CA3253017A1 - MODIFIED NUCLEASE, COMPOSITIONS AND THEIR METHODS OF USE - Google Patents

MODIFIED NUCLEASE, COMPOSITIONS AND THEIR METHODS OF USE

Info

Publication number
CA3253017A1
CA3253017A1 CA3253017A CA3253017A CA3253017A1 CA 3253017 A1 CA3253017 A1 CA 3253017A1 CA 3253017 A CA3253017 A CA 3253017A CA 3253017 A CA3253017 A CA 3253017A CA 3253017 A1 CA3253017 A1 CA 3253017A1
Authority
CA
Canada
Prior art keywords
amino acid
acid residues
sequence
seq
nucleotides
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.)
Pending
Application number
CA3253017A
Other languages
English (en)
French (fr)
Inventor
Xiao Yang
Lei S. Qi
Vincent Cutillas
Gabriella Alvarez
Tabitha Tcheau
Daniel Hart
Original Assignee
Epicrispr Biotechnologies Inc
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 Epicrispr Biotechnologies Inc filed Critical Epicrispr Biotechnologies Inc
Publication of CA3253017A1 publication Critical patent/CA3253017A1/en
Pending legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • C12N9/222Clustered regularly interspaced short palindromic repeats [CRISPR]-associated [CAS] enzymes
    • C12N9/226Class 2 CAS enzyme complex, e.g. single CAS protein
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CA3253017A 2022-03-01 2023-03-01 MODIFIED NUCLEASE, COMPOSITIONS AND THEIR METHODS OF USE Pending CA3253017A1 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202263315159P 2022-03-01 2022-03-01
US63/315,159 2022-03-01
US202263380178P 2022-10-19 2022-10-19
US63/380,178 2022-10-19
US202263385171P 2022-11-28 2022-11-28
US63/385,171 2022-11-28
PCT/US2023/063446 WO2023168242A1 (en) 2022-03-01 2023-03-01 Engineered nucleases, compositions, and methods of use thereof

Publications (1)

Publication Number Publication Date
CA3253017A1 true CA3253017A1 (en) 2023-09-07

Family

ID=87884328

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3253017A Pending CA3253017A1 (en) 2022-03-01 2023-03-01 MODIFIED NUCLEASE, COMPOSITIONS AND THEIR METHODS OF USE

Country Status (8)

Country Link
US (2) US12054756B2 (https=)
EP (1) EP4486882A4 (https=)
JP (1) JP2025507822A (https=)
KR (1) KR20240152394A (https=)
CN (1) CN119137268A (https=)
AU (1) AU2023229364A1 (https=)
CA (1) CA3253017A1 (https=)
WO (1) WO2023168242A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3192654A1 (en) 2020-09-01 2022-03-10 The Board Of Trustees Of The Leland Stanford Junior Unversity Synthetic miniature crispr-cas (casmini) system for eukaryotic genome engineering
CN119137268A (zh) 2022-03-01 2024-12-13 埃皮克里斯皮尔生物技术公司 工程化核酸酶、组合物及其使用方法
WO2025231397A1 (en) * 2024-05-03 2025-11-06 Tenaya Therapeutics, Inc. Modified hek cell lines for improved aav production
CN118315068B (zh) * 2024-05-15 2025-08-08 西湖实验室(生命科学和生物医学浙江省实验室) 基于蛋白标志物预测卵巢癌患者化疗敏感性的装置、蛋白标志物分类器的构建方法和应用
WO2025264819A1 (en) * 2024-06-18 2025-12-26 Nchroma Bio, Inc. Compositions and methods for epigenetic regulation of znf410 expression

