Presented at the Retina Society 59th Annual Scientific Meeting in Los Angeles, the data highlights that 60% of participants receiving the 1.0×10¹² GC/eye dose achieved a greater than 2-step improvement on the Diabetic Retinopathy Severity Scale (DRSS) by the three-year mark. Crucially, these patients required no additional treatment for the condition and experienced no vision-threatening events during the follow-up period.

The therapy, which utilizes suprachoroidal delivery, aims to inhibit the vascular endothelial growth factor (VEGF) pathway responsible for fluid accumulation and leaky blood vessels in the retina. Investigators noted that the safety profile remains consistent, with no new gene therapy-related signals or intraocular inflammation observed when paired with a short-course of prophylactic topical steroids. Given that current standards often force working-age patients into frequent clinical visits, the potential for a one-time, disease-modifying treatment represents a significant shift for the ophthalmology landscape. REGENXBIO is now evaluating this dosage level in the Phase IIb/III NAAVIGATE trial to further validate these outcomes.