NANTONG and SUZHOU, China, December 24, 2024 — Ractigen Therapeutics, a clinical-stage biopharmaceutical company specializing in RNA-based treatments, today reported that the first patient has been dosed in its Phase I clinical trial of RAG-17. RAG-17 is a novel siRNA therapy targeting Amyotrophic Lateral Sclerosis (ALS) linked to superoxide dismutase 1 (SOD1) gene mutations. This trial is underway at the Second Affiliated Hospital of Zhejiang University School of Medicine.
This Phase I study, a randomized, double-blind, placebo-controlled trial, will assess the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of RAG-17 in individuals with SOD1-ALS. The trial is being overseen by Dr. Yilong Wang of Beijing Tiantan Hospital, Capital Medical University, and Dr. Zhiying Wu of the Second Affiliated Hospital of Zhejiang University School of Medicine, in collaboration with Dr. Huifang Shang of West China Hospital, Sichuan University.
“Commencing the RAG-17 trial represents a significant step forward in our efforts to address ALS, a severely debilitating neurodegenerative disease,” stated Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics. “This accomplishment reaffirms our dedication to developing RNA-based therapies with the potential to significantly improve the lives of patients and their families impacted by rare and severe illnesses like ALS.”
RAG-17 received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) in March 2023, followed by FDA clearance of its Investigational New Drug (IND) application. In May 2024, the IND was approved by the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA). Positive clinical data from an Investigator-Initiated Trial (IIT) of RAG-17, presented in November 2024, further supports its potential. These results were shared at the 27th National Conference of Neurology, Neuroscience 2024, and the 35th International Symposium on ALS/MND, generating considerable interest within the global scientific community.
About RAG-17
RAG-17 is a siRNA designed to reduce SOD1 gene expression in ALS patients with disease-causing mutations. Leveraging Ractigen’s proprietary SCAD
delivery system, RAG-17 combines siRNA with an accessory oligonucleotide (ACO) to improve delivery to the central nervous system (CNS). Preclinical studies, including those using the hSOD1G93A mouse model, showed that RAG-17 effectively improved motor function and extended lifespan. The IIT data indicated that intrathecal RAG-17 was well-tolerated across all doses, with only mild side effects, and comprehensive safety assessments confirmed its favorable safety profile.
About ALS
ALS, a severe and currently incurable neurodegenerative disease, substantially shortens life expectancy, with most patients dying from respiratory failure within 3-5 years of diagnosis. Typical initial symptoms include muscle cramps, twitching, and weakness. These symptoms worsen, leading to impaired movement and speech, reliance on respiratory support, paralysis, and ultimately, death. SOD1 gene mutations account for approximately 20% of familial ALS and 5% of sporadic ALS cases.
About Ractigen Therapeutics
Ractigen Therapeutics is a leader in small activating RNA (saRNA) drug development, using the RNA activation (RNAa) mechanism to increase the production of endogenous genes. This approach uses saRNA to target specific genes to enhance transcription and restore normal protein function. Ractigen’s advanced technology is crucial for treating diseases that are difficult to address with conventional methods, such as those arising from epigenetic silencing or gene downregulation. For more information, please visit our website at .
SOURCE Ractigen Therapeutics