Ractigen Therapeutics Doses First Patient in IIT Study of RAG-18, a Potential Game-Changing saRNA Therapeutic for Duchenne Muscular Dystrophy

NANTONG, China, Dec. 12, 2025 /PRNewswire/ -- Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, today announced the successful dosing of the first patient in its investigator-initiated trial (IIT) of RAG-18 at Peking Union Medical College Hospital (PUMCH), Chinese Academy of Medical Sciences in Beijing. This milestone marks a transformative moment in the fight against Duchenne Muscular Dystrophy (DMD), positioning RAG-18 as a potential game-changer for the field. By utilizing a novel saRNA mechanism to upregulate UTRN expression, RAG-18 represents a revolutionary therapeutic approach that aims to overcome the limitations of current treatments and could provide a universal solution benefiting all patients affected by this devastating condition.

The IIT is led by Professor Dai Yi, a distinguished expert in neuromuscular disorders and the Deputy Director of the Neurology Department at PUMCH. The study aims to assess RAG-18's safety and pharmacokinetic profile while exploring its potential to improve muscle function in DMD patients. Professor Dai stated, "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."

Dr. Long-Cheng Li, the Founder and CEO of Ractigen Therapeutics, shared his enthusiasm about the trial, stating, "Starting this study is a crucial step forward for RAG-18, emphasizing its unique role in overcoming the challenges of DMD. This treatment has the potential to address all genetic mutations related to DMD, illustrating RNA activation (RNAa) technology as a revolutionary method in clinical practice and offering hope for previously untreatable conditions."

About RAG-18
RAG-18 is a first of its kind saRNA candidate designed to specifically target and activate UTRN gene expression in muscle cells via RNAa mechanism. The utrophin protein encoded by the UTRN gene is structurally and functionally similar to dystrophin, and its upregulation could potentially serve as a functional replacement for the missing dystrophin in DMD muscle cells, providing treatment for all DMD patients regardless of the specific mutation location.

Preclinical data indicate that RAG-18, delivered utilizing Ractigen's proprietary LiCO™ (lipid-conjugated oligonucleotide) technology, has effectively upregulated UTRN expression, mitigated muscle damage and improved muscle function, demonstrating significant potential in treating DMD patients. RAG-18 received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA) in 2024, underscoring its promise as a therapeutic option for patients with DMD.

About DMD
Duchenne Muscular Dystrophy (DMD) are severe genetic disorders caused by mutations in the dystrophin gene, leading to the absence or insufficiency of functional dystrophin protein. This protein is essential for muscle fiber stability. Without it, muscle cells are easily damaged and cannot repair themselves, resulting in progressive muscle weakness and degeneration. The dystrophin gene, the largest in the human body, contains 79 exons. Current disease-modifying therapeutic approaches for DMD include antisense oligonucleotides (ASO) mediated exon skipping, gene therapy, and gene editing, with exon skipping being the most common strategy. However, these treatments have significant limitations, highlighting the critical need for innovative therapies that target the root cause of DMD to provide more effective and long-lasting benefits for patients.

About RNAa
RNA activation is a clinically validated platform technology developed by Dr. Long-Cheng Li and his team. It utilizes saRNAs to target gene regulatory domains, activating gene expression and restoring therapeutic protein levels. This innovative technology holds vast potential for developing therapeutic drugs across various diseases, particularly where traditional methods fall short.

About Ractigen Therapeutics
Ractigen Therapeutics is a clinical-stage biopharmaceutical company innovating next-generation RNA therapeutics, with a primary focus on small activating RNAs (saRNAs) developed through its clinically validated RNA activation (RNAa) technology. Leveraging proprietary delivery platforms such as SCAD™, LiCO™, and GLORY™, Ractigen is advancing a robust pipeline addressing unmet medical needs in oncology, neurological diseases, and genetic disorders. Its versatile technologies also enable the rapid development of RNA-based solutions, including siRNAs, where applicable, to target life-threatening, fast-progressing conditions such as those in the CNS. Committed to scientific excellence and patient-centered innovation, Ractigen strives to transform healthcare through the power of RNA therapeutics. For more information, visit www.ractigen.com.

SOURCE Ractigen Therapeutics