CA3009637A1 - Engineered meganucleases with recognition sequences found in the human beta-2 microglobulin gene - Google Patents

Engineered meganucleases with recognition sequences found in the human beta-2 microglobulin gene Download PDF

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Publication number
CA3009637A1
CA3009637A1 CA3009637A CA3009637A CA3009637A1 CA 3009637 A1 CA3009637 A1 CA 3009637A1 CA 3009637 A CA3009637 A CA 3009637A CA 3009637 A CA3009637 A CA 3009637A CA 3009637 A1 CA3009637 A1 CA 3009637A1
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seq
meganuclease
cell
sequence
nos
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CA3009637A
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English (en)
French (fr)
Inventor
Victor Bartsevich
Christina PHAM
Aaron Martin
Derek Jantz
James Jefferson Smith
Michael G. Nicholson
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Precision Biosciences Inc
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Precision Biosciences Inc
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Publication of CA3009637A1 publication Critical patent/CA3009637A1/en
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    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/10Cellular immunotherapy characterised by the cell type used
    • A61K40/11T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/30Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/31Chimeric antigen receptors [CAR]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/40Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41Vertebrate antigens
    • A61K40/42Cancer antigens
    • A61K40/4202Receptors, cell surface antigens or cell surface determinants
    • A61K40/421Immunoglobulin superfamily
    • A61K40/4211CD19 or B4
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/69Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6901Conjugates being cells, cell fragments, viruses, ghosts, red blood cells or viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/04Immunostimulants
    • 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
    • C12N15/52Genes encoding for enzymes or proenzymes
    • 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
    • C12N15/62DNA sequences coding for fusion proteins
    • 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
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503Immunoglobulin superfamily
    • C07K14/7051T-cell receptor (TcR)-CD3 complex
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Mycology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Developmental Biology & Embryology (AREA)
CA3009637A 2015-12-23 2016-12-22 Engineered meganucleases with recognition sequences found in the human beta-2 microglobulin gene Pending CA3009637A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562387318P 2015-12-23 2015-12-23
US62/387,318 2015-12-23
US201662416513P 2016-11-02 2016-11-02
US62/416,513 2016-11-02
PCT/US2016/068289 WO2017112859A1 (en) 2015-12-23 2016-12-22 Engineered meganucleases with recognition sequences found in the human beta-2 microglobulin gene

Publications (1)

Publication Number Publication Date
CA3009637A1 true CA3009637A1 (en) 2017-06-29

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CA3009637A Pending CA3009637A1 (en) 2015-12-23 2016-12-22 Engineered meganucleases with recognition sequences found in the human beta-2 microglobulin gene

Country Status (7)

Country Link
US (3) US20190017075A1 (OSRAM)
EP (2) EP3394253B1 (OSRAM)
JP (3) JP6846429B2 (OSRAM)
AU (2) AU2016379393B2 (OSRAM)
CA (1) CA3009637A1 (OSRAM)
ES (1) ES2901000T3 (OSRAM)
WO (1) WO2017112859A1 (OSRAM)

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US10799535B2 (en) * 2015-10-05 2020-10-13 Precision Biosciences, Inc. Engineered meganucleases with recognition sequences found in the human T cell receptor alpha constant region gene
JP6816133B2 (ja) 2015-10-05 2021-01-20 プレシジョン バイオサイエンシズ,インク. 改変ヒトt細胞受容体アルファ定常領域遺伝子を含む遺伝子改変細胞
WO2017112859A1 (en) * 2015-12-23 2017-06-29 Precision Biosciences, Inc. Engineered meganucleases with recognition sequences found in the human beta-2 microglobulin gene
EP4234691A3 (en) * 2016-10-14 2023-10-18 Precision Biosciences, Inc. Engineered meganucleases specific for recognition sequences in the hepatitis b virus genome
JP7170666B2 (ja) 2017-05-08 2022-11-14 プレシジョン バイオサイエンシズ,インク. 操作された抗原受容体をコードする核酸分子及び阻害性核酸分子、並びにそれらの使用方法
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WO2019118475A1 (en) * 2017-12-13 2019-06-20 Janssen Biotech, Inc. Immortalized car-t cells genetically modified to eliminate t-cell receptor and beta 2-microglobulin expression
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US11142750B2 (en) 2018-04-12 2021-10-12 Precision Biosciences, Inc. Optimized engineered meganucleases having specificity for a recognition sequence in the Hepatitis B virus genome
CN112105420A (zh) 2018-05-11 2020-12-18 克里斯珀医疗股份公司 用于治疗癌症的方法和组合物
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CN116200342A (zh) 2019-04-03 2023-06-02 精密生物科学公司 包含microRNA适应的shRNA(shRNAmiR)的遗传修饰的免疫细胞
CA3136265A1 (en) 2019-04-05 2020-10-08 Precision Biosciences, Inc. Methods of preparing populations of genetically-modified immune cells
WO2020222176A1 (en) 2019-04-30 2020-11-05 Crispr Therapeutics Ag Allogeneic cell therapy of b cell malignancies using genetically engineered t cells targeting cd19
CA3137975A1 (en) 2019-05-07 2020-11-12 Precision Biosciences, Inc. Optimization of engineered meganucleases for recognition sequences
EP4069729B1 (en) 2019-12-06 2025-01-22 Precision BioSciences, Inc. Optimized engineered meganucleases having specificity for a recognition sequence in the hepatitis b virus genome
US20230183664A1 (en) 2020-05-11 2023-06-15 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
EP4200412A1 (en) * 2020-08-21 2023-06-28 Precision BioSciences, Inc. Engineered meganucleases having specificity for a recognition sequence in the transthyretin gene
WO2022072325A1 (en) * 2020-09-29 2022-04-07 NeuExcell Therapeutics Inc. Neurod1 combination vector
US20220098254A1 (en) * 2020-09-29 2022-03-31 NeuExcell Therapeutics Inc. NEUROD1 and DLX2 VECTOR
US11459372B2 (en) 2020-11-30 2022-10-04 Crispr Therapeutics Ag Gene-edited natural killer cells
WO2022144632A1 (en) 2020-12-30 2022-07-07 Crispr Therapeutics Ag Compositions and methods for differentiating stem cells into nk cells
EP4419660A2 (en) * 2021-10-19 2024-08-28 Precision BioSciences, Inc. Gene editing methods for treating alpha-1 antitrypsin (aat) deficiency
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US20210032664A1 (en) 2021-02-04
EP3988655A3 (en) 2022-08-03
JP6846429B2 (ja) 2021-03-24
AU2023202035A1 (en) 2023-04-27
EP3394253B1 (en) 2021-09-01
AU2016379393B2 (en) 2023-01-05
AU2016379393A1 (en) 2018-07-12
JP2023052095A (ja) 2023-04-11
JP2018538000A (ja) 2018-12-27
US20240052373A1 (en) 2024-02-15
JP7203873B2 (ja) 2023-01-13
EP3988655A2 (en) 2022-04-27
EP3394253A1 (en) 2018-10-31
ES2901000T3 (es) 2022-03-21
WO2017112859A1 (en) 2017-06-29
JP2021101704A (ja) 2021-07-15
US20190017075A1 (en) 2019-01-17

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