Tackling AAV Manufacturing Process Industrialization Challenges Using A Standard Platform Approach 

E. Gateau, A. Reniers, C. Lemaigre, S. Arnoult, C. Favre-Felix, T. Albano, J. Caballero-Montes, A. Salgado-Somoza, K. Watillon, S. Fauquenoy, V. Medvedev, H. P. Lesch


    AAV is considered as one of the most versatile tools for gene therapy applications, with its target tissue specificity and its non-replicating property benefits. Therefore, a growing number of AAV clinical trials are initiated each year and increasing safety concerns come with the need to strengthen our understanding of the manufacturing process and its impact on the final product quality. For this purpose, we developed a standardized manufacturing process following QbD principles ensuring sustainable yields for commercial stages and compliance with CMC requirements. 

    Viral Vectored Gene therapies approved worldwide