AAV-PR

AAV-PR is a peptide-engineered adeno-associated virus (AAV) capsid optimized for the selective and efficient transduction of smooth muscle cells (SMCs) within the brain and peripheral vasculature. Developed by a collaborative team from Massachusetts General Hospital, the Broad Institute, and Harvard Medical School, AAV-PR was designed to overcome the limitations of conventional vectors, such as AAV9, in crossing the endothelial barrier to reach the underlying vascular media. Preclinical evaluations in human ex vivo arterial perfusion models and non-human primate studies have demonstrated that AAV-PR achieves significantly higher gene expression and vector copy numbers in SMC layers compared to standard serotypes. This vascular-tropic capsid is intended as a delivery platform for gene therapies targeting SMC-driven genetic vascular diseases, including those affecting the aorta and cerebrovascular system.