JP7784707B2 - 系統特異的抗原の阻害のための組成物および方法 - Google Patents

系統特異的抗原の阻害のための組成物および方法

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Publication number
JP7784707B2
JP7784707B2 JP2021541017A JP2021541017A JP7784707B2 JP 7784707 B2 JP7784707 B2 JP 7784707B2 JP 2021541017 A JP2021541017 A JP 2021541017A JP 2021541017 A JP2021541017 A JP 2021541017A JP 7784707 B2 JP7784707 B2 JP 7784707B2
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cells
cell
grna
hematopoietic stem
genetically engineered
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JP2022517618A (ja
JPWO2020150478A5 (https=
JP2022517618A5 (https=
Inventor
ムカジー，シッダールタ
ボロット，フローレンス
アリ，アブドゥッラー，マムード
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Columbia University in the City of New York
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Columbia University in the City of New York
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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
    • A61P35/00—Antineoplastic agents
    • A61P35/02—Antineoplastic agents specific for leukemia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • 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
    • C07K14/70596—Molecules with a "CD"-designation not provided for elsewhere
    • C—CHEMISTRY; METALLURGY
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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2510/00—Genetically modified cells

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Cell Biology (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Hematology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Oncology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
JP2021541017A 2019-01-16 2020-01-16 系統特異的抗原の阻害のための組成物および方法 Active JP7784707B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025202676A JP2026035683A (ja) 2019-01-16 2025-11-25 系統特異的抗原の阻害のための組成物および方法

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

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JP2025202676A Division JP2026035683A (ja) 2019-01-16 2025-11-25 系統特異的抗原の阻害のための組成物および方法

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JP2022517618A JP2022517618A (ja) 2022-03-09
JPWO2020150478A5 JPWO2020150478A5 (https=) 2023-01-25
JP2022517618A5 JP2022517618A5 (https=) 2023-01-25
JP7784707B2 true JP7784707B2 (ja) 2025-12-12

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JP2025202676A Pending JP2026035683A (ja) 2019-01-16 2025-11-25 系統特異的抗原の阻害のための組成物および方法

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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
SG11201907870VA (en) 2017-02-28 2019-09-27 Vor Biopharma Inc Compositions and methods for inhibition of lineage specific proteins
CA3110837A1 (en) 2018-08-28 2020-03-05 Vor Biopharma Inc. Genetically engineered hematopoietic stem cells and uses thereof
CN114423865A (zh) * 2019-05-23 2022-04-29 Vor生物制药股份有限公司 Cd33修饰的组合物和方法
US20220290160A1 (en) * 2019-08-28 2022-09-15 Vor Biopharma Inc. Compositions and methods for cll1 modification
US20240110189A1 (en) * 2020-08-28 2024-04-04 Vor Biopharma Inc. Compositions and methods for cll1 modification
CA3199623A1 (en) * 2020-10-27 2022-05-05 Vor Biopharma Inc. Compositions and methods for treating hematopoietic malignancy
CN115443333B (zh) * 2021-04-27 2025-04-25 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
US20240384304A1 (en) 2021-07-06 2024-11-21 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

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JP2018519801A (ja) 2015-05-11 2018-07-26 エディタス・メディシン、インコーポレイテッド 幹細胞における遺伝子編集のための最適化ｃｒｉｓｐｒ／ｃａｓ９システムおよび方法
WO2018154387A1 (en) 2017-02-22 2018-08-30 Crispr Therapeutics Ag Compositions and methods for gene editing
JP2018538002A (ja) 2015-12-23 2018-12-27 クリスパー セラピューティクス アーゲー 筋萎縮性側索硬化症及び／または前頭側頭葉変性症の治療のための材料及び方法

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EP3126390B2 (en) * 2014-04-03 2026-01-07 Cellectis Cd33 specific chimeric antigen receptors for cancer immunotherapy
RU2749113C2 (ru) * 2015-04-22 2021-06-04 Куревак Аг Содержащая рнк композиция для лечения опухолевых заболеваний
CN108290939B (zh) * 2015-10-16 2023-01-13 纽约市哥伦比亚大学理事会 用于抑制谱系特异性抗原的组合物和方法
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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JP2018519801A (ja) 2015-05-11 2018-07-26 エディタス・メディシン、インコーポレイテッド 幹細胞における遺伝子編集のための最適化ｃｒｉｓｐｒ／ｃａｓ９システムおよび方法
JP2018538002A (ja) 2015-12-23 2018-12-27 クリスパー セラピューティクス アーゲー 筋萎縮性側索硬化症及び／または前頭側頭葉変性症の治療のための材料及び方法
WO2018154387A1 (en) 2017-02-22 2018-08-30 Crispr Therapeutics Ag Compositions and methods for gene editing

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Also Published As

Publication number Publication date
CA3126677A1 (en) 2020-07-23
AU2020209218A1 (en) 2021-07-29
IL284853A (en) 2021-08-31
EP3911338A4 (en) 2023-06-07
JP2022517618A (ja) 2022-03-09
WO2020150478A1 (en) 2020-07-23
JP2026035683A (ja) 2026-03-04
KR20210129048A (ko) 2021-10-27
US20210260130A1 (en) 2021-08-26
BR112021014010A2 (pt) 2021-09-21
CN113474452A (zh) 2021-10-01
EP3911338A1 (en) 2021-11-24
MX2021008490A (es) 2021-09-28
SG11202107639UA (en) 2021-08-30

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