Platform 2 is dedicated to the development and production of recombinant adeno-associated viruses (rAAV) based on gene ferries. These viral vectors are tailored to the very specific and unique needs of each application, enabling efficient and specific transduction of the respective target cells. This will contribute to the biosafety of optogenetic treatment and improve efficacy.
Therefore, we will screen the most suitable viral vector capsid variants, characterize them in detail, and combine them with additional features to improve their biosafety profile. We are also working to layer the expression of the optogenetic transgene onto the target cells. After the design freeze of the optimized GTMP candidate vectors, a scalable, GMP-compliant production process will be developed.

This platform brings together the expertise of several laboratories and Principal Investigators: vector researchers Kathrin Kusch at the German Primate Center and Hildegard Büning from the Institute of Experimental Hematology at Hannover Medical School, biostructure specialist Rubén Fernández Busnadiego at the Institute of Neuropathology, bioimaging expert Silvio Rizzoli in the Department of Neuro- and Sensory Physiology, and geneticist Bernd Wollnik at the Institute of Human Genetics.