AAN 2026: VQ-101 reaches the brain, hits target in diverse Parkinson’s patients
WHY IT MATTERS
This is one of the first oral drugs shown to cross the blood-brain barrier and activate neuroprotective pathways in genetically diverse Parkinson's patients, potentially expanding treatment options beyond GBA-mutation carriers.
Scientists tested a new pill called VQ-101 that can enter the brain and increase levels of a protective protein in people with Parkinson's disease. In early testing, the drug worked in patients both with and without a specific genetic change (GBA mutation). This is an important first step toward potentially helping different groups of Parkinson's patients.
A new experimental oral therapy called VQ-101 successfully reached the brain and increased the activity of a key protective protein in people with Parkinson’s disease, according to early trial results. The Phase 1 study showed that the treatment, developed by Vanqua Bio, effectively boosted protein levels in patients with and without GBA gene mutations. These findings were presented […] The post AAN 2026: VQ-101 reaches the brain, hits target in diverse Parkinson’s patients appeared first