#China #Beijing #Duchenne Muscular Dystrophy #Ractigen #RAG-18

Ractigen Therapeutics Celebrates First Patient Dosing in RAG-18 Study for Duchenne Muscular Dystrophy

Ractigen Therapeutics, a leader in the field of small activating RNA (saRNA) therapies, has recently achieved an impressive milestone by dosing its first patient in an investigator-initiated clinical trial (IIT) for RAG-18, which targets Duchenne Muscular Dystrophy (DMD). This landmark event took place at the prestigious Peking Union Medical College Hospital (PUMCH) in Beijing. This trial is not only a pivotal moment for Ractigen but also a beacon of hope for patients suffering from this debilitating disease.

The investigation is spearheaded by Professor Dai Yi, a renowned authority in neuromuscular disorders and Deputy Director of the Neurology Department at PUMCH. The primary objectives of the study are to gauge RAG-18's safety profile and its pharmacokinetic characteristics, alongside evaluating its efficacy in improving muscle function in individuals diagnosed with DMD. Speaking of the trial's launch, Professor Dai expressed his excitement, stating, "We are enthusiastic to embark on this clinical trial with RAG-18, representing a cutting-edge therapeutic perspective for DMD. We are hopeful for promising clinical outcomes that could lead to groundbreaking treatment options, ultimately enhancing patients' quality of life."

The trial's commencement was also lauded by Dr. Long-Cheng Li, the Founder and CEO of Ractigen Therapeutics. He articulated the significance of this study as a critical juncture for RAG-18, underscoring its potential to tackle the complexities associated with DMD. Dr. Li emphasized that this innovative treatment aims to address all genetic mutations associated with DMD, showcasing RNA activation (RNAa) technology as an advanced approach in clinical settings that may offer hope where there previously was none.

Preclinical analysis has shown that RAG-18, administered through subcutaneous injections via Ractigen's proprietary LiCO™ (lipid-conjugated oligonucleotide) technology, effectively reduced muscle damage. These results project a promising framework for the treatment of DMD. The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to RAG-18 in 2024, which reaffirms its potential as a vital therapeutic solution for DMD patients.

Understanding Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy is a severe genetic condition triggered by mutations in the dystrophin gene, responsible for encoding a crucial muscle protein called dystrophin. The absence or malfunction of dystrophin leads to unstable muscle fibers. Without adequate dystrophin, muscle cells sustain damage and face challenges in repair, resulting in progressive weakness and degeneration.

The dystrophin gene, the largest in the human genome with 79 exons, poses significant treatment hurdles. Existing therapeutic strategies include antisense oligonucleotides (ASO) aimed at mediating exon skipping, gene therapy, and gene editing. However, these methods are limited in efficacy, highlighting the urgent need for innovative therapies that can directly target the underlying causes of DMD, offering more effective and lasting benefits.

Insights on RAG-18

RAG-18 emerges as a unique saRNA candidate that selectively targets and activates UTRN gene expression within muscle cells through the RNAa mechanism. The protein produced by the UTRN gene closely resembles dystrophin functionally and structurally, suggesting that its increased expression could serve as a substitute for the absent dystrophin in muscle cells affected by DMD. This mechanism offers a universal treatment avenue for all patients suffering from DMD, regardless of the mutation type.

The Promise of RNA Activation

Developed by Dr. Long-Cheng Li and his team, RNA activation technology is a validated platform that uses saRNAs to stimulate gene regulatory systems, thus enhancing gene expression and therapeutic protein levels. This groundbreaking technology opens extensive possibilities in drug development for a range of diseases, particularly in instances where conventional treatment methods have reached their limits.

About Ractigen Therapeutics

Ractigen Therapeutics stands at the forefront of biopharmaceutical innovation, focusing on next-generation RNA therapeutics, predominantly through saRNA platforms developed via clinically validated RNA activation technology. The company's proprietary delivery systems, including SCAD™, LiCO™, and GLORY™, enable the progressive development of solutions for unmet medical challenges across a variety of conditions such as oncology, neurological diseases, and inherited disorders. By fostering scientific discovery and patient-centric innovations, Ractigen is committed to revolutionizing healthcare through the transformative potential of RNA therapeutics. For more information, please visit www.ractigen.com.