Platform 2 is dedicated to the development and production of recombinant adeno-associated viral vectors (rAAV) as gene shuttles. These viral vectors are tailored to the very specific and unique needs of each application, thus enabling efficient and specific transduction of the respective target cells. This will contribute to the biological safety of the optogenetic treatment and improve efficacy.
Therefore, we will screen the most suitable viral vector capsid variants, characterize them in depth, and combine them with additional features to enhance their biological safety profile. We are also working on restricting the expression of the optogenetic transgene to 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.