Platform 2

Viral Vector Engineering

Platform II is dedicated to the development and production of recombinant adeno-associated virus (rAAV) based gene ferries. These viral vectors will be tailored towards the very specific and unique needs of each application and thereby enable efficient and specific transduction of the respective target cells. This will contribute to biosafety of the optogenetic treatment and improve efficacy.

Therefore, we will screen for most suitable viral vector capsid variants, characterize them in depth and combine them with additional features to improve their biosafety profile. Furthermore, we work on stratifying expression of the optogenetic transgene to the target cells. Upon design freeze of the resulting optimized GTMP candidate vectors, a scalable, GMP compliant production process will be developed.

This platform combines the expertise of several labs and principal investigators: viral vector researchers Kathrin Kusch at the German Primate Center and Hildegard Büning from the Institute of Experimental Hematology at the Hannover Medical School, biostructural specialist Rubén Fernández Busnadiego at the Institute of Neuropathology, bioimaging expert Silvio Rizzoli at the Department of Neuro- and Sensory Physiology and geneticist Bernd Wollnik at the Institute for human genetics.