Berlin, Germany; Cambridge, Massachusetts, USA – February 27, 2025: In the realm of regenerative medicine, Bayer AG has reached a significant milestone working with its affiliate BlueRock Therapeutics LP. OpCT-001, an investigational cell therapy derived from induced pluripotent stem cells (iPSCs) targeted at treating primary photoreceptor diseases—an inherited class of retinal disorders responsible for severe and irreversible vision loss in children and adults—has Fast Track designation granted by the U.S. Food and Drug Administration (FDA).
Retinitis pigmentosa and cone-rod dystrophy are two primary photoreceptor illnesses for which OpCT-001 is the first investigational medication of its type to reach clinical testing. These disorders compromise the structure and operation of photoreceptor cells in the retina, which causes slow down of vision. For thousands of people afflicted by these crippling diseases, this novel therapy offers hope given the few therapeutic choices already at hand.
The Fast Track program of the FDA is meant to hasten the development and evaluation of medicines tackling major diseases with unmet medical needs. Bayer and BlueRock Therapeutics’ frequent FDA interactions made possible by this acknowledgement help to hasten the development of the medicine. Should important requirements be satisfied, the therapy could also be eligible for priority evaluation and rapid approval.
Emphasizing the urgent need for creative solutions, Chief Development and Medical Officer of BlueRock Therapeutics, Dr. Amit Rakhit, expressed excitement at this milestone. “It gives us great pleasure to have Fast Track designated for OpCT-001. Those suffering from primary photoreceptor disorders require innovative ideas able to possibly restore vision. Working with the FDA to push this exciting medicine via clinical studies excites us.
For those afflicted, primary photoreceptor illnesses seriously harm their quality of life since they cause permanent visual loss without any clear treatment. OpCT-001 is meant to replace functioning cells in the retina instead of degraded photoreceptor cells, hence perhaps restoring eyesight. Given the estimated 110,000 Americans with these hereditary disorders, new therapeutic options are desperately needed.
Emphasizing the importance of this accomplishment, Bayer Pharmaceuticals Executive Vice President and Global Head of Research & Development Christian Rommel stressed “Receiving FDA Fast Track designation for OpCT-001 emphasizes the crucial need of creative ophthalmic treatments. Bayer is dedicated to provide innovative cell therapy solutions capable of changing people’s life. It gives us great pleasure to advance in tackling the terrible effects of main photoreceptor disorders.
Originally created in association with FUJIFILM Cellular Dynamics and Opsis Therapeutics, BlueRock Therapeutics was granted exclusive licencing rights in January 2024. FUJIFILM Cellular Dynamics was important in the research, development, and clinical manufacture of the therapy as part of this strategic alliance. Established in 2021, the cooperation keeps pushing cell therapy options for eye illnesses to develop forward.
OpCT-001 is still an investigational treatment and has not yet been licenced by any regulatory body. Extensive clinical studies will help to assess its safety and effectiveness in order to ascertain its possible use as a suitable therapy for main photoreceptor disorders.
About BlueRock Therapeutics LP
Clinically-stage biotechnology startup BlueRock Therapeutics LP is committed to create cellular treatments for serious disease sufferers. BlueRock is pioneering treatments for neurological, ophthalmic, cardiovascular, and immunological conditions by utilizing the power of cell therapy. Two main investigational cell therapies on the company’s pipeline are OpCT-001, which the FDA has now cleared for Phase 1 testing, and bemdaneprocel (BRT-DA01), for Parkinson’s disease, which has received RMAT and Fast Track designation and completed Phase 1 clinical trials. Originally established in 2016 as a joint venture between Versant Ventures and Leaps by Bayer, BlueRock became a wholly owned subsidiary of Bayer AG in 2019 and is still run independently under Bayer’s Cell & Gene Therapy programme.
Relcerning Bayer
Global in nature, Bayer specializes in agriculture and healthcare. Aiming at “Health for all, Hunger for none,” Bayer is committed to inspire creativity in nutritional and medical sciences. With R&D spending €5.8 billion and reported sales of €47.6 billion in 2023 the company employs about 100,000 people globally. Through innovative research, sustainable development, and the distribution of premium products, Bayer seeks to advance human health. Visit www.bayer.com for further details.
Note: This article includes forward-looking comments on OpCT-001’s development and regulatory approval. Though the FDA’s Fast Track classification is encouraging, it does not ensure approval. Constant clinical assessment will help to determine OpCT-001’s safety and effectiveness.
