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Ocular biomarker profiling after complement factor I gene therapy in geographic atrophy secondary to age-related macular degeneration

2024-06-13 ophthalmology Title + abstract only
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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 an in vitro complement activation assay. Design, setting and participantsBiomarker data were evaluated from 28 subjects in FOCUS, a Ph...

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