E. Loeb, Sophia Fergione, Vivian Yudistyra, Marco M. Fanous, Abigail R. Benkert, Delaney G. Fisher, Joshua A. Hull, Mai K. ElMallah, Aravind Asokan
Overall, our work leverages untapped dependoparvoviral diversity to overcome pre-existing and vector-induced immunity, enabling expansion of patient eligibility and effective redosing.
The use of adeno-associated virus (AAV) as a gene therapy vector is significantly limited by pre-existing immunity. The high seroprevalence and broad antigenic cross-reactivity of primate-derived AAVs restrict patient eligibility and preclude therapeutic redosing. Here, we harness the phylogenetic diversity of non-mammalian dependoparvoviruses to engineer serologically distinct AAV capsids for immune evasion. A barcoded screen of divergent Dependoparvovirus isolates identifies AAV.div3A, a chimeric capsid with robust transduction, zero antigenic cross-reactivity, and undetectable seroprevalence. Derived from a phylogenetically distant Muscovy duck isolate, AAV.div3A fully evades neutralization in mice, even after passive immunization with NAb+ human serum or following initial vector dosing. Further engineering yields AAV.div3A-M1, a myotropic, liver-detargeted capsid with enhanced cardiac and diaphragm transduction. In a Pompe disease model, redosing with AAV.div3A or div3A-M1 significantly increases therapeutic GAA levels. Overall, our work leverages untapped dependoparvoviral diversity to overcome pre-existing and vector-induced immunity, enabling expansion of patient eligibility and effective redosing.