Arthex Biotech is a biotechnology spin-off from the University of Valencia, a leader in the research and development of therapies based on microRNA-modulating oligonucleotides through its patented ENTRYᵀᴹ platform. Arthex’s drugs are intended for the treatment of diseases with no therapeutic option or considered difficult to treat due to their complex pathogenesis. ATX-01 is Arthex’s most advanced treatment, a drug with a unique, dual mechanism of action that reverses the symptoms of myotonic dystrophy type 1 (DM1). Myotonic dystrophy is a rare, highly disabling disease that affects muscles and other tissues and reduces the life expectancy of patients, which does not exceed 55 years. It is the most common muscular atrophy in adults, with more than one million people affected, and currently untreated. ATX-01 has shown unprecedented therapeutic efficacy in models of the disease and zero toxicity. Furthermore, the development program has been validated by the FDA, which has designated ATX-01 as an orphan drug to incentivize its development. Overall, ATX-01 will enter the clinical phase in 2023. Arthex’s business model is based on the development of these microRNA modulators involved in the pathogenesis of diseases and their subsequent licensing to a pharmaceutical company. The preclinical and clinical results of ATX-01 will enable the signing of a licensing agreement, and with the revenue generated, Arthex will continue to expand its pipeline, which already includes three other potential drugs in the discovery phase for the treatment of cachexia, osteoarthritis, and Fuchs’ endothelial dystrophy. The cost of this first transaction is estimated at €500 million plus royalties. Finally, it is worth noting that Arthex has secured €8.5 million in public and private financing (three major VCs) and has a signed term sheet with a new investor who will lead a new round.