Phase 3 Ixo-Vec Program Advances

The ARTEMIS and AQUARIUS studies will evaluate the intravitreal gene therapy for neovascular AMD, after earlier trials demonstrated durable treatment effects.

By: Jim Gallagher, senior managing editor

Retinal Physician May 19, 2026

Clinical Report: Phase 3 Ixo-Vec Program Advances

Overview

The phase 3 development program for ixoberogene soroparvovec (ixo-vec) in neovascular age-related macular degeneration (nAMD) is progressing, with the ARTEMIS and AQUARIUS studies evaluating its efficacy and safety. Early trials indicate that a single injection may provide sustained aflibercept expression and significantly reduce treatment burden.

Background

Neovascular age-related macular degeneration (nAMD) is a leading cause of vision loss in older adults, necessitating frequent anti-VEGF injections. Current treatment regimens often lead to variability in retinal thickness and poorer long-term outcomes. The development of durable therapies like gene therapy could address these challenges and improve patient care.

Data Highlights

No numerical data available in the source material.

Key Findings

The ARTEMIS and AQUARIUS studies are assessing the efficacy of ixo-vec in nAMD patients.
Previous trials (OPTIC and LUNA) demonstrated sustained aflibercept expression for 4-5 years post-injection.
Patients in OPTIC averaged fewer than 2 injections annually after treatment, down from approximately 10 prior to enrollment.
Visual acuity and central subfield thickness remained stable across both OPTIC and LUNA studies.
In LUNA, treatment burden decreased by nearly 90% during the first 2 years of follow-up.
No significant safety concerns were reported, with mild to moderate inflammation manageable with corticosteroids.

Clinical Implications

The findings suggest that ixo-vec could significantly reduce the frequency of injections required for nAMD patients, addressing a major unmet need in the treatment landscape. Clinicians may consider gene therapy as a viable long-term solution for patients with high treatment burdens.

Conclusion

The ongoing phase 3 studies of ixo-vec hold promise for transforming the management of nAMD by potentially offering a durable treatment option that reduces the need for frequent injections.

PentaVision

Retinal Physician