CA3064014A1 - Cblb endonuclease variants, compositions, and methods of use - Google Patents

Cblb endonuclease variants, compositions, and methods of use Download PDF

Info

Publication number
CA3064014A1
CA3064014A1 CA3064014A CA3064014A CA3064014A1 CA 3064014 A1 CA3064014 A1 CA 3064014A1 CA 3064014 A CA3064014 A CA 3064014A CA 3064014 A CA3064014 A CA 3064014A CA 3064014 A1 CA3064014 A1 CA 3064014A1
Authority
CA
Canada
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
CA3064014A
Other languages
English (en)
French (fr)
Inventor
Jordan JARJOUR
Kyle HAVENS
Anne-Rachel KROSTAG
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.)
2Seventy Bio 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 CA3064014A1 publication Critical patent/CA3064014A1/en
Pending legal-status Critical Current

Links

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
    • 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
    • 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
    • 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 [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2863—Immunoglobulins [IGs], 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)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Toxicology (AREA)
  • Oncology (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Virology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CA3064014A 2017-05-25 2018-05-25 Cblb endonuclease variants, compositions, and methods of use Pending CA3064014A1 (en)

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
CA3064014A1 true CA3064014A1 (en) 2018-11-29

Family

ID=64395994

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3064014A Pending CA3064014A1 (en) 2017-05-25 2018-05-25 Cblb endonuclease variants, compositions, and methods of use

Country Status (12)

Country Link
US (4) US12227740B2 (enExample)
EP (1) EP3630962A4 (enExample)
JP (3) 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)
NZ (1) NZ759382A (enExample)
WO (1) WO2018218194A1 (enExample)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102622910B1 (ko) 2016-09-08 2024-01-10 2세븐티 바이오, 인코포레이티드 Pd-1 호밍 엔도뉴클레아제 변이체, 조성물 및 사용 방법
MA48797A (fr) 2017-05-25 2020-04-08 Bluebird Bio Inc Variants de l'endonucléase cblb , compositions et procédés d'utilisation
EP3806903B1 (en) 2018-06-14 2024-02-14 2seventy bio, Inc. Cd79a chimeric antigen receptors
WO2020072059A1 (en) 2018-10-04 2020-04-09 Bluebird Bio, Inc. Cblb endonuclease variants, compositions, and methods of use
EP3893886A4 (en) * 2018-12-10 2022-09-28 2seventy bio, Inc. Pdcd-1 homing endonuclease variants
KR20210102925A (ko) 2018-12-10 2021-08-20 블루버드 바이오, 인코포레이티드. 호밍 엔도뉴클레아제 변이체
US20240034768A1 (en) * 2018-12-14 2024-02-01 2Seventy Bio, Inc. Dimerizing agent regulated immunoreceptor complexes
MA56212A (fr) * 2019-06-14 2022-04-20 2Seventy Bio Inc Compositions et méthodes de traitement du cancer
HUE21846991T1 (hu) 2020-07-20 2022-09-28 Enanta Pharm Inc Vírusellenes funkcionalizált peptid szerek
US11384090B2 (en) 2020-11-23 2022-07-12 Enanta Pharmaceuticals, Inc. Spiropyrrolidine derived antiviral agents
JP2024500286A (ja) 2020-11-23 2024-01-09 エナンタ ファーマシューティカルズ インコーポレイテッド 新規スピロピロリジン誘導抗ウイルス剤
US11352363B1 (en) 2020-11-23 2022-06-07 Enanta Pharmaceuticals, Inc. Spiropyrrolidine derived antiviral agents
US20240336904A1 (en) * 2020-12-21 2024-10-10 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

Family Cites Families (43)

