MALVERN, PA — In a promising development for the treatment of Stargardt disease, Ocugen, Inc. (NASDAQ: OCGN), a biotechnology company based in Chester County, Pennsylvania, has announced the completion of dosing in the first cohort of its Phase 1/2 GARDian clinical trial for OCU410ST (AAV5-hRORA). This modifier gene therapy candidate is being developed to treat Stargardt disease, a rare inherited retinal disorder that affects approximately 35,000 people in the U.S.
“This is an important clinical milestone for our first-in-class, potential one-time therapy for the treatment of Stargardt disease,” said Dr. Arun Upadhyay, Chief Scientific Officer and Head of R&D at Ocugen. The disease currently has no viable treatment options, making it a pressing concern in the medical community.
Stargardt disease is characterized by progressive vision loss due to the degeneration of photoreceptor cells in the macula, the central portion of the retina responsible for central vision. While some peripheral vision is typically preserved, decreased central vision is a hallmark of the condition, which generally develops during childhood or adolescence.
OCU410ST utilizes an adeno-associated virus (AAV) delivery platform for the retinal delivery of the RORA (RAR Related Orphan Receptor A) gene. This represents Ocugen’s unique approach to gene therapy, which is based on Nuclear Hormone Receptor (NHR) RORA that regulates pathway links to Stargardt disease, including lipofuscin formation, oxidative stress, compliment formation, inflammation, and cell survival networks.
The GARDian clinical trial is being conducted across up to 10 leading retinal surgery centers in the U.S. In the first cohort, three subjects received 200L single subretinal administration of the low dose (3.75×1010 vg/mL) of OCU410ST.
The trial will be conducted in two phases to assess the safety of unilateral subretinal administration of OCU410ST in subjects with Stargardt disease. Phase 1 is a multicenter, open-label, dose-ranging study comprising three dose levels. Phase 2 is a randomized, outcome accessor-blinded, dose-expansion study where adult and pediatric subjects will be randomly assigned to either one of two OCU410ST dose groups or to an untreated control group.
“I am very pleased to participate in this study and to offer a novel modifier gene therapy option to my patients,” said Dr. Benjamin Bakall, Director of Clinical Research at Associated Retina Consultants and Clinical Assistant Professor at the University of Arizona, College of Medicine—Phoenix. “Until now, we have not had any effective treatment that can prevent the vision loss in patients with Stargardt disease. Now, I believe that this approach can offer a new therapeutic option to address the disease itself.”
Ocugen’s mission is to develop and commercialize novel gene and cell therapies, biologics, and vaccines that improve health and provide hope for patients worldwide. The progress made in the GARDian clinical trial represents a significant step forward in the company’s mission and offers renewed hope for those affected by Stargardt disease. As the trial proceeds into its next phase, industry watchers will be eagerly awaiting updates from Ocugen on this potentially game-changing therapy.
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