BB-AMD-231

BB-AMD-231 is an ocular DNA-directed RNA interference (ddRNAi) therapeutic developed by Benitec Biopharma in collaboration with 4D Molecular Therapeutics for the treatment of age-related macular degeneration. It utilizes proprietary adeno-associated virus (AAV) capsids from 4DMT to deliver the ddRNAi payload, aiming to enhance biodistribution and efficacy in the eye. The drug is designed to achieve long-term therapeutic activity through gene silencing, specifically targeting factors involved in the pathogenesis of wet age-related macular degeneration, such as vascular endothelial growth factor (VEGF), which stimulates abnormal blood vessel growth and leakage in the retina. The goal is to provide a sustained treatment effect via intravitreal injection, a commercially attractive route of administration.