Design

Design Therapeutics is a clinical-stage biopharmaceutical company pioneering a new class of small molecule therapies known as Gene Targeted Chimeras (GeneTACs). These molecules are designed to address the underlying causes of inherited nucleotide repeat expansion diseases by either increasing or decreasing the transcription of specific genes. The company's lead program, DT-216, is being developed for the treatment of Friedreich ataxia, a progressive neurodegenerative disorder caused by a transcriptional deficiency of the frataxin gene. Beyond Friedreich ataxia, Design is advancing a pipeline targeting other serious conditions such as myotonic dystrophy type 1 and Fuchs endothelial corneal dystrophy. By leveraging its proprietary platform, Design Therapeutics aims to provide transformative treatments for patients with limited or no therapeutic options. The company is headquartered in Carlsbad, California, and is publicly traded on the Nasdaq Global Select Market.