JP7615134B2 - 液体がんの治療のための組成物および方法 - Google Patents

液体がんの治療のための組成物および方法 Download PDF

Info

Publication number
JP7615134B2
JP7615134B2 JP2022519460A JP2022519460A JP7615134B2 JP 7615134 B2 JP7615134 B2 JP 7615134B2 JP 2022519460 A JP2022519460 A JP 2022519460A JP 2022519460 A JP2022519460 A JP 2022519460A JP 7615134 B2 JP7615134 B2 JP 7615134B2
Authority
JP
Japan
Prior art keywords
tada
cell
protein
domain
deaminase
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.)
Active
Application number
JP2022519460A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022549916A (ja
JPWO2021062227A5 (https=
JP2022549916A5 (https=
Inventor
ジェイソン マイケル ゲールケ、
アーロン ディー． エドワーズ、
ライアン マーレイ、
ワシーム カシム、
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
UCL Business Ltd
Original Assignee
UCL Biomedica PLC
UCL Business Ltd
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 UCL Biomedica PLC, UCL Business Ltd filed Critical UCL Biomedica PLC
Publication of JP2022549916A publication Critical patent/JP2022549916A/ja
Publication of JPWO2021062227A5 publication Critical patent/JPWO2021062227A5/ja
Publication of JP2022549916A5 publication Critical patent/JP2022549916A5/ja
Priority to JP2024231478A priority Critical patent/JP2025066723A/ja
Application granted granted Critical
Publication of JP7615134B2 publication Critical patent/JP7615134B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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]
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • 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
    • 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
    • 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
    • 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
    • A61K40/13—B-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
    • A61K40/15—Natural-killer [NK] cells; Natural-killer T [NKT] 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
    • A61K40/19—Dendritic cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/20—Cellular immunotherapy characterised by the effect or the function of the cells
    • A61K40/22—Immunosuppressive or immunotolerising
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41—Vertebrate antigens
    • A61K40/418—Antigens related to induction of tolerance to non-self
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41—Vertebrate antigens
    • A61K40/42—Cancer antigens
    • A61K40/4202—Receptors, cell surface antigens or cell surface determinants
    • A61K40/4214—Receptors for cytokines
    • A61K40/4215—Receptors for tumor necrosis factors [TNF], e.g. lymphotoxin receptor [LTR], CD30
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41—Vertebrate antigens
    • A61K40/42—Cancer antigens
    • A61K40/4202—Receptors, cell surface antigens or cell surface determinants
    • A61K40/4214—Receptors for cytokines
    • A61K40/4217—Receptors for interleukins [IL]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/40—Cellular immunotherapy characterised by antigens that are targeted or presented by cells of the immune system
    • A61K40/41—Vertebrate antigens
    • A61K40/42—Cancer antigens
    • A61K40/4202—Receptors, cell surface antigens or cell surface determinants
    • A61K40/4224—Molecules with a "CD" designation not provided for elsewhere
    • 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
    • C07K14/70503—Immunoglobulin superfamily
    • C07K14/7051—T-cell receptor (TcR)-CD3 complex
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/12—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from bacteria
    • C07K16/1267—Gram-positive bacteria
    • C07K16/1271—Micrococcaceae (F); Staphylococcaceae (F), e.g. Staphylococcus (G)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2803—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the immunoglobulin superfamily
    • 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
    • C12N5/0636—T lymphocytes
    • 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
    • A61K2035/122—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells for inducing tolerance or supression of immune responses
    • 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
    • A61K2035/124—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells the cells being hematopoietic, bone marrow derived or blood cells
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00—Medicinal preparations containing antigens or antibodies
    • A61K2039/80—Vaccine for a specifically defined cancer
    • A61K2039/804—Blood cells [leukemia, lymphoma]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2121/00—Preparations for use in therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2239/00—Indexing codes associated with cellular immunotherapy of group A61K40/00
    • A61K2239/46—Indexing codes associated with cellular immunotherapy of group A61K40/00 characterised by the cancer treated
    • A61K2239/48—Blood cells, e.g. leukemia or lymphoma
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K40/00—Cellular immunotherapy
    • A61K40/50—Cellular immunotherapy characterised by the use of allogeneic cells
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03—Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
    • 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
    • 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
    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector
    • C12N2740/15043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
    • C12N2800/106—Plasmid DNA for vertebrates
    • C12N2800/107—Plasmid DNA for vertebrates for mammalian

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Immunology (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Cell Biology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Engineering & Computer Science (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
  • Oncology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Developmental Biology & Embryology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
JP2022519460A 2019-09-27 2020-09-25 液体がんの治療のための組成物および方法 Active JP7615134B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024231478A JP2025066723A (ja) 2019-09-27 2024-12-27 液体がんの治療のための組成物および方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962907254P 2019-09-27 2019-09-27
US62/907,254 2019-09-27
PCT/US2020/052822 WO2021062227A2 (en) 2019-09-27 2020-09-25 Compositions and methods for treatment of liquid cancers

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024231478A Division JP2025066723A (ja) 2019-09-27 2024-12-27 液体がんの治療のための組成物および方法

Publications (4)

Publication Number Publication Date
JP2022549916A JP2022549916A (ja) 2022-11-29
JPWO2021062227A5 JPWO2021062227A5 (https=) 2023-10-02
JP2022549916A5 JP2022549916A5 (https=) 2023-10-02
JP7615134B2 true JP7615134B2 (ja) 2025-01-16

Family

ID=75166843

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022519460A Active JP7615134B2 (ja) 2019-09-27 2020-09-25 液体がんの治療のための組成物および方法
JP2024231478A Pending JP2025066723A (ja) 2019-09-27 2024-12-27 液体がんの治療のための組成物および方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024231478A Pending JP2025066723A (ja) 2019-09-27 2024-12-27 液体がんの治療のための組成物および方法

Country Status (8)

Country Link
US (1) US12594301B2 (https=)
EP (1) EP4034138A4 (https=)
JP (2) JP7615134B2 (https=)
KR (1) KR20220090512A (https=)
CN (2) CN115279398B (https=)
AU (1) AU2020355222A1 (https=)
CA (1) CA3155667A1 (https=)
WO (1) WO2021062227A2 (https=)

