RRG001

RRG001 is an adeno-associated virus (AAV) vector-based gene therapy developed by Shanghai Refreshgene Technology for the treatment of neovascular (wet) age-related macular degeneration (nAMD) and diabetic retinopathy. It is administered via subretinal injection and aims to provide long-term, stable expression of a therapeutic protein after a single dose, potentially reducing the need for repeated anti-VEGF injections in nAMD patients. The therapy uses gene transfer to deliver genetic material encoding an anti-VEGF protein directly into retinal cells, thereby inhibiting vascular endothelial growth factor activity and reducing abnormal blood vessel growth associated with these eye diseases. The precise molecular mechanism has not been fully defined but involves antagonism of VEGF signaling through genetic delivery[1][2][4][7].