Ractigen Therapeutics, a China-based developer of small activating RNA (saRNA) therapeutics, announced on Thursday that it has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for RAG-18, one of the company's lead saRNA product candidates.

This follows Rare Pediatric Disease Designation (RPDD) granted to the product last month, further strengthening the potential of this novel therapeutic strategy for Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) caused by any mutation of the DMD gene.

RAG-18, a first of its kind saRNA candidate, is aimed at specifically targeting and activating UTRN gene expression in muscle cells through RNAa mechanism.

Dr Long-Cheng Li, Ractigen Therapeutics' founder and CEO, said: "Receiving FDA Orphan Drug Designation marks a pivotal achievement for RAG-18. Combined with the recent Rare Pediatric Disease Designation, it reflects the groundbreaking work we're doing with RNA activation and reinforces our commitment to making a real difference in the lives of those affected by rare diseases. This recognition fuels our determination to push forward with RAG-18's development, aiming to bring innovative and life-changing treatments to DMD and BMD patients around the world."