AU2017226172B9 - CRISPR/Cpf1-related methods, compositions and components for cancer immunotherapy - Google Patents

CRISPR/Cpf1-related methods, compositions and components for cancer immunotherapy Download PDF

Info

Publication number
AU2017226172B9
AU2017226172B9 AU2017226172A AU2017226172A AU2017226172B9 AU 2017226172 B9 AU2017226172 B9 AU 2017226172B9 AU 2017226172 A AU2017226172 A AU 2017226172A AU 2017226172 A AU2017226172 A AU 2017226172A AU 2017226172 B9 AU2017226172 B9 AU 2017226172B9
Authority
AU
Australia
Prior art keywords
cell
certain embodiments
cpfl
molecule
grna
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
AU2017226172A
Other languages
English (en)
Other versions
AU2017226172A2 (en
AU2017226172A1 (en
AU2017226172B2 (en
Inventor
Christopher BORGES
Hariharan JAYARAM
Tongyao WANG
Gordon Grant WELSTEAD
John Anthony Zuris
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.)
Editas Medicine Inc
Original Assignee
Editas Medicine Inc
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 Editas Medicine Inc filed Critical Editas Medicine Inc
Publication of AU2017226172A1 publication Critical patent/AU2017226172A1/en
Publication of AU2017226172A2 publication Critical patent/AU2017226172A2/en
Application granted granted Critical
Publication of AU2017226172B2 publication Critical patent/AU2017226172B2/en
Publication of AU2017226172B9 publication Critical patent/AU2017226172B9/en
Priority to AU2023266284A priority Critical patent/AU2023266284B2/en
Priority to AU2026206148A priority patent/AU2026206148A1/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • 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
    • 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
    • A61K35/14—Blood; Artificial blood
    • A61K35/17—Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
    • 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
    • A61K40/30—Cellular immunotherapy characterised by the recombinant expression of specific molecules in the cells of the immune system
    • A61K40/31—Chimeric antigen receptors [CAR]
    • 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
    • A61K40/32—T-cell receptors [TCR]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • 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
    • 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
    • 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
    • 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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian 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
    • 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
    • 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]
    • 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]
    • 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
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • 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
    • 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
    • C12N2800/80—Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Hematology (AREA)
  • Epidemiology (AREA)
  • Virology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Developmental Biology & Embryology (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Oncology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
AU2017226172A 2016-03-04 2017-03-03 CRISPR/Cpf1-related methods, compositions and components for cancer immunotherapy Active AU2017226172B9 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU2023266284A AU2023266284B2 (en) 2016-03-04 2023-11-15 CRISPR/Cpf1-related methods, compositions and components for cancer immunotherapy
AU2026206148A AU2026206148A1 (en) 2016-03-04 2026-07-16 CRISPR/Cpf1-related methods, compositions and components for cancer immunotherapy

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201662304057P 2016-03-04 2016-03-04
US62/304,057 2016-03-04
PCT/US2017/020598 WO2017152015A1 (en) 2016-03-04 2017-03-03 Crispr-cpf1-related methods, compositions and components for cancer immunotherapy

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2023266284A Division AU2023266284B2 (en) 2016-03-04 2023-11-15 CRISPR/Cpf1-related methods, compositions and components for cancer immunotherapy

Publications (4)

Publication Number Publication Date
AU2017226172A1 AU2017226172A1 (en) 2018-09-27
AU2017226172A2 AU2017226172A2 (en) 2018-10-04
AU2017226172B2 AU2017226172B2 (en) 2023-08-17
AU2017226172B9 true AU2017226172B9 (en) 2023-08-24

Family

ID=58358928

Family Applications (3)

Application Number Title Priority Date Filing Date
AU2017226172A Active AU2017226172B9 (en) 2016-03-04 2017-03-03 CRISPR/Cpf1-related methods, compositions and components for cancer immunotherapy
AU2023266284A Active AU2023266284B2 (en) 2016-03-04 2023-11-15 CRISPR/Cpf1-related methods, compositions and components for cancer immunotherapy
AU2026206148A Pending AU2026206148A1 (en) 2016-03-04 2026-07-16 CRISPR/Cpf1-related methods, compositions and components for cancer immunotherapy

Family Applications After (2)

