LQT-23

LQT-23 is a first-in-class, orally bioavailable allosteric small molecule inhibitor of MSH3 (a component of the MutSβ complex) being developed by LoQus23 Therapeutics. It targets the DNA mismatch repair (MMR) pathway, which has been genetically linked to the progression of triplet repeat expansion diseases. By selectively inhibiting MSH3, LQT-23 aims to stop the somatic expansion of CAG or CTG repeats that drive the pathology of Huntington's Disease and myotonic dystrophy type 1. Preclinical data indicates that the molecule can potently and selectively modulate MutSβ to stabilize repeat lengths in cellular and animal models, representing a novel approach to treating the underlying cause of these genetic disorders.