gms | German Medical Science

VI. International Symposium on AMD – Age-Related Macular Degeneration – Emerging Concepts – Exploring known and Identifying new Pathways

11. - 12.09.2015, Baden-Baden

Gene therapy for macular degeneration

Meeting Abstract

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  • Andrew Lotery - Southhampton

VI. International Symposium on AMD – Age-Related Macular Degeneration – Emerging Concepts – Exploring known and Identifying new Pathways. Baden-Baden, 11.-12.09.2015. Düsseldorf: German Medical Science GMS Publishing House; 2015. Doc15amd07

doi: 10.3205/15amd07, urn:nbn:de:0183-15amd074

Veröffentlicht: 1. Oktober 2015

© 2015 Lotery.
Dieser Artikel ist ein Open-Access-Artikel und steht unter den Lizenzbedingungen der Creative Commons Attribution 4.0 License (Namensnennung). Lizenz-Angaben siehe



Age related macular degeneration is a disease of epidemic proportions. Our current best therapy for the neovascular form of the disease is repeated intra-vitreal injections of anti-VEGF antibodies. However most ophthalmic services around the world struggle to cope with the number of injections required. Patients also find it inconvenient to have to have repeated intra-vitreal injections. There are also potential risks such as trauma to the eye or endophthalmitis. Further, early trials for the atrophic form of the disease suggest that intra-vitreal therapy with repeated injections of complement inhibitors may soon be necessary too.

It is unlikely that our current approach of repeated intra-vitreal injections for AMD therapy is sustainable. Therefore novel methods of delivering therapeutic antibodies to the eye are necessary. Gene therapy to deliver the necessary therapeutic proteins is an attractive option for several reasons. Firstly the eye is readily accessible to deliver a viral vector to the retina. Secondly a relatively small amount of viral vector is needed which makes manufacture easier. Expertise has been gained in delivering gene therapy to the eye through clinical trials for patients affected with Leber congenital amaurosis and choroideremia. Four trials of gene therapy for AMD have already occurred or are ongoing. In this presentation, I will discuss some of the challenges of delivering gene therapy to the macula as well as some of our early work assessing inhibitors of VEGF and PDGFR in Southampton. Potential new interventions such as inducible promoters will also be discussed.