ARTHEx Biotech is a clinical-stage company specializing in targeted RNA medicines that precisely modulate gene expression across skeletal muscle, heart and brain tissues. Its lead program, ATX-01, is an oleic-acid-conjugated anti-miR oligonucleotide designed to inhibit microRNA-23b and restore MBNL protein function in myotonic dystrophy type 1 (DM1); the drug is currently being evaluated in the randomized, placebo-controlled Phase I/IIa ArthemiR™ study. ATX-01 has secured Orphan Drug Designation in both the United States and Europe, as well as Rare Pediatric Disease Designation from the FDA. Preclinical data show the therapy increases MBNL production, reduces toxic DMPK mRNA and corrects spliceopathy in animal and cell models. Beyond DM1, ARTHEx is using its delivery platform to build a pipeline for additional muscular, CNS, cardiac and pulmonary diseases with high unmet need. The company’s headquarters are in Valencia, Spain, and it recently upsized its Series B financing to support preparation for a registrational study of ATX-01 and to broaden its pipeline. Including this round, the company has attracted backing from a consortium of European and U.S. life-science investors.

Total raised

$92M

Funding rounds

2

Latest round

Series B

Latest activity

Sep 2025