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
WO2020168122A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
EP4034138A4 (en) 2019-09-27 2024-07-31 Beam Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS
WO2022056459A1 (en) * 2020-09-14 2022-03-17 Vor Biopharma, Inc. Compositions and methods for cd5 modification
WO2022061115A1 (en) * 2020-09-18 2022-03-24 Vor Biopharma Inc. Compositions and methods for cd7 modification
AU2021347359A1 (en) 2020-09-25 2023-05-18 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
US11591381B2 (en) 2020-11-30 2023-02-28 Crispr Therapeutics Ag Gene-edited natural killer cells
WO2022144632A1 (en) 2020-12-30 2022-07-07 Crispr Therapeutics Ag Compositions and methods for differentiating stem cells into nk cells
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
EP4337701A4 (en) 2021-05-10 2025-04-09 Mammoth Biosciences, Inc. EFFECTOR PROTEINS AND METHODS OF USE
WO2023034475A1 (en) * 2021-09-01 2023-03-09 Arbor Biotechnologies, Inc. Cells modified by a cas12i polypeptide
AU2022366987A1 (en) 2021-10-14 2024-05-16 Arsenal Biosciences, Inc. Immune cells having co-expressed shrnas and logic gate systems
CA3242360A1 (en) * 2021-12-10 2023-06-15 Beam Therapeutics Inc. Modified immune cells and methods of using the same
WO2023137457A2 (en) * 2022-01-13 2023-07-20 Spotlight Therapeutics Immune response specific guide rnas and uses thereof
AU2023209457A1 (en) 2022-01-21 2024-08-08 Mammoth Biosciences, Inc. Effector proteins and methods of use
KR20250027283A (ko) * 2022-05-19 2025-02-25 라이엘 이뮤노파마, 인크. nr4a3을 표적하는 폴리뉴클레오타이드 및 이의 용도
EP4587570A2 (en) * 2022-09-16 2025-07-23 Arsenal Biosciences, Inc. Immune cells with combination gene perturbations
KR20250158048A (ko) 2023-03-03 2025-11-05 아스널 바이오사이언시스, 인크. Psma 및 ca9을 표적으로 하는 시스템
EP4683650A2 (en) * 2023-03-21 2026-01-28 Beam Therapeutics Inc. Immunosuppressant-resistant modified allogeneic modified immune cells and methods for use thereof
WO2025126049A2 (en) * 2023-12-11 2025-06-19 Crispr Therapeutics Ag Multiplex gene editing
WO2026077659A1 (en) * 2024-09-17 2026-04-16 Institut National de la Santé et de la Recherche Médicale Enrichment of genetically modified hematopoietic stem cells through epigenome editing

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019524156A (ja) 2016-08-03 2019-09-05 ジョン・エフ・ディパージオＪｏｈｎ Ｆ． ＤＩＰＥＲＳＩＯ キメラ抗原受容体でのｔ細胞性悪性腫瘍の治療のためのｃａｒ−ｔ細胞の遺伝子編集

