CN110799644A - Cblb核酸内切酶变体、组合物及使用方法 - Google Patents

Cblb核酸内切酶变体、组合物及使用方法 Download PDF

Info

Publication number
CN110799644A
CN110799644A CN201880042211.8A CN201880042211A CN110799644A CN 110799644 A CN110799644 A CN 110799644A CN 201880042211 A CN201880042211 A CN 201880042211A CN 110799644 A CN110799644 A CN 110799644A
Authority
CN
China
Prior art keywords
polypeptide
cell
seq
amino acid
biologically active
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
CN201880042211.8A
Other languages
English (en)
Chinese (zh)
Inventor
乔丹·贾儒尔
凯尔·哈文斯
安妮-瑞秋·克罗斯塔格
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.)
Regeneron Pharmaceuticals Inc
Original Assignee
Bluebird Bio 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 Bluebird Bio Inc filed Critical Bluebird Bio Inc
Publication of CN110799644A publication Critical patent/CN110799644A/zh
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • C12N15/10—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
    • 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]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
    • A61K35/17—Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A61K40/15—Natural-killer [NK] cells; Natural-killer T [NKT] 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
    • A61K40/30—Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/32—T-cell receptors [TCR]
    • 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
    • A61K40/4203—Receptors for growth factors
    • A61K40/4204—Epidermal growth factor receptors [EGFR]
    • 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
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/405—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from algae
    • 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
    • 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
    • C07K14/71—Receptors; Cell surface antigens; Cell surface determinants for growth factors; for growth regulators
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2863—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against receptors for growth factors, growth regulators
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0636—T lymphocytes
    • 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
    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0646—Natural killers cells [NK], NKT 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
    • C12N9/10—Transferases (2.)
    • 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)
    • 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
    • C12N9/88—Lyases (4.)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y307/00—Hydrolases acting on carbon-carbon bonds (3.7)
    • C12Y307/01—Hydrolases acting on carbon-carbon bonds (3.7) in ketonic substances (3.7.1)
    • C12Y307/01003—Kynureninase (3.7.1.3)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y406/00—Phosphorus-oxygen lyases (4.6)
    • C12Y406/01—Phosphorus-oxygen lyases (4.6.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2121/00—Preparations for use in therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
    • A61K2239/38—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterised by the dose, timing or administration schedule
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/50—Fusion polypeptide containing protease site
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding
    • 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
    • C12N2510/00—Genetically modified 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Cell Biology (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Oncology (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Developmental Biology & Embryology (AREA)
  • Virology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN201880042211.8A 2017-05-25 2018-05-25 Cblb核酸内切酶变体、组合物及使用方法 Pending CN110799644A (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201762511194P 2017-05-25 2017-05-25
US62/511,194 2017-05-25
US201762567417P 2017-10-03 2017-10-03
US62/567,417 2017-10-03
PCT/US2018/034726 WO2018218194A1 (en) 2017-05-25 2018-05-25 Cblb endonuclease variants, compositions, and methods of use

Publications (1)

Publication Number Publication Date
CN110799644A true CN110799644A (zh) 2020-02-14

Family

ID=64395994

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201880042211.8A Pending CN110799644A (zh) 2017-05-25 2018-05-25 Cblb核酸内切酶变体、组合物及使用方法

Country Status (11)

Country Link
US (4) US12227740B2 (enExample)
EP (1) EP3630962A4 (enExample)
JP (4) JP7191042B2 (enExample)
KR (2) KR102590466B1 (enExample)
CN (1) CN110799644A (enExample)
AU (2) AU2018273979B2 (enExample)
CA (1) CA3064014A1 (enExample)
IL (2) IL270776B2 (enExample)
MA (1) MA48797A (enExample)
MX (1) MX2019014100A (enExample)
WO (1) WO2018218194A1 (enExample)

