RAG-21

RAG-21 is a novel small interfering RNA (siRNA) therapy developed by Ractigen Therapeutics for the treatment of amyotrophic lateral sclerosis (ALS), specifically targeting cases associated with mutations in the FUS gene, known as FUS-ALS. The drug leverages RNA interference to selectively bind and degrade FUS messenger RNA, thereby reducing production of toxic FUS protein that drives motor neuron degeneration in this aggressive ALS subtype. Delivered via Ractigen’s proprietary Smart Chemistry-Aided Delivery (SCAD) platform, RAG-21 is designed for efficient and durable gene knockdown within the central nervous system. Preclinical studies have demonstrated its ability to mitigate motor neuron loss and improve disease outcomes. The U.S. FDA has granted orphan drug designation to RAG-21 for ALS due to its potential benefit in this rare and severe neurodegenerative disorder[1][2][3][4][5][7][8].