Ractigen Announces U.S. FDA Rare Pediatric Disease Designation (RPDD) Granted to RAG-18 for the treatment of Duchenne Muscular Dystrophy

SUZHOU, China, July 25, 2024 /PRNewswire/ -- Ractigen Therapeutics, a pioneering developer of small activating RNA (saRNA) therapeutics, today 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."

About RAG-18

RAG-18 is a first of its kind saRNA candidate designed to specifically target and activate UTRN gene expression in muscle cells via RNAa mechanism. The utrophin protein encoded by the UTRN gene is structurally and functionally similar to dystrophin, and its upregulation could potentially serve as a functional replacement for the missing dystrophin in DMD muscle cells, providing treatment for all DMD patients regardless of the specific mutation location. Preclinical data indicate that RAG-18, delivered through subcutaneous injection utilizing Ractigen's proprietary LiCO^TM (lipid-conjugated oligonucleotide) technology, has effectively mitigated muscle damage, demonstrating significant potential in treating DMD patients..

About DMD

Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) are severe genetic disorders caused by mutations in the dystrophin gene, leading to the absence or insufficiency of functional dystrophin protein. This protein is essential for muscle fiber stability. Without it, muscle cells are easily damaged and cannot repair themselves, resulting in progressive muscle weakness and degeneration. The dystrophin gene, the largest in the human body, contains 79 exons. Current disease-modifying therapeutic approaches for DMD include antisense oligonucleotides (ASO) mediated exon skipping, gene therapy, and gene editing, with exon skipping being the most common strategy. However, these treatments have significant limitations, highlighting the critical need for innovative therapies that target the root cause of DMD to provide more effective and long-lasting benefits for patients.

About RNAa

RNAa is a clinically validated platform technology developed by Dr. Long-Cheng Li and his team. It utilizes saRNAs to target gene regulatory domains, activating gene expression and restoring therapeutic protein levels. This innovative technology holds vast potential for developing therapeutic drugs across various diseases, particularly where traditional methods fall short, including genetic disorders.

About Ractigen Therapeutics

A leader in saRNA drug development, Ractigen Therapeutics is at the forefront of developing saRNA drugs utilizing the RNAa mechanism to up-regulate endogenous gene expression. This innovative approach involves saRNA targeting specific genes to enhance transcription, thereby restoring normal protein functions. Ractigen's cutting-edge technology is pivotal in treating diseases unaddressable by conventional methods, such as those resulting from epigenetic silencing or gene downregulation. For more information, please visit our website at www.ractigen.com.

SOURCE Ractigen Therapeutics