The upcoming presentation, led by Professor Yi Dai of Peking Union Medical College Hospital, focuses on the company’s mutation-independent strategy to treat the disorder. While dystrophin deficiency causes Duchenne, researchers have long sought to boost utrophin, a structural homolog that can substitute for dystrophin at the muscle membrane. Historically, turning on this gene on demand within human muscle tissue remained an elusive biological hurdle.
In section Releases
Ractigen to Unveil First Human Data on RNA Activation in DMD Patients
Clinical-stage firm Ractigen Therapeutics will present inaugural human trial results for its RAG-18 program at the World Muscle Society Congress in Hiroshima. The data, selected as a late-breaking oral presentation, marks the first time small activating RNA has successfully upregulated an endogenous target protein in patients with Duchenne muscular dystrophy.
Professor Dai will report findings from the initial cohort of an ongoing dose-escalation trial, covering safety, muscle biopsies, and functional markers. By leveraging small activating RNA (saRNA) to induce transcriptional activation, Ractigen aims to demonstrate that its RAG-18 agent can restore cellular integrity across all patient genotypes. The full dataset, including high-resolution imaging and clinical outcomes, is scheduled for disclosure on October 3, 2026.
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