Erik Storkebaum
Erik Storkebaum

New spin-off company to develop viral gene transfer of transfer RNA as a therapeutic approach

Following research conducted by Erik Storkebaum and his lab, a new spin-off company named XTRNA Bio B.V. was recently established. The foundation of this new venture is a viral gene transfer of transfer RNA (tRNA) discovered by Storkebaum and his lab, which can be utilised in the treatment of neurodegenerative diseases.

Storkebaum's novel tRNA technology originated from research on Charcot-Marie-Tooth disease (CMT), a hereditary form of neuropathy. CMT is a hereditary neuromuscular disease characterised by impaired nerve signals for movement and sensation. Symptoms of the disease often develop in adolescence, and to date, there is no effective treatment to slow its progression. However, there is hope for change: Storkebaum has developed a viral gene transfer that could serve as a potential medication.

Mutant proteins

Since his post-doctoral research in 2006, Erik Storkebaum has been studying CMT, specifically caused by mutations in tRNA synthetases. These enzymes connect tRNAs with their corresponding amino acids, marking the initial step of protein synthesis in cells. Through motor and sensory in vivo research, Storkebaum discovered that disease-causing mutations in these enzymes result in a significant reduction in protein synthesis. Why does this happen? In his latest research, Storkebaum provides a piece of the puzzle: "The mutant enzymes bind to the tRNA but fail to release it. tRNA is essentially a translation molecule used to translate mRNA into proteins. When tRNA remains attached to the mutant tRNA synthetases, this results in a shortage of this tRNA in the cell. This shortage then leads to defective protein synthesis."


The technology developed by Storkebaum and his group involves the addition of extra tRNA molecules into the cell via viral gene transfer. In newborn mouse models with the genetic defect, such a gene transfer prevents disease symptoms. This breakthrough is significant from both a scientific and molecular perspective: the genetic defect is directly addressed at a molecular level. The next question is how effective the gene transfer can be in mouse models that already exhibit disease symptoms, where degeneration has already begun.

Spin-off company

The aim of the spin-off company is to use the tRNA technology as a platform for multiple applications. While Storkebaum's research currently focuses on one variant of CMT, the technology could also assist with other variants or other neurodegenerative diseases involving tRNA-related issues.
The spin-off company is established in collaboration with Radboud University and NLC, an organisation that sets up and builds spin-off companies in partnership with an academic partner, investing in them as well. Currently, there is a 'Research and License option Agreement' in place with NLC. Initially, a proof-of-concept study will be conducted to validate the functionality of the technology platform.


Estimating the timeline for the actual launch of a drug is challenging. Storkebaum states, "The success of the medication depends on many factors: the effectiveness of the gene transfer in a symptomatic disease stage, the efficiency and safety of the gene transfer in larger animals, whether we can proceed to clinical studies, and if and how quickly we can observe a therapeutic effect. CMT is a slowly progressing disease, so demonstrating a therapeutic effect will likely be a matter of years rather than months."