SG11202012499RA - Compositions and methods for genomic editing by insertion of donor polynucleotides - Google Patents

Compositions and methods for genomic editing by insertion of donor polynucleotides

Info

Publication number
SG11202012499RA
SG11202012499RA SG11202012499RA SG11202012499RA SG11202012499RA SG 11202012499R A SG11202012499R A SG 11202012499RA SG 11202012499R A SG11202012499R A SG 11202012499RA SG 11202012499R A SG11202012499R A SG 11202012499RA SG 11202012499R A SG11202012499R A SG 11202012499RA
Authority
SG
Singapore
Prior art keywords
compositions
methods
genomic editing
donor polynucleotides
polynucleotides
Prior art date
Application number
SG11202012499RA
Other languages
English (en)
Inventor
Troy Carlo
Roman Bogorad
Original Assignee
Crispr Therapeutics Ag
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 Crispr Therapeutics Ag filed Critical Crispr Therapeutics Ag
Publication of SG11202012499RA publication Critical patent/SG11202012499RA/en

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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
    • C12N15/111—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
    • 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
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered 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
    • 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
    • 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
    • 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 RNAses, DNAses
    • 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
    • C12N2310/18—Type of nucleic acid acting by a non-sequence specific mechanism
    • 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 [CRISPRs]
    • 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
    • C12N2320/33—Alteration of splicing
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Apparatus Associated With Microorganisms And Enzymes (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
SG11202012499RA 2018-06-28 2019-06-28 Compositions and methods for genomic editing by insertion of donor polynucleotides SG11202012499RA (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201862691573P 2018-06-28 2018-06-28
PCT/IB2019/000833 WO2020003006A2 (fr) 2018-06-28 2019-06-28 Compositions et procédés d'édition génomique par insertion de polynucléotides donneurs

Publications (1)

Publication Number Publication Date
SG11202012499RA true SG11202012499RA (en) 2021-01-28

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202012499RA SG11202012499RA (en) 2018-06-28 2019-06-28 Compositions and methods for genomic editing by insertion of donor polynucleotides

Country Status (12)

Country Link
US (3) US11827877B2 (fr)
EP (1) EP3814499A2 (fr)
JP (1) JP7550057B2 (fr)
KR (1) KR20210027389A (fr)
CN (1) CN112585268A (fr)
AU (1) AU2019293286A1 (fr)
BR (1) BR112020025824A2 (fr)
CA (1) CA3104028A1 (fr)
IL (1) IL279718A (fr)
MX (1) MX2020014248A (fr)
SG (1) SG11202012499RA (fr)
WO (1) WO2020003006A2 (fr)

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* Cited by examiner, † Cited by third party
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CN109328231A (zh) 2015-11-06 2019-02-12 克里斯普治疗股份公司 用于治疗1a型糖原贮积病的材料和方法
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
SG11202012499RA (en) 2018-06-28 2021-01-28 Crispr Therapeutics Ag Compositions and methods for genomic editing by insertion of donor polynucleotides
BR112021007229A2 (pt) * 2018-10-16 2021-08-10 Blueallele, Llc métodos para inserção dirigida de dna em genes
EP4297802A1 (fr) * 2021-04-01 2024-01-03 Duke University Compositions pour édition et procédés d'édition du génome
CN113403309B (zh) * 2021-05-24 2022-08-16 珠海舒桐医疗科技有限公司 非同源双链寡聚核苷酸片段在基因敲除系统中的应用
CN113416730B (zh) * 2021-07-07 2023-04-04 中国医学科学院血液病医院(中国医学科学院血液学研究所) 一种降低细胞因基因编辑产生的大片段删除突变的方法
WO2023201276A2 (fr) * 2022-04-12 2023-10-19 Orthobio Therapeutics, Inc. Nanoparticules lipidiques pour systèmes d'édition de gènes
CN116334000A (zh) * 2022-05-31 2023-06-27 南京艾尔普再生医学科技有限公司 一种低免疫原性iPSC细胞的制备方法及低免疫原性iPSC细胞
CN117953969B (zh) * 2023-12-18 2024-08-27 广州凯普医学检验所有限公司 一种线粒体疾病预测方法及线粒体疾病预测系统

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CN109328231A (zh) * 2015-11-06 2019-02-12 克里斯普治疗股份公司 用于治疗1a型糖原贮积病的材料和方法
WO2017093804A2 (fr) 2015-12-01 2017-06-08 Crispr Therapeutics Ag Matériaux et méthodes de traitement d'une déficience en antitrypsine alpha-1
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WO2019239361A1 (fr) * 2018-06-14 2019-12-19 Novartis Ag Procédé d'insertion de séquence à l'aide de crispr
SG11202012499RA (en) 2018-06-28 2021-01-28 Crispr Therapeutics Ag Compositions and methods for genomic editing by insertion of donor polynucleotides

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JP7550057B2 (ja) 2024-09-12
US20240167060A1 (en) 2024-05-23
EP3814499A2 (fr) 2021-05-05
US11332760B2 (en) 2022-05-17
US11827877B2 (en) 2023-11-28
CA3104028A1 (fr) 2020-01-02
KR20210027389A (ko) 2021-03-10
WO2020003006A2 (fr) 2020-01-02
AU2019293286A1 (en) 2021-01-07
BR112020025824A2 (pt) 2021-03-23
MX2020014248A (es) 2021-03-09
US20200040362A1 (en) 2020-02-06
JP2021529518A (ja) 2021-11-04
WO2020003006A3 (fr) 2020-03-05
CN112585268A (zh) 2021-03-30
US20210171985A1 (en) 2021-06-10
IL279718A (en) 2021-03-01

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