AU2020270960B2 - Engineered producer cell lines and methods of making and using the same - Google Patents

Engineered producer cell lines and methods of making and using the same

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Publication number
AU2020270960B2
AU2020270960B2 AU2020270960A AU2020270960A AU2020270960B2 AU 2020270960 B2 AU2020270960 B2 AU 2020270960B2 AU 2020270960 A AU2020270960 A AU 2020270960A AU 2020270960 A AU2020270960 A AU 2020270960A AU 2020270960 B2 AU2020270960 B2 AU 2020270960B2
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Prior art keywords
cell line
seq
raav
expression
cells
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AU2020270960A
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AU2020270960A1 (en
Inventor
Nicholas Richards
Aubrey R. Tiernan
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Ultragenyx Pharmaceutical Inc
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Ultragenyx Pharmaceutical Inc
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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
    • C12N15/11—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
    • 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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6869—Methods for sequencing
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Medicinal Chemistry (AREA)
  • Analytical Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
AU2020270960A 2019-04-12 2020-04-09 Engineered producer cell lines and methods of making and using the same Active AU2020270960B2 (en)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962833548P 2019-04-12 2019-04-12
US62/833,548 2019-04-12
US201962839207P 2019-04-26 2019-04-26
US62/839,207 2019-04-26
US202062979483P 2020-02-21 2020-02-21
US62/979,483 2020-02-21
PCT/US2020/027489 WO2020210507A1 (en) 2019-04-12 2020-04-09 Engineered producer cell lines and methods of making and using the same

Publications (2)

Publication Number Publication Date
AU2020270960A1 AU2020270960A1 (en) 2021-11-04
AU2020270960B2 true AU2020270960B2 (en) 2026-01-29

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020270960A Active AU2020270960B2 (en) 2019-04-12 2020-04-09 Engineered producer cell lines and methods of making and using the same

Country Status (15)

Country Link
US (2) US12018288B2 (https=)
EP (2) EP4603579A3 (https=)
JP (2) JP7560475B2 (https=)
KR (1) KR20210152514A (https=)
CN (1) CN113966395B (https=)
AU (1) AU2020270960B2 (https=)
BR (1) BR112021020391A2 (https=)
CA (1) CA3136246A1 (https=)
CO (1) CO2021013699A2 (https=)
IL (1) IL287106A (https=)
MX (1) MX2021012489A (https=)
PH (1) PH12021552577A1 (https=)
SG (1) SG11202110904PA (https=)
TW (1) TWI859216B (https=)
WO (1) WO2020210507A1 (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
WO2023019168A1 (en) 2021-08-11 2023-02-16 Ultragenyx Pharmaceutical Inc. Compositions and methods for treating a muscular dystrophy
AU2022373653A1 (en) 2021-10-18 2024-05-02 Regeneron Pharmaceuticals, Inc. Eukaryotic cells comprising adenovirus-associated virus polynucleotides
US20250019668A1 (en) 2021-10-29 2025-01-16 Ultragenyx Pharmaceutical Inc. Engineered cell lines for increased production of recombinant adeno-associated virus (raav)
CN119137263A (zh) 2022-03-07 2024-12-13 奥特吉尼克斯制药公司 改进的分批aav产生系统和方法
WO2025078616A1 (en) * 2023-10-12 2025-04-17 Cevec Pharmaceuticals Gmbh Cellular factors affecting recombinant aav production
WO2025226558A1 (en) 2024-04-22 2025-10-30 Ultragenyx Pharmaceutical Inc. Multi-step filtration and clarification methods for recombinant adeno-associated virus purification
WO2026078664A1 (en) 2024-10-10 2026-04-16 Instituto De Biologia Experimental E Tecnológica (Ibet) Transcription factor enhancing recombinant adeno-associated virus production, products, methods and uses thereof

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Also Published As

Publication number Publication date
MX2021012489A (es) 2021-11-12
US20200325455A1 (en) 2020-10-15
CO2021013699A2 (es) 2021-10-29
IL287106A (en) 2021-12-01
JP2022526646A (ja) 2022-05-25
US20240376440A1 (en) 2024-11-14
JP7560475B2 (ja) 2024-10-02
AU2020270960A1 (en) 2021-11-04
US12018288B2 (en) 2024-06-25
KR20210152514A (ko) 2021-12-15
EP3953458A1 (en) 2022-02-16
BR112021020391A2 (pt) 2022-02-08
SG11202110904PA (en) 2021-10-28
TWI859216B (zh) 2024-10-21
JP2024107295A (ja) 2024-08-08
EP4603579A3 (en) 2025-12-10
TW202104593A (zh) 2021-02-01
WO2020210507A1 (en) 2020-10-15
PH12021552577A1 (en) 2022-04-04
CN113966395A (zh) 2022-01-21
CA3136246A1 (en) 2020-10-15
JP7706607B2 (ja) 2025-07-11
EP4603579A2 (en) 2025-08-20
CN113966395B (zh) 2025-07-04

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