Design Therapeutics is pioneering novel small-molecule therapeutic candidates, called gene targeted chimeras (GeneTACs™), that are designed to be disease-modifying and target the underlying cause of inherited nucleotide repeat expansion diseases. Certain nucleotide repeat expansion diseases, such as Friedreich ataxia, can result in reduced expression of specific mRNAs; in other diseases, such as myotonic dystrophy type-1, Fuchs endothelial corneal dystrophy, and Huntington disease, the nucleotide repeat expansions result in the generation of toxic gene products.

Our GeneTACs are designed to selectively bind to genetic repeat sequences, modulate gene expression either by restoring or blocking transcription, and restore cellular health. As a platform, we believe that GeneTACs have broad potential applicability across monogenic nucleotide repeat expansion diseases.