The overall objective of the project is to develop a comprehensive platform for new oligonucleotide-based therapies (antimiRNAs, siRNAs and aptamers) that will boost and facilitate the arrival on the market of new therapies for the treatment of rare diseases (Rare Diseases with no medical response). In particular, the project will study retinitis pigmentosa, Leber’s congenital amaurosis, Almström syndrome, Fuch’s dystrophy, myotonic dystrophy, cachexia, Guillain-Barré syndrome and anaplastic thyroid carcinoma.

The seven constituent entities are specialised in the different activities necessary to complete the oligonucleotide-based drug development cycle, creating the industrial fabric necessary for the emergence of new RNA-based therapies.

In addition, the following specific objectives are pursued: