Ractigen Therapeutics, a clinical-stage pharmaceutical company committed to developing pioneering therapies,  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.