof, methods and kits relating to same
WO2018162702A1 (en) * 2017-03-10 2018-09-13 Institut National De La Sante Et De La Recherche Medicale (Inserm) Nuclease fusions for enhancing genome editing by homology-directed transgene integration

Also Published As

Publication number Publication date
EP3908659A4 (en) 2024-04-03
WO2020146290A1 (en) 2020-07-16
GB202307873D0 (en) 2023-07-12
US20210403922A1 (en) 2021-12-30
JP2022516647A (ja) 2022-03-01
US20240043851A1 (en) 2024-02-08
EP3908659A1 (en) 2021-11-17
GB2616539B (en) 2023-12-20
GB2596660B (en) 2023-09-13
GB202110538D0 (en) 2021-09-08
CN113811611A (zh) 2021-12-17
GB2596660A (en) 2022-01-05
US20240093207A1 (en) 2024-03-21
CA3125009A1 (en) 2020-07-16
GB2616539A (en) 2023-09-13

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MK4 Application lapsed section 142(2)(d) - no continuation fee paid for the application