WO2019178421A1 - Gene-regulating compositions and methods for improved immunotherapy - Google Patents

Gene-regulating compositions and methods for improved immunotherapy Download PDF

Info

Publication number
WO2019178421A1
WO2019178421A1 PCT/US2019/022365 US2019022365W WO2019178421A1 WO 2019178421 A1 WO2019178421 A1 WO 2019178421A1 US 2019022365 W US2019022365 W US 2019022365W WO 2019178421 A1 WO2019178421 A1 WO 2019178421A1
Authority
WO
WIPO (PCT)
Prior art keywords
gene
immune effector
nos
seq
group
Prior art date
Application number
PCT/US2019/022365
Other languages
English (en)
French (fr)
Inventor
Micah BENSON
Jason MERKIN
Gregory V. KRYUKOV
Solomon Martin SHENKER
Michael Schlabach
Noah TUBO
Original Assignee
KSQ Therapeutics, 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 KSQ Therapeutics, Inc. filed Critical KSQ Therapeutics, Inc.
Priority to KR1020207026518A priority Critical patent/KR20200130826A/ko
Priority to CA3093919A priority patent/CA3093919A1/en
Priority to JP2020573081A priority patent/JP2021518161A/ja
Priority to AU2019236204A priority patent/AU2019236204A1/en
Priority to CN201980019421.XA priority patent/CN112040987A/zh
Priority to BR112020018658-9A priority patent/BR112020018658A2/pt
Priority to EP19767975.6A priority patent/EP3765094A4/en
Publication of WO2019178421A1 publication Critical patent/WO2019178421A1/en

