Investigational Compounds

The first investigational product of the company is ATX-01, an antimiR in preclinical development for use in Myotonic Dystrophy Type 1

What are antimiRs?

MicroRNAs (miRNAs) are a class of  non-coding RNA molecules that play an essential role in the regulation of gene expression at post-transcriptional levels by binding to target mRNAs and inhibiting protein synthesis. The most common way to inhibit microRNAs is the administration of synthetic single-stranded oligonucleotides (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 bio-distribution of the antimRs, much progress has been made in the generation of new chemical modifications in the nucleotides that make them up.

DM1, a genetic disease with unmet medical needs

DM1 is caused by an abnormal expansion of a trinucleotide repeat in a non-coding region of the DMPK gene. After transcription, mutated DMPK mRNA folds into a hairpin that sequesters MBNL1 proteins, causing a lack of function of these proteins.

In the DM1 mouse model, HSALR, ATX-01 increased MBNL protein levels, reduced abnormal splicing events and improved the clinical phenotype.