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Implications of Gene Therapy in Retina, With Christina Y. Weng, MD
Gene therapy could revolutionize retinal disease treatment, promising consistent dosing, fewer injections, and improved patient outcomes, as highlighted by expert Christina Y. Weng.
In a field marked by rapid innovation, gene therapy is emerging as one of the most promising advances in retinal disease treatment. At the 2025 American Society of Cataract and Refractive Surgery (ASCRS) Annual Meeting, Christina Y. Weng, MD, MBA, a professor and the Alice R. McPherson Retina Research Foundation Chair in Ophthalmology at Baylor College of Medicine, offered perspective on the transformative potential of these therapies and what they could mean for both patients and providers.
Weng outlined how these gene therapies have the potential to raise the bar for treatment durability to new levels, highlighting three key benefits these therapies could offer: more consistent dosing, fewer injections, and greater overall efficiency in disease management.
“The first one is that we know that most patients are under-treated with anti-VEGF in the real world, and consequently, they suffer from suboptimal visual outcomes,” Weng told HCPLive. “By providing constant dosing of anti-VEGF, gene therapy might help counteract this challenge.”
Weng also emphasized the value of steady dosing, noting that current treatment methods center on bolus dosing, which often causes recurrent anatomic fluctuations and could negatively impact outcomes. Gene therapy could provide a steadier level of dosing, which could render benefits not yet fully realized, as well as lead to fewer burdensome injections.
“Those injections are not only associated with psychological burden, financial burden, but there's also substantial socioeconomic costs,” Weng told HCPLive. “Many of these patients come in each time, bringing in an accompaniment or arranging transportation, etc. All those things can also be reduced with the reduced treatment burden that gene therapy might bring.”
As several gene therapy programs reach late-stage trials, Weng is closely watching the results to see if efficacy signals seen in earlier trials are replicated with larger cohorts. She cited the importance of safety, with reported issues like intraocular inflammation and pigmentary changes becoming important factors in regulatory and clinical decision-making.
“All of the programs I've mentioned have found their gene therapy candidates to be well tolerated, but many have also been associated with adverse events such as intraocular inflammation, pigmentary changes, and episcleritis,” Weng told HCPLive. “The Phase 3 studies will provide important insights on safety, and they're also going to pressure test some of the prophylactic strategies being evaluated.
Looking ahead, Weng noted gene therapy could drive a paradigm shift in disease management,” particularly when paired with advances like home optical coherence tomography (OCT). However, questions remain about how these new options will fit into treatment algorithms.
“We’ve entered the era of gene therapy, not just for rare diseases, but also for some of our most common,” Weng told HCPLive. “Several programs are in late stages, and those results will be key in providing safety and efficacy data that will determine the viability of these gene therapy candidates. If they do emerge on the market, they have the potential of significantly reducing treatment burden and potentially improving visual outcomes for our patients.”
Weng reports relevant disclosures with Alcon, AbbVie, Apellis, Astellas/Iveric Bio, Genentech, Regeneron, and others.
