A new class of RNA biopharmaceuticals for regenerative medicine
PD Dr. Carsten Rudolph
Recipient: ETHRIS GmbH
Funding: GO-Bio Phase II (01.12.2011 - 31.05.2015, 3.571.148 Euro)
Biopharmaceuticals have to date found use in gene and protein therapies. However, such gene therapies may present health risks, including the initiation of cancer through the introduction of replacement DNA. In turn, the use of proteins as medicines is limited by an extremely short duration of action and efficacy, which makes their manufacture and application more time-consuming and expensive.
Against this backdrop, the team led by Carsten Rudolph has recognised the potential of the molecular transcription of genes, namely so-called messenger RNAs (mRNAs), as a new class of biopharmaceuticals, and has been further developing these for use in medicine. The Munich-based researchers are creating chemically modified RNA molecules that are exceptionally robust and do not trigger an immune response in the body. These ‘stabilised, non-immunogenic mRNA’ (SNIM RNA) can be administered into specific cells or tissues in the form of ‘naked’ molecules. Once in place, the transcripts begin the production of a therapeutic protein. Rudolph and his team have already successfully tested the concept of ‘transcription therapy’ in the mouse model.
In 2009, the company ethris GmbH was founded in Seefeld for the commercialisation of SNIM RNA technology, among other activities. The aim of the GO-Bio project is to further develop the transcription therapy for use in regenerative medicine. Bioactive surfaces will be produced and tested in the animal model in collaboration with ethris co-founder Christian Plank from the Klinikum rechts der Isar of the Technical University Munich. Examples of possible products include dental implants or bone replacement materials coated with SNIM-RNA that stimulate the body’s own healing processes. The project is set to lay the foundations for the start of clinical trials in the near future.