KR20220097875A - 게놈 통합을 위한 방법 및 조성물 - Google Patents

게놈 통합을 위한 방법 및 조성물 Download PDF

Info

Publication number
KR20220097875A
KR20220097875A KR1020227010704A KR20227010704A KR20220097875A KR 20220097875 A KR20220097875 A KR 20220097875A KR 1020227010704 A KR1020227010704 A KR 1020227010704A KR 20227010704 A KR20227010704 A KR 20227010704A KR 20220097875 A KR20220097875 A KR 20220097875A
Authority
KR
South Korea
Prior art keywords
sequence
mrna
cell
encoding
utr
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
KR1020227010704A
Other languages
English (en)
Korean (ko)
Inventor
대니얼 겟츠
위샤오 왕
나미타 비사리아
Original Assignee
마이얼로이드 테라퓨틱스, 인크.
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 마이얼로이드 테라퓨틱스, 인크. filed Critical 마이얼로이드 테라퓨틱스, 인크.
Publication of KR20220097875A publication Critical patent/KR20220097875A/ko
Pending legal-status Critical Current

Links

Images

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/711Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/177Receptors; Cell surface antigens; Cell surface determinants
    • A61K38/1774Immunoglobulin superfamily (e.g. CD2, CD4, CD8, ICAM molecules, B7 molecules, Fc-receptors, MHC-molecules)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/45Transferases (2)
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/465Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K39/00Medicinal preparations containing antigens or antibodies
    • A61K39/395Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum
    • A61K39/39533Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals
    • A61K39/39558Antibodies; Immunoglobulins; Immune serum, e.g. antilymphocytic serum against materials from animals against tumor tissues, cells, antigens
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705Receptors; Cell surface antigens; Cell surface determinants
    • C07K14/70503Immunoglobulin superfamily
    • C07K14/7051T-cell receptor (TcR)-CD3 complex
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K16/00Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/32Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against translation products of oncogenes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90Stable introduction of foreign DNA into chromosome
    • C12N15/902Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10Transferases (2.)
    • C12N9/12Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241Nucleotidyltransferases (2.7.7)
    • C12N9/1276RNA-directed DNA polymerase (2.7.7.49), i.e. reverse transcriptase or telomerase
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y207/00Transferases transferring phosphorus-containing groups (2.7)
    • C12Y207/07Nucleotidyltransferases (2.7.7)
    • C12Y207/07049RNA-directed DNA polymerase (2.7.7.49), i.e. telomerase or reverse-transcriptase
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/03Fusion polypeptide containing a localisation/targetting motif containing a transmembrane segment
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/01Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/09Fusion polypeptide containing a localisation/targetting motif containing a nuclear localisation signal
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/30Non-immunoglobulin-derived peptide or protein having an immunoglobulin constant or Fc region, or a fragment thereof, attached thereto
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • C07K2319/33Fusion polypeptide fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • C12N2015/8518Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic expressing industrially exogenous proteins, e.g. for pharmaceutical use, human insulin, blood factors, immunoglobulins, pseudoparticles
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/31Combination therapy
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2800/00Nucleic acids vectors
    • C12N2800/90Vectors containing a transposable element
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/20Vectors comprising a special translation-regulating system translation of more than one cistron
    • C12N2840/203Vectors comprising a special translation-regulating system translation of more than one cistron having an IRES

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Mycology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Cell Biology (AREA)
  • Oncology (AREA)
  • Toxicology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
KR1020227010704A 2019-09-03 2020-09-03 게놈 통합을 위한 방법 및 조성물 Pending KR20220097875A (ko)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962895441P 2019-09-03 2019-09-03
US62/895,441 2019-09-03
US201962908800P 2019-10-01 2019-10-01
US62/908,800 2019-10-01
US202063039261P 2020-06-15 2020-06-15
US63/039,261 2020-06-15
PCT/US2020/049240 WO2021046243A2 (en) 2019-09-03 2020-09-03 Methods and compositions for genomic integration

Publications (1)

Publication Number Publication Date
KR20220097875A true KR20220097875A (ko) 2022-07-08

Family

ID=74853338

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020227010704A Pending KR20220097875A (ko) 2019-09-03 2020-09-03 게놈 통합을 위한 방법 및 조성물

Country Status (12)

