Cambridge-based biotechnology company developing structure-based, oral small-molecule somatic expansion inhibitors for Huntington’s disease and other triplet repeat expansion diseases
Cambridge-based biotechnology company developing structure-based, oral small-molecule somatic expansion inhibitors for Huntington’s disease and other triplet repeat expansion diseases. Its lead candidate, LQT-23, is an allosteric inhibitor of MSH3/MutSβ intended to reduce DNA instability and slow neurodegeneration.