AAV9.CAG.scFvMC1

AAV9.CAG.scFvMC1 is an investigational adeno-associated virus serotype 9 (AAV9) gene therapy vector designed to deliver a single-chain variable fragment (scFv) derived from the conformation-dependent anti-tau monoclonal antibody MC1. Driven by a CAG promoter, the therapy selectively targets early pathological, misfolded conformations of the microtubule-associated protein tau (MAPT). In preclinical models of tauopathy, AAV9.CAG.scFvMC1 has been evaluated in combination with MRI-guided focused ultrasound (FUS) to facilitate non-surgical, targeted delivery across the blood-brain barrier to specific brain regions, such as the frontal cortex, resulting in significant reductions of both soluble and insoluble phosphorylated tau species. The therapy is being developed for the treatment of Alzheimer's disease and other tauopathies.

Other names

AAV9-CAG-scFvMC1AAV-9-CAG-scFvMC1AAV 9-CAG-scFvMC1AAV9-scFvMC1AAV-9-scFvMC1AAV 9-scFvMC1scFv-MC1 gene therapyscFv-MC-1 gene therapyscFv-MC 1 gene therapyscFvMC1 gene therapyscFvMC-1 gene therapyscFvMC 1 gene therapy