Country Link
US (6) US11672874B2 (https=)
EP (1) EP4025686A4 (https=)
JP (1) JP7831937B2 (https=)
KR (1) KR20220097875A (https=)
CN (1) CN114981409A (https=)
AU (1) AU2020341479B2 (https=)
BR (1) BR112022003970A2 (https=)
CA (1) CA3149897A1 (https=)
GB (2) GB2605276B (https=)
IL (1) IL291048A (https=)
MX (1) MX2022002613A (https=)
WO (1) WO2021046243A2 (https=)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP7831937B2 (ja) 2019-09-03 2026-03-17 クリエイト・メディシンズ,インコーポレーテッド ゲノム組込みのための方法および組成物
US10980836B1 (en) 2019-12-11 2021-04-20 Myeloid Therapeutics, Inc. Therapeutic cell compositions and methods of manufacturing and use thereof
EP4232583A1 (en) 2020-10-21 2023-08-30 Massachusetts Institute of Technology Systems, methods, and compositions for site-specific genetic engineering using programmable addition via site-specific targeting elements (paste)
CA3197423A1 (en) 2020-11-04 2022-05-12 Daniel Getts Engineered chimeric fusion protein compositions and methods of use thereof
CN112708636A (zh) * 2021-01-22 2021-04-27 彭双红 基因转录框架、载体系统、基因组序列编辑方法及应用
EP4308133A4 (en) 2021-03-17 2025-01-22 Myeloid Therapeutics, Inc. MANIPULATED CHIMERIC FUSION PROTEIN COMPOSITIONS AND METHODS OF USE THEREOF
AU2022272235A1 (en) * 2021-05-11 2023-12-21 Create Medicines, Inc. Methods and compositions for genomic integration
WO2023039434A1 (en) * 2021-09-08 2023-03-16 Metagenomi, Inc. Systems and methods for transposing cargo nucleotide sequences
MX2024003006A (es) * 2021-09-08 2024-03-25 Metagenomi Inc Sistemas, composiciones y metodos que implican retrotransposones y fragmentos funcionales de estos.
CN118871134A (zh) * 2022-03-21 2024-10-29 生物探险有限公司 用于增强蛋白质表达的内部核糖体进入位点(IRES)、质粒载体和环状mRNA
CN115044583A (zh) * 2022-03-21 2022-09-13 隋云鹏 用于基因编辑的rna框架和基因编辑方法
CA3262555A1 (en) * 2022-07-20 2024-01-25 Addition Therapeutics, Inc. GENOME INSERTION INTO CELLS
WO2024044767A2 (en) * 2022-08-25 2024-02-29 The Board Of Trustees Of The Leland Stanford Junior University Recruitment of donor dna from in vivo assembled plasmids for saturation genome editing
US20260078403A1 (en) * 2022-11-07 2026-03-19 The Regents Of The University Of California Zikv-based gene delivery system
WO2024263202A2 (en) * 2022-11-28 2024-12-26 Yale University Compositions and methods for enhancement of mrna vaccine performance and vaccination against mpox
EP4627071A1 (en) * 2022-12-02 2025-10-08 Averna Therapeutics Ltd Recombinant proteins comprising non-ltr retrotransposon-derived polypeptides for gene delivery and insertion
CN118222533A (zh) * 2022-12-19 2024-06-21 北京干细胞与再生医学研究院 一种在基因组插入大片段dna的系统
CN120476212A (zh) * 2022-12-22 2025-08-12 美国金斯瑞公司 自环化rna的制备方法
WO2024163805A1 (en) * 2023-02-01 2024-08-08 Myeloid Therapeutics, Inc. Methods and compositions for genomic integration
WO2024193739A1 (en) * 2023-03-23 2024-09-26 Ostravska Univerzita Method for producing proteins in host cells
WO2024220409A1 (en) * 2023-04-17 2024-10-24 Massachusetts Institute Of Technology Genomic editing with site-specific retrotransposons
WO2025064511A1 (en) * 2023-09-19 2025-03-27 Icahn School Of Medicine At Mont Sinai Bat-associated reverse transcriptase and methods of use thereof
WO2025074310A2 (en) * 2023-10-06 2025-04-10 Exsilio Therapeutics Ltd Engineered retrotransposable element proteins for gene delivery and insertion
WO2025128722A2 (en) * 2023-12-11 2025-06-19 Myeloid Therapeutics, Inc. Methods and compositions for genomic integration
CN117899207A (zh) * 2023-12-12 2024-04-19 中国医学科学院医学生物学研究所 一种轮状病毒mRNA疫苗及其制备方法和应用
WO2025213067A2 (en) * 2024-04-05 2025-10-09 Arc Research Institute Methods and compositions for increased expression of integration deficient viral vectors
WO2025217194A1 (en) * 2024-04-09 2025-10-16 Typewriter Therapeutics, Inc. Repeat dosing of retrotransposons
WO2025229499A1 (en) * 2024-04-29 2025-11-06 Averna Therapeutics Ltd Compositions and methods for modulating t cells

