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 lead disease focus, myotonic dystrophy type 1 (DM1), is the most frequent cause of adult onset muscular dystrophy. Expansion has assembled an initial focused pipeline of compositions targeting repeats that drive a number of expansion repeat disorders.
We believe that the field of small molecules interacting with RNA (SMiRNA™) has the potential to fundamentally change the therapeutic landscape by significantly opening up the small molecule “druggable” universe beyond classic protein-based targets. Advances in our understanding of the structure and function of RNA has opened up a wealth of potential small molecule drug targets. However, the ability to selectively target RNA remains difficult, encumbered by both the discovery tools available and by our emerging understanding of RNA-small molecule interactions.
Expansion has assembled key platform enabling technologies and tools to allow for the identification of specific and potent novel small molecule binders of RNA. We have demonstrated the ability to identify small, drug like compositions of matter that can target various RNAs, including mRNA and various non-coding RNAs, across multiple therapeutic areas.