Family Cites Families (243)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1997006121A1 (en) 1995-08-04 1997-02-20 Jennings, Norman, T. Compositions and method for controlling odor in organic waste material
GB9600384D0 (en) 1996-01-09 1996-03-13 Nyfotek As Dna glycosylases
CA2392490A1 (en) 1999-11-24 2001-05-31 Mcs Micro Carrier Systems Gmbh Polypeptides comprising multimers of nuclear localization signals or of protein transduction domains and their use for transferring molecules into cells
US20040003420A1 (en) 2000-11-10 2004-01-01 Ralf Kuhn Modified recombinase
US20050222030A1 (en) 2001-02-21 2005-10-06 Anthony Allison Modified annexin proteins and methods for preventing thrombosis
US20040115184A1 (en) 2001-02-27 2004-06-17 Smith Harold C Methods and compositions for modifying apolipoprotein b mrna editing
AU2002330714A1 (en) 2001-05-30 2003-01-02 Biomedical Center In silico screening for phenotype-associated expressed sequences
US7300829B2 (en) 2003-06-02 2007-11-27 Applied Materials, Inc. Low temperature process for TFT fabrication
WO2010132092A2 (en) 2009-05-12 2010-11-18 The Scripps Research Institute Cytidine deaminase fusions and related methods
US20110104787A1 (en) 2009-11-05 2011-05-05 President And Fellows Of Harvard College Fusion Peptides That Bind to and Modify Target Nucleic Acid Sequences
US20130011380A1 (en) 2009-12-18 2013-01-10 Blau Helen M Use of Cytidine Deaminase-Related Agents to Promote Demethylation and Cell Reprogramming
US20130109048A1 (en) 2010-07-09 2013-05-02 Ecole Polytechnique Federale De Lausanne (Epfl) Method for in-vitro monitoring of neuronal disorders and use thereof
WO2012145384A1 (en) 2011-04-20 2012-10-26 University Of Washington Through Its Center For Commercialization Beta-2 microglobulin-deficient cells
EP2761006B1 (en) 2011-09-28 2016-12-14 Zera Intein Protein Solutions, S.L. Split inteins and uses thereof
CN103088008B (zh) 2011-10-31 2014-08-20 中国科学院微生物研究所 胞苷脱氨酶及其编码基因和它们的应用
MX2014010185A (es) 2012-02-22 2014-11-14 Univ Pennsylvania Uso de dominio de señalizacion cd2 en receptores de antigeno quimericos de segunda generacion.
US20160289674A1 (en) 2012-04-02 2016-10-06 Moderna Therapeutics, Inc. Modified polynucleotides for the production of membrane proteins
US20150056225A1 (en) 2012-04-17 2015-02-26 University Of Washington Through Its Center For Commercialization HLA Class II Deficient Cells, HLA Class I Deficient Cells Capable of Expressing HLA Class II Proteins, and Uses Thereof
US20150017136A1 (en) 2013-07-15 2015-01-15 Cellectis Methods for engineering allogeneic and highly active t cell for immunotherapy
EP3241902B1 (en) 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
US9102936B2 (en) 2012-06-11 2015-08-11 Agilent Technologies, Inc. Method of adaptor-dimer subtraction using a CRISPR CAS6 protein
DK3431497T3 (da) 2012-06-27 2022-10-10 Univ Princeton Spaltede inteiner, konjugater og anvendelser deraf
EP3363902B1 (en) 2012-12-06 2019-11-27 Sigma Aldrich Co. LLC Crispr-based genome modification and regulation
DK2931898T3 (en) 2012-12-12 2016-06-20 Massachusetts Inst Technology CONSTRUCTION AND OPTIMIZATION OF SYSTEMS, PROCEDURES AND COMPOSITIONS FOR SEQUENCE MANIPULATION WITH FUNCTIONAL DOMAINS
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
PL3546572T3 (pl) 2013-05-13 2024-07-22 Cellectis Chimeryczny receptor antygenowy swoisty względem cd19 i jego zastosowania
WO2014186686A2 (en) 2013-05-17 2014-11-20 Two Blades Foundation Targeted mutagenesis and genome engineering in plants using rna-guided cas nucleases
US20140356956A1 (en) 2013-06-04 2014-12-04 President And Fellows Of Harvard College RNA-Guided Transcriptional Regulation
EP3004370B1 (en) 2013-06-05 2024-08-21 Duke University Rna-guided gene editing and gene regulation
JP2016528890A (ja) 2013-07-09 2016-09-23 プレジデント アンド フェローズ オブ ハーバード カレッジ ＣＲＩＳＰＲ／Ｃａｓ系を用いるゲノム編集の治療用の使用
WO2015006294A2 (en) 2013-07-10 2015-01-15 President And Fellows Of Harvard College Orthogonal cas9 proteins for rna-guided gene regulation and editing
WO2015021426A1 (en) 2013-08-09 2015-02-12 Sage Labs, Inc. A crispr/cas system-based novel fusion protein and its application in genome editing
US9388430B2 (en) 2013-09-06 2016-07-12 President And Fellows Of Harvard College Cas9-recombinase fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
WO2015069922A2 (en) 2013-11-06 2015-05-14 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Alk antibodies, conjugates, and chimeric antigen receptors, and their use
US9994831B2 (en) 2013-12-12 2018-06-12 The Regents Of The University Of California Methods and compositions for modifying a single stranded target nucleic acid
AU2014361781B2 (en) 2013-12-12 2021-04-01 Massachusetts Institute Of Technology Delivery, use and therapeutic applications of the CRISPR -Cas systems and compositions for genome editing
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
ES2918501T3 (es) 2013-12-19 2022-07-18 Novartis Ag Receptores de antígenos quiméricos de mesotelina humana y usos de los mismos
WO2015092024A2 (en) 2013-12-20 2015-06-25 Cellectis Method of engineering multi-input signal sensitive t cell for immunotherapy
SG11201609211VA (en) 2014-03-05 2016-12-29 Nat Univ Corp Univ Kobe Genomic sequence modification method for specifically converting nucleic acid bases of targeted dna sequence, and molecular complex for use in same
MX373460B (es) 2014-03-11 2020-04-07 Cellectis Metodo para generar celulas t compatibles para el trasplante alogenico.
EP3593812A3 (en) 2014-03-15 2020-05-27 Novartis AG Treatment of cancer using chimeric antigen receptor
WO2015191693A2 (en) 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
AU2015289644A1 (en) 2014-07-15 2017-02-02 Juno Therapeutics, Inc. Engineered cells for adoptive cell therapy
ES2781175T3 (es) 2014-07-31 2020-08-31 Novartis Ag Subconjunto optimizado de células T que contienen un receptor de antígeno quimérico