Family Cites Families (189)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5633234A (en) 1993-01-22 1997-05-27 The Johns Hopkins University Lysosomal targeting of immunogens
US5773244A (en) 1993-05-19 1998-06-30 Regents Of The University Of California Methods of making circular RNA
FR2709309B1 (fr) 1993-08-25 1995-11-10 Centre Nat Rech Scient Compositions cellulaires, préparation et utilisations thérapeutiques.
US5631236A (en) 1993-08-26 1997-05-20 Baylor College Of Medicine Gene therapy for solid tumors, using a DNA sequence encoding HSV-Tk or VZV-Tk
US5641875A (en) 1993-09-30 1997-06-24 University Of Pennsylvania DNA encoding chimeric IgG Fc receptor
US6150160A (en) * 1995-11-16 2000-11-21 The John Hopkins University Compositions and methods of use of mammalian retrotransposons
US20030121063A1 (en) * 1995-11-16 2003-06-26 The Trustees Of The University Of Pennsylvania Compositions and methods of use of mammalian retrotransposons
US6576463B1 (en) 1999-01-15 2003-06-10 The Regents Of The University Of California Hybrid vectors for gene therapy
AU1100201A (en) 1999-10-28 2001-05-08 Board Of Trustees Of The Leland Stanford Junior University Methods of in vivo gene transfer using a sleeping beauty transposon system
US7176243B2 (en) 2000-06-02 2007-02-13 The General Hospital Corporation CaR receptor as a mediator of migratory cell chemotaxis and/or chemokinesis
AU2001297703B2 (en) 2000-11-07 2006-10-19 City Of Hope CD19-specific redirected immune cells
US8709412B2 (en) 2001-06-29 2014-04-29 The Board Of Trustees Of The Leland Stanford Junior University Modulation of TIM receptor activity in combination with cytoreductive therapy
US20060183226A1 (en) * 2002-01-31 2006-08-17 Haruhiko Fujiwara Methods for retrotransposing long interspersed elements (lines)
CA2476452A1 (en) 2002-02-15 2003-08-28 Zycos Inc. Electroporation methods for introducing bioactive agents into cells
US20080254027A1 (en) 2002-03-01 2008-10-16 Bernett Matthew J Optimized CD5 antibodies and methods of using the same
AU2003233734C1 (en) 2002-06-05 2011-01-20 Her Majesty The Queen In Right Of Canada, As Represented By The Minister Of Agriculture And Agri-Food Retrons for gene targeting
EP1509244B1 (en) 2002-06-06 2011-07-27 Immunicum AB New method and composition for producing a cellular allogeneic vaccine
US20060239962A1 (en) 2002-12-04 2006-10-26 Banchereau Jacques F Rapid one-step generation of antigen loaded dendritic cell vaccine from precursors
US20070037759A1 (en) * 2003-02-07 2007-02-15 Administrators Of The Tulane Educational Fund, The Mammalian retrotransposable elements
US8007805B2 (en) 2003-08-08 2011-08-30 Paladin Labs, Inc. Chimeric antigens for breaking host tolerance to foreign antigens
US8198020B2 (en) 2003-08-22 2012-06-12 Potentia Pharmaceuticals, Inc. Compositions and methods for enhancing phagocytosis or phagocyte activity
CN105168250B (zh) 2004-10-25 2019-02-05 塞勒兰特治疗公司 增殖骨髓细胞群体的方法及其应用
GB2421948A (en) 2004-12-30 2006-07-12 Ist Superiore Sanita Retrotransposon inhibition to treat cancer
US20060188891A1 (en) 2005-02-23 2006-08-24 Bickmore William D Jr Methods and apparatus for controlling DNA amplification
US20060257359A1 (en) 2005-02-28 2006-11-16 Cedric Francois Modifying macrophage phenotype for treatment of disease
US20090191202A1 (en) 2005-09-29 2009-07-30 Jamieson Catriona Helen M Methods for manipulating phagocytosis mediated by CD47
EP2004237A1 (en) 2006-04-03 2008-12-24 Keele University Targeted therapy
JP2009535065A (ja) * 2006-05-04 2009-10-01 アブマクシス・インコーポレイテツド 組換えタンパク質の高レベルを産生する安定した哺乳動物細胞系を作製するための方法
EP2046347A2 (en) 2006-07-20 2009-04-15 Gourmetceuticals, LLC Phosphorylated glucomannan polysaccharide for receptor mediated activation and maturation of monocyte-derived dendritic cells
US7833789B2 (en) 2006-08-01 2010-11-16 Fondazione Centro San Raffaele Del Monte Tabor Monocyte cell
CN1951499A (zh) 2006-10-09 2007-04-25 中国农业大学 一种细胞免疫疫苗及其制备方法
EP2160461B1 (en) 2007-07-04 2012-08-08 Max-Delbrück-Centrum für Molekulare Medizin (MDC) Hyperactive variants of the transposase protein of the transposon system sleeping beauty
EP2055784A1 (en) 2007-10-31 2009-05-06 Bundesrepublik Deutschland, letztvertreten durch den Präsidenten des Paul-Ehrlich-Instituts Prof. Dr. Johannes Löwer Controlled activation of non-LTR retrotransposons in mammals
AU2009205665B2 (en) 2008-01-15 2013-12-05 The Board Of Trustees Of The Leland Stanford Junior University Methods for manipulating phagocytosis mediated by CD47
WO2010010119A1 (en) 2008-07-22 2010-01-28 Ablynx Nv Amino acid sequences directed against multitarget scavenger receptors and polypeptides
