The Phase I study, led by Professor Yi Dai of Peking Union Medical College Hospital, tracked three boys receiving monthly intravenous infusions of RAG-18. Results showed a 3.5- to 5.3-fold increase in sarcolemmal utrophin expression, a protein capable of substituting for the missing dystrophin in DMD patients. Because this mechanism acts independently of specific genetic mutations, it offers a potential therapeutic path for the entire patient population, bypassing the need for viral vectors or permanent DNA editing.
Ractigen Reports First Clinical Proof of RNA Activation in DMD Patients
Ractigen Therapeutics has unveiled the first clinical evidence that small activating RNA can safely upregulate endogenous protein production in humans. Presented at the World Muscle Society congress in Hiroshima, the data from the RAG-18 trial demonstrate successful target engagement in the skeletal muscle of boys with Duchenne muscular dystrophy.
Clinical observations through Day 169 revealed structural improvements, including increased myofiber diameter and a reduction in muscle fat fraction. Quantitative MRI scans indicated a decrease in active tissue edema, while pulmonary function tests showed positive numerical trends. The trial reported a favorable safety profile, with no serious adverse events or dose-limiting toxicities recorded among participants. With the 30 mg Cohort 2 now fully enrolled, Ractigen continues to evaluate the long-term impact of this mutation-agnostic approach on disease progression.
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