Application Number Title Priority Date Filing Date
AU2023266284A Active AU2023266284B2 (en) 2016-03-04 2023-11-15 CRISPR/Cpf1-related methods, compositions and components for cancer immunotherapy
AU2026206148A Pending AU2026206148A1 (en) 2016-03-04 2026-07-16 CRISPR/Cpf1-related methods, compositions and components for cancer immunotherapy

Country Status (11)

Country Link
US (2) US12325854B2 (cg-RX-API-DMAC10.html)
EP (1) EP3423580A1 (cg-RX-API-DMAC10.html)
JP (3) JP7251980B2 (cg-RX-API-DMAC10.html)
KR (4) KR102738283B1 (cg-RX-API-DMAC10.html)
CN (1) CN109312340A (cg-RX-API-DMAC10.html)
AU (3) AU2017226172B9 (cg-RX-API-DMAC10.html)
CA (1) CA3016331A1 (cg-RX-API-DMAC10.html)
IL (2) IL291971B2 (cg-RX-API-DMAC10.html)
MX (2) MX2018010484A (cg-RX-API-DMAC10.html)
SG (1) SG11201807538PA (cg-RX-API-DMAC10.html)
WO (1) WO2017152015A1 (cg-RX-API-DMAC10.html)

Families Citing this family (99)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013066438A2 (en) 2011-07-22 2013-05-10 President And Fellows Of Harvard College Evaluation and improvement of nuclease cleavage specificity
US10704021B2 (en) 2012-03-15 2020-07-07 Flodesign Sonics, Inc. Acoustic perfusion devices
US9163284B2 (en) 2013-08-09 2015-10-20 President And Fellows Of Harvard College Methods for identifying a target site of a Cas9 nuclease
US9359599B2 (en) 2013-08-22 2016-06-07 President And Fellows Of Harvard College Engineered transcription activator-like effector (TALE) domains and uses thereof
US9340799B2 (en) 2013-09-06 2016-05-17 President And Fellows Of Harvard College MRNA-sensing switchable gRNAs
US9322037B2 (en) 2013-09-06 2016-04-26 President And Fellows Of Harvard College Cas9-FokI fusion proteins and uses thereof
US9526784B2 (en) 2013-09-06 2016-12-27 President And Fellows Of Harvard College Delivery system for functional nucleases
US20150166984A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting alpha-antitrypsin point mutations
CN105939767B (zh) 2014-01-08 2018-04-06 弗洛设计声能学公司 具有双声电泳腔的声电泳装置
AU2015298571B2 (en) 2014-07-30 2020-09-03 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
US11708572B2 (en) 2015-04-29 2023-07-25 Flodesign Sonics, Inc. Acoustic cell separation techniques and processes
US11377651B2 (en) 2016-10-19 2022-07-05 Flodesign Sonics, Inc. Cell therapy processes utilizing acoustophoresis
WO2017070632A2 (en) 2015-10-23 2017-04-27 President And Fellows Of Harvard College Nucleobase editors and uses thereof
US11214789B2 (en) 2016-05-03 2022-01-04 Flodesign Sonics, Inc. Concentration and washing of particles with acoustics
WO2018027078A1 (en) 2016-08-03 2018-02-08 President And Fellows Of Harard College Adenosine nucleobase editors and uses thereof
AU2017308889B2 (en) 2016-08-09 2023-11-09 President And Fellows Of Harvard College Programmable Cas9-recombinase fusion proteins and uses thereof
IL264439B2 (en) 2016-08-17 2024-08-01 Factor Bioscience Inc non-viral, cell-free composition comprising a synthetic messenger RNA (MRNA) encoding a gene-editing protein for use in treating cancer, and a synthetic RNA encoding a gene-editing protein for use in treatment
WO2018039438A1 (en) 2016-08-24 2018-03-01 President And Fellows Of Harvard College Incorporation of unnatural amino acids into proteins using base editing
KR20240007715A (ko) 2016-10-14 2024-01-16 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 핵염기 에디터의 aav 전달
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
WO2018165504A1 (en) 2017-03-09 2018-09-13 President And Fellows Of Harvard College Suppression of pain by gene editing
KR20190123328A (ko) 2017-03-09 2019-10-31 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 암 백신
CN110914310A (zh) 2017-03-10 2020-03-24 哈佛大学的校长及成员们 胞嘧啶至鸟嘌呤碱基编辑器
IL306092A (en) 2017-03-23 2023-11-01 Harvard College Nucleic base editors that include nucleic acid programmable DNA binding proteins
CN108690845B (zh) 2017-04-10 2021-04-27 中国科学院动物研究所 基因组编辑系统和方法
JP2020517247A (ja) 2017-04-18 2020-06-18 イエール ユニバーシティ Ｔリンパ球ゲノム操作のためのプラットフォームおよびそのインビボハイスループットスクリーニング法
