The research, centered on the company’s proprietary BOOST-ON platform, utilizes lipid-conjugated antimiR-23b to address the underlying genetic drivers of the disease. In the study, the therapy increased MBNL1/2 protein levels and reduced toxic DMPK transcripts within the brain, resulting in the normalization of exploratory activity in animal models. These findings offer a potential pathway for treating the cognitive and behavioral impairments that often accompany the muscular symptoms of the disorder.
In section Releases
ARTHEx Biotech Reports Brain Function Recovery in DM1 Model
A preclinical study published in Cell Reports Medicine confirms that the investigational therapy ATX-01 can cross the blood-brain barrier, correcting molecular defects and improving behavioral patterns in subjects with myotonic dystrophy type 1. This marks the first evidence linking systemic RNA-based treatment to neurological restoration in DM1.
Beatriz Llamusí, co-founder and chief scientific officer of ARTHEx, noted that the results establish a clear connection between molecular correction and functional recovery. The data suggest that the BOOST-ON platform could provide a comprehensive approach to a condition previously considered difficult to treat due to the inability of most therapeutics to penetrate the central nervous system. As the company continues its Phase I/IIa ArthemiR trial, these results broaden the scope of potential applications for ATX-01, moving beyond strictly muscular manifestations toward a systemic treatment for the full spectrum of DM1.
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