Posts tagged: Pompe disease

Preliminary data from Astellas Pharma’s gene therapy study in adult patients with Pompe disease has crossed a first safety hurdle. At WORLDSymposium, Astellas presented interim safety data from a Phase I/II study assessing its AAV gene therapy for the rare, autosomal metabolic disease.

Weston Miller, M.D., senior medical director of clinical development at Astellas Gene Therapies, called the positive interim and tolerability data an important waypoint in the development of AT845, the company’s investigational adeno-associated virus (AAV) gene replacement therapy.

AT845 has been designed to deliver a functional alpha-glucosidase (GAA) gene into a patient in order to express acid alpha-glucosidase (GAA) directly into the muscle cells of adults with Late-Onset Pompe Disease (LOPD). GAA is responsible for metabolizing glycogen. The absence of GAA or its improper functioning causes the excessive accumulation of glycogen, primarily in the skeletal and cardiac muscles. Once it has accumulated in significant amounts, the glycogen causes damage to tissue structure and function, Miller said. AT845 is being assessed in the global Phase I/II FORTIS first-in-human clinical study.

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