AB-1005 gene therapy placed on fast track by regulators in US, UK

AskBio's experimental gene therapy, AB-1005, has received special designations from the FDA in the U.S. and the regulatory agency in the U.K. These designations are intended to speed up the development and review process of promising treatments for serious conditions like Parkinson’s disease, aiming to get new therapies to patients faster. AB-1005 is currently being tested in the REGENERATE-PD Phase 2 trial, which is recruiting Parkinson's patients in the U.S. and will soon expand to the EU and U.K. This trial focuses on patients with moderate Parkinson’s who have motor fluctuations despite stable medication use. The therapy works by delivering the GDNF gene to specific brain regions, helping to produce a protein that supports the survival of neurons that produce dopamine, which is crucial for motor control. This gene therapy is administered directly into the brain via a precise surgical procedure. In an earlier Phase 1 trial, AB-1005 showed promise by improving motor symptoms in patients with moderate Parkinson’s and stabilizing the condition in those with mild disease. It was generally safe with no serious side effects. The REGENERATE-PD trial aims to further evaluate the effectiveness and safety of AB-1005 in a larger group of patients over 18 months. Additionally, AskBio is testing this therapy for another condition, multiple system atrophy, in a separate Phase 1 trial.