Askbio Inc

This early-stage study is testing a single infusion of an experimental gene therapy called AB-1009 in adults with late-onset Pompe disease. The goal is to see if this one-time treatment is safe and if it can help control the disease, which causes progressive muscle weakness. Part…

This study is testing whether a gene therapy delivered directly into the brain can help control symptoms in people with moderate Parkinson's disease. Participants will receive either the real gene therapy or a sham (fake) surgery, and neither they nor the doctors will know which …

This study is testing the safety of a single-dose gene therapy called AB-1003 in adults with limb-girdle muscular dystrophy type 2I/R9, a rare genetic muscle disorder. Researchers will give increasing doses to small groups of participants to check for side effects. The goal is to…