MarketVIEW: New gene therapies – Wet AMD

Published February 2026

Wet age-related macular degeneration (wet AMD) is a progressive eye disease in which abnormal, fragile blood vessels grow under the macula and leak fluid or blood, leading to irreversible central vision loss if not treated. In many patients, untreated wet AMD can progress to legal blindness within months to a few years.

Wet AMD primarily affects older adults, with prevalence rising steeply with age. Recent analyses suggest that approximately 1 to 3% of individuals aged 75 years and older are affected, with hundreds of thousands of patients living with wet AMD in the US alone and substantial numbers of new diagnoses each year. As populations age, the overall burden of AMD is expected to increase further over the coming decades.

The current standard of care for wet AMD is based on intravitreal anti-VEGF therapies. These often require 6 to 10 injections in the first year, followed by ongoing injections in subsequent years, resulting in a long-term treatment burden for patients. Beyond the risk of complications from frequent injections, this regimen places significant pressure on healthcare services and on patients and caregivers who must attend regular, time-consuming clinic visits. Despite intensive treatment, an estimated 25 to 35% of patients with more aggressive disease have partial or inadequate responses and remain at risk of progressive, irreversible vision loss.

Given these limitations, there is a strong unmet need for more durable, disease-modifying therapies. A broad R&D pipeline is investigating new molecular targets and innovative delivery technologies, including gene therapies designed to drive long-term intraocular expression of anti-VEGF or VEGF-targeting molecules thereby reducing or eliminating the need for repeated injections. In Western markets, later-stage (pivotal Phase 3) AAV-based gene therapy candidates include 4D-150 (4D Molecular Therapeutics), ABBV-RGX-314 (sura-vec, Regenxbio/AbbVie) and ADVM-022 (ixo-vec, Adverum). These candidates have demonstrated meaningful reductions in annualized anti-VEGF injection burden in both treatment-naive and heavily pre-treated populations. If successful in Phase 3 and regulatory review, could start to reach major markets from around 2028 onwards. Other China-based contenders include Innostellar Biotherapeutics Co Ltd also in Phase 3 with their LX102 candidate.

This MarketVIEW product is a brand-new commercial opportunity assessment focused on the potential of emerging gene therapies for wet AMD through to 2040, across 10 major high-income developed markets*. It delivers a patient-based, interactive forecast model (.xls) and a comprehensive Executive presentation (~165 slides). All methodology and key assumptions are clearly documented, and the analysis incorporates four pricing case studies centred on the potential healthcare costs averted by gene therapy versus the current standard of care. The product also includes an up-to-date review of disease background, epidemiology, the current anti-VEGF market and the evolving R&D landscape. This product is ideally suited to organisations seeking a detailed, forward-looking, global forecast for this emerging therapeutic class. It is particularly relevant for pharmaceutical and biotech companies, investors, and other stakeholders evaluating the strategic and commercial potential of gene therapies in wet AMD.

THIS PRODUCT IS A SUMMARY PRESENTATION (~175 slides, .pdf) + interactive MS Excel Workbook(s) (>30 worksheets)

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