Designing Wet AMD Gene Therapies: Strategies That Drive Success

By Bharti Arya, Therapeutic area lead, Ophthalmology; Simona Stankeviciute, M.D., M.Sc., Vice President, Technical, Regulatory Consulting; Patrick Symmnds, PharmD, Health Advances

Gene therapy is redefining what’s possible in retinal disease—and momentum is building fast. For conditions like wet age‑related macular degeneration (AMD), these therapies offer the potential to move beyond traditional treatment cycles and deliver meaningful, lasting impact. But unlocking that promise requires sponsors to navigate a development path unlike anything in conventional ophthalmology programs.

This article spotlights the essential considerations for advancing retinal gene therapies with speed and confidence. It digs into the clinical design choices that matter most—precise patient selection, data‑rich dosing strategies, and endpoints that capture both anatomical change and real‑world visual improvement. It also addresses the operational realities unique to ophthalmic studies, from surgical delivery to advanced imaging, along with the regulatory expectations shaping safety, durability, and long‑term follow‑up.

By mastering these elements early, sponsors can build high‑performing programs that cut through complexity, reduce development risk, and accelerate transformative gene therapies to patients who need them most.