Astellas Gene Therapies

This study was designed to test a new gene therapy called ASP2016 for heart problems in people with Friedreich Ataxia, a rare genetic disease. The goal was to see if the treatment is safe and tolerable. However, the study was withdrawn before any participants were enrolled.

This study tested a gene therapy called AT132 for children under 5 with a rare, serious muscle disease (XLMTM). The goal was to see if it could reduce the need for breathing machines. However, the treatment caused severe liver problems and deaths, so the study was stopped early. …

This early-stage study tests a gene therapy called AT845 for adults with late-onset Pompe disease, a condition that weakens muscles. The treatment aims to deliver a working copy of the GAA gene to muscle cells. Eleven participants who have been on standard enzyme replacement ther…

This study looks at people with late-onset Pompe disease to measure antibodies against a common gene therapy carrier (AAV8) and the standard enzyme replacement therapy. It also checks for biomarkers in blood and urine that could help track the disease. No new treatment is given—j…