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UTR-HEXA

Development stage: Preclinical
Lead developer: UTR Therapeutics
Modality: RNA Therapeutics → Nucleic Acid Therapeutics

Overview

UTR-HEXA is an investigational RNA-based therapeutic being developed by UTR Therapeutics for the treatment of GM2 gangliosidosis, specifically Tay-Sachs disease. The drug utilizes a novel RNA rewriting platform that targets the 3' untranslated region (UTR) of the *HEXA* mRNA. By engineering destabilized AU-rich elements (AREs) within the 3'UTR, the therapy aims to modulate the stability and increase the expression of the *HEXA* gene, which encodes the alpha subunit of the beta-hexosaminidase A enzyme. This approach is intended to restore enzymatic activity and prevent the toxic accumulation of GM2 gangliosides in the central nervous system. It is currently in the discovery phase.

Other names: UTR-HEXA (GM2 gangliosidosis / Tay-Sachs disease program)

Targets

HEXA (Hexosaminidase subunit alpha)

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