EP3924375A4 - Universalspenderstammzellen und zugehörige verfahren - Google Patents

Universalspenderstammzellen und zugehörige verfahren Download PDF

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Publication number
EP3924375A4
EP3924375A4 EP20755906.3A EP20755906A EP3924375A4 EP 3924375 A4 EP3924375 A4 EP 3924375A4 EP 20755906 A EP20755906 A EP 20755906A EP 3924375 A4 EP3924375 A4 EP 3924375A4
Authority
EP
European Patent Office
Prior art keywords
stem cells
related methods
universal donor
donor stem
universal
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.)
Withdrawn
Application number
EP20755906.3A
Other languages
English (en)
French (fr)
Other versions
EP3924375A2 (de
Inventor
Torsten B. Meissner
Leonardo M.R. FERREIRA
Jack L. Strominger
Chad A. COWAN
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.)
Harvard College
Original Assignee
Harvard College
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 Harvard College filed Critical Harvard College
Publication of EP3924375A2 publication Critical patent/EP3924375A2/de
Publication of EP3924375A4 publication Critical patent/EP3924375A4/de
Withdrawn legal-status Critical Current

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    • 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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • C12N15/1138—Non-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 against receptors or cell surface proteins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—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
    • A61K2035/122—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells for inducing tolerance or supression of immune responses
    • 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 [CRISPRs]
    • 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
    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/50—Cell markers; Cell surface determinants
    • C12N2501/599—Cell markers; Cell surface determinants with CD designations not provided for elsewhere
    • 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
    • C12N2502/00—Coculture with; Conditioned medium produced by
    • C12N2502/11—Coculture with; Conditioned medium produced by blood or immune system cells
    • C12N2502/1114—T 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)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Reproductive Health (AREA)
  • Gynecology & Obstetrics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Cell Biology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
EP20755906.3A 2019-02-15 2020-02-16 Universalspenderstammzellen und zugehörige verfahren Withdrawn EP3924375A4 (de)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201916277913A 2019-02-15 2019-02-15
US201916596697A 2019-10-08 2019-10-08
PCT/US2020/018467 WO2020168317A2 (en) 2019-02-15 2020-02-16 Universal donor stem cells and related methods

Publications (2)

Publication Number Publication Date
EP3924375A2 EP3924375A2 (de) 2021-12-22
EP3924375A4 true EP3924375A4 (de) 2023-03-22

Family

ID=72045137

Family Applications (1)

Application Number Title Priority Date Filing Date
EP20755906.3A Withdrawn EP3924375A4 (de) 2019-02-15 2020-02-16 Universalspenderstammzellen und zugehörige verfahren

Country Status (11)

Country Link
EP (1) EP3924375A4 (de)
JP (1) JP2022526218A (de)
KR (1) KR20210128440A (de)
CN (1) CN113906048A (de)
AU (1) AU2020223192A1 (de)
BR (1) BR112021016178A2 (de)
CA (1) CA3130398A1 (de)
IL (1) IL285619A (de)
MX (1) MX2021009842A (de)
SG (1) SG11202108891QA (de)
WO (1) WO2020168317A2 (de)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20230338533A1 (en) * 2020-05-15 2023-10-26 Rxcell Inc. Hypoimmunogenic Cells and Uses Thereof in Immune Responses
CN114457021A (zh) * 2020-10-30 2022-05-10 未来智人再生医学研究院(广州)有限公司 一种表达cd47抗体的多能干细胞及其衍生物与应用
CN114525256A (zh) * 2020-10-30 2022-05-24 未来智人再生医学研究院(广州)有限公司 一种表达Siglec-15阻断物的多能干细胞或其衍生物及应用
TW202237826A (zh) 2020-11-30 2022-10-01 瑞士商克里斯珀醫療股份公司 基因編輯的自然殺手細胞
JP2023552816A (ja) * 2020-12-11 2023-12-19 インテリア セラピューティクス，インコーポレイテッド 細胞内のｍｈｃクラスｉｉを低減する組成物及び方法
AU2021410751A1 (en) * 2020-12-23 2023-07-13 Intellia Therapeutics, Inc. Compositions and methods for genetically modifying ciita in a cell
JP2024501971A (ja) 2020-12-31 2024-01-17 サナ バイオテクノロジー，インコーポレイテッド Ｃａｒ－ｔ活性を調節するための方法及び組成物
AU2022283291A1 (en) 2021-05-27 2023-11-02 Sana Biotechnology, Inc. Hypoimmunogenic cells comprising engineered hla-e or hla-g
IL310702A (en) 2021-08-11 2024-04-01 Sana Biotechnology Inc Inducible systems for altering gene expression in hypoimmunogenic cells
WO2023122337A1 (en) 2021-12-23 2023-06-29 Sana Biotechnology, Inc. Chimeric antigen receptor (car) t cells for treating autoimmune disease and associated methods
WO2023154578A1 (en) 2022-02-14 2023-08-17 Sana Biotechnology, Inc. Methods of treating patients exhibiting a prior failed therapy with hypoimmunogenic cells
WO2023191063A1 (ja) * 2022-04-01 2023-10-05 株式会社Logomix 遺伝子工学、細胞工学、および細胞医薬に適した細胞およびその製造方法
WO2024003349A1 (en) 2022-07-01 2024-01-04 Novo Nordisk A/S Enhancing neuronal differentiation of ventral midbrain neural progenitor cells

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017079673A1 (en) * 2015-11-04 2017-05-11 Fate Therapeutics, Inc. Genomic engineering of pluripotent cells

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3294342A4 (de) * 2015-05-08 2018-11-07 President and Fellows of Harvard College Universalspenderstammzellen und zugehörige verfahren
BR112019014257A2 (pt) * 2017-01-13 2020-04-28 Univ California método para gerar uma célula-tronco pluripotente hipoimunogênica, célula-tronco pluripotente hipoimunogênica humana, método para produzir uma célula pluripotente hipoimunogênica, e ss-2 microglobulina

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017079673A1 (en) * 2015-11-04 2017-05-11 Fate Therapeutics, Inc. Genomic engineering of pluripotent cells

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
XIAO HAN ET AL: "Generation of hypoimmunogenic human pluripotent stem cells", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES, vol. 116, no. 21, 30 April 2019 (2019-04-30), pages 10441 - 10446, XP055640699, ISSN: 0027-8424, DOI: 10.1073/pnas.1902566116 *

Also Published As

Publication number Publication date
JP2022526218A (ja) 2022-05-24
EP3924375A2 (de) 2021-12-22
WO2020168317A8 (en) 2021-02-25
IL285619A (en) 2021-09-30
KR20210128440A (ko) 2021-10-26
WO2020168317A2 (en) 2020-08-20
CN113906048A (zh) 2022-01-07
SG11202108891QA (en) 2021-09-29
AU2020223192A1 (en) 2021-09-09
MX2021009842A (es) 2021-12-10
BR112021016178A2 (pt) 2021-11-03
WO2020168317A3 (en) 2020-10-29
CA3130398A1 (en) 2020-08-20

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