JP2017522893A (ja) 2014-07-31 2017-08-17 セレクティスＣｅｌｌｅｃｔｉｓ Ｒｏｒ１特異的多重鎖キメラ抗原受容体
EP3633032A3 (en) 2014-08-28 2020-07-29 North Carolina State University Novel cas9 proteins and guiding features for dna targeting and genome editing
EP2990416B1 (en) 2014-08-29 2018-06-20 GEMoaB Monoclonals GmbH Universal chimeric antigen receptor expressing immune cells for targeting of diverse multiple antigens and method of manufacturing the same and use of the same for treatment of cancer, infections and autoimmune disorders
ES2753391T3 (es) 2014-10-14 2020-04-08 Halozyme Inc Composiciones de adenosina desaminasa 2 (ADA2), variantes de la misma y métodos de uso de las mismas
WO2016061368A1 (en) 2014-10-15 2016-04-21 The Children's Hospital Of Philadelphia Compositions and methods for treating b-lymphoid malignancies
US10258697B2 (en) 2014-10-29 2019-04-16 Massachusetts Eye And Ear Infirmary Efficient delivery of therapeutic molecules in vitro and in vivo
WO2016069282A1 (en) 2014-10-31 2016-05-06 The Trustees Of The University Of Pennsylvania Altering gene expression in modified t cells and uses thereof
US10920215B2 (en) 2014-11-04 2021-02-16 National University Corporation Kobe University Method for modifying genome sequence to introduce specific mutation to targeted DNA sequence by base-removal reaction, and molecular complex used therein
MA40921A (fr) 2014-11-05 2017-09-12 Abbvie Stemcentrx Llc Récepteurs antigéniques chimériques anti-cldn et procédés d'utilisation
US11072644B2 (en) 2014-11-12 2021-07-27 Allogene Therapeutics, Inc. Inhibitory chimeric antigen receptors
EA036379B1 (ru) 2014-12-12 2020-11-02 Блубёрд Био, Инк. Химерные антигенные рецепторы к bcma
US10676737B2 (en) 2014-12-17 2020-06-09 Proqr Therapeutics Ii B.V. Targeted RNA editing
CA2971444A1 (en) 2014-12-20 2016-06-23 Arc Bio, Llc Compositions and methods for targeted depletion, enrichment, and partitioning of nucleic acids using crispr/cas system proteins
WO2016115482A1 (en) 2015-01-16 2016-07-21 Novartis Pharma Ag Phosphoglycerate kinase 1 (pgk) promoters and methods of use for expressing chimeric antigen receptor
MA41613A (fr) 2015-02-23 2018-01-02 Abbvie Stemcentrx Llc Récepteurs antigéniques chimériques anti-dll3 et procédés d'utilisation desdits récepteurs
JP7237449B2 (ja) 2015-02-27 2023-03-13 アイセル・ジーン・セラピューティクス・エルエルシー 血液系腫瘍を標的としたキメラ抗体受容体（ＣＡＲｓ）の構成およびその使用方法
EP3265559B1 (en) 2015-03-03 2021-01-06 The General Hospital Corporation Engineered crispr-cas9 nucleases with altered pam specificity
KR20170137079A (ko) 2015-03-11 2017-12-12 셀렉티스 그것들의 지속을 증가시키기 위한 동종이계 t 세포의 조작 및/또는 환자들 내로 이식 방법들
KR20260048600A (ko) 2015-03-27 2026-04-10 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 변형된 t 세포 및 이의 제조 및 사용 방법
CA2982966C (en) 2015-04-24 2024-02-20 Editas Medicine, Inc. Evaluation of cas9 molecule/guide rna molecule complexes
EP3294342A4 (en) 2015-05-08 2018-11-07 President and Fellows of Harvard College Universal donor stem cells and related methods
WO2016183438A1 (en) 2015-05-14 2016-11-17 Massachusetts Institute Of Technology Self-targeting genome editing system
CN106011104B (zh) 2015-05-21 2019-09-27 清华大学 利用拆分Cas系统进行基因编辑和表达调控方法
JP6949728B2 (ja) 2015-05-29 2021-10-13 ジュノー セラピューティクス インコーポレイテッド 遺伝子操作された細胞における阻害相互作用を調節するための組成物および方法
EP3331582A4 (en) 2015-05-29 2019-08-07 Agenovir Corporation METHODS AND COMPOSITIONS FOR THE TREATMENT OF TRANSPLANTATION CELLS
WO2016205759A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Engineering and optimization of systems, methods, enzymes and guide scaffolds of cas9 orthologs and variants for sequence manipulation
CA3012607A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzymes and systems
US9790490B2 (en) 2015-06-18 2017-10-17 The Broad Institute Inc. CRISPR enzymes and systems
TWI813532B (zh) 2015-06-18 2023-09-01 美商博得學院股份有限公司 降低脱靶效應的ｃｒｉｓｐｒ酶突變
EP4043556B1 (en) 2015-06-30 2024-02-07 Cellectis Methods for improving functionality in nk cell by gene inactivation using specific endonuclease
US11479793B2 (en) 2015-07-15 2022-10-25 Rutgers, The State University Of New Jersey Nuclease-independent targeted gene editing platform and uses thereof
US20170020922A1 (en) 2015-07-16 2017-01-26 Batu Biologics Inc. Gene editing for immunological destruction of neoplasia
US9512446B1 (en) 2015-08-28 2016-12-06 The General Hospital Corporation Engineered CRISPR-Cas9 nucleases
WO2017040945A1 (en) * 2015-09-04 2017-03-09 Memorial Sloan Kettering Cancer Center Immune cell compositions and methods of use
WO2017048969A1 (en) 2015-09-17 2017-03-23 The Regents Of The University Of California Variant cas9 polypeptides comprising internal insertions
US20180258149A1 (en) 2015-09-17 2018-09-13 Novartis Ag Car t cell therapies with enhanced efficacy
JP7109784B2 (ja) 2015-10-23 2022-08-01 プレジデント アンド フェローズ オブ ハーバード カレッジ 遺伝子編集のための進化したＣａｓ９蛋白質
MX2018005618A (es) 2015-11-05 2018-08-01 Juno Therapeutics Inc Receptores quimericos que contienen dominios inductores de traf y composiciones y metodos relacionados.
US11020429B2 (en) 2015-11-05 2021-06-01 Juno Therapeutics, Inc. Vectors and genetically engineered immune cells expressing metabolic pathway modulators and uses in adoptive cell therapy
CA2999649A1 (en) 2015-11-06 2017-05-11 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1a
CA3006618A1 (en) 2015-12-01 2017-06-08 Crispr Therapeutics Ag Materials and methods for treatment of alpha-1 antitrypsin deficiency
EA201891338A1 (ru) 2015-12-04 2018-12-28 Новартис Аг Композиции и способы для иммуноонкологии
ES2949163T3 (es) 2016-01-29 2023-09-26 Univ Princeton Inteínas divididas con actividad de corte y empalme excepcional
KR102438360B1 (ko) 2016-03-04 2022-08-31 에디타스 메디신, 인코포레이티드 암 면역요법을 위한 crispr-cpf1-관련 방법, 조성물 및 구성성분
WO2017165862A1 (en) 2016-03-25 2017-09-28 Editas Medicine, Inc. Systems and methods for treating alpha 1-antitrypsin (a1at) deficiency
