White Paper: Improving AAV Vector Quality with In-Process Monitoring of Capsid Formation  

Adeno-associated viruses (AAVs) are widely used for gene therapy, however, large-scale production of high-quality vectors at a high yield remains challenging. A major issue is the inefficiency in DNA packaging, leading to a high proportion of capsids that are either empty or contain unwanted DNA fragments. Increasing the percentage of full capsids at harvest is crucial for enhancing the effectiveness of AAV-based therapies. However, conventional analytical methods struggle to monitor the ratio of full to empty capsids during upstream processing due to the complexity of the samples.  

In collaboration with Refeyn and the Cell and Gene Therapy Catapult (CGT Catapult) and supported by Innovate UK, Rentschler Biopharma has developed a rapid, small-scale purification process that revolutionizes AAV production. This innovative approach requires low-volume samples and enables in-process monitoring of full, partial, and empty capsids using mass photometry.  

Key benefits of this solution: 

• Process Optimization – streamlined production process with in-process monitoring, leading to improved efficiency and reduced costs 
• Enhanced Quality – increased percentage of full capsids, ensuring more effective delivery of therapeutic transgenes 
• Cost-effective Production – integrated solution into Rentschler’s AAV manufacturing toolbox to enable cost-effective, higher-quality AAV products  

Our white paper explores the details of this breakthrough project, providing an in-depth look at the innovative purification process established to streamline AAV manufacturing. Read this white paper to gain valuable insights into optimizing AAV production strategies and enhancing the quality of gene therapy products.  

Download white paper here: