IL284219A - Adeno-associated virus (AAV) cell line generation and related processes - Google Patents

Adeno-associated virus (AAV) cell line generation and related processes

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Publication number
IL284219A
IL284219A IL284219A IL28421921A IL284219A IL 284219 A IL284219 A IL 284219A IL 284219 A IL284219 A IL 284219A IL 28421921 A IL28421921 A IL 28421921A IL 284219 A IL284219 A IL 284219A
Authority
IL
Israel
Prior art keywords
aav
adeno
cell line
associated virus
related methods
Prior art date
Application number
IL284219A
Other languages
English (en)
Hebrew (he)
Inventor
Bingnan Gu
Caitlin M Guenther
Anandita Seth
Original Assignee
Lonza Walkersville Inc
Bingnan Gu
Caitlin M Guenther
Anandita Seth
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 Lonza Walkersville Inc, Bingnan Gu, Caitlin M Guenther, Anandita Seth filed Critical Lonza Walkersville Inc
Publication of IL284219A publication Critical patent/IL284219A/en

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    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/14—Antivirals for RNA viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—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
    • C12N15/63—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
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • C12N15/8645—Adeno-associated virus
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
    • C12N2710/00011—Details
    • C12N2710/10011—Adenoviridae
    • C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
    • C12N2710/10341—Use of virus, viral particle or viral elements as a vector
    • C12N2710/10343—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
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    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/005—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination repressible enhancer/promoter combination, e.g. KRAB
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/001—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination
    • C12N2830/005—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination repressible enhancer/promoter combination, e.g. KRAB
    • C12N2830/006—Vector systems having a special element relevant for transcription controllable enhancer/promoter combination repressible enhancer/promoter combination, e.g. KRAB tet repressible
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/40—Vector systems having a special element relevant for transcription being an insulator
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
IL284219A 2018-12-21 2021-06-20 Adeno-associated virus (AAV) cell line generation and related processes IL284219A (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862783589P 2018-12-21 2018-12-21
US201962866092P 2019-06-25 2019-06-25
PCT/US2019/067156 WO2020132059A1 (en) 2018-12-21 2019-12-18 Adeno-associated virus (aav) producer cell line and related methods

Publications (1)

Publication Number Publication Date
IL284219A true IL284219A (en) 2021-08-31

Family

ID=71099162

Family Applications (1)

Application Number Title Priority Date Filing Date
IL284219A IL284219A (en) 2018-12-21 2021-06-20 Adeno-associated virus (AAV) cell line generation and related processes

Country Status (8)

Country Link
US (2) US11739347B2 (https=)
EP (1) EP3891290A4 (https=)
JP (2) JP7650800B2 (https=)
KR (1) KR20210108423A (https=)
CN (1) CN113396222A (https=)
IL (1) IL284219A (https=)
SG (1) SG11202106217TA (https=)
WO (1) WO2020132059A1 (https=)

