In a first-in-human phase 1/2 study, GT005, an AAV2-based one-time investigational gene therapy, showed potential to regulate the complement pathway involved in the pathogenesis of geographic atrophy.
The complement system has a key role in the pathogenesis of age-related macular degeneration, Robert MacLaren, MD, said at the virtual Euretina congress. Central to this is C3b, with an amplification cycle that leads to formation via C5 convertase of the membrane attack complex, leading to secondary damage and disruption of the retinal pigment epithelium. GT005 (Gyroscope Therapeutics) is