The FDA granted orphan drug designation to OpCT-001 for the treatment of retinitis pigmentosa, according to a press release from BlueRock Therapeutics.
The investigational induced pluripotent stem cell-derived cell therapy is currently being evaluated in the phase 1/2a CLARICO study.
OpCT-001 is designed to replace lost retina cells with new functional cells, which may restore vision, according to the release.
“Receiving orphan drug designation for retinitis pigmentosa is an important milestone for the OpCT-001 program,” Amit Rakhit, MD, MBA, chief medical officer of BlueRock, said in the