Ocugen has announced the completion of subject dosing in the first cohort of its Phase I/II GARDian clinical trial of OCU410ST (AAV5-hRORA), a modifier gene therapy candidate being developed to treat Stargardt disease.
This marks a significant milestone in the study of the genetic eye disorder that leads to retinal degeneration and vision loss.
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OCU410ST employs an adeno-associated virus (AAV) delivery platform to deliver the RAR Related Orphan Receptor A (RORA) gene to the retina.
The treatment is part of modifier gene therapy strategy of Ocugen, which targets the Nuclear Hormone Receptor (NHR) RORA.
This receptor is involved in regulating pathways linked to Stargardt disease, including formation of lipofuscin, oxidative stress, complement formation, inflammation, and cell survival networks.
The GARDian trial will be conducted at up to ten retinal surgery centres in the US.
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By GlobalDataIn the first cohort of the trial, three participants received a 200L single subretinal administration of low dose OCU410ST.
The two-phase trial is designed to evaluate the safety of unilateral subretinal administration of OCU410ST in Stargardt disease patients.
Phase I portion is an open-label, multicentre, dose-ranging study assessing three dose levels while Phase II will be an outcome accessor-blinded, randomised, dose-expansion study in adult and paediatric patients.
Participants in Phase II will be randomised in a 1:1:1 ratio to one of two OCU410ST dose groups or an untreated control group.
Ocugen R&D chief scientific officer and head Dr Arun Upadhyay said: “This is an important clinical milestone for our first-in-class, potential one-time therapy for the treatment of Stargardt disease.
“Although Stargardt is one of the most common inherited retinal diseases, there remains no treatment option to address this condition. OCU410ST provides hope to these patients who may eventually lose their vision.”
Last November, the company dosed the first patient in its Phase I/II GARDian clinical study of OCU410ST (AAV5-hRORA) for Stargardt disease.
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