Ocugen Completes Dosing in OCU410ST Clinical Trial
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Ocugen Announces Completion of Dosing in Clinical Trial
Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on the discovery, development, and commercialization of novel gene and cell therapies, has completed dosing in the third cohort of its Phase 1/2 GARDian clinical trial for OCU410ST (AAV-hRORA), a modifier gene therapy candidate targeting Stargardt disease.
Understanding Stargardt Disease
Stargardt disease is a significant genetic eye disorder responsible for retinal degeneration and vision loss, affecting approximately 100,000 individuals in the U.S. and Europe. It is recognized as the most common form of inherited macular degeneration, typically resulting in progressive vision loss due to the deterioration of photoreceptors in the retina's central area, known as the macula. The condition often begins in childhood or adolescence, leading to varying degrees of vision impairment.
Significance of the GARDian Clinical Trial
The completion of dosing in the third cohort marks a pivotal moment in the trial’s progress. Dr. Huma Qamar, Chief Medical Officer at Ocugen, expressed confidence, stating, "With all patients dosed in cohort 3 (high dose), Phase 1 of the dose-escalation portion of the trial is complete. We will continue to advance the trial efficiently, working towards fulfilling an unmet medical need for Stargardt patients.”
Trial Structure and Administration
The GARDian clinical trial includes a multicenter, open-label, dose-ranging study comprised of three dose levels: low dose (3.75×1010 vg/mL), medium dose (7.5×1010 vg/mL), and high dose (2.25×1011 vg/mL). To date, three subjects have been administered a single subretinal injection utilizing the highest dose tested.
Potential of OCU410ST
Charles Wykoff, MD, PhD, the Director of Research at Retina Consultants of Texas and lead investigator for the study, has noted the substantial potential OCU410ST holds. He remarked, "OCU410ST is a novel modifier gene therapy that could provide a one-time treatment administered via subretinal injection within the operating room. The safety and tolerability profile of OCU410ST remains encouraging as the clinical trial progresses, guiding hope for those affected by Stargardt disease who currently lack FDA-approved treatment.”
Advancement in Gene Therapy Solutions
Ocugen remains steadfast in its mission to develop innovative gene therapy solutions aimed at addressing blindness and enhancing patient outcomes. The Company's commitment to researching and advancing such therapies underscores its resolve to provide lasting benefits.
About OCU410ST and Its Development
OCU410ST employs an AAV delivery mechanism to effectively transport the RORA (RAR Related Orphan Receptor A) gene. This therapeutic approach leverages the Nuclear Hormone Receptor (NHR) RORA to regulate pathways that are linked to Stargardt disease, including lipofuscin formation, oxidative stress, inflammation, and cell survival networks.
Company Overview
Ocugen, Inc. is committed to discovering, developing, and commercializing innovative gene and cell therapies that improve health and offer hope to patients around the world. The Company is recognized for its groundbreaking modifier gene therapy platform, which has the potential to address multiple retinal diseases with a single therapeutic solution, alongside advancing research in infectious diseases and orthopedic ailments.
Frequently Asked Questions
What is the GARDian clinical trial?
The GARDian clinical trial is a Phase 1/2 study examining the safety and efficacy of OCU410ST, a gene therapy for Stargardt disease.
Who is leading the clinical trial for OCU410ST?
The clinical trial is led by Charles Wykoff, MD, PhD, a recognized expert in retinal research.
What is Stargardt disease?
Stargardt disease is a genetic eye disorder leading to retinal deterioration and vision loss, primarily affecting the central vision.
How many subjects received treatment in the trial?
Three subjects have received the highest tested dose of OCU410ST in the clinical trial.
How is the OCU410ST therapy administered?
OCU410ST is administered via a single subretinal injection in an operating room setting.
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