Families Citing this family (17)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG11201901757YA (en) 2016-09-08 2019-03-28 Bluebird Bio Inc Pd-1 homing endonuclease variants, compositions, and methods of use
IL270776B2 (en) 2017-05-25 2024-11-01 Bluebird Bio Inc Cblb endonuclease variants, compositions, and methods of use
CN112368021B (zh) 2018-06-14 2025-02-21 2赛文缇生物公司 Cd79a嵌合抗原受体
WO2020072059A1 (en) 2018-10-04 2020-04-09 Bluebird Bio, Inc. Cblb endonuclease variants, compositions, and methods of use
JP2022513750A (ja) * 2018-12-10 2022-02-09 2セブンティ バイオ インコーポレイテッド ホーミングエンドヌクレアーゼバリアント
CA3122094A1 (en) 2018-12-10 2020-06-18 Bluebird Bio, Inc. Pdcd-1 homing endonuclease variants
WO2020123933A1 (en) * 2018-12-14 2020-06-18 Bluebird Bio, Inc. Dimerizing agent regulated immunoreceptor complexes
MX2021015495A (es) * 2019-06-14 2022-04-18 2Seventy Bio Inc Composiciones y métodos para tratar cáncer.
EP4635972A3 (en) 2020-07-20 2025-12-24 Enanta Pharmaceuticals, Inc. Functionalized peptides as antiviral agents
WO2022109360A1 (en) 2020-11-23 2022-05-27 Enanta Pharmaceuticals, Inc. Novel spiropyrrolidine derived antiviral agents
US11352363B1 (en) 2020-11-23 2022-06-07 Enanta Pharmaceuticals, Inc. Spiropyrrolidine derived antiviral agents
US11384090B2 (en) 2020-11-23 2022-07-12 Enanta Pharmaceuticals, Inc. Spiropyrrolidine derived antiviral agents
US12540141B2 (en) 2020-11-23 2026-02-03 Enanta Pharmaceuticals, Inc. Spiropyrrolidine derived antiviral agents
WO2022140150A1 (en) * 2020-12-21 2022-06-30 2Seventy Bio, Inc. Compositions and methods for site-directed mutagenesis
US11319325B1 (en) 2021-05-11 2022-05-03 Enanta Pharmaceuticals, Inc. Macrocyclic spiropyrrolidine derived antiviral agents
US11325916B1 (en) 2021-07-29 2022-05-10 Enanta Pharmaceuticals, Inc. Spiropyrrolidine derived antiviral agents
US11339170B1 (en) 2021-07-23 2022-05-24 Enanta Pharmaceuticals, Inc. Spiropyrrolidine derived antiviral agents

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110179506A1 (en) * 2008-01-31 2011-07-21 Sylvestre Grizot New i-crei derived single-chain meganuclease and uses thereof
US20140148361A1 (en) * 2010-06-07 2014-05-29 Barry L. Stoddard Generation and Expression of Engineered I-ONUI Endonuclease and Its Homologues and Uses Thereof
US20160102323A1 (en) * 2013-05-31 2016-04-14 Cellectis A laglidadg homing endonuclease cleaving the c-c chemokine receptor type-5 (ccr5) gene and uses thereof
US20160208243A1 (en) * 2015-06-18 2016-07-21 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2017001572A1 (en) * 2015-06-30 2017-01-05 Cellectis Methods for improving functionality in nk cell by gene inactivation using specific endonuclease

