Exploring delivery methods for gene therapy in AMD

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Gene therapy is being used in both wet and dry age-related macular degeneration in the form of a biofactor. What does that mean? Well, we're using the viral vector to infect the cells to produce some sort of protein. In patients with wet macular degeneration, that protein would be a similar thing as an anti-VEGF agent. So essentially, your own cells are producing an anti-VEGF agent. In dry macular degeneration, just like we found that complement inhibition works, they're looking at different complement modulators to produce, for instance, complement factor I or complement factor H, or CD59 which blocks the formation of the membrane attack complex. Now, in all these situations, the key area of gene therapy that we're trying to determine is what's the best way to deliver it? Historically, it was performed using surgery in a subretinal injection, so that's not a very complicated procedure, but does require surgery. Some of our clinical studies currently are looking at the idea of doing an intravitreal-injected viral vector or placing it into the suprachoroidal space. Now, you would think that intravitreal would be by far the easiest. However, there are issues with that; because of the way the eye is formed, there's the internal limiting membrane. So different areas and different cells are going to be transfected with the viral vector if you inject it into the vitreous, or you inject into the suprachoroidal space, or you do surgery, and these are all things we're trying to figure out in the clinical studies for gene therapy and wet macular degeneration, as well as dry now. Currently, the only gene therapies that are approved for the eye are for inherited retinal degenerations. We don't yet have any macular degeneration-approved gene therapy studies. However, in wet age-related macular degeneration, we have a gene therapy study in phase three, so that's the furthest along.