AAVpo2.99

AAVpo2.99 is a novel porcine-derived adeno-associated virus (AAV) capsid variant identified for its high efficiency in transducing the retinal pigment epithelium (RPE) and photoreceptors. Developed by researchers at the University of Naples Federico II and the Telethon Institute of Genetics and Medicine (TIGEM), it is being investigated as a gene delivery vector for retinal gene therapy. Specifically, it has been utilized in a homology-independent targeted integration (HITI) platform to target the rhodopsin (RHO) locus in models of autosomal dominant retinitis pigmentosa (RP4) caused by the P23H mutation. The vector supports efficient in vivo genome editing and has demonstrated sustained improvements in retinal function and architecture in preclinical studies.