UTR-FOXG1

UTR-FOXG1 is an investigational RNA-based therapeutic being developed by UTR Therapeutics for the treatment of FOXG1 Syndrome. The drug utilizes a novel RNA rewriting modality that targets the 3' untranslated region (UTR) of the FOXG1 mRNA. Specifically, it employs engineered destabilized AU-rich elements (AREs) to modulate the stability and expression levels of the FOXG1 protein. By manipulating these regulatory elements, the therapy aims to restore functional levels of FOXG1 in patients with haploinsufficiency or other mutations leading to reduced protein activity. It is currently in the discovery phase of development.

Other names

FOXG1 Syndrome programFOXG-1 Syndrome programFOXG 1 Syndrome program