An innovative hub for innovative medicines

What is the goal of a new medicine? To treat or even cure a disease, it has to interfere with the processes inside our bodies that cause that disease. Traditionally, we have been mostly confined to designing medicines that either treat symptoms or manipulate the end stages of these processes. But what if we could create new drugs that work earlier in these processes to cut off the disease-causing information flow?

In order to treat the cause of a disease, one first needs a molecular understanding of the disease biology and second the tools to interfere directly with the mRNA of target genes. This ambitious aim is the mission of the new RNAHub at Roche Pharma Research and Early Development (pRED) in Basel.

Many of the processes underlying disease are driven by molecules called proteins, which have traditionally been the target of pharmaceuticals made up of small molecules or antibodies. However, proteins ultimately come from a recipe book - our genome, made of DNA. Recipes are copied out of the book as RNA, serving as the template to make the proteins. A variety of new medicines made out of the same building blocks as RNA and DNA, called nucleic acid therapeutics, could target the recipes themselves, rather than the final protein product, thus intervening earlier in the information flow causing disease.

Given the recent progress in the understanding of nucleic acid therapies and what the remaining challenges are, Roche is committed to fully leverage the potential of this class of molecules. Unlocking this exciting avenue of medical possibility requires bringing together experts across many fields to solve challenges and deliver the rapid advances necessary to make nucleic acid therapeutics a reality for patients. The RNAHub is doing just that.

A unique multidisciplinary approach with access to innovative research capabilities

The RNA discovery hub is a fully integrated multidisciplinary research community with direct access to all of Roche’s current and future research capabilities, including theThe RNAHub is unique in bringing together scientists with deep expertise in RNA biology, medicinal chemistry, molecular design, drug safety, bioinformatics and data analytics, as well as several specific therapeutic areas. The scientists are taking an integrated and data-centric drug discovery approach to improve the safety and predictive validity of RNA therapeutics and technologies. The goal is to discover new drugs, biomarkers and targets that support Roche’s mission to create transformative medicines.

“Co-locating our research in oligonucleotides with other nucleic acid therapeutic opportunities and our leading expertise in small molecule splicing modifiers enables us to address targets and disease biology from different angles. This will help to accelerate our learnings and identify the most successful therapeutic path”, Hendrik adds.

Short sequences with big potential

Oligonucleotides - that is potential medicines made out of the building blocks of RNA or DNA — can be highly specific, fast to design and manufacture, and “programmable” to modulate a desired target. They could be used to “drug the undruggable” and give patients access to important transformative therapies while we are learning at the same time to generate additional therapeutic options.

In addition to directly meeting patients’ needs with new oligonucleotide therapeutics, these potential medicines have other uses across Roche, too. For example, we can also use these molecules to more quickly gain a deeper understanding of a target’s biology, or even understand how and why other non-oligonucleotide medicines work. The RNAHub will coordinate these different strategies, and also focus on tackling the challenges to their adoption. RNA Strategy Lead, Morten Lindow, highlights one of these challenges: “Expanding their high predictivity, when it comes to pharmacology to all drug properties including safety, will be key to increasing both their therapeutic and innovation-enhancing potential.”

How does the RNAHub work?

The RNAHub takes an integrated and data-centric drug discovery approach to improve the predictive validity of RNA therapeutics and technologies to create potentially transformative medicines for patients.

“This approach is especially important for technologies and endeavours where there is a big component of complexity, things are not completely plannable, there are many unknown unknowns and we need fast iteration,” adds Morten. “We can achieve this with the closeness and co-ownership between different areas of expertise.”

“Luckily, the ‘programmable’ nature of RNA is very amenable to data analysis”, explains Morten. As a result, the RNAHub exploits a vast data space by iteratively investing in increasing data-density, to uncover design rules and make drug properties such as distribution and delayed chronic toxicity predictable, not only in each project individually, but across the whole technology platform. “The challenges here are holistic and we can’t solve them project by project,” he adds.

After using this combination of big data and AI to design new RNA molecules, the RNAHub will carry out deeper characterisation and profiling, and closely collaborate with theto use our unique strength in human cellular model systems and organoids.

Finally, the RNAHub will combine data and experiments to build predictive models, where the experimental design and data collection fits the model and the other way around, and enables the broad integration of early and late stage data to leverage the combined strength of forward and reverse translation.

A collaborative culture

The RNAHub co-locates our RNA drug discovery in one physical location for coming up with and testing creative ideas. It’s a place to foster a culture of scientific creativity and rigour in execution and decision-making, and to deliver oligonucleotides as a nimble tool to manipulate gene expression for discovery and as a default frontrunner therapeutic approach.

“It’s a place with a heartbeat - a destination for innovation to solve the grand challenges in this area and lay out a path for an integrated drug discovery and development approach,” reports Eunice. “It’s the perfect playground for the scientifically curious and creative, combining the intimacy of a smaller organisation with the advantages of support from Roche.”

“The creativity is very stimulating,” adds Stormy. “The conversations at the coffee machine spark new ideas and ways to solve problems - which are even applicable to other projects and areas.”

An exciting opportunity to serve patients

The RNAHub is continuously looking for great collaborators to join its patient-focused and data-driven team. Highly collaborative, code-literate or code-curious, scientists across any discipline of drug development are welcome to apply.

For data scientists, the RNAHub is a thrilling opportunity to contribute directly to the drug development process while taking on exciting computational challenges, such as combining multi-modal data from characterisations of multiple biological systems. “It’s a very rich environment to apply many methods, and will be a fascinating field in the years to come,” shares Daniel Butnaru, Senior Principal Scientist in Data & Analytics, pRED.

With this foundation in oligonucleotides, the RNAHub will become a true centre of excellence for RNA biology, eventually for all modalities targeting RNA. The goal is to discover new drugs, biomarkers and targets that support Roche’s mission to create transformative medicines.

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