XtRNA Bio, Nijmegen, Netherlands
Develop a viral gene therapy for CMT2D
XtRNA Bio is looking to develop a new viral gene therapy for Charcot-Marie-Tooth disease type 2D.
CMT2D is caused by mutations in the GARS1 gene, which encodes glycyl-tRNA synthetase — an enzyme that plays a crucial role in protein production by attaching amino acids to their corresponding carrier molecules, called tRNAs. In CMT2D, the faulty version of the GARS1 protein holds onto tRNA too tightly, preventing it from functioning properly. This disrupts protein production and stresses the cells, which has been shown to contribute to nerve degeneration and peripheral neuropathy.
Research out of Dr. Erik Storkebaum’s lab demonstrated that increasing tRNA levels corrected disease symptoms in two different animal models of CMT2D.
XtRNA Bio will aim to identify the optimized components of the tRNA elevation gene therapy and then test the optimized therapy for its ability to correct disease phenotypes in CMT2D animal models.
The support from CMTRF allows us to rapidly advance a new genetic approach for treating CMT2D. We’re working to turn a clear disease mechanism into a first-in-class therapy that addresses the root cause.