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Retinal Physician

Article | 2026 ASRS Meeting

5 Years of Gene Therapy: What the OPTIC Data Show

Szilárd Kiss, MD, FASRS, reviews 5-year findings from the OPTIC program in neovascular AMD patients with high anti-VEGF treatment needs, showing stable vision, substantially reduced treatment burden, and a favorable long-term safety profile.

By: Szilárd Kiss, MD
Retinal Physician July 22, 2026
At ASRS 2026, Szilárd Kiss, MD, FASRS, of Weill Cornell Medicine discusses 5-year follow-up from the OPTIC program evaluating Ixo-vec (Adverum Biotechnologies) for neovascular AMD. The ongoing phase 3 ARTEMIS and AQUARIUS trials are expected to report topline data next year.

The following transcript has been edited for clarity.

 

Hi, this is Szilárd Kiss, MD, FASRS, here at the ASRS meeting in Montreal. I practice at Weill Cornell Medicine in New York City, and I presented the 5-year data from the OPTIC trial.

Anti-VEGF gene therapy is something that is emerging as a long-term treatment for patients who have a chronic disease. Anti-VEGF works in patients with age-related macular degeneration (AMD), but the paradigm of episodic treatment has really not changed. So it's really about extending that treatment paradigm beyond the monthly, every-2-month, or every-3-month injections.

Ixo-vec (ixoberogene soroparvovec; formerly ADVM-022, Adverum Biotechnologies) is an investigational gene therapy that’s delivered by an intravitreal injection. It provides long-term aflibercept expression in the eye. The eye actually is turned into a biofactory to deliver the anti-VEGF that controls the chronic disease of wet AMD.

At ASRS, we presented the 5-year data from the OPTIC trial, which is a phase 1 trial enrolling patients with a high anti-VEGF need who had required approximately 10 injections annualized and had been diagnosed with AMD for many years.

Patients received 1 injection of Ixo-vec and were followed out to 5 years. The 5-year data have been consistent with all the data that have been presented previously. We went from an average of 10 injections annualized to less than 2 at each year during follow-up, a remarkable feat considering the high anti-VEGF need.

Patients also maintained vision despite not needing an intravitreal rescue injection, which was consistent with what they had been needing before. The anatomic and visual acuity results were really reliant on the aflibercept expression, which remarkably remained constant throughout the 5 years.

The 5-year data not only showed efficacy, but the safety profile also looked very favorable. No new serious adverse events were noted. No vasculitis, no choroiditis, and no vascular occlusion were seen out to 5 years.

When we think about the revolution that was anti-VEGF therapy for patients with macular degeneration, we now think about a way to deliver it that’s not episodic, that doesn’t require repeated injections for the lifetime of the patient.

So that’s why we’re all excited about the OPTIC trial. Now, as a phase 1 trial, it really is hypothesis-generating. So the phase 3 trials, ARTEMIS and AQUARIUS, are ongoing. ARTEMIS and AQUARIUS are enrolling both treatment-naïve and treatment-experienced patients with wet AMD, and there’s going to be a direct comparison to aflibercept 2 mg (Eylea; Regeneron) given via bolus intravitreal injections every 8 weeks.

We await the results of those phase 3 trials and hope that the results are consistent with what we’ve seen in the 5-year OPTIC data.

This is Szilárd Kiss, MD, FASRS, from the ASRS meeting in Montreal. RP