MX2011000970A (es) 2008-08-29 2011-03-15 Symphogen As Anticuerpos anti-cd5.
US9080211B2 (en) 2008-10-24 2015-07-14 Epicentre Technologies Corporation Transposon end compositions and methods for modifying nucleic acids
EP2248903A1 (en) * 2009-04-29 2010-11-10 Universitat Autònoma De Barcelona Methods and reagents for efficient and targeted gene transfer to monocytes and macrophages
AU2011239569B2 (en) 2010-04-16 2014-07-24 Baylor College Of Medicine Method for treating solid tumors
WO2012005763A1 (en) 2010-07-06 2012-01-12 The Scripps Research Institute Use of myeloid-like progenitor cell populations to treat tumors
WO2012043651A1 (ja) 2010-09-30 2012-04-05 国立大学法人 熊本大学 ミエロイド系血液細胞の製造方法
US9206479B2 (en) 2011-02-09 2015-12-08 University Of Rochester Methods and compositions related to Staufen 1 binding sites formed by duplexing Alu elements
EP3792359A1 (en) 2011-03-30 2021-03-17 Transine Therapeutics Limited Functional nucleic acid molecule and use thereof
JP6153526B2 (ja) 2011-09-06 2017-06-28 エージェンシー フォー サイエンス,テクノロジー アンド リサーチ ポリペプチドワクチン
WO2013051718A1 (ja) 2011-10-07 2013-04-11 国立大学法人三重大学 キメラ抗原受容体
US9149519B2 (en) 2012-01-17 2015-10-06 New York University Chimeric human immunodeficiency virus type 1 (HIV-1) with enhanced dendritic cell and macrophage tropism comprising the simian immunodeficiency virus (SIV) minimal Vpx packaging domain
KR102338833B1 (ko) 2012-02-06 2021-12-13 인히브릭스, 인크. Cd47 항체 및 그 사용 방법
US20140140989A1 (en) 2012-02-06 2014-05-22 Inhibrx Llc Non-Platelet Depleting and Non-Red Blood Cell Depleting CD47 Antibodies and Methods of Use Thereof
JP6850528B2 (ja) 2012-02-13 2021-03-31 シアトル チルドレンズ ホスピタル ドゥーイング ビジネス アズ シアトル チルドレンズ リサーチ インスティテュート 二重特異性キメラ抗原受容体およびその治療的使用
WO2013123088A1 (en) 2012-02-14 2013-08-22 Loma Linda University Agents and method for treating inflammation-related conditions and diseases
EP2639313A1 (en) 2012-03-14 2013-09-18 Rheinische Friedrich-Wilhelms-Universität Bonn High-resolution transcriptome of human macrophages
AU2013243948A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of proteins associated with human disease
KR102437522B1 (ko) 2012-05-25 2022-08-26 셀렉티스 면역요법을 위한 동종이형 및 면역억제제 저항성인 t 세포의 조작 방법
ES2528892T3 (es) 2012-07-30 2015-02-13 Nbe-Therapeutics Llc Identificación mediada por trasposición de proteínas de unión o funcionales específicas
US20140037606A1 (en) 2012-08-06 2014-02-06 Eyal Amiel Cell-based, anti-cancer vaccines
MX2015007446A (es) 2012-12-12 2015-12-07 Vasculox Inc Anticuerpos terapeuticos para cd47.
US9221908B2 (en) 2012-12-12 2015-12-29 Vasculox, Inc. Therapeutic CD47 antibodies
WO2014121840A1 (en) 2013-02-07 2014-08-14 Medizinische Hochschule Hannover Induced dendritic cells and uses thereof
SE537429C2 (sv) 2013-02-14 2015-04-28 Scania Cv Ab Samtidig skattning av åtminstone massa och rullmotstånd förett fordon
WO2014134412A1 (en) 2013-03-01 2014-09-04 Regents Of The University Of Minnesota Talen-based gene correction
WO2014153114A1 (en) 2013-03-14 2014-09-25 Fred Hutchinson Cancer Research Center Compositions and methods to modify cells for therapeutic objectives
EP2970426B1 (en) 2013-03-15 2019-08-28 Michael C. Milone Targeting cytotoxic cells with chimeric receptors for adoptive immunotherapy
MX389160B (es) 2013-10-31 2025-03-20 Hutchinson Fred Cancer Res Células no t efectoras y madre/progenitoras hematopoyéticas modificadas y usos de estas.
EP3656864A1 (en) 2014-02-14 2020-05-27 Board of Regents, The University of Texas System Chimeric antigen receptors and methods of making
AU2015229448B2 (en) 2014-03-11 2020-09-03 The Board Of Trustees Of The Leland Stanford Junior University Anti SIRP-alpha antibodies and Bi-specific Macrophage Enhancing antibodies
WO2015164627A1 (en) 2014-04-23 2015-10-29 Discovery Genomics, Inc. Chimeric antigen receptors specific to avb6 integrin and methods of use thereof to treat cancer
CN104004095B (zh) 2014-06-04 2016-11-23 博生吉医药科技(苏州)有限公司 一种cd7纳米抗体、其编码序列及应用
TWI750110B (zh) 2014-07-21 2021-12-21 瑞士商諾華公司 使用人類化抗-bcma嵌合抗原受體治療癌症
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
WO2016033331A1 (en) 2014-08-28 2016-03-03 Bioatla, Llc Conditionally active chimeric antigen receptors for modified t-cells
WO2016030501A1 (en) * 2014-08-28 2016-03-03 Centre National De La Recherche Scientifique - Cnrs - Synthetic alu-retrotransposon vectors for gene therapy
US20180133252A9 (en) 2014-09-09 2018-05-17 Unum Therapeutics Inc. Chimeric receptors and uses thereof in immune therapy
ES2946697T3 (es) 2014-09-10 2023-07-24 Miltenyi Biotec Bv & Co Kg Una población celular para su uso en el tratamiento de cáncer
CA2961950A1 (en) 2014-09-28 2016-03-31 The Regents Of The University Of California Modulation of stimulatory and non-stimulatory myeloid cells
