Ractigen Therapeutics' saRNA Drug RAG-18 Receives FDA Rare Pediatric Disease Designation for Duchenne Muscular Dystrophy

Rare Pediatric Disease Designation (RPDD)It is the FDA's qualification designation for pediatric rare disease drugs that affect fewer than 200,000 patients in the United States and severely threaten the lives of children under 18, aiming to encourage the development and approval of treatments for rare pediatric diseases. If RAG-18's New Drug Application (NDA) is approved, Ractigen Therapeutics may be eligible for a Priority Review Voucher (PRV), which can be used to expedite the review of subsequent market applications for different products or sold to other companies. Its historical transaction value has exceeded 100 million US dollars, reaching up to 350 million US dollars.

Founder and CEO of RactigenDr. Li Longcheng said:"RAG-18 has successfully obtained RPDD approval, marking the recognition of RAG-18 and RNA activation technology by the U.S. FDA. This highlights Ractigen's strength in innovative drug development and further strengthens Ractigen Therapeutics' confidence in developing innovative therapies for rare diseases. We will rapidly advance the development and clinical research of RAG-18, hoping to bring a safe and effective revolutionary treatment to DMD and BMD patients worldwide."

RAG-18 is a first-in-class double-stranded saRNA drug with a novel mechanism of action, specifically targeting and activating the expression of the UTRN gene in muscle cells through the RNA activation (RNAa) mechanism. Utrophin, encoded by the UTRN gene, is structurally and functionally similar to Dystrophin. Its upregulation can functionally compensate for the missing Dystrophin in DMD muscle cells, thereby treating all mutation types of DMD and BMD patients. Preclinical studies have shown that, using Ractigen Therapeutics' self-developed small nucleic acid delivery system LiCO™ and administered via subcutaneous injection, RAG-18 significantly reduces muscle damage in DMD model mice, demonstrating potential for treating DMD patients.

Duchenne Muscular Dystrophy (DMD) is a severe X-linked recessive genetic disorder caused by mutations in the DMD gene, which encodes dystrophin. The disease primarily affects male children, with symptoms such as muscle inflammation, fibrosis, and reduced motor ability typically appearing between the ages of 3 and 5. Without treatment, most patients lose the ability to walk independently before the age of 20 and may die from cardiac and diaphragmatic (respiratory) muscle failure before the age of 30. The global incidence of DMD is approximately 1 in every 3,600 to 6,000 newborn males, with about 70,000 patients in China. Current treatments for DMD include antisense oligonucleotide (ASO)-mediated exon skipping, gene therapy, and gene editing, but the efficacy of these therapies is very limited, indicating a significant unmet clinical need in the development of DMD drugs.

RNA activation (RNAa) is a platform technology pioneered internationally by Dr. Longcheng Li, the founder of Ractigen, and his team, which has already been clinically validated. This technology uses double-stranded RNA targeting the promoter region of genes to activate gene expression, restoring the levels of therapeutic proteins. RNA activation technology is a very rare platform technology in the pharmaceutical field, with broad application prospects in drug development, including genetic diseases, chronic diseases, metabolic diseases, cardiovascular and cerebrovascular diseases, and cancer.

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

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™ (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.