Ocugen (NASDAQ:OCGN) has announced that the Data and Safety Monitoring Board (DSMB) has approved enrollment for the second phase of its Phase 1/2 clinical trial of OCU410ST GARDian for Stargardt disease.
The GARDian clinical trial is underway at six leading retinal surgery centers across the U.S. OCU410ST (AAV5-hRORA) is a novel modifier gene therapy candidate being developed for Stargardt disease. Stargardt disease is the most common form of inherited macular dystrophy. Symptoms of bilateral central vision loss typically begin in childhood and gradually worsen over time.
A lead study investigator, Charles Wykoff, MD, PhD, director of research, Retina Consultants of Texas and Retina Consultants of America, said, “The DSMB has recommended moving forward with Phase 2 enrollment, as safety data indicate that OCU410ST appears to be safe and well-tolerated to date. The safety and tolerability profile of OCU410ST remains encouraging as the clinical trial has progressed and continues to bring hope to patients with Stargardt disease, which still has no FDA-approved treatments.”
“We are enthusiastic about the potential of OCU410ST to be the first one-time novel modifier gene therapy for Stargardt disease,” said Huma Qamar, MD, MPH, CMO of Ocugen. “We are encouraged by the prospect of addressing a substantial unmet medical need for the estimated 100,000 Stargardt patients in the U.S. and Europe.”
