Huntington's Disease GeneTAC molecules

Design Therapeutics is developing a GeneTAC® (Gene Targeted Chimera) small molecule program for the treatment of Huntington's disease (HD). These molecules are designed to selectively modulate gene expression by targeting the CAG repeat expansion in the HTT gene. The program aims to achieve allele-selective reduction of mutant huntingtin (mHTT) protein expression while preserving the expression of wild-type HTT. The GeneTAC® molecules consist of a DNA-targeting moiety that binds to CAG repeats in Exon 1 of the HTT gene, linked to a ligand moiety that recruits transcriptional machinery to downregulate transcription of the mutant allele. Preclinical studies have demonstrated over 50% reduction of mutant HTT expression in the brain striatum following systemic administration. The program is currently in the preclinical stage, with the company working toward the selection of a development candidate for future IND submission.

Other names

HTT GeneTACHuntington's Disease GeneTAC program