Design Therapeutics is pioneering novel small-molecule therapeutic candidates, called GeneTAC™ molecules (Gene Targeted Chimera), that are designed to be disease-modifying and target the underlying cause of inherited nucleotide repeat expansion diseases.

Our GeneTAC™ molecules 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 GeneTAC™ molecules have broad potential applicability across monogenic nucleotide repeat expansion diseases.