CA3178670A1 - Programmable nucleases and methods of use - Google Patents

Programmable nucleases and methods of use Download PDF

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Publication number
CA3178670A1
CA3178670A1 CA3178670A CA3178670A CA3178670A1 CA 3178670 A1 CA3178670 A1 CA 3178670A1 CA 3178670 A CA3178670 A CA 3178670A CA 3178670 A CA3178670 A CA 3178670A CA 3178670 A1 CA3178670 A1 CA 3178670A1
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CA
Canada
Prior art keywords
nuclease
programmable
nucleic acid
region
sequence
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Pending
Application number
CA3178670A
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English (en)
French (fr)
Inventor
Lucas Benjamin HARRINGTON
William Douglass WRIGHT
Pei-Qi Liu
Benjamin Julius RAUCH
Wiputra Jaya HARTONO
Bridget Ann Paine MCKAY
Danuta Sastre PHIPPS
Yuxuan Zheng
Nerea SANVISENS
Sean Chen
David PAEZ-ESPINO
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.)
Mammoth Biosciences Inc
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Mammoth Biosciences Inc
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Filing date
Publication date
Application filed by Mammoth Biosciences Inc filed Critical Mammoth Biosciences Inc
Publication of CA3178670A1 publication Critical patent/CA3178670A1/en
Pending legal-status Critical Current

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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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
    • C12N15/52—Genes encoding for enzymes or proenzymes
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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
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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
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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
    • C12N15/63—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
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
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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
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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]
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6897—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids involving reporter genes operably linked to promoters
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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
    • C12N2310/30—Chemical structure
    • C—CHEMISTRY; METALLURGY
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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
    • 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)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
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  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Cell Biology (AREA)
  • Analytical Chemistry (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
CA3178670A 2020-06-03 2021-06-03 Programmable nucleases and methods of use Pending CA3178670A1 (en)

Applications Claiming Priority (17)

Application Number Priority Date Filing Date Title
US202063034346P 2020-06-03 2020-06-03
US63/034,346 2020-06-03
US202063037535P 2020-06-10 2020-06-10
US63/037,535 2020-06-10
US202063040998P 2020-06-18 2020-06-18
US63/040,998 2020-06-18
US202063092481P 2020-10-15 2020-10-15
US63/092,481 2020-10-15
US202063116083P 2020-11-19 2020-11-19
US63/116,083 2020-11-19
US202063124676P 2020-12-11 2020-12-11
US63/124,676 2020-12-11
US202163156883P 2021-03-04 2021-03-04
US63/156,883 2021-03-04
US202163178472P 2021-04-22 2021-04-22
US63/178,472 2021-04-22
PCT/US2021/035781 WO2021247924A1 (en) 2020-06-03 2021-06-03 Programmable nucleases and methods of use

Publications (1)

Publication Number Publication Date
CA3178670A1 true CA3178670A1 (en) 2021-12-09

Family

ID=78829953

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3178670A Pending CA3178670A1 (en) 2020-06-03 2021-06-03 Programmable nucleases and methods of use

Country Status (7)

Country Link
US (3) US20230357735A1 (https=)
EP (1) EP4162052A4 (https=)
JP (1) JP2023529151A (https=)
CN (1) CN116209755A (https=)
AU (1) AU2021282578A1 (https=)
CA (1) CA3178670A1 (https=)
WO (1) WO2021247924A1 (https=)

Families Citing this family (22)

