Design Therapeutics

Design Therapeutics is a clinical-stage biotechnology company developing a new class of small molecule therapies called GeneTAC® (gene-targeted chimeras) for the treatment of genetic disorders caused by nucleotide repeat expansions. The company was founded with a vision of restoring health to patients with genetic disorders by harnessing their body's own biology. Design's platform targets diseases at the DNA level, modulating transcription to address the underlying cause of repeat expansion diseases rather than their symptoms. The company's pipeline spans multiple high-need indications including Friedreich Ataxia, Fuchs Endothelial Corneal Dystrophy, Myotonic Dystrophy, and Huntington's Disease. Design Therapeutics was co-founded by Pratik Shah, Ph.D. (President and CEO) and Aseem Ansari, Ph.D. (Scientific Advisor), who pioneered the development of synthetic transcription factors over 25 years of foundational research. The company is headquartered in Carlsbad, California, and is publicly traded on the Nasdaq under the ticker DSGN. Its team consists of recognized global leaders in DNA-targeted small molecules, transcription modulation, medicinal chemistry, and clinical development.