Viral gene transfer
CMT is the most common hereditary neuromuscular disorder. In this disease, which often manifests during the teenage years, nerves do not properly transmit movement and sensory signals. Until now, treatment has only been available for the symptoms, not for the disease itself.
In his previous ERC project, Storkebaum unraveled the molecular mechanism underlying a form of CMT caused by mutations in glycyl-tRNA synthetase. "In patients with this form of CMT, we see that mutant enzymes sequester the tRNA and no longer release it. This hampers normal protein synthesis in the cell," Storkebaum explains. "Our solution is to add extra tRNA molecules to the cell via viral gene transfer. In newborn mouse models with this disorder, the gene transfer prevents disease symptoms: the mouse does not become ill."
Further development
With the ERC PoC, Storkebaum and his team will investigate how effective the gene transfer is in adult mouse models that already show disease symptoms - a crucial step towards possible application in patients. For this, the researchers will first work on optimizing the gene transfer to reach adult motor and sensory nerves as effectively as possible, testing various doses and administration routes.
Business plan
Additionally, the project will focus on developing a business roadmap for XtRNA Bio, the spin-off company Storkebaum founded with NLC Health Ventures. Upon successful proof-of-concept experiments, XtRNA Bio will seek funding for further preclinical and clinical development of the viral gene transfer AAV-tRNA-Gly.
ERC Proof of Concept Funding
The ERC Proof of Concept funding is made available only to those who already have an ERC award to establish proof of concept of an idea that was generated in the course of their ERC-funded projects.
Proof of Concept Grants aim at maximizing the value of the excellent research that the ERC funds, by funding further work (i.e. activities which were not scheduled to be funded by the original ERC frontier research grant) to verify the innovation potential of ideas arising from ERC funded projects.