The primary objective of Horizontal Spokes is to develop and share their expertise on the methodologies for designing, delivering, and manufacturing gene therapy products and RNA drugs. Their research focus is thus the technological advancement in Key Enabling Technologies (KET) and the establishment of core facilities for the production of RNA and gene therapy products, working in close collaborations with the Vertical Spokes aimed at identifying and developing individual drug targets.
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Goals of the project The fundamental science questions The therapeutic applications ProgramLed by National Research Council of Italy (CNR), Spoke 6 coordinates activities related to RNA-based technologies for RNA Drug Development.
Led by University of Bari Aldo Moro, Spoke 7 coordinates Biocomputing activities related to RNA-based technologies.
Led by University of Naples Federico II, Spoke 8 coordinates activities related to Platforms for RNA/DNA Delivery.
Led by University of Milan, Spoke 9 coordinates the Competence Center on RNA Drug Pharmacology – From target to therapy: pharmacology, safety, and regulatory competence center.
Led by IRCCS Bambino Gesù Children’s Hospital in Rome (OPBG), Spoke 10 coordinates activities related to pre-clinical development, Good Manufacturing Practices (GMP) and clinical trials of Gene Therapy Medicinal Products (GTMPs).