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WO2024196669A3 - Methods for in vivo editing of b cells - Google Patents

Methods for in vivo editing of b cells Download PDF

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Publication number
WO2024196669A3
WO2024196669A3 PCT/US2024/019736 US2024019736W WO2024196669A3 WO 2024196669 A3 WO2024196669 A3 WO 2024196669A3 US 2024019736 W US2024019736 W US 2024019736W WO 2024196669 A3 WO2024196669 A3 WO 2024196669A3
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WO
WIPO (PCT)
Prior art keywords
vivo
cells
methods
specific cell
vivo editing
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
PCT/US2024/019736
Other languages
French (fr)
Other versions
WO2024196669A2 (en
Inventor
Mark Selby
Hangil Park
Rosa Romano
David T. Curiel
Paul Boucher
Zhi Hong LU
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.)
Washington University in St Louis WUSTL
Walking Fish Therapeutics Inc
Original Assignee
Washington University in St Louis WUSTL
Walking Fish Therapeutics Inc
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Filing date
Publication date
Application filed by Washington University in St Louis WUSTL, Walking Fish Therapeutics Inc filed Critical Washington University in St Louis WUSTL
Publication of WO2024196669A2 publication Critical patent/WO2024196669A2/en
Publication of WO2024196669A3 publication Critical patent/WO2024196669A3/en
Anticipated expiration legal-status Critical
Pending legal-status Critical Current

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    • 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
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2803—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2878—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the NGF-receptor/TNF-receptor superfamily, e.g. CD27, CD30, CD40, CD95
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2887—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against CD20
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • 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
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0635—B lymphocytes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0636—T lymphocytes
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/08—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from viruses
    • C07K16/10—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from viruses from RNA viruses
    • C07K16/1036—Retroviridae, e.g. leukemia viruses
    • C07K16/1045—Lentiviridae, e.g. HIV, FIV, SIV
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
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    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/56—Immunoglobulins specific features characterized by immunoglobulin fragments variable (Fv) region, i.e. VH and/or VL
    • C07K2317/569—Single domain, e.g. dAb, sdAb, VHH, VNAR or nanobody®
    • C—CHEMISTRY; METALLURGY
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    • C12N2501/00—Active agents used in cell culture processes, e.g. differentation
    • C12N2501/20—Cytokines; Chemokines
    • C12N2501/23—Interleukins [IL]
    • C12N2501/2304—Interleukin-4 (IL-4)
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    • C12N2510/00—Genetically modified cells
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    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10322—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10345—Special targeting system for viral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/80—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates
    • C12N2810/85—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian
    • C12N2810/859—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian from immunoglobulins

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  • Health & Medical Sciences (AREA)
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  • Chemical & Material Sciences (AREA)
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  • Engineering & Computer Science (AREA)
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  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
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  • Zoology (AREA)
  • Biochemistry (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Cell Biology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Hematology (AREA)
  • Plant Pathology (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Mycology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Disclosed herein are novel targeting technologies to deliver gene therapies to specific cell types both in vivo and ex vivo, comprising a system that includes an adenoviral particle modified to incorporate one or more peptide tags into one or more components of the capsid protein, and a recombinant protein that comprises a first portion capable of forming a high affinity bond with said peptide tag, and a second portion comprising a ligand or antigen binding protein capable of targeting a specific cell type (for example, a B cell).
PCT/US2024/019736 2023-03-17 2024-03-13 Methods for in vivo editing of b cells Pending WO2024196669A2 (en)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US202363452960P 2023-03-17 2023-03-17
US63/452,960 2023-03-17
US202363495050P 2023-04-07 2023-04-07
US63/495,050 2023-04-07
US202363510822P 2023-06-28 2023-06-28
US63/510,822 2023-06-28

Publications (2)

Publication Number Publication Date
WO2024196669A2 WO2024196669A2 (en) 2024-09-26
WO2024196669A3 true WO2024196669A3 (en) 2024-11-21

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ID=92842234

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2024/019736 Pending WO2024196669A2 (en) 2023-03-17 2024-03-13 Methods for in vivo editing of b cells

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Citations (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011038290A2 (en) * 2009-09-25 2011-03-31 The U. S. A., As Represented By The Secretary, Department Of Health And Human Services Neutralizing antibodies to hiv-1 and their use
US20180153946A1 (en) * 2015-05-04 2018-06-07 Vcn Biosciences Sl Oncolytic adenoviruses with mutations in immunodominant adenovirus epitopes and their use in cancer treatment
US20180362975A1 (en) * 2015-12-04 2018-12-20 Novartis Ag Compositions and methods for immunooncology
US20190178878A1 (en) * 2010-02-11 2019-06-13 Oxford University Innovation Limited Peptide tag systems that spontaneously form an irreversible link to protein partners via isopeptide bonds
US20190352614A1 (en) * 2017-01-26 2019-11-21 Sangamo Therapeutics, Inc. B-cell engineering
US20200354782A1 (en) * 2017-11-21 2020-11-12 Expansion Technologies Expansion microscopy compatible and multiplexed in situ hybridization of formalin fixed paraffin embedded tissue sections for spatially resolved transcriptomics
US20210163894A1 (en) * 2017-07-21 2021-06-03 Washington University Methods and compositions for t cell activation
WO2021202810A2 (en) * 2020-03-31 2021-10-07 Walking Fish Therapeutics Modified b cells and methods of use thereof
US20220168342A1 (en) * 2016-09-12 2022-06-02 Regents Of The University Of Minnesota Genome edited primary b cell and methods of making and using
WO2022226020A2 (en) * 2021-04-20 2022-10-27 Walking Fish Therapeutics Engineering b cell-based protein factories to treat serious diseases
US20220372514A1 (en) * 2019-11-01 2022-11-24 SpyBiotech Limited Viruses with modified capsid proteins

Patent Citations (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011038290A2 (en) * 2009-09-25 2011-03-31 The U. S. A., As Represented By The Secretary, Department Of Health And Human Services Neutralizing antibodies to hiv-1 and their use
US20190178878A1 (en) * 2010-02-11 2019-06-13 Oxford University Innovation Limited Peptide tag systems that spontaneously form an irreversible link to protein partners via isopeptide bonds
US20180153946A1 (en) * 2015-05-04 2018-06-07 Vcn Biosciences Sl Oncolytic adenoviruses with mutations in immunodominant adenovirus epitopes and their use in cancer treatment
US20180362975A1 (en) * 2015-12-04 2018-12-20 Novartis Ag Compositions and methods for immunooncology
US20220168342A1 (en) * 2016-09-12 2022-06-02 Regents Of The University Of Minnesota Genome edited primary b cell and methods of making and using
US20190352614A1 (en) * 2017-01-26 2019-11-21 Sangamo Therapeutics, Inc. B-cell engineering
US20210163894A1 (en) * 2017-07-21 2021-06-03 Washington University Methods and compositions for t cell activation
US20200354782A1 (en) * 2017-11-21 2020-11-12 Expansion Technologies Expansion microscopy compatible and multiplexed in situ hybridization of formalin fixed paraffin embedded tissue sections for spatially resolved transcriptomics
US20220372514A1 (en) * 2019-11-01 2022-11-24 SpyBiotech Limited Viruses with modified capsid proteins
WO2021202810A2 (en) * 2020-03-31 2021-10-07 Walking Fish Therapeutics Modified b cells and methods of use thereof
WO2022226020A2 (en) * 2021-04-20 2022-10-27 Walking Fish Therapeutics Engineering b cell-based protein factories to treat serious diseases

Also Published As

Publication number Publication date
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