Links

Classifications

    • 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
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0634Cells from the blood or the immune system
    • C12N5/0636T lymphocytes
    • 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
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4747Apoptosis related proteins
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/46Cellular immunotherapy
    • A61K39/461Cellular immunotherapy characterised by the cell type used
    • A61K39/4611T-cells, e.g. tumor infiltrating lymphocytes [TIL], lymphokine-activated killer cells [LAK] or regulatory T cells [Treg]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/46Cellular immunotherapy
    • A61K39/463Cellular immunotherapy characterised by recombinant expression
    • A61K39/4631Chimeric Antigen Receptors [CAR]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/46Cellular immunotherapy
    • A61K39/463Cellular immunotherapy characterised by recombinant expression
    • A61K39/4632T-cell receptors [TCR]; antibody T-cell receptor constructs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/46Cellular immunotherapy
    • A61K39/464Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643Vertebrate antigens
    • A61K39/4644Cancer antigens
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/46Cellular immunotherapy
    • A61K39/464Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643Vertebrate antigens
    • A61K39/4644Cancer antigens
    • A61K39/464402Receptors, cell surface antigens or cell surface determinants
    • A61K39/464403Receptors for growth factors
    • A61K39/464404Epidermal growth factor receptors [EGFR]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/46Cellular immunotherapy
    • A61K39/464Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643Vertebrate antigens
    • A61K39/4644Cancer antigens
    • A61K39/464402Receptors, cell surface antigens or cell surface determinants
    • A61K39/464403Receptors for growth factors
    • A61K39/464406Her-2/neu/ErbB2, Her-3/ErbB3 or Her 4/ ErbB4
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/46Cellular immunotherapy
    • A61K39/464Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643Vertebrate antigens
    • A61K39/4644Cancer antigens
    • A61K39/464402Receptors, cell surface antigens or cell surface determinants
    • A61K39/464411Immunoglobulin superfamily
    • A61K39/464412CD19 or B4
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/46Cellular immunotherapy
    • A61K39/464Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643Vertebrate antigens
    • A61K39/4644Cancer antigens
    • A61K39/464452Transcription factors, e.g. SOX or c-MYC
    • A61K39/464453Wilms tumor 1 [WT1]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/46Cellular immunotherapy
    • A61K39/464Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643Vertebrate antigens
    • A61K39/4644Cancer antigens
    • A61K39/464484Cancer testis antigens, e.g. SSX, BAGE, GAGE or SAGE
    • A61K39/464488NY-ESO
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/46Cellular immunotherapy
    • A61K39/464Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643Vertebrate antigens
    • A61K39/4644Cancer antigens
    • A61K39/46449Melanoma antigens
    • A61K39/464491Melan-A/MART
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/46Cellular immunotherapy
    • A61K39/464Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643Vertebrate antigens
    • A61K39/4644Cancer antigens
    • A61K39/46449Melanoma antigens
    • A61K39/464492Glycoprotein 100 [Gp100]
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • 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
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
    • C07K14/4703Inhibitors; Suppressors
    • 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
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4702Regulators; Modulating activity
    • C07K14/4705Regulators; Modulating activity stimulating, promoting or activating activity
    • 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
    • C07K14/46Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4728Calcium binding proteins, e.g. calmodulin
    • 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
    • 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
    • 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
    • C07K14/72Receptors; Cell surface antigens; Cell surface determinants for hormones
    • C07K14/723G protein coupled receptor, e.g. TSHR-thyrotropin-receptor, LH/hCG receptor, FSH receptor
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/81Protease inhibitors
    • C07K14/8107Endopeptidase (E.C. 3.4.21-99) inhibitors
    • C07K14/811Serine protease (E.C. 3.4.21) inhibitors
    • C07K14/8121Serpins
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/82Translation products from oncogenes
    • 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
    • C12N15/10Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102Mutagenizing nucleic acids
    • 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
    • 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
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10Transferases (2.)
    • C12N9/1048Glycosyltransferases (2.4)
    • C12N9/1077Pentosyltransferases (2.4.2)
    • 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
    • C12N9/10Transferases (2.)
    • C12N9/12Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • 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)
    • 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 RNAses, DNAses
    • 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
    • C12N9/88Lyases (4.)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y204/00Glycosyltransferases (2.4)
    • C12Y204/02Pentosyltransferases (2.4.2)
    • C12Y204/0201Orotate phosphoribosyltransferase (2.4.2.10)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y207/00Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11001Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y301/00Hydrolases acting on ester bonds (3.1)
    • C12Y301/04Phosphoric diester hydrolases (3.1.4)
    • C12Y301/04001Phosphodiesterase I (3.1.4.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y401/00Carbon-carbon lyases (4.1)
    • C12Y401/01Carboxy-lyases (4.1.1)
    • C12Y401/01023Orotidine-5'-phosphate decarboxylase (4.1.1.23)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00Indexing codes associated with cellular immunotherapy of group A61K39/46
    • A61K2239/27Indexing codes associated with cellular immunotherapy of group A61K39/46 characterized by targeting or presenting multiple antigens
    • A61K2239/28Expressing multiple CARs, TCRs or antigens
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00Indexing codes associated with cellular immunotherapy of group A61K39/46
    • A61K2239/27Indexing codes associated with cellular immunotherapy of group A61K39/46 characterized by targeting or presenting multiple antigens
    • A61K2239/29Multispecific CARs
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00Indexing codes associated with cellular immunotherapy of group A61K39/46
    • A61K2239/31Indexing codes associated with cellular immunotherapy of group A61K39/46 characterized by the route of administration
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00Indexing codes associated with cellular immunotherapy of group A61K39/46
    • A61K2239/38Indexing codes associated with cellular immunotherapy of group A61K39/46 characterised by the dose, timing or administration schedule
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00Indexing codes associated with cellular immunotherapy of group A61K39/46
    • A61K2239/46Indexing codes associated with cellular immunotherapy of group A61K39/46 characterised by the cancer treated
    • A61K2239/48Blood cells, e.g. leukemia or lymphoma
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00Indexing codes associated with cellular immunotherapy of group A61K39/46
    • A61K2239/46Indexing codes associated with cellular immunotherapy of group A61K39/46 characterised by the cancer treated
    • A61K2239/50Colon
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00Indexing codes associated with cellular immunotherapy of group A61K39/46
    • A61K2239/46Indexing codes associated with cellular immunotherapy of group A61K39/46 characterised by the cancer treated
    • A61K2239/57Skin; melanoma
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • 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
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • 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 [CRISPRs]
    • 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
    • C12N2510/00Genetically modified cells
PCT/US2019/022365 2018-03-15 2019-03-14 Gene-regulating compositions and methods for improved immunotherapy WO2019178421A1 (en)

Priority Applications (7)

