AAV-DIDA-CBE

AAV-DIDA-CBE is an adeno-associated virus (AAV) vector-based gene therapy component that functions as a cytosine base editor (CBE). Developed by Shenzhen Eye Hospital, it is designed for the treatment of neovascular (wet) age-related macular degeneration (nAMD), particularly in patients who are resistant to standard anti-VEGF antibody therapies. The therapy is part of a dual-vector CRISPR system, where AAV-DIDA-CBE is co-administered with AAV-nCas9 (encoding a Cas9 nickase) to target the Vascular Endothelial Growth Factor (VEGF) gene. This approach enables precise C-to-T base editing to silence or downregulate VEGF expression directly in retinal cells following subretinal injection. The goal of this intervention is to provide a durable, long-term therapeutic effect, potentially eliminating the need for chronic intravitreal anti-VEGF injections.