A single, subretinal administration of ABBV-RGX-314 gene therapy was well-tolerated, with no clinically recognized immune response, in the treatment of neovascular (wet) age-related macular degeneration (nAMD).

Published in The Lancet, the two-year Phase 1/2a data indicate the novel approach may provide a pathway for sustained vascular endothelial growth factor (VEGF)-A suppression with a single dose, potentially safely maintaining vision and reducing treatment burden in nAMD.

In an interview with HCPLive, Peter A. Campochiaro, MD, the director of the retinal cell and molecular laboratory and professor of ophthalmology and neuroscience at the Wilmer Eye Institute, Johns Hopkins University School of Medicine, described the notability of a potential, one-time gene therapy for wet AMD, given the burden resulting from the current treatment landscape.

https://www.hcplive.com/view/peter-a-...

Interview Highlights

0:08 Importance of a ‘one-time’ gene therapy
2:27 Phase 1/2a trial results
3:52 Ongoing pivotal trials
4:51 RGX-314 timeline
6:00 Innovation in ophthalmology
7:19: Upcoming data at medical meetings

#ophthalmology #genetherapy