JP2017531430A (ja) 2014-10-07 2017-10-26 セレクティスCellectis Carによって誘導される免疫細胞の活性を調節するための方法
MA41538A (fr) 2014-10-17 2017-12-26 Baylor College Medicine Cellules immunitaires bipartites et tripartites de signalisation
JP7523203B2 (ja) 2014-10-31 2024-07-26 マサチューセッツ インスティテュート オブ テクノロジー 生体分子の免疫細胞への送達
CA2967379A1 (en) 2014-11-18 2016-05-26 Janssen Pharmaceutica Nv Cd47 antibodies, methods, and uses
WO2016106184A1 (en) 2014-12-22 2016-06-30 AgBiome, Inc. Methods for making a synthetic gene
US11161907B2 (en) 2015-02-02 2021-11-02 Novartis Ag Car-expressing cells against multiple tumor antigens and uses thereof
CN113713091A (zh) 2015-02-06 2021-11-30 新加坡国立大学 工程免疫细胞及其用途和生产方法
US20170151281A1 (en) 2015-02-19 2017-06-01 Batu Biologics, Inc. Chimeric antigen receptor dendritic cell (car-dc) for treatment of cancer
WO2016138034A1 (en) 2015-02-24 2016-09-01 The Regents Of The University Of California Binding-triggered transcriptional switches and methods of use thereof
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
WO2016149254A1 (en) 2015-03-17 2016-09-22 Chimera Bioengineering, Inc. Smart car devices, de car polypeptides, side cars and uses thereof
US10214591B1 (en) * 2015-04-03 2019-02-26 Alienor Farma Monoclonal antibody to human line-1 ORF2 protein and method for early detection of transforming cells in pre-neoplastic tissues of a human subject
EP3286225B1 (en) 2015-04-23 2020-07-01 Baylor College of Medicine Cd5 chimeric antigen receptor for adoptive t cell therapy
CA2994935A1 (en) 2015-05-18 2016-11-24 Ab Initio Biotherapeutics, Inc. Sirp polypeptide compositions and methods of use
US10434153B1 (en) 2015-05-20 2019-10-08 Kim Leslie O'Neill Use of car and bite technology coupled with an scFv from an antibody against human thymidine kinase 1 to specifically target tumors
EP3302558A4 (en) 2015-06-01 2019-01-16 The Rockefeller University ANTI-TUMOR MEDIUM AND METHOD OF USE
GB201509413D0 (en) 2015-06-01 2015-07-15 Ucl Business Plc Fusion protein
CA3012607A1 (en) 2015-06-18 2016-12-22 The Broad Institute Inc. Crispr enzymes and systems
RU2766690C2 (ru) 2015-07-28 2022-03-15 Дзе Трастиз Оф Дзе Юниверсити Оф Пенсильвания Модифицированные моноциты/макрофаги, экспрессирующие химерные антигенные рецепторы, и их применения
GB2592821B (en) 2015-07-31 2022-01-12 Univ Minnesota Modified cells and methods of therapy
EP3328377A4 (en) 2015-07-31 2019-03-13 Tarveda Therapeutics, Inc. COMPOSITIONS AND METHODS FOR IMMUNE ONCOLOGY THERAPIES
WO2017027422A1 (en) 2015-08-07 2017-02-16 Alexo Therapeutics Inc. Constructs having a sirp-alpha domain or variant thereof
US11352439B2 (en) 2015-08-13 2022-06-07 Kim Leslie O'Neill Macrophage CAR (MOTO-CAR) in immunotherapy
CN108243607A (zh) 2015-09-09 2018-07-03 西雅图儿童医院(Dba西雅图儿童研究所) 用于免疫疗法的巨噬细胞的遗传工程
EP3350335B1 (en) 2015-09-16 2019-11-27 T-CURX GmbH Improved transposon system for gene delivery
EP4706680A3 (en) 2015-09-22 2026-03-25 Julius-Maximilians-Universität Würzburg A method for high level and stable gene transfer in lymphocytes
CN105154473B (zh) 2015-09-30 2019-03-01 上海细胞治疗研究院 一种高效安全的转座子整合系统及其用途
KR20180054600A (ko) 2015-10-13 2018-05-24 브라이엄 영 유니버시티 면역치료에서의 대식세포 키메라 항원 수용체(moto-car)
CA3004738A1 (en) 2015-11-09 2017-05-18 Aperisys, Inc. Modified immune cells and uses thereof
US10946042B2 (en) 2015-12-01 2021-03-16 The Trustees Of The University Of Pennsylvania Compositions and methods for selective phagocytosis of human cancer cells
EP3202783A1 (en) 2016-02-02 2017-08-09 Ecole Polytechnique Fédérale de Lausanne (EPFL) Engineered antigen presenting cells and uses thereof
WO2017133175A1 (en) 2016-02-04 2017-08-10 Nanjing Legend Biotech Co., Ltd. Engineered mammalian cells for cancer therapy
US12220451B2 (en) 2016-02-04 2025-02-11 Duke University Cell-based vaccine compositions and methods of use
GB201602473D0 (en) 2016-02-11 2016-03-30 Horizon Discovery Ltd Replicative transposon system
US10617749B1 (en) 2016-02-24 2020-04-14 Duke University Composition of matter and methods for alteration of dendritic cell metabolism to augment cancer vaccine efficacy
US20170275665A1 (en) 2016-02-24 2017-09-28 Board Of Regents, The University Of Texas System Direct crispr spacer acquisition from rna by a reverse-transcriptase-cas1 fusion protein
US20200063157A9 (en) 2016-02-26 2020-02-27 Poseida Therapeutics, Inc. Transposon system and methods of use
EP3219803A1 (en) 2016-03-15 2017-09-20 Max-Delbrück-Centrum für Molekulare Medizin Enhanced sleeping beauty transposons, kits and methods of transposition
US20180186855A1 (en) 2016-03-23 2018-07-05 Alector Llc Chimeric receptors and methods of use thereof
