Myotonic dystrophy type 1 (DM1) is a clinically and genetically heterogeneous disorder with more than 1 million diagnosed patients worldwide, making it the most common adult muscular dystrophy. Available treatments only relief symptoms. Given this need, we offer ATX-01, a first in class miRNA therapy that inhibits miR-23b without changing the DNA, which rescues DM1 pathogenic mis-splicing. We have demonstrated improved target engagement effects in skeletal and respiratory muscle in in-vitro and in-vivo studies, granting us an FDA pre-IND approval and orphan drug designation. Our approach, antimiRs conjugated to fatty acids, constitutes the ENTRY platform, which guarantees efficient drug delivery to target muscular and extra-muscular tissue at low doses. This patented technology will be validated through a Phase I/IIa clinical trial and chronic preclinical studies, to then be extended to applications as other myotonic dystrophies, Fuchs dystrophy, cachexia, among other opportunities.
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