Article

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

2024-06-13

Abstract excerpt

<h4>Objective</h4> Complement 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 ass...

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Literature Corpus work: 92af515f-5a51-5d61-885b-4ad87c4fdbd7
DOI: 10.1101/2024.06.12.24308796

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