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The enormous achievements in RNA technology, immunology (vaccines) and nanotechnology in the last 10 years have paved the way for the development of RNA-based medicines. The particular advantage of RNA-based drugs is that they switch off or change the information read from the genes (DNA) at the RNA level before it is converted into proteins. RNA drugs do not change the genetic material itself and are therefore not gene therapies. RNA drugs can prevent the expression of a genetic defect and are therefore causally effective therapies for hereditary diseases. RNA therapeutics have the advantage that they can also be directed against proteins that cannot be reached by classical small-molecule drugs or biologics because they are localised intracellularly or do not offer specific targets.

However, the challenges for clinical development are considerable and conformity with "Good Manufacturing Practice" (GMP) is a prerequisite for first use in humans. The GMP area of Charité Research Organisation enables us to serve also these exciting new therapeutic approaches of RNA technologies. RNA agents are usually rapidly degraded and need to be stabilised by modifications to reach the cell nucleus and remain there long enough. The efficiency of RNA transport into the cytoplasm by overcoming extracellular and intracellular barriers remains crucial for successful RNA therapy. To solve the challenges in terms of pharmacodynamics and pharmacokinetics, various chemical modifications and formulations for RNA drug delivery are being tested. Many of the hurdles have now been overcome. Several RNA drugs, including RNA vaccines, have already been approved and many more are in clinical development. In this respect, this could mean the beginning of a new era in drug therapy.

Due to its research orientation and expertise, Charité Research Organisation GmbH is able to conduct and support innovative research projects with new RNA-based substances in the early phase of clinical development.

In this white paper, we provide an overview of the RNA-based drugs currently already available, their mechanisms of action, the challenges of RNA administration and current possible solutions.