article reprinted from the McPherson Eye Research Institute’s Fall 2024 InSights newsletter
Stem cell-derived photoreceptor replacement therapy has been one of the goals of Dr. David Gamm’s lab for a decade and a half, beginning with the development of a process to create human photoreceptor cells (cones and rods) from induced pluripotent stem cells (iPSCs). In September 2024, the FDA approved a plan to begin initial human trials using iPSC-derived photoreceptors for individuals with advanced forms of retinitis pigmentosa.
BlueRock Therapeutics LP, a wholly owned subsidiary of Bayer, Inc., will manage the trial, and has licensed the cell therapy (termed OpCT-001) from Opsis Therapeutics and FUJIFILM Cellular Dynamics, which supported the research, development, and manufacturing of OpCT-Trial participants will be enrolled in multiple locations, with the goal of beginning the trial in at least one location in 2025. In the press release announcing FDA clearance, Amit Rakhit,
Chief Development and Medical Officer at BlueRock Therapeutics, noted, “We believe that OpCT-001 has 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.”
Of note, photoreceptors are also lost in macular degenerative diseases like AMD, Best disease, and Stargardt disease, but these conditions also require replacement of retinal pigment epithelium (RPE) cells. Efforts are well underway to develop products to address these diseases as well, so stay tuned.