Family Cites Families (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4751180A (en) 1985-03-28 1988-06-14 Chiron Corporation Expression using fused genes providing for protein product
US4935233A (en) 1985-12-02 1990-06-19 G. D. Searle And Company Covalently linked polypeptide cell modulators
US4873192A (en) 1987-02-17 1989-10-10 The United States Of America As Represented By The Department Of Health And Human Services Process for site specific mutagenesis without phenotypic selection
GB8918616D0 (en) 1989-08-15 1989-09-27 Univ Glasgow Herpes simplex virus type 1 mutant
US5804413A (en) 1992-07-31 1998-09-08 University Of Pittsburgh Of The Commonwealth System Of Higher Education Herpes simplex virus strains for gene transfer
GB9415319D0 (en) 1994-07-29 1994-09-21 Medical Res Council HSV viral vector
US5846782A (en) 1995-11-28 1998-12-08 Genvec, Inc. Targeting adenovirus with use of constrained peptide motifs
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
EP1002119A1 (en) 1997-07-31 2000-05-24 University Of Pittsburgh Of The Commonwealth System Of Higher Education Targeted hsv vectors
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
FR2777909B1 (fr) 1998-04-24 2002-08-02 Pasteur Institut Utilisation de sequences d'adn de structure triplex pour le tranfert de sequences de nucleotides dans des cellules, vecteurs recombinants contenant ces sequences triplex
US6692736B2 (en) 2000-03-24 2004-02-17 Cell Genesys, Inc. Cell-specific adenovirus vectors comprising an internal ribosome entry site
US7465583B2 (en) 2000-06-01 2008-12-16 The University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
US20060206949A1 (en) 2003-01-28 2006-09-14 Sylvain Arnould Custom-made meganuclease and use thereof
FR2872170B1 (fr) 2004-06-25 2006-11-10 Centre Nat Rech Scient Cnrse Lentivirus non interactif et non replicatif, preparation et utilisations
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
ES2422291T3 (es) 2007-10-31 2013-09-10 Prec Biosciences Inc Meganucleasas monocatenarias diseñadas racionalmente con secuencias de reconocimiento no palindrómicas
US20110239315A1 (en) 2009-01-12 2011-09-29 Ulla Bonas Modular dna-binding domains and methods of use
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
WO2010093966A2 (en) * 2009-02-12 2010-08-19 Fred Hutchinson Cancer Research Center Generation of a dna nicking enzyme that stimulates site-specific gene conversion from a homing endonuclease
AU2010325563B2 (en) * 2009-11-27 2017-02-02 Basf Plant Science Company Gmbh Chimeric endonucleases and uses thereof
US9169494B2 (en) 2010-01-12 2015-10-27 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
WO2011097456A2 (en) 2010-02-05 2011-08-11 The University Of North Carolina At Chapel Hill Compositions and methods for enhanced parvovirus transduction
SG189482A1 (en) 2010-10-27 2013-05-31 Cellectis Method for increasing the efficiency of double-strand break-induced mutagenesis
US8673557B2 (en) 2011-02-28 2014-03-18 Seattle Children's Research Institute Coupling endonucleases with end-processing enzymes drives high efficiency gene disruption
US20140115726A1 (en) 2011-04-05 2014-04-24 Cellectis New tale-protein scaffolds and uses thereof
BR112013030652A2 (pt) 2011-06-10 2016-12-13 Basf Plant Science Co Gmbh polinucleotídeo que codifica um polipeptídeo, molécula de ácido nucleico, vetor, organismo não humano, polipeptídeo e método para introduzir um ácido nucleico de interesse em um genoma de um organismo não humano
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
JP6608807B2 (ja) 2013-05-29 2019-11-20 セレクティス Rnaガイドcasヌクレアーゼ系を用いることによって免疫療法のためにt細胞を操作するための方法
CA2913872C (en) 2013-05-31 2022-01-18 Cellectis A laglidadg homing endonuclease cleaving the t-cell receptor alpha gene and uses thereof
SG11201608701QA (en) 2014-04-18 2016-11-29 Editas Medicine Inc Crispr-cas-related methods, compositions and components for cancer immunotherapy
CA3031785A1 (en) 2016-07-25 2018-02-01 Bluebird Bio, Inc. Bcl11a homing endonuclease variants, compositions, and methods of use
EP3500696A4 (en) 2016-08-16 2020-04-08 Bluebird Bio, Inc. VARIANTS OF IL-10 ALPHA RECEPTOR HOMING ENDONUCLEASE, COMPOSITIONS AND METHODS OF USE
US20190262398A1 (en) 2016-08-23 2019-08-29 Bluebird Bio, Inc. Tim3 homing endonuclease variants, compositions, and methods of use
SG11201901757YA (en) 2016-09-08 2019-03-28 Bluebird Bio Inc Pd-1 homing endonuclease variants, compositions, and methods of use
IL270776B2 (en) 2017-05-25 2024-11-01 Bluebird Bio Inc Cblb endonuclease variants, compositions, and methods of use
WO2020072059A1 (en) 2018-10-04 2020-04-09 Bluebird Bio, Inc. Cblb endonuclease variants, compositions, and methods of use

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20110179506A1 (en) * 2008-01-31 2011-07-21 Sylvestre Grizot New i-crei derived single-chain meganuclease and uses thereof
US20140148361A1 (en) * 2010-06-07 2014-05-29 Barry L. Stoddard Generation and Expression of Engineered I-ONUI Endonuclease and Its Homologues and Uses Thereof
US20160102323A1 (en) * 2013-05-31 2016-04-14 Cellectis A laglidadg homing endonuclease cleaving the c-c chemokine receptor type-5 (ccr5) gene and uses thereof
US20160208243A1 (en) * 2015-06-18 2016-07-21 The Broad Institute, Inc. Novel crispr enzymes and systems
WO2017001572A1 (en) * 2015-06-30 2017-01-05 Cellectis Methods for improving functionality in nk cell by gene inactivation using specific endonuclease

