EP4175651A4 - METHODS AND COMPOSITIONS FOR MODIFYING THE B2M LOCUS IN B CELLS - Google Patents

METHODS AND COMPOSITIONS FOR MODIFYING THE B2M LOCUS IN B CELLS Download PDF

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Publication number
EP4175651A4
EP4175651A4 EP21832140.4A EP21832140A EP4175651A4 EP 4175651 A4 EP4175651 A4 EP 4175651A4 EP 21832140 A EP21832140 A EP 21832140A EP 4175651 A4 EP4175651 A4 EP 4175651A4
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EP
European Patent Office
Prior art keywords
editing
locus
compositions
cells
methods
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
EP21832140.4A
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German (de)
English (en)
French (fr)
Other versions
EP4175651A1 (en
Inventor
David J. Rawlings
Richard G. James
Claire Marie STOFFERS
Peter J. Cook
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.)
Seattle Childrens Hospital
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Seattle Childrens Hospital
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Filing date
Publication date
Application filed by Seattle Childrens Hospital filed Critical Seattle Childrens Hospital
Publication of EP4175651A1 publication Critical patent/EP4175651A1/en
Publication of EP4175651A4 publication Critical patent/EP4175651A4/en
Pending legal-status Critical Current

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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/10—Cellular immunotherapy characterised by the cell type used
    • A61K40/13—B-cells
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/20—Cellular immunotherapy characterised by the effect or the function of the cells
    • A61K40/22—Immunosuppressive or immunotolerising
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
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    • A61K40/24—Antigen-presenting cells [APC]
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    • A61K40/00—Cellular immunotherapy
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    • A61K40/00—Cellular immunotherapy
    • A61K40/30—Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/35—Cytokines
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • 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
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    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
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    • C07K2319/00—Fusion polypeptide
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  • Developmental Biology & Embryology (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
EP21832140.4A 2020-07-03 2021-06-30 METHODS AND COMPOSITIONS FOR MODIFYING THE B2M LOCUS IN B CELLS Pending EP4175651A4 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063047978P 2020-07-03 2020-07-03
US202063114131P 2020-11-16 2020-11-16
PCT/US2021/039953 WO2022006309A1 (en) 2020-07-03 2021-06-30 Methods and compositions for editing the b2m locus in b cells

Publications (2)

Publication Number Publication Date
EP4175651A1 EP4175651A1 (en) 2023-05-10
EP4175651A4 true EP4175651A4 (en) 2024-08-14

Family

ID=79315579

Family Applications (1)

Application Number Title Priority Date Filing Date
EP21832140.4A Pending EP4175651A4 (en) 2020-07-03 2021-06-30 METHODS AND COMPOSITIONS FOR MODIFYING THE B2M LOCUS IN B CELLS

Country Status (10)

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EP (1) EP4175651A4 (https=)
JP (1) JP2023532917A (https=)
KR (1) KR20230035048A (https=)
CN (1) CN116033910A (https=)
AU (1) AU2021300358A1 (https=)
BR (1) BR112022026534A2 (https=)
CA (1) CA3186620A1 (https=)
IL (1) IL299491A (https=)
MX (1) MX2022015788A (https=)
WO (1) WO2022006309A1 (https=)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20250222138A1 (en) * 2022-03-28 2025-07-10 Ramot At Tel-Aviv University Ltd. Site-specific in vivo t cell engineering, systems, compositions and methods thereof
CA3261942A1 (en) * 2022-08-01 2024-02-08 Idexx Laboratories, Inc. FRACTIONAL ABUNDANCE DOSES OF HUMAN MOUSE
EP4698226A2 (en) * 2023-04-17 2026-02-25 Be Biopharma, Inc. Engineered cell preparations for treatment of hemophilia
CN120866304A (zh) * 2024-04-23 2025-10-31 南京奇迹生物科技有限公司 对b2m基因座的基因编辑方法

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016073955A2 (en) * 2014-11-06 2016-05-12 President And Fellows Of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
WO2020049535A1 (en) * 2018-09-07 2020-03-12 Crisper Therapeutics Ag Universal donor cells

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IL297018A (en) * 2015-12-18 2022-12-01 Sangamo Therapeutics Inc Directed cleavage of cell mhc receptor
US12144825B2 (en) * 2017-06-30 2024-11-19 Cellectis Cellular immunotherapy for repetitive administration
US20200407713A1 (en) * 2017-11-16 2020-12-31 Mogam Institute For Biomedical Research Transformed human cell and use thereof
CA3122131A1 (en) * 2018-09-21 2020-03-26 Zonghai Li Method for gene editing of cell on the basis of crispr/cas system
UY38427A (es) * 2018-10-26 2020-05-29 Novartis Ag Métodos y composiciones para terapia con células oculares

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2016073955A2 (en) * 2014-11-06 2016-05-12 President And Fellows Of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
WO2020049535A1 (en) * 2018-09-07 2020-03-12 Crisper Therapeutics Ag Universal donor cells

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
See also references of WO2022006309A1 *

Also Published As

Publication number Publication date
KR20230035048A (ko) 2023-03-10
JP2023532917A (ja) 2023-08-01
CA3186620A1 (en) 2022-01-06
EP4175651A1 (en) 2023-05-10
MX2022015788A (es) 2023-02-27
WO2022006309A1 (en) 2022-01-06
CN116033910A (zh) 2023-04-28
AU2021300358A1 (en) 2023-02-02
IL299491A (en) 2023-02-01
BR112022026534A2 (pt) 2023-04-18

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