TWI773666B (zh) 2016-03-30 2022-08-11 美商英特利亞醫療公司 Crispr/cas 組分之脂質奈米粒子調配物
EP3443001B1 (en) 2016-04-11 2025-04-30 Obsidian Therapeutics, Inc. REGULATED BIOCIRCUIT SYSTEMS
WO2017180993A1 (en) 2016-04-14 2017-10-19 Bluebird Bio, Inc. Salvage chimeric antigen receptor systems
US20200263190A1 (en) 2016-04-19 2020-08-20 The Broad Institute, Inc. Novel crispr enzymes and systems
CN110382692A (zh) 2016-04-19 2019-10-25 博德研究所 新型crispr酶以及系统
BR112019000057A2 (pt) 2016-07-05 2019-04-02 The Johns Hopkins University composições e métodos com base em crispr/cas9 para o tratamento de degeneração de retina
WO2018020323A2 (en) 2016-07-25 2018-02-01 Crispr Therapeutics Ag Materials and methods for treatment of fatty acid disorders
KR102547316B1 (ko) 2016-08-03 2023-06-23 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
US11661590B2 (en) 2016-08-09 2023-05-30 President And Fellows Of Harvard College Programmable CAS9-recombinase fusion proteins and uses thereof
WO2018035388A1 (en) 2016-08-17 2018-02-22 The Broad Institute, Inc. Novel crispr enzymes and systems
US10941402B2 (en) 2016-09-01 2021-03-09 Proqr Therapeutics Ii B.V. Chemically modified single-stranded RNA-editing oligonucleotides
EP3526320A1 (en) 2016-10-14 2019-08-21 President and Fellows of Harvard College Aav delivery of nucleobase editors
KR20240005168A (ko) 2016-11-04 2024-01-11 2세븐티 바이오, 인코포레이티드 항-bcma car t 세포 조성물
WO2018089664A1 (en) 2016-11-11 2018-05-17 The Regents Of The University Of California Variant rna-guided polypeptides and methods of use
CN107043779B (zh) 2016-12-01 2020-05-12 中国农业科学院作物科学研究所 一种CRISPR/nCas9介导的定点碱基替换在植物中的应用
AU2017374044B2 (en) 2016-12-08 2023-11-30 Intellia Therapeutics, Inc. Modified guide RNAs
AU2017379073B2 (en) 2016-12-22 2023-12-14 Intellia Therapeutics, Inc. Compositions and methods for treating alpha-1 antitrypsin deficiency
GB201622044D0 (en) 2016-12-22 2017-02-08 Ucl Business Plc T cell-targeted T cells
EP3559223A1 (en) 2016-12-23 2019-10-30 President and Fellows of Harvard College Gene editing of pcsk9
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
WO2018129129A1 (en) 2017-01-05 2018-07-12 Rutgers, The State University Of New Jersey Targeted gene editing platform independent of dna double strand break and uses thereof
JP7717439B2 (ja) * 2017-01-10 2025-08-04 ザ ジェネラル ホスピタル コーポレイション 修飾されたｔ細胞及びその使用方法
EP4043485A1 (en) 2017-01-26 2022-08-17 Novartis AG Cd28 compositions and methods for chimeric antigen receptor therapy
WO2018152197A1 (en) 2017-02-15 2018-08-23 Massachusetts Institute Of Technology Dna writers, molecular recorders and uses thereof
SG11201907870VA (en) 2017-02-28 2019-09-27 Vor Biopharma Inc Compositions and methods for inhibition of lineage specific proteins
JP2020510038A (ja) * 2017-03-09 2020-04-02 プレジデント アンド フェローズ オブ ハーバード カレッジ がんワクチン
JP2020510439A (ja) 2017-03-10 2020-04-09 プレジデント アンド フェローズ オブ ハーバード カレッジ シトシンからグアニンへの塩基編集因子
CA3057192A1 (en) 2017-03-23 2018-09-27 President And Fellows Of Harvard College Nucleobase editors comprising nucleic acid programmable dna binding proteins
WO2018183888A2 (en) 2017-03-31 2018-10-04 The Board Of Trustees Of The Leland Stanford Junior University Methods of treating t cell exhaustion by inhibiting or modulating t cell receptor signaling
CN106916852B (zh) 2017-04-13 2020-12-04 上海科技大学 一种碱基编辑系统及其构建和应用方法
WO2018195545A2 (en) 2017-04-21 2018-10-25 The General Hospital Corporation Variants of cpf1 (cas12a) with altered pam specificity
US20200078404A1 (en) * 2017-05-01 2020-03-12 Juno Therapeutics, Inc. Combination of a cell therapy and an immunomodulatory compound
EP3625342B1 (en) 2017-05-18 2022-08-24 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
WO2018213708A1 (en) 2017-05-18 2018-11-22 The Broad Institute, Inc. Systems, methods, and compositions for targeted nucleic acid editing
WO2018218066A1 (en) 2017-05-24 2018-11-29 The Regents Of The University Of California Cmv vaccine and method of making and using the same
US20200172895A1 (en) 2017-05-25 2020-06-04 The General Hospital Corporation Using split deaminases to limit unwanted off-target base editor deamination
JP2020525537A (ja) 2017-06-12 2020-08-27 エモリー ユニバーシティー Ｔ細胞抗原標的キメラ抗原受容体（ｃａｒ）、及び細胞療法での使用
AU2018290843B2 (en) 2017-06-26 2025-04-24 Massachusetts Institute Of Technology CRISPR/Cas-adenine deaminase based compositions, systems, and methods for targeted nucleic acid editing
WO2019005886A1 (en) 2017-06-26 2019-01-03 The Broad Institute, Inc. CRISPR / CAS-CYTIDINE DEAMINASE COMPOSITIONS, SYSTEMS AND METHODS FOR TARGETED EDITING OF NUCLEIC ACIDS
US11168322B2 (en) 2017-06-30 2021-11-09 Arbor Biotechnologies, Inc. CRISPR RNA targeting enzymes and systems and uses thereof
JP7210534B2 (ja) 2017-07-12 2023-01-23 ザ・ボード・オブ・トラスティーズ・オブ・ザ・リーランド・スタンフォード・ジュニア・ユニバーシティ Ｈ３ｋ２７ｍ変異を有するがんの処置のための組成物および方法
US11740242B2 (en) 2017-07-14 2023-08-29 Dana-Farber Cancer Institute, Inc. Modulating biomarkers to increase tumor immunity and improve the efficacy of cancer immunotherapy
WO2019023680A1 (en) 2017-07-28 2019-01-31 President And Fellows Of Harvard College METHODS AND COMPOSITIONS FOR EVOLUTION OF BASIC EDITORS USING PHAGE-ASSISTED CONTINUOUS EVOLUTION (PACE)
CN118530967A (zh) 2017-08-23 2024-08-23 通用医疗公司 具有改变的PAM特异性的工程化的CRISPR-Cas9核酸酶
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
EP4389889A3 (en) 2017-10-06 2024-09-04 Oregon Health & Science University Compositions and methods for editing rna
KR20250107288A (ko) * 2017-10-16 2025-07-11 더 브로드 인스티튜트, 인코퍼레이티드 아데노신 염기 편집제의 용도
US11400117B2 (en) 2017-12-15 2022-08-02 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods for inhibiting T cell exhaustion
CN109957569B (zh) 2017-12-22 2022-10-25 苏州齐禾生科生物科技有限公司 基于cpf1蛋白的碱基编辑系统和方法
CN112534044A (zh) * 2018-02-16 2021-03-19 凯德药业股份有限公司 经修饰的多能干细胞及制备和使用方法
CN112292139B (zh) * 2018-03-13 2024-03-08 明尼苏达大学董事会 使用Cas9碱基编辑器的淋巴造血改造
