CAMBRIDGE, Mass.– BlueRock Therapeutics has dosed the first patient in a clinical trial of OpCT-001, a stem cell-based therapy developed to treat inherited retinal conditions that cause progressive and irreversible vision loss.
The therapy, derived from induced pluripotent stem cells (iPSCs), is the first of its kind to enter human trials for primary photoreceptor diseases—a group of rare genetic disorders affecting the retina, including retinitis pigmentosa and cone-rod dystrophy. These conditions lead to the deterioration of photoreceptor cells, often resulting in blindness in both children and adults.
The ongoing Phase 1/2a trial, called CLARICO, will enroll up to 54 adult patients across multiple sites. The first phase will assess the safety and tolerability of OpCT-001 using a dose-escalation design. The second phase will collect additional safety data and evaluate the therapy’s potential to improve visual function and restore retinal structure.
“This trial marks a major step forward for OpCT-001 and for cell therapy in vision restoration,” said Dr. Amit Rakhit, Chief Development and Medical Officer at BlueRock. “We’re now able to test whether this approach can safely deliver functional cells to the retina and offer hope to people who currently have very limited treatment options.”
The investigational therapy is designed to replace damaged or missing photoreceptor cells with lab-grown healthy ones, with the goal of restoring visual capacity. According to estimates, around 110,000 people in the U.S. are affected by these retinal conditions.
Christian Rommel, head of R&D at Bayer’s Pharmaceuticals Division—which owns BlueRock—said the launch of the CLARICO trial represents a key milestone for the company’s broader regenerative medicine pipeline. “This is the first clinical trial of an iPSC-derived therapy for primary photoreceptor disease, and we believe it may change the landscape of treatment for irreversible vision loss,” Rommel said.
OpCT-001 remains an investigational product and has not yet been approved by any regulatory authority. The safety and effectiveness of the therapy are still under evaluation.
