JP2024503291A - ユニバーサルドナー細胞 - Google Patents

ユニバーサルドナー細胞 Download PDF

Info

Publication number
JP2024503291A
JP2024503291A JP2023540003A JP2023540003A JP2024503291A JP 2024503291 A JP2024503291 A JP 2024503291A JP 2023540003 A JP2023540003 A JP 2023540003A JP 2023540003 A JP2023540003 A JP 2023540003A JP 2024503291 A JP2024503291 A JP 2024503291A
Authority
JP
Japan
Prior art keywords
locus
nucleotide sequence
nucleic acid
target
vitro method
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
JP2023540003A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024503291A5 (enExample
Inventor
アリレザ レザニア，
バレンティン スルチ，
Original Assignee
クリスパー セラピューティクス アクチェンゲゼルシャフト
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 クリスパー セラピューティクス アクチェンゲゼルシャフト filed Critical クリスパー セラピューティクス アクチェンゲゼルシャフト
Publication of JP2024503291A publication Critical patent/JP2024503291A/ja
Publication of JP2024503291A5 publication Critical patent/JP2024503291A5/ja
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
    • 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
    • C12N5/0676—Pancreatic 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
    • C12N5/0603—Embryonic cells ; Embryoid bodies
    • C12N5/0606—Pluripotent embryonic cells, e.g. embryonic stem cells [ES]
    • 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
    • A61K35/37—Digestive system
    • A61K35/39—Pancreas; Islets of Langerhans
    • 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
    • A61K35/48—Reproductive organs
    • A61K35/54—Ovaries; Ova; Ovules; Embryos; Foetal cells; Germ cells
    • A61K35/545—Embryonic stem cells; Pluripotent stem cells; Induced pluripotent stem cells; Uncharacterised stem cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/18—Drugs for disorders of the alimentary tract or the digestive system for pancreatic disorders, e.g. pancreatic enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • C07K14/475—Growth factors; Growth regulators
    • 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
    • C07K14/475—Growth factors; Growth regulators
    • C07K14/495—Transforming growth factor [TGF]
    • 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
    • C07K14/52—Cytokines; Lymphokines; Interferons
    • C07K14/525—Tumour necrosis factor [TNF]
    • 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
    • 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
    • C07K14/70532—B7 molecules, e.g. CD80, CD86
    • 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
    • C07K14/70539—MHC-molecules, e.g. HLA-molecules
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal 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
    • 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
    • C12N5/0696—Artificially induced pluripotent stem cells, e.g. iPS
    • 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.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • 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
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • 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
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/48—Hydrolases (3) acting on peptide bonds (3.4)
    • C12N9/485—Exopeptidases (3.4.11-3.4.19)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y207/00—Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/11—Protein-serine/threonine kinases (2.7.11)
    • C12Y207/11001—Non-specific serine/threonine protein kinase (2.7.11.1), i.e. casein kinase or checkpoint kinase
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y301/00—Hydrolases acting on ester bonds (3.1)
    • C12Y301/03—Phosphoric monoester hydrolases (3.1.3)
    • C12Y301/03005—5'-Nucleotidase (3.1.3.5)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
    • C12Y304/19—Omega peptidases (3.4.19)
    • C12Y304/19012—Ubiquitinyl hydrolase 1 (3.4.19.12)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
    • C12Y306/01—Hydrolases acting on acid anhydrides (3.6) in phosphorus-containing anhydrides (3.6.1)
    • C12Y306/01005—Apyrase (3.6.1.5), i.e. ATP diphosphohydrolase
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/02—Fusion polypeptide containing a localisation/targetting motif containing a signal sequence
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/035—Fusion polypeptide containing a localisation/targetting motif containing a signal for targeting to the external surface of a cell, e.g. to the outer membrane of Gram negative bacteria, GPI- anchored eukaryote proteins
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09—Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • 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
    • C12N2506/00—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
    • C12N2506/02—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from embryonic 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
    • C12N2506/00—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
    • C12N2506/03—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from non-embryonic pluripotent stem 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
    • C12N2506/00—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells
    • C12N2506/45—Differentiation of animal cells from one lineage to another; Differentiation of pluripotent cells from artificially induced pluripotent stem 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
    • C12N2510/00—Genetically modified cells

