Integrating different approaches for establishing a multi-scale functional validation platform for RNA-based drugs in the CNS (MULTIVAL)

The National Center for Gene Therapy and RNA Technology, funded by the Next Generation EU PNRR project of the Italian Ministry of Research, aims to establish a collaborative hub where leading academic groups from Italian research institutions collaborate with national and international pharmaceutical, diagnostic companies, and contract research organizations. Together, they are dedicated to advancing RNA drug development. As part of this initiative, Spoke#9 harnesses cutting-edge methodologies to devise novel protocols ensuring the reproducible, rapid, and safe analysis of RNA drug absorption, distribution, metabolism, excretion, and toxicity (ADMET), as well as pharmacokinetics and pharmacodynamics. This abstract will showcase the partnership between the Institute of Neuroscience of the CNR and Humanitas University, highlighting their integrated neuropharmacology platform. This platform employs diverse approaches to facilitate functional testing of RNA drugs for central nervous system (CNS) diseases, with a keen emphasis on biomarker analysis and animal models to enrich our understanding of therapeutic outcomes within the CNS milieu. Moreover, it explores multimodal strategies to enhance the efficacy and safety assessment of RNA therapeutics within the CNS context.