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RTX-317

Development stage: Preclinical
Lead developer: ReviR Therapeutics
Modality: Small Molecules
Administration: Oral

Overview

RTX-317 is an orally administered, brain-penetrant small molecule mRNA splicing modulator being developed by ReviR Therapeutics for the treatment of Huntington's disease. Discovered using ReviR's proprietary VoyageR AI platform, RTX-317 acts as a dual-target agent that reduces the expression of both mutant huntingtin (mHTT) and PMS1 (PMS1 homolog 1, a mismatch repair system component) mRNA levels. It achieves this by inducing the inclusion of poison exons within the pre-mRNAs, leading to degradation via nonsense-mediated mRNA decay (NMD). By lowering mHTT and PMS1, RTX-317 aims to reduce somatic CAG repeat expansion and mutant huntingtin protein aggregation, addressing the underlying genetic pathology of Huntington's disease. The candidate is currently undergoing IND-enabling studies.

Targets

HTT (Huntingtin protein)

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