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On July 25, Ractigen Therapeutics announced that its self-developedSmall activating RNA (saRNA) drugsRAG-18 Receives U.S. FDAPediatric Rare Disease Drug Designation (RPDD)RAG-18 is an innovative therapeutic strategy currently under development for the treatment of anyDMDCaused by gene mutationsDuchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD)。
RNA activation (RNAa) technology uses double-stranded RNA targeting the promoter region of genes to activate gene expression, restoring the levels of therapeutic proteins. RNA activation technology has broad application prospects in the development of therapeutic drugs, including cancer, genetic diseases, chronic diseases, metabolic diseases, and cardiovascular and cerebrovascular diseases. Ractigen Therapeutics is committed to developing breakthrough small nucleic acid drugs and has established multiple small nucleic acid drug pipelines based on RNA activation technology. Public data shows,Ractigen Therapeutics already has two innovative small nucleic acid drugs in clinical development, respectively small activating RNA (saRNA).DrugRAG-01and double-stranded small interfering RNA drugsRAG-17。RAG-18 isDeveloped by RactigenAnother innovative dual-strand saRNA drug with a unique mechanism of action specifically targets and activates muscle cells through the RNA activation (RNAa) mechanism.UTRNGene Expression. Utrophin, encoded by the UTRN gene, is structurally and functionally similar to dystrophin. Its upregulation can functionally replace the missing dystrophin in DMD muscle cells, thereby treating all mutation types of DMD and BMD patients.Preclinical studies have shown that using Ractigen's self-developed small nucleic acid delivery system LiCO™ andBy Subcutaneous InjectionRAG-18 significantly reduced muscle damage in DMD model mice, demonstrating potential for treating DMD patients.Duchenne Muscular Dystrophy (DMD)It 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 patients typically showing symptoms such as muscle inflammation, fibrosis, and reduced mobility for the first time between the ages of 3 and 5. Without treatment, most patients will lose the ability to walk independently before the age of 20 and may die before the age of 30 due to cardiac and diaphragm (respiratory) muscle failure. Current treatments for DMD include antisense oligonucleotide (ASO)-mediated exon skipping, gene therapy, and gene editing, but their effectiveness is still limited, and there remains a significant clinical need for DMD drugs.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 to receive 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.Founder and Chief Executive Officer of RactigenDr. Longcheng LiIndicates:"RAG-18 has successfully obtained RPDD approval, marking the recognition by the U.S. FDA of both RAG-18 and RNA activation technology. This highlights Ractigen's strength in innovative drug development and further strengthens Ractigen Therapeutics' confidence in developing groundbreaking 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."[1] Breakthrough Therapy! Ractigen's RAG-18 Granted FDA Rare Pediatric Disease Designation, Poised to Transform the Treatment Landscape for Duchenne Muscular Dystrophy. Retrieved July 25, 2024, from https://mp.weixin.qq.com/s/2S1WQxQEqJpOAhiehGKCZQ
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