Thursday, January 23, 2025

Stem Cell Therapy for Photoreceptor Diseases Granted License by BlueRock

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BlueRock Therapeutics has licensed the OpCT-001 induced pluripotent stem cell (iPSC) therapy candidate aimed at treating primary photoreceptor diseases. This represents the first licensing from the research and development partnership established in 2021 between BlueRock, FUJIFILM Cellular Dynamics, and Opsis Therapeutics.

As part of the agreement, FUJIFILM Cellular Dynamics and Opsis Therapeutics will receive licensing fees and may also receive payments contingent on specific development and commercial milestones. An Investigational New Drug (IND) filing for OpCT-001 is expected in 2024.

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A Promising Breakthrough in Restoring Vision for Retinal Disease Patients

Ahmed Enayetallah, the senior vice-president and development head at BlueRock Therapeutics, expressed belief in the potential of cell therapy to restore vision in patients suffering from retinal diseases. The collaboration with FUJIFILM Cellular Dynamics and Opsis Therapeutics has allowed for crucial IND-enabling activities, he noted, intending to advance OpCT-001 toward clinical trials.

Primary photoreceptor diseases, including retinitis pigmentosa and cone-rod dystrophies, affect the retina’s photoreceptor cells, leading to irreversible vision loss in both children and adults. Currently, there are no available treatment options for these conditions.

OpCT-001 is engineered to substitute degenerated retinal tissue with functional cells, potentially restoring vision. The innovative approach of OpCT-001 could be an important step in addressing the unmet medical needs of patients with these diseases.

Photoreceptor Diseases

Licensing Agreement Marks Milestone in Advancing Stem Cell Therapy for Photoreceptor Diseases

This licensing agreement signifies a significant milestone in the development of innovative therapies for retinal diseases and is a testament to the potential impact of stem cell technology on medical treatments.

The licensing of OpCT-001 by BlueRock Therapeutics represents crucial progress in the treatment of primary photoreceptor diseases. The lack of existing treatment options highlights the pressing need for innovative approaches like OpCT-001. This unique therapy could potentially restore vision by replacing degenerated retinal tissue with functional cells, thus offering hope to patients suffering from these debilitating diseases.


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