Optimizing vector design at R&D and preclinical stages for improved safety and efficacy
The rAAV vector design affects not only the duration and localization of the therapeutic sequence transcription, but also manufacturing scalability and generation of manufacturing by-products, such as process- and product-related impurities, that compromise the safety and efficacy of rAAV vector particles.
SIRION BIOTECH will illustrate via gap analysis case studies how undersized rAAV vectors affect the level of process-related impurities such as co-packaged DNA plasmid sequences. Experimental data will be also presented to compare design strategies, such as adding “DNA stuffer sequences”, to minimize this safety concern. We will also discuss case studies in which oversized rAAV vectors are the root cause of suboptimal rAAV batch potency due to high levels of product-related impurities. Strategies for analytical quantification of these type of impurities will also be demonstrated with experimental data.
In addition to the impact on quality and safety, rAAV vector design contributes to the therapeutic sequence expression level and persistence in the target tissue. We will exemplify this point by focusing on the promoter element.
Key Learning Objectives
- The safety challenges of undersized and oversized rAAV vectors and design strategies to address them.
- The impact of rAAV vector design on manufacturing productivity and batch potency.
- The contribution of rAAV vector design to the therapeutic sequence expression level and persistence in the target tissue.
– Dr. Michael Salomon, SVP Development
– Dr. Irene Ferreira, Head of Clinical Support, Cell & Gene Therapy
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