DT-MyD1

DT-MyD1 is a preclinical small-molecule Gene Targeted Chimera (GeneTAC) developed by Design Therapeutics for the treatment of Myotonic Dystrophy Type-1 (DM1). DM1 is a multisystemic genetic disorder caused by an unstable CTG trinucleotide repeat expansion in the 3' untranslated region of the *DMPK* gene. This expansion results in the production of toxic RNA that sequesters key splicing proteins, leading to widespread splicing defects. DT-MyD1 is designed to selectively bind to the expanded CTG repeats and recruit transcriptional machinery to modulate gene expression, thereby reducing the accumulation of toxic *DMPK* RNA and addressing the underlying cause of the disease.