Core Insights - Design Therapeutics has developed a novel class of small molecules that can modulate the expression of specific genes in the genome, functioning similarly to transcription factors [1] - The company is focusing on several monogenic disorders, including Friedreich's ataxia, Fuchs endothelial corneal dystrophy, myotonic dystrophy, and Huntington's disease [2] Group 1 - The unique mechanism of Design Therapeutics' molecules involves recognizing DNA sequences through minor groove interactions and recruiting transcriptional machinery to adjust gene expression levels [1] - Friedreich's ataxia is characterized by low expression levels of the frataxin gene, which is the root cause of the disease [3]