RTX-117

RTX-117 is an investigational **small molecule drug** being developed as a disease-modifying therapy for Charcot-Marie-Tooth disease (CMT), a progressive inherited peripheral neuropathy. It is designed to **activate eIF2B**, thereby restoring the translation of cap-dependent mRNAs to normalize protein expression and address underlying disease mechanisms in CMT, rather than just alleviating symptoms. This therapeutic approach has the potential to slow or halt disease progression and improve neuromuscular function. The drug is being advanced by ReviR Therapeutics, which received FDA Orphan Drug Designation for RTX-117 for CMT. A Phase 1 clinical trial in healthy volunteers is planned for early 2026, to be followed by a Phase Ib/IIa study in CMT patients[1]. Some sources also indicate Xili Technology (Shenzhen) as an early originator, though ReviR Therapeutics appears to be the primary developer in current development[1][3]. RTX-117 represents a novel approach with no current approved disease-modifying therapies for CMT.

Brand names

RTX-117RTX117RTX 117

Other names

RTX-117RTX117RTX 117