* Cited by examiner, † Cited by third party
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AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
EP3830301B1 (en) 2018-08-01 2024-05-22 Mammoth Biosciences, Inc. Programmable nuclease compositions and methods of use thereof
US20240026351A1 (en) * 2020-10-30 2024-01-25 Arbor Biotechnologies, Inc. Compositions comprising an rna guide targeting trac and uses thereof
WO2023173110A1 (en) * 2022-03-11 2023-09-14 Epicrispr Biotechnologies, Inc. Compositions, systems, and methods for treating familial hypercholesterolemia by targeting pcsk9
WO2023220654A2 (en) * 2022-05-10 2023-11-16 Mammoth Biosciences, Inc. Effector protein compositions and methods of use thereof
WO2023220649A2 (en) * 2022-05-10 2023-11-16 Mammoth Biosciences, Inc. Effector protein compositions and methods of use thereof
IL317874A (en) 2022-06-24 2025-02-01 Tune Therapeutics Inc Compounds, systems and methods for reducing low density lipoproteins through targeted gene suppression
WO2024047587A1 (en) * 2022-08-31 2024-03-07 Regel Therapeutics, Inc. Cas-phi compositions and methods of use
WO2024073114A1 (en) * 2022-09-30 2024-04-04 Vedabio, Inc. Delivery of therapeutics in vivo via a crispr-based cascade system
WO2024091958A1 (en) * 2022-10-25 2024-05-02 Mammoth Biosciences, Inc. Effector proteins, compositions, systems and methods for the modification of serpina1
WO2024107665A1 (en) * 2022-11-14 2024-05-23 Mammoth Biosciences, Inc. Effector proteins, compositions, systems and methods of use thereof
WO2024127370A1 (en) * 2022-12-16 2024-06-20 LifeEDIT Therapeutics, Inc. Guide rnas that target trac gene and methods of use
EP4637841A1 (en) * 2022-12-22 2025-10-29 Mammoth Biosciences, Inc. Compositions and methods for modifying dux4
EP4673550A2 (en) * 2023-02-27 2026-01-07 Mammoth Biosciences, Inc. Compositions and methods for the modification and regulation of liver gene expression
AU2024236558A1 (en) 2023-03-15 2025-10-09 Renagade Therapeutics Management Inc. Delivery of gene editing systems and methods of use thereof
WO2024238321A1 (en) * 2023-05-12 2024-11-21 Mammoth Biosciences, Inc. Compositions and methods for targeted epigenetic modification
CN116844639B (zh) * 2023-06-14 2025-09-12 上海诚明融鑫科技有限公司 一种具有自适应学习控制能力的人类mtDNA快速比对方法及装置
AU2024335327A1 (en) 2023-09-01 2026-03-26 Renagade Therapeutics Management Inc. Gene editing systems, compositions, and methods for treatment of vexas syndrome
WO2025050015A1 (en) * 2023-09-01 2025-03-06 Script Biosciences, Inc. Compositions and methods for targeted modification of msh3
WO2025174765A1 (en) 2024-02-12 2025-08-21 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
CN119351374B (zh) * 2024-12-23 2025-03-25 内蒙古大学 工程化改造超紧凑型CRISPR/Cas12j-8及其应用
CN120330238B (zh) * 2025-04-10 2025-12-23 北京林业大学 一种ect2基因在调控杨树次生木质部发育中的应用

Family Cites Families (9)

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US20050108791A1 (en) * 2001-12-04 2005-05-19 Edgerton Michael D. Transgenic plants with improved phenotypes
US20110275135A1 (en) * 2010-05-05 2011-11-10 Battelle Energy Alliance, Llc Genetic elements, proteins, and associated methods including application of additional genetic information to gram (+) thermoacidophiles
WO2012164565A1 (en) * 2011-06-01 2012-12-06 Yeda Research And Development Co. Ltd. Compositions and methods for downregulating prokaryotic genes
WO2015161276A2 (en) * 2014-04-18 2015-10-22 Editas Medicine, Inc. Crispr-cas-related methods, compositions and components for cancer immunotherapy
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
AU2016380351B2 (en) * 2015-12-29 2023-04-06 Monsanto Technology Llc Novel CRISPR-associated transposases and uses thereof
EP3445853A1 (en) * 2016-04-19 2019-02-27 The Broad Institute, Inc. Cpf1 complexes with reduced indel activity
EP3830301B1 (en) * 2018-08-01 2024-05-22 Mammoth Biosciences, Inc. Programmable nuclease compositions and methods of use thereof
MX2021010559A (es) * 2019-03-07 2021-12-15 Univ California Polipéptidos efectores de crispr-cas y métodos de uso de estos.

Also Published As

Publication number Publication date
WO2021247924A1 (en) 2021-12-09
AU2021282578A1 (en) 2023-02-02
CN116209755A (zh) 2023-06-02
EP4162052A4 (en) 2024-07-10
US20230167454A1 (en) 2023-06-01
EP4162052A1 (en) 2023-04-12
US20230357735A1 (en) 2023-11-09
JP2023529151A (ja) 2023-07-07
US20250066797A1 (en) 2025-02-27

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