FDA clears investigational new drug application for photoreceptor disease therapy
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Key takeaways:
- BlueRock Therapeutics is preparing to initiate a phase 1/2a clinical study of OpCT-001.
- OpCT-001 will be evaluated for treating primary photoreceptor diseases.
The FDA cleared an investigational new drug application for OpCT-001, an investigational cell therapy for the treatment of primary photoreceptor diseases, according to a press release from BlueRock Therapeutics.
“We believe that, if approved, OpCT-001 has potential as a treatment for people living with primary photoreceptor diseases, which are a subgroup of inherited retinal disorders that includes retinitis pigmentosa and cone-rod dystrophy,” Amit Rakhit, MD, MBA, chief development and medical officer at BlueRock Therapeutics, told Healio. “Currently, no treatment options exist for these diseases, which affect the function and structure of the photoreceptor cells in the retina, leading to irreversible vision loss in both children and adults.”
The induced pluripotent stem cell-derived cell therapy is the first to be clinically evaluated for primary photoreceptor diseases, including disorders such as retinitis pigmentosa and cone-rod dystrophy. The therapy was exclusively licensed in January 2024 as part of a collaboration between BlueRock Therapeutics, Fujifilm Cellular Dynamics and Opsis Therapeutics.
BlueRock plans to evaluate the safety and tolerability of subretinal administration of several dose levels of OpCT-001 in a phase 1/2a clinical study. The trial will enroll participants from across the U.S. and assess the effect of the therapy on retinal structure, visual function and functional vision, according to the release.
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