WO2020168317A8 - Cellules souches donnatrices universelles et méthodes associées - Google Patents

Cellules souches donnatrices universelles et méthodes associées Download PDF

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Publication number
WO2020168317A8
WO2020168317A8 PCT/US2020/018467 US2020018467W WO2020168317A8 WO 2020168317 A8 WO2020168317 A8 WO 2020168317A8 US 2020018467 W US2020018467 W US 2020018467W WO 2020168317 A8 WO2020168317 A8 WO 2020168317A8
Authority
WO
WIPO (PCT)
Prior art keywords
stem cells
universal donor
donor stem
related methods
disclosed
Prior art date
Application number
PCT/US2020/018467
Other languages
English (en)
Other versions
WO2020168317A2 (fr
WO2020168317A3 (fr
Inventor
Torsten B. Meissner
Leonardo M.R. FERREIRA
Jack L. Strominger
Chad A. COWAN
Original Assignee
President And Fellows Of 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
Priority to MX2021009842A priority Critical patent/MX2021009842A/es
Priority to KR1020217029445A priority patent/KR20210128440A/ko
Priority to EA202192264A priority patent/EA202192264A1/ru
Priority to BR112021016178A priority patent/BR112021016178A2/pt
Priority to JP2021547755A priority patent/JP2022526218A/ja
Priority to EP20755906.3A priority patent/EP3924375A4/fr
Priority to CN202080021860.7A priority patent/CN113906048A/zh
Priority to CA3130398A priority patent/CA3130398A1/fr
Application filed by President And Fellows Of Harvard College filed Critical President And Fellows Of Harvard College
Priority to AU2020223192A priority patent/AU2020223192A1/en
Priority to SG11202108891QA priority patent/SG11202108891QA/en
Publication of WO2020168317A2 publication Critical patent/WO2020168317A2/fr
Publication of WO2020168317A3 publication Critical patent/WO2020168317A3/fr
Publication of WO2020168317A8 publication Critical patent/WO2020168317A8/fr
Priority to IL285619A priority patent/IL285619A/en

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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
    • 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
    • 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
    • C12N15/10—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)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Developmental Biology & Embryology (AREA)
  • Microbiology (AREA)
  • Reproductive Health (AREA)
  • Biochemistry (AREA)
  • Gynecology & Obstetrics (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

L'invention concerne des cellules souches donnatrices universelles et leurs méthodes associées d'utilisation et de production. Les cellules souches donnatrices universelles selon l'invention sont utiles pour surmonter le rejet immunitaire dans des thérapies de transplantation à base de cellules. Selon certains modes de réalisation, les cellules souches donnatrices universelles révélées ici ont une expression modulée d'un ou de plusieurs antigènes leucocytaires humains MHC-I et MHC-II et d'un ou de plusieurs facteurs tolérogéniques.
PCT/US2020/018467 2019-02-15 2020-02-16 Cellules souches donnatrices universelles et méthodes associées WO2020168317A2 (fr)

Priority Applications (11)

Application Number Priority Date Filing Date Title
CN202080021860.7A CN113906048A (zh) 2019-02-15 2020-02-16 通用供体干细胞和相关方法
EA202192264A EA202192264A1 (ru) 2019-10-08 2020-02-16 Универсальные донорские стволовые клетки и соответствующие способы
BR112021016178A BR112021016178A2 (pt) 2019-02-15 2020-02-16 Células-tronco doadoras universais e métodos relacionados
JP2021547755A JP2022526218A (ja) 2019-02-15 2020-02-16 汎用ドナー幹細胞及び関連する方法
EP20755906.3A EP3924375A4 (fr) 2019-02-15 2020-02-16 Cellules souches donnatrices universelles et méthodes associées
MX2021009842A MX2021009842A (es) 2019-02-15 2020-02-16 Células madre de donantes universales y métodos relacionados.
CA3130398A CA3130398A1 (fr) 2019-02-15 2020-02-16 Cellules souches donnatrices universelles et methodes associees
KR1020217029445A KR20210128440A (ko) 2019-02-15 2020-02-16 범용 공여자 줄기 세포 및 관련 방법
AU2020223192A AU2020223192A1 (en) 2019-02-15 2020-02-16 Universal donor stem cells and related methods
SG11202108891QA SG11202108891QA (en) 2019-02-15 2020-02-16 Universal donor stem cells and related methods
IL285619A IL285619A (en) 2019-02-15 2021-08-15 Universal donor stem cells and related methods

