Ractigen Therapeutics Receives CDE Approval for Phase I Clinical Trial of RAG-17, a First-in-Class CNS-Targeted siRNA Therapy for SOD1-ALS

On May 15, 2024, Ractigen Therapeutics announced that RAG-17, the company's first self-developed small nucleic acid drug targeting central nervous system diseases, received clinical trial approval from the Center for Drug Evaluation (CDE) of China's National Medical Products Administration.

RAG-17 is used to treat Amyotrophic Lateral Sclerosis (ALS) caused by mutations in the Superoxide Dismutase 1 (SOD1) gene. This open-label study is a randomized, double-blind, placebo-controlled Phase I clinical trial aimed at evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of RAG-17 in ALS patients carrying SOD1 mutations.

Dr. Longcheng Li, Founder/CEO of Ractigen, stated：“RAG-17 is one of the few small nucleic acid drugs globally that can be efficiently delivered to the central nervous system. We are thrilled that our first IND application in China has been granted clinical trial approval by the CDE, marking a significant milestone for us. We eagerly anticipate RAG-17’s safety and efficacy being validated in ALS patients soon. We believe that, compared with existing treatment options, RAG-17 has the potential to offer better therapeutic outcomes for ALS patients with SOD1 gene mutations.”

Famous ALS (Amyotrophic Lateral Sclerosis) fighter Mr. Cai Lei stated:“The preliminary efficacy of RAG-17 innovative therapy is encouraging, and I am confident in the potential of this drug. I believe it can bring significant therapeutic effects and hope to ALS patients.”

RAG-17 received Orphan Drug Designation (ODD) from the U.S. FDA in March 2023, followed by FDA approval for clinical trials. Additionally, an Investigator-Initiated Trial (IIT) (NCT05903690) of RAG-17 is currently underway, with six subjects recruited and treated. Preliminary results have shown promising efficacy and good safety.

RAG-17 is a double-stranded small interfering RNA (siRNA) targeting the SOD1 gene, which treats ALS patients caused by SOD1 mutations by reducing SOD1 protein expression. RAG-17 utilizes Ractigen Therapeutics' independently developed SCAD™ (Smart Chemical-Assisted Delivery) platform with independent intellectual property rights to achieve efficient delivery to the central nervous system. Preclinical efficacy studies have shown that RAG-17 treatment significantly delays disease onset, prolongs animal survival time, and improves motor function.

Ractigen Therapeutics is a platform-based new drug research and development company based in China and targeting the global market. It is 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 multiple internationally leading small nucleic acid drug delivery platform technologies with independent intellectual property rights, such as 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 and neuromuscular diseases, cancer, metabolic and hematological disorders, providing innovative therapeutic solutions for undruggable targets and currently incurable diseases across various disease areas.