Tides Express | Ractigen Therapeutics Submits IND Application for RAG-17 Injection, a Small Interfering RNA Therapy for SOD1-ALS

February 2024On the 23rd, the official website of the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) announced that Ractigen Therapeutics' "RAG-17 Injection" has applied for clinical trials.This candidate drug is developed based on Ractigen Therapeutics' independently developed Smart Chemical-Assisted Delivery (SCAD™) platform, which ensures efficient and safe delivery of the target drug to the central nervous system. Preclinical studies have shown that RAG-17 significantly delays disease onset, extends animal survival time, and improves motor function.

RAG-17 is an siRNA drug that targets and inhibits the expression of the SOD1 gene, used for treating ALS caused by SOD1 mutations. RAG-17 utilizes Ractigen Therapeutics' proprietary SCAD delivery platform technology, which achieves efficient and long-lasting delivery in the central nervous system (CNS) by conjugating double-stranded RNA with auxiliary oligonucleotides (ACO). Multiple preclinical efficacy studies have shown that RAG-17 exhibits superior efficacy compared to reference drugs in ALS disease models (e.g., the hSOD1G93A mouse model).

About Ractigen

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 very few small nucleic acid pharmaceutical companies in the world that simultaneously masters both intrahepatic and extrahepatic delivery technologies, having developed several internationally leading proprietary small nucleic acid drug delivery platform technologies, including SCADTM and LiCOTM, with independent intellectual property rights. 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 and neuromuscular diseases, cancer, metabolic and hematological disorders, providing innovative therapeutic solutions for undruggable targets and currently incurable diseases across various disease areas.