BlueRock Therapeutics Receives FDA Clearance for OpCT-001 IND Application

BlueRock Therapeutics, a subsidiary of Bayer, announced that the FDA has cleared its investigational new drug (IND) application for OpCT-001, a stem cell therapy designed to treat primary photoreceptor diseases. The clearance allows BlueRock to initiate a phase 1/2a clinical trial to assess the safety and impact of the iPSC-derived therapy on retinal structure and visual function in participants with conditions like retinitis pigmentosa and cone-rod dystrophy.

OpCT-001: A Novel Approach to Photoreceptor Diseases

OpCT-001 is the first investigational iPSC-derived cell therapy to undergo clinical evaluation for primary photoreceptor diseases, offering the potential to restore vision in patients suffering from inherited retinal disorders. Primary photoreceptor diseases, such as retinitis pigmentosa and cone-rod dystrophy, affect the photoreceptor cells in the retina, leading to progressive vision loss. With limited treatment options available, OpCT-001 seeks to address this unmet need by replacing degenerated retinal cells with functional ones.

Amit Rakhit, Chief Development and Medical Officer at BlueRock Therapeutics, expressed optimism, stating, "We believe that OpCT-001 has the potential to restore vision in people living with primary photoreceptor diseases and look forward to working with the ophthalmology community in initiating our phase 1/2a clinical study.”

Clinical Trial and Goals

The upcoming phase 1/2a study will evaluate the safety and tolerability of subretinal administration of OpCT-001, testing several dose levels. The trial will also explore its effects on retinal structure, visual function, and overall vision improvement in participants, with multiple study sites across the United States. The study represents the first human trial to investigate the potential benefits of OpCT-001 for individuals affected by primary photoreceptor diseases.

Collaborative Development

OpCT-001 is the result of a strategic partnership formed in 2021 between BlueRock Therapeutics, FUJIFILM Cellular Dynamics, and Opsis Therapeutics. In January 2024, BlueRock Therapeutics exclusively licensed OpCT-001 from FUJIFILM and Opsis. FUJIFILM Cellular Dynamics supported the development of the therapy by contributing to key IND-enabling activities, including the clinical manufacturing of OpCT-001 at their cGMP facility in Wisconsin.

This marks a significant step forward in developing a novel therapeutic option for an estimated 110,000 individuals affected by primary photoreceptor diseases in the U.S.However, OpCT-001 is still under clinical investigation, and its safety and efficacy are yet to be fully established.

About BlueRock Therapeutics

BlueRock Therapeutics LP is a clinical-stage cell therapy company developing cellular medicines for neurological, ophthalmic, cardiovascular, and immunological diseases. Founded in 2016, BlueRock's pipeline includes two investigational therapies: bemdaneprocel for Parkinson’s disease, which has completed Phase 1 trials, and OpCT-001 for primary photoreceptor diseases, which has just received FDA clearance for Phase 1 testing. BlueRock was established as a joint venture of Versant Ventures and Leaps by Bayer and became a wholly owned subsidiary of Bayer AG in 2019.

For further details, you can read the official announcement from BlueRock Therapeutics.