Ractigen Reports Positive Human Trial Data for SOD1-ALS Therapy

The Phase 1 trial, conducted on six patients with SOD1-ALS, met all primary safety endpoints without any serious adverse events. Beyond safety, the data showed a 69% mean reduction in cerebrospinal fluid SOD1 protein and a 62% decrease in plasma neurofilament light chain (NfL), a key indicator of neuroaxonal damage. Some participants even exhibited respiratory function stabilization or improvement during the study period.

Dr. Yilong Wang, the trial's lead investigator from Beijing Tiantan Hospital, noted that the therapy's ability to achieve such responses in humans, combined with its performance in advanced-stage animal models, positions RAG-17 as a potential best-in-class treatment. The drug utilizes Ractigen’s Smart Chemistry-Aided Delivery (SCAD™) technology, which conjugates siRNA to an accessory oligonucleotide to ensure deep penetration into the central nervous system.

Preclinical results included in the Nature Medicine paper underscore the durability of the therapy. In non-human primate studies, RAG-17 achieved a 91% reduction of SOD1 mRNA in the lumbar spinal cord that persisted for over two months. These findings suggest that patients might eventually require less frequent dosing than current treatment regimens allow, potentially easing the burden of care for those with the rapidly progressive condition.