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Immunology (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Reproductive Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Gynecology & Obstetrics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Diabetes (AREA)
  • Mycology (AREA)
  • Transplantation (AREA)
JP2023540003A 2020-12-31 2021-12-31 ユニバーサルドナー細胞 Pending JP2024503291A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202063132890P 2020-12-31 2020-12-31
US63/132,890 2020-12-31
US202163234997P 2021-08-19 2021-08-19
US63/234,997 2021-08-19
US202163288356P 2021-12-10 2021-12-10
US63/288,356 2021-12-10
PCT/IB2021/062526 WO2022144856A1 (en) 2020-12-31 2021-12-31 Universal donor cells

Publications (2)

Publication Number Publication Date
JP2024503291A true JP2024503291A (ja) 2024-01-25
JP2024503291A5 JP2024503291A5 (enExample) 2025-01-14

Family

ID=80112324

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2023540003A Pending JP2024503291A (ja) 2020-12-31 2021-12-31 ユニバーサルドナー細胞
JP2023540001A Pending JP2024503290A (ja) 2020-12-31 2021-12-31 ユニバーサルドナー細胞

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2023540001A Pending JP2024503290A (ja) 2020-12-31 2021-12-31 ユニバーサルドナー細胞

Country Status (8)

Country Link
US (6) US11566230B2 (enExample)
EP (2) EP4271796A1 (enExample)
JP (2) JP2024503291A (enExample)
KR (2) KR20230146007A (enExample)
AU (2) AU2021414617A1 (enExample)
CA (2) CA3203707A1 (enExample)
TW (2) TW202241472A (enExample)
WO (2) WO2022144855A1 (enExample)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US12378572B2 (en) 2018-09-07 2025-08-05 Crispr Therapeutics Ag Universal donor cells
US11116798B2 (en) 2019-09-05 2021-09-14 Crispr Therapeutics Ag Universal donor cells
US11566230B2 (en) 2020-12-31 2023-01-31 Crispr Therapeutics Ag Universal donor cells
CN116179495B (zh) * 2022-11-28 2025-06-06 上海恩凯细胞技术有限公司 转基因免疫细胞及其应用
WO2025202874A1 (en) 2024-03-25 2025-10-02 Crispr Therapeutics Ag Genetically modified cells expressing glp-1 receptor agonist
WO2025259836A1 (en) * 2024-06-13 2025-12-18 Beth Israel Deaconess Medical Center, Inc. Therapy for ocular neovascularization
WO2026069163A1 (en) 2024-09-25 2026-04-02 Crispr Therapeutics Ag Differentiation method for producing immature beta cells

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170274048A1 (en) * 2014-03-28 2017-09-28 Buck Institute For Research On Aging Methods and compositions for modulating the imune system
WO2020049535A1 (en) * 2018-09-07 2020-03-12 Crisper Therapeutics Ag Universal donor cells

