SUZHOU, China I May 15, 2024 I Ractigen Therapeutics, a clinical-stage pharmaceutical company committed to developing pioneering therapies, today announces that the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) has approved the Investigational New Drug (IND) application for the initiation of Phase 1 clinical trials in China for RAG-17, targeting Amyotrophic Lateral Sclerosis (ALS).

The IND-opening study is a Phase I, randomized, double-blind, placebo-controlled study evaluating the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of RAG-17 in subjects with ALS carrying the Superoxide Dismutase 1 (SOD1) mutation.

Dr. Long-Cheng Li, Founder, President, and CEO of Ractigen, expressed enthusiasm about this significant milestone: “This marks a pivotal moment for our company, as RAG-17 is our first siRNA drug targeting the central nervous system to receive CDE approval. We are excited to advance this therapy into clinical trials for ALS patients and are optimistic about its potential to bring meaningful benefits to those with SOD1 mutation.”

Mr. Lei Cai, a prominent ALS advocate and fighter against the disease, who himself battles ALS in China, shared his hopeful perspective on RAG-17: “I hold a strong sense of optimism for RAG-17. Its innovative approach and promising preliminary results give me great confidence in its potential. I believe this drug could offer significant hope and tangible benefits to the ALS community, especially for those with the SOD1 mutation.”

RAG-17 previously received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) in March 2023 and has been cleared for clinical trials in the U.S. Additionally, an Investigator-Initiated Trial (IIT) study (NCT05903690) for RAG-17 is currently ongoing, with 6 patients recruited and treated, showing encouraging preliminary safety and efficacy results.

About RAG-17

RAG-17 is a therapeutic siRNA specifically designed to suppress the SOD1 gene in ALS patients with pathogenic mutations. Utilizing Ractigen’s proprietary SCAD™ delivery platform, RAG-17 combines siRNA with an accessory oligonucleotide (ACO) for enhanced effectiveness in central nervous system (CNS) tissues. Preclinical studies, including those using the hSOD1G93A mouse model, have demonstrated RAG-17’s remarkable therapeutic efficacy in ameliorating motor function and prolonging survival. 

About ALS

ALS, a severe neurodegenerative disease with no cure, significantly reduces life expectancy, with most patients succumbing to respiratory failure within 3-5 years of diagnosis. Initial symptoms typically include muscle cramps, twitching, and weakness. These symptoms progress to difficulties with movement and speech, the need for assisted breathing, paralysis, and ultimately death. Of the more than 50 genes associated to ALS, mutations in the SOD1 gene account for approximately 20% of the cases.

About Ractigen Therapeutics

Ractigen Therapeutics, a clinical-stage pharmaceutical company, is committed to developing innovative oligonucleotide drugs and therapeutic approaches. Ractigen has pioneered several globally leading oligonucleotide drug delivery platforms, including SCAD™ and LiCO™, and boasts a highly diverse pipeline of oligonucleotide drugs. These drugs are aimed to target a wide variety of indications, from neurodegenerative and neuromuscular diseases to cancer, metabolic disorders, and hematological diseases. The company is dedicated to delivering innovative therapeutic solutions for previously undruggable targets and addressing incurable diseases across various fields. For more information, please visit our website at

SOURCE: Ractigen Therapeutics