This year’s American Academy of Ophthalmology meeting in San Francisco had significant data releases from pharmaceutical companies focused on the treatment of neovascular age-related macular degeneration with gene therapy.
Regenxbio announced data about its lead compound RGX-314 delivered via subretinal delivery. The study tested five different doses in 42 patients previously treated with anti-VEGF agents. Overall, the subretinal delivery was well tolerated without significant intraocular inflammation. There was a dose-dependent increase of the protein in aqueous samples across the five arms. The data presented showed six patients with 1.5 years of follow-up and an impressive +9 letter visual gain from baseline. Three of the six patients were injection-free at 18 months, and the number of patients who were injection-free increased with the amount of protein delivered in cohort 4 and 5.
So, is gene therapy ready for prime time? While the data are very encouraging, the procedure is still invasive and requires surgery rather than a simple intravitreal injection, so it’s not likely to be the entry-level treatment. Will retina specialists commit all patients to the therapy if a small minority of patients do not require more than two to three injections over 2 years? That still remains to be determined. Lastly, with the advent of different mechanisms of action like pan-VEGF agents and anti-angiopoietin 2 agents, this will likely be step therapy for those patients with persistent or recurrent disease.
Nonetheless, there are really promising results on a platform that appears to be safe and effective in the management of active neovascular AMD.
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Disclosure: Singh reports he is a consultant to Zeiss, Novartis, Regeneron, Genentech and Alcon and receives grant support from Apellis and Graybug.