Abstract
RNA-binding proteins (RBPs) regulate post-transcriptional gene expression and participate in essential cellular processes such as splicing and mRNA transport. Alterations in their functions are associated with diseases such as cancer, neurodegeneration, and viral infections. Despite their therapeutic potential, RBPs remain poorly explored in drug development.In this PITE-Grandes Projetos grant, we propose to advance molecules (hit to lead) identified during the INCT project. Our partner, the Structural Genomics Consortium, is committed to co-financing this effort. The project will focus on four RNA-binding proteins associated with cancer and drug resistance, which have demonstrated interest from our national pharmaceutical industry partners.In the INCT project, we developed protein purification protocols, structural and cellular assays, and chemical screenings that identified promising compounds (hits) using two complementary methodologies: one conducted at CQMED and another at SGC-Toronto. Here, we propose activities integrating chemical synthesis, structural biology, and cellular biology to develop lead molecules, which will be evaluated for cell penetration, metabolic stability, toxicity, and disease-associated cellular models. The project aims to develop innovative molecular prototypes, offering new therapeutic approaches for cancer and neurodegenerative diseases, thus contributing to the advancement of medicinal chemistry and pharmaceutical innovation at an international level. The established relationship with the national pharmaceutical industry will be crucial for the absorption of the project's outcomes. (AU)
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