Matilde Helbo Sørensen

AMD causes vision loss in millions of adults, with wAMD representing the most severe form. The current standard of care involves repeated anti-VEGF injections, yet this approach is insufficient in many patients, who subsequently develop subretinal fibrosis resulting in irreversible vision loss. This PhD project will explore a novel approach to wAMD treatment, using gene therapy and RNA interference to target subretinal fibrosis and angiogenesis. By engineering a multi-mechanistic RNAi platform to target a pro-fibrotic mediator, we aim to reduce fibrosis and, combined with an anti-angiogenic approach, we aim to improve therapeutic efficacy. Using in vitro, in vivo, and ex vivo models, we will investigate both efficacy and safety of our RNAi platform. The potential impact of this innovative project is substantial and would be beneficial for both patients and the healthcare system.