AU2022237663A1 - Multiplex editing with cas enzymes - Google Patents

Multiplex editing with cas enzymes Download PDF

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Publication number
AU2022237663A1
AU2022237663A1 AU2022237663A AU2022237663A AU2022237663A1 AU 2022237663 A1 AU2022237663 A1 AU 2022237663A1 AU 2022237663 A AU2022237663 A AU 2022237663A AU 2022237663 A AU2022237663 A AU 2022237663A AU 2022237663 A1 AU2022237663 A1 AU 2022237663A1
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Australia
Prior art keywords
sequence
cell
target
locus
rna
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Pending
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AU2022237663A
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AU2022237663A9 (en
Inventor
Lisa ALEXANDER
Christopher Brown
Cristina Butterfield
Greg COST
Audra DEVOTO
Diego Espinosa
Daniela S. A. GOLTSMAN
Rebecca LAMOTHE
Brian Thomas
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Metagenomi Therapeutics Inc
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Metagenomi Inc
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Publication of AU2022237663A1 publication Critical patent/AU2022237663A1/en
Publication of AU2022237663A9 publication Critical patent/AU2022237663A9/en
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    • 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
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A61K40/11—T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/30—Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/31—Chimeric antigen receptors [CAR]
    • C—CHEMISTRY; METALLURGY
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/72—Receptors; Cell surface antigens; Cell surface determinants for hormones
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    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
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    • 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
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
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    • 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]
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Cell Biology (AREA)
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  • Gastroenterology & Hepatology (AREA)
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  • Crystallography & Structural Chemistry (AREA)
  • Endocrinology (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Immobilizing And Processing Of Enzymes And Microorganisms (AREA)
  • Investigating Or Analysing Biological Materials (AREA)
  • Graft Or Block Polymers (AREA)
AU2022237663A 2021-03-19 2022-03-18 Multiplex editing with cas enzymes Pending AU2022237663A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163163510P 2021-03-19 2021-03-19
US63/163,510 2021-03-19
US202163186506P 2021-05-10 2021-05-10
US63/186,506 2021-05-10
US202163241916P 2021-09-08 2021-09-08
US63/241,916 2021-09-08
PCT/US2022/021004 WO2022198080A1 (en) 2021-03-19 2022-03-18 Multiplex editing with cas enzymes

Publications (2)

Publication Number Publication Date
AU2022237663A1 true AU2022237663A1 (en) 2023-10-12
AU2022237663A9 AU2022237663A9 (en) 2023-10-26

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AU2022237663A Pending AU2022237663A1 (en) 2021-03-19 2022-03-18 Multiplex editing with cas enzymes

Country Status (9)

Country Link
US (1) US20250075233A1 (https=)
EP (1) EP4308699A4 (https=)
JP (1) JP2024515936A (https=)
KR (1) KR20230157387A (https=)
AU (1) AU2022237663A1 (https=)
BR (1) BR112023018948A2 (https=)
CA (1) CA3210361A1 (https=)
MX (1) MX2023010969A (https=)
WO (1) WO2022198080A1 (https=)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2021178933A2 (en) 2020-03-06 2021-09-10 Metagenomi Ip Technologies, Llc Class ii, type v crispr systems
WO2021226363A1 (en) * 2020-05-08 2021-11-11 Metagenomi Ip Technologies, Llc Enzymes with ruvc domains
CA3205865A1 (en) * 2021-01-22 2022-07-28 Brian C. Thomas Novel engineered and chimeric nucleases
AU2022335499A1 (en) 2021-08-27 2024-02-22 Metagenomi Therapeutics, Inc. Enzymes with ruvc domains
AU2022396533A1 (en) 2021-11-24 2024-05-02 Metagenomi Therapeutics, Inc. Endonuclease systems
WO2024127370A1 (en) * 2022-12-16 2024-06-20 LifeEDIT Therapeutics, Inc. Guide rnas that target trac gene and methods of use

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003099215A2 (en) * 2002-05-20 2003-12-04 Pharmacia Corporation Antisense modulation of glucocorticoid receptor expression
US20150017136A1 (en) * 2013-07-15 2015-01-15 Cellectis Methods for engineering allogeneic and highly active t cell for immunotherapy
CA2985650A1 (en) * 2015-05-13 2016-11-17 Seattle Children's Hospital (dba Seattle Children's Research Institute) Enhancing endonuclease based gene editing in primary cells
EP3423580A1 (en) * 2016-03-04 2019-01-09 Editas Medicine, Inc. Crispr-cpf1-related methods, compositions and components for cancer immunotherapy
MX2019013514A (es) * 2017-05-12 2020-01-20 Crispr Therapeutics Ag Materiales y metodos para modificar celulas por ingenieria genetica y usos de los mismos en inmunooncologia.
EP3686275A4 (en) * 2017-09-18 2021-09-29 Edigene Inc. GENE EDITING LYMPHOCYTE T AND RELATED USE
EP3746554A1 (en) * 2018-01-30 2020-12-09 Editas Medicine, Inc. Systems and methods for modulating chromosomal rearrangements
US12275951B2 (en) * 2018-02-06 2025-04-15 Ohio State Innovation Foundation Engineered guide RNA and uses thereof
CA3089587A1 (en) * 2018-02-08 2019-08-15 Sangamo Therapeutics, Inc. Engineered target specific nucleases
CA3241703C (en) * 2019-02-14 2026-02-24 Metagenomi Therapeutics, Inc. Enzymes with ruvc domains
US10913941B2 (en) * 2019-02-14 2021-02-09 Metagenomi Ip Technologies, Llc Enzymes with RuvC domains
CN113825834A (zh) * 2019-03-11 2021-12-21 索伦托药业有限公司 使用rna引导的核酸内切酶进行dna构建体整合的改进工艺
GB201907733D0 (en) * 2019-05-31 2019-07-17 Governing Council Of The Univ Of Toronto Methods and compositions for multiplex gene editing

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Publication number Publication date
WO2022198080A1 (en) 2022-09-22
BR112023018948A2 (pt) 2023-10-17
MX2023010969A (es) 2023-09-27
US20250075233A1 (en) 2025-03-06
EP4308699A1 (en) 2024-01-24
JP2024515936A (ja) 2024-04-11
KR20230157387A (ko) 2023-11-16
CA3210361A1 (en) 2022-09-22
AU2022237663A9 (en) 2023-10-26
EP4308699A4 (en) 2025-12-31

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