In a significant advancement for ophthalmology treatment, BlueRock Therapeutics, a Bayer AG subsidiary, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to OpCT-001, their groundbreaking induced pluripotent stem cell (iPSC)-derived cell therapy targeting primary photoreceptor diseases.
The designation marks a crucial milestone as OpCT-001 becomes the first iPSC-derived cell therapy to enter clinical testing for primary photoreceptor diseases, including retinitis pigmentosa and cone-rod dystrophy. This regulatory decision enables more frequent interactions with the FDA and potential eligibility for accelerated approval and priority review.
Addressing Critical Unmet Medical Need
Primary photoreceptor diseases, affecting approximately 110,000 Americans, cause irreversible vision loss in both pediatric and adult populations. These inherited retinal disorders progressively damage photoreceptor cells in the retina, with limited treatment options currently available.
"People living with primary photoreceptor diseases need new treatments that have the potential to restore vision," stated Amit Rakhit, MD, MBA, BlueRock's Chief Development and Medical Officer. "We look forward to working closely with the FDA to advance this program through clinical trials."
Innovative Therapeutic Approach
OpCT-001's mechanism of action represents a novel approach to treating retinal disorders. The therapy aims to restore vision by replacing degenerated retinal cells with functional cells derived from induced pluripotent stem cells. This regenerative approach could potentially offer hope to patients facing progressive vision loss.
Christian Rommel, Executive Vice President and Global Head of Research and Development at Bayer, emphasized the significance of the Fast Track designation: "This demonstrates the urgent need for innovative treatments in the field of primary photoreceptor diseases. We are proud to deliver on our commitment to advancing groundbreaking cell therapies."
Strategic Collaboration and Development
The development of OpCT-001 stems from a strategic collaboration established in 2021 between BlueRock Therapeutics, FUJIFILM Cellular Dynamics, and Opsis Therapeutics. In January 2024, BlueRock exclusively licensed the therapy, with FUJIFILM Cellular Dynamics providing crucial support in research, development, and clinical manufacturing activities.
Clinical Development Status
While OpCT-001 has received FDA clearance to begin Phase 1 clinical testing, it remains an investigational therapy whose safety and efficacy have not yet been fully evaluated. The Fast Track designation is expected to expedite its development process, potentially bringing this innovative treatment option to patients more quickly.
