ES2901000T3 - Meganucleasas de ingeniería con secuencias de reconocimiento encontradas en el gen de la microglobulina beta-2 humana - Google Patents

Meganucleasas de ingeniería con secuencias de reconocimiento encontradas en el gen de la microglobulina beta-2 humana Download PDF

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Publication number
ES2901000T3
ES2901000T3 ES16825979T ES16825979T ES2901000T3 ES 2901000 T3 ES2901000 T3 ES 2901000T3 ES 16825979 T ES16825979 T ES 16825979T ES 16825979 T ES16825979 T ES 16825979T ES 2901000 T3 ES2901000 T3 ES 2901000T3
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seq
meganuclease
cells
sequence
cell
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ES16825979T
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English (en)
Spanish (es)
Inventor
Victor Bartsevich
Christina Pham
Aaron Martin
Derek Jantz
James Smith
Michael Nicholson
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Precision Biosciences Inc
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Precision Biosciences Inc
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A61K40/11—T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/30—Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/31—Chimeric antigen receptors [CAR]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41—Vertebrate antigens
    • A61K40/42—Cancer antigens
    • A61K40/4202—Receptors, cell surface antigens or cell surface determinants
    • A61K40/421—Immunoglobulin superfamily
    • A61K40/4211—CD19 or B4
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal 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
    • A61K47/69—Medicinal 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
    • A61K47/6901—Conjugates being cells, cell fragments, viruses, ghosts, red blood cells or viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A61P37/02—Immunomodulators
    • A61P37/04—Immunostimulants
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/62—DNA sequences coding for fusion proteins
    • 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
    • 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]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503—Immunoglobulin superfamily
    • C07K14/7051—T-cell receptor (TcR)-CD3 complex
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03—Fusion 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)
  • Pharmacology & Pharmacy (AREA)
  • Immunology (AREA)
  • Mycology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (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)
ES16825979T 2015-12-23 2016-12-22 Meganucleasas de ingeniería con secuencias de reconocimiento encontradas en el gen de la microglobulina beta-2 humana Active ES2901000T3 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562387318P 2015-12-23 2015-12-23
US201662416513P 2016-11-02 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
ES2901000T3 true ES2901000T3 (es) 2022-03-21

Family

ID=57794384

Family Applications (1)

Application Number Title Priority Date Filing Date
ES16825979T Active ES2901000T3 (es) 2015-12-23 2016-12-22 Meganucleasas de ingeniería con secuencias de reconocimiento encontradas en el gen de la microglobulina beta-2 humana

Country Status (7)

Country Link
US (4) US20190017075A1 (enExample)
EP (2) EP3988655A3 (enExample)
JP (3) JP6846429B2 (enExample)
AU (2) AU2016379393B2 (enExample)
CA (1) CA3009637A1 (enExample)
ES (1) ES2901000T3 (enExample)
WO (1) WO2017112859A1 (enExample)

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ES2768984T3 (es) 2015-10-05 2020-06-24 Prec Biosciences Inc Meganucleasas diseñadas con secuencias de reconocimiento encontradas en el gen de la región constante alfa del receptor de células T humanas
ES2803728T3 (es) 2015-10-05 2021-01-29 Prec Biosciences Inc Células genéticamente modificadas que comprenden un gen de región constante alfa del receptor de linfocitos T humano modificado
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KR20230010826A (ko) * 2016-10-14 2023-01-19 프리시젼 바이오사이언시스 인코포레이티드 B형 간염 바이러스 게놈 내의 인식 서열에 대해 특이적인 조작된 메가뉴클레아제
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
US11166985B2 (en) 2017-05-12 2021-11-09 Crispr Therapeutics Ag Materials and methods for engineering cells and uses thereof in immuno-oncology
BR112019023608A2 (pt) 2017-05-12 2020-05-26 Crispr Therapeutics Ag Materiais e métodos para células manipuladas e seus usos em imuno-oncologia
EP4775669A2 (en) 2017-06-30 2026-07-15 Precision Biosciences, Inc. Genetically-modified t cells comprising a modified intron in the t cell receptor alpha gene
TW201923073A (zh) * 2017-11-20 2019-06-16 新加坡商泰莎治療私人有限公司 經修飾之k562細胞
CA3082204A1 (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
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
KR102617818B1 (ko) * 2018-04-12 2023-12-27 프리시젼 바이오사이언시스 인코포레이티드 인간 t 세포 수용체 알파 불변 영역 유전자에 대한 특이성을 갖는 최적화된 조작된 뉴클레아제
US20200063100A1 (en) 2018-05-11 2020-02-27 Crispr Therapeutics Ag Methods and compositions for treating cancer
WO2020146807A1 (en) * 2019-01-10 2020-07-16 Precision Biosciences, Inc. Genetically-modified cells comprising a modified transferrin gene
EP4332115A3 (en) 2019-04-03 2024-05-29 Precision Biosciences, Inc. Genetically-modified immune cells comprising a microrna-adapted shrna (shrnamir)
EP3947646A1 (en) 2019-04-05 2022-02-09 Precision BioSciences, Inc. Methods of preparing populations of genetically-modified immune cells
CA3138633A1 (en) 2019-04-30 2020-11-05 Crispr Therapeutics Ag Allogeneic cell therapy of b cell malignancies using genetically engineered t cells targeting cd19
CN114026228A (zh) 2019-05-07 2022-02-08 精密生物科学公司 用于识别序列的工程化大范围核酸酶的优化
US12410418B2 (en) 2019-12-06 2025-09-09 Precision Biosciences, Inc. Optimized engineered meganucleases having specificity for a recognition sequence in the Hepatitis B virus genome
WO2021231259A1 (en) 2020-05-11 2021-11-18 Precision Biosciences, Inc. Self-limiting viral vectors encoding nucleases
US20240011003A1 (en) * 2020-08-21 2024-01-11 Precision Biosciences, Inc. Engineered meganucleases having specificity for a recognition sequence in the transthyretin gene
JP2023543361A (ja) * 2020-09-29 2023-10-13 ニューエクセル・セラピューティクス・インコーポレイテッド Ｎｅｕｒｏｄ１及びｄｌｘ２ベクター
EP4221761A4 (en) * 2020-09-29 2025-02-26 Neuexcell Therapeutics Inc. Neurod1 combination vector
US11459372B2 (en) 2020-11-30 2022-10-04 Crispr Therapeutics Ag Gene-edited natural killer cells
WO2022112596A1 (en) * 2020-11-30 2022-06-02 Cellectis Sa Use of aminoquinoline compounds for higher gene integration
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CA3009637A1 (en) 2017-06-29
JP2023052095A (ja) 2023-04-11
JP2021101704A (ja) 2021-07-15
AU2023202035A1 (en) 2023-04-27
AU2016379393B2 (en) 2023-01-05
US20260201422A1 (en) 2026-07-16
US20190017075A1 (en) 2019-01-17
WO2017112859A1 (en) 2017-06-29
US20210032664A1 (en) 2021-02-04
JP2018538000A (ja) 2018-12-27
JP7203873B2 (ja) 2023-01-13
EP3394253B1 (en) 2021-09-01
US20240052373A1 (en) 2024-02-15
EP3394253A1 (en) 2018-10-31
JP6846429B2 (ja) 2021-03-24
AU2016379393A1 (en) 2018-07-12
EP3988655A2 (en) 2022-04-27
EP3988655A3 (en) 2022-08-03

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