Ractigen Therapeutics' siRNA Therapy RAG-21 Receives FDA Orphan Drug Designation for Treatment of ALS

November 202419thRactigen Therapeutics announced that its self-developed FUS gene-targeting small interfering RNA (siRNA) therapy, RAG-21, has successfully received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for the treatment of Amyotrophic Lateral Sclerosis (ALS).

RAG-21 is a siRNA therapy specifically targeting FUS gene mutations, effectively reducing the level of FUS protein and addressing the root cause of motor neuron degeneration. The drug functions through the RNA interference (RNAi) mechanism, alleviating the mislocalization and abnormal aggregation of FUS protein by lowering its levels. Preclinical studies have shown that RAG-21 exhibits significant knockdown effects and favorable safety. As a novel RNA therapy, RAG-21 offers new hope for improving the prognosis of FUS-ALS patients, who currently lack disease-modifying treatments. This marks our second FDA Orphan Drug Designation in the ALS field, following the previous designation of RAG-17 for SOD1-ALS, demonstrating our steadfast commitment to conquering ALS.

Orphan Drug Designation Granted by FDA Aims to Encourage the Development of Drugs and Biologics for Rare Diseases (Diseases affecting fewer than 200,000 people in the United States). Companies receiving Orphan Drug Designation will enjoy several incentives, including...Seven years of market exclusivity from the date of market approval and a waiver of the new drug application fee.

About RAG-21

RAG-21 selectively knocks down the expression of the FUS gene through the RNA interference (RNAi) mechanism, thereby reducing the production of toxic FUS protein and targeting the root pathological issue of FUS-ALS. With Ractigen's SCAD™ delivery platform, RAG-21 achieves targeted, efficient, and long-lasting gene knockdown in the central nervous system (CNS). Preclinical data shows that RAG-21 has the potential to alleviate motor neuron degeneration and improve disease prognosis.Brings hope to patients with this aggressive ALS subtype.

About ALS and FUS Gene Mutations

ALS is a severe and incurable neurodegenerative disease that selectively damages motor neurons in the cortex and spinal cord. Most patients die from respiratory failure within 2-5 years after diagnosis. FUS gene mutations are associated with an especially aggressive form of ALS, characterized by early onset and rapid progression. These mutations result in toxic FUS proteins.The accumulation, mislocalization, and formation of abnormal inclusions within neurons ultimately lead to motor neuron degeneration.

AboutRactigen

Ractigen Therapeutics is a platform-based new drug development company based in China and targeting the global market, committed to developing breakthrough small nucleic acid drugs and disease treatment methods. Ractigen Therapeutics is one of the few global small nucleic acid drug companies that simultaneously possess both intrahepatic and extrahepatic delivery technologies, having developed several internationally leading proprietary delivery platform technologies, including SCAD™ and LiCO™. Based on RNA activation technology and its self-developed Smart-TTC saRNA drug development platform, the company has established a highly differentiated small nucleic acid drug pipeline with indications covering neurodegenerative diseases, neuromuscular diseases, cancer, metabolic diseases, and hematological disorders, providing innovative therapeutic solutions for undruggable targets and currently incurable diseases across various disease areas.