Design Therapeutics is a clinical-stage biopharmaceutical company developing small-molecule drugs for rare inherited genetic diseases caused by nucleotide repeat expansions — conditions where abnormally long stretches of repeated DNA sequences either silence a critical gene or generate toxic gene products that damage cells. Design's core technology is its proprietary GeneTAC platform, which produces heterobifunctional small molecules with two functional ends: one that targets the abnormal repeat sequence in the disease-causing gene, and one that interacts with the cell's transcriptional machinery to either increase or decrease gene expression. This approach is designed to modify disease at the root genetic level, rather than manage downstream symptoms, using a conventional small-molecule format rather than a biologic or gene therapy. Design has four active programs: its lead program targets Friedreich Ataxia (FA), a fatal neurodegenerative disease with no approved disease-modifying therapy; a second program targets Fuchs Endothelial Corneal Dystrophy (FECD), formulated as an eye drop; a third program targets Myotonic Dystrophy Type 1 (DM1), entering Phase 1 in 2026; and a fourth preclinical program targets Huntington's Disease. Design has no approved products and no product revenue. The company funds operations through capital raises and outsources all drug manufacturing to third parties. If products are approved, Design would likely price them as orphan drugs given the small patient populations involved, and intends to commercialize independently in key markets while potentially partnering in other geographies.