Pilot-scale process development for recombinant adeno-associated virus (rAAV) production based on high-density Sf9 cell culture

Abstract Background In recent years, gene therapy drugs have been widely marketed, and their effectiveness and potential have been confirmed. Thus, increasing their production on an industrial scale is critical. Recombinant adeno-associated viruses (rAAVs) are optimal vectors for gene therapy applic...

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Main Authors: Xinran Li, Jieyi Gu, Haoquan Wu, Yuanyuan Xie
Format: Article
Language:English
Published: BMC 2024-11-01
Series:Virology Journal
Subjects:
Online Access:https://doi.org/10.1186/s12985-024-02550-4
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author Xinran Li
Jieyi Gu
Haoquan Wu
Yuanyuan Xie
author_facet Xinran Li
Jieyi Gu
Haoquan Wu
Yuanyuan Xie
author_sort Xinran Li
collection DOAJ
description Abstract Background In recent years, gene therapy drugs have been widely marketed, and their effectiveness and potential have been confirmed. Thus, increasing their production on an industrial scale is critical. Recombinant adeno-associated viruses (rAAVs) are optimal vectors for gene therapy applications, and the baculovirus expression vector system (BEVS), which is based on Sf9 cell culture, is a common tool for rAAV production. Methods In this work, an Sf9 cell fed-batch process was developed using shake flasks. In the laboratory-scale bioreactor, four processes were selected as the key factors when carrying out the orthogonal experiment. On the basis of the equal P/V principle and considering the problem posed by air bubbles, a pilot-scale level bioreactor process was established. Results Here, we describe a method in which a BEVS was used to produce rAAV vectors, with the cell density increasing to 22.8 × 106 cells/mL and the rAAV titre increasing to 20 × 1011 VG/mL upon adding feed material. By resolving the problems associated with high-density cell culture and air bubbles, this process was successfully scaled to a 50 L pilot-scale level. Conclusions This successful experiment not only provides a technological basis for further scale-up but also guarantees product capacity. We hope that this development process can provide reference data for studying cell culture-based drug production.
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institution Kabale University
issn 1743-422X
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spelling doaj-art-df07bcbad12f42859b6236a861c13e712024-11-10T12:06:38ZengBMCVirology Journal1743-422X2024-11-0121111010.1186/s12985-024-02550-4Pilot-scale process development for recombinant adeno-associated virus (rAAV) production based on high-density Sf9 cell cultureXinran Li0Jieyi Gu1Haoquan Wu2Yuanyuan Xie3College of Pharmaceutical Science, Zhejiang University of TechnologyKanglin Biotec (Hangzhou) Co., LtdKanglin Biotec (Hangzhou) Co., LtdCollege of Pharmaceutical Science, Zhejiang University of TechnologyAbstract Background In recent years, gene therapy drugs have been widely marketed, and their effectiveness and potential have been confirmed. Thus, increasing their production on an industrial scale is critical. Recombinant adeno-associated viruses (rAAVs) are optimal vectors for gene therapy applications, and the baculovirus expression vector system (BEVS), which is based on Sf9 cell culture, is a common tool for rAAV production. Methods In this work, an Sf9 cell fed-batch process was developed using shake flasks. In the laboratory-scale bioreactor, four processes were selected as the key factors when carrying out the orthogonal experiment. On the basis of the equal P/V principle and considering the problem posed by air bubbles, a pilot-scale level bioreactor process was established. Results Here, we describe a method in which a BEVS was used to produce rAAV vectors, with the cell density increasing to 22.8 × 106 cells/mL and the rAAV titre increasing to 20 × 1011 VG/mL upon adding feed material. By resolving the problems associated with high-density cell culture and air bubbles, this process was successfully scaled to a 50 L pilot-scale level. Conclusions This successful experiment not only provides a technological basis for further scale-up but also guarantees product capacity. We hope that this development process can provide reference data for studying cell culture-based drug production.https://doi.org/10.1186/s12985-024-02550-4rAAVSf9Scale-upProcess developmentGene therapy
spellingShingle Xinran Li
Jieyi Gu
Haoquan Wu
Yuanyuan Xie
Pilot-scale process development for recombinant adeno-associated virus (rAAV) production based on high-density Sf9 cell culture
Virology Journal
rAAV
Sf9
Scale-up
Process development
Gene therapy
title Pilot-scale process development for recombinant adeno-associated virus (rAAV) production based on high-density Sf9 cell culture
title_full Pilot-scale process development for recombinant adeno-associated virus (rAAV) production based on high-density Sf9 cell culture
title_fullStr Pilot-scale process development for recombinant adeno-associated virus (rAAV) production based on high-density Sf9 cell culture
title_full_unstemmed Pilot-scale process development for recombinant adeno-associated virus (rAAV) production based on high-density Sf9 cell culture
title_short Pilot-scale process development for recombinant adeno-associated virus (rAAV) production based on high-density Sf9 cell culture
title_sort pilot scale process development for recombinant adeno associated virus raav production based on high density sf9 cell culture
topic rAAV
Sf9
Scale-up
Process development
Gene therapy
url https://doi.org/10.1186/s12985-024-02550-4
work_keys_str_mv AT xinranli pilotscaleprocessdevelopmentforrecombinantadenoassociatedvirusraavproductionbasedonhighdensitysf9cellculture
AT jieyigu pilotscaleprocessdevelopmentforrecombinantadenoassociatedvirusraavproductionbasedonhighdensitysf9cellculture
AT haoquanwu pilotscaleprocessdevelopmentforrecombinantadenoassociatedvirusraavproductionbasedonhighdensitysf9cellculture
AT yuanyuanxie pilotscaleprocessdevelopmentforrecombinantadenoassociatedvirusraavproductionbasedonhighdensitysf9cellculture