Day One (13th May 2025)Tuesday, 13 May 2025 - CET/CEST (Cent Europe Summer, GMT+2)

Current cell therapy manufacturing sciences are evolving rapidly to enable more reliable and faster scale-up and scale-out of lab-based processes to GMP manufacturing. The key challenge is striking a balance between significantly reducing time-to-market and integrating as many commercialization solutions as possible into the process. In this review, we share key lessons learned from recent engineering and GMP runs, highlighting the impact of automation and process modeling in enhancing readiness for final product supply.

In this presentation we will discuss key aspects related to rAAV production systems' scalability, addressing challenges and future directions for product characterization. Our research lies in improving stable cell line-based production systems for rAAV production. How to optimise the cell line generation process and improve the cell host by CRISPR-Cas9 genetic screen and single cell RNA-seq approaches will be discussed.

Recombinant protein expression is a highly regulated process consisting of transcription, translation, and protein folding. CHO-based expression often stays challenging for artificial therapeutic proteins, like Bispecific T-cell Engagers (BiTE®) due to reduced productivity compared to monoclonal antibodies. A differentiated insight into the evaluation of expression steps will be given, as well as strategies to find bottlenecks.