BlueRock Therapeutics LP has announced that the U.S. Food and Drug Administration (FDA) has cleared OpCT-001, its investigational product, an induced pluripotent stem cell (iPSC)- derived cell therapy, through an Investigational New Drug (IND) application. This means that the company is now approved to begin clinical trials. BlueRock Therapeutics LP is investigating OpCT-001 as a potential treatment for primary photoreceptor diseases, including retinitis pigmentosa.
OpCT-001 will be the first iPSC-derived cell therapy to be clinically studied as a treatment for primary photoreceptor diseases. iPSC-derived cell therapy uses specialized cells made from bodily adult cells to grow new, healthy cells that can help treat diseases. These cells are reprogrammed to resemble embryonic stem cells that can become almost any type of cell the body needs, such as retinal cells. BlueRock Therapeutics LP aims to start a Phase 1/2a research study with plans to have sites across the United States where participants will be enrolled. The goals of this study will be to understand the tolerability and safety of OpCT-001 as well as retinal structure, visual function, and functional vision following treatment with the new cells. OpCT-001 could potentially restore vision loss by replacing damaged cells in the eyes with working cells.
What this means for Usher syndrome: Currently, there are limited therapy options for photoreceptor diseases, including retinitis pigmentosa. Although OpCT-001 is in the very early stages of research, it could be a future therapy for retinitis pigmentosa. It would replace damaged cells with working cells in the eyes to restore vision.
