Current overview of intriguing RNA therapies

RNA is at the prime focus of attention. Not only because of the various corona vaccines that are based on RNA technology, but also because of the therapeutic applications that are rapidly increasing in number. In an article in EMBO Molecular Medicine, researchers from Radboudumc, together with European colleagues, provide an up-to-date overview of the (im)possibilities of these intriguing RNA therapies.

Until recently, drugs targeting the RNA were considered a curiosity. They were mainly used in the research of rare diseases and personalized therapies. These RNA therapies are aimed at altering the RNA, which functions as an intermediate step between DNA (genes) and proteins. If there is an error in the DNA, you can try to correct that error in the RNA. This is possible by "masking" the wrong RNA piece, for example, with a piece of RNA that has been made specifically for a certain condition. With this strategy, the mistake can be corrected or made illegible.

RNA therapies on the rise

This new research area has undergone an enormous development in recent years. More than a dozen RNA drugs have now been approved for use in patients. Many other RNA-based drugs are at various stages of development. Although rare diseases are still a major target, many of these drugs are being developed for diseases that are common and that many people suffer from, such as hyperlipidemia (too much cholesterol in the blood) or certain types of cancers.

Delivery is key

Alex Garanto, researcher at the Department of Pediatrics at Radboudumc and one of the authors of the paper: “The main hurdle for this type of medicine is delivery. The RNA must be delivered to the correct tissue or organ, because it has to do its job just there and nowhere else. In addition, you also have to consider how you can effectively get it into the cells, because not all cells actively absorb an RNA drug. This is an essential step, because the RNA can only do its job if it penetrates the right cells.”

Progress

The article in EMBO Molecular Medicine, written with colleagues from Leiden and thirteen European countries who are all studying the development of RNA therapies, focuses on this issue of the ‘delivery’. Professor of Molecular Therapy for Inherited Retinal Diseases Rob Collin, also a co-author in this publication, was recently interviewed about these developments in the NRC: “In recent years, we have made enormous progress in a better and more effective delivery of these RNA drugs to patients, whereby safety is also an important aspect in the development and evaluation of these drugs.”

European cooperation

The 22 authors of the paper are part of the European COST (Cooperation of Science and Technology) consortium. Via this European network (www.antisenserna.eu) they bundle their knowledge and expertise to develop meaningful RNA therapies for patients.

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Paper in EMBO Molecular Medicine: Delivery of oligonucleotide-based therapeutics: challenges and opportunities - Suzan M Hammond, Annemieke Aartsma-Rus, Sandra Alves, Sven E Borgos, Ronald A M Buijsen, Rob W J Collin, Giuseppina Covello, Michela A Denti, Lourdes R Desviat, Lucía Echevarría, Camilla Foged, Gisela Gaina, Alejandro Garanto, Aurelie T Goyenvalle, Magdalena Guzowska, Irina Holodnuka, David R Jones, Sabine Krause, Taavi Lehto, Marisol Montolio, Willeke Van Roon-Mom & Virginia Arechavala-Gomeza

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