Wet AMD gene therapy: Success depends on a comprehensive development strategy

Wet age-related macular degeneration (AMD) is a crowded, competitive therapeutic market dominated by highly effective, well-established, and increasingly affordable anti-vascular endothelial growth factor (VEGF) injectables. New wet AMD therapies will compete against a range of products, from biosimilars for ranibizumab (Lucentis), aflibercept (Eylea), and compounded bevacizumab (Avastin) to branded drugs like 8 mg aflibercept (Eylea HD), faricimab (Vabysmo), and the continuous delivery ocular implant ranibizumab injection (Susvimo)—initially approved by the FDA in 2021 and under review by the EMA as of September 3, 2025.¹  

Existing treatments have set a high bar for efficacy, improving or stabilizing vision for more than 90 percent of patients in clinical trials.^2,3,4,5  Meanwhile, retina specialists have built profitable practice models around their use. However, these products necessitate frequent eye injections (ranging from every 4 to 24 weeks), and real-world results have not equaled those of clinical trials due to factors such as fewer injections resulting in undertreatment.^6,7

Retinal gene therapies offer the promise of a one-time, long-lasting treatment for wet AMD. However, for a wet AMD gene therapy to succeed commercially, drug developers must present a clear and compelling value proposition that aligns with the sometimes-different priorities of key stakeholders: regulators, clinicians, patients, and payers.