Bayer AG and its wholly owned, independently operated subsidiary BlueRock Therapeutics LP, announced in a press release that its investigational cell therapy OpCT-001 has received Orphan Drug Designation from the US Food and Drug Administration (FDA) for treating retinitis pigmentosa (RP).

OpCT-001 is an investigational induced pluripotent stem cell (iPSC)–derived cell therapy being tested in a phase 1/2a clinical study (CLARICO) for the treatment of primary photoreceptor diseases, which are a subgroup of inherited retinal disorders that include RP and cone-rod dystrophy. According to the press release, OpCT-001 aims to restore vision in people living with RP by replacing lost cells in the retina with new functional cells.

OpCT-001 has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated, the press release noted. 

CLARICO is a multisite, 2-part interventional study to evaluate the safety, tolerability, and the effect on clinical outcomes of OpCT-001 in up to approximately 54 adults with primary photoreceptor disease. Phase 1 will focus on safety and features a dose-escalation design. Phase 2 is designed to gather additional safety data and assess the effect of OpCT-001 on measures of visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups, according to the press release. RP