BlueRock Therapeutics Wins FDA Approval For New Treatment

BlueRock Therapeutics, a clinical-stage cell therapy firm fully owned and operated independently under Bayer, has received approval from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application. This green light pertains to OpCT-001, a novel cell therapy derived from induced pluripotent stem cells (iPSCs) aimed at treating primary photoreceptor diseases.

Amit Rakhit, chief development and medical officer at BlueRock Therapeutics, said in a statement, “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. 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 pioneering as the first iPSC-derived cell therapy being tested for primary photoreceptor diseases. Preparations are in progress for a Phase 1/2a clinical trial, which will be the inaugural human study of this treatment. This trial aims to assess the safety and tolerability of delivering OpCT-001 directly into the retina of individuals with these diseases. Additionally, it will examine how OpCT-001 impacts retinal health, visual function, and overall vision. The study will explore various dosages of OpCT-001 and is set to include participants at multiple locations throughout the United States

Primary photoreceptor diseases, which encompass conditions like retinitis pigmentosa and cone-rod dystrophy, are a type of inherited retinal disorder. These conditions impair the photoreceptor cells in the retina, leading to permanent vision loss in both children and adults. In the U.S., approximately 110,000 people are affected by these diseases, and currently, there are very few treatment options available.

OpCT-001 is a promising new treatment designed to address vision loss caused by these disorders. It works by replacing damaged retinal cells with healthy ones. This innovative approach comes from a collaboration that began in 2021 between BlueRock Therapeutics, FUJIFILM Cellular Dynamics, and Opsis Therapeutics. In January 2024, FUJIFILM Cellular Dynamics and Opsis Therapeutics exclusively licensed OpCT-001 to BlueRock Therapeutics. 

The development of this therapy has been supported by FUJIFILM Cellular Dynamics through research and essential IND-enabling activities, including the clinical manufacturing of OpCT-001 at their cGMP facility in Madison, Wisconsin. It's important to note that OpCT-001 is still in the investigational phase and has not yet received regulatory approval. Its safety and effectiveness are still under evaluation.