adeno-associated virus-engineered anti-amyloid beta assembly antibody construct

This adeno-associated virus (AAV)-based gene therapy is designed for the treatment of early Alzheimer's disease. It utilizes a neuron-targeted AAV vector to deliver a genetic sequence encoding an engineered antibody-based construct (intrabody). This construct selectively recognizes and binds to neurotoxic intracellular amyloid-beta (Aβ) assemblies, such as the 30-mer ASPD (amylospheroid), and facilitates their trafficking toward intracellular degradative pathways. By clearing these assemblies before they form extracellular plaques, the therapy aims to preserve neuronal integrity and prevent neuroinflammation. Preclinical studies in 5xFAD mice have demonstrated reduced Aβ accumulation and preservation of neuronal markers without vascular injury.

Other names

neuron-targeted gene therapy enabling intracellular clearance of amyloid-β assembliesAAV-engineered anti-amyloid beta assembly antibody construct