ReviR Therapeutics, a previous CMT Research Foundation grantee, announced in March 2026 that the first human has been dosed with RTX-117, an experimental drug for Charcot-Marie-Tooth disease that ReviR says targets multiple CMT subtypes. The drug was administered in a Phase 1 clinical trial in China, and there’s already a path cleared for bringing it to the United States.
Because this is a safety trial, the drug was administered to healthy volunteers, not CMT patients, who would receive the experimental treatment in a Phase 2 trial.
What Is RTX-117?
RTX-117 is an oral pill (no injections) that works by targeting a cellular pathway called the Integrated Stress Response, or ISR. In CMT, this pathway gets stuck, preventing nerve cells from making the proteins they need to function. RTX-117 helps release that block by activating expression of the eIF2B gene, restoring normal protein production.
What makes this approach particularly exciting is that it isn’t tied to a single genetic mutation. Because the ISR is a pathway affected in many subtypes of CMT, RTX-117 has the potential to help patients across multiple CMT subtypes.
The Technology Behind It
RTX-117 was discovered using artificial intelligence. ReviR built an AI platform called VoyageR that analyzes RNA to identify promising drug candidates, working in partnership with another AI and robotics drug discovery company called XtalPi. AI-driven drug discovery can compress timelines that would otherwise take decades, and RTX-117’s rapid path to a human trial reflects that.
What This Means for Patients from the US
The current trial is in China, and the groundwork for testing patients in the US is already laid. In 2025, RTX-117 received FDA Investigational New Drug (IND) approval, meaning the FDA has reviewed the science and clinical plans, clearing it for human testing in the US. RTX-117 also received Orphan Drug Designation for CMT, a special status that recognizes the unmet need and provides development incentives.
While a US trial hasn’t been announced yet, ReviR hopes this China trial can facilitate development of their US expansion strategy.