RNA for targeted therapies
RNA-based therapeutics are a drug class with huge potential for medicine — and scientists have barely scratched the surface of their possible use. Fraunhofer ITEM researchers develop novel drugs and methods for RNA-based therapeutic concepts.
The targeted use of RNA compounds based on coding or non-coding RNA sequences enables a tailored response of the respective target cells under certain pathological conditions. Fraunhofer researchers use a wide range of RNA-based compounds, such as small interfering RNA (siRNA), nucleoside-modified messenger RNA (modRNA), or RNA blockers – from target discovery through to clinical use. Bioinformatic models play a key role when it comes to selecting disease-associated RNAs, studying their interaction with other genes, and optimizing drug design.
Special viral and non-viral administration technologies, both at the molecular and equipment level, are being developed for the targeted use of RNA compounds. Local delivery via the airways is a promising solution in particular for RNA therapeutics used to combat lung diseases, as this enables the therapeutic agent to reach the site of action directly, thus minimizing systemic drug exposure.
Safety and efficacy testing of RNA compounds
For safety and efficacy testing of therapeutics, Fraunhofer ITEM researchers use existing and develop new model systems that are based on human cells and tissues. They can also test RNA-based therapeutics in early-phase clinical trials.
Process development and GMP production of RNA-based drugs
Fraunhofer ITEM researchers develop bioprocessing methods and production technologies for modular and automated manufacturing of RNA molecules and RNA nanocarriers, and these can be scaled right up to industry level.
Rapid, safe and reliable production technologies for the manufacture of RNA-based vaccines and drugs are a fundamental requirement for successful translation into marketable products.
To support this, process development and GMP production of mRNA therapeutics for early-phase clinical trials take place directly at Fraunhofer ITEM. The early-phase clinical trials can also be conducted in-house.