Design Therapeutics

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.