UX053: mRNA Therapy for the potential treatment of glycogen storage disease type III (GSD III)

Program overview

Stage: Phase 1/2
Disease: GSD III
Prevalence: 10,000 people in our territories
Disease Mechanism: Glycogen debranching enzyme deficiency
Symptoms: Hepatomegaly, hypoglycemia, hyperlipidemia, progressive liver cirrhosis, muscle disease
Treatment Modality: mRNA therapy
Treatment Mechanism: Encode glycogen debranching enzyme

GSD III: accumulation of glycogen in the liver and muscle may result in cirrhosis and worsening muscle disease

GSD III is caused by a glycogen debranching enzyme deficiency, which results in accumulation of abnormally structured glycogen (limit dextrins) in the liver and muscles. Patients with GSD III experience hepatomegaly (enlarged liver), hypoglycemia, hyperlipidemia, progressive liver fibrosis, cirrhosis and muscle disease. No pharmacologic therapies are approved for GSD III, which affects an estimated 10,000 people in our territories.

Evaluating UX053 to provide the deficient enzyme in patients with GSD III

UX053 is an investigational mRNA therapy encoding glycogen debranching enzyme that is encapsulated in a lipid nanoparticle. The lipid nanoparticle facilitates its uptake into target cells, where the mRNA is released and then translated into functional glycogen debranching enzyme. Preclinical studies of UX053 demonstrated reductions in glycogen content in the liver and improvement in fasting hypoglycemia. The U.S. Food and Drug Administration (FDA) cleared the Investigational New Drug (IND) application, and a Phase 1/2 study is ongoing.

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