AAV9-R3VQ

AAV9-R3VQ is an adeno-associated virus (AAV) mediated gene therapy designed to deliver the anti-amyloid-beta (Aβ) nanobody R3VQ directly to the brain for the treatment of Alzheimer's disease. Developed by researchers at the Institut du Cerveau (Paris Brain Institute) and the Pasteur Institute, the therapy utilizes a modified AAV9 vector (pHp.e.B) to cross the blood-brain barrier and express the R3VQ nanobody in neurons and astrocytes. The R3VQ nanobody is a camelid-derived single-domain antibody (VHH) that binds to Aβ aggregates, including both intracellular and extracellular plaques. By enabling in situ production and secretion (facilitated by an IL-2 signaling peptide), the therapy aims to overcome the delivery challenges associated with traditional monoclonal antibodies, such as poor blood-brain barrier penetration and limited parenchymal diffusion, potentially improving brain bioavailability and therapeutic efficacy against amyloid-driven neurodegeneration.

Other names

R3VQ gene therapyR-3VQ gene therapyR 3VQ gene therapy