GT005

GT005 is an investigational gene therapy developed for the treatment of geographic atrophy (GA) secondary to dry age-related macular degeneration (AMD). It is a one-time adeno-associated virus serotype 2 (AAV2)-based gene therapy designed to be administered as a single subretinal injection. The mechanism of action involves delivering a functional copy of the complement factor I (CFI) gene to retinal cells, aiming to increase CFI protein production and restore balance in an overactive complement system—a key driver in AMD progression. By increasing CFI levels, GT005 seeks to dampen complement-mediated inflammation and slow GA progression. The drug was originally developed by Gyroscope Therapeutics and later acquired by Novartis. Clinical development included Phase 1/2 and Phase 2 trials; however, Novartis discontinued its development for GA in September 2023.