WO2023250433A3 - Engineered human t cells comprising a switchable chimeric antigen cell surface receptor and methods for generating them - Google Patents

Engineered human t cells comprising a switchable chimeric antigen cell surface receptor and methods for generating them Download PDF

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Publication number
WO2023250433A3
WO2023250433A3 PCT/US2023/068901 US2023068901W WO2023250433A3 WO 2023250433 A3 WO2023250433 A3 WO 2023250433A3 US 2023068901 W US2023068901 W US 2023068901W WO 2023250433 A3 WO2023250433 A3 WO 2023250433A3
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WO
WIPO (PCT)
Prior art keywords
engineered human
generating
chimeric antigen
surface receptor
cell surface
Prior art date
Application number
PCT/US2023/068901
Other languages
French (fr)
Other versions
WO2023250433A2 (en
Inventor
Surbhi GOEL
Reynald Michael LESCARBEAU
Yong Zhang
Srijani SRIDHAR
William Frederick HARRINGTON
Armin Ehninger
Marc CARTELLIERI
Gerhard Ehninger
Andrew Schiermeier
Original Assignee
Goel Surbhi
Lescarbeau Reynald Michael
Yong Zhang
Sridhar Srijani
Harrington William Frederick
Armin Ehninger
Cartellieri Marc
Gerhard Ehninger
Andrew Schiermeier
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 claimed from EP22180820.7A external-priority patent/EP4295860A1/en
Application filed by Goel Surbhi, Lescarbeau Reynald Michael, Yong Zhang, Sridhar Srijani, Harrington William Frederick, Armin Ehninger, Cartellieri Marc, Gerhard Ehninger, Andrew Schiermeier filed Critical Goel Surbhi
Publication of WO2023250433A2 publication Critical patent/WO2023250433A2/en
Publication of WO2023250433A3 publication Critical patent/WO2023250433A3/en

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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/46—Cellular immunotherapy
    • A61K39/461—Cellular immunotherapy characterised by the cell type used
    • A61K39/4611—T-cells, e.g. tumor infiltrating lymphocytes [TIL], lymphokine-activated killer cells [LAK] or regulatory T cells [Treg]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/46—Cellular immunotherapy
    • A61K39/463—Cellular immunotherapy characterised by recombinant expression
    • A61K39/4631—Chimeric Antigen Receptors [CAR]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K39/46—Cellular immunotherapy
    • A61K39/464—Cellular immunotherapy characterised by the antigen targeted or presented
    • A61K39/4643—Vertebrate antigens
    • A61K39/4644—Cancer antigens
    • A61K39/464402—Receptors, cell surface antigens or cell surface determinants
    • A61K39/464416—Receptors for cytokines
    • A61K39/464419—Receptors for interleukins [IL]
    • 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
    • C07K14/7051—T-cell receptor (TcR)-CD3 complex
    • 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
    • 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
    • C07K16/2866—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against receptors for cytokines, lymphokines, interferons
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K39/46
    • A61K2239/10—Indexing codes associated with cellular immunotherapy of group A61K39/46 characterized by the structure of the chimeric antigen receptor [CAR]
    • A61K2239/11—Antigen recognition domain
    • A61K2239/15—Non-antibody based
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K39/46
    • A61K2239/10—Indexing codes associated with cellular immunotherapy of group A61K39/46 characterized by the structure of the chimeric antigen receptor [CAR]
    • A61K2239/23—On/off switch
    • A61K2239/24—Dimerizable CARs; CARs with adapter
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K39/46
    • A61K2239/26—Universal/off- the- shelf cellular immunotherapy; Allogenic cells or means to avoid rejection
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K39/46
    • A61K2239/46—Indexing codes associated with cellular immunotherapy of group A61K39/46 characterised by the cancer treated
    • A61K2239/48—Blood cells, e.g. leukemia or lymphoma
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/30—Immunoglobulins specific features characterized by aspects of specificity or valency
    • C07K2317/31—Immunoglobulins specific features characterized by aspects of specificity or valency multispecific
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/60—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments
    • C07K2317/62—Immunoglobulins specific features characterized by non-natural combinations of immunoglobulin fragments comprising only variable region components
    • C07K2317/622—Single chain antibody (scFv)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/40—Fusion polypeptide containing a tag for immunodetection, or an epitope for immunisation
    • 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]