WO2019183000A1 (en) 2018-03-19 2019-09-26 University Of Massachusetts Modified guide rnas for crispr genome editing
EP3781176A4 (en) 2018-04-09 2022-05-25 The Trustees of the University of Pennsylvania METHODS AND COMPOSITIONS USING A VIRAL VECTOR FOR THE EXPRESSION OF A TRANSGENE AND AN EFFECTOR
US12473342B2 (en) 2018-04-27 2025-11-18 The Trustees Of The University Of Pennsylvania Chimeric antigen receptor T regulatory cells for the treatment of atherosclerosis
JP7558929B2 (ja) 2018-05-11 2024-10-01 ビーム セラピューティクス インク． プログラム可能塩基エディターシステムを用いて病原性変異を抑制する方法
AU2019266327B2 (en) 2018-05-11 2025-11-06 Beam Therapeutics Inc. Methods of editing single nucleotide polymorphism using programmable base editor systems
CN121555430A (zh) 2018-05-11 2026-02-24 比姆医疗股份有限公司 使用可编程碱基编辑器系统取代病原性氨基酸的方法
EP3797160A1 (en) 2018-05-23 2021-03-31 The Broad Institute Inc. Base editors and uses thereof
CN110616189B (zh) 2018-06-20 2022-02-18 西安桑尼赛尔生物医药有限公司 通用型靶向cd19抗原嵌合受体t细胞的制备及其应用
CN108753823B (zh) 2018-06-20 2022-09-23 李广磊 利用碱基编辑技术实现基因敲除的方法及其应用
WO2020010239A1 (en) 2018-07-06 2020-01-09 The Board Of Trustees Of The Leland Stanford Junior University Chimeric antigen receptor polypeptides and methods of using same
CN108949825A (zh) 2018-07-30 2018-12-07 苏州茂行生物科技有限公司 一种靶向her2的car-t细胞的制备方法及应用
AU2019316094B2 (en) 2018-08-03 2026-02-19 Beam Therapeutics Inc. Multi-effector nucleobase editors and methods of using same to modify a nucleic acid target sequence
EP3841203A4 (en) 2018-08-23 2022-11-02 The Broad Institute Inc. CAS9 VARIANTS WITH NON-CANONICAL PAM SPECIFICITIES AND THEIR USES
KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
CN109295186B (zh) 2018-09-30 2023-10-03 中山大学 一种基于全基因组测序检测腺嘌呤单碱基编辑系统脱靶效应的方法及其在基因编辑中的应用
US20220023348A1 (en) 2018-11-28 2022-01-27 Forty Seven, Inc. Genetically modified hspcs resistant to ablation regime
US20220047637A1 (en) 2018-11-29 2022-02-17 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
CA3121911A1 (en) 2018-12-06 2020-06-11 The Board Of Trustees Of The Leland Stanford Junior University Regulatable cell surface receptors and related compositions and methods
WO2020132327A1 (en) 2018-12-19 2020-06-25 The Trustees Of The University Of Pennsylvania Use of cd2/5/7 knock-out anti-cd2/5/7 chimeric antigen receptor t cells against t cell lymphomas and leukemias
US20220133790A1 (en) 2019-01-16 2022-05-05 Beam Therapeutics Inc. Modified immune cells having enhanced anti-neoplasia activity and immunosuppression resistance
CN109706121A (zh) * 2019-01-25 2019-05-03 苏州茂行生物科技有限公司 一种基于碱基编辑的通用型car-t细胞及其制备方法和应用
KR20210121113A (ko) 2019-01-31 2021-10-07 빔 테라퓨틱스, 인크. 비-표적 탈아미노화가 감소된 핵염기 편집기 및 핵염기 편집기를 특성규명하기 위한 분석
EP3918077A4 (en) 2019-01-31 2023-03-29 Beam Therapeutics, Inc. NUCLEOBASE EDITORS WITH REDUCED OFF-TARGET DEAMINATION AND METHODS OF USING THEM TO MODIFY A NUCLEOBASE TARGET SEQUENCE
US11946040B2 (en) 2019-02-04 2024-04-02 The General Hospital Corporation Adenine DNA base editor variants with reduced off-target RNA editing
WO2020168122A1 (en) 2019-02-13 2020-08-20 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
KR20210126680A (ko) 2019-02-13 2021-10-20 빔 테라퓨틱스, 인크. 알파-1 항트립신 결핍증을 치료하기 위한 조성물 및 방법
AU2020223306A1 (en) 2019-02-13 2021-08-05 Beam Therapeutics Inc. Methods of editing a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease
US20220235347A1 (en) 2019-02-13 2022-07-28 Beam Therapeutics Inc. Compositions and methods for treating hemoglobinopathies
US20220127594A1 (en) 2019-02-13 2022-04-28 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
US20220098593A1 (en) 2019-02-13 2022-03-31 Beam Therapeutics Inc. Splice acceptor site disruption of a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease
KR20210139265A (ko) 2019-02-13 2021-11-22 빔 테라퓨틱스, 인크. 표적 서열에서 핵염기를 변형하기 위한 아데노신 데아미나제 염기 편집기 및 이의 사용 방법
EP3924467A1 (en) 2019-02-15 2021-12-22 Editas Medicine, Inc. Modified natural killer (nk) cells for immunotherapy
WO2020176897A1 (en) 2019-02-28 2020-09-03 The Board Of Trustees Of The Leland Stanford Junior University Chimeric antigen receptors and uses thereof
JP7654557B2 (ja) 2019-03-27 2025-04-01 ザ トラスティーズ オブ ザ ユニバーシティ オブ ペンシルバニア Ｔｎ－ＭＵＣ１キメラ抗原受容体（ＣＡＲ）Ｔ細胞療法
US12473543B2 (en) 2019-04-17 2025-11-18 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CA3139319A1 (en) 2019-05-07 2020-11-12 The Board Of Trustees Of The Leland Stanford Junior University Enhancement of polypeptides and chimeric antigen receptors via hinge domains
WO2020227447A1 (en) 2019-05-07 2020-11-12 The Board Of Trustees Of The Leland Stanford Junior University Chimeric antigen receptors targeting glypican-2
US20200370013A1 (en) 2019-05-20 2020-11-26 The Trustees Of The University Of Pennsylvania Engineered Expression of Cell Surface and Secreted Sialidase by CAR T Cells for Increased Efficacy in Solid Tumors
WO2020236982A1 (en) 2019-05-20 2020-11-26 The Broad Institute, Inc. Aav delivery of nucleobase editors
US20220387622A1 (en) 2019-05-21 2022-12-08 Beam Therapeutics Inc. Methods of editing a single nucleotide polymorphism using programmable base editor systems
CN118852456A (zh) 2019-05-29 2024-10-29 Cue生物制药股份有限公司 多聚体t细胞调节多肽及其使用方法
AU2020322021A1 (en) 2019-07-30 2022-02-10 Pairwise Plants Services, Inc. Morphogenic regulators and methods of using the same
KR102258713B1 (ko) 2019-07-31 2021-05-31 한양대학교 산학협력단 사이토신 염기교정용 조성물 및 이의 용도
US20230017979A1 (en) 2019-08-29 2023-01-19 Beam Therapeutics Inc. Compositions and methods for non-toxic conditioning
WO2021042062A2 (en) 2019-08-30 2021-03-04 Joung J Keith Combinatorial adenine and cytosine dna base editors
CN114667149A (zh) 2019-09-09 2022-06-24 比姆医疗股份有限公司 新型核碱基编辑器及其使用方法
JP7814052B2 (ja) 2019-09-17 2026-02-16 ラトガース，ザ ステート ユニバーシティ オブ ニュー ジャージー 標的ゲノム修飾のための高度に効率的なｒｎａ－アプタマー動員媒介性ｄｎａ塩基エディターおよびそれらの使用
EP4034138A4 (en) 2019-09-27 2024-07-31 Beam Therapeutics, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF LIQUID CANCERS