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201916277913A 2019-02-15 2019-02-15
US16/277,913 2019-02-15
US201916596697A 2019-10-08 2019-10-08
US16/596,697 2019-10-08

Publications (3)

Publication Number Publication Date
WO2020168317A2 WO2020168317A2 (fr) 2020-08-20
WO2020168317A3 WO2020168317A3 (fr) 2020-10-29
WO2020168317A8 true WO2020168317A8 (fr) 2021-02-25

Family

ID=72045137

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2020/018467 WO2020168317A2 (fr) 2019-02-15 2020-02-16 Cellules souches donnatrices universelles et méthodes associées

Country Status (11)

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

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4149525A1 (fr) * 2020-05-15 2023-03-22 Rxcell Inc. Cellules hypo-immunogènes et leurs utilisations dans des réponses immunitaires
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
US11459372B2 (en) 2020-11-30 2022-10-04 Crispr Therapeutics Ag Gene-edited natural killer cells
AU2021396403A1 (en) * 2020-12-11 2023-06-29 Intellia Therapeutics, Inc. Compositions and methods for reducing mhc class ii in a cell
WO2022140587A1 (fr) * 2020-12-23 2022-06-30 Intellia Therapeutics, Inc. Compositions et procédés pour modifier génétiquement le ciita dans une cellule
CA3200509A1 (fr) 2020-12-31 2022-07-07 Sonja SCHREPFER Methodes et compositions pour moduler une activite de car-t
AU2022283291A1 (en) 2021-05-27 2023-11-02 Sana Biotechnology, Inc. Hypoimmunogenic cells comprising engineered hla-e or hla-g
CA3227613A1 (fr) 2021-08-11 2023-02-16 William Dowdle Systemes inductibles pour modifier l'expression genique dans des cellules hypoimmunogenes
WO2023122337A1 (fr) 2021-12-23 2023-06-29 Sana Biotechnology, Inc. Lymphocytes t à récepteur antigénique chimérique (car) pour le traitement d'une maladie auto-immune et méthodes associées
WO2023154578A1 (fr) 2022-02-14 2023-08-17 Sana Biotechnology, Inc. Méthodes de traitement de patients présentant une thérapie préalable ayant échoué avec des cellules hypoimmunogènes
WO2023191063A1 (fr) * 2022-04-01 2023-10-05 株式会社Logomix Cellule appropriée pour l'ingénierie génique, l'ingénierie cellulaire et la médecine cellulaire, et son procédé de production
WO2024003349A1 (fr) 2022-07-01 2024-01-04 Novo Nordisk A/S Amélioration de la différenciation neuronale de cellules progénitrices neurales du mésencéphale ventral

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3294342A4 (fr) * 2015-05-08 2018-11-07 President and Fellows of Harvard College Cellules souches de donneur universel et procédés associés
CN108368520B (zh) * 2015-11-04 2023-01-17 菲特治疗公司 多能细胞的基因组工程改造
KR20190103373A (ko) * 2017-01-13 2019-09-04 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 면역조작된 만능 세포

Also Published As

Publication number Publication date
IL285619A (en) 2021-09-30
KR20210128440A (ko) 2021-10-26
JP2022526218A (ja) 2022-05-24
SG11202108891QA (en) 2021-09-29
CA3130398A1 (fr) 2020-08-20
EP3924375A2 (fr) 2021-12-22
AU2020223192A1 (en) 2021-09-09
CN113906048A (zh) 2022-01-07
WO2020168317A2 (fr) 2020-08-20
MX2021009842A (es) 2021-12-10
EP3924375A4 (fr) 2023-03-22
WO2020168317A3 (fr) 2020-10-29
BR112021016178A2 (pt) 2021-11-03

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