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Engineering & Computer Science (AREA)
  • Cell Biology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Mycology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Toxicology (AREA)
  • Oncology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

The present invention relates to an engineered human T cell comprising a switchable chimeric antigen cell surface receptor, a pharmaceutical composition comprising the engineered human T cell, a kit comprising the engineered human T cell and a targeting module and a method for generating the engineered human T cell.
PCT/US2023/068901 2022-06-23 2023-06-22 Engineered human t cells comprising a switchable chimeric antigen cell surface receptor and methods for generating them WO2023250433A2 (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
EP22180820.7 2022-06-23
EP22180820.7A EP4295860A1 (en) 2022-06-23 2022-06-23 Engineered human t cells comprising a switchable chimeric antigen cell surface receptor and methods for generating them
EP23168261 2023-04-17
EP23168261.8 2023-04-17

Publications (2)

Publication Number Publication Date
WO2023250433A2 WO2023250433A2 (en) 2023-12-28
WO2023250433A3 true WO2023250433A3 (en) 2024-01-18

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PCT/US2023/068901 WO2023250433A2 (en) 2022-06-23 2023-06-22 Engineered human t cells comprising a switchable chimeric antigen cell surface receptor and methods for generating them
PCT/EP2023/067034 WO2023247727A2 (en) 2022-06-23 2023-06-22 Engineered human t cells comprising a switchable chimeric antigen cell surface receptor and methods for generating them

Family Applications After (1)

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PCT/EP2023/067034 WO2023247727A2 (en) 2022-06-23 2023-06-22 Engineered human t cells comprising a switchable chimeric antigen cell surface receptor and methods for generating them

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Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3170361A1 (en) * 2020-02-07 2021-08-12 Juntendo Educational Foundation Cytotoxic t cells derived from human t cell-derived ips cells

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019238722A1 (en) * 2018-06-13 2019-12-19 Gemoab Monoclonals Gmbh Reversed universal chimeric antigen receptor expressing immune cells for targeting of diverse multiple antigens and method of manufacturing the same and use of the same for treatment of cancer, infections and autoimmune disorders

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2688035T3 (en) 2014-08-29 2018-10-30 Gemoab Monoclonals Gmbh Universal antigen receptor that expresses immune cells for addressing multiple multiple antigens, procedure for manufacturing it and using it for the treatment of cancer, infections and autoimmune diseases

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019238722A1 (en) * 2018-06-13 2019-12-19 Gemoab Monoclonals Gmbh Reversed universal chimeric antigen receptor expressing immune cells for targeting of diverse multiple antigens and method of manufacturing the same and use of the same for treatment of cancer, infections and autoimmune disorders

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
ELLIS GAVIN I ET AL: "Genetic engineering of T cells for immunotherapy", NATURE REVIEWS GENETICS, NATURE PUBLISHING GROUP, GB, vol. 22, no. 7, 18 February 2021 (2021-02-18), pages 427 - 447, XP037484731, ISSN: 1471-0056, [retrieved on 20210218], DOI: 10.1038/S41576-021-00329-9 *
LIU DELONG ET AL: "Engineering switchable and programmable universal CARs for CAR T therapy", vol. 12, no. 1, 4 July 2019 (2019-07-04), pages 1 - 9, XP055772193, Retrieved from the Internet <URL:http://link.springer.com/article/10.1186/s13045-019-0763-0/fulltext.html> DOI: 10.1186/s13045-019-0763-0 *
WASEEM QASIM ET AL: "Molecular remission of infant B-ALL after infusion of universal TALEN gene-edited CAR T cells", SCIENCE TRANSLATIONAL MEDICINE, vol. 9, no. 374, 25 January 2017 (2017-01-25), pages eaaj2013, XP055498786, ISSN: 1946-6234, DOI: 10.1126/scitranslmed.aaj2013 *

Also Published As

Publication number Publication date
WO2023247727A3 (en) 2024-02-08
WO2023250433A2 (en) 2023-12-28
WO2023247727A2 (en) 2023-12-28

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