This year’s American Academy of Ophthalmology meeting in San Francisco, CA had significant data releases from pharmaceutical companies focused on the treatment of neovascular AMD with gene therapy. Data from Regenxbio and their 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 a 1.5 years of follow 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 is very encouraging, the procedure is still invasive and requires a surgical procedure rather than a simple intravitreal injection so it’s not likely to be the entry level treatment for patients. Will retina specialists commit all patients to the therapy if a small minority of patients do not require more than 2-3 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 angiopoetin-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.