Family Cites Families (33)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2014093655A2 (en) 2012-12-12 2014-06-19 The Broad Institute, Inc. Engineering and optimization of systems, methods and compositions for sequence manipulation with functional domains
EP3080259B1 (en) 2013-12-12 2023-02-01 The Broad Institute, Inc. Engineering of systems, methods and optimized guide compositions with new architectures for sequence manipulation
US10648020B2 (en) 2015-06-18 2020-05-12 The Broad Institute, Inc. CRISPR enzymes and systems
WO2016205749A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
EP4485466A3 (en) 2016-08-17 2025-04-02 The Broad Institute Inc. Novel crispr enzymes and systems
US11970719B2 (en) * 2017-11-01 2024-04-30 The Regents Of The University Of California Class 2 CRISPR/Cas compositions and methods of use
AU2018358051B2 (en) 2017-11-01 2025-01-09 The Regents Of The University Of California CasZ compositions and methods of use
AU2018370588B2 (en) 2017-11-21 2022-09-08 Genkore Co. Ltd. Composition for Genome Editing Using Crispr/CPF1 System and Use Thereof
KR102929494B1 (ko) 2018-10-29 2026-02-20 차이나 어그리컬처럴 유니버시티 신규 CRISPR/Cas12f 효소 및 시스템
US12258594B2 (en) 2018-12-05 2025-03-25 The Broad Institute, Inc. Cas proteins with reduced immunogenicity and methods of screening thereof
WO2020123887A2 (en) * 2018-12-14 2020-06-18 Pioneer Hi-Bred International, Inc. Novel crispr-cas systems for genome editing
MX2021010559A (es) * 2019-03-07 2021-12-15 Univ California Polipéptidos efectores de crispr-cas y métodos de uso de estos.
WO2020223634A1 (en) 2019-05-01 2020-11-05 Mammoth Biosciences, Inc. Programmable nucleases and methods of use
EP3772329A1 (fr) * 2019-08-08 2021-02-10 PKvitality Systeme de surveillance corporelle comprenant une microaiguille
WO2021084533A1 (en) * 2019-10-28 2021-05-06 Targetgene Biotechnologies Ltd Pam-reduced and pam-abolished cas derivatives compositions and uses thereof in genetic modulation
CN114846146B (zh) 2019-10-29 2024-04-12 基恩科雷有限责任公司 用于增加CRISPR/Cas12f1系统的效率的工程化引导RNA及其用途
CA3192654A1 (en) * 2020-09-01 2022-03-10 The Board Of Trustees Of The Leland Stanford Junior Unversity Synthetic miniature crispr-cas (casmini) system for eukaryotic genome engineering
CN116568803A (zh) 2020-10-08 2023-08-08 基恩科雷有限责任公司 用于增加CRISPR/Cas12f1系统的效率的包含富含U的尾部的经工程化的引导RNA及其用途
CA3198435A1 (en) 2020-10-08 2022-04-14 Genkore Inc. Engineered guide rna for optimized crispr/cas12f1 (cas14a1) system and use thereof
EP4227411A4 (en) 2020-10-08 2025-07-09 Genkore Inc MODIFIED GUIDE RNA TO INCREASE THE EFFICIENCY OF THE CRISPR/CAS12F1 SYSTEM, AND ITS USE
EP4267252A4 (en) 2020-12-23 2025-07-23 Mammoth Biosciences Inc COMPOSITIONS AND METHODS OF USING PROGRAMMABLE NUCLEASES TO INDUCE CELL DEATH
WO2022150372A1 (en) 2021-01-05 2022-07-14 Horizon Discovery Ltd. Guide rna designs and complexes for type v cas systems
WO2022266324A1 (en) 2021-06-17 2022-12-22 Epicrispr Biotechnologies, Inc. Systems and methods for regulating aberrant gene expressions
KR20230007218A (ko) * 2021-07-05 2023-01-12 주식회사 진코어 초소형 염기교정 시스템 및 이의 용도
IL310116A (en) 2021-07-20 2024-03-01 Epicrispr Biotechnologies Inc Systems and methods for regulating target genes
US20240252684A1 (en) 2021-07-30 2024-08-01 Tune Therapeutics, Inc. Compositions and methods for modulating expression of methyl-cpg binding protein 2 (mecp2)
CN119137268A (zh) 2022-03-01 2024-12-13 埃皮克里斯皮尔生物技术公司 工程化核酸酶、组合物及其使用方法
WO2023172995A1 (en) 2022-03-11 2023-09-14 Epicrispr Biotechnologies, Inc. Systems and methods for genetic modulation to treat ocular diseases
WO2023173072A1 (en) 2022-03-11 2023-09-14 Epicrispr Biotechnologies, Inc. Systems and methods for genetic modulation to treat liver disease
EP4490295A4 (en) 2022-03-11 2026-03-18 Epicrispr Biotechnologies Inc GENETIC MODULATION SYSTEMS AND METHODS FOR TREATING EYE DISEASES
WO2023173110A1 (en) 2022-03-11 2023-09-14 Epicrispr Biotechnologies, Inc. Compositions, systems, and methods for treating familial hypercholesterolemia by targeting pcsk9
CA3253453A1 (en) 2022-03-24 2023-09-28 Epicrispr Biotechnologies, Inc. MODIFIED GENE EFFECTORS, COMPOSITIONS AND THEIR METHODS OF USE

Also Published As

Publication number Publication date
JP2025507822A (ja) 2025-03-21
WO2023168242A1 (en) 2023-09-07
EP4486882A4 (en) 2025-09-17
US20250011742A1 (en) 2025-01-09
AU2023229364A1 (en) 2024-09-19
US12054756B2 (en) 2024-08-06
EP4486882A1 (en) 2025-01-08
CN119137268A (zh) 2024-12-13
US20240035009A1 (en) 2024-02-01
KR20240152394A (ko) 2024-10-21

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