CAP-003

CAP-003 is an intravenously administered, engineered adeno-associated virus (AAV)-based gene therapy developed by Capsida Biotherapeutics. It is designed to deliver a functional copy of the GBA gene to nerve cells in the brain, thereby increasing levels and activity of the enzyme glucocerebrosidase (GCase). Mutations in GBA are the most common genetic risk factor for Parkinson’s disease (PD), leading to reduced GCase activity and lysosomal dysfunction. CAP-003 aims to restore normal GCase enzyme activity, slow or halt disease progression, and provide a disease-modifying treatment for Parkinson’s disease associated with GBA mutations (PD-GBA). Preclinical studies in non-human primates demonstrated that a single IV infusion of CAP-003 resulted in dose-dependent increases in brain GCase activity—substantially above efficacy thresholds—with strong biomarker correlations and no significant safety concerns. The therapy leverages an engineered capsid capable of crossing the blood-brain barrier, offering a non-invasive alternative to previous approaches that required direct CNS administration[1][2][5][8].