CN115315518A - 重组aav的生产 - Google Patents

重组aav的生产 Download PDF

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Publication number
CN115315518A
CN115315518A CN202180023263.2A CN202180023263A CN115315518A CN 115315518 A CN115315518 A CN 115315518A CN 202180023263 A CN202180023263 A CN 202180023263A CN 115315518 A CN115315518 A CN 115315518A
Authority
CN
China
Prior art keywords
aav
nucleic acid
raav
cell line
cells
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
CN202180023263.2A
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English (en)
Chinese (zh)
Inventor
J·史密斯
J·格里格
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
AskBio Inc
Original Assignee
Asklepios Biopharmaceutical 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 Asklepios Biopharmaceutical Inc filed Critical Asklepios Biopharmaceutical Inc
Publication of CN115315518A publication Critical patent/CN115315518A/zh
Pending legal-status Critical Current

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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
    • 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
    • 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

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  • Life Sciences & Earth Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Production Of Liquid Hydrocarbon Mixture For Refining Petroleum (AREA)
CN202180023263.2A 2020-01-17 2021-01-15 重组aav的生产 Pending CN115315518A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202062962911P 2020-01-17 2020-01-17
US62/962,911 2020-01-17
PCT/US2021/013689 WO2021146591A2 (en) 2020-01-17 2021-01-15 Recombinant aav production

Publications (1)

Publication Number Publication Date
CN115315518A true CN115315518A (zh) 2022-11-08

Family

ID=76864730

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180023263.2A Pending CN115315518A (zh) 2020-01-17 2021-01-15 重组aav的生产

Country Status (8)

Country Link
US (1) US20230048994A1 (https=)
EP (1) EP4090750A4 (https=)
JP (2) JP7846625B2 (https=)
CN (1) CN115315518A (https=)
AU (1) AU2021207683A1 (https=)
CA (1) CA3162520A1 (https=)
IL (1) IL294775A (https=)
WO (1) WO2021146591A2 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4189077A1 (en) 2020-07-30 2023-06-07 Shape Therapeutics Inc. Stable cell lines for inducible production of raav virions
AU2022373653A1 (en) 2021-10-18 2024-05-02 Regeneron Pharmaceuticals, Inc. Eukaryotic cells comprising adenovirus-associated virus polynucleotides
WO2024129882A1 (en) * 2022-12-14 2024-06-20 Astellas Gene Therapies, Inc. Compositions and methods for improved production of adeno-associated viral particles
AU2024223271A1 (en) * 2023-02-17 2025-09-18 Synplogen Co., Ltd. Itr-rep gene complex
EP4527938A1 (en) * 2023-09-22 2025-03-26 LenioBio GmbH Method for cell-free polypeptide synthesis

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020160501A1 (en) * 1997-09-05 2002-10-31 Atkinson Edward M. Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US20100284971A1 (en) * 2006-06-19 2010-11-11 Asklepios Biopharmaceutical, Inc. Modified factor viii and factor ix genes and vectors for gene therapy
US20130052717A1 (en) * 2006-09-15 2013-02-28 Medimmune, Llc Mdck cell lines supporting viral growth to high titers and bioreactor process using the same
US20140242671A1 (en) * 2011-10-28 2014-08-28 The University Of North Carolina At Chapel Hill Cell Line for Production of Adeno-associated Virus
US20190292561A1 (en) * 2015-12-01 2019-09-26 Spark Therapeutics, Inc. Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use

Family Cites Families (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB0901593D0 (en) * 2009-01-30 2009-03-11 Touchlight Genetics Ltd Production of closed linear DNA
EP3039129B1 (en) * 2013-08-30 2018-06-27 Amgen Inc. High titer recombinant aav vector production in adherent and suspension cells
EP3810782A2 (en) * 2018-06-22 2021-04-28 Asklepios Biopharmaceutical, Inc. Vectors for gene delivery that persist within cells

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20020160501A1 (en) * 1997-09-05 2002-10-31 Atkinson Edward M. Methods for generating high titer helper-free preparations of released recombinant AAV vectors
US20100284971A1 (en) * 2006-06-19 2010-11-11 Asklepios Biopharmaceutical, Inc. Modified factor viii and factor ix genes and vectors for gene therapy
US20130052717A1 (en) * 2006-09-15 2013-02-28 Medimmune, Llc Mdck cell lines supporting viral growth to high titers and bioreactor process using the same
US20140242671A1 (en) * 2011-10-28 2014-08-28 The University Of North Carolina At Chapel Hill Cell Line for Production of Adeno-associated Virus
US20190292561A1 (en) * 2015-12-01 2019-09-26 Spark Therapeutics, Inc. Scalable methods for producing recombinant adeno-associated viral (aav) vector in serum-free suspension cell culture system suitable for clinical use

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
JOSHUA C GRIEGER 等: ""Production of Recombinant Adeno-associated Virus Vectors Using Suspension HEK293 Cells and Continuous Harvest of Vector From the Culture Media for GMP FIX and FLT1 Clinical Vector"", 《MOLECULAR THERAPY》, vol. 24, no. 2, 3 November 2015 (2015-11-03), pages 288 *
KINGA KARBOWNICZEK 等: ""Doggybone™ DNA: an advanced platform for AAV production"", 《CELL AND GENE THERAPY INSIGHTS》, 16 November 2017 (2017-11-16), pages 2 - 7 *

Also Published As

Publication number Publication date
WO2021146591A3 (en) 2021-10-28
JP2026032042A (ja) 2026-02-25
JP2023510590A (ja) 2023-03-14
EP4090750A4 (en) 2024-03-13
AU2021207683A1 (en) 2022-08-11
US20230048994A1 (en) 2023-02-16
CA3162520A1 (en) 2021-07-22
JP7846625B2 (ja) 2026-04-15
EP4090750A2 (en) 2022-11-23
IL294775A (en) 2022-09-01
WO2021146591A2 (en) 2021-07-22

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