A novel modifier gene therapy to treat Stargardt disease: Phase 1 GARDian1 Trial Insights

Stargardt disease type 1 (STGD1) is an orphan inherited macular dystrophy affecting over 100,000 individuals in the US and EU, commonly presenting in childhood or adolescence [ 1 ]. Biallelic ABCA4 mutations lead to toxic bisretinoid lipofuscin accumulation, retinal pigment epithelium (RPE) atrophy, and progressive central vision loss [ 2 ]. With no approved treatments, STGD1 remains a significant unmet medical need. OCU410ST (AAV5-hRORA) is designed to modulate RPE and photoreceptor homeostasis, thereby preserving retinal integrity independent of ABCA4 genotype. This first-in-human phase 1 trial (NCT05956626) evaluated safety, tolerability, and exploratory efficacy of a single subretinal dose of OCU410ST in adults with advanced STGD1.