AU2021300358A1 - Methods and compositions for editing the B2M locus in B cells - Google Patents

Methods and compositions for editing the B2M locus in B cells Download PDF

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Publication number
AU2021300358A1
AU2021300358A1 AU2021300358A AU2021300358A AU2021300358A1 AU 2021300358 A1 AU2021300358 A1 AU 2021300358A1 AU 2021300358 A AU2021300358 A AU 2021300358A AU 2021300358 A AU2021300358 A AU 2021300358A AU 2021300358 A1 AU2021300358 A1 AU 2021300358A1
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cell
modified
cells
gene
nuclease
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AU2021300358A
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Peter J. Cook
Richard G. James
David J. Rawlings
Claire Marie STOFFERS
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Seattle Childrens Hospital
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Seattle Childrens Hospital
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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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    • A61K40/00—Cellular immunotherapy
    • A61K40/20—Cellular immunotherapy characterised by the effect or the function of the cells
    • A61K40/22—Immunosuppressive or immunotolerising
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    • A61K40/24—Antigen-presenting cells [APC]
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
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    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
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    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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    • 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
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    • 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
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    • C12N9/14—Hydrolases (3)
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    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2121/00—Preparations for use in therapy
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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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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
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    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
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    • 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 [CRISPR]
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Gastroenterology & Hepatology (AREA)
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  • Virology (AREA)
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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2021300358A 2020-07-03 2021-06-30 Methods and compositions for editing the B2M locus in B cells Pending AU2021300358A1 (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202063047978P 2020-07-03 2020-07-03
US63/047,978 2020-07-03
US202063114131P 2020-11-16 2020-11-16
US63/114,131 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 (1)

Publication Number Publication Date
AU2021300358A1 true AU2021300358A1 (en) 2023-02-02

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AU2021300358A Pending AU2021300358A1 (en) 2020-07-03 2021-06-30 Methods and compositions for editing the B2M locus in B cells

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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

Family Cites Families (7)

* 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
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
US12378572B2 (en) * 2018-09-07 2025-08-05 Crispr Therapeutics Ag Universal donor cells
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

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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
EP4175651A4 (en) 2024-08-14
IL299491A (en) 2023-02-01
BR112022026534A2 (pt) 2023-04-18

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