Application Number Priority Date Filing Date Title
KR1020207026518A KR20200130826A (ko) 2018-03-15 2019-03-14 개선된 면역요법을 위한 유전자-조절 조성물 및 방법
CA3093919A CA3093919A1 (en) 2018-03-15 2019-03-14 Gene-regulating compositions and methods for improved immunotherapy
JP2020573081A JP2021518161A (ja) 2018-03-15 2019-03-14 免疫療法の改善のための遺伝子調節組成物及び遺伝子調節方法
AU2019236204A AU2019236204A1 (en) 2018-03-15 2019-03-14 Gene-regulating compositions and methods for improved immunotherapy
CN201980019421.XA CN112040987A (zh) 2018-03-15 2019-03-14 用于改进的免疫疗法的基因调控组合物和方法
BR112020018658-9A BR112020018658A2 (pt) 2018-03-15 2019-03-14 Composições de regulação gênica e métodos para imu-noterapia aprimorada
EP19767975.6A EP3765094A4 (en) 2018-03-15 2019-03-14 GENE REGULATION COMPOSITIONS AND METHODS FOR IMPROVING IMMUNOTHERAPY

Applications Claiming Priority (24)

Application Number Priority Date Filing Date Title
US201862643598P 2018-03-15 2018-03-15
US201862643587P 2018-03-15 2018-03-15
US201862643597P 2018-03-15 2018-03-15
US201862643578P 2018-03-15 2018-03-15
US62/643,587 2018-03-15
US62/643,578 2018-03-15
US62/643,598 2018-03-15
US62/643,597 2018-03-15
US201862692019P 2018-06-29 2018-06-29
US201862692010P 2018-06-29 2018-06-29
US201862692100P 2018-06-29 2018-06-29
US62/692,100 2018-06-29
US62/692,010 2018-06-29
US62/692,019 2018-06-29
US201862768443P 2018-11-16 2018-11-16
US201862768458P 2018-11-16 2018-11-16
US201862768448P 2018-11-16 2018-11-16
US201862768428P 2018-11-16 2018-11-16
US62/768,443 2018-11-16
US62/768,448 2018-11-16
US62/768,428 2018-11-16
US62/768,458 2018-11-16
US201962804265P 2019-02-12 2019-02-12
US62/804,265 2019-02-12

Publications (1)

Publication Number Publication Date
WO2019178421A1 true WO2019178421A1 (en) 2019-09-19

Family

ID=67906919

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2019/022365 WO2019178421A1 (en) 2018-03-15 2019-03-14 Gene-regulating compositions and methods for improved immunotherapy

Country Status (6)

Country Link
JP (1) JP2021518161A (ko)
KR (1) KR20200130826A (ko)
CN (1) CN112040987A (ko)
AU (1) AU2019236204A1 (ko)
CA (1) CA3093919A1 (ko)
WO (1) WO2019178421A1 (ko)

Cited By (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019057102A1 (en) 2017-09-20 2019-03-28 Tsinghua University HPK1 TARGETING ARNG AND HPK1 GENE EDITING METHOD
CN110777147A (zh) * 2019-11-29 2020-02-11 上海科技大学 一种ikzf3基因沉默的t细胞及其应用
WO2021108455A1 (en) * 2019-11-25 2021-06-03 KSQ Therapeutics, Inc. Methods for activation and expansion of tumor infiltrating lymphocytes
WO2022159496A3 (en) * 2021-01-19 2022-10-06 The J. David Gladstone Institutes, A Testamentary Trust Under The Will Of J. David Gladstone Gene activation targets for enhanced human t cell function
US11497773B2 (en) 2020-09-23 2022-11-15 Crispr Therapeutics Ag Genetically engineered t cells with regnase-1 and/or TGFBRII disruption have improved functionality and persistence
WO2023011433A1 (zh) * 2021-08-03 2023-02-09 苏州沙砾生物科技有限公司 一种修饰的肿瘤浸润淋巴细胞及其用途
WO2023225665A1 (en) * 2022-05-19 2023-11-23 Lyell Immunopharma, Inc. Polynucleotides targeting nr4a3 and uses thereof

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN117355600A (zh) * 2021-05-20 2024-01-05 上海药明生物技术有限公司 遗传修饰的nk细胞及其用途
CN113750218B (zh) * 2021-08-10 2022-05-20 南通市肿瘤医院 多肽zc3h12a及其突变体在制备抗肝癌药物中的应用
WO2023082640A1 (zh) * 2021-11-10 2023-05-19 清华大学 增强免疫细胞持久性的方法
WO2023143515A1 (zh) * 2022-01-29 2023-08-03 苏州沙砾生物科技有限公司 一种修饰的肿瘤浸润淋巴细胞及其用途
WO2024011335A1 (zh) * 2022-07-10 2024-01-18 清华大学 经修饰的免疫细胞

