Ractigen Therapeutics, a China-based, clinical-stage biotechnology company, announced on Saturday the presentation of positive first-in-human clinical data from its ongoing Phase I trial of RAG-18 during a Late-Breaking Oral Presentation at the 31st Annual Congress of the World Muscle Society (WMS 2026), held in Hiroshima, Japan.

According to the company, the data establish the first clinical proof-of-mechanism for RNA activation (RNAa) in a human monogenic disease, demonstrating that a systemically delivered small activating RNA (saRNA) can safely enter human skeletal muscle and upregulate an endogenous, therapeutically relevant target protein.

The presentation, titled 'First-in-human evidence of RNA activation-mediated sarcolemmal utrophin upregulation in Duchenne muscular dystrophy,' was delivered orally by the study's Principal Investigator, Professor Yi Dai, MD, PhD, Department of Neurology, Peking Union Medical College Hospital (PUMCH), Chinese Academy of Medical Sciences.

RAG-18 is developed on Ractigen's proprietary RNAa platform, and delivered using the company's Lipid-Conjugated Oligonucleotide (LiCO) technology as an aqueous saline formulation via monthly intravenous (IV) infusion. Reactigen says that it harnesses the cell's endogenous transcriptional machinery to increase 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 US Food and Drug Administration (FDA).