Ocular biomarker profiling after complement factor I gene therapy in geographic atrophy secondary to age-related macular degeneration
Thomas M Hallam,
Emanuela Gardenal,
Fraser McBlane
et al.
Abstract:ObjectiveComplement biomarker analysis in ocular fluid samples from subjects with geographic atrophy (GA) in a Phase I/II clinical trial of subretinal AAV2 complement factor I (CFI; FI) gene therapy, PPY988 (formerly GT005), to understand target pharmacokinetics/pharmacodynamics. Clinical findings were subsequently utilized to investigate the therapeutic dose in anin vitrocomplement activation assay.Design, setting and participantsBiomarker data were evaluated from 28 subjects in FOCUS, a Phase I/II clinical t… Show more
Set email alert for when this publication receives citations?
scite is a Brooklyn-based organization that helps researchers better discover and understand research articles through Smart Citations–citations that display the context of the citation and describe whether the article provides supporting or contrasting evidence. scite is used by students and researchers from around the world and is funded in part by the National Science Foundation and the National Institute on Drug Abuse of the National Institutes of Health.