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.
One of the major problems in the field of non-viral gene therapy is the inefficient and safe delivery of genetic material for therapeutic use in patients.
The ENDOSCAPE brings together an interdisciplinary chain of European partners with academic and industrial technologies and expertise in order to develop a beyond state of the art non-viral clinically applicable gene delivery technology .
ENDOSCAPE will overcome a longstanding and major bottleneck in the field of gene delivery, namely efficient, safe and cost-effective transfer of gene therapeutic products into the cytosol.
The ENDOSCAPE technology will be designed to allow targeting of any addressable cell type with all known genetic agents, thereby ensuring better patient therapy not limited to inherited disorders, but also for cancer therapy and therefore of importance for large patient groups. ENDOSCAPE technology is exploiting a collection of unique molecules for enabling the delivery of genes into the cell. Development of new targeting ligands is under continuous investigation by numerous research groups and companies worldwide. ENDOSCAPE represents a toolbox which enables targeted drug delivery, allowing for customized drug applications and for future developments in the field of cell targeting techniques.
In the present project, we will focus on commonly used targeting concepts to demonstrate proof of concept of the technology. The long-term vision of ENDOSCAPE is market uptake of a novel technology that can be used for intracellular delivery of any applicable drug for medical treatment, as well as its application in personalized medicine.
To reach the goals of ENDOSCAPE, secondary metabolites will be produced in contained systems.
Only a strong interdisciplinary European collaboration of researchers, biotech, clinicians, and manufacturing organizations, together with translation and regulatory knowledge of the ENDOSCAPE Advisory Committee, allows successful development and exploitation of the technology.
Public summaries and progress reports will be published here soon
The worldwide market of gene therapeutics will rapidly grow in the coming years covering potential treatments for a wide range of disease areas. The current viral vector-based gene therapeutic technologies have significant challenges, such as safety, manufacturing logistics, and associated high costs.The ENDOSCAPE technology platform might represent a good alternative for a viral gene delivery technology, Of course, the long-term effects and safety of viral gene delivery still have to be evaluated.
The gene delivery technology developed by the ENDOSCAPE consortium can be incorporated into existing medicinal drug candidates, its safe and can be produced at relatively low costs thereby impacting and changing the future landscape of gene therapeutic delivery, significantly.