Introducing Xcite® AAV platform technology

With the AAV market gaining momentum, gene therapy developers are under increasing pressure to advance their AAV-based therapeutics for clinical trials quickly, while managing the risks and the time-consuming steps of gene therapy drug development.

Leveraging Lonza expertise and the same tools and technologies implemented for our platform process, you can balance risk and speed, while maintaining high AAV productivity. Xcite®  AAV platform technology is established based on our proprietary suspension HEK293 cell line and plasmid vectors, proven to enhance AAV production. These materials are now available for use in your own laboratory.

High Performing Production HEK293 Cell Line and Plasmids

AAV-based therapies require a scalable GMP manufacturing platform that is robust and can support high titers during production. Traditionally AAV manufacturing for gene therapy entailed the use of labor-intensive adherent-based HEK293 processes not amenable to scale-up. Suspension adaptation of HEK293 cells has enabled more scalable processes but remains time-consuming. Low AAV productivity and lack of platform processes continue to be key challenges driving AAV production cost up. In response to these challenges, Lonza has established a robust and scalable suspension manufacturing platform for AAV production.

At the heart of our Xcite® AAV platform process is our proprietary HEK293 Host Cell Line (5B8) and AAV Production Plasmids. The 5B8 cell line is a HEK293 suspension host cell line, selected and developed at Lonza for high AAV productivity. The 5B8 cell line is cultured in animal-component- free conditions and has demonstrated high expression and scalability for AAV production (Figure 1). Research and cGMP cell banks are available, under license, to take to your laboratory.

Figure 1: AAV-GFP Expression in Lonza HEK293 Cell Line versus alternate cell line. Anc80 is an in-silico-derived serotype (Zinn et al., Cell Reports, 2015).

Figure 2: Superior performance of Lonza proprietary AAV production plasmids compared to standard plasmids in Lonza 5B8 cell line. Anc80 is an in-si/ico- derived serotype (Zinn et al., Cell Reports, 2015).

The Xcite®AAV platform technology is now available to take to your laboratory

5B8 Production Host Cell Line

  • Suspension HEK 293 cell line
  • High AAV productivity for multiple serotypes and GOIs
  • Proven Scalability for AAV production
  • Research and cGMP cell banks available under license

AAV Production Plasmids

  • Proprietary know-how on pHelper plasmid and promoter for balance Redp/Cap expression
  • Higher productivity and titers1
  • Optimized for multiple AAV serotypes and GOIs

1 Lonza in-house data, comparison to standard commercially available plasmids.

Learn more about how improved and rebalanced Helper and RepCap Plasmids can ramp up your AAV productivity.

Contact us today to find out more and discuss how Lonza's AAV platform can ramp up your AAV production.

Disclaimer: The information contained herein is intended for general marketing purposes only. While Lonza makes efforts to include accurate and up-to-date information, it makes no representations or warranties, expressed or implied, as to the accuracy or completeness of the information provided herein and disclaims any liability for the use of this publication and that all access and use of the information contained herein are at their own risk. Lonza may change the content of this publication at any time without notice but does not assume any responsibility to update it. All trademarks belong to Lonza and are registered in Switzerland, EU and/or USA or belong to their respective third party owners. © 2023 Lonza. All rights reserved 05.2023.1

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