Kseniia Pidlisna
PIPS Host Organisation: Charles River Discovery Research (Industry Partner)
Project Title: Unravelling Genome Packaging during Recombinant AAV (rAAV) Gene Therapy Viral Vector Production

The main objective of the work carried out during Kseniia’s placement was to investigate different approaches to improving rAAV vector packaging by engineering new plasmid genomes with different ITR serotypes. Different genome-containing constructs were prepared with eGFP as the model genome flanked by alternative ITRs. These were assessed in terms of their impact on AAV production and genome packaging by producing rAAV in HEK293 cells under industrially relevant conditions at CRL. This work will form a distinct chapter of her thesis, and there is potential for publication depending on the study results.

This experience provided Kseniia with occupational knowledge and an understanding of the skills, attributes, and qualifications required for different roles within the industry. She was able to further expand her molecular cloning skills and learn new industry-specific techniques. She was also introduced to cell and gene therapy commercialisation and the development of such therapeutics, including the pathway from innovation to clinic. Kseniia learned to think outside the box when solving experimental problems and troubleshooting experiments. She also had the opportunity to further improve her presentation skills in front of new audiences and gained new professional connections.

Kseniia is very pleased with the work completed at CRL during her placement and the results gathered so far. She will continue this work for approximately two more months at the University of Kent to complete this section of the work, with a view to generating data and a story for a potential publication.