Research and Development

About

The Research and Development team at Design Therapeutics focuses on pioneering novel small-molecule therapeutic candidates, specifically GeneTACs™, to modify diseases by targeting the underlying causes of inherited nucleotide repeat expansion diseases. This multidisciplinary team, comprising bioinformatics experts, computational chemists, and biologists, works collaboratively to design, synthesize, and evaluate the efficacy of these therapeutics through advanced research and innovative drug discovery techniques.