Primary photoreceptor diseases could result in irreversible vision loss in both children and adults. Credit: Andrey Sayfutdinov/Shutterstock.

The US Food and Drug Administration (FDA) has granted clearance for BlueRock Therapeutics’ investigational new drug (IND) application to commence a Phase I/IIa trial of OpCT-001, an investigational iPSC-derived cell therapy to treat primary photoreceptor diseases.

This first-in-human study will evaluate the safety, tolerability, and efficacy of the therapy in treating primary photoreceptor diseases, which lead to vision loss.

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It will also evaluate the impact of the subretinal administration of OpCT-001 on retinal structure, visual function, and functional vision in individuals.

The study is set to explore various dose levels of OpCT-001 and will recruit participants across multiple US sites.

BlueRock exclusively licensed OpCT-001 from FUJIFILM Cellular Dynamics and Opsis Therapeutics in January this year, as a result of a strategic research and development and clinical manufacturing collaboration that was established in 2021.

As part of this deal, FUJIFILM Cellular Dynamics provided support to BlueRock by researching, developing, and executing critical IND-enabling activities.

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This included the clinical manufacturing of OpCT-001 at its cGMP facility in Madison, Wisconsin.

BlueRock Therapeutics chief development and medical officer Amit Rakhit said: “We are pleased with the immense progress we have made to date and thrilled that the FDA has cleared our IND application to initiate clinical testing for OpCT-001.

“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 I/IIa clinical study.”

Primary photoreceptor diseases, which include conditions such as retinitis pigmentosa and cone-rod dystrophy, impact the structure and function of the photoreceptor cells in the retina.

These diseases can result in irreversible vision loss in both children and adults.

In March 2022, BlueRock announced plans to commence a Phase I Study of MSK-DA01 cell therapy for advanced Parkinson’s disease after it received permission from Health Canada.