Ractigen Therapeutics to Present First-in-Human RAG-18 Data in Duchenne at WMS 2026

Key Takeaways

• Ractigen Therapeutics will present first-in-human findings for RAG-18, an investigational small activating RNA (saRNA) for Duchenne muscular dystrophy (DMD), as a late-breaking oral presentation at the World Muscle Society (WMS) Congress, September 29 to October 3 in Hiroshima, Japan.
• The data come from Cohort 1 of an ongoing open-label, dose-escalation trial in ambulatory boys with genetically confirmed DMD. The company says it is the first clinical evidence that an saRNA can upregulate an endogenous target protein in patients with a monogenic disease.
• RAG-18 is designed to increase utrophin, a protein related to dystrophin, an approach that could apply to patients regardless of their specific DMD mutation. Detailed results will be released only at the presentation, under congress embargo rules.

Ractigen Therapeutics, a clinical-stage biotechnology company focused on RNA activation (RNAa) therapeutics, announced that first-in-human findings from its RAG-18 program have been accepted as a late-breaking presentation at the 31st Annual Congress of the World Muscle Society. The company describes the late-breaking designation as reserved for findings the program committee considers of immediate importance to the field.

RAG-18 is an investigational saRNA administered systemically. It targets regulatory regions of the human UTRN gene to induce transcriptional activation and increase production of utrophin, a structural and functional homolog of dystrophin. DMD is a rare, severe, X-linked neuromuscular disorder caused by mutations in the DMD gene that lead to a deficiency or absence of dystrophin. Because utrophin can substitute for dystrophin at the muscle membrane, raising its levels is considered a mutation-independent strategy with theoretical relevance across all DMD genotypes.

The presentation will report available Cohort 1 findings from the ongoing open-label, dose-escalation trial (NCT07282652) in ambulatory boys with genetically confirmed DMD. Assessments include safety and tolerability, paired pre- and post-treatment muscle biopsies with quantitative immunofluorescence of sarcolemmal utrophin, muscle histopathology, serum creatine kinase, quantitative muscle MRI, spirometry, and motor function measures. The company has not yet released results and says detailed data will be unveiled at the presentation, in line with WMS embargo regulations.

The oral presentation, titled "First-in-human evidence of RNA activation-mediated sarcolemmal utrophin upregulation in Duchenne muscular dystrophy" (Abstract No. 02LBO), will be delivered by principal investigator Professor Yi Dai, MD, PhD, of the Department of Neurology at Peking Union Medical College Hospital. It is scheduled for Saturday, October 3, from 12:00 to 12:15 PM JST in Phoenix Hall at the International Congress Center Hiroshima. RAG-18 is investigational and has not been approved by any regulatory authority.

Ractigen Therapeutics, based in Nantong, China, develops next-generation RNA therapeutics, with a primary focus on saRNAs built on its RNAa platform. Its pipeline targets oncology, neurological diseases, and genetic disorders.