SG11202003083PA - Modified cpf1 guide rna - Google Patents

Modified cpf1 guide rna

Info

Publication number
SG11202003083PA
SG11202003083PA SG11202003083PA SG11202003083PA SG11202003083PA SG 11202003083P A SG11202003083P A SG 11202003083PA SG 11202003083P A SG11202003083P A SG 11202003083PA SG 11202003083P A SG11202003083P A SG 11202003083PA SG 11202003083P A SG11202003083P A SG 11202003083PA
Authority
SG
Singapore
Prior art keywords
guide rna
cpf1 guide
modified cpf1
modified
rna
Prior art date
Application number
SG11202003083PA
Other languages
English (en)
Inventor
Kunwoo Lee
Original Assignee
Genedit 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 Genedit Inc filed Critical Genedit Inc
Publication of SG11202003083PA publication Critical patent/SG11202003083PA/en

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    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • 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
    • 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]
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid
    • 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
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • C12N2310/531Stem-loop; Hairpin

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Mycology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Enzymes And Modification Thereof (AREA)
SG11202003083PA 2017-10-02 2018-10-02 Modified cpf1 guide rna SG11202003083PA (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762567123P 2017-10-02 2017-10-02
US201862617138P 2018-01-12 2018-01-12
US201862697327P 2018-07-12 2018-07-12
PCT/US2018/054027 WO2019070762A1 (en) 2017-10-02 2018-10-02 RNA GUIDE CPF1 MODIFIED

Publications (1)

Publication Number Publication Date
SG11202003083PA true SG11202003083PA (en) 2020-05-28

Family

ID=64184173

Family Applications (1)

Application Number Title Priority Date Filing Date
SG11202003083PA SG11202003083PA (en) 2017-10-02 2018-10-02 Modified cpf1 guide rna

Country Status (12)

Country Link
US (1) US12606823B2 (https=)
EP (1) EP3692154A1 (https=)
JP (2) JP2020537540A (https=)
KR (2) KR102907044B1 (https=)
CN (2) CN120290559A (https=)
AU (2) AU2018345683B2 (https=)
BR (1) BR112020006601A2 (https=)
CA (1) CA3077189A1 (https=)
IL (2) IL273595B2 (https=)
MX (1) MX2020003339A (https=)
SG (1) SG11202003083PA (https=)
WO (1) WO2019070762A1 (https=)

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GB201616590D0 (en) 2016-09-29 2016-11-16 Oxford Nanopore Technologies Limited Method
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
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
CN120290559A (zh) 2017-10-02 2025-07-11 基因编辑有限公司 修饰的cpf1引导rna
US11268092B2 (en) 2018-01-12 2022-03-08 GenEdit, Inc. Structure-engineered guide RNA
WO2019178426A1 (en) * 2018-03-14 2019-09-19 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
GB201905651D0 (en) * 2019-04-24 2019-06-05 Lightbio Ltd Nucleic acid constructs and methods for their manufacture
GB201913997D0 (en) * 2019-09-27 2019-11-13 Oxford Nanopore Tech Ltd Method
EP4076479A4 (en) * 2019-12-18 2024-05-22 Editas Medicine, Inc. GENETICALLY MODIFIED CELLS FOR THERAPY
CN113234701B (zh) * 2020-10-20 2022-08-16 珠海舒桐医疗科技有限公司 一种Cpf1蛋白及基因编辑系统
CN116848240A (zh) * 2020-12-07 2023-10-03 因思科瑞普特公司 核酸引导的切口酶编辑中的gRNA稳定
IL316048A (en) * 2022-04-13 2024-11-01 Caribou Biosciences Inc Therapeutic applications of V-type CRISPR systems
US20240167029A1 (en) * 2022-06-27 2024-05-23 University Of Massachusetts Modified guide rnas for crispr genome editing
IL324634A (en) 2023-05-15 2026-01-01 Nchroma Bio Inc Compositions and methods for epigenetic regulation of hbv gene expression
WO2025212120A1 (en) * 2024-04-05 2025-10-09 Arbor Biotechnologies, Inc. Chemical modifications of guide rnas for crispr nucleases

Family Cites Families (26)

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US4863457A (en) 1986-11-24 1989-09-05 Lee David A Drug delivery device
US5378475A (en) 1991-02-21 1995-01-03 University Of Kentucky Research Foundation Sustained release drug delivery devices
US5443505A (en) 1993-11-15 1995-08-22 Oculex Pharmaceuticals, Inc. Biocompatible ocular implants
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
US20140349400A1 (en) 2013-03-15 2014-11-27 Massachusetts Institute Of Technology Programmable Modification of DNA
US10563225B2 (en) 2013-07-26 2020-02-18 President And Fellows Of Harvard College Genome engineering
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
WO2016022866A1 (en) 2014-08-07 2016-02-11 Agilent Technologies, Inc. Cis-blocked guide rna
EP3183358B1 (en) 2014-08-19 2020-10-07 President and Fellows of Harvard College Rna-guided systems for probing and mapping of nucleic acids
US20170306306A1 (en) 2014-10-24 2017-10-26 Life Technologies Corporation Compositions and Methods for Enhancing Homologous Recombination
US10900034B2 (en) 2014-12-03 2021-01-26 Agilent Technologies, Inc. Guide RNA with chemical modifications
EP3889260A1 (en) 2014-12-12 2021-10-06 The Broad Institute, Inc. Protected guide rnas (pgrnas)
AU2016261358B2 (en) 2015-05-11 2021-09-16 Editas Medicine, Inc. Optimized CRISPR/Cas9 systems and methods for gene editing in stem cells
WO2016183402A2 (en) 2015-05-13 2016-11-17 President And Fellows Of Harvard College Methods of making and using guide rna for use with cas9 systems
WO2017040511A1 (en) 2015-08-31 2017-03-09 Agilent Technologies, Inc. Compounds and methods for crispr/cas-based genome editing by homologous recombination
US20180237800A1 (en) 2015-09-21 2018-08-23 The Regents Of The University Of California Compositions and methods for target nucleic acid modification
US20190048340A1 (en) 2015-09-24 2019-02-14 Crispr Therapeutics Ag Novel family of rna-programmable endonucleases and their uses in genome editing and other applications
DK3350327T3 (en) * 2015-10-23 2019-01-21 Caribou Biosciences Inc CONSTRUCTED CRISPR CLASS-2-NUCLEIC ACID TARGETING-NUCLEIC ACID
WO2017099494A1 (ko) * 2015-12-08 2017-06-15 기초과학연구원 Cpf1을 포함하는 유전체 교정용 조성물 및 그 용도
EP3443088B1 (en) 2016-04-13 2024-09-18 Editas Medicine, Inc. Grna fusion molecules, gene editing systems, and methods of use thereof
JP7184648B2 (ja) 2016-04-29 2022-12-06 ビーエーエスエフ プラント サイエンス カンパニー ゲーエムベーハー 標的核酸の改変のための改善された方法
CN106244591A (zh) * 2016-08-23 2016-12-21 苏州吉玛基因股份有限公司 修饰crRNA在CRISPR/Cpf1基因编辑系统中的应用
JP2019517261A (ja) 2016-06-01 2019-06-24 カーヴェーエス ザート ソシエタス・ヨーロピアKws Saat Se ゲノム操作のためのハイブリッド核酸配列
KR20190089175A (ko) 2016-11-18 2019-07-30 진에딧 인코포레이티드 표적 핵산 변형을 위한 조성물 및 방법
CA3062165A1 (en) * 2017-05-04 2018-11-08 The Trustees Of The University Of Pennsylvania Compositions and methods for gene editing in t cells using crispr/cpf1
CN120290559A (zh) 2017-10-02 2025-07-11 基因编辑有限公司 修饰的cpf1引导rna

Also Published As

Publication number Publication date
EP3692154A1 (en) 2020-08-12
AU2018345683A1 (en) 2020-04-23
US12606823B2 (en) 2026-04-21
US20200299689A1 (en) 2020-09-24
IL273595B2 (en) 2026-02-01
CN111770994B (zh) 2025-03-18
IL273595B1 (en) 2025-10-01
AU2018345683B2 (en) 2025-05-08
MX2020003339A (es) 2020-11-06
CA3077189A1 (en) 2019-04-11
CN120290559A (zh) 2025-07-11
JP2020537540A (ja) 2020-12-24
WO2019070762A1 (en) 2019-04-11
IL273595A (en) 2020-05-31
BR112020006601A2 (pt) 2020-12-08
KR102907044B1 (ko) 2026-01-02
KR20260011199A (ko) 2026-01-22
AU2025202594A1 (en) 2025-05-22
IL323269A (en) 2025-11-01
KR20200106485A (ko) 2020-09-14
CN111770994A (zh) 2020-10-13
AU2025202594A9 (en) 2025-09-04
JP2024116119A (ja) 2024-08-27

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