US11560566B2 (en) 2017-05-12 2023-01-24 President And Fellows Of Harvard College Aptazyme-embedded guide RNAs for use with CRISPR-Cas9 in genome editing and transcriptional activation
EP3658573A1 (en) 2017-07-28 2020-06-03 President and Fellows of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (pace)
EP3676376B1 (en) 2017-08-30 2025-01-15 President and Fellows of Harvard College High efficiency base editors comprising gam
EP3686275A4 (en) * 2017-09-18 2021-09-29 Edigene Inc. GENE EDITING LYMPHOCYTE T AND RELATED USE
CA3082251A1 (en) 2017-10-16 2019-04-25 The Broad Institute, Inc. Uses of adenosine base editors
JP2021502077A (ja) * 2017-11-06 2021-01-28 エディタス・メディシン，インコーポレイテッド 免疫療法のためのｔ細胞におけるｃｂｌｂのｃｒｉｓｐｒ−ｃａｓ９編集のための方法、組成物および構成要素
SG11202004529XA (en) * 2017-11-16 2020-06-29 Mogam Inst Biomedical Res Transformed human cell and use thereof
AU2018383712B2 (en) * 2017-12-11 2025-09-04 Editas Medicine, Inc. Cpf1-related methods and compositions for gene editing
CN114900773A (zh) 2017-12-14 2022-08-12 弗洛设计声能学公司 声泳系统及其操作方法、控制声换能器及声学系统的方法
US12406749B2 (en) 2017-12-15 2025-09-02 The Broad Institute, Inc. Systems and methods for predicting repair outcomes in genetic engineering
WO2019173942A1 (en) * 2018-03-12 2019-09-19 Nanjing Bioheng Biotech Co., Ltd Engineered chimeric guide rna and uses thereof
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
CA3096708A1 (en) 2018-04-18 2019-10-24 Yale University Compositions and methods for multiplexed tumor vaccination with endogenous gene activation
WO2019226953A1 (en) 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
US20210363206A1 (en) * 2018-06-18 2021-11-25 The Regents Of The University Of California Proteins that inhibit cas12a (cpf1), a cripr-cas nuclease
EP3820495A4 (en) 2018-07-09 2022-07-20 The Broad Institute Inc. RNA-PROGRAMMABLE EPIGENETIC RNA MODIFIERS AND THEIR USES
US20210322473A1 (en) * 2018-07-18 2021-10-21 The General Hospital Corporation Modified t cells and methods of their use
US20200080056A1 (en) * 2018-09-06 2020-03-12 The Trustees Of The University Of Pennsylvania CRISPR-Cas9 Knock-out of SHP-1/2 to Reduce T cell Exhaustion in Adoptive Cell Therapy
AU2019341365A1 (en) * 2018-09-21 2021-05-27 Crage Medical Co., Limited Method for gene editing of cell on the basis of CRISPR/Cas system
CA3116132A1 (en) 2018-10-16 2020-04-23 Intellia Therapeutics, Inc. Compositions and methods for immunotherapy
WO2020092453A1 (en) 2018-10-29 2020-05-07 The Broad Institute, Inc. Nucleobase editors comprising geocas9 and uses thereof
CN121759517A (zh) * 2019-01-04 2026-03-31 赋生元公司 使用单链环状dna靶向修饰基因组
US12351837B2 (en) 2019-01-23 2025-07-08 The Broad Institute, Inc. Supernegatively charged proteins and uses thereof
US12600971B2 (en) 2019-02-13 2026-04-14 Beam Therapeutics Inc. Modified immune cells having adenosine deaminase base editors for modifying a nucleobase in a target sequence
US10913941B2 (en) 2019-02-14 2021-02-09 Metagenomi Ip Technologies, Llc Enzymes with RuvC domains
BR112021016046A2 (pt) 2019-02-15 2021-11-09 Editas Medicine Inc Células assassinas naturais modificadas (nk) para imunoterapia
KR20210137085A (ko) 2019-03-05 2021-11-17 엔카르타, 인크. Cd19 유도된 키메라 항원 수용체 및 면역 요법에서 이의 용도
WO2020191241A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020190925A1 (en) 2019-03-19 2020-09-24 Seattle Children's Hospital (dba Seattle Children's Research Institute) Methods and compositions for engineering cd4-deficient car t cells and anti-cd4 car t cells and uses thereof
WO2020214842A1 (en) 2019-04-17 2020-10-22 The Broad Institute, Inc. Adenine base editors with reduced off-target effects
CN114174325A (zh) * 2019-06-04 2022-03-11 恩卡尔塔公司 用于免疫疗法的工程化自然杀伤细胞和工程化t细胞的组合
WO2020264117A1 (en) * 2019-06-26 2020-12-30 Integrated Dna Technologies, Inc. Compositions and methods for improved detection of genomic editing events
CA3145425A1 (en) 2019-07-03 2021-01-07 Factor Bioscience Inc. Cationic lipids and uses thereof
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
AU2020324386B2 (en) * 2019-08-02 2026-04-23 Monsanto Technology Llc Methods and compositions to promote targeted genome modifications using HUH endonucleases
US20220340966A1 (en) * 2019-09-09 2022-10-27 Oregon Health & Science University Crispr-mediated capture of nucleic acids
WO2021062267A1 (en) * 2019-09-26 2021-04-01 Nantbio, Inc. Primary t-cell expansion
US12594301B2 (en) 2019-09-27 2026-04-07 Beam Therapeutics Inc. Compositions and methods for treatment of liquid cancers
US12435330B2 (en) 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA
CN110904239B (zh) * 2019-12-25 2020-11-10 武汉博杰生物医学科技有限公司 肺癌相关分子标志物基因突变的检测试剂盒与检测方法
AU2021216418A1 (en) * 2020-02-05 2022-09-01 Danmarks Tekniske Universitet Compositions and methods for targeting, editing or modifying human genes
CN116096892A (zh) * 2020-05-08 2023-05-09 宏基因组学公司 具有RuvC结构域的酶
CA3177481A1 (en) 2020-05-08 2021-11-11 David R. Liu Methods and compositions for simultaneous editing of both strands of a target double-stranded nucleotide sequence
JP2023525579A (ja) * 2020-05-15 2023-06-16 アールエックスセル インコーポレーテッド 低免疫原性の細胞および免疫応答におけるそれらの使用
JP2023527145A (ja) * 2020-05-20 2023-06-27 ジ アドミニストレイターズ オブ ザ チューレン エデュケイショナル ファンド 試料中の病原体を検出するｃｒｉｓｐｒベースアッセイ
MX2022015586A (es) 2020-06-12 2023-03-15 Nkarta Inc Linfocitos citolíticos naturales modificados genéticamente para inmunoterapia de cáncer dirigida a cd70.
AU2021347359A1 (en) 2020-09-25 2023-05-18 Beam Therapeutics Inc. Fratricide resistant modified immune cells and methods of using the same
CA3198905A1 (en) * 2020-10-19 2022-04-28 Caribou Biosciences, Inc. Dna-containing polynucleotides and guides for crispr type v systems, and methods of making and using the same
KR20230093328A (ko) * 2020-10-30 2023-06-27 아버 바이오테크놀로지스, 인크. B2m을 표적화하는 rna 가이드를 포함하는 조성물 및 이의 용도
AU2021368740A1 (en) * 2020-10-30 2023-06-01 Arbor Biotechnologies, Inc. Compositions comprising an rna guide targeting trac and uses thereof
CN113881652B (zh) * 2020-11-11 2022-11-22 山东舜丰生物科技有限公司 新型Cas酶和系统以及应用
US11459372B2 (en) 2020-11-30 2022-10-04 Crispr Therapeutics Ag Gene-edited natural killer cells
IL303269A (en) 2020-12-03 2023-07-01 Century Therapeutics Inc Genetically engineered cells and uses thereof
US11661459B2 (en) 2020-12-03 2023-05-30 Century Therapeutics, Inc. Artificial cell death polypeptide for chimeric antigen receptor and uses thereof
EP4255453A4 (en) * 2020-12-03 2025-03-26 Nkarta, Inc. Methods for manipulating immune cells for increased potency and persistence, as well as uses of engineered cells in immunotherapy
CN116848241A (zh) * 2020-12-17 2023-10-03 孟山都技术公司 工程化无ssDNA酶的CRISPR核酸内切酶
TW202241935A (zh) 2020-12-18 2022-11-01 美商世紀治療股份有限公司 具有可調適受體專一性之嵌合抗原受體系統
EP4271798A1 (en) 2020-12-30 2023-11-08 CRISPR Therapeutics AG Compositions and methods for differentiating stem cells into nk cells
JP7877351B2 (ja) * 2021-03-19 2026-06-22 メタゲノミ セラピューティクス，インク． Ｃａｓ酵素を用いたマルチプレックス編集
KR102428773B1 (ko) * 2021-04-21 2022-08-05 (주)지플러스생명과학 분리형 crRNA 및 이를 이용한 CRISPR-Cas 시스템
MX2023014356A (es) * 2021-06-02 2024-02-15 Metagenomi Inc Sistemas crispr de clase ii, tipo v.
WO2022272292A2 (en) 2021-06-23 2022-12-29 Editas Medicine, Inc. Engineered cells for therapy
KR20240051112A (ko) 2021-07-01 2024-04-19 인답타 세라뷰틱스 인코포레이티드 조작된 자연 살해(nk) 세포 및 관련 방법
CN119452085A (zh) * 2021-08-27 2025-02-14 宏基因组学公司 具有ruvc结构域的酶
KR20240110603A (ko) 2021-11-24 2024-07-15 메타지노미, 인크. 엔도뉴클레아제 시스템
TWI870769B (zh) * 2022-01-24 2025-01-21 美洛生物科技股份有限公司 合成rna片段及其用於rna依賴擴增之用途
JP2023107748A (ja) * 2022-01-24 2023-08-03 美洛生物科技股▲ふん▼有限公司 合成ｒｎａ断片及びｒｎａ依存性増幅のためのその使用
WO2023164625A2 (en) * 2022-02-25 2023-08-31 Georgia Tech Research Corporation Modified plant virus system for delivery of nucleic acids into mammalian cells
US20250375524A1 (en) 2022-06-30 2025-12-11 Indapta Therapeutics, Inc. Combination of engineered natural killer (nk) cells and antibody therapy and related methods
WO2024263564A2 (en) * 2023-06-21 2024-12-26 Lonza Cologne Gmbh Multi-chambered electroporation cartridge and methods of use
WO2025213127A1 (en) 2024-04-05 2025-10-09 Indapta Therapeutics, Inc. Treatment of autoimmune diseases or conditions with natural killer cells
WO2025245399A1 (en) 2024-05-23 2025-11-27 Indapta Therapeutics, Inc. Method of treating cancer with natural killer cells
KR102955030B1 (ko) * 2025-11-04 2026-04-23 대한민국 신규한 가이드 rna 및 이의 용도

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015136001A1 (en) * 2014-03-11 2015-09-17 Cellectis Method for generating t-cells compatible for allogenic transplantation
WO2015161276A2 (en) * 2014-04-18 2015-10-22 Editas Medicine, Inc. Crispr-cas-related methods, compositions and components for cancer immunotherapy
WO2016073955A2 (en) * 2014-11-06 2016-05-12 President And Fellows Of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
WO2016160721A1 (en) * 2015-03-27 2016-10-06 President And Fellows Of Harvard College Modified t cells and methods of making and using the same
WO2016205711A1 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4286517A3 (en) * 2013-04-04 2024-03-13 President and Fellows of Harvard College Therapeutic uses of genome editing with crispr/cas systems
JP6608807B2 (ja) 2013-05-29 2019-11-20 セレクティス Ｒｎａガイドｃａｓヌクレアーゼ系を用いることによって免疫療法のためにｔ細胞を操作するための方法
ES2803728T3 (es) * 2015-10-05 2021-01-29 Prec Biosciences Inc Células genéticamente modificadas que comprenden un gen de región constante alfa del receptor de linfocitos T humano modificado
WO2017093969A1 (en) * 2015-12-04 2017-06-08 Novartis Ag Compositions and methods for immunooncology

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2015136001A1 (en) * 2014-03-11 2015-09-17 Cellectis Method for generating t-cells compatible for allogenic transplantation
WO2015161276A2 (en) * 2014-04-18 2015-10-22 Editas Medicine, Inc. Crispr-cas-related methods, compositions and components for cancer immunotherapy
WO2016073955A2 (en) * 2014-11-06 2016-05-12 President And Fellows Of Harvard College Cells lacking b2m surface expression and methods for allogeneic administration of such cells
WO2016160721A1 (en) * 2015-03-27 2016-10-06 President And Fellows Of Harvard College Modified t cells and methods of making and using the same
WO2016205711A1 (en) * 2015-06-18 2016-12-22 The Broad Institute Inc. Novel crispr enzymes and systems

