BlueRock Therapeutics Receives FDA Fast Track Designation for OpCT-001 for the Treatment of Primary Photoreceptor Diseases

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Berlin, Germany, and Cambridge, MA, USA February 27, 2025 – Bayer AG and BlueRock Therapeutics LP, a clinical-stage cell therapy company and wholly owned subsidiary of Bayer AG, today 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 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.

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.

¹ https://www.ncbi.nlm.nih.gov/books/NBK519518/;https://www.ncbi.nlm.nih.gov/pmc/articles/PMC1808442/; https://www.nidcd.nih.gov/health/usher-syndrome#bAbout BlueRock Therapeutics LPBlueRock Therapeutics LP is a clinical stage cell therapy company focused on creating cellular medicines to treat devastating diseases. We are harnessing the power of cell therapy to create a pipeline of new medicines for people suffering from neurological, ophthalmic, cardiovascular and immunological diseases. Two of our novel investigational cell therapies, bemdaneprocel (BRT-DA01) for the treatment Parkinson’s disease and OpCT-001 for the treatment of primary photoreceptor disease are clinical stage programs. Bemdaneprocel has RMAT (Regenerative Medicine Advanced Therapy) and Fast Track designation from the U.S. FDA (Food and Drug Administration); has completed Phase 1 clinical trial and is progressing to the next stage of clinical development. OpCT-001 has been cleared by the FDA to begin Phase 1 clinical testing. BlueRock was founded in 2016 as a joint venture of Versant Ventures and Leaps by Bayer, the impact investing arm of Bayer AG that invests in paradigm-shifting breakthrough innovation. In late 2019, BlueRock became a wholly owned, independently operated subsidiary of Bayer AG as a cornerstone of its newly formed Cell & Gene Therapy platform. Our culture is defined by the courage to persist regardless of the challenge, the urgency to transform medicine and deliver hope, integrity guided by mission, and community-mindedness with the understanding that we are all part of something bigger than ourselves. For more information, visit www.bluerocktx.com.

About BayerBayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population. Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2023, the Group employed around 100,000 people and had sales of 47.6 billion euros. R&D expenses before special items amounted to 5.8 billion euros. For more information, go to www.bayer.com.Forward-Looking Statements BlueRock TherapeuticsCertain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “plan,” and “intend,” among others. These forward-looking statements are based on BlueRock’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, the initiation, timing, progress, activities, goals and reporting of results of any preclinical programs and clinical trials and research and development programs, the potential benefits, timing and future operation of the agreements with FUJIFILM Cellular Dynamics and Opsis Therapeutics, the ability to advance therapies into, and successfully initiate, enroll and complete clinical trials, the potential clinical utility of product candidates, the regulatory pathway of, and the timing or likelihood of any regulatory filings and approvals for, any product candidates, and the ability to, and extent of, potentially commercializing any product candidate, are forward looking. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Except as expressly required by law, BlueRock does not undertake an obligation to update or revise any forward-looking statement. All of BlueRock’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date hereof.Forward-Looking Statements BayerThis release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer’s public reports which are available on the Bayer website at www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

Posted : 2025-03-01 18:00

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