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

Eye 2026 Open access

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Authors: Khanani AM, Lejla Vajzovic, Benjamin Bakall, Venkata Ramana Murthy Chavali, Huma Qamar
DOI: 10.1038/s41433-025-04202-5
Cited by: 3
References: 5

Abstract

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.

Fields of Study & Subjects

Retinal Development and Disorders Connexins and lens biology Virus-based gene therapy research

Publication details

Journal / source
Eye
Publisher
Springer Science and Business Media LLC
Volume
40
Issue
3
Pages
429–430
Publication date
10 January 2026
Language
EN