AU2020266587B2 - Novel OMNI-50 CRISPR nuclease - Google Patents

Novel OMNI-50 CRISPR nuclease Download PDF

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Publication number
AU2020266587B2
AU2020266587B2 AU2020266587A AU2020266587A AU2020266587B2 AU 2020266587 B2 AU2020266587 B2 AU 2020266587B2 AU 2020266587 A AU2020266587 A AU 2020266587A AU 2020266587 A AU2020266587 A AU 2020266587A AU 2020266587 B2 AU2020266587 B2 AU 2020266587B2
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Australia
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molecule
sequence
seq
target
nuclease
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AU2020266587A
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AU2020266587A1 (en
Inventor
David Baram
Joseph GEORGESON
Asael Herman
Lior IZHAR
Nadav MARBACH-BAR
Nurit MERON
Liat ROCKAH
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Emendobio Inc
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Emendobio Inc
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • 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
    • 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]
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • 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
    • 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
    • C12N15/52—Genes encoding for enzymes or proenzymes
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/20—Fusion polypeptide containing a tag with affinity for a non-protein ligand
    • C07K2319/21—Fusion polypeptide containing a tag with affinity for a non-protein ligand containing a His-tag
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • C07K2319/42—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation containing a HA(hemagglutinin)-tag
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/50—Fusion polypeptide containing protease site
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/60—Fusion polypeptide containing spectroscopic/fluorescent detection, e.g. green fluorescent protein [GFP]
    • 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
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/11—Applications; Uses in screening processes for the determination of target sites, i.e. of active 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
    • 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)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2020266587A 2019-04-30 2020-04-30 Novel OMNI-50 CRISPR nuclease Active AU2020266587B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2024202171A AU2024202171A1 (en) 2019-04-30 2024-04-04 Novel OMNI-50 CRISPR nuclease

Applications Claiming Priority (11)

Application Number Priority Date Filing Date Title
US201962841046P 2019-04-30 2019-04-30
US62/841,046 2019-04-30
US201962897806P 2019-09-09 2019-09-09
US62/897,806 2019-09-09
US201962931630P 2019-11-06 2019-11-06
US62/931,630 2019-11-06
US202062959672P 2020-01-10 2020-01-10
US62/959,672 2020-01-10
US202062991285P 2020-03-18 2020-03-18
US62/991,285 2020-03-18
PCT/US2020/030782 WO2020223514A2 (en) 2019-04-30 2020-04-30 Novel omni-50 crispr nuclease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2024202171A Division AU2024202171A1 (en) 2019-04-30 2024-04-04 Novel OMNI-50 CRISPR nuclease

Publications (2)

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AU2020266587A1 AU2020266587A1 (en) 2021-12-16
AU2020266587B2 true AU2020266587B2 (en) 2024-01-04

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AU2020266587A Active AU2020266587B2 (en) 2019-04-30 2020-04-30 Novel OMNI-50 CRISPR nuclease
AU2024202171A Pending AU2024202171A1 (en) 2019-04-30 2024-04-04 Novel OMNI-50 CRISPR nuclease

Family Applications After (1)

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AU2024202171A Pending AU2024202171A1 (en) 2019-04-30 2024-04-04 Novel OMNI-50 CRISPR nuclease

Country Status (10)

Country Link
US (2) US11666641B2 (https=)
EP (1) EP3963061A4 (https=)
JP (2) JP2022531347A (https=)
KR (2) KR20250060327A (https=)
CN (1) CN114127274A (https=)
AU (2) AU2020266587B2 (https=)
BR (1) BR112021021912A2 (https=)
CA (1) CA3135759A1 (https=)
IL (1) IL287693A (https=)
WO (1) WO2020223514A2 (https=)

