CTx001

CTx001 is an investigational adeno-associated virus serotype 2 (AAV2)-based gene therapy developed by Complement Therapeutics for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The therapy is designed to deliver a gene encoding mini-complement receptor 1 (mini-CR1), which is a truncated and secreted form of the naturally occurring complement receptor 1 (CR1). Once expressed in the retinal cells following a single subretinal injection, the mini-CR1 protein acts as a potent cofactor for factor I-mediated proteolysis of C3b and C4b. This activity effectively modulates both the classical and alternative complement pathways locally within the retina, inhibiting complement overactivation and preventing the formation of the membrane attack complex (MAC). By addressing the dysregulation of the complement system—specifically targeting the roles of factor H-related proteins—CTx001 aims to provide a durable, long-term therapeutic effect to slow or prevent the progression of vision loss in patients with late-stage dry AMD.

Other names

mini-CR1 gene therapymini-CR-1 gene therapymini-CR 1 gene therapy