RAG-17

RAG-17 is an investigational small interfering RNA (siRNA) therapeutic specifically designed to suppress the superoxide dismutase 1 (SOD1) gene in patients with amyotrophic lateral sclerosis (ALS) who have pathogenic SOD1 mutations. It utilizes Ractigen Therapeutics’ proprietary Smart Chemistry-Aided Delivery (SCAD) platform, which conjugates the siRNA with an accessory oligonucleotide for enhanced delivery and durability in central nervous system tissues. By binding to SOD1 mRNA, RAG-17 promotes its degradation, thereby reducing production of the toxic mutant SOD1 protein that drives neurodegeneration in familial ALS. Preclinical studies demonstrated improved motor function and survival in disease models, while early clinical trials showed favorable safety and tolerability profiles along with significant reductions in cerebrospinal fluid SOD1 levels and promising signs of clinical benefit[1][2][4][5][6][7][8].

Other names

RAG-17 sodium saltRAG17 sodium saltRAG 17 sodium salt