A stem cell therapy for geographic atrophy improved vision in a phase Ib trial, and the first gene therapy for optic neuropathies was administered. Next-generation anti-VEGF agents are also extending treatment intervals in retinal vascular disease.
Recent studies in retinitis pigmentosa showed mixed results for gene and optogenetic therapies. UGX-201 and MCO-010 were associated with visual acuity improvements, while AAV8.hPDE6A showed no significant functional gains at 1 year.
Nicox SA received positive written feedback from a pre-NDA meeting with the FDA for NCX 470, a nitric oxide-donating bimatoprost eye drop for glaucoma. The NDA submission remains on track for summer 2026.