RAG-18

RAG-18 is an investigational small activating RNA (saRNA) therapy developed by Ractigen Therapeutics for the treatment of Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD), regardless of the specific mutation in the DMD gene. It is designed to activate expression of the UTRN gene in muscle cells, leading to increased production of utrophin—a protein structurally and functionally similar to dystrophin, which is deficient or absent in DMD/BMD patients. By upregulating utrophin via RNA activation (RNAa), RAG-18 aims to compensate for dystrophin deficiency and mitigate muscle damage. The therapy uses a proprietary lipid-conjugated oligonucleotide delivery technology and has shown efficacy in preclinical models, including cell lines and mouse models with humanized UTRN promoters. As of mid-2025, it has received both Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA but remains in preclinical development[1][2][3][6][7].