KR20230126707A - Cd34 유전자 변형을 위한 조성물 및 방법 - Google Patents

Cd34 유전자 변형을 위한 조성물 및 방법 Download PDF

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KR20230126707A
KR20230126707A KR1020237020742A KR20237020742A KR20230126707A KR 20230126707 A KR20230126707 A KR 20230126707A KR 1020237020742 A KR1020237020742 A KR 1020237020742A KR 20237020742 A KR20237020742 A KR 20237020742A KR 20230126707 A KR20230126707 A KR 20230126707A
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cell
cells
grna
genetically engineered
hematopoietic
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Korean (ko)
Inventor
엘리자베스 팩
알레잔드라 팔라
티르타 차크라보티
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보르 바이오파마 인크.
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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/28Bone marrow; Haematopoietic stem cells; Mesenchymal stem cells of any origin, e.g. adipose-derived stem cells
    • A61K39/4611
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/10Cellular immunotherapy characterised by the cell type used
    • A61K40/11T-cells, e.g. tumour infiltrating lymphocytes [TIL] or regulatory T [Treg] cells; Lymphokine-activated killer [LAK] cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/30Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/31Chimeric antigen receptors [CAR]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00Cellular immunotherapy
    • A61K40/40Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41Vertebrate antigens
    • A61K40/42Cancer antigens
    • A61K40/4202Receptors, cell surface antigens or cell surface determinants
    • A61K40/4224Molecules with a "CD" designation not provided for elsewhere
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
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    • C12N15/113Non-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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    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
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    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2510/00Genetically modified cells

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  • Virology (AREA)
  • Mycology (AREA)
  • Oncology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020237020742A 2020-12-31 2021-12-31 Cd34 유전자 변형을 위한 조성물 및 방법 Pending KR20230126707A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
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

Publications (1)

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KR20230126707A true KR20230126707A (ko) 2023-08-30

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KR1020237020742A Pending KR20230126707A (ko) 2020-12-31 2021-12-31 Cd34 유전자 변형을 위한 조성물 및 방법

Country Status (8)

Country Link
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=)
WO (1) WO2022147347A1 (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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US20140310830A1 (en) 2012-12-12 2014-10-16 Feng Zhang CRISPR-Cas Nickase Systems, Methods And Compositions For Sequence Manipulation in Eukaryotes
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Publication number Publication date
WO2022147347A9 (en) 2023-05-25
WO2022147347A1 (en) 2022-07-07
AU2021413252A1 (en) 2023-06-08
CA3202219A1 (en) 2022-07-07
JP2024502820A (ja) 2024-01-23
EP4271801A1 (en) 2023-11-08
US20240366675A1 (en) 2024-11-07
CN116724109A (zh) 2023-09-08

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