BlueRock Receives FDA Clearance for IND Application of OpCT-001 for Primary Photoreceptor Diseases
Bayer subsidiary BlueRock Therapeutics announced that the FDA has cleared its investigational new drug (IND) application for OpCT-001, an investigational induced pluripotent stem cell (iPSC)-derived therapy designed to treat primary photoreceptor diseases. The clearance allows BlueRock to initiate clinical testing for the investigational stem cell therapy.
"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," Amit Rakhit, Chief Development and Medical Officer at BlueRock Therapeutics, said in a company news release. "We believe that OpCT-001 has the 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 first investigational iPSC-derived cell therapy to enter clinical evaluation for primary photoreceptor diseases. BlueRock Therapeutics is preparing to launch a phase 1/2a study, which will be the first human trial to assess the safety and tolerability of subretinal administration of OpCT-001. The study will also evaluate the impact of the therapy on retinal structure, visual function, and overall functional vision in participants. Several dose levels of OpCT-001 will be tested, and the trial will enroll participants at multiple sites across the United States.
Primary photoreceptor diseases, including retinitis pigmentosa and cone-rod dystrophy, are a subset of inherited retinal disorders that severely affect the structure and function of the photoreceptor cells in the retina, leading to irreversible vision loss in both children and adults. With an estimated 110,000 individuals affected in the US, there are limited treatment options available for this patient population. OpCT-001 aims to address this urgent need by replacing degenerated retinal cells with functional ones, offering the possibility of vision restoration.
The development of OpCT-001 stems from a strategic collaboration formed in 2021 between BlueRock Therapeutics, FUJIFILM Cellular Dynamics, and Opsis Therapeutics. In January 2024, BlueRock Therapeutics exclusively licensed OpCT-001 from FUJIFILM Cellular Dynamics and Opsis Therapeutics. FUJIFILM Cellular Dynamics played a critical role in supporting BlueRock Therapeutics by contributing to the research, development, and execution of key IND-enabling activities, including the clinical manufacturing of OpCT-001 at their cGMP facility in Madison, Wisconsin.