WO2022129472A1 - Safe immuno-stealth cells - Google Patents

Safe immuno-stealth cells Download PDF

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Publication number
WO2022129472A1
WO2022129472A1 PCT/EP2021/086399 EP2021086399W WO2022129472A1 WO 2022129472 A1 WO2022129472 A1 WO 2022129472A1 EP 2021086399 W EP2021086399 W EP 2021086399W WO 2022129472 A1 WO2022129472 A1 WO 2022129472A1
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WO
WIPO (PCT)
Prior art keywords
cell
hla
gene
genes
hsv
Prior art date
Application number
PCT/EP2021/086399
Other languages
English (en)
French (fr)
Inventor
Jay Chaplin
Ulrik DØHN
Thomas FROGNE
Original Assignee
Novo Nordisk A/S
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 Novo Nordisk A/S filed Critical Novo Nordisk A/S
Publication of WO2022129472A1 publication Critical patent/WO2022129472A1/en

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    • 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
    • C12N5/0603Embryonic cells ; Embryoid bodies
    • C12N5/0606Pluripotent embryonic cells, e.g. embryonic stem cells [ES]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/48Reproductive organs
    • A61K35/54Ovaries; Ova; Ovules; Embryos; Foetal cells; Germ cells
    • A61K35/545Embryonic stem cells; Pluripotent stem cells; Induced pluripotent stem cells; Uncharacterised stem 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
    • C12N2510/00Genetically modified cells

Definitions

  • B2M/HLA-E fusion gene such as a B2M/HLA-E*0101 gene and/or a B2M/HLA-E*0103 gene, into said mammalian cell
  • the expression "at distinct locations” as used herein means "at different loci on the genome”.
  • the expression refers for example to more than one nucleic acid sequence insertion, where said 2 or more nucleic acid sequences are not inserted on the same locus on the genome, i.e. on the one same position on the genome. Rather, said 2 or more nucleic acid sequences are inserted at different loci on the genome. For example, if inserted on the same chromosome, the 2 or more sequences are separated from each other by a number of nucleotides after insertion.
  • the expression "distinct locations” may include the same locus located on 2 chromosomes of a pair of chromosomes.
  • HLA-II deficient cell means a cell which comprises no HLA-II protein on its cell surface.
  • the absence of HLA-II proteins on the cell surface may result from the absence of any expressible HLA-II gene in the cell, e.g. due to inactivation of all HLA-II genes.
  • the absence of HLA-II proteins on the cell surface may result from the cell being CIITA deficient.
  • HSV-TK gene and TK-sr39 :
  • the present invention provides a mammalian cell which has knock-ins of both B2M/HLA-E*0101 and B2M/HLA-E*0103 genes into an otherwise B2M deficient cell.
  • Said differentiated cell may be derived from a stem cell, a pluripotential cell or an iPS cell of the invention according to one of the differentiation methods described in the publications referred to in the below list:
  • a clone containing four HSV-TK copies from the protocol above is electroporated with a total of 200ng TALEN® mRNA pair (ThermoFisher®, forward target sequence: TCTCGCTCCGTGGCCTT (SEQ ID NO 15), reverse target sequence: AGCCTCCAGGCCAGAAAG (SEQ ID NO 16)) against B2M and 200ng donor plasmid containing 300bp homology arms flanking the TALEN® cut site in B2M, a B2M- HLAIE0101 fusion cassette followed by a mCherry selection cassette and 200ng donor plasmid containing 300bp homology arms flanking the TALEN® cut site in B2M, a B2M-HLAIE0103 fusion cassette followed by a eGFP selection cassette.
  • TALEN® mRNA pair ThermoFisher®, forward target sequence: TCTCGCTCCGTGGCCTT (SEQ ID NO 15), reverse target sequence: AGCCTCCAGGCCAGAAAG

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Developmental Biology & Embryology (AREA)
  • Engineering & Computer Science (AREA)
  • Cell Biology (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Reproductive Health (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Gynecology & Obstetrics (AREA)
  • General Health & Medical Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Immunology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
PCT/EP2021/086399 2020-12-18 2021-12-17 Safe immuno-stealth cells WO2022129472A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP20215272 2020-12-18
EP20215272.4 2020-12-18

Publications (1)

Publication Number Publication Date
WO2022129472A1 true WO2022129472A1 (en) 2022-06-23