WO2017172981A2 (en) 2016-03-29 2017-10-05 University Of Southern California Chimeric antigen receptors targeting cancer
US11649284B2 (en) 2016-04-18 2023-05-16 Baylor College Of Medicine Cancer gene therapy targeting CD47
US10875919B2 (en) 2016-04-26 2020-12-29 Alector Llc Chimeric receptors and methods of use thereof
US11390658B2 (en) 2016-06-06 2022-07-19 St. Jude Children's Research Hospital Anti-CD7 chimeric antigen receptor and method of use thereof
WO2017214553A1 (en) 2016-06-09 2017-12-14 The General Hospital Corporation Modulating the cellular stress response
CA3028158A1 (en) 2016-06-17 2017-12-21 The Broad Institute, Inc. Type vi crispr orthologs and systems
CN107523545A (zh) 2016-06-20 2017-12-29 上海细胞治疗研究院 一种高效稳定表达抗体的杀伤性细胞及其用途
EP3504244A4 (en) 2016-08-26 2020-08-19 Agency for Science, Technology and Research MACROPHAGE-STIMULATING PROTEIN RECEPTOR ANTIBODIES (OR OF NANTAIS ORIGINAL RON-RECEPTOR) AND THEIR USES
JOP20190009A1 (ar) 2016-09-21 2019-01-27 Alx Oncology Inc أجسام مضادة ضد بروتين ألفا منظم للإشارات وطرق استخدامها
EP4089116A1 (en) 2016-09-27 2022-11-16 Cero Therapeutics, Inc. Chimeric engulfment receptor molecules
WO2018064626A1 (en) 2016-09-30 2018-04-05 Baylor College Of Medicine Adaptive chimeric antigen receptor t-cell design
WO2018071058A1 (en) 2016-10-15 2018-04-19 Baylor College Of Medicine Platform for enhanced targeted cell delivery
WO2018073394A1 (en) 2016-10-19 2018-04-26 Cellectis Cell death inducing chimeric antigen receptors
EP3529276A4 (en) 2016-10-21 2020-06-17 Arch Oncology, Inc. CD47 THERAPEUTIC ANTIBODIES
SG11201903693QA (en) 2016-11-01 2019-05-30 Genmab Bv Polypeptide variants and uses thereof
KR102709829B1 (ko) 2016-11-11 2024-09-24 바이오 래드 래버러토리스 인코오포레이티드 핵산 샘플을 프로세싱하는 방법
CN118027200A (zh) 2016-12-09 2024-05-14 艾利妥 抗SIRPα抗体及其使用方法
CA3049961A1 (en) 2016-12-09 2018-06-14 The Broad Institute, Inc. Crispr effector system based diagnostics
WO2018140831A2 (en) 2017-01-27 2018-08-02 Silverback Therapeutics, Inc. Tumor targeting conjugates and methods of use thereof
AU2018235756B2 (en) 2017-03-13 2024-08-08 Poseida Therapeutics, Inc. Compositions and methods for selective elimination and replacement of hematopoietic stem cells
CN111108220B (zh) 2017-03-15 2024-11-19 博德研究所 用于病毒检测的基于crispr效应系统的诊断
WO2018191490A1 (en) 2017-04-13 2018-10-18 The Trustees Of The University Of Pennsylvania Use of gene editing to generate universal tcr re-directed t cells for adoptive immunotherapy
US10415017B2 (en) 2017-05-17 2019-09-17 Thunder Biotech, Inc. Transgenic macrophages, chimeric antigen receptors, and associated methods
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
TWI828625B (zh) 2017-06-25 2024-01-11 美商西雅圖免疫公司 引導及導航控制蛋白質以及彼之製造及使用方法
WO2019010551A1 (pt) 2017-07-10 2019-01-17 Instituto De Biologia Molecular Do Paraná - Ibmp Plataforma genética para superexpressão heteróloga associada à seleção de células altamente produtoras de proteínas
US20210087548A1 (en) 2017-07-24 2021-03-25 The Board Of Trustees Of The Leland Stanford Junior University Compositions and Methods for Inducing Protein Function
WO2019023347A1 (en) 2017-07-26 2019-01-31 Forty Seven, Inc. ANTI-SIRP-ALPHA ANTIBODIES AND ASSOCIATED METHODS
CN109337872B (zh) 2017-07-27 2023-06-23 上海细胞治疗研究院 高效扩增car-t细胞的人工抗原递呈细胞及其用途
CN109306340B (zh) 2017-07-27 2023-06-06 上海细胞治疗研究院 一种高效扩增全t细胞的人工抗原递呈细胞及其用途
WO2019032624A1 (en) 2017-08-08 2019-02-14 Pionyr Immunotherapeutics, Inc. COMPOSITIONS AND METHODS FOR DEACTIVATING TREM1-EXPRESSING MESHLOID CELLS
WO2019050948A1 (en) 2017-09-05 2019-03-14 Regeneron Pharmaceuticals, Inc. ADMINISTRATION OF A GENE EDITION SYSTEM HAVING ONLY ONE RETROVIRAL PARTICLE AND METHODS OF GENERATING AND USING
AU2018329741B2 (en) 2017-09-08 2025-02-20 Poseida Therapeutics, Inc. Compositions and methods for chimeric ligand receptor (CLR)-mediated conditional gene expression
CN111479921B (zh) 2017-09-18 2024-08-02 埃克苏马生物技术公司 用于以基因方式修饰且扩增淋巴细胞以及调节其活性的方法及组合物
EP3688032B1 (en) 2017-09-26 2025-11-05 Cero Therapeutics Holdings, Inc. Chimeric engulfment receptor molecules and methods of use
US12467050B2 (en) 2017-10-02 2025-11-11 Georgia Tech Research Corporation Methods and compositions for engineering synthetic bioswitches for remote control of biological activity
US11352611B2 (en) 2017-10-03 2022-06-07 Board Of Regents, The University Of Texas System Non-LTR-retroelement reverse transcriptase and uses thereof
US20200323964A1 (en) 2017-10-16 2020-10-15 Aadigen, Llc Peptides and nanoparticles for intracellular delivery of mrna
GB201717974D0 (en) 2017-10-31 2017-12-13 Univ Court Of The Univ Of Aberdeen Modified receptors
WO2019090175A1 (en) 2017-11-02 2019-05-09 Arbor Biotechnologies, Inc. Novel crispr-associated transposon systems and components
CN109971716B (zh) 2017-12-28 2023-08-01 上海细胞治疗研究院 自分泌cd47抗体的egfr特异性car-t细胞及其用途
AU2019226526B2 (en) * 2018-03-02 2025-12-04 Generation Bio Co. Identifying and characterizing genomic safe harbors (GSH) in humans and murine genomes, and viral and non-viral vector compositions for targeted integration at an identified GSH loci
GB2572005A (en) 2018-03-16 2019-09-18 Univ Court Univ Of Edinburgh Macrophage-based therapy
US20210015865A1 (en) 2018-03-28 2021-01-21 Cero Therapeutics, Inc. Chimeric engulfment receptors and uses thereof for neurodegenerative diseases
WO2019191334A1 (en) 2018-03-28 2019-10-03 Cero Therapeutics, Inc. Chimeric tim4 receptors and uses thereof
JP7444781B2 (ja) 2018-03-28 2024-03-06 セロ・セラピューティクス・インコーポレイテッド 細胞免疫療法組成物およびその使用
SG11202011240YA (en) 2018-06-26 2020-12-30 Broad Inst Inc Crispr/cas and transposase based amplification compositions, systems and methods
US20210222163A1 (en) 2018-07-11 2021-07-22 X Gen Us Co. Transposome enabled dna/rna-sequencing (ted rna-seq)
JP2021530212A (ja) 2018-07-13 2021-11-11 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアThe Regents Of The University Of California レトロトランスポゾンベースの送達媒体及びその使用方法
CN113286880A (zh) 2018-08-28 2021-08-20 旗舰先锋创新Vi有限责任公司 调控基因组的方法和组合物
EP3628309A1 (en) 2018-09-28 2020-04-01 Universität Heidelberg Method of making oral dosage forms
EP3876977A1 (en) 2018-11-06 2021-09-15 The Regents Of The University Of California Chimeric antigen receptors for phagocytosis
GB201818110D0 (en) 2018-11-06 2018-12-19 Macrophox Ltd Monocytes for cancer targeting
CA3124110A1 (en) 2018-12-17 2020-06-25 The Broad Institute, Inc. Crispr-associated transposase systems and methods of use thereof
CA3117950A1 (en) 2018-12-19 2020-06-25 Amgen Inc. Apparatus for resolving imaging problems caused by the meniscus
US20220133790A1 (en) * 2019-01-16 2022-05-05 Beam Therapeutics Inc. Modified immune cells having enhanced anti-neoplasia activity and immunosuppression resistance
JP7657726B2 (ja) 2019-03-19 2025-04-07 ザ ブロード インスティテュート,インコーポレーテッド 編集ヌクレオチド配列を編集するための方法および組成物
US11013764B2 (en) 2019-04-30 2021-05-25 Myeloid Therapeutics, Inc. Engineered phagocytic receptor compositions and methods of use thereof
US20220378824A1 (en) 2019-04-30 2022-12-01 Myeloid Therapeutics, Inc. Engineered chimeric fusion protein compositions and methods of use thereof
US20220001031A1 (en) 2019-04-30 2022-01-06 Myeloid Therapeutics, Inc. Engineered chimeric fusion protein compositions and methods of use thereof
CA3135531A1 (en) 2019-04-30 2020-11-05 Daniel Getts Engineered chimeric fusion protein compositions and methods of use thereof
MX2021015411A (es) 2019-06-11 2022-04-18 Myeloid Therapeutics Inc Composiciones acopladoras especificas de macrofagos y metodos de uso de las mismas.
JP2022536951A (ja) 2019-06-19 2022-08-22 フラッグシップ パイオニアリング イノベーションズ シックス,エルエルシー 環状ポリリボヌクレオチドを投与する方法
WO2021016075A1 (en) 2019-07-19 2021-01-28 Flagship Pioneering Innovations Vi, Llc Recombinase compositions and methods of use
JP7831937B2 (ja) 2019-09-03 2026-03-17 クリエイト・メディシンズ,インコーポレーテッド ゲノム組込みのための方法および組成物
US20230040216A1 (en) 2019-11-19 2023-02-09 The Broad Institute, Inc. Retrotransposons and use thereof
BR112022011339A2 (pt) 2019-12-11 2022-10-04 Myeloid Therapeutics Inc Composições de células terapêuticas e métodos para fabricação e usos das mesmas
CA3174483A1 (en) 2020-03-04 2021-09-10 Flagship Pioneering Innovations Vi, Llc Improved methods and compositions for modulating a genome
IL296024A (en) 2020-03-04 2022-10-01 Flagship Pioneering Innovations Vi Llc Methods and compositions for modulating a genome
JP2023516692A (ja) 2020-03-04 2023-04-20 フラッグシップ パイオニアリング イノベーションズ シックス,エルエルシー ゲノムを調節するための方法及び組成物
AU2021232069A1 (en) 2020-03-05 2022-11-03 Flagship Pioneering Innovations Vi, Llc Host defense suppressing methods and compositions for modulating a genome
EP4232583A1 (en) 2020-10-21 2023-08-30 Massachusetts Institute of Technology Systems, methods, and compositions for site-specific genetic engineering using programmable addition via site-specific targeting elements (paste)
CA3197423A1 (en) 2020-11-04 2022-05-12 Daniel Getts Engineered chimeric fusion protein compositions and methods of use thereof
AU2022272235A1 (en) 2021-05-11 2023-12-21 Create Medicines, Inc. Methods and compositions for genomic integration
EP4399315A2 (en) 2021-09-10 2024-07-17 Myeloid Therapeutics, Inc. Macrophage specific engager compositions and methods of use thereof
WO2025128722A2 (en) 2023-12-11 2025-06-19 Myeloid Therapeutics, Inc. Methods and compositions for genomic integration

