REGENXBIO Launches Phase IIb/III Trial for Gene Therapy Sura-vec

The NAAVIGATE study is a multicenter, randomized, masked, and sham-controlled trial designed to assess the safety and efficacy of the therapy using suprachoroidal delivery. The study focuses on patients with non-proliferative diabetic retinopathy who do not have center-involved diabetic macular edema. Participants will receive a 1.0x10^12 genome copies per eye dose, supported by a short course of topical steroids. The primary objective is to achieve a greater than 2-step improvement on the diabetic retinopathy severity scale within one year.

Dr. Arshad Khanani, Director of Clinical Research at Sierra Eye Associates, noted that the current standard of care often relies on repeated, ongoing interventions that can lead to undertreatment. By providing a one-time, in-office solution, the therapy aims to make early intervention more practical and scalable. REGENXBIO plans to enroll approximately 135 participants in the United States for the Phase IIb portion of the trial.

Supporting this expansion, the company intends to share updated data from its previous ALTITUDE trial at the upcoming American Society of Retina Specialists meeting in July 2026. Previous two-year results from that study indicated a durable efficacy profile with no observed intraocular inflammation, reinforcing the potential for gene therapy to address chronic retinal conditions before permanent vision loss occurs.