Article

CTx001 for geographic atrophy: a gene therapy expressing soluble, truncated complement receptor 1 (mini-CR1)

2025-06-03

Abstract excerpt

Over-activation of the complement system is strongly associated with geographic atrophy (GA), a late-stage form of age-related macular degeneration (AMD) and major cause of blindness. Here we report the development of a candidate GA treatment that is a potent complement modifier (mini-CR1) that addresses consequences of complement over-activation including membrane attack complex (MAC) formation. Mini-CR1 acts a p...

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Literature Corpus work: 29d63c2e-a7a2-5cf9-9371-623b27be31f6
DOI: 10.1101/2025.06.03.657557

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