Its lead candidate is ATX-01, an antimiR targeting microRNA-23b (miR-23b), designed to correct underlying molecular causes in patients with Myotonic Dystrophy Type 1 (DM1), a progressive muscle-wasting disease. The approach aims to restore expression of the MBNL protein and reduce toxicity associated with mutant DMPK transcripts. 

The company raised €42 million (~US $45 million) in a Series B financing round in May 2023. Investors included Columbus Venture Partners (lead), the European Innovation Council, Hadean Ventures, Sound Bioventures, Invivo Capital, AdBio Partners, and CDTI via its Innvierte initiative. The funds are being used to advance ATX-01 into a Phase I/IIa trial (named ArthemiR) for DM1, scheduled to begin in the second half of 2023. 

Dr. Frédéric Legros serves as CEO (having been previously Chairman), and Dr. Beatriz Llamusí is co-founder and CSO.