MALVERN, PA — Ocugen, Inc. (NASDAQ: OCGN) has announced a significant development in its efforts to treat Stargardt disease, as the Data and Safety Monitoring Board (DSMB) has greenlit the enrollment for the second phase of their Phase 1/2 clinical trial for OCU410ST. This innovative gene therapy candidate aims to address a condition with no current FDA-approved treatments, offering new hope to patients.
The decision to proceed follows favorable safety data from the first phase, in which nine patients received varying doses of OCU410ST through subretinal injection. The DSMB confirmed that the high dose was well-tolerated, with no serious adverse events reported, marking an important milestone in the therapy’s development.
Stargardt disease is the most prevalent form of inherited macular degeneration, characterized by progressive central vision loss starting in childhood or adolescence. It affects approximately 100,000 people across the U.S. and Europe, yet remains without an approved treatment. The disease results from the degeneration of photoreceptor cells in the retina’s central region, the macula, often preserving some peripheral vision.
OCU410ST represents a novel gene therapy approach, utilizing an AAV delivery system to introduce the RORA gene to the retina. This method aims to modify disease pathways related to Stargardt disease, addressing factors such as oxidative stress and inflammation, crucial for cell survival.
Dr. Charles Wykoff, a leading study investigator, expressed optimism about the trial’s progress, emphasizing the importance of moving forward given the promising safety profile observed so far. Ocugen’s Chief Medical Officer, Dr. Huma Qamar, echoed this sentiment, highlighting the potential impact of OCU410ST as a one-time treatment.
The GARDian clinical trial is being conducted at six prominent retinal centers across the U.S., aiming to fulfill the unmet medical needs of Stargardt patients and pave the way for future innovations in retinal disease treatment.
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