ENDOSCAPE – a new EU project aims for a clinically applicable non-viral gene delivery technology
VIB participates in the EU-funded ENDOSCAPE project, a research and innovation action that aims to develop a novel gene delivery technology. ENDOSCAPE is a 6.8 M€ project funded by the European Union’s Horizon 2020 Research and Innovation programme under grant agreement No 825730 and is coordinated by scientists at the Charité in Berlin with participation of 10 industrial and academic partners from 7 European countries.
Gene therapy is one of the most promising treatment options for future advanced therapies in a broad range of diseases. Successful gene delivery requires the recognition of target cells as well as cytosolic and nucleosolic uptake of the gene. Currently, non-viral based gene delivery such as transfection reagents are only suitable for in vitro applications and clinical gene therapeutics delivery is accomplished via viral vectors, which still has major safety concerns and complex and costly manufacturing procedures, preventing future implementation for the treatment of diseases with large patients groups.
In the last 15 years, a class of specialized plant metabolites has been discovered that selectively mediates endosomal escape and cytoplasmic delivery of macromolecules only at low endosomal pH, thereby inducing a 40-fold enhanced gene delivery efficacy, in vivo. The currently employed methods of applying endosomal escape enhancers and gene therapeutic product, however, do not ensure that both compounds end up at the same time at the site of interaction.
The ENDOSCAPE technology platform will develop and collect proof of concept for a non-viral gene delivery technology with increased synchronization (in time and place) of both compounds. Proof of concept of the ENDOSCAPE technology has a major impact on the therapeutic opportunities for current and future macromolecule drugs for a broad range of diseases. The non-viral bases ENDOSCAPE technology will enhance therapeutic efficacy with lower therapeutic dose thereby reducing costs of healthcare, improving the health of patients worldwide, and strengthening the competitive landscape of the EU in the worldwide quest for such an advanced technology.
At the VIB-UGent Center for Plant Systems Biology, Prof. Alain Goossens and his team will have an important contribution to ENDOSCAPE by designing and optimizing a sustainable plant-based production pipeline of endosomal endoscape enhancers. The team of Alain Goossens will establish plant (hairy) root and hydroponic plant production lines from the medicinal plants that naturally produce the endosomal endoscape enhancers. Furthermore, they will genetically engineer these plant and/or root lines for enhanced production of the endosomal endoscape enhancers.
ENDOSCAPE was launched in January 2019 in Berlin and is a 4-year project.
- Log in to post comments