Key Account Manager
Key Account Manager
Global Product Manager
Challenges of building an AAV platform in 2023: March 15, 2023 – 12:30 CET/CEST
Presented by Quentin Bazot, PhD, Viral Vector Manufacturing & Process Development Specialist, ABL Europe
AAV has emerged as a leading vector for gene delivery for treating various diseases due to its safety profile and efficient transduction of various target tissues. With the shift beyond ultrarare indications and the use of engineered synthetic AAV capsids, there is a need for new, scalable and easy-to-implement AAV platform processes compatible with different AAV serotypes. This presentation will discuss the challenges of starting the development of such a platform in 2023.
Quality by Design Process Development for rAAV manufacturing: March 15, 2023 – 5:40 pm CET/CEST
Presented by Nic Preyat, PhD, Associate Director, Gene Therapy CMC, UCB Pharma
Recombinant adeno-associated viral vectors (rAAV) are extensively used in gene therapy to introduce a genetic modification, and rAAV have been proven to be extremely effective and safe modalities for in vivo applications. The manufacturing of rAAV often relies on transient transfection of plasmids, and transfection reagents are used to convey plasmids through the plasma membrane. The selection of the best transfection reagents and the optimization of the transfection parameters is therefore key to achieving improved bioprocess productivity, consistency, and scalability. Our quality-by-design approach indicates that FectoVIR®-AAV (Polyplus) is a very efficient tool for GMP-compliant rAAV production.
Development of a novel helper plasmid: one step closer to the next generation rAAV vectors.
Laure Robert, Eric Mauro, Jonathan Havard, Sylvain Julien, Laure Robert, Carine Morel, Patrick Erbacher
Harnessing rAAVs as viral vectors for therapeutic transgene delivery still requires improvements in yields and specificity to lower vector doses, and therefore manufacturing cost, as well as to improve patient safety. To this end, our research is focused on developing novel technologies to ensure manufacturing of high yielding rAAV particles using transient transfection, as well as enhancing features of rAAV vectors that act on the overall size of packaged material and specificity of delivery. Here we present our state-of-the art approach to design new helper plasmids (phelpers) with the aim of improving both the infectiosity (TU/mL) and the quality (full/empty ratio) of the viral particle obtained from suspension cultures. We took the opportunity to exploit our proprietary DNA assembly method technology to explore the synergies of multiple genetic features modularly assembled in synthetic plasmids. Comparison of the biological activity of several versions of rationally designed pHelpers led us to identify the optimal configuration able to outperform existing helper plasmids in every tested bioproduction conditions. Our expertise in DNA plasmid design and assembly together with our scalable transfection solutions for rAAV manufacturing gives us the potential to improve both productivity and specificity of gene therapy products.
Discover our product & services:
FectoVIR®-AAV RUO and FectoVIR®-AAV GMP for large scale rAAV production in suspension cells.
Plasmid DNA engineering services and plasmid DNA manufacturing capabilities with Xpress Biologics, now a Polylplus company.