The project is divided into 5 work packages that will complete the full cycle of processes necessary for the development of oligonucleotide-based therapies for rare deseases:

PT1. New drug discovery strategies.

OLIGOFASTX has several companies with expertise in the development of RNA-based therapies that are in charge of the project to search for new therapeutic targets for rare diseases and the design of oligonucleotides for the treatment of these diseases.

PT2. New release systems.

Consortium members design and develop new formulations for targeted nanoliberation of oligonucleotides and perform scale-up strategies for in vivo studies.

PT3. Validation of new therapies for rare deseases.

The aim is to obtain the efficacy and toxicity profile of naked, modified, bioconjugated and/or formulated siRNAs, modified antimiRs and aptamers using in vitro functional models, animal models and preclinical toxicology for different pathologies.

PT4. Synthesis and scaling strategies.

OLIGOFASTX encompasses the task of research, optimisation and development with the generation of know-how and innovation in production for industrial purposes. Oligonucleotide production processes are developed and optimised for both the production of active ingredients and the search for cross-cutting solutions in the field of RNA-based compounds for therapeutic applications.

PT5. Exploitation of results.

The consortium members are oriented towards the exploitation of the project results through IP analysis and technology watch.