BR112023021963A2 - Sistemas de produção estáveis para produção do vírus adenoassociado - Google Patents

Sistemas de produção estáveis para produção do vírus adenoassociado

Info

Publication number
BR112023021963A2
BR112023021963A2 BR112023021963A BR112023021963A BR112023021963A2 BR 112023021963 A2 BR112023021963 A2 BR 112023021963A2 BR 112023021963 A BR112023021963 A BR 112023021963A BR 112023021963 A BR112023021963 A BR 112023021963A BR 112023021963 A2 BR112023021963 A2 BR 112023021963A2
Authority
BR
Brazil
Prior art keywords
associated virus
production
stable production
production systems
genetically modified
Prior art date
Application number
BR112023021963A
Other languages
English (en)
Inventor
A K Nielsen Alec
S Stach Christopher
J Gam Jeremy
T Leonard Michael
Original Assignee
Asimov 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 Asimov Inc filed Critical Asimov Inc
Publication of BR112023021963A2 publication Critical patent/BR112023021963A2/pt

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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
    • 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
    • C12N5/0684—Cells of the urinary tract or kidneys
    • C12N5/0686—Kidney 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
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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 RNAses, DNAses
    • 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
    • C12N9/93—Ligases (6)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y601/00—Ligases forming carbon-oxygen bonds (6.1)
    • C12Y601/01—Ligases forming aminoacyl-tRNA and related compounds (6.1.1)
    • C12Y601/01026—Pyrrolysine-tRNAPyl ligase (6.1.1.26)
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

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)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Urology & Nephrology (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

sistemas de produção estáveis para produção do vírus adenoassociado. a presente invenção refere-se a células geneticamente modificadas para produção do aav. a célula geneticamente modificada compreende sistemas moleculares para controle temporal da expressão de genes necessários para a produção de aav. também são aqui descritos os métodos de utilização de células geneticamente modificadas para produção do aav.
BR112023021963A 2021-04-21 2022-04-21 Sistemas de produção estáveis para produção do vírus adenoassociado BR112023021963A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163177760P 2021-04-21 2021-04-21
PCT/US2022/025755 WO2022226189A1 (en) 2021-04-21 2022-04-21 Stable production systems for adeno-associated virus production

Publications (1)

Publication Number Publication Date
BR112023021963A2 true BR112023021963A2 (pt) 2024-01-16

Family

ID=83723162

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112023021963A BR112023021963A2 (pt) 2021-04-21 2022-04-21 Sistemas de produção estáveis para produção do vírus adenoassociado

Country Status (10)

Country Link
EP (1) EP4326883A1 (pt)
JP (1) JP2024515369A (pt)
KR (1) KR20240019759A (pt)
CN (1) CN117529558A (pt)
AU (1) AU2022262365A1 (pt)
BR (1) BR112023021963A2 (pt)
CA (1) CA3217226A1 (pt)
IL (1) IL307830A (pt)
MX (1) MX2023012518A (pt)
WO (1) WO2022226189A1 (pt)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20240101620A (ko) * 2021-11-09 2024-07-02 아시모브 인코포레이티드 Aav 벡터 생산을 위한 안정한 생산 시스템

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU775679B2 (en) * 1998-08-20 2004-08-12 Cell Genesys, Inc. Use of suppressor tRNA's to regulate cytotoxicity during the production of recombinant gene products
WO2005080581A2 (en) * 2004-02-17 2005-09-01 University Of Florida Research Foundation, Inc. Insulated herpesvirus-derived gene expression cassettes for sustained and regulatable gene expression
WO2010114948A2 (en) * 2009-04-02 2010-10-07 University Of Florida Research Foundation, Inc. An inducible system for highly efficient production of recombinant adeno-associated virus (raav) vectors
AU2013320184B2 (en) * 2012-09-24 2017-10-12 Medimmune Limited Cell lines
US11702673B2 (en) * 2018-10-18 2023-07-18 University Of Florida Research Foundation, Incorporated Methods of enhancing biological potency of baculovirus system-produced recombinant adeno-associated virus
EP3870148A4 (en) * 2018-10-25 2022-11-09 Takeda Pharmaceutical Company Limited AAV TRIPLE PLASMID SYSTEM
US20220290121A1 (en) * 2019-08-30 2022-09-15 The General Hospital Corporation Combinatorial Adenine and Cytosine DNA Base Editors
AU2020361533A1 (en) * 2019-10-08 2022-04-28 Trustees Of Boston College Proteins containing multiple, different unnatural amino acids and methods of making and using such proteins

Also Published As

Publication number Publication date
AU2022262365A1 (en) 2023-11-09
CA3217226A1 (en) 2022-10-27
EP4326883A1 (en) 2024-02-28
WO2022226189A1 (en) 2022-10-27
MX2023012518A (es) 2024-02-08
KR20240019759A (ko) 2024-02-14
JP2024515369A (ja) 2024-04-09
IL307830A (en) 2023-12-01
CN117529558A (zh) 2024-02-06
AU2022262365A9 (en) 2023-11-16

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