Unlock Scalable, High-Quality AAV Manufacturing with Xcite® Transient Transfection and Stable Producer Cell Line Platforms
Join our experts, Suparna Sanyal and Peng Wang, as they explore innovative solutions for industrial-scale AAV (Adeno-Associated Virus) manufacturing. This session introduces two powerful production platforms: Xcite® Transient Transfection and Stable Producer Cell Lines designed to help gene therapy developers meet growing demands for efficiency, scalability, and quality.
Through real-world case studies and performance data, you'll discover how these platforms streamline both upstream and downstream processes, boost packaging efficiency, and enable seamless scale-up from shake flasks to 250L bioreactors.
We’ll also showcase our proprietary analytical tools, including advanced 3D digital droplet PCR (ddPCR), which deliver deep insights into AAV genome integrity and identity, critical for ensuring product quality. Whether you're working with standard or novel capsids, the Xcite® platform offers unmatched flexibility and performance, achieving titers up to 1E16 VG and packaging efficiencies of over 90%.
For teams focused on reducing the cost of goods and enhancing process robustness, our Stable Producer Cell Line platform offers a transformative alternative. By stably integrating all AAV components into the genome, this system eliminates the need for plasmids and helper viruses, enabling safer, more consistent, and scalable production.
This webinar walks you through the full development journey from GENE sequence to GMP readiness backed by compelling feasibility data and client success stories.
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FAQ from the webinar
Here is a summary of popular questions from the full webinar.
Q: What is Lonza's Excite® AAV Transient Transfection Platform?
The Excite® platform is a scalable, high-performance AAV manufacturing solution using Lonza's proprietary HEK293 suspension cell line and plasmids. It supports multiple serotypes and GOIs, delivering titers of 10¹¹–10¹² VG/mL and up to 94% full capsids post-polishing.
Q: How does the Excite® platform ensure scalability and consistency?
The process is validated from shake flask to 250L scale, with consistent titers and packaging efficiency. A comprehensive feasibility assessment at 3L scale establishes a reliable baseline, enabling smooth scale-up and GMP readiness.
Q: What are the benefits of Lonza's proprietary plasmids and cell line?
Using Lonza's plasmids and cell line boosts AAV titers by 2–9x and increases packaging efficiency by up to 4x compared to standard systems. This results in higher purity, reduced downstream burden, and improved safety profiles.
Q: Can Lonza accommodate novel capsids or customer plasmids?
Yes. Lonza can integrate customer-specific capsid sequences and GOIs into its platform. Side-by-side comparisons with Lonza's system can help determine the optimal path forward.
Q: What is the Excite® Stable Producer Cell Line (PCL) Platform?
This next-gen platform integrates all AAV components (Rep, Cap, Helper, GOI) into a stable HEK293 suspension cell line. It enables virus-free, cost-efficient, and scalable AAV production with titers exceeding 10¹² VG/mL and stable performance over 30+ passages.
Q: How does the PCL platform compare to transient transfection?
Top PCL clones deliver 2–4x higher titers than transient systems, with comparable packaging efficiency and infectivity. The platform eliminates the need for plasmids and enhances process robustness and reproducibility.
Q: What analytics does Lonza use to assess AAV quality?
Lonza employs advanced 3D digital droplet PCR (3D ddPCR) to measure AAV genome integrity and identity. This method correlates well with nanopore sequencing and functional assays, offering early insights into product quality.
Q: Is the Excite® platform suitable for fastidious GOIs or challenging serotypes?
Yes. Lonza uses DOE-based optimization (via shake flasks or AMBR systems) to fine-tune parameters like plasmid ratios, media, and transfection conditions, ensuring high productivity and packaging efficiency even for difficult constructs.
Q: What is included in Lonza's feasibility assessment?
The assessment includes shake flask screening, a 3L production run, analytical characterization, and a detailed GMP path plan. It's offered at a discounted rate with a typical turnaround of ~3 months.
Q: How can I get started with Lonza's AAV platforms?
Simply provide your GOI and capsid sequences. Lonza will integrate them into its system and guide you through each development stage. Contact the team to discuss your project and receive tailored support.