As part of Falling Walls’ Berlin Science Week, Elsevier’s 2nd Berlin Translational Dialogue (BTD) brought together world leaders in Ribonucleic Acid (RNA) research, genome-editing technologies and biotechs at the forefront of harnessing RNA-based technologies. The daylong event November 8 emphasized recent scientific discoveries with the potential for clinical translation, communicated in an easily accessible way. The format was designed to present the most relevant information from experts to help policymakers, the public and other stakeholders realize the tremendous therapeutic potential of RNA.
Amid recent landmark discoveries in RNA-research and gene-editing technologies, the event attracted journalists, leaders of public organizations, firms with vested interests, and researchers at various stages of their careers to learn about the complex world of RNA and routes to future RNA-based medicines.
Why RNA and why now?
Until recently, RNA was considered short-lived and uncontrollable for therapeutic or clinical opportunities. New technologies such as next-generation sequencing helped researchers discover many novel classes of RNA (particularly so called “non-coding” RNAs). Genome-scale analyses, such as the Human Genome Project and ENCODE, dramatically changed our view on the function of RNA: away from a simple “messenger” to a versatile regulator and effector, presumably involved in most, if not all, cellular decisions and therefore also of relevance for the majority of human diseases.
These findings certainly accelerated the interest in better basic understanding and its transformation into RNA-based therapeutics. As a result, a few visionaries have already founded RNA-medicine focused biomedical research centers, and a small number of biopharmceutical companies entered first clinical trials for RNA-based products.
“It’s an RNA world – we just live in it,” said Dr. Frank Slack, Director of the Institute for RNA-Medicine at Beth Israel Deaconess Medical Center in Boston. “RNA has been around for a long time. Only now we recognize how wonderfully diverse it is. Understanding its biology and being able to target it, makes RNA a very attractive medical tool that is indispensable for future medical therapies.”
Challenges for RNA research and RNA-based medicine
The BTD served as vibrant forum for some of these global initiatives, featuring speakers from Boston, Michigan, Lisbon, Würzburg, Berlin and the German-based Biotechs CureVac and BioNTech. (You can find the program and speaker bios here).
Despite substantial achievements, the scale of RNA-based biological regulation has not been effectively communicated to the greater scientific community or received sufficient recognition from the larger part of society. This lack of awareness hinders rapid advances of this very exciting research and its vast therapeutic potential.
“The RNA revolution is no different from any other revolution; there are many ups and downs at the beginning,” said Dr. Nils Walter, Director of the Center for RNA Biomedicine and professor at the University of Michigan.
So far, big pharmaceutical companies in Germany, more than in the US, are unwilling to invest considerably into RNA-research. This might change once the first RNA-based drug has been developed. Biotechs like CureVac and BioNTech are convinced they can bring first products soon into the clinic. Once that happened, “big pharma” might rethink and turn into a dependable driver to successful technologies.
“It is remarkable that most of our funding comes from US investors, despite two of the three globally leading RNA Biotechs operating very successfully in Germany,” said Dr. Mariola Fotin-Mleczek, Chief Scientific Officer at CureVac, based in Tübingen.
So who can step up to fill that gap? Bio-tech companies such as CureVac or BioNTech combine academic research and product development while co-operating in a transparent and open manner.
“We should remind our students and funding bodies that most landmark discoveries in the biomedical sciences originate from serendipitous observations of completely unbiased basic research,” said Prof. Emmanuelle Charpentier, Director at the Max Planck Institute for Infection Biology in Berlin.
Dr. Charpentier's statement was strongly resonated by Dr. Jörg Vogel, Professor at the University of Würzburg and future founding director of the new Helmholtz Institute for RNA-based Infection Research (HIRI). He stressed the relevance of regulatory RNAs during infections, a stream of research rather neglected so far. He is certain that new disease-relevant target structures will be identified in focused institutions such as HIRI, generously supported by investments and vision of the Helmholtz Association.
Dr. Walter presented a similar RNA-centric view that involves harnessing high-resolution imaging technologies. He proposed that three main factors could make RNA research more successful in the future: Vision, Proximity and Resources (VPR).
There was general agreement that interdisciplinary RNA-focused infrastructures supported by new funding schemes from governments, private-public partnerships or philanthropic sources, would attract many skilled scientists and accelerate RNA-discoveries and RNA-based treatments. Establishing RNA-flagship institutions around the globe and fostering their tangible collaboration with innovative instruments might trigger new biotechs to translate the “RNA revolution” into novel diagnostics and therapies, benefiting society.
Driving research in the context of Berlin Science Week
The Berlin Translational Dialogue is structured to involve and engage relevant scientific actors and societal stakeholders. The format boosts visibility among policy makers and politicians as the presentations and discussions are targeted towards a broader audience. Integrated into the Berlin Science Week and closely affiliated with the Falling Walls conference, the BTD offers a general perspective, draws experts from around the world and opens multiple opportunities to interact with the most relevant strategists, politicians and decision makers.
The scientific co-organizers, Markus Landthaler of the Berlin Institute for Medical Systems Biology at the MDC-Berlin and Dr. Gunter Meister of the University of Regensburg, and participants emphasized the unique profile of the Berlin Translational Dialogue and expressed great interest in continuing this collaboration in one form or another. Berlin, as an emerging center of excellence in biomedical research, digitization, health, politics and entrepreneurship, is an ideal focal point for Elsevier to foster collaborations and exchange, leading the way with the most visionary partners. At Elsevier, we develop digital solutions to support the research of millions of scientists; we’re looking forward to continuing the Berlin Translational Dialogue as a way to advance science and technology for the benefit of society.