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
BERND ZETSCHE, JONATHAN S. GOOTENBERG, OMAR O. ABUDAYYEH, IAN M. SLAYMAKER, KIRA S. MAKAROVA, PATRICK ESSLETZBICHLER, SARA E. VOLZ: "Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System", CELL, ELSEVIER, AMSTERDAM NL, vol. 163, no. 3, 1 October 2015 (2015-10-01), Amsterdam NL , pages 759 - 771, XP055267511, ISSN: 0092-8674, DOI: 10.1016/j.cell.2015.09.038 *

Also Published As

Publication number Publication date
AU2023266284A1 (en) 2023-12-07
WO2017152015A1 (en) 2017-09-08
KR20180118204A (ko) 2018-10-30
MX2023007841A (es) 2023-07-07
IL261163A (en) 2018-10-31
KR20230146127A (ko) 2023-10-18
KR20240173370A (ko) 2024-12-11
AU2017226172A2 (en) 2018-10-04
KR102738283B1 (ko) 2024-12-09
SG11201807538PA (en) 2018-09-27
AU2017226172A1 (en) 2018-09-27
JP2019507599A (ja) 2019-03-22
JP7251980B2 (ja) 2023-04-04
IL261163B2 (en) 2023-02-01
AU2026206148A1 (en) 2026-08-06
US20250320491A1 (en) 2025-10-16
IL261163B (en) 2022-10-01
EP3423580A1 (en) 2019-01-09
AU2023266284B2 (en) 2026-04-16
KR102587132B1 (ko) 2023-10-11
CA3016331A1 (en) 2017-09-08
MX2018010484A (es) 2019-01-10
IL291971B2 (en) 2025-04-01
CN109312340A (zh) 2019-02-05
JP2026016399A (ja) 2026-02-03
JP2023089005A (ja) 2023-06-27
US12325854B2 (en) 2025-06-10
IL291971A (en) 2022-06-01
US20190062735A1 (en) 2019-02-28
AU2017226172B2 (en) 2023-08-17
KR20220124278A (ko) 2022-09-13
IL291971B1 (en) 2024-12-01
KR102438360B1 (ko) 2022-08-31

Similar Documents

Publication Publication Date Title
AU2023266284B2 (en) CRISPR/Cpf1-related methods, compositions and components for cancer immunotherapy
AU2021236446B2 (en) CRISPR-Cas-related methods, compositions and components for cancer immunotherapy
EP3981876A1 (en) Crispr/cas-related methods and compositions for treating sickle cell disease
US20170007679A1 (en) Crispr/cas-related methods and compositions for treating hiv infection and aids
WO2023015307A1 (en) Method for producing genetically modified cells
HK40050007A (en) Crispr-cas-related methods, compositions and components for cancer immunotherapy

Legal Events

Date Code Title Description
DA3 Amendments made section 104

Free format text: THE NATURE OF THE AMENDMENT IS AS SHOWN IN THE STATEMENT FILED 12 SEP 2018

SREP Specification republished
FGA Letters patent sealed or granted (standard patent)