P21

Improving AAV Purification : A Novel Affinity Chromatography Approach

L Le Dortz(1,5,6) N Vitoux(1,5,6) C Robin(2,3,4) O Adjali(2,3,4) F Ducongé(1,5,6)

1:Université Paris Saclay; 2:Université de Nantes; 3:CHU de Nantes; 4:Inserm U1089; 5:CNRS UMR 9199; 6:CEA MIRCen

Adeno-associated virus (AAV) vectors are highly promising for gene therapy applications, with numerous preclinical and clinical successes demonstrating their potential. However, clinical translation requires high doses of AAV, and scaling up production remains a significant bottleneck. Efficient purification is essential for isolating AAV particles from cell lysates, as impurities can compromise both therapeutic efficacy and safety. Chromatographic techniques, particularly affinity chromatography, are the gold standard for large-scale AAV purification. Most ligands used in this approach are nanobodies derived from single-domain antibody fragments of the Camelidae family. However, these antibodies have several notable drawbacks, including high production costs, low yields and insufficient purity. Our project aims to develop innovative synthetic ligands designed to enhance AAV purification. These chemically synthesized ligands offer a cost-effective, scalable, and reproducible solution and are well-suited for GMP-compliant manufacturing.