US FDA Nods BlueRock's IND Application For iPSC-derived Cell Therapy

BlueRock Therapeutics LP, a company developing cell therapy in clinical stage and a subsidiary of Bayer AG, announced the FDA's approval of its IND application for OpCT-001, a treatment for primary photoreceptor diseases.

“We are pleased with the immense progress we have made to date and thrilled that the FDA has cleared our IND application to initiate clinical testing for OpCT-001," said Amit Rakhit, chief development and medical officer at BlueRock Therapeutics. "We believe that OpCT-001 has potential to restore vision in people living with primary photoreceptor diseases and look forward to working with the ophthalmology community in initiating our phase 1/2a clinical study.”

OpCT-001 is the initial iPSC-derived cell therapy being clinically studied for treating primary photoreceptor diseases. Activities for starting a phase 1/2a study are currently in progress. The phase 1/2a trial aims to assess the safety and tolerance of OpCT-001 given subretinally in individuals with primary photoreceptor disorders, and also to study OpCT-001's impact on retinal structure, visual function, and functional vision. The research will evaluate various doses of OpCT-001 and aims to recruit participants from various locations in the United States.

Inherited retinal disorders such as retinitis pigmentosa and cone-rod dystrophy belong to the category of primary photoreceptor diseases. These conditions impact the structure and function of the photoreceptor cells located in the retina, resulting in permanent vision impairment in individuals of all ages. Approximately 110,000 individuals in the United States are impacted by primary photoreceptor diseases, and there are few available treatment choices for this group. OpCT-001 seeks to repair vision impairment from these conditions by substituting degenerated cells in the retina with healthy cells.