AAV-Abeta-F20P

AAV-Abeta-F20P is an investigational adeno-associated virus (AAV)-based gene therapy designed to treat Alzheimer's disease. Developed by researchers at Baylor College of Medicine, Washington University in St. Louis, and the spinout company Aβrain, the therapy delivers a minigene encoding a sequence variant of the amyloid-beta (Aβ) peptide with a proline substitution at residue 20 (F20P). This variant peptide is designed to be expressed and secreted into the extracellular space of the brain, where it acts as an aggregation inhibitor. It physically binds to wild-type Aβ, preventing its fibrilization into neurotoxic aggregates and promoting the compaction of diffuse amyloid plaques rather than their clearance, thereby improving cognitive function and reducing neuroinflammation.

Other names

AAV-AbetaF20PAAV-AbetaF-20PAAV-AbetaF 20PAAV-Aβ-F20PAAV-AβF20PAbeta F20PAβ-F20PAβF20P