16 Mar 2021Ended

Engineering AAV Lead Vectors for Gene Therapy


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upto 100
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Mar 2021


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The rAAV vector design affects not only the duration and localization of the therapeutic sequence transcription, but also manufacturing scalability .. Read more 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. We will exemplify this point by focusing on the promoter element. Join SIRION BIOTECH´s Dr Michael Salomon and Dr Irene Ferreira as they discuss the strategies for engineering rAAV lead vectors for gene therapy that address safety and efficacy requirements. Michael received his PhD from LMU in Munich, Germany.

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