DT-FXS

DT-FXS is a preclinical-stage small molecule GeneTAC (Gene Targeted Chimera) candidate being developed by Design Therapeutics for the treatment of Fragile X Syndrome (FXS). FXS is a neurodevelopmental disorder caused by a CGG triplet repeat expansion in the 5' untranslated region of the FMR1 gene, which leads to hypermethylation, epigenetic silencing, and a subsequent deficiency of the Fragile X Mental Retardation Protein (FMRP). DT-FXS is designed to selectively bind to these expanded CGG repeats and recruit the cell's endogenous transcriptional machinery to the FMR1 promoter, thereby reactivating the silenced gene and restoring the production of functional FMRP protein.