FDA Fast-Tracks OPCT-001

Bayer AG and BlueRock Therapeutics LP, a clinical-stage cell therapy company and wholly owned subsidiary of Bayer AG, announced that the FDA has granted Fast Track designation for OpCT-001, an investigational induced pluripotent stem cell (iPSC)-derived cell therapy for the treatment of primary photoreceptor diseases. OpCT-001 is the first iPSC-derived investigational cell therapy to be clinically tested for treating primary photoreceptor diseases, which are a subgroup of inherited retinal disorders that includes retinitis pigmentosa and cone-rod dystrophy.  

Primary photoreceptor diseases affect the structure and function of the photoreceptor cells in the retina, leading to irreversible vision loss in both children and adults. OpCT-001 aims to restore vision in patients with primary photoreceptor diseases by replacing degenerated cells in the retina with functional cells. Limited treatment options currently exist for treating primary photoreceptor diseases which affect an estimated 110,000 people in the U.S. 

“We are thrilled to receive Fast Track designation from the FDA for OpCT-001,” said Dr. Amit Rakhit,  BlueRock’s Chief Development and Medical Officer. “People living with primary photo receptor diseases need new treatments that have the potential to restore vision, and we look forward to working closely with the FDA to advance this program through clinical trials.”  

“Receiving FDA Fast Track designation for OpCT-001 demonstrates the urgent need for innovative treatments in the field of primary photoreceptor diseases,” said Christian Rommel, Executive Vice President, Global Head of Research and Development, and Member of the Pharmaceuticals Leadership Team at Bayer. “We are proud to deliver on our commitment to advancing groundbreaking cell therapies by seeking to develop the first iPSC-derived investigational cell therapy for patients facing devastating consequences from primary photoreceptor diseases.”