Ocugen doses first patient in Phase 1/2 clinical trial evaluating safety, efficacy of OCU410ST for Stargardt disease


According to the company, OCU410ST uses an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene.

©Viacheslav Yakobchuk / adobe.stock.com

Image Credit: ©Viacheslav Yakobchuk / adobe.stock.com

Ocugen Inc announced the first patient has been dosed in its Phase 1/2 GARDian clinical trial for OCU410ST (AAV5-hRORA), a modifier gene therapy candidate being developed to treat Stargardt disease.

Stargardt disease is a genetic eye disorder that causes retinal degeneration and vision loss, and is the most common form of inherited macular degeneration. The progressive vision loss associated with Stargardt disease is caused by the degeneration of photoreceptor cells in the central portion of the retina called the macula.1

Shankar Musunuri, PhD, MBA, noted there is a significant unmet medical need for the approximate 35,000 patients in the US living with Stargardt disease.

“It is critical to our mission to develop innovative treatments for inherited retinal diseases and this milestone is an important step in bringing our novel modifier gene therapies to people who desperately need them,” Musunuri said in a news release.

The company also noted decreased central vision due to loss of photoreceptors in the macula is the hallmark of Stargardt disease. Some peripheral vision is usually preserved. The disease typically develops during childhood or adolescence, but the age of onset and rate of progression can vary. The retinal pigment epithelium (RPE), a layer of cells supporting photoreceptors, is also affected in people with Stargardt disease.

According to the company, the Phase 1/2 trial will assess the safety of unilateral subretinal administration of OCU410ST in subjects with Stargardt Disease and will be conducted in two phases. Phase 1 is a multicenter, open-label, dose ranging study. Phase 2 is a randomized, outcome accessor-blinded, dose-expansion study in which adult and pediatric subjects will be randomized in a 1:1:1 ratio to either one of two OCU410ST dose groups or to an untreated control group.1

Moreover, the company noted OCU410ST uses an AAV delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. It represents Ocugen’s modifier gene therapy approach, which is based on nuclear hormone receptor (NHR) RORA that regulates pathway links to Stargardt disease such as lipofuscin formation, oxidative stress, compliment formation, inflammation, and cell survival networks.1

“It is important and exciting to pursue novel therapies for untreatable blinding diseases,” Charles Wykoff, MD, PhD, director of Research, Retina Consultants of Texas, said in the news release. “Initiation of this trial program investigating a new mechanism of action for the treatment of Stargardt disease is inspiring and brings hope to patients and families.”


1. Ocugen Inc Announces First Patient Dosed in Phase 1/2 Clinical Trial Evaluating the Safety and Efficacy of OCU410ST-Modifier Gene Therapy-for Stargardt Disease. Ocugen website. Published November 10, 2023. Accessed November 11, 2023. https://ir.ocugen.com/news-releases/news-release-details/ocugen-inc-announces-first-patient-dosed-phase-12-clinical-0
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