Ractigen Therapeutics Reports Positive Data for RAG-18 in Duchenne Muscular Dystrophy
Ractigen Therapeutics has presented positive first-in-human data for its RAG-18 drug candidate in Duchenne muscular dystrophy. The drug demonstrated increased utrophin expression and improved muscle pathology.
Ractigen Therapeutics has presented initial human clinical data for its drug candidate RAG-18, targeting Duchenne muscular dystrophy (DMD). The findings, shared in March 2026 at the World Muscle Society (WMS 2026) congress, indicate the drug can safely increase endogenous utrophin protein expression in human skeletal muscle.
The study provides the first clinical proof-of-mechanism for RNA activation (RNAa) therapy in a monogenic disease. RAG-18 is delivered systemically as a small activating RNA (saRNA). Analysis of muscle biopsies revealed a 3.5- to 5.3-fold increase in sarcolemmal utrophin, alongside improvements in muscle histopathological remodeling.
Patients in the trial also exhibited favorable trends in motor and pulmonary function endpoints. The drug maintained a positive safety and tolerability profile, with no dose-limiting toxicities (DLTs) or serious adverse events (SAEs) reported. Patient enrollment for the second dose cohort is now complete.
RAG-18 utilizes Ractigen's RNAa platform and LiCO™ lipid-conjugate technology for monthly intravenous infusions. This approach activates the cell's endogenous transcription machinery to boost utrophin production without viral vectors or permanent DNA editing. RAG-18 has received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA).