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:
To create an industrial niche to accelerate the development of oligonucleotide-based targeted therapies, as well as to reduce the translational gap between academia and industry.
Facilitating new oligonucleotide-based therapies to the market and bridging the gap between research and translation into therapies for rare diseases.
Generate new niches for therapeutic action through the development of appropriate delivery systems; strategies for targeted nanoliberation of oligonucleotides.
Providing a health response to RRDs with no effective therapeutic alternative.
Improve oligonucleotide synthesis and purification processes and develop new oligonucleotide synthesis strategies as alternatives to solid support based on biological and enzymatic processes to provide a more sustainable response to the oligonucleotide production market.
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