Anticipating the Ophthalmology Gene Therapy Pipeline


From curative stem cell approaches to in-clinic subretinal injections, Arshad Khanani, MD, MA, navigates what the future drug class may entail.

Gene and stem cell therapy research is beginning to gain stride in ophthalmology, as various agents for an array of indications have begun reporting phase 2/3 outcomes with benefits ranging from reduced treatment burden to reversal of chronic vision loss.

One such product, single-injection suprachoroidal-administered agent RGX-314 from Regenxbio, reported promising phase 2 tolerability and efficacy findings at the Association for Research in Vision and Ophthalmology (ARVO) 2022 Meeting this week. Though it’s not the furthest advanced agent in cell- or gene-based therapy, it represents the continual exploration and evidencing of unique means of altering ophthalmic disease through innovation.

In the second segment of an interview with HCPLive during ARVO 2022, RGX-314 investigator Arshad Khanani, MD, MA, director of clinical research at Sierra Eye Associates, discussed the fields of ophthalmic research and care already being formed by the anticipated arrival of gene and cell therapies.

“The bottom line is the long-term data for efficacy and safety will dictate what patients are good for gene therapy,” Khanani said. “For suprachoroidal gene therapy, because it’s an in-clinic procedure, it’s something that can be done anywhere in the world for the clinic. I think it’s a space that needs to be looked at more, but to me, it will depend on the patient’s need.”

Khanani also discussed the potential of gene therapies for vision loss, stressing that benefit can range from curative to simply more efficacious than standard care. The rate of patients with age-related macular degeneration (AMD) or other forms of vision loss who may benefit from products in investigation remains to be seen.

“I don’t have a number for you—that’s something everybody asks, but I will say that the majority of patients who are sitting between every 1-2 months of treatment should be able to decrease their treatment burden with this approach,” Khanani said regarding RGX-314.

Moving forward, Khanani anticipates it will be another 3 years until the most advanced subretinal gene therapy programs may complete pivotal phase 3 assessments. The field will require more industry- and organizational-led guidance in defining ideal patient populations, but Khanani sees this as no worrisome challenge. After all, everyone is learning more about these agents together.

“It’s a paradigm shift, and we’re coming closer to curing a subset of patients,” Khanani said. “But it’s not easy—innovation with a new paradigm shift is always challenging. The key for me is seeing that different companies and leaders in gene therapy…everybody is coming together so we can have a treatment that will benefit our patients.”

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