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
PATRIK LUCAS等: "Rapid evolution of the Dna-binding site in LAGLIDADG homing endonucleases", 《NUCLEIC ACIDS RESEARCH》 *

Also Published As

Publication number Publication date
JP2026034640A (ja) 2026-02-27
JP2020521456A (ja) 2020-07-27
US20200087653A1 (en) 2020-03-19
US11732255B2 (en) 2023-08-22
IL270776B1 (en) 2024-07-01
US20210214714A1 (en) 2021-07-15
JP7450683B2 (ja) 2024-03-15
IL313037A (en) 2024-07-01
JP7793669B2 (ja) 2026-01-05
AU2025200323A1 (en) 2025-02-13
US10927367B2 (en) 2021-02-23
AU2018273979B2 (en) 2024-10-17
WO2018218194A1 (en) 2018-11-29
JP2022183262A (ja) 2022-12-08
US20210147818A1 (en) 2021-05-20
RU2019141853A3 (enExample) 2021-08-09
RU2019141853A (ru) 2021-06-25
EP3630962A4 (en) 2021-01-20
MX2019014100A (es) 2020-02-07
US20250179469A1 (en) 2025-06-05
IL270776B2 (en) 2024-11-01
CA3064014A1 (en) 2018-11-29
AU2025200323B2 (en) 2026-04-09
JP2024055980A (ja) 2024-04-19
MA48797A (fr) 2020-04-08
KR20230147767A (ko) 2023-10-23
US12227740B2 (en) 2025-02-18
KR102742817B1 (ko) 2024-12-17
JP7191042B2 (ja) 2022-12-16
AU2018273979A1 (en) 2019-12-12
NZ759382A (en) 2025-09-26
KR102590466B1 (ko) 2023-10-19
EP3630962A1 (en) 2020-04-08
KR20200011953A (ko) 2020-02-04
IL270776A (en) 2020-01-30

Similar Documents

Publication Publication Date Title
KR102742817B1 (ko) Cblb 엔도뉴클레아제 변이체, 조성물 및 사용 방법
JP7538301B2 (ja) Pd-1ホーミングエンドヌクレアーゼバリアント、組成物、および使用方法
US12297465B2 (en) CBLB endonuclease variants, compositions, and methods of use
AU2017346683B2 (en) TGFβR2 endonuclease variants, compositions, and methods of use
CN110446781A (zh) 供体修复模板多重基因组编辑
EP3500696A1 (en) Il-10 receptor alpha homing endonuclease variants, compositions, and methods of use
US20190262398A1 (en) Tim3 homing endonuclease variants, compositions, and methods of use
WO2019126558A1 (en) Ahr homing endonuclease variants, compositions, and methods of use
RU2779097C2 (ru) Варианты, композиции и способы применения эндонуклеазы cblb
HK40101654A (en) Pd-1 homing endonuclease variants, compositions, and methods of use
CN116887853A (zh) 用于定点诱变的组合物和方法
HK40005329A (en) Pd-1 homing endonuclease variants, compositions, and methods of use
HK40005329B (en) Pd-1 homing endonuclease variants, compositions, and methods of use

Legal Events

Date Code Title Description
PB01 Publication
PB01 Publication
SE01 Entry into force of request for substantive examination
SE01 Entry into force of request for substantive examination
TA01 Transfer of patent application right

Effective date of registration: 20211102

Address after: 60 Benny street, Cambridge, Massachusetts, USA

Applicant after: 2 savinti biology

Address before: 60 Benny street, Cambridge, Massachusetts, USA

Applicant before: BLUEBIRD BIO, Inc.

TA01 Transfer of patent application right
TA01 Transfer of patent application right

Effective date of registration: 20240926

Address after: USA New York

Applicant after: REGENERON PHARMACEUTICALS, Inc.

Country or region after: U.S.A.

Address before: 60 Benny street, Cambridge, Massachusetts, USA

Applicant before: 2 savinti biology

Country or region before: U.S.A.

TA01 Transfer of patent application right