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060183687A1 (en) * 1997-09-17 2006-08-17 Suzanne Cory Novel therapeutic molecules
US20150218636A1 (en) * 2012-04-12 2015-08-06 Aarhus Universitet Mutations In Calmodulin Genes
WO2017001572A1 (en) * 2015-06-30 2017-01-05 Cellectis Methods for improving functionality in nk cell by gene inactivation using specific endonuclease
WO2017040186A1 (en) * 2015-08-28 2017-03-09 Bioincept, Llc Compositions and methods for the treatment of neurodamage
WO2017048995A1 (en) * 2015-09-15 2017-03-23 Mirimus, Inc. Inducible crispr/cas9 and rnai systems and methods of use
WO2017079642A1 (en) * 2015-11-04 2017-05-11 Emory University Immune cells with dnmt3a gene modifications and methods related thereto

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPWO2010098429A1 (ja) * 2009-02-27 2012-09-06 国立大学法人大阪大学 免疫アジュバント組成物、及びその利用
KR20160089527A (ko) * 2013-12-12 2016-07-27 더 브로드 인스티튜트, 인코퍼레이티드 게놈 편집을 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료적 응용
EP3303381A1 (en) * 2015-05-29 2018-04-11 Fred Hutchinson Cancer Research Center Compositions for cellular immunotherapy
BE1023924B1 (nl) * 2015-12-09 2017-09-13 Stairsolutions Vof Systeem van elementen voor het samenstellen van een trap en een trap samengesteld met elementen van zulk systeem

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20060183687A1 (en) * 1997-09-17 2006-08-17 Suzanne Cory Novel therapeutic molecules
US20150218636A1 (en) * 2012-04-12 2015-08-06 Aarhus Universitet Mutations In Calmodulin Genes
WO2017001572A1 (en) * 2015-06-30 2017-01-05 Cellectis Methods for improving functionality in nk cell by gene inactivation using specific endonuclease
WO2017040186A1 (en) * 2015-08-28 2017-03-09 Bioincept, Llc Compositions and methods for the treatment of neurodamage
WO2017048995A1 (en) * 2015-09-15 2017-03-23 Mirimus, Inc. Inducible crispr/cas9 and rnai systems and methods of use
WO2017079642A1 (en) * 2015-11-04 2017-05-11 Emory University Immune cells with dnmt3a gene modifications and methods related thereto

Non-Patent Citations (6)

* Cited by examiner, † Cited by third party
Title
ANONYMOUS: "Mouse Assembly", UCSC GENOME BROWSER GATEWAY, December 2011 (2011-12-01), pages 1 - 4, XP009523756, Retrieved from the Internet <URL:https://genome.ucsc.edu/cgi-bin/hgGateway> [retrieved on 20190630] *
AUBREY, BJ ET AL.: "An Inducible Lentiviral Guide RNA Platform Enables the Identification of Tumor-Essential Genes and Tumor-Promoting Mutations In Vivo", CELL REPORTS, vol. 10, no. 8, 26 February 2015 (2015-02-26), pages 1422 - 1432, XP055303271, DOI: 10.1016/j.celrep.2015.02.002 *
DEWITT, MA ET AL.: "Genome editing via delivery of Cas9 ribonucleoprotein", METHODS, vol. 121 -122, 12 April 2017 (2017-04-12), pages 9 - 15, XP055563766 *
NG, KP ET AL.: "A common BIM deletion polymorphism mediates intrinsic resistance and inferior responses to tyrosine kinase inhibitors in cancer", NATURE MEDICINE, vol. 18, no. 4, 18 March 2012 (2012-03-18), pages 521 - 528, XP055144177, DOI: 10.1038/nm.2713 *
SABBAGH, L ET AL.: "A critical role for TNF receptor-associated factor 1 and Bim down-regulation in CD 8 memory T cell survival", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE USA, vol. 103, no. 49, 5 December 2006 (2006-12-05), pages 18703 - 18708, XP055636439 *
SOH, SX ET AL.: "Multi-Agent Chemotherapy Overcomes Glucocorticoid Resistance Conferred by a BIM Deletion Polymorphism in Pediatric Acute Lymphoblastic Leukemia", PLOS ONE, vol. 9, no. 8, 4 August 2014 (2014-08-04), pages 1 - 12, XP055636442 *