KR20220095193A (ko) 2019-10-11 2022-07-06 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 조절 가능한 세포 국재화를 위한 재조합 폴리펩티드
US11591607B2 (en) 2019-10-24 2023-02-28 Pairwise Plants Services, Inc. Optimized CRISPR-Cas nucleases and base editors and methods of use thereof
KR20220110739A (ko) 2019-10-30 2022-08-09 페어와이즈 플랜츠 서비시즈, 인크. 유형 v crispr-cas 염기 편집제 및 그의 사용 방법
US20220380447A1 (en) 2019-11-01 2022-12-01 The Trustees Of The University Of Pennsylvania Fibronectin targeting chimeric antigen receptors (cars)
CN120905142A (zh) 2019-11-20 2025-11-07 卡瑟里克斯私人有限公司 用于提供具有增强功能的免疫细胞的方法
US20230086199A1 (en) 2019-11-26 2023-03-23 The Broad Institute, Inc. Systems and methods for evaluating cas9-independent off-target editing of nucleic acids
EP4076479A4 (en) 2019-12-18 2024-05-22 Editas Medicine, Inc. GENETICALLY MODIFIED CELLS FOR THERAPY
US20210230246A1 (en) 2020-01-15 2021-07-29 The Trustees Of The University Of Pennsylvania Use of inhibitory chimeric receptors to prevent t cell-induced blood brain barrier damage
WO2021158921A2 (en) 2020-02-05 2021-08-12 The Broad Institute, Inc. Adenine base editors and uses thereof
WO2021158999A1 (en) 2020-02-05 2021-08-12 The Broad Institute, Inc. Gene editing methods for treating spinal muscular atrophy
CA3167572A1 (en) 2020-02-14 2021-08-19 Robbie G. MAJZNER Chimeric antigen receptors with cd2 activation
US20230313234A1 (en) 2020-03-04 2023-10-05 Suzhou Qi Biodesign Biotechnology Company Limited Improved cytosine base editing system
AU2021236683A1 (en) 2020-03-19 2022-11-17 Intellia Therapeutics, Inc. Methods and compositions for directed genome editing
WO2021207651A2 (en) 2020-04-09 2021-10-14 Verve Therapeutics, Inc. Chemically modified guide rnas for genome editing with cas12b
EP4179079A1 (en) 2020-07-10 2023-05-17 Horizon Discovery Limited Method for producing genetically modified cells
JP2023534693A (ja) 2020-07-15 2023-08-10 ライフエディット セラピューティクス，インコーポレイティド ウラシル安定化タンパク質並びにその活性断片及びバリアント並びに使用方法
CA3192224A1 (en) 2020-09-11 2022-03-17 Jyun-Liang LIN Base editing enzymes
TWI910226B (zh) 2020-09-11 2026-01-01 美商生命編輯治療學公司 Ｄｎａ修飾酶及活性片段，及其變異體與使用方法
WO2022061115A1 (en) * 2020-09-18 2022-03-24 Vor Biopharma Inc. Compositions and methods for cd7 modification
AU2021347359A1 (en) 2020-09-25 2023-05-18 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
US20230383277A1 (en) 2020-10-14 2023-11-30 Beam Therapeutics Inc. Compositions and methods for treating glycogen storage disease type 1a
IT202000028688A1 (it) 2020-11-27 2022-05-27 Consiglio Nazionale Ricerche Varianti della citidina deaminasi per l’editazione di basi
JP2024502348A (ja) 2021-01-05 2024-01-18 ホライズン・ディスカバリー・リミテッド Ｔｒａｃｒ不含 Ｖ型Ｃａｓシステム用のガイドＲＮＡデザインおよび複合体
WO2022150372A1 (en) 2021-01-05 2022-07-14 Horizon Discovery Ltd. Guide rna designs and complexes for type v cas systems
CA3207144A1 (en) 2021-01-05 2022-07-14 Horizon Discovery Limited Method for producing genetically modified cells
EP4274897A4 (en) 2021-01-08 2025-03-05 The General Hospital Corporation GENOMEDITING APPROACHES TO THE TREATMENT OF SPINAL MUSCLE ATROPHY
WO2022178307A1 (en) 2021-02-19 2022-08-25 Beam Therapeutics Inc. Recombinant rabies viruses for gene therapy
CA3214494A1 (en) 2021-03-26 2022-09-29 Beam Therapeutics Inc. Adenosine deaminase variants and uses thereof
AU2022299551A1 (en) 2021-06-23 2024-01-18 Editas Medicine, Inc. Engineered cells for therapy
US20240327859A1 (en) 2021-07-02 2024-10-03 University Of Maryland, College Park Cytidine deaminases and methods of genome editing using the same
AU2022311013A1 (en) 2021-07-16 2024-02-08 President And Fellows Of Harvard College Context-specific adenine base editors and uses thereof
CA3228879A1 (en) 2021-08-16 2023-02-23 Beam Therapeutics Inc. Persistent allogeneic modified immune cells and methods of use thereof
US20240352439A1 (en) 2021-09-03 2024-10-24 The University Of Chicago Polypeptides and methods for modifying nucleic acids
AU2022349449A1 (en) 2021-09-22 2024-04-18 Verve Therapeutics, Inc. Gene editing of pcsk9 or angptl3 and compositions and methods of using same for treatment of disease
WO2023047338A1 (en) 2021-09-24 2023-03-30 Crispr Therapeutics Ag PRODRUG INCORPORATED sgRNA SYNTHESIS
CA3242360A1 (en) 2021-12-10 2023-06-15 Beam Therapeutics Inc. Modified immune cells and methods of using the same
WO2023125814A1 (zh) 2021-12-29 2023-07-06 华东师范大学 腺嘌呤脱氨酶及其应用
CN119013310A (zh) 2022-02-17 2024-11-22 正序(上海)生物科技有限公司 具有改善的编辑精确度的突变的胞嘧啶脱氨酶
CN117925585A (zh) 2022-04-07 2024-04-26 尧唐(上海)生物科技有限公司 腺苷脱氨酶、碱基编辑器融合蛋白、碱基编辑器系统及用途
WO2023227669A2 (en) 2022-05-26 2023-11-30 UCB Biopharma SRL Novel nucleic acid-editing proteins
EP4532697A2 (en) 2022-06-03 2025-04-09 Beam Therapeutics Inc. Modified regulatory t cells and methods of using the same
EP4540378A1 (en) 2022-06-20 2025-04-23 CRISPR Therapeutics AG Base editing proteins and uses thereof
AU2023288909A1 (en) 2022-06-23 2025-01-09 Basf Agricultural Solutions Us Llc Diversifying base editing
WO2024006772A2 (en) 2022-06-27 2024-01-04 Beam Therapeutics Inc. Adenosine deaminase base editors and methods for use thereof
CA3263821A1 (en) 2022-08-16 2024-02-22 The Broad Institute, Inc. ADVANCED CYTOSIN DESAMINASE AND DNA EDITING METHODS USING IT
CN119998447A (zh) 2022-09-23 2025-05-13 基础科学研究院 新型腺嘌呤脱氨酶变体及使用其进行碱基编辑的方法
CA3267375A1 (en) 2022-09-26 2024-04-04 Beam Therapeutics Inc. SYNTHETIC POLYPEPTIDES AND THEIR USES
WO2024179426A2 (en) 2023-02-28 2024-09-06 Accuredit Therapeutics (Suzhou) Co., Ltd. Deaminases for use in base editing
WO2024226156A1 (en) 2023-04-27 2024-10-31 University Of Massachusetts Cas-embedded cytidine deaminase ribonucleoprotein complexes having improved base editing specificity and efficiency
WO2024227047A2 (en) 2023-04-28 2024-10-31 Beam Therapeutics Inc. Modified guide rna
CN121889499A (zh) 2023-06-15 2026-04-17 比姆医疗股份有限公司 用于治疗α1-抗胰蛋白酶缺乏症的方法和组合物
CN117965505A (zh) 2023-06-28 2024-05-03 微光基因(苏州)有限公司 工程化的腺苷脱氨酶及碱基编辑器

