In WP4 novel techniques of gene therapy and gene editing we be developed as new therapeutic strategies for cancer prone inherited diseases and novel approaches of HSC gene therapy and gene editing for cancer treatment.

SPECIFIC OBJECTIVES

• Design optimized ex vivo gene therapies to prevent leukemia in patients with inherited cancer-prone hematopoietic syndromes and harness ex vivo expansion to potentiate polyclonal long-term repopulation in transplanted recipients. 
• Apply novel HSC gene therapy approaches to cancer patients capitalizing on tumor-cell purging, ex vivo expansion to obtain stable engraftment upon standard-of-care, alkylator-free chemotherapy and HDR-mediated knock-ins of anti-cancer genes into loci activated in tumor-infiltrating myeloid cells.

The activities of WP5 will generate resources to be integrated into future gene-therapy projects by collecting, integrating and reanalysing high-content multidimensional data by machine-learning to develop an Open Source tool for deep and reproducible characterization of genetically-engineered ATMPs.

SPECIFIC OBJECTIVES

• Generation of a Gene Therapy DB/Atlas to organize, annotate and share the multiomics data. 
• Design a Systems biology/AI toolkit to extract additional information from the combined datasets to identify generic and disease-specific fingerprints associated with gene therapy. 
• Generation of Open Source gene therapy applications/packages to characterize ATMPs

The dissemination and communication activities of X-PAND will be mainly targeted to raise awareness about project outcomes, engaging stakeholders to underpin the potential of future innovation advancements towards new markets. The WP6 is focused on Exploitation, Dissemination and Communication to ensure that the generated know-how and good practices, as well as the relevant results obtained are properly promoted to reach a broad audience and create an impact beyond the project itself. 

The exploitation measures will be carried out by the Business Development Office (BDO) of OSR to envisages the Identification and evaluation of potential valuable IP, a Market Analysis and Assessment and partnering activities aimed to negotiate agreements or strategic alliances with selected interested partners in order to carry out further development of our platform into ATMPs and, eventually, commercialization.

SPECIFIC OBJECTIVES

• Align project activities and results to the Portfolio strategy 
• Maximize X-PAND impact, focusing on activities to boost the commercial exploitation 
• Disseminate project results to targeted audiences 
• Increase visibility of project activities and results to the public through a proper communication strategy