JP2024502820A - Ｃｄ３４遺伝子改変のための組成物及び方法 - Google Patents

Ｃｄ３４遺伝子改変のための組成物及び方法 Download PDF

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JP2024502820A
JP2024502820A JP2023540486A JP2023540486A JP2024502820A JP 2024502820 A JP2024502820 A JP 2024502820A JP 2023540486 A JP2023540486 A JP 2023540486A JP 2023540486 A JP2023540486 A JP 2023540486A JP 2024502820 A JP2024502820 A JP 2024502820A
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cells
cell
grna
genetically engineered
hematopoietic
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パイク，エリザベス
ファリャ，アレハンドラ
チャクラボーティ，ティルタ
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Vor Biopharma Inc
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    • A—HUMAN NECESSITIES
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    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28—Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • 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
    • A61K40/11—T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/30—Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/31—Chimeric antigen receptors [CAR]
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    • A61K40/4202—Receptors, cell surface antigens or cell surface determinants
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    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
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    • C07K14/7051—T-cell receptor (TcR)-CD3 complex
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    • 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
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    • 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
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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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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JP2023540486A 2020-12-31 2021-12-31 Ｃｄ３４遺伝子改変のための組成物及び方法 Pending JP2024502820A (ja)

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US202063132852P 2020-12-31 2020-12-31
US63/132,852 2020-12-31
PCT/US2021/065813 WO2022147347A1 (en) 2020-12-31 2021-12-31 Compositions and methods for cd34 gene modification

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JP2024502820A true JP2024502820A (ja) 2024-01-23
JP2024502820A5 JP2024502820A5 (https=) 2025-01-15

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US (1) US20240366675A1 (https=)
EP (1) EP4271801A1 (https=)
JP (1) JP2024502820A (https=)
KR (1) KR20230126707A (https=)
CN (1) CN116724109A (https=)
AU (1) AU2021413252A1 (https=)
CA (1) CA3202219A1 (https=)
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CA3110837A1 (en) * 2018-08-28 2020-03-05 Vor Biopharma Inc. Genetically engineered hematopoietic stem cells and uses thereof
CN116656681B (zh) * 2023-05-09 2024-03-19 佛山市第一人民医院 一种cd34转基因模式小鼠的构建方法与应用
WO2025030010A1 (en) 2023-08-01 2025-02-06 Vor Biopharma Inc. Compositions comprising genetically engineered hematopoietic stem cells and methods of use thereof
CN118325842B (zh) * 2024-06-17 2025-01-24 天海元祺生物科技(天津)有限公司 一种工程化的细胞及其应用

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KR20230126707A (ko) 2023-08-30
WO2022147347A1 (en) 2022-07-07
AU2021413252A1 (en) 2023-06-08
CA3202219A1 (en) 2022-07-07
EP4271801A1 (en) 2023-11-08
US20240366675A1 (en) 2024-11-07
CN116724109A (zh) 2023-09-08

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