Speaker: Dr. Ariadna Boloix Amenós, Main researcher Childhood Cancer and Blood Disorders (VHIR)

I am a postdoctoral researcher in the Neural tumors Laboratory  in the group of Childhood Cancer and Blood Disorders. Specifically, I am working on project based on the development and implementation of RNA-based therapies in clinical practice for the treatment of pediatric solid tumors.
Despite my Pharmacist background, I started my PhD in a collaboration between the Childhood Cancer and Blood Disorders group at VHIR and the Nanomol group at ICMAB-CSIC, focusing on developing a non-liposomal lipid nanocarrier for RNA delivery. After defending my thesis, I continued my work at VHIR, evaluating the effectiveness of the nanomedicine that we have developed from in vitro studies to in vivo models. My goal is to bridge the gap between cutting-edge research and practical clinical applications to benefit young patients.

RNA therapeutics have revolutionized cancer treatment by targeting the entire transcriptome. Small RNA molecules, such as microRNA (miRNA) and small interfering RNA (siRNA), are commonly used in preclinical and clinical settings. However, challenges in stability, biodistribution, and tissue targeting hinder their translation into effective treatments. Nanotechnology offers a promising solution to enhance the efficacy and selectivity of these therapies, particularly in targeting cancer cells. Lipid-based nanovesicles, including liposomes, have been extensively utilized for delivering nucleic acids and were among the first to reach the market. However, the lack of a standardized clinical formulation is a hurdle, mainly due to limited industrial-scale manufacturing capabilities and challenges in tissue specificity.
Our objective is to advance the development of a nanomedicine platform utilizing non-liposomal lipid-based nanovesicles known as Quatsomes (QS). This platform aims to deliver small RNAs with tumor-suppressive properties, ultimately improving cancer treatment.

Host: Dr. Miguel Segura Ginard, Main researcher Childhood Cancer and Blood Disorders (VHIR)