CORDIS Project
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ATX-01 is a pioneering miRNA therapy aimed at treating myotonic dystrophy type 1 by targeting specific RNA molecules to correct mis-splicing. This innovative approach has shown promising results in preclinical studies and is set for clinical trials to validate its efficacy.
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…
ARTHEX BIOTECH SL
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