BR112023026523A2 - Métodos para fabricar aav - Google Patents

Métodos para fabricar aav

Info

Publication number
BR112023026523A2
BR112023026523A2 BR112023026523A BR112023026523A BR112023026523A2 BR 112023026523 A2 BR112023026523 A2 BR 112023026523A2 BR 112023026523 A BR112023026523 A BR 112023026523A BR 112023026523 A BR112023026523 A BR 112023026523A BR 112023026523 A2 BR112023026523 A2 BR 112023026523A2
Authority
BR
Brazil
Prior art keywords
cells
polynucleotide sequence
aav
methods
culture medium
Prior art date
Application number
BR112023026523A
Other languages
English (en)
Inventor
Ana Valinhas
Bastiaan Leewis
Florian Dziopa
Original Assignee
Meiragtx Uk Ii Ltd
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 Meiragtx Uk Ii Ltd filed Critical Meiragtx Uk Ii Ltd
Publication of BR112023026523A2 publication Critical patent/BR112023026523A2/pt

Links

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
    • 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

Landscapes

  • 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)

Abstract

a presente invenção se refere a métodos para produzir partículas de vírus adenoassociado recombinante (raav) que incluem: uma fase de expansão que inclui aumentar o número de células em pelo menos um recipiente de cultura que contém o meio de cultura; introduzir nas células uma primeira sequência polinucleotídica que inclui um transgene flanqueado por repetições terminais invertidas de aav e, opcionalmente, uma segunda sequência polinucleotídica que inclui genes rep e cap de aav e/ou uma terceira sequência polinucleotídica que inclui um ou mais genes auxiliares; uma fase de produção que inclui cultivar as células nas quais a uma ou mais sequências polinucleotídicas foram introduzidas e adicionar íons de cálcio (ca) (isto é, sais de ca2+) ao meio de cultura de fase de produção; e isolar as partículas de raav.
BR112023026523A 2021-06-17 2022-06-17 Métodos para fabricar aav BR112023026523A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163211877P 2021-06-17 2021-06-17
PCT/IB2022/000347 WO2022263930A2 (en) 2021-06-17 2022-06-17 Aav manufacturing methods

Publications (1)

Publication Number Publication Date
BR112023026523A2 true BR112023026523A2 (pt) 2024-03-05

Family

ID=82850181

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112023026523A BR112023026523A2 (pt) 2021-06-17 2022-06-17 Métodos para fabricar aav

Country Status (8)

Country Link
EP (1) EP4355887A2 (pt)
KR (1) KR20240054958A (pt)
CN (1) CN117730154A (pt)
AU (1) AU2022292164A1 (pt)
BR (1) BR112023026523A2 (pt)
CA (1) CA3222723A1 (pt)
IL (1) IL309351A (pt)
WO (1) WO2022263930A2 (pt)

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2017112948A1 (en) * 2015-12-24 2017-06-29 University Of Florida Research Foundation, Inc. Improved aav production using suspension adapted cells
MA44546B1 (fr) 2016-06-15 2021-03-31 Univ California Virus adéno-associés variants et procédés d'utilisation
CA3112824A1 (en) * 2018-09-21 2020-03-26 Nightstarx Limited Compositions and methods for manufacturing gene therapy vectors
CN114829391A (zh) * 2019-11-14 2022-07-29 生物马林药物股份有限公司 用肝特异性基因疗法载体治疗遗传性血管性水肿

Also Published As

Publication number Publication date
CA3222723A1 (en) 2022-12-22
IL309351A (en) 2024-02-01
CN117730154A (zh) 2024-03-19
WO2022263930A2 (en) 2022-12-22
WO2022263930A3 (en) 2023-01-26
EP4355887A2 (en) 2024-04-24
AU2022292164A1 (en) 2024-02-01
KR20240054958A (ko) 2024-04-26

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