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2021/086399 WO2022129472A1 (en) 2020-12-18 2021-12-17 Safe immuno-stealth cells

Country Status (3)

Country Link
AR (1) AR124419A1 (zh)
TW (1) TW202242095A (zh)
WO (1) WO2022129472A1 (zh)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024008979A1 (en) 2022-09-30 2024-01-11 Novo Nordisk A/S A sirp-alpha binding chimeric protein

Citations (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003046141A2 (en) 2001-11-26 2003-06-05 Advanced Cell Technology, Inc. Methods for making and using reprogrammed human somatic cell nuclei and autologous and isogenic human stem cells
WO2003055992A2 (en) 2001-12-28 2003-07-10 Cellartis Ab A method for the establishment of a pluripotent human blastocyst-derived stem cell line
US20040225112A1 (en) 2003-05-06 2004-11-11 Crew Mark D. Genes encoding single chain human leukocyte antigen E (HLA-E) proteins to prevent natural killer cell-mediated cytotoxicity
WO2007042225A2 (en) 2005-10-07 2007-04-19 Cellartis Ab A method for obtaining a xeno-free hbs cell line
WO2012145384A1 (en) 2011-04-20 2012-10-26 University Of Washington Through Its Center For Commercialization Beta-2 microglobulin-deficient cells
US8586358B2 (en) 2007-03-09 2013-11-19 University Of Washington HLA homozygous cells
WO2015028614A1 (en) 2013-08-30 2015-03-05 Novo Nordisk A/S Generation of endocrine progenitor cells from human pluripotent stem cells using small molecules
WO2017079673A1 (en) * 2015-11-04 2017-05-11 Fate Therapeutics, Inc. Genomic engineering of pluripotent cells
WO2017144695A1 (en) 2016-02-24 2017-08-31 Novo Nordisk A/S Generation of functional beta cells from human pluripotent stem cell-derived endocrine progenitors
WO2018005556A1 (en) 2016-06-27 2018-01-04 Juno Therapeutics, Inc. Mhc-e restricted epitopes, binding molecules and related methods and uses
WO2019032675A1 (en) 2017-08-08 2019-02-14 Sangamo Therapeutics, Inc. CELL TARGETING MEDIATED BY A CHIMERIC ANTIGEN RECEPTOR
WO2019241400A1 (en) * 2018-06-12 2019-12-19 The Regents Of The University Of California Stem cell-engineered inkt cell-based off -the-shelf cellular therapy
WO2020012033A1 (en) * 2018-07-13 2020-01-16 Lothar Germeroth Non-immunogenic engineered tissue and methods of producing and using the same
WO2020072390A1 (en) * 2018-10-01 2020-04-09 Caribou Biosciences, Inc. Suicide module compositions and methods

Patent Citations (14)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003046141A2 (en) 2001-11-26 2003-06-05 Advanced Cell Technology, Inc. Methods for making and using reprogrammed human somatic cell nuclei and autologous and isogenic human stem cells
WO2003055992A2 (en) 2001-12-28 2003-07-10 Cellartis Ab A method for the establishment of a pluripotent human blastocyst-derived stem cell line
US20040225112A1 (en) 2003-05-06 2004-11-11 Crew Mark D. Genes encoding single chain human leukocyte antigen E (HLA-E) proteins to prevent natural killer cell-mediated cytotoxicity
WO2007042225A2 (en) 2005-10-07 2007-04-19 Cellartis Ab A method for obtaining a xeno-free hbs cell line
US8586358B2 (en) 2007-03-09 2013-11-19 University Of Washington HLA homozygous cells
WO2012145384A1 (en) 2011-04-20 2012-10-26 University Of Washington Through Its Center For Commercialization Beta-2 microglobulin-deficient cells
WO2015028614A1 (en) 2013-08-30 2015-03-05 Novo Nordisk A/S Generation of endocrine progenitor cells from human pluripotent stem cells using small molecules
WO2017079673A1 (en) * 2015-11-04 2017-05-11 Fate Therapeutics, Inc. Genomic engineering of pluripotent cells
WO2017144695A1 (en) 2016-02-24 2017-08-31 Novo Nordisk A/S Generation of functional beta cells from human pluripotent stem cell-derived endocrine progenitors
WO2018005556A1 (en) 2016-06-27 2018-01-04 Juno Therapeutics, Inc. Mhc-e restricted epitopes, binding molecules and related methods and uses
WO2019032675A1 (en) 2017-08-08 2019-02-14 Sangamo Therapeutics, Inc. CELL TARGETING MEDIATED BY A CHIMERIC ANTIGEN RECEPTOR
WO2019241400A1 (en) * 2018-06-12 2019-12-19 The Regents Of The University Of California Stem cell-engineered inkt cell-based off -the-shelf cellular therapy
WO2020012033A1 (en) * 2018-07-13 2020-01-16 Lothar Germeroth Non-immunogenic engineered tissue and methods of producing and using the same
WO2020072390A1 (en) * 2018-10-01 2020-04-09 Caribou Biosciences, Inc. Suicide module compositions and methods

