Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to RAG-18, one of the company's lead saRNA product candidates for the treatment of Duchenne Muscular Dystrophy (DMD). RAG-18 could represent a novel and translatable therapeutic strategy for DMD and Becker Muscular Dystrophy (BMD) caused by any mutation of the DMD gene.

The RPDD is granted by the FDA to drugs that treat serious or life-threatening diseases primarily affecting children under 18 years of age with a prevalence of fewer than 200,000 in the United States. If a New Drug Application (NDA) for RAG-18 is approved, Ractigen may be eligible to receive a Priority Review Voucher (PRV), which can be used to expedite the review of a subsequent marketing application for a different product or sold to another company with historical transaction values exceeding $100 million and reaching as high as $350 million, representing a substantial financial opportunity for Ractigen.

Dr. Long-Cheng Li, Founder and CEO of Ractigen Therapeutics, stated: "Receiving the RPDD for RAG-18 is a milestone for Ractigen and reinforces our commitment to developing innovative therapies for rare diseases. This designation not only accelerates the development of RAG-18 but also opens opportunities for future advancements in RNAa therapies. We are dedicated to improving the lives of patients with DMD and other rare diseases."