Bayer and its wholly owned, independently operated subsidiary BlueRock Therapeutics, announced that its investigational cell therapy OpCT-001 has received Orphan Drug Designation from FDA for the treatment of retinitis pigmentosa (RP).

OpCT-001 is an investigational induced pluripotent stem cell (iPSC)–derived cell therapy currently being evaluated in a phase 1/2a clinical study known as CLARICO. The study is designed to assess OpCT-001 in people living with primary photoreceptor diseases, a subgroup of inherited retinal disorders that includes retinitis pigmentosa and cone-rod dystrophy.

Orphan Drug Designation is granted to drugs and biologics intended to treat rare diseases or conditions affecting fewer than 200,000 people in the United States. The designation is intended to encourage the development of therapies for rare conditions by providing certain regulatory and financial incentives.

“The FDA’s Orphan Drug Designation for OpCT-001 to treat retinitis pigmentosa underscores the importance of developing urgently needed innovative therapies for patients living with inherited retinal disorders,” said Christian Rommel, Executive Vice President and Global Head of Research and Development for Bayer’s Pharmaceuticals Division. “Together with BlueRock, we are excited to be advancing the first-ever clinical trial of an iPSC-derived cell therapy in primary photoreceptor diseases.”

About the CLARICO Study

CLARICO is a Phase 1/2a, first-in-human, multisite, two-part interventional study designed to evaluate the safety, tolerability, and effects on clinical outcomes of OpCT-001 in up to approximately 54 adults with primary photoreceptor disease.

Phase 1 of the study focuses on safety and uses a dose-escalation design across four planned dose levels administered in four cohorts. Dose escalation follows a standard 3+3 design, enrolling approximately 12 to 24 legally blind participants.

Phase 2 is designed to further assess safety and evaluate the effect of OpCT-001 on visual function, functional vision, and anatomical measures of retinal engraftment. Up to 30 participants will be enrolled across two cohorts and randomized 1:1 to one of two selected dose levels based on phase 1 data. Participants and investigators, excluding the surgical team, will be masked to dose assignments.