FDA Clears BlueRock's IND for iPSC Cell Therapy OpCT-001 for Primary Photoreceptor Diseases

BlueRock Therapeutics LP, a clinical stage cell therapy company and wholly owned subsidiary of Bayer AG, announced that its Investigational New Drug (IND) application for OpCT-001 has been cleared by the U.S. Food and Drug Administration (FDA). OpCT-001 is an investigational induced pluripotent stem cell (iPSC)-derived therapy designed to treat primary photoreceptor diseases, marking a significant advancement in the field of cell-based treatments for vision loss.

Amit Rakhit, Chief Development and Medical Officer at BlueRock Therapeutics, expressed enthusiasm over the FDA's clearance, highlighting the potential of OpCT-001 to restore vision in individuals affected by primary photoreceptor diseases. The company is gearing up to initiate a Phase 1/2a clinical study, which will evaluate the safety and tolerability of subretinal administration of OpCT-001 in patients. Additionally, the study aims to assess the impact of OpCT-001 on retinal structure, visual function, and overall functional vision. The clinical trial will explore multiple dose levels and is set to enroll participants at various sites across the United States.

Primary photoreceptor diseases, including retinitis pigmentosa and cone-rod dystrophy, are inherited retinal disorders that impair the function of photoreceptor cells in the retina, leading to irreversible vision loss in both children and adults. In the U.S., approximately 110,000 people are affected by these conditions, and current treatment options are limited. OpCT-001 seeks to address this gap by replacing degenerated retinal cells with functional ones, potentially restoring lost vision.

The development of OpCT-001 is part of a broader collaboration between BlueRock Therapeutics, FUJIFILM Cellular Dynamics, and Opsis Therapeutics, initiated in 2021. In January 2024, BlueRock exclusively licensed OpCT-001 from these partners, who have supported the project through research, development, and the execution of critical IND-enabling activities, including clinical manufacturing at FUJIFILM Cellular Dynamics’ facility in Madison, Wisconsin.

It's important to note that OpCT-001 remains an investigational therapy and has not been approved by any regulatory body. Its safety and efficacy are still under evaluation in clinical studies.

BlueRock Therapeutics, established in 2016, focuses on developing cellular medicines for severe diseases, leveraging cell therapy technologies. The company has other notable investigational cell therapies in its pipeline, such as bemdaneprocel (BRT-DA01) for Parkinson’s disease, which has already advanced beyond Phase 1 clinical trials. BlueRock became a wholly owned subsidiary of Bayer AG in 2019, positioning itself as a central component of Bayer's Cell & Gene Therapy platform.

Bayer AG, a global enterprise with expertise in health care and nutrition, emphasizes sustainability and innovation in its operations. The company aims to address significant global challenges through its products and services, promoting growth and creating value. In 2022, Bayer employed around 101,000 people worldwide and reported sales of 50.7 billion euros, with substantial investments in research and development.