The research program has two main goals: increasing the technological know-how necessary to design and deliver RNA-based and gene therapy medicinal products and identifying promising candidate drugs/genes in five major areas of human diseases (genetic diseases, cancer, metabolic/cardiovascular diseases, neurodegenerative disorders and inflammatory/infectious diseases). Two aims will thus be pursued. The first is to identify the most promising candidate targets for RNA-based drugs in five major areas of human diseases (genetic diseases, cancer, metabolic and cardiovascular, neurodegenerative, and inflammatory/infectious disorders) by supporting the progress in TRL of projects presented by highly qualified biomedical and clinical scientists (“Vertical Spokes”). The second is to build and disseminate the technological know-how necessary for designing, delivering and producing RNA drugs (“Horizontal Spokes”). For this purpose, a network of experts is created with skills covering biophysical, computational and experimental aspects of RNA biology, in order to support the activity and the competitiveness of the scientists focused on the development of RNA drugs for specific targets and simultaneously train a new generation of scientists that would ensure the implementation of the most updated RNA-based technologies.
National Center for Gene Therapy and Drugs based on RNA Technology
Abstract
People involved
Project information
Acronym
National Center for Gene Therapy and Drugs based on RNA Technology
Start date
01/11/2022
End date
31/10/2025
Role
Coordinator
Funds
National
Budget
Total budget: 12.663.229,33€
Total contribution: 9.394.395,03€
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