Advances in gene therapy delivery: next-generation transfection reagent for large scale AAV manufacturingPublished: April 20, 2020
The number of ATMP therapeutic-based medicines for inherited genetic disorders is in constant growth, with a global 32% increase in new clinical trials in the last 4 years. ATMPs have demonstrated their success with already more than ten approved for commercialization.
The success of AAV as the most promising viral vector for gene therapy is due to low immunogenicity, broad tropism and non-integrating properties. One major challenge for translation of promising research to clinical development is the manufacture of sufficient quantities of AAV. Transient transfection of suspension cells is the most commonly used production platform, as it offers significant flexibility for cell and gene therapy development. However, this method shows some limitations in large scale bioreactors: inadequate transfection protocol, reduced transfection efficiency and lower productivity.
To address this concern, we present data on the novel transfection reagent showing:
i) increased AAV titers
ii) improved transfection protocol for large scale bioreactors
iii) reproducibility of viral titers at different production scale
The aforementioned optimized parameters make this novel transfection reagent ideal for cell and gene therapy developers by combining the flexibility of transient transfection with scalability and speed to market.
- How to improve AAV viral vector yields by focusing on transfection step
- How to perform transfection at large scale
- How to ensure reproducibility at different production scales
Mathieu Porte, R&D Manager, BioProduction, Polyplus-transfection
Mathieu Porte is Bioproduction R&D Manager at Polyplus-transfection®. After obtaining his Biotechnology Engineering Degree from the renowned European School ESBS (Strasbourg, France), he worked as Research Assistant at New England Biolabs and UCSF. He joined Polyplus-transfection® in 2009. During 10 years in the Bioproduction R&D group, he carried out the development of several innovative transfection solutions dedicated to biomanufacturing.
Malik Hellal, PhD, Senior Scientist, Chemistry, Polyplus-transfection
Malik Hellal is a Senior Scientist in Chemistry at Polyplus-transfection®. With a PhD in Organic and Medicinal chemistry from the University of Strasbourg, a post-doctoral experience at Harvard NeuroDiscovery Center, and a Marie Curie fellow position at the Institut de Science et d’ingénierie Supramoléculaire, Malik is an expert in the conception and synthesis of biopolymers for the development of innovative targeted delivery solutions.