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

계통 특이적 항원의 저해를 위한 조성물 및 방법 Download PDF

Info

Publication number
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
Authority
KR
South Korea
Prior art keywords
cell
cells
grna
hematopoietic stem
genetically engineered
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
KR1020217025362A
Other languages
English (en)
Korean (ko)
Inventor
시드하르타 무케르지
플로렌스 보로트
압둘라 마흐무드 알리
Original Assignee
더 트러스티스 오브 컬럼비아 유니버시티 인 더 시티 오브 뉴욕
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by 더 트러스티스 오브 컬럼비아 유니버시티 인 더 시티 오브 뉴욕 filed Critical 더 트러스티스 오브 컬럼비아 유니버시티 인 더 시티 오브 뉴욕
Publication of KR20210129048A publication Critical patent/KR20210129048A/ko
Pending legal-status Critical Current

Links

Images

Classifications

    • 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
    • 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
    • C12N15/10—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

Landscapes

  • 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)
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

Family

ID=71613437

Family Applications (1)

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
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

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
AU2016261358B2 (en) 2015-05-11 2021-09-16 Editas Medicine, Inc. Optimized CRISPR/Cas9 systems and methods for gene editing in stem cells
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
BR112018012894A2 (en) 2015-12-23 2018-12-04 Crispr Therapeutics Ag Materials and Methods for Treatment of Amyotrophic Lateral Sclerosis and / or Frontotemporal Lobular Degeneration
JP2020508056A (ja) 2017-02-22 2020-03-19 クリスパー・セラピューティクス・アクチェンゲゼルシャフトＣＲＩＳＰＲ Ｔｈｅｒａｐｅｕｔｉｃｓ ＡＧ 遺伝子編集のための組成物および方法
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

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
JP7784707B2 (ja) 2025-12-12
JP2026035683A (ja) 2026-03-04
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

Similar Documents

Publication Publication Date Title
JP7680384B2 (ja) 系統特異的抗原の阻害のための組成物および方法
US20240166721A1 (en) Cd33 exon 2 deficient donor stem cells for use with cd33 targeting agents
JP7784707B2 (ja) 系統特異的抗原の阻害のための組成物および方法
US20240041923A1 (en) Compositions and methods for inhibition of lineage specific antigens using crispr-based base editor systems
HK1258310B (en) Compositions and methods for inhibition of lineage specific antigens

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

E13-X000 Pre-grant limitation requested

St.27 status event code: A-2-3-E10-E13-lim-X000

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

T11-X000 Administrative time limit extension requested

St.27 status event code: U-3-3-T10-T11-oth-X000

T11 Administrative time limit extension requested

Free format text: ST27 STATUS EVENT CODE: U-3-3-T10-T11-OTH-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

T11-X000 Administrative time limit extension requested

St.27 status event code: U-3-3-T10-T11-oth-X000

T11 Administrative time limit extension requested

Free format text: ST27 STATUS EVENT CODE: U-3-3-T10-T11-OTH-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

T11-X000 Administrative time limit extension requested

St.27 status event code: U-3-3-T10-T11-oth-X000

T11 Administrative time limit extension requested

Free format text: ST27 STATUS EVENT CODE: U-3-3-T10-T11-OTH-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

T11-X000 Administrative time limit extension requested

St.27 status event code: U-3-3-T10-T11-oth-X000

P11 Amendment of application requested

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P11-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000