DT-818

DT-818 is a small molecule developed by Design Therapeutics as a selective gene-targeted chimera (GeneTAC) for the treatment of myotonic dystrophy type-1 (DM1). It targets the CTG repeat expansion in the DMPK gene, the root cause of DM1, by selectively reducing transcription of the mutant expanded allele. In preclinical studies, DT-818 demonstrated greater than 90% reduction in toxic RNA foci, corresponding splicing correction, and selective targeting of mutant DMPK. It is intended as a potentially best-in-disease treatment for DM1, addressing the disease at its genetic cause[1][2][3][4][5][7][11].

Other names

GeneTAC small molecule for myotonic dystrophy type 1