RNA Therapeutics

Platform lead: Prof. Dr. Christian Bär

Potential pathway of an RNA-based drug

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Innovative therapeutics based on ribonucleic acid (RNA) have become known to a broad public in the course of vaccine development. However, RNA-based vaccines are only one example of their application. In many organ diseases (for example, lung, heart, liver), altered gene expression signatures are known to influence disease progression. This is where RNA therapy comes in to provide leverage on pathophysiological processes.

Targeted RNA-based therapies

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

The dovetailing of large clinical datasets, molecular biology data, and functional training of organ dysfunction has been demonstrated in many ways. Molecular RNA structures thus provide a prerequisite for the development of novel targeted RNA therapeutics.

Development of an RNA-based drug

The RNA Therapeutics platform will map a potential pathway that an RNA-based drug will follow from discovery to clinical use. Bioinformatic models will be used to select disease-associated RNAs that can be modulated via various RNA technologies. The RNA therapeutics will be transferred into protective encapsulation technologies (nanoparticles), qualitatively verified, and transferred into appropriate preclinical model systems to investigate therapeutic safety and efficacy.

Advanced RNA-based analytical methods to determine pharmacokinetic pathways complete the portfolio.

The processed proof-of-concept for RNA-based therapy development can be transferred to many other disease indications. In particular, the development of an appropriate encapsulation technology can be supportive here.

Synergistic effects will be achieved via the harmony of bioinformatics, in vitro/ex vivo models and the specific requirements of the encapsulation technologies. The platform therefore has a strong interdisciplinary character with dynamic control for future application models.

Another important development step will be the focus on RNA therapeutics production. This platform will act as a general realization platform for RNA-based therapies. Starting with small synthesis scales, RNA production will be available in larger quantities and incorporated into appropriate therapeutic strategies. Furthermore, the development of specific encapsulations/nanotechnology will be key for targeted therapy.


Building on the results of the RNA therapeutics platform, further research activities in the field of RNA production and targeted nanoparticles will be necessary to optimize RNA-based drugs for therapeutic application. We are in intensive exchange with other Fraunhofer sites and external partners on this and see convincing potential for a new class of drugs in a broad clinical application.


Expertise within the platform

  • Library screening and network analysis for the identification of key RNA factors in fibrosis
  • Bioinformatic prediction of sequence-optimized siRNAs or LNAs, or modified RNAs (modRNA) for mRNA overexpression
  • Development of a radiomics signature
  • Formulation of RNA therapeutics administered by inhalation
  • Biofunctionalized polymers which exhibit improved biocompatibility with simultaneous functional integration
    • Polyethylenimine (PEI)-based siRNA formulations
    • Liposomal formulations
    • Silica gel particles

Technologies within the platform

  • Omics pipeline for integrative RNA analysis and pathway stratification
  • Microscale Thermophoresis (MST) including validation of molecular binding for (nc)RNA inhibitor (match and mismatch, robustness and reproducibility analysis and evaluation; quality control of data (aggregates, sample adsorption, fluorescence variation); calculation and fitting of binding curves (KD vs. hill fitting))
  • Multi-organ in vitro study (skin, cornea, blood-brain barrier, intestine) to test the biocompatibility of siRNA candidates
  • Ex vivo tissue slices for safety and efficacy testing of RNA therapeutics and their delivery systems
  • Chromatography-based (HPLC) method for the quantification of RNA therapeutics in body fluids and tissues for the collection of pharmacokinetic (PK) parameters
  • NanoAssemblr® system from Precision Nanosystems at Fraunhofer ITEM
  • Visual dashboard for the analysis of the omics data and the corresponding decision support

Infrastructure within the platform

GXP – quality assurance according to international standards

To guarantee that the work performed within the platform "RNA technologies" satisfies internationally accepted quality standards, Fraunhofer has implemented the GXP quality assurance systems.

The GXP quality assurance systems include Good Laboratory Practice (GLP), Good Clinical Practice (GCP), and Good Manufacturing Practice (GMP). With their respective scopes of application, these quality assurance systems cover the translational approach in the platform’s spectrum of activities. 

Here you can find more information about our GXP platform at Fraunhofer ITEM as well as our certificates. 

RNA Therapeutics: Current platform projects

  • SARS-CoV-2 infection timeline in hiPSC cardiomyocytes
  • RNA sequencing to identify dysregulated circRNAs
  • Modulation of circRNAs followed by infection in hiPSC cardiomyocytes
  • circRNAs based regulation of SARS-CoV-2 infection
  • siRNA targeting of circRNAs for COVID-19 therapy

  • Multimodal in silico analysis of human antifibrotic gene expression fingerprints
  • Endogenous silencing of identified druggable RNA targets in vitro 
  • Packaging with nanoparticle carrier systems 
  • Validation of identified RNA targets ex vivo (PCLS, LMS) 
  • Nebulization trials to bridge translational gap

  • Single-cell analysis of human BAL cells 
  • In silico analysis of transcription factors in T cell subsets 
  • siRNA generation and in vitro evaluation on human BAL cells 
  • Generation of nanoparticles for targeted siRNA delivery 
  • In vivo experiments to test efficacy of intratracheal application of targeted delivery approaches using different nanoparticles/-siRNAs 

Your contact persons for RNA Therapeutics

Christian Bär

Contact Press / Media

Prof. Dr. Christian Bär

Platform Lead RNA Therapeutics

Phone +49 511 5350-120

Sabine Kafert-Kasting

Contact Press / Media

Dr. Sabine Kafert-Kasting

Project management RNA Therapeutics

Phone +49 511 5350-363