IMPACT
EUROPE FRAMEWORK. ECONOMIC/TECHNOLOGICAL IMPACT
Our platform holds the potential to bring the development of new ATMPs based on genetic HSC engineering to the next level, accelerating product optimization, validation and characterization. This potentially impacts on different categories of industries active in the cell and gene therapy arena enhancing the European technological leadership in the field. The interaction of X-PAND consortium with industrial companies which have complementary/non-overlapping interests in different aspects of the project such as HSC expansion, HSC gene editing technologies and HSC-based treatments for cancer, would provide ample space for exploiting the results obtained by X-PAND in different fields.
SCIENTIFIC IMPACT
X-PAND will contribute to fully engage and reinforce the EU’s scientific talent pool thanks to the i) involvement of leading investigators with complementary expertise in the consortium; ii) planned portfolio activities aimed at consolidating a European-wide network of researchers working on cell and gene therapy. Though this project has a clear development focus, we envisage that its concepts and tools will foster new fundamental research, feeding the EU innovation pipeline: e.g., optimization of the HSC platform and study of clonal HSC dynamics will provide valuable insight into HSC biology; the editing technologies and analysis of patient cells carrying mutations in DNA repair genes will inform cell biology questions about DDR; the bioinformatics tools developed in the project will be made available to the scientific community, with potential applications well beyond the scope of this project.
SOCIETAL IMPACT
Successful completion of this project bears the potential to turn the cell and gene therapy promise into clinical reality for an increasing number of patient categories within an accelerated time frame. These comprise patients with genetic diseases affecting hematopoietic cells and oncological patients. In the long run, this will improve the overall social-economic welfare of EU citizens impacting specifically those connected with patients via personal, familial, professional or social links. Moreover, the reduced costs associated to optimized protocols will positively impact on the production chain ultimately improving sustainability of HSC gene therapy for the healthcare system.