Lyna Azzouz, MD
PGY4 Resident
Byers Eye Institute, Stanford University
Late-breaking data presented at AAO 2025 highlighted emerging gene-independent treatment strategies for inherited retinal diseases.
Allen C. Ho, MD (Wills Eye Hospital, Philadelphia) presented the three-year extension results from the RESTORE trial evaluating MCO-010, an investigational intravitreal optogenetic therapy for severe retinitis pigmentosa. The treatment delivers a light-sensitive protein to surviving retinal bipolar cells, aiming to restore light responsiveness even in the absence of functional photoreceptors. In long-term follow-up, a meaningful proportion of treated eyes demonstrated sustained functional improvement over the three year follow up period. There was a good safety profile and no serious inflammatory events. As a mutation-agnostic approach, MCO-010 may hold a potential across a broad range of retinal degenerations.
In a complementary strategy targeting Stargardt disease, Michel Michaelides, MD (Moorfields Eye Hospital, London) presented the interim analysis of the Phase 3 DRAGON trial investigating Tinlarebant, an oral RBP4 antagonist designed to reduce accumulation of toxic bisretinoids associated with ABCA4 dysfunction. Visual function remained stable through the first treatment year, and treatment-related effects such as delayed dark adaptation were consistent with the drug’s mechanism and generally manageable. Tinlarebant has received FDA Breakthrough Therapy designation, and global regulatory discussions are ongoing.
While long-term outcomes and real-world applicability will require further study, both presentations sought to advance the broader goal of developing mutation-independent therapies for patients who currently have limited or no treatment options.