Highly Efficient and selective RNA delivery systems for the treatment of MEtabolic and cardiovascular diseaseS” – HERMES

Funder: Project funded under the National Recovery and Resilience Plan (NRRP), Mission 4 Component 2 Investment 1.3 – Cascade Call related to the Program "National Center for Gene Therapy and Drugs based on RNA Technology (CN RNA & GENE THERAPY) – Spoke 4” funded by the European Union – NextGenerationEU

Award Number: Cascade Call related to the Program “National Center for Gene Therapy and Drugs based on RNA Technology (CN RNA & GENE THERAPY) – Spoke 4 ”, CN00000041 CUP C93C22002780006, IRIS AP Archive number AE1E1DD55E;

Start date: 01/06/2024

End date: 31/05/2025

Total NRRP funding for the cascade call: € 2.873.667,83

Total NRRP funding for Unipr: € 290.000,00

Objectives

The National Center focuses on Gene therapy and RNA-based treatments, which represent a ground-breaking approach to healthcare. These personalized interventions leverage an individual’s molecular profile, precisely targeting disease pathways while minimizing effects on normal physiological mechanisms. Their flexibility allows adaptation to changing disease patterns, and their duration can vary from short-term (as seen with mRNA vaccines) to lifelong results through DNA modification.

Activities

The project focuses on the development of innovative RNA-based therapies to tackle age-related diseases that affect a significant portion of the elderly population. These conditions, which include cardiovascular and metabolic disorders, cancer, and muscle decline, contribute to global health challenges and pharmaceutical costs. The initiative aims to discover and validate new RNA targets that can regulate muscle growth, obesity, metabolic syndrome, diabetes, hypertension, inflammation, kidney disease, and heart conditions, ultimately leading to more effective treatments for these ailments.

To design, synthesize, and optimize novel RNA delivery vectors, structurally well-defined and monodispersed, able to selectively transfect different organs/tissues and cell lines, focusing the attention onto the target cells of metabolic and cardiovascular diseases such as macrophage, pancreatic beta cells, liver and bone marrow hematopoietic cells.

• Department of Chemistry, Life Sciences and Environmental Sustainability
• Department of Food and Drug

• University of Parma

The final target of the project will be: 

1. Definition and synthesis of macrocyclic RNA vectors for precision delivery of NAs with selectivity for their target cells. 
2. Definition of protocols for the synthesis of macrocyclic RNA vectors with reactive groups to be easily grafted with targeting ligands using very simple and fast preparations. 

The development of more sustainable and green procedures for the scale-up of the synthesis of the improved RNA vectors will potentially help to boost industrial competitiveness of Italian pharmaceutical companies. The results obtained by HERMES will also help the Spoke and CN to highly increase the Italian international reputation and attractiveness for the development of nucleic acid therapeutics and personalized medicine, contributing to change the social consideration and reputation of nucleic acid therapeutics, thus deeply impacting the global health system.

National Center for Gene Therapy and Drugs based on RNA Technology (rna-genetherapy.eu)