Family Cites Families (112)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US1724052A (en) 1928-06-13 1929-08-13 Luther S Swearengin Fire-door construction
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5283058A (en) 1990-08-30 1994-02-01 The General Hospital Corporation Methods for inhibiting rejection of transplanted tissue
EP0555428A1 (en) 1991-07-30 1993-08-18 Baxter International Inc. Foramenous implant
US7101543B2 (en) 2000-03-31 2006-09-05 Novarx Genetically modified cells expressing a TGFβ inhibitor, the cells being lung cancer cells
JP2004514407A (ja) 2000-04-28 2004-05-20 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 異種キャプシド中にシュードタイピングされたａａｖ５キャプシドおよびａａｖ５ベクターを含む組換えａａｖベクター
US20060222633A1 (en) 2000-05-11 2006-10-05 Yale University Prevention, decrease, and/or treatment of immunoreactivity by depleting and/or inactivating antigen presenting cells in the host
US6635750B1 (en) 2000-07-20 2003-10-21 Millennium Pharmaceuticals, Inc. B7-H2 nucleic acids, members of the B7 family
US7432104B2 (en) 2001-08-06 2008-10-07 Bresgen Inc. Methods for the culture of human embryonic stem cells on human feeder cells
EP2722387B1 (en) 2003-12-23 2019-12-11 Viacyte, Inc. Definitive endoderm
US8586357B2 (en) 2003-12-23 2013-11-19 Viacyte, Inc. Markers of definitive endoderm
US20050266554A1 (en) 2004-04-27 2005-12-01 D Amour Kevin A PDX1 expressing endoderm
US7985585B2 (en) 2004-07-09 2011-07-26 Viacyte, Inc. Preprimitive streak and mesendoderm cells
US7541185B2 (en) 2003-12-23 2009-06-02 Cythera, Inc. Methods for identifying factors for differentiating definitive endoderm
US8187878B2 (en) 2004-08-13 2012-05-29 University Of Georgia Research Foundation, Inc. Methods for increasing definitive endoderm differentiation of pluripotent human embryonic stem cells with PI-3 kinase inhibitors
EP2110667A1 (en) 2005-01-20 2009-10-21 University Of Rochester Thoredoxin interacting protein (TXNIP) as regulator of vascular function
ES2687233T3 (es) 2005-10-27 2018-10-24 Viacyte, Inc. Endodermo de intestino proximal dorsal y ventral que expresa PDX-1
EP2420565B1 (en) 2006-02-23 2017-08-30 ViaCyte, Inc. Compositions and methods useful for culturing differentiable cells
NZ571427A (en) 2006-03-02 2012-07-27 Viacyte Inc Endocrine precursor cells, pancreatic hormone-expressing cells and methods of production
US7695965B2 (en) 2006-03-02 2010-04-13 Cythera, Inc. Methods of producing pancreatic hormones
WO2007139929A2 (en) 2006-05-25 2007-12-06 The Burnham Institute For Medical Research Methods for culture and production of single cell populations of human embryonic stem cells
RU2473685C2 (ru) 2007-07-31 2013-01-27 Лайфскен, Инк. Дифференцировка человеческих эмбриональных стволовых клеток
US7695963B2 (en) 2007-09-24 2010-04-13 Cythera, Inc. Methods for increasing definitive endoderm production
US8623650B2 (en) 2007-10-19 2014-01-07 Viacyte, Inc. Methods and compositions for feeder-free pluripotent stem cell media containing human serum
CN107574142B (zh) 2007-11-27 2021-07-06 生命扫描有限公司 人胚胎干细胞的分化
US8338170B2 (en) 2008-04-21 2012-12-25 Viacyte, Inc. Methods for purifying endoderm and pancreatic endoderm cells derived from human embryonic stem cells
US7939322B2 (en) 2008-04-24 2011-05-10 Centocor Ortho Biotech Inc. Cells expressing pluripotency markers and expressing markers characteristic of the definitive endoderm
US20120058105A1 (en) 2008-06-27 2012-03-08 Martin Kean Chong Ng Method of treatment of vascular complications
EP2346988B1 (en) 2008-10-31 2017-05-31 Janssen Biotech, Inc. Differentiation of human embryonic stem cells to the pancreatic endocrine lineage
JP2012507289A (ja) 2008-10-31 2012-03-29 ヤンセン バイオテツク，インコーポレーテツド ヒト胚性幹細胞の膵内分泌系への分化
US8008075B2 (en) 2008-11-04 2011-08-30 Viacyte, Inc. Stem cell aggregate suspension compositions and methods of differentiation thereof
US8895300B2 (en) 2008-11-04 2014-11-25 Viacyte, Inc. Scalable primate pluripotent stem cell aggregate suspension culture and differentiation thereof
EP2356227B1 (en) 2008-11-14 2018-03-28 Viacyte, Inc. Encapsulation of pancreatic cells derived from human pluripotent stem cells
US20100272710A1 (en) 2009-03-26 2010-10-28 Abdelhadi Rebbaa Compositions and methods for treating and preventing aging-associated diseases
ES2849181T3 (es) 2009-04-22 2021-08-16 Viacyte Inc Composiciones celulares derivadas de células reprogramadas desdiferenciadas
