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Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study by Peter A Campochiaro; Robert Avery; David M Brown; Jeffrey S Heier; Allen C Ho; Stephen M Huddleston; Glenn J Jaffe; Arshad M Khanani; Stephen Pakola; Dante J Pieramici; Charles C Wykoff; Sherri Van Everen is a Social Sciences article available to read on EtoBox.
What is Gene therapy for neovascular age-related macular degeneration by subretinal delivery of RGX-314: a phase 1/2a dose-escalation study about?
Background Frequent anti-vascular endothelial growth factor A (VEGF-A) injections reduce the risk of rapid and severe vision loss in patients with neovascular age-related macular degeneration (nAMD); however, due to undertreatment, many patients lose vision over time. New treatments that provide sustained suppression of VEGF-A are needed. RGX-314 (currently known as ABBV-RGX-314) is an adeno-associated virus serotype 8 vector that expresses an anti-VEGF-A antigen-binding fragment, which provides potential for continuous VEGF-A suppression after a single subretinal injection. We report results on the safety and efficacy of subretinal injection of RGX-314 in patients with nAMD. ## Methods For this open-label, multiple-cohort, multicentre, phase 1/2a, dose-escalation study conducted at eight sites in the USA, we enrolled participants with nAMD aged 50-89 years who had previously been treated with anti-VEGF injections into five cohorts (with five different doses of RGX-314). To be eligible, participants had to have macular neovascularisation secondary to nAMD with subretinal or intraretinal fluid in the centre subfield, be pseudophakic (after cataract removal), and have a best-correcte
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- Author
- Peter A Campochiaro; Robert Avery; David M Brown; Jeffrey S Heier; Allen C Ho; Stephen M Huddleston; Glenn J Jaffe; Arshad M Khanani; Stephen Pakola; Dante J Pieramici; Charles C Wykoff; Sherri Van Everen
- Publisher
- Elsevier BV
- Published
- 2024
- Language
- EN
- Field
- Social Sciences