Ractigen Therapeutics Presents Positive First-in-Human Data for RAG-18 at WMS 2026, Establishing Clinical Proof-of-Mechanism for RNA Activation in Duchenne Muscular Dystrophy

Initial clinical evidence showed that systemically delivered small activating RNA (saRNA) safely upregulated an endogenous target protein in human skeletal muscle in a monogenic disease. Paired muscle biopsies demonstrated 3.5- to 5.3-fold increases in sarcolemmal utrophin expression, supporting biological activity for the investigational therapy. The findings are encouraging but remain early-stage clinical data, with no efficacy outcomes or broader safety details provided.