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

🌐 Laotian Times (Laos) —

AI Summary

Ractigen Therapeutics reported positive first-in-human results for RAG-18, an RNA-activation treatment being studied for Duchenne muscular dystrophy. Muscle biopsies showed increased utrophin expression, with encouraging trends also reported in motor and pulmonary measures.

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, accompanied by definitive tissue histopathological remodeling Concordant positive trajectories observed across motor and pulmonary functional endpoints […]

Health AI & Tech Ractigen Therapeutics RAG-18 Duchenne muscular dystrophy RNA activation clinical trial utrophin first-in-human

Read original source →