Ocugen has dosed the first subject in its Phase II/III GARDian3 trial of modifier gene therapy candidate, OCU410ST (AAV5-hRORA), targeting Stargardt disease (ABCA4-associated retinopathies).
This follows positive findings from the preceding Phase I GARDian study, which indicated a 48% slowdown in lesion growth at 12 months follow-up period in evaluable treated eyes against untreated eyes and showed improved visual acuity in treated eyes.
Go deeper with GlobalData
Discover B2B Marketing That Performs
Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.
The current study aims to enrol 51 Stargardt disease patients, with 34 set to receive a single subretinal injection of OCU410ST, which contains 200μL with a concentration of 1.5×10¹¹ vector genomes per millilitre, into the eye that has lesser visual acuity.
The remaining 17 subjects will be placed in a control group that will not receive the treatment.
GARDian3’s primary goal is to assess the reduction in atrophic lesion size, with key secondary endpoints focusing on best corrected visual acuity (BCVA) improvements and low-luminance visual acuity (LLVA) improvements.
A one-year follow-up data of the trial will support Ocugen’s future biologics licence application (BLA).
US Tariffs are shifting - will you react or anticipate?
Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis.
By GlobalDataOcugen co-founder, CEO and chairman Dr Shankar Musunuri said: “Dosing the first patient is an especially significant milestone and brings us closer to our goal of addressing the unmet medical need that exists for all Stargardt patients—100,000 in the US and Europe and one million worldwide.
“Progressing our second modifier gene therapy candidate into a registration clinical trial is a pivotal step in potentially providing a one-time therapy for life for the millions of patients affected by inherited retinal diseases.”
Ocugen’s strategic plan includes filing three BLAs over the next three years, with the submission for OCU410ST anticipated in 2027.
The company noted that the therapy leverages an adeno-associated virus (AAV) delivery platform for retinal delivery of the RAR-Related Orphan Receptor A (RORA) gene.
A genetic eye disorder, Stargardt disease, is the most prevalent form of inherited macular degeneration, leading to vision loss and retinal degeneration.
In March 2025, Ocugen obtained the Data and Safety Monitoring Board (DSMB) approval to start dosing the second cohort of individuals in a Phase I trial of OCU200, a fusion protein for treating diabetic macular oedema.
Cell & Gene therapy coverage on Clinical Trials Arena is supported by Cytiva.
Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.
