MicroRNAs (miRNAs) are a class of recently identified non-coding RNA molecules that play an essential role in gene expression regulation at post-transcriptional levels by binding to target mRNAs and blocking the protein synthesis. The most common way to inhibit microRNAs is the administration of synthetic single-stranded RNA molecules (called antimiRs) with a sequence complementary to that of the miRNA to be inhibited. These antimiRs bind to the target miRNA by blocking or degrading it. To improve the binding affinity, stability and biodistribution of the antimRs, much progress has been made in the generation of new chemical modifications in the nucleotides that make them up.
The first product of the company, ARTHEx-01, is a new drug (RNA-modified single stranded molecule), which is an antimiR therapy addressed to the treatment of Myotonic Dystrophy type 1 (DM1). For manufacturing and quality purposes and considering the manufacturing procedure followed to synthesize the miRNA inhibitors, the product is considered a small molecule and not an advanced therapy medicinal product.