* 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
AU8605598A (en) 1997-07-31 1999-02-22 University Of Pittsburgh 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
ES2256265T3 (es) 2000-06-01 2006-07-16 University Of North Carolina At Chapel Hill Vectores de parvovirus duplicados.
WO2009095742A1 (en) * 2008-01-31 2009-08-06 Cellectis New i-crei derived single-chain meganuclease and uses thereof
AU2004208031B2 (en) 2003-01-28 2009-10-08 Cellectis Use of meganucleases for inducing homologous recombination ex vivo and in toto in vertebrate somatic tissues and application 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
ES2732735T3 (es) * 2007-10-31 2019-11-25 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
US20110294217A1 (en) 2009-02-12 2011-12-01 Fred Hutchinson Cancer Research Center Dna nicking enzyme from a homing endonuclease that stimulates site-specific gene conversion
WO2011064750A1 (en) 2009-11-27 2011-06-03 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
US9169492B2 (en) 2010-02-05 2015-10-27 The University Of North Carolina At Chapel Hill Compositions and methods for enhanced parvovirus transduction
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
US20130337454A1 (en) 2010-10-27 2013-12-19 Philippe Duchateau Method for increasing the efficiency of double-strand break-induced mutagenesis
EP4062927A1 (en) 2011-02-28 2022-09-28 Seattle Children's Research Institute Coupling endonucleases with end-processing enzymes drives high efficiency gene disruption
JP5996630B2 (ja) 2011-04-05 2016-09-21 セレクティスＣｅｌｌｅｃｔｉｓ コンパクトｔａｌｅ−ヌクレアーゼを作製する方法及びその使用
WO2012168910A1 (en) 2011-06-10 2012-12-13 Basf Plant Science Company Gmbh Nuclease fusion protein and uses thereof
US8450107B1 (en) 2011-11-30 2013-05-28 The Broad Institute Inc. Nucleotide-specific recognition sequences for designer TAL effectors
ES2883131T3 (es) 2013-05-29 2021-12-07 Cellectis Métodos para la modificación de células T para inmunoterapia utilizando el sistema de nucleasa CAS guiado por ARN
US10000746B2 (en) 2013-05-31 2018-06-19 Cellectis LAGLIDADG homing endonuclease cleaving the T cell receptor alpha gene and uses thereof
EP3004149B1 (en) 2013-05-31 2018-12-19 Cellectis S.A. A laglidadg homing endonuclease cleaving the c-c chemokine receptor type-5 (ccr5) gene and uses thereof
SG10201809157VA (en) 2014-04-18 2018-11-29 Editas Medicine Inc Crispr-cas-related methods, compositions and components for cancer immunotherapy
US9790490B2 (en) * 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
EP3317399B1 (en) 2015-06-30 2024-06-26 Cellectis Methods for improving functionality in nk cell by gene inactivation using specific endonuclease
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
WO2018039333A1 (en) 2016-08-23 2018-03-01 Bluebird Bio, Inc. Tim3 homing endonuclease variants, compositions, and methods of use
KR102622910B1 (ko) 2016-09-08 2024-01-10 2세븐티 바이오, 인코포레이티드 Pd-1 호밍 엔도뉴클레아제 변이체, 조성물 및 사용 방법
MA48797A (fr) 2017-05-25 2020-04-08 Bluebird Bio Inc Variants de l'endonucléase cblb , compositions et procédés d'utilisation
WO2020072059A1 (en) 2018-10-04 2020-04-09 Bluebird Bio, Inc. Cblb endonuclease variants, compositions, and methods of use

Also Published As

Publication number Publication date
US20210147818A1 (en) 2021-05-20
IL270776A (en) 2020-01-30
JP7191042B2 (ja) 2022-12-16
AU2018273979B2 (en) 2024-10-17
US20250179469A1 (en) 2025-06-05
EP3630962A1 (en) 2020-04-08
AU2018273979A1 (en) 2019-12-12
MA48797A (fr) 2020-04-08
IL313037A (en) 2024-07-01
AU2025200323A1 (en) 2025-02-13
JP2022183262A (ja) 2022-12-08
KR20230147767A (ko) 2023-10-23
EP3630962A4 (en) 2021-01-20
US20200087653A1 (en) 2020-03-19
WO2018218194A1 (en) 2018-11-29
CN110799644A (zh) 2020-02-14
KR20200011953A (ko) 2020-02-04
JP2020521456A (ja) 2020-07-27
US12227740B2 (en) 2025-02-18
IL270776B1 (en) 2024-07-01
IL270776B2 (en) 2024-11-01
US11732255B2 (en) 2023-08-22
RU2019141853A (ru) 2021-06-25
US10927367B2 (en) 2021-02-23
RU2019141853A3 (enExample) 2021-08-09
KR102590466B1 (ko) 2023-10-19
MX2019014100A (es) 2020-02-07
NZ759382A (en) 2025-09-26
JP7450683B2 (ja) 2024-03-15
JP2024055980A (ja) 2024-04-19
KR102742817B1 (ko) 2024-12-17
US20210214714A1 (en) 2021-07-15

Similar Documents

Publication Publication Date Title
US11912746B2 (en) PD-1 homing endonuclease variants, compositions, and methods of use
US20250179469A1 (en) Cblb endonuclease variants, compositions, and methods of use
US12297465B2 (en) CBLB endonuclease variants, compositions, and methods of use
US11530395B2 (en) TGFBetaR2 endonuclease variants, compositions, and methods of use
CA3053499A1 (en) Donor repair templates multiplex genome editing
US20190262398A1 (en) Tim3 homing endonuclease variants, compositions, and methods of use
HK40101654A (en) Pd-1 homing endonuclease variants, compositions, and methods of use
US20210002621A1 (en) Ctla4 homing endonuclease variants, compositions, and methods of use
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
EEER Examination request

Effective date: 20220816

EEER Examination request

Effective date: 20220816

EEER Examination request

Effective date: 20220816

EEER Examination request

Effective date: 20220816

EEER Examination request

Effective date: 20220816