Main Conference – Day 2Thursday, 19 March 2026 - CET (Central European Time, GMT+01:00)

• circVec technology utilizes the process of back-splicing in order to substitute mRNA by engineered, transcribable circular RNA with high intracellular stability leading to enhanced and sustained protein expression
• incorporation of circVec cassettes into AAV or DNA vectors is used to develop more effective and safer gene therapies for both genetic and acquired diseases affecting various organs and tissues in which circular RNA outperforms mRNA
• circular RNA produced from circVec vectors can be loaded intracellularly into and delivered through virus like particles as an alternative to delivery of synthetic circular RNA through lipid nano-particles
• circVec technology has broad potential applications in different therapeutic areas

Cardiovascular disease is the leading cause of death worldwide; heart failure carries high mortality, and substantial health-economic burden. Cardiac infarcts can lead to loss of heart function and heart failure. Cardiomyocytes have limited regenerative capacity and current therapies only improve residual function and are not curative.

• Small non-coding microRNAs delivered as synthetic mimics in cardiomyocyte specific lipid nanoparticles can reactivate cardiomyocyte proliferation in the adult heart. Preclinical studies in mice and pigs demonstrate increased cardiomyocyte division, reduced infarct size, and improved function after myocardial infarction.
• The miRNA–LNP approach provides potent, transient, and repeatable effects with efficient myocardial delivery; Compared with AAV, protein and cell therapies, miRNA–LNPs offer better control of dose and optimal duration
• miRNA-based regenerative therapy represents a promising, scalable strategy for curative treatment against heart failure, warranting further clinical development and safety evaluation.
  
  

• DM1 is a rare neuromuscular disease with no disease modifying treatments. Arthex’s therapeutic approach in DM1 is miR-23b inhibition, which demonstrated a dual beneficial effect on DM1 models.
• In order to alleviate functional and molecular symptoms of DM1 disease, preferentially in the tissues affected by the disease, Arthex is developing a safe lipid-conjugated antimiR-23b oligonucleotide (ATX-01).
• Explore how the conjugated antimiR reaches muscle, heart, brain and diaphragm more efficiently than the naked molecule in the HSALR mice and shows improved PK/PD properties. Importantly, the level of compound delivered into brain by IV injection in the DMSXL animals was enough to produce important target engagement and concomitant rescue of exploratory behaviour of the mice.