RGX-314 - (ALTITUDE)

July 18, 2026: REGENXBIO Inc. (NASDAQ: RGNX) reported positive long-term follow-up data for investigational gene therapy surabgene lomparvovec (sura-vec, ABBV-RGX-314) in wet age-related macular degeneration (wet AMD) and diabetic retinopathy (DR), presented at the American Society of Retina Specialists (ASRS) 44th Annual Meeting.

Wet AMD (subretinal delivery): In Phase I/IIa Cohorts 3 and 4 at doses similar to ongoing pivotal trials, participants showed stable to improved visual acuity and meaningful reductions in anti-VEGF injection burden through 5 years; no new safety signals and no intraocular inflammation (IOI) in long-term follow-up. An external control analysis versus real-world anti-VEGF injections showed vision preservation at 1 year for sura-vec participants, with maintained/improved BCVA gains, less central retinal thickness fluctuation, and significantly fewer injections.

Upcoming catalyst: Topline data (with AbbVie) from the pivotal wet AMD trials ATMOSPHERE and ASCENT are expected in Q4 2026; both are randomized, active-controlled studies (vs ranibizumab and aflibercept) with primary non-inferiority endpoints on BCVA change at 54 weeks/1 year; >1,200 participants enrolled across >200 sites.

DR/NPDR (suprachoroidal delivery): Phase II ALTITUDE long-term follow-up at Dose Level 3 (1.0×10¹² GC/eye) showed durability through 2.5 years with short-course prophylactic topical steroids: 55% achieved >2-step DRSS improvement without additional treatment and 70% had no vision-threatening events; no IOI observed through 2.5 years (n=17) as of May 25, 2026. The dose matches the ongoing Phase IIb/III NAAVIGATE trial.