SELECTIVE HDACI’S FOR NON-ONCOLOGICAL APPLICATIONS

Steven Harper, Jesus Ontoria, Federica Ferrigno, Simona Ponzi, Emanuela Nizi, Ilaria Biancofiore, Savina Malancona, Giacomo Paonessa, Alberto Bresciani, Edith Monteagudo, Annalise Di Marco and Vincenzo Summa

IRBM-Science Park, Via Pontina km 30,600, Pomezia, 00071 Rome, Italy

Abstract

Epigenetic effects – often modulated by histone deacetylase (HDAC) enzymes– can result in dramatic changes to gene expression, and consequently HDAC inhibitors have evolved into a major class of anti-cancer agents. However, the potential to modulate gene expression by HDAC inhibition is also stimulating increasing interest for non-oncological indications, including rare and neglected diseases such as muscular dystrophies, hemoglobinopathies and parasitic diseases [1]. The present talk will provide a brief overview of our efforts and results in some of these fields, where several related but highly complementary selective series of inhibitors have been discovered.

REFERENCES

[1] Ontoria, J.M. et al. Discovery of a Selective Series of Inhibitors of Plasmodium falciparum HDACs, ACS Med. Chem. Lett., 2016, 7 (5), pp 454–459