Sep 15, 2026 —
Ractigen Therapeutics will present first-in-human data for RAG-18, an RNA activation (RNAa) therapy for Duchenne muscular dystrophy (DMD), as a late-breaking oral presentation at the World Muscle Society Congress at the end of September.
RAG-18 uses a small activating RNA (saRNA) to increase production of utrophin, a natural structural relative of dystrophin. Because utrophin can substitute for the missing dystrophin regardless of a patient’s specific mutation, the approach is mutation-independent. Ractigen says the results are expected to provide the first clinical evidence that a small activating RNA can raise an endogenous target protein in patients with a monogenic disease.
The late-breaking slot is reserved for findings the program committee considers of immediate importance to the field.
