Pursuing the Vast Potential of Small Molecule Medicines for RNA-Mediated Diseases

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 molecule-RNA interactions 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.

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Expansion has exclusively licensed enabling technologies and intellectual property from The Disney Lab at The Scripps Research Institute.

The Scripps Research Institute Florida chemist has been trying to do something that defies drug discovery dogma: Invent small-molecule drugs that can target RNA.

The RNA Drug Hunters — C&EN

Our understanding of RNA has evolved considerably in recent years, driven by advances in molecular biology and efforts to catalogue all functional elements in the human genome, including elements that act at the protein and RNA levels. RNA is now known to play a diverse role in cell biology and is implicated in many diseases, making it an attractive therapeutic target.

Platform Technologies

Expansion has assembled key enabling platform technologies to allow for identification of specific and potent RNA targeted small molecule medicines across multiple therapeutic areas.

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Pipeline of Compositions

Expansion is advancing a focused pipeline of compositions initially targeting expansion repeat disorders, a set of approximately 30 genetic diseases for which there are currently no satisfactory treatments.

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