Expansion Therapeutics is a drug discovery and development company pursuing the vast potential of small molecule medicines for RNA-mediated diseases. We are initially focused on expansion repeat disorders, a set of ~30 genetic diseases for which there are no satisfactory therapies. Our initial disease focus, myotonic dystrophy type 1 (DM1), is the most frequent cause of adult onset muscular dystrophy.
We believe that the field of small molecules interacting with RNA (SMiRNA™) has the potential to fundamentally change the therapeutic landscape by significantly expanding the small molecule “druggable” universe beyond classic protein-based targets. We have assembled a platform of proprietary enabling technologies, including discovery and validation tools, that allow for the efficient identification and evaluation of small molecule drug candidates.