Seeing the Unseen | Breakthroughs in Back-of-the-Eye Therapies

Rishi Singh MD moderated a wide-ranging ophthalmology discussion with Lance Baldo MD, Jason Comander MD, PhD, Maha Radhakrishnan MD, Richard Small and Cheng Zhang on how gene therapy, cell-based approaches, genetics and better clinical trial design are changing treatment for retinal disease.

Baldo discussed positive Phase III gene therapy results in X-linked retinitis pigmentosa and the lessons behind them. Better vector and capsid design mattered, but so did patient selection, surgical delivery and choosing endpoints that capture changes patients actually experience. Comander put that progress into perspective through patients with inherited retinal disease who are now seeing functional improvements that would once have been difficult to imagine.

Zhang described how Character Bio is combining genetic, imaging and longitudinal clinical data from thousands of patients with age-related macular degeneration to understand why disease progresses differently across patients. Small discussed the path from encapsulated cell technology to commercialization, while Radhakrishnan brought the investor perspective, emphasizing the importance of clearly defined patient populations, meaningful endpoints and differentiated benefits.

Looking ahead, the panel pointed to improved gene therapy, durable delivery, cell-based treatments and better trial design. Zhang offered another possibility: the biggest advance may come from treating retinal disease earlier, before irreversible vision loss has occurred.