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
First clinical evidence demonstrating that a systemically delivered small activating RNA (saRNA) can safely upregulate an endogenous target protein in human skeletal muscle in a monogenic disease. Paired muscle biopsies reveal 3.5- to 5.3-fold increases in sarcolemmal utrophin expression,...