Work

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

Year: 2026

Type: article

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.

Source: Eye

DOI: 10.1038/s41433-025-04202-5

Authors: Arshad M. Khanani, Lejla Vajzovic, Benjamin Bakall, Venkata Ramana Murthy Chavali, Huma Qamar

Institutions: University of Nevada, Reno, Sierra Eye Associates, Duke University, University of Arizona College of Medicine- Phoenix, Ocugen, Inc. (United States)

Language: English

Topic: Retinal Development and Disorders

Subfield: Ophthalmology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: hybrid

Estimated APC paid: $3,590