Longevity & Aging

Harness Therapeutics Targets Root Cause of Parkinson's With Novel Gene-Silencing Drug

Harness Therapeutics has received a major grant from the Michael J. Fox Foundation to develop a potential disease-modifying drug for Parkinson's disease linked to GBA1 gene mutations — the most common genetic risk factor for the condition. The two-year program uses a cutting-edge microRNA-blocking technology to simultaneously boost two key proteins involved in cellular waste clearance in the brain. GBA1 mutations disrupt how cells process certain fats, leading to earlier onset and faster progression of Parkinson's. By restoring proper protein function inside cellular recycling compartments called lysosomes, researchers hope to slow or halt disease progression. This work also has implications for related brain diseases including Alzheimer's and other synucleinopathies.