Non-Patent Citations (27)

* Cited by examiner, † Cited by third party
Title
ACKERMANN M. ET AL., NAT COMMUN., vol. 9, no. 1, 30 November 2018 (2018-11-30), pages 5088
AKTINSON-DELL R. ET AL., ADV EXP MED BIOL., vol. 1175, 2019, pages 383 - 405
BEN M'BAREK K ET AL., BIOMATERIALS, 6 November 2019 (2019-11-06), pages 119603
BLACK ET AL., CANCER RESEARCH, vol. 61, 1 April 2001 (2001-04-01), pages 3022 - 3026
CANDELARIO K.M ET AL., J COMP NEUROL., 19 November 2019 (2019-11-19)
CHEN B, STEM CELL RES THER., vol. 10, no. 1, 21 May 2019 (2019-05-21), pages 142
CHEN KH, AM J TRANSL RES., vol. 11, no. 9, 15 September 2019 (2019-09-15), pages 6232 - 6248
COLL M., CELL STEM CELL, vol. 23, no. 1, 5 July 2018 (2018-07-05), pages 101 - 113
DEUSE ET AL., NATURE BIOTECHNOLOGY, 2019
DOUGHERTY J.A. ET AL., FRONT PHYSIOL, vol. 9, 14 December 2018 (2018-12-14), pages 1794
GOOD ML. ET AL., J VIS EXP., no. 152, 24 October 2019 (2019-10-24)
GORNALUSSE G. ET AL., NATURE BIOTECHNOLOGY, 2017
HUANG CY ET AL., J MOL CELL CARDIOL., vol. 138, 23 October 2019 (2019-10-23), pages 1 - 11
JEONG M. ET AL., CELL DEATH DIS., vol. 9, no. 9, 11 September 2018 (2018-09-11), pages 922
KIRKEBY A. ET AL., CELL REP., vol. 1, no. 6, 28 June 2012 (2012-06-28), pages 703 - 14
KITADANI J. ET AL., SCI REP., vol. 8, no. 1, 15 March 2018 (2018-03-15), pages 4569
LEES EA ET AL., J VIS EXP., 12 May 2019 (2019-05-12), pages 147
LI Z. ET AL., CELL DEATH DIS, vol. 10, no. 10, 10 October 2019 (2019-10-10), pages 763
MIYAKE T, INT J RADIAT ONCOL BIOL PHYS., vol. 105, no. 1, 1 September 2019 (2019-09-01), pages 193 - 205
NEGORO R. ET AL., STEM CELL REPORTS, vol. 11, no. 6, 11 December 2018 (2018-12-11), pages 1539 - 1550
NI P. ET AL., MOL THER METHODS CLIN DEV., vol. 13, 8 April 2019 (2019-04-08), pages 414 - 430
NOLBRANT S. ET AL., NAT. PROTOC., no. 9, 12 September 2017 (2017-09-12), pages 1962 - 1979
SUN X. ET AL., FRONT CELL NEUROSCI., vol. 13, 3 September 2019 (2019-09-03), pages 394
VANSLAMBROUCK JM ET AL., J AM SOC NEPHROL., no. 10, 30 October 2019 (2019-10-30), pages 1811 - 1823
YANG R. ET AL., FRONT IMMUNOL., vol. 10, 16 October 2019 (2019-10-16), pages 2346
YOUNG ET AL., CANCER GEN. THERAPY, vol. 7, 2000, pages 240 - 246
ZHU H. ET AL., METHODS MOL BIOL., vol. 2048, 2019, pages 107 - 119

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024008979A1 (en) 2022-09-30 2024-01-11 Novo Nordisk A/S A sirp-alpha binding chimeric protein

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TW202242095A (zh) 2022-11-01
AR124419A1 (es) 2023-03-29

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