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EP4189077A1 (en) * 2020-07-30 2023-06-07 Shape Therapeutics Inc. Stable cell lines for inducible production of raav virions
EP4229205B1 (en) * 2020-10-15 2025-05-14 F. Hoffmann-La Roche AG Nucleic acid constructs for va rna transcription
CN112501209B (zh) * 2020-12-07 2024-02-13 和元生物技术（上海）股份有限公司 外源基因可控表达的腺相关病毒包装方法
CN116829725A (zh) * 2021-02-12 2023-09-29 富士胶片株式会社 用于产生腺相关病毒的试剂盒及其利用
EP4322975A4 (en) * 2021-04-14 2025-03-26 Capsida, Inc. PLASMIDS AND PROCESSES FOR PRODUCING ADENO-ASSOCIATED VIRUSES
EP4410971A4 (en) * 2021-09-30 2025-06-04 FUJIFILM Corporation METHOD FOR PRODUCING AN ADENO-ASSOCIATED VIRUS, CELLS AND EXPRESSION VECTOR
AU2022373653A1 (en) * 2021-10-18 2024-05-02 Regeneron Pharmaceuticals, Inc. Eukaryotic cells comprising adenovirus-associated virus polynucleotides
EP4473123A4 (en) * 2022-02-01 2026-03-11 Shape Therapeutics Inc STABLE CELL LINES FOR INDUCIBLE VAAR VIRION PRODUCTION
EP4491717A4 (en) 2022-03-08 2025-11-12 Fujifilm Corp PRODUCING CELL, PRODUCTION METHOD FOR PRODUCING PRODUCING CELLS AND PRODUCTION METHOD FOR ADENE-ASSOCIATED VIRUS
CN120092093A (zh) * 2022-08-25 2025-06-03 富士胶片株式会社 靶病毒的制造方法及培养组合物
EP4624584A1 (en) * 2022-12-27 2025-10-01 Synplogen Co., Ltd. Helper gene control
US12410436B2 (en) * 2023-10-31 2025-09-09 Lonza Houston, Inc. Adeno-associated virus (AAV) producer cell lines
WO2025122676A1 (en) * 2023-12-05 2025-06-12 Shape Therapeutics Inc. Systems and methods for raav production using a biphasic culture
WO2025168663A1 (en) 2024-02-09 2025-08-14 F. Hoffmann-La Roche Ag Method for producing recombinant adeno-associated viral particles
WO2025252480A1 (en) 2024-06-07 2025-12-11 F. Hoffmann-La Roche Ag Method for purifying plasmid dna
CN118460545B (zh) * 2024-07-08 2025-02-11 凌意(杭州)生物科技有限公司 一种诱导型表达Rep多肽的表达框
US20260022401A1 (en) 2024-07-22 2026-01-22 Hoffmann-La Roche Inc. Novel AAV rep ORFs and Rep polypeptides
WO2026037757A1 (en) 2024-08-13 2026-02-19 F. Hoffmann-La Roche Ag Modified aav particles
WO2026044087A1 (en) * 2024-08-21 2026-02-26 Shape Therapeutics Inc. Increased cellular stability for aav production

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US5622856A (en) 1995-08-03 1997-04-22 Avigen High efficiency helper system for AAV vector production
US6037177A (en) * 1997-08-08 2000-03-14 Cell Genesys, Inc. Method for increasing the efficiency of recombinant AAV production
JP2002153278A (ja) * 2000-11-22 2002-05-28 Hisamitsu Pharmaceut Co Inc ウイルスベクターの製造に用いられる細胞、その製法およびその細胞を用いたウイルスベクターの製造方法
AU2002305778A1 (en) * 2001-05-31 2002-12-09 The Rockefeller University Method for generating replication defective viral vectors that are helper free
AU2002367943A1 (en) * 2001-12-18 2003-12-22 University Of North Carolina At Chapel Hill Improved reagents and methods for producing parvoviruses
EP1844148A2 (en) 2005-01-31 2007-10-17 University of Iowa Research Foundation Nucleic acid silencing of huntington's disease gene
US20130023033A1 (en) * 2010-03-29 2013-01-24 The Trustees Of The University Of Pennsylvania Pharmacologically induced transgene ablation system
JP6661544B2 (ja) 2014-04-24 2020-03-11 ミルテニイ バイオテック ゲゼルシャフト ミット ベシュレンクテル ハフツング 遺伝子改変したｔ細胞の自動生成法
JP2018508223A (ja) * 2015-03-16 2018-03-29 ボード・オブ・トラスティーズ、サザン・イリノイ・ユニバーシティ 安定な遺伝子発現のための系
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US10858631B2 (en) * 2017-04-18 2020-12-08 Glaxosmithkline Intellectual Property Development Limited Methods for adeno-associated viral vector production

Also Published As

Publication number Publication date
JP7650800B2 (ja) 2025-03-25
CN113396222A (zh) 2021-09-14
US11739347B2 (en) 2023-08-29
EP3891290A1 (en) 2021-10-13
KR20210108423A (ko) 2021-09-02
JP2022516004A (ja) 2022-02-24
US20240018545A1 (en) 2024-01-18
WO2020132059A1 (en) 2020-06-25
EP3891290A4 (en) 2022-11-02
US12529074B2 (en) 2026-01-20
SG11202106217TA (en) 2021-07-29
US20200199627A1 (en) 2020-06-25
JP2025102801A (ja) 2025-07-08

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