KR20210129048A - 계통 특이적 항원의 저해를 위한 조성물 및 방법 - Google Patents

계통 특이적 항원의 저해를 위한 조성물 및 방법 Download PDF

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KR20210129048A
KR20210129048A KR1020217025362A KR20217025362A KR20210129048A KR 20210129048 A KR20210129048 A KR 20210129048A KR 1020217025362 A KR1020217025362 A KR 1020217025362A KR 20217025362 A KR20217025362 A KR 20217025362A KR 20210129048 A KR20210129048 A KR 20210129048A
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cell
cells
grna
hematopoietic stem
genetically engineered
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Korean (ko)
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시드하르타 무케르지
플로렌스 보로트
압둘라 마흐무드 알리
Original Assignee
더 트러스티스 오브 컬럼비아 유니버시티 인 더 시티 오브 뉴욕
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Publication of KR20210129048A publication Critical patent/KR20210129048A/ko
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • 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
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    • 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
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    • 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
    • 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
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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06—Animal cells or tissues; Human cells or tissues
    • C12N5/0602—Vertebrate cells
    • C12N5/0634—Cells from the blood or the immune system
    • C12N5/0647—Haematopoietic stem cells; Uncommitted or multipotent progenitors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2510/00—Genetically modified cells

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  • Animal Behavior & Ethology (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Oncology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
KR1020217025362A 2019-01-16 2020-01-16 계통 특이적 항원의 저해를 위한 조성물 및 방법 Pending KR20210129048A (ko)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962793210P 2019-01-16 2019-01-16
US62/793,210 2019-01-16
US201962852573P 2019-05-24 2019-05-24
US62/852,573 2019-05-24
PCT/US2020/013887 WO2020150478A1 (en) 2019-01-16 2020-01-16 Compositions and methods for inhibition of lineage specific antigens

Publications (1)

Publication Number Publication Date
KR20210129048A true KR20210129048A (ko) 2021-10-27

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Application Number Title Priority Date Filing Date
KR1020217025362A Pending KR20210129048A (ko) 2019-01-16 2020-01-16 계통 특이적 항원의 저해를 위한 조성물 및 방법

Country Status (12)

Country Link
US (1) US20210260130A1 (https=)
EP (1) EP3911338A4 (https=)
JP (2) JP7784707B2 (https=)
KR (1) KR20210129048A (https=)
CN (1) CN113474452A (https=)
AU (1) AU2020209218A1 (https=)
BR (1) BR112021014010A2 (https=)
CA (1) CA3126677A1 (https=)
IL (1) IL284853A (https=)
MX (1) MX2021008490A (https=)
SG (1) SG11202107639UA (https=)
WO (1) WO2020150478A1 (https=)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2019130504A (ru) 2017-02-28 2021-03-30 Вор Байофарма, Инк. Композиции и способы ингибирования линиеспецифических белков
CA3110837A1 (en) 2018-08-28 2020-03-05 Vor Biopharma Inc. Genetically engineered hematopoietic stem cells and uses thereof
AU2020280103A1 (en) * 2019-05-23 2021-12-23 Vor Biopharma Inc. Compositions and methods for CD33 modification
AU2020338011A1 (en) * 2019-08-28 2022-03-10 Vor Biopharma Inc. Compositions and methods for CLL1 modification
WO2022047168A1 (en) * 2020-08-28 2022-03-03 Vor Biopharma Inc. Compositions and methods for cll1 modification
WO2022093983A1 (en) * 2020-10-27 2022-05-05 Vor Biopharma, Inc. Compositions and methods for treating hematopoietic malignancy
CN120249215A (zh) * 2021-04-27 2025-07-04 上海驯鹿生物技术有限公司 一种基因编辑的造血干细胞及其与car-t细胞的联合应用
WO2023283585A2 (en) 2021-07-06 2023-01-12 Vor Biopharma Inc. Inhibitor oligonucleotides and methods of use thereof
AU2022324093A1 (en) 2021-08-02 2024-02-08 Vor Biopharma Inc. Compositions and methods for gene modification
WO2024015925A2 (en) 2022-07-13 2024-01-18 Vor Biopharma Inc. Compositions and methods for artificial protospacer adjacent motif (pam) generation
WO2024073751A1 (en) 2022-09-29 2024-04-04 Vor Biopharma Inc. Methods and compositions for gene modification and enrichment
WO2025030010A1 (en) 2023-08-01 2025-02-06 Vor Biopharma Inc. Compositions comprising genetically engineered hematopoietic stem cells and methods of use thereof
BG113932A (bg) * 2024-07-19 2026-01-30 Медицински университет – Пловдив Панели и китове от реагенти за измерване на минимална резидуална болест при детска в-клетъчна прекурсорна остра лимфобластна левкемия чрез имунофенотипизация

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN106795221B (zh) * 2014-04-03 2022-06-07 塞勒克提斯公司 用于癌症免疫治疗的cd33特异性嵌合抗原受体
CN108064176A (zh) * 2015-04-22 2018-05-22 库瑞瓦格股份公司 用于治疗肿瘤疾病的含有rna的组合物
CA2986310A1 (en) * 2015-05-11 2016-11-17 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
AU2016337525B2 (en) * 2015-10-16 2022-01-20 The Trustees Of Columbia University In The City Of New York Compositions and methods for inhibition of lineage specific antigens
KR20180118111A (ko) * 2015-12-23 2018-10-30 크리스퍼 테라퓨틱스 아게 근위축성 측색 경화증 및/또는 전두측두엽 퇴행의 치료 물질 및 방법
EP3585895A1 (en) * 2017-02-22 2020-01-01 CRISPR Therapeutics AG Compositions and methods for gene editing
US11718659B2 (en) * 2017-08-28 2023-08-08 The Trustees Of Columbia University In The City Of New York CD33 exon 2 deficient donor stem cells for use with CD33 targeting agents

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Publication number Publication date
BR112021014010A2 (pt) 2021-09-21
JP2022517618A (ja) 2022-03-09
JP7784707B2 (ja) 2025-12-12
JP2026035683A (ja) 2026-03-04
CA3126677A1 (en) 2020-07-23
SG11202107639UA (en) 2021-08-30
MX2021008490A (es) 2021-09-28
WO2020150478A1 (en) 2020-07-23
EP3911338A4 (en) 2023-06-07
EP3911338A1 (en) 2021-11-24
AU2020209218A1 (en) 2021-07-29
US20210260130A1 (en) 2021-08-26
CN113474452A (zh) 2021-10-01
IL284853A (en) 2021-08-31

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