Design Therapeutics, Inc.

Design Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on the research and development of GeneTAC® molecules. These are a new class of small-molecule gene targeted chimera therapeutic candidates. The company designs these molecules to modify diseases by addressing the root cause of conditions resulting from inherited nucleotide repeat expansion mutations. Some diseases, like Friedreich ataxia (FA) and fragile X syndrome, involve reduced gene expression and a lack of vital proteins. Other diseases, such as myotonic dystrophy type-1 (DM1), Fuchs endothelial corneal dystrophy (FECD), and Huntington's disease (HD), are caused by nucleotide repeat expansions that produce toxic gene products, often linked to pathological nuclear foci, broad splicing disruptions, or mutant protein aggregates. Design Therapeutics' GeneTAC® small molecules are engineered to specifically target expanded genetic repeat sequences. They modulate gene expression by either increasing or decreasing mRNA transcription, depending on the disease's cause, with the goal of restoring cellular health. The company believes GeneTAC® molecules have broad potential for various unaddressed degenerative, monogenic nucleotide repeat expansion diseases globally. In preclinical studies for their lead FA program, the company observed restoration of frataxin (FXN) levels in multiple cell types from FA patients and in an in vivo murine model of FA using their FA GeneTAC® molecules. At well-tolerated doses in rodents and non-human primates (NHPs), FA GeneTAC® molecules reached the brain and heart, key organs affected by FA, at concentrations exceeding those needed to restore FXN levels in FA patient cells. Consistent with this biodistribution, increased endogenous FXN expression was observed in the brain and heart in an animal model of FA after treatment with FA GeneTAC® molecules.