Cited By (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019057102A1 (en) 2017-09-20 2019-03-28 Tsinghua University HPK1 TARGETING ARNG AND HPK1 GENE EDITING METHOD
EP3682000A4 (en) * 2017-09-20 2021-01-20 Beijing Yufan Biotechnologies Co., Ltd. HPK1 TARGETING ARNG AND HPK1 GENE EDITING METHOD
US11786550B2 (en) 2017-09-20 2023-10-17 Beijing Synthetic Vaccine Biosciences Co., Ltd gRNA targeting HPK1 and a method for editing HPK1 gene
WO2021108455A1 (en) * 2019-11-25 2021-06-03 KSQ Therapeutics, Inc. Methods for activation and expansion of tumor infiltrating lymphocytes
CN115003387A (zh) * 2019-11-25 2022-09-02 Ksq治疗公司 用于活化和扩增肿瘤浸润淋巴细胞的方法
CN110777147A (zh) * 2019-11-29 2020-02-11 上海科技大学 一种ikzf3基因沉默的t细胞及其应用
US11497773B2 (en) 2020-09-23 2022-11-15 Crispr Therapeutics Ag Genetically engineered t cells with regnase-1 and/or TGFBRII disruption have improved functionality and persistence
US11679130B2 (en) 2020-09-23 2023-06-20 Crispr Therapeutics Ag Genetically engineered t cells with Regnase-1 and/or TGFBRII disruption have improved functionality and persistence
US11679131B2 (en) 2020-09-23 2023-06-20 Crispr Therapeutics Ag Genetically engineered T cells with regnase-1 and/or TGFBRII disruption have improved functionality and persistence
US11857574B2 (en) 2020-09-23 2024-01-02 Crispr Therapeutics Ag Genetically engineered T cells with Regnase-1 and/or TGFBRII disruption have improved functionality and persistence
WO2022159496A3 (en) * 2021-01-19 2022-10-06 The J. David Gladstone Institutes, A Testamentary Trust Under The Will Of J. David Gladstone Gene activation targets for enhanced human t cell function
WO2023011433A1 (zh) * 2021-08-03 2023-02-09 苏州沙砾生物科技有限公司 一种修饰的肿瘤浸润淋巴细胞及其用途
WO2023225665A1 (en) * 2022-05-19 2023-11-23 Lyell Immunopharma, Inc. Polynucleotides targeting nr4a3 and uses thereof

Also Published As

Publication number Publication date
JP2021518161A (ja) 2021-08-02
CA3093919A1 (en) 2019-09-19
KR20200130826A (ko) 2020-11-20
AU2019236204A1 (en) 2020-10-08
CN112040987A (zh) 2020-12-04

Similar Documents

Publication Publication Date Title
US20230088186A1 (en) Gene-regulating compositions and methods for improved immunotherapy
US11608500B2 (en) Gene-regulating compositions and methods for improved immunotherapy
US11459544B2 (en) Gene-regulating compositions and methods for improved immunotherapy
AU2019236204A1 (en) Gene-regulating compositions and methods for improved immunotherapy
US20200347386A1 (en) Combination gene targets for improved immunotherapy
JP2021518161A6 (ja) 免疫療法の改善のための遺伝子調節組成物及び遺伝子調節方法
US11332713B2 (en) Gene-regulating compositions and methods for improved immunotherapy
US20230119142A1 (en) Guide rna combinations and methods of use

Legal Events

Date Code Title Description
121 Ep: the epo has been informed by wipo that ep was designated in this application

Ref document number: 19767975

Country of ref document: EP

Kind code of ref document: A1

ENP Entry into the national phase

Ref document number: 3093919

Country of ref document: CA

Ref document number: 2020573081

Country of ref document: JP

Kind code of ref document: A

NENP Non-entry into the national phase

Ref country code: DE

ENP Entry into the national phase

Ref document number: 2019236204

Country of ref document: AU

Date of ref document: 20190314

Kind code of ref document: A

WWE Wipo information: entry into national phase

Ref document number: 2019767975

Country of ref document: EP

ENP Entry into the national phase

Ref document number: 2019767975

Country of ref document: EP

Effective date: 20201015

REG Reference to national code

Ref country code: BR

Ref legal event code: B01A

Ref document number: 112020018658

Country of ref document: BR

ENP Entry into the national phase

Ref document number: 112020018658

Country of ref document: BR

Kind code of ref document: A2

Effective date: 20200914