#China #ALS #Ractigen Therapeutics #RAG-17 #Nantong

Ractigen Therapeutics Begins Phase II Trials for RAG-17

Ractigen Therapeutics has recently announced a significant advancement in their clinical research, marking the beginning of the Phase II clinical trial of RAG-17, a promising siRNA therapy aimed at treating SOD1-mutated amyotrophic lateral sclerosis (ALS). On January 13, 2026, the first patient was dosed at the Second Affiliated Hospital, Zhejiang University School of Medicine, with renowned expert Dr. Zhi-Ying Wu leading the effort.

The primary objective of this Phase II trial is to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and initial efficacy of repeated intrathecal injections of RAG-17 for those afflicted with SOD1 mutations. Key participant sites for this trial include prestigious institutions such as Beijing Tiantan Hospital and West China Hospital of Sichuan University, among others.

This trial represents a logical progression from the successful completion of the Phase I single ascending dose (SAD) portion, which provided encouraging results, indicating a strong safety profile along with promising biomarkers. Notably, the SAD trial showed significant reductions in cerebrospinal fluid (CSF) SOD1 protein levels and improvements in plasma neurofilament light chain (NfL) concentrations, suggesting the potential efficacy of RAG-17 in altering disease progression. These findings strongly validate Ractigen's proprietary SCAD™ delivery system for effective administration in the central nervous system (CNS).

Dr. Long-Cheng Li, the founder and CEO of Ractigen Therapeutics, highlighted the importance of this milestone, stating, "The successful dosing of the first patient represents a significant milestone in our mission to develop transformative therapies for ALS. The positive results from the Phase I trial affirm the promise of RAG-17 and provide us with strong confidence in its potential to markedly improve the lives of patients suffering from SOD1-mutated ALS."

The enthusiasm surrounding the trial was echoed by Dr. Zhi-Ying Wu, who expressed optimism about RAG-17's potential to significantly enhance the quality of life for ALS patients. "We are excited to be part of this important study. Based on the improvements we have observed clinically, we look forward to further investigating RAG-17's potential to enhance the quality of life for ALS patients as we progress," said Dr. Wu.

The therapeutic candidate, RAG-17, is crafted specifically to target and silence the superoxide dismutase 1 (SOD1) gene mRNA, which is implicated in causing familial ALS due to toxic gain-of-function mutations. By focusing on reducing the toxic mutant SOD1 protein's production, RAG-17 aspires to slow down or ideally halt the devastating progression of SOD1-ALS.

The U.S. Food and Drug Administration (FDA) has granted RAG-17 Orphan Drug Designation (ODD), further endorsing its potential as a vital treatment option for ALS. Additionally, RAG-17 has been enlisted in the Center for Drug Evaluation's CARE Program of the National Medical Products Administration (NMPA), which is dedicated to accelerating the development of therapies for rare diseases.

ALS, a cruel and progressive neurodegenerative disorder, primarily targets nerve cells in the brain and spinal cord, leading to muscle weakness and paralysis, with a fatal prognosis typically within three to five years post-diagnosis. Approximately 10-20% of familial ALS cases are attributed to SOD1 gene mutations, and there remains an urgent need for effective treatments to combat this debilitating disease.

Ractigen Therapeutics, at the forefront of RNA therapeutics, is committed to innovating next-generation treatments. The company focuses on small activating RNAs (saRNAs) developed through its advanced RNA activation (RNAa) technology. Their proprietary delivery platforms, including SCAD™, LiCO™, and GLORY™, facilitate the development of effective RNA solutions targeting severe conditions, especially those affecting the CNS. With a mission rooted in scientific excellence and patient-centered innovation, Ractigen Therapeutics is poised to revolutionize healthcare with its advancements in RNA therapeutics.

For further insights, visit Ractigen Therapeutics.