Key takeaways from the RGX-314 (Regenxbio) phase I/IIa clinical trial indicate that the novel gene therapy is a promising approach for treating wet age-related macular degeneration (AMD). The messages were presented by Jeffrey Heier, MD, at the American Academy of Ophthalmology 2019 Retina Subspecialty Day meeting.
The investigational agent is designed to deliver a gene encoding for an anti-VEGF fab protein and uses a novel viral vector (adeno-associated virus 8; AAV8). The phase I/IIa dose-escalating trial enrolled 42 patients across five dose cohorts.
Eligible patients had severe wet AMD requiring frequent anti-VEGF injections and were required to have demonstrated a level of response to anti-VEGF therapy. They received a single subretinal injection of RGX-314 and were followed monthly for safety and need for rescue therapy.
Jeffrey Heier, MD
Dr. Heier is scientific advisor to Regenxbio.