Gene
therapies aim to deliver a therapeutic payload to specified tissues with underlying
protein deficiency. Since the 1990s, gene
therapies have been explored as potential treatments for
chronic conditions requiring lifetime care and medical management. Ocular gene
therapies target a range of ocular disorders, but
retinal diseases are of particular importance due to the prevalence of
retinal disease and the current treatment burden of such diseases on affected patients, as well as the challenge of properly delivering these
therapies to the target tissue. The purpose of this review is to provide an update on the most current data available for five different
retinal gene
therapies currently undergoing clinical trials for use against
age-related macular degeneration (AMD) and the development of novel delivery routes for the administration of such
therapies. Research has been performed and compiled from PubMed and the select authors of this manuscript on the treatment and effectiveness of five current
retinal gene
therapies: Luxturna, ADVM-022, RGX-314, GT-005, and HMR59. We present the available data of current clinical trials for the treatment of neovascular and dry
age-related macular degeneration with different AAV-based gene
therapies. We also present current research on the progress of developing novel routes of administration for ocular gene
therapies.
Retinal gene
therapies offer the potential for life-changing treatment for
chronic conditions like
age-related macular degeneration with a single administration. In doing so, gene
therapies change the landscape of treatment options for these
chronic conditions for both patient and provider.