AAV Transient Transfection And Producer Cell Line Platforms To Accelerate Your Path To IND

Current AAV manufacturing has challenges, including low production, scalability issues, expensive materials, complex supply chains, and inconsistent results between batches. To address these limitations and make AAV manufacturing more industrial, they have created two high-performing Xcite® AAV platforms that utilize transient transfection and stable producer cell line (PCL) technologies.

Lonza's transient transfection platform delivers some of the highest AAV titers in the industry (ranging from E11 to E12 viral genomes per milliliter), a high ratio of full to empty capsids, and a good overall yield (recovering 20-40% of the product). This platform integrates Lonza's proprietary suspension HEK293 cell line and plasmids, optimized manufacturing processes, and innovative AAV analysis techniques. The result is a robust and scalable platform (functional from 3 liters to 250 liters) with proven effectiveness for various serotypes (AAV2, 5, 6, 8, 9, and even novel capsids) and transgenes. This streamlined platform simplifies your path to obtaining an Investigational New Drug (IND) application by reducing complexity and shortening the timeline for lengthy process and analytical development.

Taking their proprietary HEK293 cells a step further, Lonza developed a high-producing, helper virus-free AAV stable PCL technology. AAV production in this system is tightly controlled and triggered using doxycycline. They have identified specific PCL clones that deliver even higher AAV titers (greater than 1E12 vg/mL) compared to the transient transfection process, while maintaining a comparable ratio of full to empty capsids (over 30% full capsids at harvest). These stable clonal cells can be maintained for long periods (over 30 passages) without a decline in AAV productivity and packaging efficiency. Since this helper-virus free AAV PCL eliminates the need for expensive plasmids, it reduces complexities in the supply chain and manufacturing process. Additionally, it can be easily scaled to much larger volumes (2000 liters and beyond).

Lonza's Xcite® AAV platforms offer a powerful combination that can deliver high quantities of top-quality AAV suitable for commercial manufacturing at the scale required for your valuable therapies.