Bayer and BlueRock Therapeutics’ iPSC-derived cell therapy OpCT-001 receives FDA Orphan Drug Designation for retinitis pigmentosa, supporting development of a novel vision-restoring treatment now in Phase 1/2a CLARICO trial.
Written By: Pharmacally Medical News Desk
Bayer AG and its wholly owned, independently operated cell therapy subsidiary BlueRock Therapeutics LP have announced that their investigational cell therapy, OpCT-001, has received Orphan Drug Designation from the U.S. Food and Drug Administration for the treatment of retinitis pigmentosa (RP). The designation marks a key regulatory milestone for a program targeting inherited retinal disorders with high unmet medical need.
Christian Rommel, Executive Vice President and Global Head of Research and Development for Bayer’s Pharmaceuticals Division, highlighted the broader significance of the program, emphasizing that OpCT-001 represents the first clinical trial of an iPSC-derived cell therapy in primary photoreceptor diseases.
About OpCT-001
OpCT-001 is an induced pluripotent stem cell (iPSC)-derived cell therapy designed to replace lost photoreceptor cells in the retina. Retinitis pigmentosa is characterized by progressive degeneration of rod and cone photoreceptors, leading to gradual vision loss and eventual blindness. By transplanting new, functional photoreceptor cells, OpCT-001 aims to restore retinal function and potentially improve vision in affected patients.
The therapy is currently being evaluated in the Phase 1/2a CLARICO clinical study, which focuses on primary photoreceptor diseases. This disease group includes retinitis pigmentosa and cone-rod dystrophy, both of which are inherited retinal disorders with limited treatment options.
Clinical Development: The CLARICO Study
CLARICO (NCT06789445) is a first-in-human, multicenter, two-part interventional study enrolling up to approximately 54 adult patients with primary photoreceptor disease.
Phase 1 of the study is designed to evaluate safety and tolerability using a dose-escalation approach. Four planned dose levels will be tested across four cohorts using a standard 3+3 design. Between 12 and 24 legally blind participants are expected to receive escalating doses of OpCT-001, with dose advancement dependent on acceptable safety outcomes.
Phase 2 will expand enrolment to a maximum of 30 participants across two cohorts. Two dose levels selected from Phase 1 data will be evaluated. Participants will be randomized in a 1:1 ratio, and masking will be applied to participants and study personnel outside the surgical team. This phase will assess additional safety data as well as effects on visual function, functional vision, and anatomical measures of retinal cell engraftment.
Amit Rakhit, MD, MBA, Chief Medical Officer of BlueRock Therapeutics, noted that the designation represents an important milestone for the program and reinforces the potential of OpCT-001 as a future treatment option for patients with retinitis pigmentosa and related disorders.
References
FDA grants Orphan Drug Designation to BlueRock Therapeutics’ investigational cell therapy OpCT-001, 22 January 2026, FDA grants Orphan Drug Designation to BlueRock Therapeutics’ investigational cell therapy OpCT-001
A Study to Investigate the Safety of OpCT-001 in Adults Who Have Primary Photoreceptor Disease (CLARICO) (CLARICO), ClinicalTrials.gov ID NCT06789445, https://clinicaltrials.gov/study/NCT06789445