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Ractigen Therapeutics Presents Positive Phase I Data for RAG-17 in SOD1-ALS at the 2026 AAN Annual Meeting, Demonstrating 81% Reduction in Neurofilament Light Chain
Blinded Phase I data showed an 81.2% drop in plasma NfL and no serious adverse events, while Phase II dosing has begun.
- On Tuesday, Ractigen Therapeutics announced positive preliminary Phase I data for RAG-17, an investigational RNA-based therapeutic targeting amyotrophic lateral sclerosis , while confirming Phase II dosing has commenced.
- RAG-17 utilizes Ractigen's proprietary Smart Chemistry-Aided Delivery platform to silence the SOD1 gene, aiming to slow or halt disease progression in patients with SOD1-ALS mutations.
- In the 180 mg cohort, plasma neurofilament light chain levels dropped by 81.2% by Day 150, while the 150 mg cohort achieved a 58.1% reduction in cerebrospinal fluid SOD1 protein by Day 90.
- Ractigen CEO Dr. Long-Cheng Li called the 81% reduction in plasma NfL "unprecedented," while Principal Investigator Dr. Zhi-Ying Wu noted patients face a "very limited treatment landscape."
- Data were presented on Sunday at the American Academy of Neurology Annual Meeting in Chicago, and RAG-17 has received Orphan Drug Designation from the U.S. Food and Drug Administration .
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Ractigen Therapeutics Presents Positive Phase I Data for RAG-17 in SOD1-ALS at the 2026 AAN Annual Meeting, Demonstrating 81% Reduction in Neurofilament Light Chain
— Single intrathecal dose of SCAD-siRNA therapeutic RAG-17 yields favorable safety profile and profound, durable biomarker reductions —
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