Also Published As

Publication number Publication date
AU2020341479B2 (en) 2026-02-26
WO2021046243A9 (en) 2022-06-23
WO2021046243A3 (en) 2021-06-03
MX2022002613A (es) 2022-06-02
US11672874B2 (en) 2023-06-13
IL291048A (en) 2022-05-01
US20220184230A1 (en) 2022-06-16
BR112022003970A2 (pt) 2022-06-21
EP4025686A4 (en) 2023-09-13
US20230067484A1 (en) 2023-03-02
GB2605276B (en) 2024-08-07
GB202409022D0 (en) 2024-08-07
US20230364265A1 (en) 2023-11-16
JP2022546592A (ja) 2022-11-04
US20230364266A1 (en) 2023-11-16
AU2020341479A1 (en) 2022-03-31
GB2605276A (en) 2022-09-28
GB202203647D0 (en) 2022-04-27
EP4025686A2 (en) 2022-07-13
JP7831937B2 (ja) 2026-03-17
US12599678B2 (en) 2026-04-14
GB2628935A (en) 2024-10-09
GB2628935B (en) 2025-03-05
WO2021046243A2 (en) 2021-03-11
US20220411817A1 (en) 2022-12-29
CN114981409A (zh) 2022-08-30
CA3149897A1 (en) 2021-03-11
US20220364110A1 (en) 2022-11-17

Similar Documents

Publication Publication Date Title
US12599678B2 (en) Methods and compositions for genomic integration
JP7601916B2 (ja) 蝸牛および前庭細胞に核酸を送達するための物質および方法
KR102729768B1 (ko) 트랜스제닉 선택 방법 및 조성물
AU2019252285B2 (en) Viral vectors and packaging cell lines
AU774643B2 (en) Compositions and methods for use in recombinational cloning of nucleic acids
AU2016232146B2 (en) Optimized liver-specific expression systems for FVIII and FIX
KR20240037192A (ko) 게놈 통합을 위한 방법 및 조성물
KR102628872B1 (ko) 세포의 증식을 제어하기 위해 세포 분열 좌위를 사용하기 위한 도구 및 방법
KR20240004253A (ko) 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 방법
KR20210151785A (ko) 비바이러스성 dna 벡터 및 fviii 치료제 발현을 위한 이의 용도
KR20240022575A (ko) 아머링된 키메라 수용체 및 이의 사용 방법
CN115666722A (zh) 包封的rna复制子和使用方法
KR20200085812A (ko) 바이러스 벡터-유도된 염증 반응을 억제하기 위한 조성물 및 방법
CN116323942A (zh) 用于基因组编辑的组合物及其使用方法
WO2024163805A1 (en) Methods and compositions for genomic integration
RU2812852C2 (ru) Невирусные днк-векторы и варианты их применения для экспрессии терапевтического средства на основе фактора viii (fviii)
NL2027815B1 (en) Genomic integration
HK40082332A (zh) 使用细胞分裂基因座控制细胞增殖的工具和方法
HK40082332B (zh) 使用细胞分裂基因座控制细胞增殖的工具和方法

Legal Events

Date Code Title Description
PA0105 International application

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

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

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

E13 Pre-grant limitation requested

Free format text: ST27 STATUS EVENT CODE: A-2-3-E10-E13-LIM-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

E13-X000 Pre-grant limitation requested

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

P13 Application amended

Free format text: ST27 STATUS EVENT CODE: A-2-2-P10-P13-NAP-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

P13-X000 Application amended

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

D21 Rejection of application intended

Free format text: ST27 STATUS EVENT CODE: A-1-2-D10-D21-EXM-PE0902 (AS PROVIDED BY THE NATIONAL OFFICE)

PE0902 Notice of grounds for rejection

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

PN2301 Change of applicant

St.27 status event code: A-3-3-R10-R13-asn-PN2301

St.27 status event code: A-3-3-R10-R11-asn-PN2301

R11 Change to the name of applicant or owner or transfer of ownership requested

Free format text: ST27 STATUS EVENT CODE: A-3-3-R10-R11-ASN-PN2301 (AS PROVIDED BY THE NATIONAL OFFICE)

R13 Change to the name of applicant or owner recorded

Free format text: ST27 STATUS EVENT CODE: A-3-3-R10-R13-ASN-PN2301 (AS PROVIDED BY THE NATIONAL OFFICE)

E13 Pre-grant limitation requested

Free format text: ST27 STATUS EVENT CODE: A-2-3-E10-E13-LIM-X000 (AS PROVIDED BY THE NATIONAL OFFICE)

E13-X000 Pre-grant limitation requested

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