Ractigen Therapeutics Announces Breakthrough IIT Results for RAG-17, a Novel siRNA Therapy for SOD1-ALS

These positive clinical results demonstrate the high translatability of Ractigen's previously published preclinical data for RAG-17. Ractigen’s preclinical studies have shown that RAG-17 exhibits significant therapeutic effects in SOD1-G93A ALS mouse and rat models. The studies indicate that RAG-17 significantly delays disease progression and improves survival rates, providing strong preclinical evidence for its potential as an effective therapy for ALS-SOD1.

Dr. Longcheng Li, founder and CEO of Ractigen, said:"We are encouraged by these preliminary clinical results, which bring us one step closer to our goal of bringing new hope to ALS patients. The positive outcomes of this trial highlight the potential of RAG-17 in treating ALS-SOD1. We will continue to advance its clinical development with the aim of ultimately delivering a life-changing therapy to patients."

The data from this study will be presented at three major conferences this year: the 27th National Congress of Neurology in China in September, Neuroscience 2024 in Chicago, USA, in October, and the 35th International ALS/MND Symposium in Montreal, Canada, in December — one of the largest annual conferences on ALS and motor neuron disease research worldwide.

RAG-17 received Orphan Drug Designation (ODD) from the U.S. FDA in March 2023, followed by FDA approval for clinical trials. In May 2024, it also obtained clinical trial authorization from the Center for Drug Evaluation (CDE) of China's National Medical Products Administration.

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 gene 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 treatment with RAG-17 significantly delays disease onset, extends animal survival time, and improves motor function.

ALS is a chronic progressive neurodegenerative disease characterized by the prominent involvement of upper and lower motor neurons, primarily manifesting as muscle weakness, muscle atrophy, bulbar paralysis, and pyramidal tract signs. ALS is an incurable disease, with patients typically surviving 3-5 years after onset. ALS can be divided into sporadic and familial forms. Familial ALS can be caused by mutations in various genes, with SOD1 being one of the most common. In China, SOD1 is the most frequent mutated gene causing ALS, accounting for approximately 20% of familial ALS cases and 5% of sporadic ALS cases.