Bayer-BlueRock Therapeutics bags USFDA Fast Track Designation for primary photoreceptor diseases treatment
Berlin: Bayer AG and BlueRock Therapeutics LP, a clinical-stage cell therapy company and wholly owned subsidiary of Bayer AG, has announced that the U.S. Food and Drug Administration (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 a 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.
The FDA’s Fast Track designation is intended to facilitate the development and review of drug candidates that treat serious conditions and address an unmet medical need. A drug candidate that receives Fast Track designation may be eligible for more frequent interaction with the FDA to discuss the drug candidate’s development plan as well as eligibility for accelerated approval and priority review, if relevant criteria are met.
“We are thrilled to receive Fast Track designation from the FDA for OpCT-001,” said Amit Rakhit, MD, MBA, 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.”
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 estimated110,000¹ people in the U.S.
“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.”
OpCT-001 was exclusively licensed from FUJIFILM Cellular Dynamics and Opsis Therapeutics in January 2024 as part of the strategic R&D and clinical manufacturing collaboration between BlueRock Therapeutics, FUJIFILM Cellular Dynamics, and Opsis Therapeutics forged in 2021. As part of the collaboration, FUJIFILM Cellular Dynamics supported BlueRock Therapeutics via research, development and the execution of critical IND-enabling activities including the clinical manufacturing of OpCT-001.
OpCT-001 is an investigational cell therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated.
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