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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
US11946077B2 (en) 2020-06-04 2024-04-02 Emendobio Inc. OMNI-59, 61, 67, 76, 79, 80, 81, and 82 CRISPR nucleases
WO2022098693A1 (en) * 2020-11-04 2022-05-12 Emendobio Inc. Novel omni-50 crispr nuclease-rna complexes
US20240175013A1 (en) * 2020-12-14 2024-05-30 Emendobio Inc. Biallelic knockout of trac
WO2022132765A1 (en) * 2020-12-14 2022-06-23 Emendobio Inc. Biallelic knockout of b2m
US12091688B2 (en) 2021-02-08 2024-09-17 Emendobio Inc. OMNI-103 CRISPR nuclease
KR20230142740A (ko) 2021-02-08 2023-10-11 에멘도바이오 인코포레이티드 Omni-103 crispr 뉴클레아제
IL307855A (en) * 2021-04-22 2023-12-01 Emendobio Inc OMNI CRISPR Nucleases 117, 140, 150-158, 160-165, 167-177, 180-188, 191-198, 200, 201, 203, 205-209, 211-217, 219, 220, 222, 223, 226 , 227, 229, 231-236, 238-245, 247, 250, 254, 256, 257, 260 and 262 news
IL310730A (en) * 2021-08-12 2024-04-01 Emendobio Inc Engineered high fidelity omni-50 nuclease variants
JP2024540558A (ja) * 2021-11-19 2024-10-31 エメンドバイオ・インコーポレイテッド 新規なｏｍｎｉ ｃｒｉｓｐｒヌクレアーゼ
US20250043260A1 (en) * 2021-12-01 2025-02-06 Emendobio Inc. Engineered High Activity Omni-79 Nuclease Variants
US20260021141A1 (en) * 2022-07-15 2026-01-22 Emendobio Inc. Strategies For Knock-Ins At APLP2 Safe Harbor Sites
WO2024042165A2 (en) * 2022-08-26 2024-02-29 UCB Biopharma SRL Novel rna-guided nucleases and nucleic acid targeting systems comprising such rna-guided nucleases
WO2025043140A1 (en) * 2023-08-23 2025-02-27 Emendobio Inc. Methods and compositions for modifying expression of a mutant transforming growth factor beta induced (tgfbi) allele

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EP2334794B8 (en) 2008-09-15 2017-04-19 The Children's Medical Center Corporation Modulation of bcl11a for treatment of hemoglobinopathies
EP3241902B1 (en) 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
WO2015134812A1 (en) * 2014-03-05 2015-09-11 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating usher syndrome and retinitis pigmentosa
WO2017024047A1 (en) 2015-08-03 2017-02-09 Emendobio Inc. Compositions and methods for increasing nuclease induced recombination rate in cells
ES2942309T3 (es) 2015-11-04 2023-05-31 Vertex Pharma Materiales y métodos para el tratamiento de hemoglobinopatías
CN105567735A (zh) * 2016-01-05 2016-05-11 华东师范大学 一种凝血因子基因突变的定点修复载体系统及方法

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
DATABASE GenPept [online] 10 November 2017 (2017-11-10), "type II CRISPR RNA-guided endonuclease Cas9 [Ezakiella peruensis]", XP093010135, retrieved from NCBI accession no. RefSeq:WP_099951290 Database accession no. WP_099951290 *

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Publication number Publication date
KR20250060327A (ko) 2025-05-07
BR112021021912A2 (pt) 2022-01-18
AU2020266587A1 (en) 2021-12-16
IL287693A (en) 2021-12-01
EP3963061A2 (en) 2022-03-09
KR20220012245A (ko) 2022-02-03
US20240131121A1 (en) 2024-04-25
KR102802134B1 (ko) 2025-05-07
JP2022531347A (ja) 2022-07-06
EP3963061A4 (en) 2023-02-08
US11666641B2 (en) 2023-06-06
US20220202913A1 (en) 2022-06-30
JP2024050582A (ja) 2024-04-10
AU2024202171A1 (en) 2024-06-27
WO2020223514A2 (en) 2020-11-05
WO2020223514A3 (en) 2020-12-24
US20240226246A9 (en) 2024-07-11
CN114127274A (zh) 2022-03-01
CA3135759A1 (en) 2020-11-05

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