BlueRock Therapeutics Begins Phase 1/2a Trial of Stem Cell Therapy OpCT-001 for Retinal Diseases
The Bayer owned company just starting dosing patients with OpCT-001, an iPSC-derived cell therapy aiming to restore vision in inherited retinal diseases.
Bayer AG and its subsidiary BlueRock Therapeutics have announced the dosing of the first patient in the CLARICO clinical trial, evaluating OpCT-001, an investigational induced pluripotent stem cell (iPSC)-derived therapy for primary photoreceptor (PR) diseases. These diseases, including retinitis pigmentosa and cone-rod dystrophy, are inherited retinal disorders that cause irreversible vision loss by damaging photoreceptor cells in the retina. Primary photoreceptor diseases affect approximately 110,000 people in the U.S.
OpCT-001 is the first iPSC-derived investigational cell therapy clinically tested for primary photoreceptor diseases. The therapeutic approach involves replacing degenerated retinal cells with functional ones to potentially restore vision.
The CLARICO trial is a first-in-human, multisite, Phase 1/2a, two-part interventional study that will assess the safety, tolerability, and clinical outcomes of OpCT-001 in approximately 54 adult participants.
- Phase 1: will focus primarily on safety through a dose-escalation design. It will include four planned dose levels across four patient cohorts, with a total of 12 to 24 legally blind participants (approximately 3-6 per cohort) receiving OpCT-001.
- Phase 2: will further evaluate safety and examine the effect of OpCT-001 on visual function, functional vision, and anatomic measures of engraftment in different clinical subgroups. Phase 2 plans to enroll up to 15 participants randomized 1:1 into two cohorts. Participants and study personnel (except for the surgical team) will be masked to dose assignments.
Amit Rakhit, MD, MBA, Chief Development and Medical Officer at BlueRock, stated: “The initiation of the CLARICO trial represents a key milestone for the OpCT-001 program. We believe OpCT-001 holds significant promise as a novel therapeutic approach for restoring vision in people living with primary photoreceptor diseases, and we look forward to assessing its safety and tolerability profile as we advance this important program in our pipeline.”
Christian Rommel, Executive Vice President and Global Head of Research and Development of the Pharmaceuticals Division at Bayer, added: “We aim to transform treatment options for patients facing irreversible vision loss. OpCT-001, our investigational iPSC-derived cell therapy, has the potential to restore vision for individuals with primary photoreceptor disease. We are excited to announce the first patient in the CLARICO trial, the first-ever clinical trial for an iPSC-derived treatment in this field.”
OpCT-001 has Fast Track designation from the U.S. FDA (Food and Drug Administration). The treatment remains investigational and has not been approved by any regulatory authority, with its efficacy and safety still to be fully established.
