The FDA has approved the initiation of the Opti-GAIN phase 1/2 clinical trial for CTx001, an adeno-associated virus (AAV)-based gene therapy targeting geographic atrophy (GA) secondary to age-related macular degeneration (AMD). Developed by Complement Therapeutics, CTx001 delivers a truncated version of complement receptor 1 (mini-CR1) to modulate both the classical and alternative complement pathways. The trial aims to evaluate the safety, tolerability, and preliminary efficacy of this novel therapy.
Source: Retinal Physician