#MDA2022 – Gene Therapy ACT-101 Boosts GAA Levels in Early Trial

The investigational gene therapy ACT-101 overall was well-tolerated among three people with late-onset Pompe disease (LOPD) who were treated in a Phase 1/2 clinical trial. Moreover, the trial results also showed that treatment markedly increased levels of the GAA enzyme — deficient in Pompe disease — in the patients’ muscle…

Bayer Acquires AskBio and its Gene Therapy Program for Pompe Disease

Bayer reached an agreement to acquire Asklepios BioPharmaceutical (AskBio), including its clinical program of ACT-101, an experimental one-time gene therapy for late-onset Pompe disease. AskBio, which will remain an independent entity, has a portfolio that comprises early and clinical stage gene therapies to treat neuromuscular,…