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2019524156A (ja) 2016-08-03 2019-09-05 ジョン・エフ・ディパージオＪｏｈｎ Ｆ． ＤＩＰＥＲＳＩＯ キメラ抗原受容体でのｔ細胞性悪性腫瘍の治療のためのｃａｒ−ｔ細胞の遺伝子編集

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Steven M. Banik et. al.，Lysosome-targeting chimaeras for degradation of extracellular proteins，Nature，2020年07月29日，Vol 584，Page 291-297, Methods, Fig.1-9, etc.，https://doi.org/10.1038/s41586-020-2545-9

Also Published As

Publication number Publication date
WO2021062227A2 (en) 2021-04-01
CA3155667A1 (en) 2021-04-01
US20230021636A1 (en) 2023-01-26
JP2022549916A (ja) 2022-11-29
JP2025066723A (ja) 2025-04-23
CN115279398A (zh) 2022-11-01
CN120905310A (zh) 2025-11-07
EP4034138A4 (en) 2024-07-31
KR20220090512A (ko) 2022-06-29
US12594301B2 (en) 2026-04-07
WO2021062227A3 (en) 2021-05-06
EP4034138A2 (en) 2022-08-03
AU2020355222A1 (en) 2022-03-24
CN115279398B (zh) 2025-07-04

Similar Documents

Publication Publication Date Title
JP7615134B2 (ja) 液体がんの治療のための組成物および方法
JP7672982B2 (ja) 標的配列中の核酸塩基を改変するためのアデノシンデアミナーゼ塩基エディターを有する改変された免疫細胞
AU2020336969B2 (en) Compositions and methods for non-toxic conditioning
US11155803B2 (en) Adenosine deaminase base editors and methods of using same to modify a nucleobase in a target sequence
CN114072509B (zh) 脱氨反应脱靶减低的核碱基编辑器和使用其修饰核碱基靶序列的方法
US11952408B2 (en) HPV-specific binding molecules
US20240287453A1 (en) Persistent allogeneic modified immune cells and methods of use thereof
US12227776B2 (en) Engineered cascade components and cascade complexes
JP2021523739A (ja) プログラム可能な塩基エディターシステムを用いて単一ヌクレオチド多型を編集する方法
US12576151B2 (en) Fratricide resistant modified immune cells and methods of using the same
US20230083383A1 (en) Compositions and methods for targeting, editing or modifying human genes
US20240325533A1 (en) Modified immune cells and methods of using the same
US20250034558A1 (en) Compositions and methods for targeting, editing or modifying human genes
CN118234865A (zh) 持久性同种异体修饰免疫细胞及其使用方法
US20260071197A1 (en) Engineered cascade components and cascade complexes
EP4388097A1 (en) Method

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20230922

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20230922

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20241203

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20241227

R150 Certificate of patent or registration of utility model

Ref document number: 7615134

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150