RGT-DM1

RGT-DM1 is a preclinical small molecule candidate being developed by Rgenta Therapeutics for the treatment of Myotonic Dystrophy. The drug targets PMS1, a protein involved in the DNA mismatch repair (MMR) pathway that has been identified as a key driver of somatic trinucleotide repeat expansion. In Myotonic Dystrophy, the expansion of CTG repeats in the DMPK gene leads to toxic RNA gain-of-function; by inhibiting PMS1, RGT-DM1 aims to stabilize these repeats and prevent further expansion, thereby addressing the underlying genetic instability of the disease. Rgenta leverages its proprietary discovery platform to identify small molecules that can modulate RNA-mediated pathways, positioning RGT-DM1 as a potential disease-modifying therapy for Myotonic Dystrophy.