Christian Doppler Laboratory Knowledge-Based Production of Gene Therapy Vectors

Vision

Our goal is the advancement of modern gene therapy, as it is one of the most promising treatments against gene defects, cancer, auto immune diseases as well as infectious diseases. With highly efficient tools and reliable and scalable production platforms, new and better therapeutics will be made available and contribute to human health and well-being.

Mission

This initiative intends to join expertise available at three different Institutes of the Department of Biotechnology at BOKU University. The integrated application of expertise in functional genomics, applied virology, up/downstream processing and analytics and the expertise at Boehringer Ingelheim on up-stream processing allows to establish new strategies for developing highly efficient gene therapy vectors. Fundamental understanding of cellular processes, viral packaging and influences by the production process will lead to new strategies for a robust production platform for future gene therapy applications.

Translation

The mission of the CD Laboratory is to guide production of gene therapy vectors from empiricism driven approaches towards knowledge-based approaches which are in line with the Quality–by–Design concept for pharmaceutical production. This will be achieved by better understanding of the cellular response to virus production to be used for cell line optimisation. Advanced offline analysis will be established for better product characterisation. This information integrated with online analysis will be used as soft sensors for model based real time monitoring of the process enabling process control strategies. A miniaturized process development platform will enhance process understanding based on the investigation of interrelationship of subsequent process steps. The availability of all these methods will enhance efficiency and safety of rAAV production and will increase accessibility of these valuable pharmaceuticals.