UX053: mRNA therapy for Glycogen Storage Disease Type III (GSDIII)

GSDIII: a disease involving accumulation of glycogen in the liver and muscle

GSDIII is caused by a glycogen debranching enzyme deficiency, which results in glycogen accumulation in the liver and muscle and the inability to release glucose as needed to provide energy and regulate blood glucose levels. Patients with GSDIII experience hepatomegaly (enlarged liver), hypoglycemia, hyperlipidemia, progressive liver cirrhosis and muscle disease later in life. No pharmacologic therapies are approved for GSDIII, which affects an estimated 10,000 people in the developed world.

Evaluating UX053 to provide the deficient protein in patients with GSDIII

UX053 is an investigational mRNA therapy encoding glycogen debranching enzyme that is encapsulated in a lipid nanoparticle. It is designed to provide the deficient protein in patients with GSDIII. Preclinical studies of UX053 demonstrated reductions in glycogen content in the liver in multiple models of the disease. The U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application, and Ultragenyx plans to conduct a Phase 1/2 study.

Learn more about UX053 clinical studies