RNA therapeutics

Innovative Therapeutics based on ribonucleic acid (RNA) have become known to the general public in the context of vaccine development. However, RNA-based vaccines are only one example of their application. In many organ diseases (e.g. lung, heart, liver), altered gene expression signatures are known to influence the progression of the disease. RNA therapy offers a starting point for influencing pathophysiological processes.

Targeted RNA-based therapies

Understanding the altered gene expression signatures in the disease is key to developing a targeted RNA-based therapeutic strategy. In particular, the field of medical informatics and bioinformatics is an essential component for identifying important switch molecules and building a bridge between patient data and applied research.

The interlocking of large clinical data volumes, molecular biological data and functional vocational training of an organ dysfunction has been demonstrated in many ways. Molecular RNA structures thus form a prerequisite for the development of novel, targeted RNA therapeutics.

Development of a drug based on RNA

The RNA Therapeutics platform will outline a possible pathway for an RNA-based drug from discovery to clinical application. Using bioinformatic modeling, disease-associated RNAs will be selected that can be modulated using various RNA technologies. The RNA therapeutics are transferred into protective encapsulation technologies (nanoparticles), qualitatively verified and transferred into suitable preclinical model systems to investigate their therapeutic safety and efficacy.

Advanced RNA-based analytical methods for determining pharmacokinetic pathways round off the portfolio.

The proof-of-concept process for the development of an RNA-based therapy can be transferred to many other disease indications. In particular, the development of a suitable encapsulation technology can be beneficial here.

Synergies are achieved through the interaction of bioinformatics, in vitro/ex vivo models and the specific requirements of encapsulation technologies. The platform is therefore highly interdisciplinary in nature, with dynamic control for future application models.

A further important development step will be the focus on the production of RNA therapeutics. This platform will serve as a general realization platform for RNA-based therapies. Starting with smaller syntheses, the aim is to make RNA production available in larger quantities and to incorporate it into appropriate therapeutic strategies. Furthermore, the development of specific encapsulation/nanotechnologies will be an important aspect for targeted therapies.

Outlook

Based on the results of the RNA therapeutics platform, further research activities in the section of RNA production and targeted nanoparticles will be necessary to optimize RNA-based drugs for therapeutic application. To this end, we are in close contact with other Fraunhofer locations and see convincing potential for a new class of drugs in broad clinical application.