Spanish biotech company ARTHEx has published what it says is the first demonstration that a targeted RNA therapy can improve behavioural deficits in a preclinical model of myotonic dystrophy type 1, a rare and severely disabling genetic disease that also affects the brain.
The results show that the company's BOOST-ON platform, which pairs selective oligonucleotides with enhanced tissue delivery, successfully reached the brain, corrected the underlying disease biology and produced measurable behavioural improvements. Myotonic dystrophy is the most common form of muscular dystrophy in adults and causes progressive muscle wasting, cardiac problems and cognitive impairment.
ARTHEx's lead programme, ATX-01, is already in a Phase I/IIa clinical trial called ArthemiR for the neuromuscular aspects of the disease. The company said the new preclinical data highlights the platform's potential to address central nervous system symptoms, which include intellectual disability and behavioural abnormalities.
The company, a spin-off from the University of Valencia, was previously granted FDA Fast Track Designation for ATX-01 in the treatment of myotonic dystrophy type 1.