Astellas Gene Therapies

This was an early-stage study to test a new gene therapy called ASP2016 for heart problems in adults with Friedreich ataxia, a rare genetic disease. The main goals were to check if the treatment was safe and how well people could tolerate it. Participants would receive a single i…

This early-stage study is testing a one-time gene therapy called AT845 for adults with late-onset Pompe disease. The goal is to see if delivering a working copy of the faulty gene directly into the body is safe and can help control the disease. Participants receive a single infus…

This study tested a one-time gene therapy called AT132 for young boys with X-linked myotubular myopathy (XLMTM), a rare and life-threatening muscle disease. The goal was to see if delivering a healthy gene could improve muscle function and reduce the need for breathing machines. …

This study aims to gather information that could help develop future gene therapies for late-onset Pompe disease, a genetic condition that causes progressive muscle weakness. Researchers are measuring antibody levels against a common virus used in gene therapy (AAV8) and against …