Rishi Singh, MD’s, blog takes an evidence-based approach to hot topics in eye care and examines their clinical relevance.

BLOG: Gene therapy promising for treatment of neovascular AMD

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.