The study, conducted across five major Chinese medical centers, evaluates the safety and efficacy of multiple ascending doses of RAG-17. Developed via the company’s proprietary SCAD delivery platform, the drug is designed to silence the mRNA of the SOD1 gene, preventing the production of toxic proteins linked to the progression of familial ALS. Clinical teams at institutions including Beijing Tiantan Hospital and West China Hospital of Sichuan University are managing the trial, which follows positive safety and biomarker data recently published in Nature Medicine.

Long-Cheng Li, founder and CEO of Ractigen, noted that the trial builds on Phase I results showing significant reductions in CSF SOD1 protein and plasma neurofilament light chain levels. With the recruitment phase closed, the research now moves into an intensive data collection period. RAG-17 has already secured Orphan Drug Designation from the FDA and inclusion in the NMPA’s CARE program, both of which provide accelerated regulatory pathways for therapies addressing rare, life-threatening neurological conditions.