SciLifeLab leads new national investment in oligonucleotides

Kristian Sandberg, SciLifeLab Drug Discovery and Development Platform.

Gene-based drugs is a rapidly evolving field that enables more and more medical treatments. Among the tools that raise the greatest hopes are oligonucleotide therapeutics, molecules with the potential to affect specific gene fragments without causing lasting changes in the genome. Oligonucleotides are already being used successfully against, among other things, the muscle disease Spinal muscular atrophy type 1. Now Sweden is taking the next step with OligoNova – a new initiative where academia and industry will guide innovative projects on to road towards clinical studies.

“Oligonucleotides are expected to be a key ingredient in future therapies. The fact that our national research funders are already investing in a Swedish infrastructure is exactly what we need to position our country on the very front line, and the decision to integrate OligoNova into SciLifeLab right from the start provides further strength to anchor the platform as a national resource. Now the process is underway with the aim to enter full operations as early as 2022,” says Kristian Sandberg, head of SciLifeLab's Drug Discovery and Development Platform.

OligoNova draws inspiration from Boston's RNA Therapeutics Institute, an already established structure that brings together a range of the region's leading organisations. In Sweden, the University of Gothenburg is the academic host. AstraZeneca provides expertise during the construction phase, and a network of selected scientific environments have already united in, among other things, joint applications. An experienced force in this collaboration is Per Artursson, Professor of Dosage Form Design at Uppsala University:

“This is an exciting area that in all probability contains enormous opportunities. Currently, a multitude of oligonucleotide therapeutics are already in clinical trials and the number is increasing rapidly. Still, any potential success depends on our ability to direct the substances to the intended place in the body and to activate the uptake into the cell. These are challenges that our group has been researching for over 25 years, and today we possess the knowledge and skills required to find answers to several of the key questions surrounding oligonucleotides.”

Per Artursson was, more than 20 years ago, involved in establishing DNA/RNA-based drugs and gene therapy as a discipline within Swedish research. Today, his scientific output includes more than 200 publications and 30,000 citations. His current research is carried out with the aim to increase the proportion of oligonucleotide that is transported to its subcellular target in the cytoplasm, experiences that Kristian Sandberg attributes important values ​​in the infrastructure's future work:

“We are aware of the enormous competition that prevails in this field, yet our ambition is to develop OligoNova into an internationally attractive partner that identifies and supports the country's most promising projects. One of our main strengths is that we can provide absolute cutting-edge expertise such as Lab Artursson in supply and delivery-mechanisms. Once on the track, I am convinced that OligoNova will be of great importance for both Sweden and future drug development.”