RAG-18 Receives FDA Orphan Drug Status for Duchenne and Becker Muscular Dystrophy

Ractigen Therapeutics, a forward-thinking company in the realm of small activating RNA (saRNA) therapeutics, has announced that its leading product candidate, RAG-18, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA). This follows the Rare Pediatric Disease Designation (RPDD) that RAG-18 received last month, reinforcing the potential of this innovative treatment for Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) regardless of the mutation in the DMD gene. These recognitions mark RAG-18 as the first saRNA drug to gain such notable regulatory endorsements from the FDA.

The FDA's Orphan Drug Designation is designed to support the development of treatments for rare diseases affecting fewer than 200,000 individuals in the United States. Benefits of this designation include development incentives like a seven-year market exclusivity post-approval and the waiver of the New Drug Application fee.

Dr. Long-Cheng Li, the Founder and CEO of Ractigen Therapeutics, expressed the significance of this achievement: "Securing the FDA Orphan Drug Designation for RAG-18 is a major milestone. Coupled with the Rare Pediatric Disease Designation, it underscores our pioneering work in RNA activation and our dedication to improving the lives of those with rare diseases. This recognition boosts our resolve to advance RAG-18's development and deliver transformative treatments to DMD and BMD patients globally."

RAG-18 is a groundbreaking saRNA candidate aimed at specifically activating the UTRN gene expression in muscle cells via RNAa mechanisms. The utrophin protein, produced by the UTRN gene, is similar both structurally and functionally to dystrophin. By upregulating utrophin, RAG-18 could potentially replace the missing dystrophin in DMD muscle cells, offering a treatment option for all patients irrespective of their specific gene mutation. Preclinical studies have demonstrated that RAG-18, administered through subcutaneous injection using Ractigen's proprietary lipid-conjugated oligonucleotide technology (LiCO™), effectively reduces muscle damage, showing substantial promise in DMD treatment. The FDA granted RAG-18 the Rare Pediatric Disease Designation in July of this year.

Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) are serious genetic disorders caused by mutations in the dystrophin gene, leading to the lack or insufficiency of functional dystrophin protein. Dystrophin is crucial for the stability of muscle fibers; its absence results in progressive muscle weakness and degeneration as muscle cells become easily damaged and fail to repair. The dystrophin gene, the largest in the human genome with 79 exons, has made current therapeutic approaches like antisense oligonucleotides (ASO) mediated exon skipping, gene therapy, and gene editing possible, with exon skipping being the most prevalent. However, these methods have limitations, emphasizing the necessity for novel therapies that address the fundamental causes of DMD to provide more effective, lasting benefits.

RNAa is a validated platform technology pioneered by Dr. Long-Cheng Li and his team. It employs saRNAs to target gene regulatory domains, thereby activating gene expression and restoring therapeutic protein levels. This innovative approach holds significant promise for developing treatments across a range of diseases, especially those where conventional methods are ineffective, such as certain genetic disorders.

Ractigen Therapeutics is a leader in the development of saRNA drugs using the RNAa mechanism to boost endogenous gene expression. This involves targeting specific genes with saRNA to enhance transcription and restore normal protein functions. The technology developed by Ractigen is crucial for addressing diseases that conventional treatments cannot adequately treat, particularly those resulting from epigenetic silencing or gene downregulation.