US9109245B2 (en) 2009-04-22 2015-08-18 Viacyte, Inc. Cell compositions derived from dedifferentiated reprogrammed cells
WO2010129294A2 (en) 2009-04-27 2010-11-11 Schulz Thomas C Small molecules supporting pluripotent cell growth and methods thereof
EP2456858B1 (en) 2009-07-20 2018-08-29 Janssen Biotech, Inc. Differentiation of human embryonic stem cells
RU2540016C2 (ru) 2009-07-20 2015-01-27 Янссен Байотек, Инк. Дифференцировка эмбриональных стволовых клеток человека
RU2664864C1 (ru) 2009-12-23 2018-08-23 Янссен Байотек, Инк. Способы увеличения экспрессии ngn3 и nkx6.1 в эндокринных клетках поджелудочной железы
DK2516625T3 (da) 2009-12-23 2024-09-09 Janssen Biotech Inc Differentiering af humane embryonale stamceller
KR101836850B1 (ko) 2010-08-31 2018-03-09 얀센 바이오테크 인코포레이티드 인간 배아 줄기 세포의 분화
WO2012030540A2 (en) 2010-08-31 2012-03-08 Janssen Biotech, Inc. Differentiation of pluripotent stem cells
JP5875597B2 (ja) 2010-12-08 2016-03-02 ヴィアサイト，インコーポレイテッド ヒト万能性幹細胞の成長を阻害する薬剤および方法
DK2699593T4 (da) 2011-04-20 2021-02-08 Univ Washington Through Its Center For Commercialization Beta-2 mikroglobulin-deficiente celler
USD726306S1 (en) 2011-12-12 2015-04-07 Viacyte, Inc. Cell encapsulation device
USD728095S1 (en) 2011-12-12 2015-04-28 Viacyte, Inc. Cell encapsulation device
USD726307S1 (en) 2011-12-12 2015-04-07 Viacyte Inc. Cell encapsulation device
ES2923757T3 (es) 2011-12-16 2022-09-30 Modernatx Inc Composiciones de ARNm modificado
RU2705001C2 (ru) 2011-12-22 2019-11-01 Янссен Байотек, Инк. Дифференцировка эмбриональных стволовых клеток человека в одногормональные инсулинположительные клетки
DK2657340T3 (en) 2012-04-25 2017-02-27 Deutsches Krebsforsch Thioredoxin-interacting protein (TXNIP) inhibitors for therapy
US20140162359A1 (en) 2012-05-07 2014-06-12 Janssen Biotech, Inc. Differentiation of Human Embryonic Stem Cells into Pancreatic Endoderm
ES2728782T3 (es) 2012-05-25 2019-10-28 Univ California Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN
USD734847S1 (en) 2012-05-31 2015-07-21 Viacyte, Inc. Cell encapsulation device
CN104334719B (zh) 2012-06-08 2018-02-13 詹森生物科技公司 人胚胎干细胞向胰腺内分泌细胞的分化
RU2015100900A (ru) 2012-06-14 2016-08-10 Янссен Байотек, Инк. Обработка плюрипотентных клеток
MX380562B (es) 2012-12-12 2025-03-12 Broad Inst Inc Modificaciones de sistemas, métodos y composiciones guía optimizadas para la manipulación de secuencias.
US10370644B2 (en) 2012-12-31 2019-08-06 Janssen Biotech, Inc. Method for making human pluripotent suspension cultures and cells derived therefrom
DK2938722T3 (da) 2012-12-31 2022-02-14 Janssen Biotech Inc Suspension og klyngedannelse af humane pluripotente celler til differentiering til endokrine pankreasceller
MX2015008578A (es) 2012-12-31 2015-09-07 Janssen Biotech Inc Diferenciacion de celulas madre embrionarias humanas en celulas endocrinas pancreaticas mediante el uso de relugadores de hb9.
KR102084561B1 (ko) 2012-12-31 2020-03-04 얀센 바이오테크 인코포레이티드 췌장 내분비 세포로의 분화를 위한 공기-액체 계면에서의 인간 배아 줄기세포의 배양
USD720469S1 (en) 2013-03-07 2014-12-30 Viacyte, Inc. Cell encapsulation device
JP6234485B2 (ja) 2013-03-07 2017-11-22 ヴィアサイト インコーポレイテッド ３次元大収容力細胞封入デバイスアセンブリー
US8859286B2 (en) 2013-03-14 2014-10-14 Viacyte, Inc. In vitro differentiation of pluripotent stem cells to pancreatic endoderm cells (PEC) and endocrine cells
US9101597B2 (en) 2013-03-14 2015-08-11 The Administration Of The Tulane Educational Fund Immunoprotective primary mesenchymal stem cells and methods
WO2014195159A1 (en) 2013-06-03 2014-12-11 Theravectys Lentiviral vectors containing an mhc class i, mhc class ii, or beta-2 microglobulin upstream promoter sequence
AU2014278178B2 (en) 2013-06-11 2021-01-21 President And Fellows Of Harvard College SC-beta cells and compositions and methods for generating the same
KR20140144964A (ko) 2013-06-12 2014-12-22 한국생명공학연구원 티오레독신-결합 단백질을 유효성분으로 포함하는 약학적 조성물 및 이의 용도
KR20250012194A (ko) 2013-06-17 2025-01-23 더 브로드 인스티튜트, 인코퍼레이티드 바이러스 구성성분을 사용하여 장애 및 질환을 표적화하기 위한 crispr-cas 시스템 및 조성물의 전달, 용도 및 치료 적용
RU2689710C2 (ru) 2013-11-01 2019-05-29 Янссен Байотек, Инк. Суспендирование и кластеризация плюрипотентных стволовых клеток человека с целью их дифференцировки в панкреатические эндокринные клетки
USD718472S1 (en) 2014-03-07 2014-11-25 Viacyte, Inc. 3-dimensional large capacity cell encapsulation device
USD718469S1 (en) 2014-03-07 2014-11-25 Viacyte, Inc. 3-dimensional large capacity cell encapsulation device
USD718471S1 (en) 2014-03-07 2014-11-25 Viacyte, Inc. 3-dimensional large capacity cell encapsulation device
