Astellas Pharma Inc. announced an update on preliminary safety and efficacy data from its ongoing FORTIS phase1/2 clinical trial evaluating the safety, tolerability and exploratory efficacy of investigational AT845 in adults with late-onset Pompe disease (LOPD).
The data presented during a platform presentation at the 19th Annual WORLDSymposium in Orlando, FL (Poster: #95). AT845 is an investigational adeno-associated virus (AAV) gene replacement therapy designed to deliver a functional human acid alpha-glucosidase (GAA) gene directly in muscle cells in adults with LOPD.
