Ractigen Therapeutics, an innovator in small activating RNA therapies, announced the first patient has been successfully dosed in its investigator-initiated trial of RAG-18 at Peking Union Medical College Hospital, part of the Chinese Academy of Medical Sciences in Beijing. This milestone represents an important step in advancing potential treatments for Duchenne Muscular Dystrophy, as RAG-18 employs a novel saRNA mechanism to boost UTRN expression. By targeting this gene, RAG-18 aims to overcome the limitations of current therapies and offer a treatment approach that could benefit all patients living with this severe genetic disorder.
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The trial is being led by Professor Dai Yi, Deputy Director of the Neurology Department at Peking Union Medical College Hospital and a recognized expert in neuromuscular diseases. Professor Dai emphasized the importance of this study, noting, “We are excited to begin this clinical trial with RAG-18, a cutting-edge therapeutic approach for treating DMD. We anticipate positive clinical results that could open up new, innovative treatment options for patients and significantly improve their quality of life.” The study will primarily evaluate the safety and pharmacokinetics of RAG-18 while also exploring its effects on muscle function in DMD patients.
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Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics, highlighted the transformative potential of RAG-18, saying, “Starting this study is a crucial step forward for RAG-18, emphasizing its unique role in addressing the challenges of DMD. This treatment could target all genetic mutations associated with the disease and demonstrates RNA activation technology as a revolutionary method in clinical practice, offering hope to patients with previously untreatable conditions.”
RAG-18 is a pioneering saRNA therapy designed to activate UTRN gene expression in muscle cells, producing utrophin, a protein that mimics dystrophin function. Preclinical studies using Ractigen’s proprietary LiCO technology show that RAG-18 increases utrophin levels, reduces muscle damage, and improves muscle function. The therapy has received Orphan Drug and Rare Pediatric Disease designations from the FDA, underscoring its potential as a widely applicable treatment for DMD patients, regardless of mutation type.
Duchenne Muscular Dystrophy is caused by mutations in the dystrophin gene, which is critical for muscle fiber stability. Patients suffer progressive muscle weakness because damaged muscle cells cannot repair themselves. Current therapies, including exon skipping, gene therapy, and gene editing, provide partial solutions but are limited in scope and durability. RNA activation technology, pioneered by Dr. Li, uses saRNAs to upregulate gene expression, offering a new strategy to restore therapeutic protein levels where traditional approaches are insufficient, representing a promising advance in the treatment of DMD and other genetic conditions.
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Source- PR Newswire