USD718466S1 (en) 2014-03-07 2014-11-25 Viacyte, Inc. 3-Dimensional large capacity cell encapsulation device
USD718467S1 (en) 2014-03-07 2014-11-25 Viacyte, Inc. 3-dimensional large capacity cell encapsulation device
USD718468S1 (en) 2014-03-07 2014-11-25 Viacyte, Inc. 3-dimensional large capacity cell encapsulation device
USD718470S1 (en) 2014-03-07 2014-11-25 Viacyte, Inc. 3-dimensional large capacity cell encapsulation device
USD714956S1 (en) 2014-03-07 2014-10-07 Viacyte, Inc. 3-dimensional large capacity cell encapsulation device
RU2752933C2 (ru) 2014-03-11 2021-08-11 Селлектис Способ создания т-клеток, пригодных для аллогенной трансплантации
USD761424S1 (en) 2014-04-16 2016-07-12 Viacyte, Inc. Deployment tool for an encapsulation device
USD761423S1 (en) 2014-04-16 2016-07-12 Viacyte, Inc. Sizing tool for an encapsulation device
USD760399S1 (en) 2014-04-16 2016-06-28 Viacyte, Inc. Case for an encapsulation device
USD734166S1 (en) 2014-04-16 2015-07-14 Viacyte, Inc. Fill pouch assembly for encapsulation device
SG11201609473XA (en) 2014-05-16 2016-12-29 Janssen Biotech Inc Use of small molecules to enhance mafa expression in pancreatic endocrine cells
WO2016073955A2 (en) 2014-11-06 2016-05-12 President And Fellows Of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
CN107250349A (zh) 2014-12-19 2017-10-13 詹森生物科技公司 多能干细胞的悬浮培养
EP3294342A4 (en) * 2015-05-08 2018-11-07 President and Fellows of Harvard College Universal donor stem cells and related methods
EP4339287A3 (en) 2015-07-31 2024-05-22 Regents Of The University Of Minnesota Modified cells and methods of therapy
US12642852B1 (en) * 2015-11-04 2026-06-02 Fate Therapeutics, Inc. Genomic engineering of pluripotent cells
CA3027879A1 (en) 2016-06-21 2017-12-28 Janssen Biotech, Inc. Generation of human pluripotent stem cell derived functional beta cells showing a glucose-dependent mitochondrial respiration and two-phase insulin secretion response
WO2018035387A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
US9982267B2 (en) 2016-10-12 2018-05-29 Feldan Bio Inc. Rationally-designed synthetic peptide shuttle agents for delivering polypeptide cargos from an extracellular space to the cytosol and/or nucleus of a target eukaryotic cell, uses thereof, methods and kits relating to same
EP4595951A3 (en) 2016-11-10 2025-08-27 ViaCyte, Inc. Pdx1 pancreatic endoderm cells in cell delivery devices
KR20240095477A (ko) 2017-01-13 2024-06-25 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 면역조작된 만능 세포
EP3596117A4 (en) 2017-03-17 2021-01-13 The Johns Hopkins University TARGETED EPIGENETIC THERAPY AGAINST THE DISTAL EXPRESSION REGULATORY ELEMENT OF TGFB2
CN110869494B (zh) 2017-06-12 2025-02-18 西奈卫生系统公司 无需全身性免疫遏制的同种异体移植物耐受
US10391156B2 (en) 2017-07-12 2019-08-27 Viacyte, Inc. University donor cells and related methods
JP7621795B2 (ja) 2017-10-19 2025-01-27 セレクティス 改善された免疫細胞療法のためのｎｋ阻害物質遺伝子の標的指向遺伝子組み込み
CN107723275B (zh) 2017-10-20 2020-09-04 重庆精准生物技术有限公司 通用型car-t细胞及其制备方法和应用
SG11202004529XA (en) 2017-11-16 2020-06-29 Mogam Inst Biomedical Res Transformed human cell and use thereof
CN111556892A (zh) 2017-12-08 2020-08-18 菲特治疗公司 使用增强的iPSC衍生的效应细胞的免疫疗法
US20190223416A1 (en) 2018-01-23 2019-07-25 Cotyledon, LLC Canine genome editing
EP3754018A4 (en) 2018-02-16 2021-11-24 Kyoto University METHOD OF MANUFACTURING A WEAK ANTIGENT CELL
CA3120576A1 (en) 2018-11-26 2020-06-04 Massachusetts Institute Of Technology Compositions and methods for immune tolerance
CA3123102A1 (en) 2018-12-13 2020-06-18 Sinai Health System Immunomodulatory cells and uses thereof
WO2020206055A1 (en) 2019-04-02 2020-10-08 Yale University Methods of treatment of diseases and disorders associated with increased tet level, increased h19 level, increased level of tgf signaling, or any combination thereof
US20220213434A1 (en) * 2019-05-10 2022-07-07 The Regents Of The University Of California Modified pluripotent cells
WO2020228039A1 (en) 2019-05-16 2020-11-19 Egenesis, Inc. Cells, tissues, organs, and/or animals having one or more modified genes for enhanced xenograft survival and/or tolerance
CN110373389A (zh) * 2019-06-10 2019-10-25 云南农业大学 一种适合胰岛细胞及皮肤等组织异种移植供体猪的构建方法
US11116798B2 (en) 2019-09-05 2021-09-14 Crispr Therapeutics Ag Universal donor cells
US11104918B2 (en) 2019-09-05 2021-08-31 Crispr Therapeutics Ag Universal donor cells
US11566230B2 (en) 2020-12-31 2023-01-31 Crispr Therapeutics Ag Universal donor cells

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20170274048A1 (en) * 2014-03-28 2017-09-28 Buck Institute For Research On Aging Methods and compositions for modulating the imune system
WO2020049535A1 (en) * 2018-09-07 2020-03-12 Crisper Therapeutics Ag Universal donor cells

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
TRANSGENIC RES, vol. 26, JPN6025049384, 2017, pages 153 - 163, ISSN: 0005897193 *

Also Published As

Publication number Publication date
JP2024503290A (ja) 2024-01-25
US12559726B2 (en) 2026-02-24
US20230127586A1 (en) 2023-04-27
US20220204940A1 (en) 2022-06-30
KR20230146008A (ko) 2023-10-18
CA3203707A1 (en) 2022-07-07
KR20230146007A (ko) 2023-10-18
US20230193209A1 (en) 2023-06-22
US20230323309A1 (en) 2023-10-12
EP4271796A1 (en) 2023-11-08
TW202242094A (zh) 2022-11-01
AU2021414405A1 (en) 2023-08-10
US20220218760A1 (en) 2022-07-14
WO2022144856A1 (en) 2022-07-07
EP4271795A1 (en) 2023-11-08
WO2022144855A1 (en) 2022-07-07
TW202241472A (zh) 2022-11-01
US11578309B2 (en) 2023-02-14
US11566230B2 (en) 2023-01-31
US20230193210A1 (en) 2023-06-22
AU2021414617A1 (en) 2023-08-10
CA3203392A1 (en) 2022-07-07

Similar Documents

Publication Publication Date Title
US12577582B2 (en) Universal donor cells
US11434505B2 (en) Universal donor cells
US11433103B2 (en) Universal donor cells
US12559726B2 (en) Universal donor cells
HK40114886A (en) Universal donor cells

Legal Events

Date Code Title Description
RD03 Notification of appointment of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7423

Effective date: 20230929

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20231005

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20241227

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20241227

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20251202

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20260227

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20260521

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20260630