Ractigen Reports First Human Proof-of-Mechanism for RNA Activation in Duchenne Muscular Dystrophy

Ractigen announced the first human proof-of-mechanism for RNA activation in Duchenne Muscular Dystrophy (DMD), with Phase I data showing significant increases in sarcolemmal utrophin expression. This marks the first clinical evidence that a systemically delivered small activating RNA (saRNA) can safely upregulate an endogenous target protein in human skeletal muscle for a monogenic disease.