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Riposizionamento E Sviluppo Economico di Attività di Ricerca su Composti HDACi



Description of the project

The RESEARCH project falls under the scope of epigenetics and specifically aims to evaluate the possibility of reprogramming the cell fate through a pharmacological intervention. This project will leverage on different levels of biological and medical knowledge, in order to:

  • Provide better understanding of epigenetic mechanisms underlying tissue regeneration phenomena
  • Optimize preexisting active molecules on these mechanisms in order to make them attractive for pharmaceutical development
  • Demonstrate the efficacy of the above mentioned molecules both in the context of a rare disease (Duchenne muscular dystrophy), in the cardiovascular/antifibrotic fields and in areas where HDAC inhibition proves to be beneficial


Malfunctions in the normal mechanisms of gene expression control are the basis of many pathologies, and the possibility of modifying gene expression therefore represents a therapeutic approach. One of the main methods of transcriptional activation depends on the relaxation of chromatin by inhibition of the deacetylating enzymes (HDAC’s). Molecules capable of performing this function have been approved for human use, however their use to date has been limited by their toxicity.

IRBM have already identified a proprietary class of HDAC inhibitors having low toxicity, and aims to prove its effectiveness in Duchenne muscular dystrophy through the collaboration with the University La Sapienza, where similar molecules have given encouraging results. These studies are essential in order to expand our understanding of epigenetic modulation and to identify therapeutic approaches that benefit patients.
A further objective of the project is to obtain isoform selective inhibitors (e.g. for the isoform 3, 6 or others), which can reduce the toxicity linked to inhibition of all HDAC isoforms and which may be relevant in areas in which these isoforms are involved (such as, for example, cardiovascular or anti-fibrotic indications).


The RESEARCH project is positioned in the field of industrial pharmaceutical research and will advance, thanks to the collaboration between the IRBM and the “Charles Darwin” Department of Biology and Biotechnology of the “La Sapienza” University, expertise and know-how developed in recent years by both these research groups.The results obtained during this project will be the subject of patents and scientific publications.

Financial support received

The project is the subject of the funding POR FESR LAZIO 2014-2020. Progetto T0002E0001. Riposizionamento competitivo. Avviso Pubblico “LIFE 2020”. Impegno di spesa sui capitoli del bilancio regionale A42155, A42156, A42157, A42161, A42162 e A42163 – Esercizio Finanziario 2016.

Unione Europea
Repubblica Italiana
Regione Lazio
Fesr fondo europeo di sviluppo regionale
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