Clinical-stage biotechnology company developing oral small-molecule medicines that target RNA and modulate splicing to address historically undruggable disease targets
Clinical-stage biotechnology company developing oral small-molecule medicines that target RNA and modulate splicing to address historically undruggable disease targets. Its pipeline focuses on oncology and central nervous system disorders, including MYB-driven cancers and repeat expansion diseases such as Huntington’s disease. The company also offers RSwitch, a regulatable gene-expression switch for gene and cell therapies.